Alnylam Pharmaceuticals Inc (ALNY)

Alnylam to Host Tenth “RNAi Roundtable” Webcast SeriesJune 12, 2026 9:00 AM
Business Wire Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it plans to host its 10th series of “RNAi Roundtable” webinars over the coming months. The series will offer presentations from Alnylam scientists and physicians who will review recent progress in a selection of the Company’s pipeline programs, as well as medical thought leaders who will provide their perspectives on unmet clinical needs. Each event will be webcast live on the Investors section of the Company’s website at investors.alnylam.com/events, and a replay will be posted on the Alnylam website approximately three hours after each event. The 2026 RNAi Roundtable schedule is as follows: ALN-6400: Targeting Plasminogen to Address a Wide Range of Bleeding Disorders Thursday, June 25, 11:00 am ET Zilebesiran: Targeting Angiotensinogen to Achieve Continuous Control of Blood Pressure Thursday, September 17, 10:30 am ET ALN-HTT02: Targeting Exon 1 of Huntingtin Gene to Reduce Progression of Huntington’s Disease Monday, October 26, 10:00 am ET Alnylam’s leadership in RNAi therapeutics is underpinned by a broad and rapidly advancing pipeline spanning multiple therapeutic areas and stages of development. The Company is focused on its Alnylam 2030 goals, including driving Growth Through Sustainable Innovation, by advancing an industry-leading pipeline of RNAi therapeutics designed to prevent, halt, or reverse disease. Specifically, the Company plans to achieve over 40 clinical programs by 2030—from over 25 today—and to continue expanding into new tissues, while harnessing the latest AI technologies to accelerate and optimize its discovery platform. Alnylam is investing meaningfully in R&D to extend the reach of RNAi and deliver multiple new transformative medicines for patients with serious diseases and high unmet need. About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com or follow Alnylam on X, LinkedIn, Facebook, Instagram, or YouTube. Forward Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding Alnylam’s ability to achieve the goals in its Alnylam 2030 strategy, including to drive growth through innovation; the potential to expand its pipeline to over 40 clinical programs by 2030; the potential to expand into new tissues; the potential to utilize AI technologies to accelerate and optimize its discovery platform; and the potential to deliver multiple new transformative medicines for patients should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its Alnylam 2030 strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, and Regeneron; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. View source version on businesswire.com: https://www.businesswire.com/news/home/20260612239247/en/ Alnylam Pharmaceuticals, Inc.
Christine Akinc
(Investors and Media)
617-682-4340 Josh Brodsky
(Investors)
617-551-8276 Original: Alnylam to Host Tenth “RNAi Roundtable” Webcast Series

Alnylam and Inceptive Form Strategic AI Collaboration to Accelerate the Discovery of RNAi TherapeuticsJune 3, 2026 4:45 PM
Business Wire - Collaboration is valued at up to $2B with upfront consideration of $30M - - Alliance pairs Alnylam’s RNAi platform and 20+ years of proprietary data with Inceptive’s foundation models and AI expertise to catalyze progress beyond rational drug design – - Partnership designed to accelerate timelines and unlock innovative oligonucleotide designs - Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Inceptive Nucleics, Inc., which builds foundation models of life, today announced a strategic collaboration agreement to increase the pace of therapeutic innovation. The collaboration is valued at up to $2B with upfront consideration of $30M, including cash and the purchase of Inceptive equity. Inceptive is eligible to receive additional payments based on the achievement of preclinical, regulatory, and commercial sales milestones. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260603131911/en/Yvonne Greenstreet By integrating Inceptive’s generative AI models with Alnylam’s R&D engine, Alnylam aims to accelerate the discovery of novel RNAi therapeutics as it advances ambitious pipeline expansion goals as part of its Alnylam 2030 strategy. “We’re thrilled to partner with Inceptive to push the boundaries of what is possible in the discovery of RNAi medicines,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “Inceptive stands apart as one of the most visionary companies working at the intersection of AI and biology. It is led by pioneers of the AI revolution and driven by an ambitious mission to fundamentally reinvent how RNA medicines are designed. Together, we have an extraordinary opportunity to accelerate the creation of transformative medicines with a speed, ingenuity, and sophistication that simply has not been possible before.” Inceptive’s foundation model learns the patterns underlying biology and hence can adapt to diverse therapeutic modalities without retraining. In joint exploratory work, the model achieved exceptional performance within weeks, uncovering meaningful biological insights from relatively small datasets to characterize siRNA molecules, the active ingredient in RNAi therapeutics. "Most drug design still works through a process of trial and error, testing thousands of molecules and hoping something sticks,” said Jakob Uszkoreit, Inceptive co-founder and CEO. “Inceptive was built on a different premise: that life follows rules of such complexity that only AI can learn them. Alnylam’s breakthrough platform and scientific vision are an ideal match for AI. Together, we’re not just accelerating drug discovery; we’re changing the way we understand and improve life.” Generalizable Platforms Open New Therapeutic Design Spaces The collaboration pairs Alnylam’s RNAi leadership with Inceptive’s foundation models and AI expertise to catalyze and accelerate progress in nucleic-acid based drug design. Inceptive focuses on developing models for sequence-based medicines such as RNAi therapeutics, which were pioneered by Alnylam. Alnylam’s platform: An R&D engine that has produced six approved drugs, backed by 20+ years of proprietary siRNA data. Inceptive’s foundation models of life: AI models for sequence-based medicines that generalize across programs and continuously improve. The collaboration seeks to advance siRNA design, by modeling target mRNAs and jointly exploring sequence space and novel chemical modifications to enhance potency and efficacy, and by predicting top-performing therapeutic candidates in preclinical models for further development by Alnylam. The goal is to help Alnylam prioritize the most promising molecules and improve experimental productivity. By combining Alnylam’s deep biological expertise with Inceptive’s frontier models, the alliance advances Alnylam’s ambition to unlock new therapeutic innovation through frontier AI. The collaboration gives Alnylam access to Inceptive’s AI expertise and talent, including CEO Jakob Uszkoreit, co-inventor of the Transformer architecture (i.e., the “T” in ChatGPT), and pioneers of scalable, AI-enabled wet-lab training data generation methods. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com or follow Alnylam on X, LinkedIn, Facebook, Instagram, or YouTube. About Inceptive Inceptive builds AI foundation models of life that extrapolate from data to design breakthrough biological medicines beyond the reach of nature and traditional drug discovery. The company’s antedisciplinary team of AI researchers, biochemists, and engineers trains models on diverse biological data and designs experiments to generate missing training data at unprecedented scale. Inceptive partners with leading drugmakers to customize molecule-design models for discovery of sequence-based medicines such as siRNA, ASOs, peptides, and mRNA for applications such as in-vivo cell therapies. Founded in 2021 and backed by a16z, NVIDIA, S32 and Obvious, Inceptive is headquartered in Palo Alto with offices in Berlin and Zurich. Learn more at inceptive.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential for Alnylam’s collaboration with Inceptive to achieve the goals for which it was established, including to increase the pace of therapeutic innovation, to catalyze and accelerate progress in nucleic-acid based drug design, accelerate timelines and unlock innovative oligonucleotide designs, and to accelerate the creation of transformative medicines with a speed, ingenuity and sophistication that has not been possible before; Alnylam’s ability to accelerate the discovery of novel RNAi therapeutics and to push the boundaries of what is possible in the discovery of RNAi medicines; and Alnylam’s ability to achieve the goals in its Alnylam 2030 strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its Alnylam 2030 strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, and Regeneron; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. View source version on businesswire.com: https://www.businesswire.com/news/home/20260603131911/en/ Media Contact — Alnylam:
Christine Akinc |

De nouvelles analyses de données présentées lors du congrès Heart Failure 2026 démontrent les bénéfices cliniques solides et constants du vutrisiran en tant qu’option thérapeutique de première intention chez toutes les populationsMay 13, 2026 3:53 PM
Business Wire - La réduction de la mortalité toutes causes confondues et des événements cardiovasculaires récurrents s’est maintenue dans tous les sous-groupes clés de patients, y compris chez ceux suivant un large éventail de traitements contre l’insuffisance cardiaque – - Une analyse groupée portant sur plus de 25 000 années-patients d’expérience avec les traitements par ARNi inhibiteurs de la TTR montre un profil de sécurité cohérent, sans effet oculaire cliniquement significatif lié à la réduction de la vitamine A – - L’étude DemonsTTRate est conçue pour générer des données cliniques réelles à long terme chez plus de 2 000 patients atteints d’ATTR-CM - De nouvelles analyses de données présentées lors du congrès Heart Failure 2026 démontrent les bénéfices cliniques solides et constants du vutrisiran en tant qu’option thérapeutique de première intention chez toutes les populations de patients atteints d’ATTR-CM, y compris ceux présentant une charge de morbidité élevée Alnylam Pharmaceuticals, Inc. (Nasdaq : ALNY), la société leader dans le domaine des thérapies par ARNi, a annoncé aujourd’hui de nouvelles analyses issues de l’étude de phase 3 HELIOS-B sur le vutrisiran chez des patients atteints de cardiomyopathie liée à l’amylose à transthyrétine de type sauvage ou héréditaire (ATTR-CM), venant s’ajouter à l’ensemble croissant de données étayant le vutrisiran et renforçant la durabilité de l’inhibition de la transthyrétine (TTR) ainsi que son profil de sécurité bien caractérisé. Le vutrisiran est le premier et le seul inhibiteur de la TTR approuvé pour l’ATTR-CM, conçu pour induire une inhibition rapide de la TTR à la source. Les données présentées lors du congrès Heart Failure 2026, le congrès annuel de l’Association sur l’insuffisance cardiaque de la Société européenne de cardiologie, montrent un bénéfice clinique constant chez les populations de patients couramment rencontrées en pratique clinique, y compris celles présentant une charge de morbidité élevée, ce qui soutient son utilisation en tant qu’option thérapeutique de première intention pour cette maladie à progression rapide et potentiellement mortelle. « Les analyses présentées lors du congrès Heart Failure 2026 fournissent des informations importantes sur les performances du vutrisiran chez les patients que nous voyons en pratique clinique, y compris ceux qui présentent des caractéristiques telles que la fibrillation auriculaire, une pression artérielle systolique basse et une charge élevée de comorbidités », déclare Scott Solomon, professeur de médecine à la Harvard Medical School et cardiologue au Brigham and Women’s Hospital. « Ces nouvelles analyses de l’étude HELIOS-B montrent que les bénéfices cliniques du vutrisiran ont été maintenus chez ces groupes de patients cliniquement complexes, ainsi que chez les patients recevant des traitements de fond, notamment des stabilisateurs de la TTR et des traitements modificateurs de l’évolution de l’insuffisance cardiaque, ce qui renforce à la fois la cohérence de l’effet thérapeutique et sa pertinence dans la pratique clinique réelle. Dans l’ensemble, ces résultats soutiennent l’utilisation du vutrisiran comme option thérapeutique de première intention pour l’ATTR-CM chez un large éventail de populations de patients. » Chez les patients atteints de fibrillation auriculaire, représentant environ 65 % de la population de l’étude HELIOS-B et associés à une maladie plus avancée, le vutrisiran a réduit de manière significative le risque de mortalité toutes causes confondues et d’événements cardiovasculaires (CV) récurrents par rapport au placebo. Les effets du traitement ont également été maintenus chez les patients présentant une pression artérielle systolique (PAS) basse, un phénotype à haut risque, le vutrisiran ralentissant le déclin progressif de la PAS observé au fil du temps. Les bénéfices cliniques étaient tout aussi constants, indépendamment de la charge de comorbidités ou de l’utilisation concomitante de traitements modificateurs de la maladie, notamment le tafamidis et les médicaments contre l’insuffisance cardiaque tels que les inhibiteurs du SGLT2, les MRA, les bêtabloquants et les IECA/ARA/ARNI. Des effets constants ont également été observés chez les femmes, une population historiquement sous-représentée dans les essais sur l’ATTR-CM. Une analyse groupée distincte des données de sécurité issues des essais cliniques et de la période post-commercialisation a évalué la relation entre les traitements par ARNi réduisant la transthyrétine et les événements indésirables liés à une carence en vitamine A. Il est recommandé aux patients traités par le vutrisiran et le patisiran de prendre l’apport journalier recommandé en vitamine A. L’analyse a porté sur plus de 25 000 années-patients d’exposition au traitement dans le cadre des programmes sur le vutrisiran et le patisiran. Les taux d’effets indésirables oculaires potentiellement associés à une carence en vitamine A étaient faibles et comparables à ceux observés sous placebo. Aucun cas de carence en vitamine A cliniquement significative n’a été observé. « La vitamine A joue un rôle essentiel dans la vision et d’autres fonctions physiologiques clés. Si la transthyrétine contribue à son transport, de multiples voies assurent son acheminement dans l’organisme », déclare William S. Blaner, professeur de médecine nutritionnelle à l’université Columbia et expert en métabolisme et transport de la vitamine A. « Les taux faibles et comparables à ceux du placebo d’événements indésirables liés à une carence en vitamine A observés dans cette vaste analyse fournissent une forte assurance que la réduction de la transthyrétine n’augmente pas de manière significative ces événements chez les patients atteints d’amylose ATTR. » Alnylam a également présenté le protocole et la justification de l’étude DemonsTTRate, une étude prospective et observationnelle à l’échelle mondiale évaluant les résultats en conditions réelles chez les patients atteints d’ATTR-CM. L’étude devrait recruter plus de 2 000 patients et les suivre pendant une période pouvant aller jusqu’à cinq ans, générant ainsi des données longitudinales sur les résultats cliniques, les schémas thérapeutiques et le recours aux soins de santé dans le cadre de la pratique clinique courante. Entre l’ATTR-CM et l’amylose héréditaire médiée par la transthyrétine avec polyneuropathie (hATTR-PN), l’expérience mondiale avec le vutrisiran dépasse à ce jour 13 000 années-patients, ce qui reflète un ensemble solide et croissant de données cliniques pour les deux formes de la maladie. Pour consulter les présentations d’Alnylam à Heart Failure 2026, veuillez vous rendre sur Capella. INDICATIONS ET INFORMATIONS IMPORTANTES RELATIVES À LA SÉCURITÉ d’AMVUTTRA® (vutrisiran) Indications Dans l’UE, AMVUTTRA® (vutrisiran) est indiqué pour le traitement : de l’amylose héréditaire à transthyrétine chez les patients adultes atteints de polyneuropathie de stade 1 ou 2 (hATTR-PN) ; de l’amylose à transthyrétine de type sauvage ou héréditaire chez les patients adultes atteints de cardiomyopathie (ATTR-CM). La disponibilité dans l’UE est soumise aux délais de remboursement locaux. Informations de sécurité importantes Réduction des taux sériques de vitamine A et supplémentation recommandée Le traitement par vutrisiran entraîne une diminution des taux sériques de vitamine A. Une supplémentation d’environ 2 500 à 3 000 UI de vitamine A par jour, sans dépasser cette dose, est recommandée pour les patients prenant du vutrisiran. Les patients doivent être orientés vers un ophtalmologiste s’ils développent des symptômes oculaires suggérant une carence en vitamine A (par ex. cécité nocturne). Effets indésirables Les effets indésirables fréquemment rapportés avec le vutrisiran étaient des réactions au site d’injection et une augmentation des taux sanguins de phosphatase alcaline et d’alanine transaminase. Pour plus d’informations sur le vutrisiran, veuillez consulter le résumé complet des caractéristiques du produit . À propos d’AMVUTTRA® (vutrisiran) AMVUTTRA® (vutrisiran) est un inhibiteur de la transthyrétine (TTR) qui permet une réduction rapide de la TTR à la source afin de traiter la cause sous-jacente de l’amylose à transthyrétine (ATTR). Dans une étude clinique, AMVUTTRA a rapidement réduit la TTR dès six semaines et a diminué les taux de TTR de 87 % après deux ans et demi de traitement. Il est approuvé dans le monde entier dans divers pays pour le traitement de la polyneuropathie associée à l’amylose héréditaire à transthyrétine (hATTR-PN) chez l’adulte et de la cardiomyopathie associée à l’amylose à transthyrétine de type sauvage ou héréditaire (ATTR-CM) chez l’adulte. Administré tous les trimestres par injection sous-cutanée, AMVUTTRA est le premier et le seul inhibiteur approuvé pour le traitement de l’ATTR-CM et de l’hATTR-PN. À propos de l’amylose à transthyrétine (ATTR) L’amylose à transthyrétine (ATTR) est une maladie sous-diagnostiquée, à progression rapide, invalidante et mortelle, causée par des protéines transthyrétine (TTR) pathogènes qui s’accumulent sous forme de dépôts amyloïdes dans diverses parties du corps, notamment les nerfs, le cœur et le tractus gastro-intestinal. Les patients peuvent présenter une polyneuropathie, une cardiomyopathie, ou les deux manifestations de la maladie. Il existe deux formes différentes d’ATTR : l’ATTR héréditaire (hATTR), causée par une variante du gène TTR, et l’ATTR de type sauvage (wtATTR), qui survient sans variante du gène TTR. On estime que plus de 500 000 personnes dans le monde vivent avec l’ATTR. À propos de l’iARN L’iARN (interférence ARN) est un processus cellulaire naturel de silençage génique qui représente aujourd’hui l’un des horizons les plus prometteurs et les plus avancés de la biologie et du développement de médicaments. Sa découverte a été annoncée comme « une avancée scientifique majeure qui se produit une fois tous les dix ans environ » et a été récompensée par le prix Nobel 2006 de physiologie ou médecine. En exploitant le processus biologique naturel de l’iARN se produisant dans nos cellules, une nouvelle classe de médicaments connue sous le nom de thérapeutique iARN est désormais une réalité. Les petits ARN interférents (ARNsi), les molécules qui médient l’iARN et composent la plateforme thérapeutique iARN d’Alnylam, fonctionnent en amont des médicaments actuels en inhibant puissamment l’ARN messager (ARNm), c’est-à-dire les précurseurs génétiques, qui codent pour les protéines pathogènes ou causant des maladies, empêchant ainsi leur fabrication. Il s’agit d’une approche révolutionnaire qui a le potentiel de transformer les soins aux patients atteints de maladies génétiques et autres. À propos d’Alnylam Pharmaceuticals Alnylam (Nasdaq : ALNY) est une société biopharmaceutique mondiale de premier plan et le pionnier de la révolution de l’interférence ARN (RNAi). La société se concentre sur le développement de thérapies transformatrices ayant le potentiel de prévenir, d’arrêter ou d’inverser la maladie. Depuis plus de deux décennies, Alnylam fait progresser la science de l’ARNi, récompensée par un prix Nobel, en réalisant des avancées décisives et en mettant au point six médicaments approuvés. Alnylam commercialise des médicaments dans plus de 70 pays et dispose d’un pipeline solide en pleine expansion, tout en étant régulièrement reconnue comme un lieu de travail exceptionnel et une organisation socialement responsable. La société met en œuvre sa stratégie Alnylam 2030 afin d’accélérer l’innovation et d’amplifier son impact pour transformer la santé humaine. Déclarations prospectives d’Alnylam Ce communiqué de presse contient des déclarations prospectives au sens de la section 27A du Securities Act de 1933 et de la section 21E du Securities Exchange Act de 1934. Toutes les déclarations autres que les déclarations factuelles historiques concernant les attentes, les convictions, les objectifs, les projets ou les perspectives d’Alnylam, y compris, sans s’y limiter, les déclarations concernant le potentiel du vutrisiran en tant que traitement de première intention pour l’ATTR-CM ; l’efficacité potentielle du vutrisiran chez les patients présentant des caractéristiques telles que la fibrillation auriculaire, une pression artérielle systolique basse et un fardeau élevé de comorbidités ; le nombre de patients qui seront recrutés dans l’étude DemonsTTRate, la durée de la période de suivi de ces patients et les données que l’étude générera ; et la capacité d’Alnylam à mettre en œuvre sa stratégie Alnylam 2030 visant à accélérer l’innovation et à amplifier son impact pour transformer la santé humaine, doivent être considérées comme des déclarations prospectives. Les résultats réels et les plans futurs peuvent différer matériellement de ceux indiqués dans ces déclarations prospectives en raison de divers risques, incertitudes et autres facteurs importants, y compris, mais sans s’y limiter, les risques et incertitudes liés à : la capacité d’Alnylam à mettre en œuvre avec succès sa stratégie « Alnylam 2030 » ; la capacité d’Alnylam à lancer, commercialiser et vendre avec succès ses produits approuvés à l’échelle mondiale, y compris l’AMVUTTRA ; la capacité d’Alnylam à découvrir et développer de nouveaux candidats-médicaments et de nouvelles approches d’administration, et à démontrer avec succès l’efficacité et la sécurité de ses candidats-produits ; les résultats précliniques et cliniques des produits candidats d’Alnylam ; les mesures ou avis des autorités réglementaires et la capacité d’Alnylam à obtenir et à conserver l’autorisation réglementaire pour ses produits candidats, ainsi qu’une tarification et un remboursement favorables ; les retards, interruptions ou défaillances dans la fabrication et la fourniture des produits commercialisés par Alnylam ou de ses produits candidats ; l’obtention, le maintien et la protection de la propriété intellectuelle ; la capacité d’Alnylam à gérer sa croissance et ses dépenses d’exploitation grâce à des investissements rigoureux dans ses activités ; la capacité d’Alnylam à maintenir des collaborations commerciales stratégiques ; la dépendance d’Alnylam vis-à-vis de tiers pour le développement et la commercialisation de certains produits ; l’issue des litiges et des enquêtes gouvernementales ; le risque de litiges et d’enquêtes gouvernementales futurs ; et les dépenses imprévues ; ainsi que les risques et incertitudes décrits plus en détail dans la section « Facteurs de risque » du rapport annuel 2025 d’Alnylam sur formulaire 10-K déposé auprès de la Securities and Exchange Commission (SEC), qui peuvent être mis à jour de temps à autre dans les rapports trimestriels ultérieurs d’Alnylam sur formulaire 10-Q, et dans d’autres documents déposés par Alnylam auprès de la SEC. En outre, toute déclaration prospective représente les opinions d’Alnylam uniquement à ce jour et ne doit pas être considérée comme représentant ses opinions à une date ultérieure. Alnylam décline explicitement toute obligation, sauf dans la mesure requise par la loi, de mettre à jour les déclarations prospectives. Le texte du communiqué issu d’une traduction ne doit d’aucune manière être considéré comme officiel. La seule version du communiqué qui fasse foi est celle du communiqué dans sa langue d’origine. La traduction devra toujours être confrontée au texte source, qui fera jurisprudence. Consultez la version source sur businesswire.com : https://www.businesswire.com/news/home/20260511170195/fr/ Alnylam Pharmaceuticals, Inc. Christine Akinc
(Investisseurs et médias)
+1-617-682-4340 Josh Brodsky
(Investisseurs)
+1-617-551-8276 Original: De nouvelles analyses de données présentées lors du congrès Heart Failure 2026 démontrent les bénéfices cliniques solides et constants du vutrisiran en tant qu’option thérapeutique de première intention chez toutes les populations

Heart Failure 2026: Neue Datenanalysen belegen robusten und konsistenten klinischen Nutzen von Vutrisiran als Erstlinien-Behandlungsoption für ATTR-CM-Patientenpopulationen – auch bei hoher KrankheitslastMay 13, 2026 2:28 PM
Business Wire – Die Verringerung der Gesamtmortalität und wiederkehrender kardiovaskulärer Ereignisse blieb in wichtigen Patientensubgruppen erhalten, einschließlich Patienten unter einem breiten Spektrum an Herzinsuffizienztherapien – - Eine gepoolte Analyse von Daten aus über 25.000 Patientenjahren mit TTR-silenzierenden RNAi-Therapien zeigt ein konsistentes Sicherheitsprofil ohne klinisch relevante okuläre Auswirkungen einer Vitamin-A-Senkung – - Die DemonsTTRate-Studie soll Real-World-Langzeitevidenz zu mehr als 2.000 Patienten mit ATTR-CM liefern - Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), das führende Unternehmen auf dem Gebiet der RNAi-Therapeutika, gab heute neue Analysen aus der HELIOS-B-Phase-3-Studie zu Vutrisiran bei Patienten mit Kardiomyopathie infolge einer Wildtyp- oder erblichen Transthyretin-vermittelten Amyloidose (ATTR-CM) bekannt. Diese ergänzen die stetig wachsende Evidenzbasis für Vutrisiran und untermauern den dauerhaften Knockdown von Transthyretin (TTR) sowie das gut charakterisierte Sicherheitsprofil von Vutrisiran. Vutrisiran ist der erste und einzige für ATTR-CM zugelassene TTR-Silencer, der einen schnellen Knockdown von TTR direkt an der Quelle bewirkt. Die auf dem Jahreskongress Heart Failure 2026 der Heart Failure Association der Europäischen Gesellschaft für Kardiologie präsentierten Daten zeigen einen konsistenten klinischen Nutzen bei allen Patientengruppen, die in der klinischen Praxis häufig anzutreffen sind – einschließlich solcher mit hoher Krankheitslast. Dies unterstützt den Einsatz des Arzneimittels als Option für die Erstlinienbehandlung dieser schnell fortschreitenden und lebensbedrohlichen Erkrankung. „Die auf der Heart Failure 2026 vorgestellten Analysen liefern wichtige Erkenntnisse über die Wirksamkeit von Vutrisiran bei den Patienten, die wir in der klinischen Praxis behandeln. Dazu gehören auch Patienten mit Begleiterkrankungen wie Vorhofflimmern, niedrigem systolischem Blutdruck und einer hohen Komorbiditätslast“, erklärt Scott Solomon, MD, Professor für Medizin an der Harvard Medical School und Kardiologe am Brigham and Women’s Hospital. „Diese neuen Analysen der HELIOS-B-Studie zeigen, dass der klinische Nutzen von Vutrisiran sowohl in diesen klinisch komplexen Patientengruppen als auch bei Patienten unter Begleittherapien, einschließlich TTR-Stabilisatoren und krankheitsmodifizierender Herzinsuffizienztherapien, erhalten blieb. Dies bekräftigt sowohl die Konsistenz des Behandlungseffekts als auch dessen Relevanz für die klinische Praxis unter Alltagsbedingungen. Insgesamt stützen diese Ergebnisse den Einsatz von Vutrisiran als Erstlinien-Behandlungsoption bei ATTR-CM in einem breiten Spektrum von Patientenpopulationen.“ Bei Patienten mit Vorhofflimmern, die etwa 65 % der HELIOS-B-Studienpopulation ausmachten und mit einer weiter fortgeschrittenen Erkrankung assoziiert waren, senkte Vutrisiran im Vergleich zu Placebo die Gesamtmortalität sowie das Risiko für wiederkehrende kardiovaskuläre Ereignisse signifikant. Die Behandlungseffekte blieben auch bei Patienten mit niedrigem systolischem Blutdruck (SBP) erhalten – einem Phänotyp mit erhöhtem Risiko. Vutrisiran verlangsamte dabei den im Zeitverlauf beobachteten allmählichen Rückgang des SBP. Der klinische Nutzen blieb unabhängig von der Komorbiditätslast oder der gleichzeitigen Anwendung krankheitsmodifizierender Therapien erhalten, insbesondere Tafamidis und Arzneimittel gegen Herzinsuffizienz wie SGLT2-Hemmer, MRA, ß-Blocker sowie ACE-Hemmer, ARB und ARNI. Eine konsistente Wirkung wurde auch bei Frauen festgestellt – einer Population, die in Studien zu ATTR-CM bislang unterrepräsentiert war. In einer separaten gepoolten Analyse klinischer Studiendaten und Sicherheitsdaten nach der Markteinführung wurde der Zusammenhang zwischen Transthyretin-senkenden RNAi-Therapien und unerwünschten Ereignissen im Zusammenhang mit Vitamin-A-Mangel untersucht. Patienten, die sich einer Behandlung mit Vutrisiran oder Patisiran unterziehen, wird empfohlen, die empfohlene Tagesdosis an Vitamin A einzunehmen. Die Analyse umfasste eine Behandlungsexposition von mehr als 25.000 Patientenjahren im Rahmen der Vutrisiran- und Patisiran-Programme. Die Häufigkeit von okulären unerwünschten Ereignissen, die möglicherweise mit einem Vitamin-A-Mangel in Zusammenhang stehen, war gering und mit der Placebo-Gruppe vergleichbar. Es wurden keine Fälle eines klinisch signifikanten Vitamin-A-Mangels beobachtet. „Vitamin A hat eine wichtige Funktion für das Sehvermögen und andere wesentliche Körperfunktionen. Obwohl Transthyretin an seinem Transport beteiligt ist, sorgen mehrere Stoffwechselwege für seine Verteilung im gesamten Körper“, erläutert William S. Blaner, Ph.D., Professor für Ernährungsmedizin an der Columbia University und Experte für den Vitamin-A-Stoffwechsel und -Transport. „Die in dieser breit angelegten Studie beobachteten niedrigen und mit Placebo vergleichbaren Häufigkeiten unerwünschter Ereignisse im Zusammenhang mit Vitamin-A-Mangel bestätigen, dass eine Senkung des Transthyretin-Spiegels diese Ereignisse bei Patienten mit ATTR-Amyloidose nicht maßgeblich erhöht.“ Des Weiteren stellte Alnylam das Studiendesign und die Rationale der DemonsTTRate-Studie vor, einer globalen, prospektiven Beobachtungsstudie zur Bewertung von Ergebnissen unter Alltagsbedingungen bei Patienten mit ATTR-CM. Insgesamt sollen mehr als 2.000 Patienten in die Studie aufgenommen und über einen Zeitraum von bis zu fünf Jahren beobachtet werden, um Längsschnittdaten zu klinischen Ergebnissen, Behandlungsmustern und der Inanspruchnahme von Gesundheitsleistungen im Rahmen der routinemäßigen klinischen Praxis zu gewinnen. Bei der ATTR-CM und der hereditären Transthyretin-vermittelten Amyloidose mit Polyneuropathie (hATTR-PN) übersteigt die weltweite Erfahrung mit Vutrisiran bislang 13.000 Patientenjahre und liefert damit eine solide und wachsende klinische Evidenzbasis für beide Krankheitsformen. Die Präsentationen von Alnylam auf der Heart Failure 2026 finden Sie unter Capella. AMVUTTRA® (Vutrisiran) INDIKATIONEN UND WICHTIGE SICHERHEITSINFORMATIONEN INDIKATIONEN In der EU ist AMVUTTRA® (Vutrisiran) zur Behandlung folgender Erkrankungen angezeigt: hereditäre Transthyretin-Amyloidose bei erwachsenen Patienten mit einer Polyneuropathie der Stadien 1 oder 2 (hATTR-PN) Wildtyp- oder hereditäre Transthyretin-Amyloidose bei erwachsenen Patienten mit Kardiomyopathie (ATTR-CM) Die Verfügbarkeit in der EU richtet sich nach den jeweiligen nationalen Erstattungsverfahren. Wichtige Sicherheitshinweise Verminderter Vitamin-A-Spiegel im Serum und empfohlene Nahrungsergänzung Die Behandlung mit Vutrisiran bewirkt eine Senkung des Vitamin-A-Spiegels im Serum. Für Patienten, die Vutrisiran einnehmen, wird eine Nahrungsergänzung von etwa 2500 IE bis maximal 3000 IE Vitamin A pro Tag empfohlen. Patienten sollten an einen Augenarzt überwiesen werden, wenn sie Augensymptome entwickeln, die auf einen Vitamin-A-Mangel hindeuten (beispielsweise Nachtblindheit). Unerwünschte Reaktionen Häufig berichtete Nebenwirkungen während der Behandlung mit Vutrisiran waren Reaktionen an der Injektionsstelle sowie erhöhte Werte der alkalischen Phosphatase und der Alaninaminotransferase im Blut. Weitere Informationen zu Vutrisiran finden Sie in der vollständigen Zusammenfassung der Merkmale des Arzneimittels. Über AMVUTTRA® (Vutrisiran) AMVUTTRA® (Vutrisiran) ist ein Transthyretin-(TTR-)Silencer, der einen raschen Knockdown von TTR direkt an der Quelle bewirkt, um die zugrunde liegende Ursache der Transthyretin-Amyloidose (ATTR) zu bekämpfen. In einer klinischen Studie führte AMVUTTRA innerhalb von nur sechs Wochen zu einer raschen Senkung des TTR-Spiegels und senkte diesen nach zweieinhalbjähriger Behandlung um 87 %. Das Arzneimittel ist in verschiedenen Ländern weltweit zur Behandlung der Polyneuropathie bei der hereditären Transthyretin-vermittelten Amyloidose (hATTR-PN) bei Erwachsenen sowie der Kardiomyopathie bei der Wildtyp- oder hereditären Transthyretin-vermittelten Amyloidose (ATTR-CM) bei Erwachsenen zugelassen. AMVUTTRA wird vierteljährlich als subkutane Injektion verabreicht und ist das erste und einzige Silencer-Medikament, das für die Behandlung von ATTR-CM und hATTR-PN zugelassen ist. Über Transthyretin-Amyloidose (ATTR) Transthyretin-Amyloidose (ATTR) ist eine unterdiagnostizierte, rapide fortschreitende, schwächende und tödliche Erkrankung, die durch pathogene Transthyretin-(TTR-)Proteine hervorgerufen wird, die sich als Amyloid-Ablagerungen in verschiedenen Teilen des Körpers ansammeln, insbesondere in den Nerven, im Herzen und im Magen-Darm-Trakt. Die Patienten können an einer Polyneuropathie, einer Kardiomyopathie oder an beiden Erscheinungsformen der Krankheit leiden. Es gibt zwei verschiedene Formen von ATTR – die hereditäre ATTR (hATTR), die durch eine TTR-Genvariante hervorgerufen wird, und die Wildtyp-ATTR (wtATTR), die ohne eine TTR-Genvariante auftritt. Schätzungen zufolge leben weltweit mehr als 500.000 Menschen mit ATTR. Über RNAi RNAi (RNA-Interferenz) ist ein natürlicher, zellulärer Prozess zur Stummschaltung von Genen („Gene Silencing“), der einen der derzeit vielversprechendsten und am schnellsten voranschreitenden Grenzbereiche in Biologie und Arzneimittelentwicklung darstellt. Ihre Entdeckung wurde gewürdigt als „bedeutender wissenschaftlicher Durchbruch, wie er nur einmal pro Jahrzehnt vorkommt“ und 2006 mit dem Nobelpreis für Physiologie oder Medizin ausgezeichnet. Durch Nutzung des natürlichen biologischen Prozesses der RNAi in unseren Zellen ist eine bedeutende neue Klasse von Medikamenten entstanden, die als RNAi-Therapeutika bekannt sind. Small interfering RNA (siRNA) – die Moleküle, die RNAi vermitteln und die therapeutische RNAi-Plattform von Alnylam bilden, funktionieren auf einer den heutigen Arzneimitteln vorgelagerten Ebene durch wirksame Stummschaltung der Messenger-RNA (mRNA) – genetische Vorläufer, die die krankheitsauslösenden Proteine kodieren – und verhindern auf diese Weise deren Bildung. Es handelt sich hier um einen bahnbrechenden Ansatz mit dem Potenzial, die Versorgung von Patienten mit genetischen und anderen Erkrankungen grundlegend zu verändern. Über Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) ist ein weltweit führendes biopharmazeutisches Unternehmen und Wegbereiter der RNA-Interferenz-Revolution (RNAi). Das Unternehmen widmet sich der Entwicklung bahnbrechender Therapien mit dem Potenzial, Krankheiten vorzubeugen, sie aufzuhalten oder ihren Verlauf umzukehren. Seit über zwei Jahrzehnten treibt Alnylam die mit dem Nobelpreis gekrönte Wissenschaft der RNAi voran. Dabei hat das Unternehmen entscheidende Durchbrüche erzielt und sechs zugelassene Arzneimittel hervorgebracht. Alnylam bietet Arzneimittel in mehr als 70 Ländern an und verfügt über eine schnell wachsende und starke Pipeline. Darüber hinaus wird das Unternehmen regelmäßig als hervorragender Arbeitgeber und sozial verantwortliches Unternehmen ausgezeichnet. Das Unternehmen setzt seine Strategie Alnylam 2030 um, um Innovationen zu beschleunigen und seine Wirkung zu skalieren – mit dem Ziel, die menschliche Gesundheit grundlegend zu verbessern. Zukunftsgerichtete Aussagen von Alnylam Diese Pressemitteilung enthält zukunftsgerichtete Aussagen im Sinne von Abschnitt 27A des Securities Act von 1933 und Abschnitt 21E des Securities Exchange Act von 1934. Alle Aussagen, die sich nicht auf historische Fakten beziehen und die Alnylams Erwartungen, Überzeugungen, Ziele, Pläne oder Aussichten betreffen, unter anderem Aussagen zum Potenzial von Vutrisiran als Erstlinienbehandlung bei ATTR-CM, zur potenziellen Wirksamkeit von Vutrisiran bei Patienten mit Symptomen wie Vorhofflimmern, niedrigem systolischem Blutdruck und hoher Komorbiditätslast, zur Anzahl der Patienten, die in die DemonsTTRate-Studie aufgenommen werden, die Dauer der Nachbeobachtungszeit für diese Patienten und die Daten, die die Studie generieren wird, sowie zur Fähigkeit von Alnylam, seine Strategie Alnylam 2030 umzusetzen, um Innovationen zu beschleunigen und die Transformation der Gesundheitsversorgung voranzutreiben, sind als zukunftsgerichtete Aussagen zu betrachten. Die tatsächlichen Ergebnisse und Zukunftspläne können aufgrund verschiedener wichtiger Risiken, Unwägbarkeiten und anderer Faktoren erheblich von denen abweichen, die in diesen zukunftsgerichteten Aussagen enthalten sind, einschließlich von Risiken und Unwägbarkeiten in Bezug auf: die Fähigkeit von Alnylam, seine Strategie Alnylam 2030 erfolgreich umzusetzen; die Fähigkeit von Alnylam, seine zugelassenen Produkte, einschließlich AMVUTTRA, weltweit erfolgreich einzuführen, zu vermarkten und zu verkaufen; die Fähigkeit von Alnylam, neuartige Wirkstoffkandidaten und Verabreichungsansätze zu entdecken und zu entwickeln sowie die Wirksamkeit und Sicherheit seiner Produktkandidaten erfolgreich nachzuweisen; die präklinischen und klinischen Ergebnisse für die Produktkandidaten von Alnylam; Maßnahmen oder Empfehlungen von Aufsichtsbehörden und die Fähigkeit von Alnylam, behördliche Zulassungen für seine Produktkandidaten zu erlangen und aufrechtzuerhalten sowie günstige Preis- und Erstattungsbedingungen zu erreichen; Verzögerungen, Unterbrechungen oder Ausfälle bei der Herstellung und Lieferung der vermarkteten Produkte oder Produktkandidaten von Alnylam; die Erlangung, Aufrechterhaltung und der Schutz geistigen Eigentums; die Fähigkeit von Alnylam, sein Wachstum und seine Betriebsausgaben durch disziplinierte Investitionen in den Geschäftsbetrieb zu steuern; die Fähigkeit von Alnylam, strategische geschäftliche Kooperationen aufrechtzuerhalten; die Abhängigkeit des Unternehmens von Dritten bei der Entwicklung und Vermarktung bestimmter Produkte; der Ausgang von Rechtsstreitigkeiten und behördlichen Untersuchungen; das Risiko künftiger Rechtsstreitigkeiten und behördlicher Untersuchungen; unerwartete Ausgaben; sowie die Risiken und Unwägbarkeiten, die ausführlicher unter „Risk Factors“ im Annual Report 2025 von Alnylam auf Form 10-K erörtert werden, der bei der Securities and Exchange Commission (SEC) eingereicht wurde, und die von Zeit zu Zeit in den nachfolgenden Quarterly Reports von Alnylam auf Form 10-Q sowie in anderen von Alnylam bei der SEC eingereichten Unterlagen aktualisiert werden können. Alle zukunftsgerichteten Aussagen spiegeln die Einschätzungen von Alnylam nur am Tag der Veröffentlichung wider und gelten nicht für einen späteren Zeitpunkt als das Datum der Veröffentlichung dieser Pressemitteilung. Außerhalb des gesetzlich vorgegebenen Rahmens lehnt Alnylam ausdrücklich jegliche Verpflichtung zur Aktualisierung von zukunftsgerichteten Aussagen ab. Die Ausgangssprache, in der der Originaltext veröffentlicht wird, ist die offizielle und autorisierte Version. Übersetzungen werden zur besseren Verständigung mitgeliefert. Nur die Sprachversion, die im Original veröffentlicht wurde, ist rechtsgültig. Gleichen Sie deshalb Übersetzungen mit der originalen Sprachversion der Veröffentlichung ab. Originalversion auf businesswire.com ansehen: https://www.businesswire.com/news/home/20260511183054/de/ Alnylam Pharmaceuticals, Inc. Christine Akinc
(Investoren und Medien)
+1-617-682-4340 Josh Brodsky
(Investoren)
+1-617-551-8276 Original: Heart Failure 2026: Neue Datenanalysen belegen robusten und konsistenten klinischen Nutzen von Vutrisiran als Erstlinien-Behandlungsoption für ATTR-CM-Patientenpopulationen – auch bei hoher Krankheitslast

New Data Analyses Presented at Heart Failure 2026 Demonstrate Robust and Consistent Clinical Benefit of Vutrisiran as a First-Line Treatment Option Across ATTR-CM Patient Populations, Including Patients with a High Disease BurdenMay 12, 2026 2:30 AM
Business Wire - Reductions in All-Cause Mortality and Recurrent Cardiovascular Events Maintained Across Key Patient Subgroups, Including Patients Taking a Broad Range of Heart Failure Therapies – - Pooled Analysis of Over 25,000 Patient-Years of Experience with TTR-Silencing RNAi Therapies Shows a Consistent Safety Profile, Including No Clinically Meaningful Ocular Effects of Vitamin A Lowering – - DemonsTTRate Study Designed to Generate Long-Term Real-World Evidence in More Than 2,000 Patients with ATTR-CM - Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new analyses from the HELIOS-B Phase 3 study of vutrisiran in patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), adding to the growing body of evidence supporting vutrisiran and reinforcing the durability of transthyretin (TTR) knockdown and its well-characterized safety profile. Vutrisiran is the first and only TTR silencer approved for ATTR-CM that is designed to deliver rapid knockdown of TTR at the source. The data presented at Heart Failure 2026, the annual congress of the Heart Failure Association of the European Society of Cardiology, show consistent clinical benefit across patient populations commonly encountered in clinical practice, including those with a high disease burden, supporting its use as a first-line treatment option for this rapidly progressive and life-threatening disease. “The analyses presented at Heart Failure 2026 provide important insights into how vutrisiran performs across the patients we see in clinical practice, including those who present with features such as atrial fibrillation, low systolic blood pressure and a high comorbidity burden,” said Scott Solomon, M.D., Professor of Medicine at Harvard Medical School and cardiologist at Brigham and Women’s Hospital. “These new HELIOS-B analyses show that the clinical benefits of vutrisiran were maintained across these clinically complex patient groups, as well as in patients receiving background therapies, including TTR stabilizers and disease-modifying heart failure therapies, reinforcing both the consistency of the treatment effect and its relevance in real-world clinical practice. Taken together, these findings support the use of vutrisiran as a first-line treatment option for ATTR-CM across a broad range of patient populations.” In patients with atrial fibrillation, representing approximately 65% of the HELIOS-B study population and associated with more advanced disease, vutrisiran significantly reduced the risk of all-cause mortality and recurrent cardiovascular (CV) events compared with placebo. Treatment effects were also maintained in patients with low systolic blood pressure (SBP), a higher-risk phenotype, with vutrisiran slowing the progressive decline in SBP observed over time. Clinical benefits were similarly consistent regardless of comorbidity burden or concomitant use of disease-modifying therapies, including tafamidis and heart failure medications such as SGLT2 inhibitors, MRAs, ß-blockers and ACEi/ARB/ARNI. Consistent effects were also observed in women, a historically underrepresented population in ATTR-CM trials. A separate pooled analysis of clinical trial and post-marketing safety data evaluated the relationship between transthyretin-lowering RNAi therapies and vitamin A deficiency-related adverse events. Patients treated with vutrisiran and patisiran are suggested to take the recommended daily allowance of vitamin A. The analysis included more than 25,000 patient-years of treatment exposure across vutrisiran and patisiran programs. Rates of ocular adverse events potentially associated with vitamin A deficiency were low and comparable to placebo. No cases of clinically meaningful vitamin A deficiency were observed. “Vitamin A plays an essential role in vision and other key physiological functions. While transthyretin contributes to its transport, multiple pathways support its delivery throughout the body,” said William S. Blaner, Ph.D., Professor of Nutritional Medicine at Columbia University and expert in vitamin A metabolism and transport. “The low and comparable to placebo rates of vitamin A deficiency-related adverse events observed in this large analysis provide strong reassurance that lowering transthyretin does not meaningfully increase these events in patients with ATTR amyloidosis.” Alnylam also presented the design and rationale of the DemonsTTRate study, a global, prospective, observational study evaluating real-world outcomes in patients with ATTR-CM. The study is expected to enroll more than 2,000 patients and follow them for up to five years, generating longitudinal data on clinical outcomes, treatment patterns and healthcare utilization across routine clinical practice. Across ATTR-CM and hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN), worldwide experience with vutrisiran to date exceeds 13,000 patient-years, reflecting a robust and expanding body of clinical evidence across both manifestations of the disease. To view Alnylam’s Heart Failure 2026 presentations please visit Capella. AMVUTTRA® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION Indications In the EU, AMVUTTRA® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Availability across the EU is subject to local reimbursement timelines. Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. For additional information about vutrisiran, please see the full Summary of Product Characteristics. About AMVUTTRA® (vutrisiran) AMVUTTRA® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN. About Transthyretin Amyloidosis (ATTR) Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding the potential for vutrisiran to be used as a first-line treatment for ATTR-CM; the potential efficacy of vutrisiran in patients who present with features such as atrial fibrillation, low systolic blood pressure and a high comorbidity burden; the number of patients who will be enrolled in the DemonsTTRate study, the duration of the follow-up period for those patients, and the data the study will generate; and Alnylam’s ability to execute on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam 2030” strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. View source version on businesswire.com: https://www.businesswire.com/news/home/20260511111257/en/ Alnylam Pharmaceuticals, Inc. Christine Akinc
(Investors and Media)
+1-617-682-4340 Josh Brodsky
(Investors)
+1-617-551-8276 Original: New Data Analyses Presented at Heart Failure 2026 Demonstrate Robust and Consistent Clinical Benefit of Vutrisiran as a First-Line Treatment Option Across ATTR-CM Patient Populations, Including Patients with a High Disease Burden

Alnylam präsentiert auf der „Heart Failure 2026“ neue Analysen, die das konsistente klinische Profil von Vutrisiran bei ATTR-CM unterstreichenMay 4, 2026 8:39 PM
Business Wire Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), das führende Unternehmen im Bereich der RNAi-Therapeutika, gab heute bekannt, dass es auf der „Heart Failure 2026“, einem wissenschaftlichen Kongress der European Society of Cardiology, der vom 9. bis 12. Mai 2026 in Barcelona, Spanien, stattfindet, neue Datenanalysen zu Vutrisiran bei Patienten mit Kardiomyopathie aufgrund einer Wildtyp- oder erblichen Transthyretin-vermittelten Amyloidose (ATTR-CM) vorstellen wird. ATTR-CM ist eine heterogene und schnell fortschreitende Erkrankung, und Patienten weisen oft komplexe klinische Profile auf. Auf der „Heart Failure 2026“ wird Alnylam mehrere neue Analysen aus der Phase-3-Studie HELIOS-B zu Vutrisiran vorstellen, die dessen Einsatz als Erstlinientherapie für Patienten mit ATTR-CM weiterhin stützen und dessen klinisches Profil bei Patienten mit hoher Krankheitslast sowie im Kontext von Begleittherapien weiter charakterisieren. Vutrisiran ist der erste und einzige Transthyretin (TTR)-Silencer für ATTR-CM, der eine schnelle Herunterregulierung von TTR an der Quelle bewirkt. Zu den weiteren auf dem Kongress vorgestellten Analysen gehören eine pharmakodynamische Analyse der TTR-Herabsetzung durch Vutrisiran in verschiedenen Subgruppen, eine gepoolte Sicherheitsanalyse zur Bewertung der Häufigkeit von TTR-Senkungen in Verbindung mit Vitamin-A-bezogenen Endpunkten anhand von Daten aus klinischen Studien und der Praxis sowie das Design von DemonsTTRate, einer globalen Langzeit-Beobachtungsstudie zur Bewertung der Anwendung in der Praxis bei Patienten mit ATTR-CM. Details zur Präsentation Design und Begründung von DemonsTTRate: Eine globale, langfristige Beobachtungsstudie zur Bewertung von Vutrisiran bei Patienten mit Transthyretin-Amyloidose mit Kardiomyopathie
Sitzung: Weiterentwicklung der Behandlung von Kardiomyopathien: Früherkennung, Präzisionsdiagnostik und neue Therapien (Moderierte ePosters 3)
Sonntag, 10. Mai, 08:30 – 09:15 Uhr MESZ, 02:30 – 03:15 Uhr EST
Vortragender Autor: Pablo Garcia-Pavia, Spanien Einfluss von Vutrisiran auf den systolischen Blutdruck bei ATTR-CM: Erkenntnisse aus HELIOS-B
Sitzung: Herzamyloidose: therapeutische Strategien und klinische Ergebnisse (Moderierte ePosters 4)
Sonntag, 10. Mai, 15:30 – 16:15 Uhr MESZ, 9:30 – 10:15 Uhr EST
Vortragender Autor: Awais Sheikh, Großbritannien Wirkung von Vutrisiran bei Patienten mit und ohne Vorhofflimmern oder Vorhofflattern: Analyse aus HELIOS-B
Sitzung: Fortschritte bei Amyloidose und hypertropher Kardiomyopathie: von der Diagnose bis zur Behandlung (Moderierte ePosters 2)
Montag, 11. Mai, 10:30 – 11:15 Uhr MESZ, 4:30 – 5:15 Uhr EST
Vortragender Autor: Xiaowen Wang, USA Die Behandlung mit Transthyretin-senkenden RNA-Interferenz-Therapeutika ist nicht mit Augenproblemen oder anderen klinischen Ereignissen aufgrund einer Vitamin-A-Reduktion assoziiert: Pooled-Analyse der Daten zu Vutrisiran und Patisiran
Sitzung: Fortschritte bei Amyloidose und hypertropher Kardiomyopathie: von der Diagnose bis zur Behandlung (Moderierte ePosters 2)
Montag, 11. Mai, 10:30 – 11:15 Uhr MESZ, 4:30 – 5:15 Uhr EST
Vortragender Autor: Mathew Maurer, USA Vutrisiran-vermittelter Knockdown von Transthyretin bei Patienten mit Transthyretin-Amyloidose
Sitzung: Transthyretin-Amyloidose: Langzeitergebnisse und neue Therapien (Rapid Fire Abstracts)
Montag, 11. Mai, 16:45 – 17:45 Uhr MESZ, 10:45 – 11:45 Uhr EST
Vortragender Autor: Vincent Algalarrondo, Frankreich Einfluss einer krankheitsmodifizierenden Therapie auf die Wirksamkeit von Vutrisiran bei Transthyretin-Herzamyloidose
Sitzung: Transthyretin-Amyloidose: Langzeitergebnisse und neue Therapien (Rapid Fire Abstracts)
Montag, 11. Mai, 16:45 – 17:45 Uhr MESZ, 10:45 – 11:45 Uhr EST
Vortragende Autorin: Arielle Abovich, USA Konsistente Wirksamkeit von Vutrisiran bei beiden Geschlechtern bei ATTR-CM, HELIOS-B-Studie – vorab festgelegte geschlechtsspezifische Analyse
Sitzung: Transthyretin-Amyloidose: Langzeitergebnisse und neue Therapien (Rapid Fire Abstracts)
Montag, 11. Mai, 16:45 – 17:45 Uhr MESZ, 10:45 – 11:45 Uhr EST
Vortragende Autorin: Josephine Mansell, Großbritannien Komorbiditätslast bei Transthyretin-Amyloidose mit Kardiomyopathie: Erkenntnisse aus der HELIOS-B-Studie
Sitzung: Risikofaktoren und Komorbiditäten (Moderierte ePosters 3)
Dienstag, 12. Mai, 08:30 bis 09:15 Uhr MESZ, 02:30 – 03:15 Uhr EST
Vortragende Autorin: Sarah Birkhoelzer, Großbritannien AMVUTTRA® (Vutrisiran) INDIKATIONEN UND WICHTIGE SICHERHEITSHINWEISE Indikationen
In der EU ist AMVUTTRA® (Vutrisiran) indiziert zur Behandlung von: hereditärer Transthyretin-Amyloidose bei erwachsenen Patienten mit Polyneuropathie im Stadium 1 oder 2 (hATTR-PN). Wildtyp- oder hereditärer Transthyretin-Amyloidose bei erwachsenen Patienten mit Kardiomyopathie (ATTR-CM). Die Verfügbarkeit in der EU hängt von den lokalen Erstattungsfristen ab. Wichtige Sicherheitsinformationen
Verminderte Vitamin-A-Serumspiegel und empfohlene Supplementierung
Die Behandlung mit Vutrisiran führt zu einer Abnahme der Vitamin-A-Serumspiegel. Patienten, die Vutrisiran einnehmen, wird eine Supplementierung von etwa 2500 IE bis 3000 IE Vitamin A pro Tag empfohlen, wobei diese Menge nicht überschritten werden sollte. Patienten sollten an einen Augenarzt überwiesen werden, wenn sie Augensymptome entwickeln, die auf einen Vitamin-A-Mangel hindeuten (z. B. Nachtblindheit). Unerwünschte Nebenwirkungen
Häufig berichtete unerwünschte Nebenwirkungen unter Vutrisiran waren Reaktionen an der Injektionsstelle sowie ein Anstieg der alkalischen Phosphatase und der Alaninaminotransferase im Blut. Weitere Informationen zu Vutrisiran finden Sie in der vollständigen Zusammenfassung der Merkmale des Arzneimittels. Über AMVUTTRA® (Vutrisiran)
AMVUTTRA® (Vutrisiran) ist ein Transthyretin (TTR)-Silencer, der eine rasche Herunterregulierung von TTR an der Quelle bewirkt, um die zugrunde liegende Ursache der Transthyretin-Amyloidose (ATTR) zu bekämpfen. In einer klinischen Studie führte AMVUTTRA bereits nach sechs Wochen zu einer raschen Herunterregulierung von TTR und senkte die TTR-Spiegel nach zweieinhalbjähriger Behandlung um 87 %. Es ist weltweit in verschiedenen Ländern zur Behandlung der Polyneuropathie bei hereditärer Transthyretin-vermittelter Amyloidose (hATTR-PN) bei Erwachsenen und der Kardiomyopathie bei Wildtyp- oder hereditärer Transthyretin-vermittelter Amyloidose (ATTR-CM) bei Erwachsenen zugelassen. AMVUTTRA wird vierteljährlich als subkutane Injektion verabreicht und ist der erste und einzige Silencer, der für die Behandlung von ATTR-CM und hATTR-PN zugelassen ist. Über Transthyretin Amyloidosis (ATTR)
Die Transthyretin-Amyloidose (ATTR) ist eine unterdiagnostizierte, schnell fortschreitende, schwerwiegende und tödliche Erkrankung, die durch pathogene Transthyretin (TTR)-Proteine verursacht wird, welche sich als Amyloidablagerungen in verschiedenen Körperregionen, darunter Nerven, Herz und Magen-Darm-Trakt, ansammeln. Bei Patienten können Polyneuropathie, Kardiomyopathie oder beide Krankheitsbilder auftreten. Es gibt zwei verschiedene Formen von ATTR – die hereditäre ATTR (hATTR), die durch eine TTR-Genvariante verursacht wird, und die Wildtyp-ATTR (wtATTR), die ohne eine TTR-Genvariante auftritt. Schätzungen zufolge leben weltweit mehr als 500.000 Menschen mit ATTR. Über RNAi
RNAi (RNA-Interferenz) ist ein natürlicher zellulärer Prozess der Gen-Silenzierung, der heute eine der vielversprechendsten und sich am schnellsten entwickelnden Bereiche in der Biologie und Arzneimittelentwicklung darstellt. Seine Entdeckung wurde als „ein bedeutender wissenschaftlicher Durchbruch, wie er etwa einmal pro Jahrzehnt vorkommt“ gepriesen und 2006 mit dem Nobelpreis für Physiologie oder Medizin gewürdigt. Durch die Nutzung des natürlichen biologischen Prozesses der RNAi, der in unseren Zellen abläuft, ist eine neue Klasse von Medikamenten, bekannt als RNAi-Therapeutika, nun Realität geworden. Kleine interferierende RNA (siRNA), die Moleküle, die die RNAi vermitteln und die RNAi-Therapieplattform von Alnylam bilden, wirken vorgelagert zu den heutigen Medikamenten, indem sie die Boten-RNA (mRNA) – die genetischen Vorläufer –, die für krankheitsverursachende oder krankheitsrelevante Proteine kodiert, wirksam stilllegen und so deren Bildung verhindern. Dies ist ein revolutionärer Ansatz mit dem Potenzial, die Versorgung von Patienten mit genetischen und anderen Erkrankungen grundlegend zu verändern. Über Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) ist ein weltweit führendes biopharmazeutisches Unternehmen und Pionier der RNA-Interferenz (RNAi)-Revolution. Das Unternehmen konzentriert sich auf die Entwicklung transformativer Therapien, die das Potenzial haben, Krankheiten zu verhindern, zu stoppen oder rückgängig zu machen. Seit mehr als zwei Jahrzehnten treibt Alnylam die mit dem Nobelpreis ausgezeichnete Wissenschaft der RNAi voran und hat entscheidende Durchbrüche sowie sechs zugelassene Medikamente hervorgebracht. Alnylam verfügt über Medikamente, die in mehr als 70 Ländern erhältlich sind, sowie über eine schnell wachsende und robuste Pipeline. Darüber hinaus wird das Unternehmen durchweg als außergewöhnlicher Arbeitgeber und sozial verantwortliches Unternehmen anerkannt. Das Unternehmen setzt seine Strategie Alnylam 2030 um, um Innovationen zu beschleunigen und die Wirkung zu vergrößern, um die menschliche Gesundheit zu transformieren. Zukunftsgerichtete Aussagen von Alnylam
Diese Pressemitteilung enthält zukunftsgerichtete Aussagen im Sinne von Abschnitt 27A des Securities Act von 1933 und Abschnitt 21E des Securities Exchange Act von 1934. Alle Aussagen, die keine historischen Tatsachen darstellen und sich auf die Erwartungen, Überzeugungen, Ziele, Pläne oder Aussichten von Alnylam beziehen, einschließlich, aber nicht beschränkt auf Aussagen zum Potenzial von AMVUTTRA als Erstlinienbehandlung für ATTR-CM sowie zur Fähigkeit von Alnylam, seine Strategie Alnylam 2030 umzusetzen, um Innovationen zu beschleunigen und die Wirkung zu vergrößern, um die menschliche Gesundheit zu transformieren, sind als zukunftsgerichtete Aussagen zu betrachten. Die tatsächlichen Ergebnisse und zukünftigen Pläne können aufgrund verschiedener wichtiger Risiken, Ungewissheiten und anderer Faktoren erheblich von den in diesen zukunftsgerichteten Aussagen dargestellten abweichen, einschließlich, aber nicht beschränkt auf Risiken und Ungewissheiten in Bezug auf: die Fähigkeit von Alnylam, seine „Alnylam 2030“-Strategie erfolgreich umzusetzen; die Fähigkeit von Alnylam, seine zugelassenen Produkte, einschließlich AMVUTTRA, weltweit erfolgreich auf den Markt zu bringen, zu vermarkten und zu verkaufen; die Fähigkeit von Alnylam, neuartige Wirkstoffkandidaten und Verabreichungsansätze zu entdecken und zu entwickeln sowie die Wirksamkeit und Sicherheit seiner Produktkandidaten erfolgreich nachzuweisen; die präklinischen und klinischen Ergebnisse für die Produktkandidaten von Alnylam; Maßnahmen oder Empfehlungen von Aufsichtsbehörden und die Fähigkeit von Alnylam, behördliche Zulassungen für seine Produktkandidaten zu erhalten und aufrechtzuerhalten sowie günstige Preisgestaltung und Erstattungen zu erzielen; Verzögerungen, Unterbrechungen oder Ausfälle bei der Herstellung und Lieferung der vermarkteten Produkte oder Produktkandidaten von Alnylam; die Erlangung, Aufrechterhaltung und der Schutz von geistigem Eigentum; die Fähigkeit von Alnylam, sein Wachstum und seine Betriebsausgaben durch disziplinierte Investitionen in den Betrieb zu steuern; die Fähigkeit von Alnylam, strategische Geschäftskooperationen aufrechtzuerhalten; die Abhängigkeit von Alnylam von Dritten bei der Entwicklung und Vermarktung bestimmter Produkte; der Ausgang von Rechtsstreitigkeiten und behördlichen Untersuchungen; das Risiko künftiger Rechtsstreitigkeiten und behördlicher Untersuchungen; sowie unerwartete Ausgaben; sowie jene Risiken und Ungewissheiten, die ausführlicher in den „Risikofaktoren“ des Jahresberichts 2025 von Alnylam auf Formular 10-K, der bei der Securities and Exchange Commission (SEC) eingereicht wurde, erörtert werden und die von Zeit zu Zeit in den nachfolgenden Quartalsberichten von Alnylam auf Formular 10-Q sowie in anderen bei der SEC eingereichten Unterlagen aktualisiert werden können. Darüber hinaus geben zukunftsgerichtete Aussagen lediglich die Ansichten von Alnylam zum heutigen Zeitpunkt wieder und sollten nicht als Darstellung der Ansichten des Unternehmens zu einem späteren Zeitpunkt herangezogen werden. Alnylam lehnt ausdrücklich jede Verpflichtung ab, zukunftsgerichtete Aussagen zu aktualisieren, sofern dies nicht gesetzlich vorgeschrieben ist. Die Ausgangssprache, in der der Originaltext veröffentlicht wird, ist die offizielle und autorisierte Version. Übersetzungen werden zur besseren Verständigung mitgeliefert. Nur die Sprachversion, die im Original veröffentlicht wurde, ist rechtsgültig. Gleichen Sie deshalb Übersetzungen mit der originalen Sprachversion der Veröffentlichung ab. Originalversion auf businesswire.com ansehen: https://www.businesswire.com/news/home/20260504195993/de/ Alnylam Pharmaceuticals, Inc.
Christine Akinc
(Investoren und Medien)
+1-617-682-4340 Josh Brodsky
(Investoren)
+1-617-551-8276 Original: Alnylam präsentiert auf der „Heart Failure 2026“ neue Analysen, die das konsistente klinische Profil von Vutrisiran bei ATTR-CM unterstreichen

Alnylam présentera de nouvelles analyses lors du congrès Heart Failure 2026, soulignant le profil clinique constant du vutrisiran dans l’ATTR-CMMay 4, 2026 8:39 PM
Business Wire Alnylam Pharmaceuticals, Inc. (Nasdaq : ALNY), société leader dans le domaine des thérapies par ARNi, a annoncé aujourd’hui qu’elle présentera de nouvelles analyses de données concernant le vutrisiran chez des patients atteints de cardiomyopathie liée à l’amylose à transthyrétine de type sauvage ou héréditaire (ATTR-CM) lors du congrès Heart Failure 2026, un congrès scientifique de la Société européenne de cardiologie qui se tiendra du 9 au 12 mai 2026 à Barcelone, en Espagne. L’ATTR-CM est une maladie hétérogène et à progression rapide, et les patients présentent souvent des profils cliniques complexes. Lors du congrès Heart Failure 2026, Alnylam présentera plusieurs nouvelles analyses issues de l’étude de phase 3 HELIOS-B sur le vutrisiran, qui continuent de soutenir son utilisation en tant que traitement de première intention chez les patients atteints d’ATTR-CM, et qui caractérisent davantage son profil clinique chez les patients présentant une charge de morbidité élevée, ainsi que dans le contexte de traitements concomitants. Le vutrisiran est le premier et le seul inhibiteur de la transthyrétine (TTR) pour l’ATTR-CM qui permet une réduction rapide de la TTR à la source. Parmi les analyses supplémentaires présentées lors du congrès figurent une analyse pharmacodynamique de la réduction de la TTR par le vutrisiran dans différents sous-groupes, une analyse de sécurité groupée évaluant l’incidence de la réduction de la TTR sur les résultats liés à la vitamine A à partir de données issues d’essais cliniques et de la pratique clinique, ainsi que la conception de DemonsTTRate, une étude observationnelle mondiale à long terme évaluant l’utilisation en pratique clinique chez les patients atteints d’ATTR-CM. Détails de la présentation Conception et justification de DemonsTTRate : une étude observationnelle mondiale à long terme visant à évaluer le vutrisiran chez les patients atteints d’amylose à transthyrétine avec cardiomyopathie
Session : Évolution de la prise en charge des cardiomyopathies : détection précoce, diagnostics de précision et traitements émergents (ePosters modérés 3)
Dimanche 10 mai, 08h30 – 09h15 CEST, 02h30 – 03h15 EST
Auteur de la présentation : Pablo Garcia-Pavia, Espagne Influence du vutrisiran sur la pression artérielle systolique dans l’ATTR-CM : conclusions de l’étude HELIOS-B
Session : Amylose cardiaque : stratégies thérapeutiques et résultats cliniques (ePosters modérés 4)
Dimanche 10 mai, 15h30 – 16h15 CEST, 09h30 – 10h15 EST
Auteur de la présentation : Awais Sheikh, Royaume-Uni Effet du vutrisiran chez les patients avec ou sans fibrillation ou flutter auriculaire : analyse tirée de l’étude HELIOS-B
Session : Progrès en matière d’amylose et de cardiomyopathie hypertrophique : du diagnostic au traitement (ePosters modérés 2)
Lundi 11 mai, 10h30 – 11h15 CEST, 4h30 – 5h15 EST
Auteur de la présentation : Xiaowen Wang, États-Unis d’Amérique Le traitement par des agents thérapeutiques à interférence ARN réduisant la transthyrétine n’est pas associé à des événements oculaires ou autres événements cliniques dus à une réduction de la vitamine A : Analyse groupée des données sur le vutrisiran et le patisiran
Session : Progrès en matière d’amylose et de cardiomyopathie hypertrophique : du diagnostic au traitement (ePosters modérés 2)
Lundi 11 mai, 10h30 – 11h15 CEST, 4h30 – 5h15 EST
Auteur de la présentation : Mathew Maurer, États-Unis d’Amérique Inhibition de la transthyrétine induite par le vutrisiran chez les patients atteints d’amylose à transthyrétine
Session : Amylose à transthyrétine : résultats à long terme et nouvelles thérapies (Résumés éclair)
Lundi 11 mai, 16h45 – 17h45 CEST, 10h45 – 11h45 EST
Auteur de la présentation : Vincent Algalarrondo, France Influence du traitement modificateur de la maladie sur l’efficacité du vutrisiran dans l’amylose cardiaque à transthyrétine
Session : Amylose à transthyrétine : résultats à long terme et nouvelles thérapies (Résumés éclair)
Lundi 11 mai, 16h45 – 17h45 CEST, 10h45 – 11h45 EST
Auteur de la présentation : Arielle Abovich, États-Unis Efficacité constante du vutrisiran chez les deux sexes dans l’ATTR-CM, essai HELIOS-B – Analyse prédéfinie selon le sexe
Session : Amylose à transthyrétine : résultats à long terme et nouvelles thérapies (Résumés éclair)
Lundi 11 mai, 16h45 – 17h45 CEST, 10h45 – 11h45 EST
Auteur de la présentation : Josephine Mansell, Royaume-Uni Le fardeau des comorbidités dans l’amylose à transthyrétine avec cardiomyopathie : enseignements tirés de l’essai HELIOS-B
Session : Facteurs de risque et comorbidités (ePosters modérés 3)
Mardi 12 mai, de 08h30 à 09h15 CEST, de 2h30 à 3h15 EST
Auteur de la présentation : Sarah Birkhoelzer, Royaume-Uni INDICATIONS ET INFORMATIONS IMPORTANTES RELATIVES À LA SÉCURITÉ D’AMVUTTRA® (vutrisiran) Indications
Dans l’UE, AMVUTTRA® (vutrisiran) est indiqué pour le traitement : de l’amylose héréditaire à transthyrétine chez les patients adultes atteints de polyneuropathie de stade 1 ou 2 (hATTR-PN) ; de l’amylose à transthyrétine de type sauvage ou héréditaire chez les patients adultes atteints de cardiomyopathie (ATTR-CM). La disponibilité dans l’UE est soumise aux délais de remboursement locaux. Informations de sécurité importantes
Réduction des taux sériques de vitamine A et supplémentation recommandée
Le traitement par vutrisiran entraîne une diminution des taux sériques de vitamine A. Une supplémentation d’environ 2 500 à 3 000 UI de vitamine A par jour, sans dépasser cette dose, est recommandée pour les patients prenant du vutrisiran. Les patients doivent être orientés vers un ophtalmologiste s’ils développent des symptômes oculaires suggérant une carence en vitamine A (par ex. cécité nocturne). Effets indésirables
Les effets indésirables fréquemment rapportés avec le vutrisiran étaient des réactions au site d’injection et une augmentation des taux sanguins de phosphatase alcaline et d’alanine transaminase. Pour plus d’informations sur le vutrisiran, veuillez consulter le résumé complet des caractéristiques du produit . À propos d’AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) est un inhibiteur de la transthyrétine (TTR) qui permet une réduction rapide de la TTR à la source afin de traiter la cause sous-jacente de l’amylose à transthyrétine (ATTR). Dans une étude clinique, AMVUTTRA a rapidement réduit la TTR dès six semaines et a diminué les taux de TTR de 87 % après deux ans et demi de traitement. Il est approuvé dans le monde entier dans divers pays pour le traitement de la polyneuropathie associée à l’amylose héréditaire à transthyrétine (hATTR-PN) chez l’adulte et de la cardiomyopathie associée à l’amylose à transthyrétine de type sauvage ou héréditaire (ATTR-CM) chez l’adulte. Administré tous les trimestres par injection sous-cutanée, AMVUTTRA est le premier et le seul inhibiteur approuvé pour le traitement de l’ATTR-CM et de l’hATTR-PN. À propos de l’amylose à transthyrétine (ATTR)
L’amylose à transthyrétine (ATTR) est une maladie sous-diagnostiquée, à progression rapide, invalidante et mortelle, causée par des protéines transthyrétine (TTR) pathogènes qui s’accumulent sous forme de dépôts amyloïdes dans diverses parties du corps, notamment les nerfs, le cœur et le tractus gastro-intestinal. Les patients peuvent présenter une polyneuropathie, une cardiomyopathie, ou les deux manifestations de la maladie. Il existe deux formes différentes d’ATTR : l’ATTR héréditaire (hATTR), causée par une variante du gène TTR, et l’ATTR de type sauvage (wtATTR), qui survient sans variante du gène TTR. On estime que plus de 500 000 personnes dans le monde vivent avec l’ATTR. À propos de l’iARN
L’iARN (interférence ARN) est un processus cellulaire naturel de silençage génique qui représente aujourd’hui l’un des horizons les plus prometteurs et les plus avancés de la biologie et du développement de médicaments. Sa découverte a été annoncée comme « une avancée scientifique majeure qui se produit une fois tous les dix ans environ » et a été récompensée par le prix Nobel 2006 de physiologie ou médecine. En exploitant le processus biologique naturel de l’iARN se produisant dans nos cellules, une nouvelle classe de médicaments connue sous le nom de thérapeutique iARN est désormais une réalité. Les petits ARN interférents (ARNsi), les molécules qui médient l’iARN et composent la plateforme thérapeutique iARN d’Alnylam, fonctionnent en amont des médicaments actuels en inhibant puissamment l’ARN messager (ARNm), c’est-à-dire les précurseurs génétiques, qui codent pour les protéines pathogènes ou causant des maladies, empêchant ainsi leur fabrication. Il s’agit d’une approche révolutionnaire qui a le potentiel de transformer les soins aux patients atteints de maladies génétiques et autres. À propos d’Alnylam Pharmaceuticals
Alnylam (Nasdaq : ALNY) est une société biopharmaceutique mondiale de premier plan et le pionnier de la révolution de l’interférence ARN (RNAi). La société se concentre sur le développement de thérapies transformatrices ayant le potentiel de prévenir, d’arrêter ou d’inverser la maladie. Depuis plus de deux décennies, Alnylam fait progresser la science de l’ARNi, récompensée par un prix Nobel, en réalisant des avancées décisives et en mettant au point six médicaments approuvés. Alnylam commercialise des médicaments dans plus de 70 pays et dispose d’un pipeline solide en pleine expansion, tout en étant régulièrement reconnue comme un lieu de travail exceptionnel et une organisation socialement responsable. La société met en œuvre sa stratégie Alnylam 2030 afin d’accélérer l’innovation et d’amplifier son impact pour transformer la santé humaine. Déclarations prospectives d’Alnylam
Ce communiqué de presse contient des déclarations prospectives au sens de la section 27A du Securities Act de 1933 et de la section 21E du Securities Exchange Act de 1934. Toutes les déclarations autres que des faits historiques concernant les attentes, convictions, objectifs, plans ou perspectives d’Alnylam, y compris, sans s’y limiter, les déclarations concernant le potentiel d’AMVUTTRA à être utilisé comme traitement de première intention pour l’ATTR-CM ; et la capacité d’Alnylam à mettre en œuvre sa stratégie Alnylam 2030 visant à accélérer l’innovation et à amplifier son impact pour transformer la santé humaine, doivent être considérées comme des déclarations prospectives. Les résultats réels et les plans futurs peuvent différer matériellement de ceux indiqués dans ces déclarations prospectives en raison de divers risques, incertitudes et autres facteurs importants, y compris, mais sans s’y limiter, les risques et incertitudes liés à : la capacité d’Alnylam à mettre en œuvre avec succès sa stratégie « Alnylam 2030 » ; la capacité d’Alnylam à lancer, commercialiser et vendre avec succès ses produits approuvés à l’échelle mondiale, y compris l’AMVUTTRA ; la capacité d’Alnylam à découvrir et développer de nouveaux candidats-médicaments et de nouvelles approches d’administration, et à démontrer avec succès l’efficacité et la sécurité de ses candidats-produits ; les résultats précliniques et cliniques des produits candidats d’Alnylam ; les mesures ou avis des autorités réglementaires et la capacité d’Alnylam à obtenir et à conserver l’autorisation réglementaire pour ses produits candidats, ainsi qu’une tarification et un remboursement favorables ; les retards, interruptions ou défaillances dans la fabrication et la fourniture des produits commercialisés par Alnylam ou de ses produits candidats ; l’obtention, le maintien et la protection de la propriété intellectuelle ; la capacité d’Alnylam à gérer sa croissance et ses dépenses d’exploitation grâce à des investissements rigoureux dans ses activités ; la capacité d’Alnylam à maintenir des collaborations commerciales stratégiques ; la dépendance d’Alnylam vis-à-vis de tiers pour le développement et la commercialisation de certains produits ; l’issue des litiges et des enquêtes gouvernementales ; le risque de litiges et d’enquêtes gouvernementales futurs ; et les dépenses imprévues ; ainsi que les risques et incertitudes décrits plus en détail dans la section « Facteurs de risque » du rapport annuel 2025 d’Alnylam sur formulaire 10-K déposé auprès de la Securities and Exchange Commission (SEC), qui peuvent être mis à jour de temps à autre dans les rapports trimestriels ultérieurs d’Alnylam sur formulaire 10-Q, et dans d’autres documents déposés par Alnylam auprès de la SEC. En outre, toute déclaration prospective représente les opinions d’Alnylam uniquement à ce jour et ne doit pas être considérée comme représentant ses opinions à une date ultérieure. Alnylam décline explicitement toute obligation, sauf dans la mesure requise par la loi, de mettre à jour les déclarations prospectives. Le texte du communiqué issu d’une traduction ne doit d’aucune manière être considéré comme officiel. La seule version du communiqué qui fasse foi est celle du communiqué dans sa langue d’origine. La traduction devra toujours être confrontée au texte source, qui fera jurisprudence. Consultez la version source sur businesswire.com : https://www.businesswire.com/news/home/20260504732378/fr/ Alnylam Pharmaceuticals, Inc.
Christine Akinc
(Investisseurs et médias)
+1-617-682-4340 Josh Brodsky
(Investisseurs)
+1-617-551-8276 Original: Alnylam présentera de nouvelles analyses lors du congrès Heart Failure 2026, soulignant le profil clinique constant du vutrisiran dans l’ATTR-CM

When Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in BiotechApril 20, 2026 9:00 AM
InvestorsHub NewsWireWhen Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in BiotechBioMedWire Editorial Coverage: Biotechnology is undergoing a quiet but profound transformation, one that is reshaping how investors understand value in a sector historically defined by long timelines and uncertain outcomes. As drug candidates advance closer to commercialization, scientific progress is no longer viewed solely as research expenditure but increasingly as a measurable financial asset. This shift is being reinforced by fair-value accounting under U.S. GAAP, which allows life sciences companies to reflect clinical progress, probability of success and commercialization timing as measurable balance sheet value. Leading companies, such as Oncotelic Therapeutics Inc. (OTCQB: OTLC) (Profile), are keenly aware of this evolution and are leveraging their expertise as pioneers in this space. Through its diversified pipeline and strategic holdings, including a 45% ownership stake in GMP Bio, which was recently measured at more than $1 billion enterprise value, the company exemplifies how advancing science can directly influence financial positioning. As the biotech sector increasingly aligns valuation with progress rather than revenue alone, Oncotelic provides a case study in how innovation is becoming a recognized asset class. Oncotelic joins an elite group of other key companies focused on advanced biotech platforms that target disease at the genetic or molecular level, including Sarepta Therapeutics Inc. (NASDAQ: SRPT), Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY), Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) and Denali Therapeutics Inc. (NASDAQ: DNLI).The biotechnology sector is evolving from a model centered on long-term R&D investment into one where scientific advancement can be reflected as measurable financial value.As drug candidates advance through clinical development, their value increases significantly due to rising probabilities of success and proximity to commercialization.The rise of fair-value accounting in biotechnology is enabling companies to provide more transparent and timely representations of asset value.Institutional investors are increasingly recognizing clinical-stage biotech assets as investable opportunities, even in the absence of current revenue.Oncotelic Therapeutics operates directly within this convergence, combining oncology-focused drug development with AI-enabled platforms and strategic manufacturing exposure.Click here to view the custom infographic of the Oncotelic Therapeutics editorial.Scientific Progress Is Becoming Financial ValueThe biotechnology sector is evolving from a model centered on long-term R&D investment into one where scientific advancement can be reflected as measurable financial value. Historically, investors relied heavily on discounted cash-flow projections tied to eventual commercialization, often leaving a gap between innovation and valuation. However, the adoption of fair-value frameworks under U.S. GAAP is beginning to bridge that gap by allowing companies to reassess asset value as clinical milestones are achieved.Under Financial Accounting Standards Board guidance, particularly ASC 820, companies can measure the fair value of assets, including illiquid or early-stage investments, based on observable and unobservable inputs. This includes Level 3 assets, which are commonly used in biotech to value pipeline-stage programs and strategic investments. These frameworks allow companies to incorporate scientific progress, regulatory advancement and probability of success into financial reporting.Advisory firms such as Deloitte and PwC note that in life sciences, valuation can reflects forward-looking indicators such as clinical progress and expected commercialization potential, as GAAP fair-value frameworks require assets to be measured based on market participant assumptions about future economic benefits. This approach aligns financial reporting more closely with operational reality, particularly in sectors where revenue may lag innovation by years.This shift is particularly relevant for Oncotelic Therapeutics, which operates in a space where pipeline advancement is a primary driver of value. By aligning its financial reporting with the progression of its therapeutic programs and strategic investments, the company reflects the broader industry movement toward recognizing science itself as an asset.Pipeline Proximity Drives Valuation InflectionAs drug candidates advance through clinical development, their value increases significantly due to rising probabilities of success and proximity to commercialization. This phenomenon — often referred to as valuation inflection — is well documented across the biotech industry. Late-stage assets, particularly those approaching regulatory approval, tend to command a disproportionate share of enterprise value.According to McKinsey & Company, value creation in biopharma is heavily concentrated in late-stage assets, with companies generating the majority of returns as products progress toward approval and commercialization, reflecting reduced risk and clearer revenue visibility. Additional industry analysis shows that assets approaching commercialization drive significant increases in company valuation due to higher probability of success and expected market entry.This dynamic highlights a critical change in how investors assess biotech companies. Rather than focusing solely on current revenue or earnings, market participants increasingly evaluate the maturity and trajectory of pipeline assets. As a result, companies with strong late-stage programs can experience significant valuation increases even before generating commercial sales.For Oncotelic Therapeutics, this trend is particularly relevant, given the company's focus on advancing oncology therapeutics and its strategic positioning within high-value development pathways. As its programs progress and move closer to commercialization, the company's valuation profile reflects the broader industry shift toward pipeline-driven value creation.Fair-Value Accounting Gains Ground in BiotechThe rise of fair-value accounting in biotechnology is enabling companies to provide more transparent and timely representations of asset value. Under GAAP, Level 3 valuations are used to measure assets without observable market prices, relying on models that incorporate forward-looking assumptions such as expected cash flows and probability-weighted outcomes.The ASC 820 framework defines fair value as the exit price that would be received to sell an asset in an orderly transaction between market participants at the measurement date. It provides a structured hierarchy for the subsequent measurement of complex, illiquid assets using market-participant assumptions and the best available information, aligning financial reporting with a current, market-based view of an asset's value.Oncotelic Therapeutics exemplifies this approach through the remeasurement of its stake in GMP Bio, which reflects an approximate $1 billion-plus enterprise value. This demonstrates how clinical and strategic progress can translate directly into balance sheet strength, reinforcing the concept of science as a financial asset.Institutional Capital Embraces Prerevenue BiotechInstitutional investors are increasingly recognizing clinical-stage biotech assets as investable opportunities, even in the absence of current revenue. This shift reflects a growing confidence in structured valuation methodologies that incorporate scientific progress, rather than relying solely on traditional financial metrics.Many biotechnology companies are valued primarily on the strength of their pipelines rather than their existing revenue streams, reflecting the long development timelines and lack of early-stage earnings typical of the sector. Instead, valuation is closely tied to scientific progress, with factors such as clinical development stage, probability of success and regulatory milestones serving as key drivers of equity value. This approach underscores that value creation in biotech often occurs well before commercialization, as investors increasingly rely on milestone-based and probability-adjusted models to assess future potential.Within this context, Oncotelic Therapeutics is positioned to benefit from growing institutional interest in pipeline-driven valuation. Its diversified portfolio and strategic investments align with the types of assets that are increasingly attracting capital in the biotech sector.AI, GMP and Commercial Readiness ConvergeAdvancements in AI-driven drug development and GMP-compliant manufacturing are accelerating timelines and improving scalability across the biotech industry. These technologies enable more efficient clinical development, better data analysis and streamlined production processes, reducing both time and cost to market.AI is increasingly being used to identify drug targets, optimize trial design and analyze complex datasets, improving the probability of success in clinical programs. At the same time, GMP-compliant manufacturing platforms are enabling more reliable and scalable production, which is critical as therapies move toward commercialization.Research indicates that data-driven approaches are becoming central to biopharma value creation, with AI and advanced analytics playing a key role in accelerating development timelines. These advancements enhance the reliability of valuation models by reducing uncertainty and improving execution. This convergence of AI, manufacturing and clinical development is reshaping how value is created and measured in biotech. Companies that can integrate these capabilities are better positioned to advance therapies efficiently while maintaining regulatory compliance.Oncotelic Therapeutics operates directly within this convergence, combining oncology-focused drug development with AI-enabled platforms and strategic manufacturing exposure. This integrated approach supports both scientific advancement and financial valuation, positioning the company within one of the most significant growth trends in the biotechnology sector.The biotechnology sector is entering a new phase in which scientific progress is no longer viewed solely as a cost center but as a measurable financial asset. As fair-value accounting, pipeline maturity and AI-driven development reshape the industry, the traditional gap between innovation and valuation is narrowing.Companies such as Oncotelic Therapeutics illustrate how this transformation is unfolding in real time, demonstrating that clinical advancement can materially strengthen financial positioning even before revenue generation begins. As investors increasingly focus on pipeline-driven value, the ability to translate science into measurable economic impact is becoming one of the defining characteristics of modern biotech.From Research Spend to Revenue-Ready AssetsA new wave of biotechnology innovation is increasingly blurring the line between scientific progress and financial value creation. As drug candidates targeting disease at the genetic and molecular level advance through clinical development in 2026, progress is no longer viewed solely as research expenditure but as a measurable and evolving asset. Recent updates across the sector highlight how advancements in RNA interference, gene therapy and targeted delivery platforms are not only improving clinical outcomes but also enhancing the underlying value of biotech pipelines.Sarepta Therapeutics Inc. (NASDAQ: SRPT) announced approval of its clinical trial application for SRP-1005, its investigational treatment for Huntington's disease. Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA ("siRNA") therapeutic. INSIGHTT is a phase 1, multicenter, dose-escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants.Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) reported new clinical and real-world data from its cardiovascular ("CV") portfolio. The data was presented at the American College of Cardiology's Annual Scientific Session and Expo ("ACC.26"). The new data continues to support the benefits of vutrisiran in transthyretin amyloid cardiomyopathy (ATTR-CM), as well as the potential of zilebesiran for hypertension, underscoring the versatility of RNAi therapeutics across cardiovascular diseases.Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) is advancing a suite of messenger RNA ("mRNA")–based therapies. The company's Q4 update highlights progress in both rare diseases and vaccine platforms, including its lead candidate, ARCT-032, an inhaled mRNA therapy for cystic fibrosis, which has demonstrated favorable safety and tolerability and is advancing into a phase 2 clinical study expected to begin in 2026. In addition, the update noted advancement with its ARCT-810 program for ornithine transcarbamylase ("OTC") deficiency, a rare genetic disorder, with ongoing regulatory interactions aimed at supporting future pivotal studies.Denali Therapeutics Inc. (NASDAQ: DNLI) is making significant progress in 2026 with its TransportVehicle(TM) platform. The platform is designed to enable the delivery of biologics across the blood-brain barrier, one of the most persistent challenges in treating neurological diseases. The company has outlined 2026 as a pivotal year, with plans to deliver its first TransportVehicle-enabled medicine to patients and advance two TV-enabled programs into clinical studies for Alzheimer's disease and a program for Pompe disease.These latest developments underscore a broader industry transition from discovery to execution, where scientific innovation is increasingly quantified in financial terms. As genetic and molecular therapies move closer to commercialization, the ability to demonstrate clinical efficacy, scalability and delivery precision is translating into tangible asset value within company pipelines. This convergence of science and finance is reshaping how biotechnology is evaluated, positioning advanced therapeutic platforms not just as research initiatives, but as structured, value-generating assets.For further information about Oncotelic Therapeutics Inc., visit the Oncotelic Therapeutics profile.About BioMedWireBioMedWire ("BMW") is a specialized communications platform with a focus on the latest developments in the Biotechnology (BioTech), Biomedical Sciences (BioMed) and Life Sciences sectors. 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Original: When Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in Biotech

New Data Presented at ACC.26 Further Support Benefits of Vutrisiran in ATTR-CM and Potential of Zilebesiran for Hypertension ManagementMarch 30, 2026 1:00 PM
Business Wire
- Vutrisiran, which Silences Transthyretin at the Source, Shown to Improve Health-Related Quality-of-Life in Those with ATTR-CM, with Treatment Effects Comparable to Patients More than Ten Years Younger –


- Findings Reinforce Consistent Benefits of Vutrisiran Across ATTR-CM Disease Spectrum, Including Patients with Most Advanced Disease and Diastolic Dysfunction -


- Real-World Data Demonstrate High Adherence and Persistence of Quarterly HCP-Administered Dosing with Vutrisiran –


- Pooled Phase 2 Safety Data Reinforce Encouraging Safety Profile for Zilebesiran Across a Broad Population of Patients with Hypertension -


Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new clinical and real-world data from its cardiovascular (CV) portfolio presented at the American College of Cardiology’s Annual Scientific Session and Expo (ACC.26), reinforcing the potential of RNAi to deliver fundamentally differentiated, effective, and durable impact for patients living with cardiovascular disease (CVD).


New data continue to support the use of vutrisiran – the first and only transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source – as a first-line treatment for patients with the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). The HELIOS-B analyses demonstrate that vutrisiran delivered meaningful improvements in how patients with ATTR-CM feel and function, with benefits sustained across disease severity, reinforcing how TTR silencing at its source translates into durable clinical and health-related quality-of-life (QoL) benefits for patients.


Alnylam also presented pooled Phase 2 safety data for zilebesiran, an investigational RNAi therapeutic being evaluated for cardiovascular risk reduction in patients with hypertension, with the potential to provide continuous control of blood pressure. The results support continued evaluation of zilebesiran in patients with hypertension and high CV risk in ZENITH, a global Phase 3 Cardiovascular Outcomes Trial (CVOT) that was initiated in September 2025.


Vutrisiran Analyses:

New analyses expand the totality of HELIOS-B evidence demonstrating improvements in patients’ health-related QoL, consistent efficacy on CV outcomes across a range of patient subgroups, including those with advanced disease and diastolic dysfunction, and real-world data showing high treatment adherence with four healthcare professional (HCP)-administered doses per year. In addition to clinical studies, vutrisiran has more than 13,000 patient-years of experience for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.


HELIOS-B Analysis of QoL as Measured by KCCQ

This analysis evaluated placebo-corrected mean score changes of vutrisiran-treated patients across the 23-item Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS) from baseline to 30 Months. Compared to placebo, vutrisiran demonstrated improvements in nearly all components, most notably in physical limitations and QoL. Moreover, the magnitude of treatment effect in age-adjusted KCCQ-OSS favoring vutrisiran was comparable to the difference observed in patients 11 years (95% CI: 3-20 years) apart in age. These findings were simultaneously published in the European Journal of Heart Failure.


HELIOS-B Post Hoc Analysis of Patients with Advanced Disease During the Double-Blind (DB) Period

The analysis assessed the risk of patients developing advanced disease as defined by transitioning to New York Heart Association (NYHA) class III and National Amyloidosis Center (NAC) stage 3, or NYHA class IV, with vutrisiran versus placebo during the HELIOS-B DB period. In the overall population, fewer patients receiving vutrisiran developed advanced disease compared to patients receiving placebo (8.0% vs. 10.7%, respectively). The analysis also assessed the effect of vutrisiran on outcomes in patients with ATTR-CM who developed advanced disease. In these patients, treatment with vutrisiran reduced the risk of the composite of all-cause mortality (ACM) and recurrent CV events during the DB period by 40% in the overall population and 46% in the monotherapy population, versus placebo. Treatment with vutrisiran also reduced the risk of ACM during the DB period plus up to six months of open-label extension by 56% in the overall population and 77% in the monotherapy population, versus placebo. Vutrisiran had a favorable safety profile in patients with advanced disease, with a similar or fewer proportion of patients experiencing adverse events, compared to placebo.


“These new data present further evidence of the meaningful impact vutrisiran offers patients with ATTR-CM and provide deeper insight into its benefits for patients with more advanced disease,” said Ronald Witteles, M.D., HELIOS-B Investigator, Professor of Medicine at Stanford University School of Medicine and Co-Director of the Stanford Amyloid Center. “Vutrisiran not only meaningfully reduced the risk of progression to advanced disease, but among patients who did progress, it demonstrated compelling improvements in both cardiovascular outcomes and survival. These findings provide important evidence to help inform treatment decisions for the highest-risk ATTR-CM patients.”


Real-World Vutrisiran Adherence and Persistence Study

A retrospective cohort study of real-world data in patients with amyloidosis indicated high adherence and persistence to vutrisiran treatment, with most patients still receiving treatment after 12 months. Patients were followed up for a mean of 613.8 days. Over the treatment period, 93.8% were adherent to vutrisiran, defined as PDC (proportion of days covered) ≥0.8.


HELIOS-B Post Hoc Analysis of Diastolic Dysfunction

Diastolic dysfunction is prognostic of poor outcomes in patients with ATTR-CM. To evaluate the effect of vutrisiran in patients across a range of diastolic dysfunction grades (DDGs) at baseline, a post hoc analysis of HELIOS-B assessed outcomes at Month 30 in patients who had evaluable DDG at baseline. Higher grade DDG at baseline corresponded with adverse outcomes in ATTR-CM. Vutrisiran was associated with a lower risk of worsening DDG, with a greater proportion of patients with baseline DDG III showing stable or improved NYHA class from baseline to Month 30 in the overall and monotherapy populations (70.8% and 70.8%, respectively), compared to patients receiving placebo (55.6% and 53.2%, respectively). Vutrisiran reduced the risk of ACM and CV events during the DB period, irrespective of baseline DDG.


Zilebesiran Analysis

Building on the efficacy and safety results of the KARDIA Phase 2 program, these data reinforce the rationale for evaluating zilebesiran in patients with hypertension and established or at high risk of CVD, despite the use of at least two or more antihypertensives, in ZENITH. By targeting liver-expressed angiotensinogen (AGT), the most upstream precursor in the renin-angiotensin-aldosterone system (RAAS), zilebesiran has the potential to provide continuous control of blood pressure with biannual dosing, offering a potentially differentiated approach to hypertension and CV risk management in patients with the highest unmet need.


Pooled Safety Analysis from Phase 2 Studies

A comprehensive analysis of safety across the Phase 2 KARDIA program demonstrated an acceptable safety profile for zilebesiran, both as monotherapy and in combination with standard-of-care antihypertensives, across patients with mild-to-moderate hypertension, those at high CV risk, or with lower eGFR at baseline. In the analysis, clinically relevant safety events, such as hypotension, hyperkalemia, and eGFR decline, were low across the patient populations, including those receiving zilebesiran in combination with an angiotensin-converting enzyme inhibitor (ACEi) or an angiotensin II receptor blocker (ARB). As previously reported, the majority of these events were transient and resolved without intervention.


To view Alnylam’s ACC.26 presentations, please visit Capella.


Indications and Important Safety Information


Indications Approved by the U.S. FDA

AMVUTTRA® (vutrisiran) is indicated for the treatment of the:



cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.



polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.



Important Safety Information


Reduced Serum Vitamin A Levels and Recommended Supplementation


AMVUTTRA treatment leads to a decrease in serum vitamin A levels.


Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.


Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).


Adverse Reactions

In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).


In a study of patients with ATTR-CM, no new safety issues were identified.


For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025)


About AMVUTTRA® (vutrisiran)

AMVUTTRA® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN.


About Transthyretin Amyloidosis (ATTR)

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating, and fatal disease caused by pathogenic transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart, and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant. It is estimated that more than 500,000 people worldwide live with ATTR.


About Zilebesiran

Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for cardiovascular (CV) risk reduction in patients with hypertension. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the renin-angiotensin-aldosterone system (RAAS), which plays a role in blood pressure (BP) regulation and impacts CV and renal health. Clinical trial results have shown the potential for zilebesiran to provide continuous control of BP with biannual dosing in a broad population of patients with hypertension. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA, or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche.


About Cardiovascular Disease and Hypertension

Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. Hypertension is the primary cause of and number one modifiable risk factor for CVD. An estimated one in three adults worldwide have hypertension, and despite wide availability of antihypertensives, up to 80% of all patients, and up to one-third of treated patients, do not reach and maintain blood pressure (BP) targets. Even when BP appears well-managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period, and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage.


About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.


About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt, or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030 strategy to accelerate innovation and scale impact to transform human health. For more information, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.


Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding the potential efficacy, safety or treatment effect of AMVUTTRA, zilebesiran or any of Alnylam’s other products or product candidates; the potential of RNAi to deliver fundamentally differentiated, effective, and durable impact for patients living with cardiovascular disease; the potential of AMVUTTRA to be a first line treatment for ATTR-CM, to provide treatment effects across the ATTR-CM disease spectrum, and to offer meaningful impact to patients with ATTR-CM; the potential for TTR silencing at the source to disease to translate into durable clinical benefit and health related quality of life benefits for patients; the potential of zilebesiran to provide continuous control of blood pressure with biannual dosing and to offer a potentially differentiated approach to hypertension and CV risk management in patients with highest unmet need; and Alnylam’s ability to achieve the goals in its “Alnylam 2030” strategy, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam 2030” strategy; Alnylam’s ability to successfully launch, market and sell Alnylam’s approved products globally, including AMVUTTRA and zilebesiran; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam’s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, and Regeneron; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260330643261/en/
Alnylam Pharmaceuticals, Inc.



Christine Akinc

(Investors and Media)

+1-617-682-4340
Josh Brodsky

(Investors)

+1-617-551-8276


Original: New Data Presented at ACC.26 Further Support Benefits of Vutrisiran in ATTR-CM and Potential of Zilebesiran for Hypertension Management

Alnylam teams up with Viz.ai and American Heart Association to advance ATTR-CM careMarch 24, 2026 8:36 AM
IH Market News
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said it has entered into strategic collaborations with Viz.ai and the American Heart Association to improve the detection and care management of patients with transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM).Under the agreement with Viz.ai, the companies will develop an AI-driven care pathway for ATTR-CM that integrates the FDA-cleared echocardiography algorithm Us2.ai with electronic health record systems. The initiative will also include the AWARE study, a prospective multi-system implementation trial designed to assess how AI-based screening can be incorporated into routine clinical workflows. The program is expected to begin later this year across five pilot healthcare systems.In a separate effort, Alnylam will support a three-year initiative led by the American Heart Association that will bring together a network of ten multidisciplinary health systems through a national learning collaborative. The program is intended to identify gaps in patient care, exchange best practices and develop scalable approaches for diagnosing and treating ATTR-CM.ATTR-CM is a progressive form of heart failure that frequently goes undiagnosed. According to the company, the condition affects around 350,000 people worldwide, with about 80% of cases currently unidentified.“Earlier recognition can fundamentally change a patient’s course of disease,” said Sameer Bansilal, Vice President, Global TTR Medical Lead at Alnylam Pharmaceuticals.Tim Showalter, Chief Medical Officer at Viz.ai, said the collaboration is focused on “closing the gap between the first clinical signal and meaningful clinical action.”Alnylam also hosted an investor webinar focused on its TTR programs to discuss the initiatives. The company develops RNA interference-based therapies and currently has six approved medicines available in more than 70 countries.Alnylam Pharmaceuticals stock price

Original: Alnylam teams up with Viz.ai and American Heart Association to advance ATTR-CM care

https://www.businesswire.com/news/home/20241117936734/en/Alnylam-Announces-Interim-Phase-1-Data-of-Nucresiran-ALN-TTRsc04-Showing-Rapid-Knockdown-of-TTR-that-is-Sustained-at-Six-Months-Following-a-Single-Dose

The next LLY will be the RNAI king!

$ALNY

Just In: $ALNY Can Either of These Little-Known Biotechs Find New Life Fighting Coronavirus?

In 1962, the Nobel Prize was awarded to Francis Crick and James Watson for their discoveries related to deoxyribonucleic acid (DNA), the genetic code that provides instructions to cells. Messenger ribonucleic acid (mRNA), the intermediate step between DNA and proteins, can be bound and degr...

Got this from ALNY - Can Either of These Little-Known Biotechs Find New Life Fighting Coronavirus?

Vir and Alnylam Identify RNAi Therapeutic Development Candidate, VIR-2703 (ALN-COV), Targeting SARS-CoV-2 for the Treatment of COVID-19

https://investors.alnylam.com/press-release?id=24796

May 04, 2020

- Highly Potent and Broadly Cross-Reactive RNAi Therapeutic Development Candidate Selected Only Three Months After Program Initiation -

- Companies Expect to Start Human Clinical Trials at or Around Year-End 2020 –

$ALNY short squeeze article Alnylam Pharmaceuticals Inc Nasdaq Alny Short Squeeze

News: $ALNY Alnylam to Webcast Presentations at Upcoming December Investor Conferences

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences: Evercore ISI 2 nd Annual HealthCONx Conference on Wednesday, December 4, 2019 at 1:15 pm ET at the Four Se...

Got this from ALNY - Alnylam to Webcast Presentations at Upcoming December Investor Conferences

Stock is going to the moon

News: $ALNY Alnylam Announces New and Enhanced Framework for Value-Based Agreements to Accelerate Patient and Provider Access to GIVLAARI(TM) (givosiran)

− Framework Ties Payment for GIVLAARI to its Delivery of Patient Outcomes in the Real-World Setting - − Includes New Approach Designed for Ultra-Rare Diseases that Gives Participating Payers Greater Financial Certainty if Disease Prevalence is Higher Than Anticipated ...

Read the whole news ALNY - Alnylam Announces New and Enhanced Framework for Value-Based Agreements to Accelerate Patient and Provider Access to GIVLAARI(TM) (givosiran)

News: $ALNY Alnylam Announces Approval of GIVLAARI(TM) (givosiran) by the U.S. Food and Drug Administration (FDA)

− GIVLAARI Approved for the Treatment of Adults with Acute Hepatic Porphyria (AHP) Based on ENVISION Phase 3 Study Results Showing Significant Reduction in the Rate of Porphyria Attacks in Patients with AHP – − FDA Approval Received in Less Than Four Months after N...

Read the whole news ALNY - Alnylam Announces Approval of GIVLAARI(TM) (givosiran) by the U.S. Food and Drug Administration (FDA)

News: $ALNY Alnylam Initiates ILLUMINATE-C Phase 3 Study of Lumasiran for the Treatment of Advanced Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study

− Initial Results From ILLUMINATE-C Expected in Late 2020 – − In Phase 2 OLE, Lumasiran Treatment Resulted in 76 percent Mean Maximal Reduction in Urinary Oxalate Relative to Phase 1/2 Baseline – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leadi...

Got this from ALNY - Alnylam Initiates ILLUMINATE-C Phase 3 Study of Lumasiran for the Treatment of Advanced Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study

Phase 3 data, due in the 3rd quarter of 2019.

It's going a bit crazy in the after hours market.

Agree.

It will be interesting to see the following weeks prices.

Who would have predicted that ALNY would get to this price without significant revenues yet?

IMO of course.

Recent Statement of Changes in Beneficial Ownership.

On November 15 2017 John Marangamore, Chief Executive Officer, in my opinion Acquired 74000 shares at $31.39 and Disposed 74000 at $126.864 for a computed amount of $9,387,951.301


W O R K S H E E T

22460 * $125.544 = 2819718.24
28399 * $126.603 = 3595398.597
07120 * $127.540 = 0908084.80
10571 * $128.484 = 1358204.364
04850 * $129.570 = 0628414.5
00600 * $130.218 = 0078130.8
------------------------------------
74000 * $126.864 = 9387951.301

Compare these computed numbers to other numbers in the most recent 10-q and draw your own conclusions.

IMO of course.

IMO of course.

Summary of recent Statement of Changes in Beneficial Ownership.

On September 20 2017 Barry E Greene, President, in my opinion Acquired 76815 shares at $31.39 and Disposed 76815 at $100 for a computed amount of $5,270,277.15

On October 2 2017 Barry E Greene, President, in my opinion Acquired 85316 shares at $21.35 and Disposed 85316 at $125 for a computed amount of $8,843,003.40

Barry E Greene, President has thus in my opinion realized a computed total gain of $14,113,280.55 from the option exercises noted above.

Compare these computed numbers to other numbers in the most recent 10-q and draw your own conclusions.

IMO of course.

About RNAi

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding protein synthesis in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, with the goal of preventing disease-causing proteins from being made.

$ALNY Alnylam Pharmaceuticals Inc. https://www.barchart.com/story/stocks/quotes/OTIC/news/3925902/best-relative-performance-in-the-biotechnology-industry-detected-in-shares-of-coherus-bioscien-chrs-otic-tsro-imgn-alny

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has initiated a Phase 2 clinical study with cemdisiran (formerly known as ALN-CC5), a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. The trial is being conducted in patients with atypical hemolytic-uremic syndrome (aHUS) and will evaluate the effect of C5 knockdown on hematologic response and renal function, as well as safety and tolerability. The Company expects to report initial clinical proof of concept data from this trial in late 2018.

I've been following developments in RNAI for years. It's great to finally see progress in this powerful tech.

https://seekingalpha.com/article/4109199-alnylam-sanofi-achieve-rnai-first

In addition to new data on the Company’s ESC+ platform, Alnylam scientists and collaborators presented additional pre-clinical findings showing continued leadership in RNAi technologies and delivery. First, Alnylam scientists presented further advances toward optimizing the Company’s GalNAc-siRNA conjugate platform. These included studies to further improve the mechanistic understanding of conjugate duration of activity as well as the development of advanced ESC designs with significantly improved metabolic stability and in vivo efficacy. Further, new data on Alnylam’s Reversir™ platform were presented. Specifically, optimizations were implemented that enable rapid reversal of siRNA-mediated mRNA silencing, providing the means to fine-tune the pharmacology of GalNAc-siRNA conjugates. Finally, pre-clinical data demonstrating extra-hepatic siRNA delivery, involving Centyrins, a novel class of highly stable FN3 domain proteins, were also presented as part of a research collaboration with Janssen Research & Development, LLC. Centyrin-siRNA conjugates showed excellent cross-tumor penetration and mediated robust target knockdown in a mouse xenograft tumor model. Also, the generalizability of this approach was demonstrated in vitro using Centyrins for a number of different receptors and gene targets.

“At Alnylam, we’ve been successful in advancing our ESC GalNAc-siRNA conjugate platform with many potent and generally well tolerated investigational RNAi therapeutics in clinical development, including in Phase 3 studies. Nevertheless, we continue to strive to even further optimize our RNAi therapeutics platform to achieve improved target specificity and an even greater therapeutic index. Accordingly, we were pleased to share these new pre-clinical results at this year's OTS meeting, highlighting our ESC+ GalNAc conjugate platform,” said Kevin Fitzgerald, Ph.D., Senior Vice President, Research at Alnylam. “Our goal is to employ the ESC+ chemistry in all future development candidates, beginning with ALN-AAT02, which we plan to advance into clinical development in 2018. We believe that by implementing platform improvements that preemptively address potential off-target effects we can further enhance the therapeutic index of our investigational RNAi therapeutics for all future programs in our pipeline.”

ESC+ GalNAc conjugates utilize advanced design features to further improve specificity, including a glycol nucleic acid (GNA) modification in the antisense seed region of the siRNA, while maintaining potency and durability. The data presented at OTS indicated that incorporation of GNA destabilizes seed-driven pairing with partially complementary transcripts, thus greatly reducing potential off-target effects while maintaining on-target pairing and activity. Additionally, it was shown that GNA modifications confer enhanced specificity and a greater-than 6-fold improvement in therapeutic index as observed in pre-clinical studies in rodents. The ESC+ design is now being applied to all of Alnylam’s pre-clinical programs and has shown successful translation of potency from rodents to non-human primates. Alnylam intends to employ its ESC+ siRNA-conjugate platform in all future development programs.

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, today announced that the Company presented new pre-clinical data highlighting its next generation “Enhanced Stabilization Chemistry Plus” (ESC+) GalNAc-siRNA conjugate platform at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held September 24 – 27, 2017 in Bordeaux, France.

Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) shares jumped more than 40% on Wednesday after the pharma company, together with Sanofi Genzyme, the specialty care global business unit of Sanofi, announced that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic designed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint and all secondary endpoints. "We are very proud to report the first ever positive Phase 3 results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines. This moment is the culmination of a 15-year journey of tireless work by countless contributors who have overcome enormous scientific and business challenges to make RNAi therapeutics a reality," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.

I am a finance graduate and have always been interested in trading but have never truly started researching until now. after looking at what has happened with this company and its stock price. such a spike would potentially lead to just as dramatic a drop in price, thus giving way to a great opportunity to short which you have stated correct?

FANTASTIC SHORT OPP TODAY...

$10 BILLION UNPROFITABLE BUBBLE FRAUD WHICH BURNING $120+ MILLION A QUARTER AN HAVE $200 MILLION DEBT = BRUTAL PONZI SCEHEME FRAUD ONGOING HERE TO ATTRACT NEWBIES SO THE CRIMINAL BANKSTERS AND BOARD MEMEBRS CAN UNLOAD THEIR TRASH = NEXT MEGA SCAM LIKE VALEANT VRX = FAIR VALUE $10 AT BEST

https://seekingalpha.com/article/4101274-alnylam-q3-catalysts-play

https://seekingalpha.com/article/4086761-alnylam-sanofi-produce-monster-alternative-hemophilia-market

ALNY buy option 71.49

buy 16 JUN 17 75 call for 2.40









normal chart




log chart

Back over 40/share by Tuesday.

Anyone ready?!

Liking it putting in a new high since earlier. Keep rolling on up ALNY.

Back to over 38 we go today alone.

Weeeee

So far hod 36.07, keep it rolling :)

Picked up a starter in pm.

After hours news prompting investigation... Ffs,
I'm flipping hard. GAP UP in am???

Buyers stepping up now $35.90 after the two hour short pullback. If they cover tomorrow....easy $40+

Shorts are in control right now $36.70. Looks like it is heading for a new LOD unless some covering takes place before EOD.