US Market News
1月前
Acadia Pharmaceuticals Announces Planned Year-End Retirement of Elizabeth H.Z. Thompson, Ph.D., Head of Research and Development, Following a Distinguished CareerApril 30, 2026 4:05 PM
Business Wire
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the planned retirement of Elizabeth H.Z. Thompson, Ph.D., Head of Research and Development. Dr. Thompson has decided to retire for personal reasons and informed Acadia of her plans. She will continue in her role as Head of Research and Development until a successor is appointed. After her retirement, Acadia plans to retain Dr. Thompson as a consultant through at least the end of 2026 to ensure scientific and leadership continuity through the planned readout of the Phase 2 clinical study of remlifanserin in Alzheimer’s disease psychosis (ADP) and into early Phase 3 clinical study execution.
Acadia has initiated a search for a new Head of Research and Development and is committed to identifying an experienced R&D leader who will build on the strong scientific foundation already in place. The Company continues to advance its clinical development programs as planned. All ongoing clinical trials, including the Phase 2 studies of remlifanserin in ADP and Lewy Body Dementia Psychosis (LBDP), continue to recruit and remain blinded, and the Company, including Dr. Thompson, does not yet know study outcomes.
“Liz’s leadership has been instrumental in strengthening Acadia’s research and development organization, both scientifically and operationally, and in advancing a clear, disciplined R&D strategy across our pipeline,” said Catherine Owen Adams, Chief Executive Officer of Acadia. “She has been a strong advocate for the patients we serve and at every step has ensured thoughtful execution and prioritization of our programs in neurological and rare diseases. Just as importantly, Liz fostered a culture of clarity, accountability, and engagement across R&D. We are grateful for her continued commitment to Acadia during the transition period and for her willingness to remain engaged as a consultant, providing continuity of scientific leadership and institutional perspective in support of our mission and, most importantly, the patients we serve.”
“Leading Acadia’s research and development organization has been a defining chapter in my career and one in which I take a great deal of pride,” said Dr. Thompson. “We started from a great foundation, and have been able to build and strengthen the Acadia R&D team as well as make good progress in shaping and advancing a curated pipeline. I am particularly pleased with how we’ve been able to strengthen the remlifanserin program, especially by expanding it into Lewy Body Dementia Psychosis. I remain excited by the potential for remlifanserin and confident in the disciplined development strategy the Acadia team is executing. Beyond remlifanserin, our pipeline contains multiple programs that could represent truly meaningful options for patients living with neurological and rare diseases. While I have decided to retire for personal reasons, I am committed to continuing to lead the organization until the appointment of a successor and to supporting Acadia through the transition and the remainder of the year.”
Dr. Thompson joined Acadia in 2024 serving as Head of Research and Development. During her tenure, she helped strengthen the Company’s scientific and clinical capabilities, advance multiple development programs, and foster a culture of collaboration across research, clinical development and regulatory teams.
About Acadia Pharmaceuticals
Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “expects,” “anticipates,” “continues,” “intends,” “planned,” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) Dr. Thompson’s planned retirement, and her continued service in her role through a transitional period and as a consultant with the Company, (ii) our ability to recruit and timely hire a new Head of Research and Development, and (iii) the advancement of the Company’s clinical development programs, including the timely achievement of anticipated clinical milestones. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to Dr. Thompson’s continued services to the Company, future executive recruitment and the advancement of clinical development programs. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2025 filed with the Securities and Exchange Commission on February 26, 2026, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260430350268/en/
Investor Contact:
Acadia Pharmaceuticals Inc.
Al Kildani
(858) 261-2872
ir@acadia-pharm.com
Acadia Pharmaceuticals Inc.
Jessica Tieszen
(858) 261-2950
ir@acadia-pharm.com
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Original: Acadia Pharmaceuticals Announces Planned Year-End Retirement of Elizabeth H.Z. Thompson, Ph.D., Head of Research and Development, Following a Distinguished Career
US Market News
2月前
Acadia Pharmaceuticals to Present Data at the 2026 American Academy of Neurology (AAN) Annual MeetingApril 17, 2026 9:05 AM
Business Wire
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that it will present multiple original data presentations spanning its portfolio at the 2026 American Academy of Neurology (AAN) Annual Meeting, taking place April 18–22, 2026 in Chicago, IL.
The Company will present real-world data from a sub-group analysis of the ongoing, Phase 4, prospective, observational, open-label LOTUS study evaluating the benefits and tolerability of DAYBUE® (trofinetide) in adults with Rett syndrome in routine clinical practice. In support of NUPLAZID® (pimavanserin) in Parkinson’s disease psychosis (PDP), Acadia will present exploratory analyses evaluating heterogeneity in treatment response trajectories and the impact of baseline sleep disturbances among PDP patients treated with pimavanserin. The Company is also debuting translational and pharmacokinetic research supporting the continued development of ACP-711, an investigational drug, for essential tremor. Collectively, these data reflect Acadia’s ongoing commitment to advancing scientific knowledge across a wide range of neurological conditions.
AAN Poster Presentations
P11.005: Real-world Benefits and Tolerability of Trofinetide for the Treatment of Adults with Rett Syndrome: the LOTUS Study, Wednesday, April 22, 11:45 AM – 12:45 PM CT
P11.006: Response Trajectories of Patients with Parkinson’s Disease Psychosis Treated with Pimavanserin: An Exploratory Cluster Analysis, Wednesday, April 22, 11:45 AM – 12:45 PM CT
P11.007: Impact of Baseline Sleep Disturbances on Pimavanserin Response in Parkinson’s Disease Psychosis: A Post Hoc Analysis, Wednesday, April 22, 11:45 AM – 12:45 PM CT
P7.007: Development of ACP-711, a Selective Modulator of GABA-A Receptor a3, for Essential Tremor: Use of First-in-Human Phase 1 Pharmacokinetics and Pharmacodynamics to Identify Target Dose/Exposure, Tuesday, April 21, 8:00 AM – 9:00 AM CT
P8.012: Mechanism of Action, Preclinical Efficacy, and Safety Evaluation of ACP-711 (SAN711): A Novel GABAA Subunit a3 Selective Modulator, Tuesday, April 21, 11:45 AM – 12:45 PM CT
About DAYBUE® (trofinetide) and DAYBUE® STIX (trofinetide)
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor-1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.1
Indication and Important Safety Information for DAYBUE® (trofinetide) and DAYBUE® STIX (trofinetide)
Indication
DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
Important Safety Information
Warnings and Precautions
Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was mild or moderate in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE.
Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if severe diarrhea occurs or if dehydration is suspected.
Vomiting: In a 12-week study, vomiting occurred in 29% of patients treated with DAYBUE and in 12% of patients who received placebo.
Patients with Rett syndrome are at risk for aspiration and aspiration pneumonia. Aspiration and aspiration pneumonia have been reported following vomiting in patients being treated with DAYBUE. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if vomiting is severe or occurs despite medical management.
Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if significant weight loss occurs.
Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).
Drug Interactions: Effect of DAYBUE and DAYBUE STIX on other Drugs
Trofinetide, a weak inhibitor of CYP3A and an inhibitor of P-gp, can increase the plasma concentrations of CYP3A and/or P-gp substrates (e.g., loperamide), which may increase the risk of adverse reactions associated with these substrates .
Closely monitor patients when DAYBUE or DAYBUE STIX is administered concomitantly with sensitive CYP3A and/or P-gp substrates for which a minimal increase in substrate plasma concentration (i.e., drugs with a narrow therapeutic index) may lead to serious adverse reactions.
Use in Specific Population: Renal Impairment
DAYBUE and DAYBUE STIX are not recommended for patients with severe renal impairment.
DAYBUE is available as an oral solution (200 mg/mL).
DAYBUE STIX for oral solution powder is available in 5,000 mg, 6,000 mg, and 8,000 mg packets.
Please read the full Prescribing Information also available at DAYBUEhcp.com.
About NUPLAZID® (pimavanserin)
Pimavanserin is a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. These receptors are thought to play an important role in neuropsychiatric disorders. In vitro, pimavanserin demonstrated no appreciable binding affinity for dopamine (including D2), histamine, muscarinic, or adrenergic receptors. Pimavanserin was approved for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis by the U.S. Food and Drug Administration in April 2016 under the trade name NUPLAZID.
Indication
NUPLAZID is indicated for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.
Important Safety Information
WARNING: INCREASED MORTALITY IN ELDERLY PATIENTS WITH DEMENTIA-RELATED PSYCHOSIS
Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death.
NUPLAZID is not approved for the treatment of patients with dementia who experience psychosis unless their hallucinations and delusions are related to Parkinson’s disease.
Contraindication: NUPLAZID is contraindicated in patients with a history of a hypersensitivity reaction to pimavanserin or any of its components. Rash, urticaria, and reactions consistent with angioedema (e.g., tongue swelling, circumoral edema, throat tightness, and dyspnea) have been reported.
Warnings and Precautions: QT Interval Prolongation
NUPLAZID prolongs the QT interval. The use of NUPLAZID should be avoided in patients with known QT prolongation or in combination with other drugs known to prolong QT interval (e.g., Class 1A antiarrhythmics, Class 3 antiarrhythmics, certain antipsychotics or antibiotics).
NUPLAZID should also be avoided in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes and/or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and presence of congenital prolongation of the QT interval.
Adverse Reactions: The adverse reactions (≥2% for NUPLAZID and greater than placebo) were peripheral edema (7% vs 2%), nausea (7% vs 4%), confusional state (6% vs 3%), hallucination (5% vs 3%), constipation (4% vs 3%), and gait disturbance (2% vs
US Market News
2月前
Acadia Pharmaceuticals Announces DAYBUE® STIX (trofinetide) is Now Broadly Available in the United States for the Treatment of Rett SyndromeApril 7, 2026 9:05 AM
Business Wire
-- DAYBUE STIX is a new powder formulation of trofinetide
-- Recently published expert consensus recognizes trofinetide as part of the standard of care for Rett syndrome and highlights the importance of flexible, patient-centered treatment approaches
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced DAYBUE® STIX (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide, is now broadly available in the United States for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. The new formulation, approved by the U.S. Food and Drug Administration (FDA) in December 2025, is bioequivalent to the original DAYBUE® oral solution, delivering the same efficacy and safety profile, while offering children and adults living with Rett syndrome new flexibility and choice regarding the dose volume and taste of their DAYBUE treatment.1
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260407618433/en/
"Initial feedback from a small group of caregivers following the limited launch revealed that more than 80% of early users reported satisfaction with DAYBUE STIX, highlighting the added flexibility and portability of this new formulation,"2 said Tom Garner, Acadia’s Chief Commercial Officer. “We are hearing that the new formulation may allow for more customized care in real-world settings. Ongoing evaluation from patients and caregivers remains a priority; their perspectives are essential as we identify ways to better assist families managing this complex condition.”
The importance of flexible, patient-centered approaches was reinforced in a recent publication of expert recommendations for real-world use of trofinetide in Rett syndrome. A steering group comprised of experts based at International Rett Syndrome Foundation (IRSF)-designated centers of excellence (COEs) reached consensus recognizing trofinetide oral solution as part of the standard of care for individuals with Rett syndrome. They also aligned on key real-world considerations such as early initiation and sustained use over time. The recommendations also reflect shared perspectives on the need for individualized decision making in clinical practice to help optimize outcomes for patients, families, and caregivers.3
“The availability of DAYBUE STIX gives us an additional, flexible way to administer trofinetide, which allows us more options to address unique patient and caregiver needs,” said Arthur Beisang, M.D., Department of Pediatrics, Gillette Children's Specialty Healthcare, Saint Paul, Minn. “This patient-centered approach aligns with recently published expert consensus recommendations, which advocate for the integration of trofinetide as part of the standard of care and comprehensive Rett syndrome management. This new option provides additional customization, supporting individualized care for people with Rett syndrome.”
DAYBUE STIX is a for oral solution powder that caregivers can mix with a variety of water-based liquids such as juice, tea, lemonade, limeade, or liquid hydration so that caregivers have the ability to customize to their loved ones' taste.4 The product comes in individual packets that are easily portable.
The efficacy and safety of DAYBUE STIX is based on the results of the pivotal Phase 3 LAVENDER™ study with DAYBUE oral solution in patients with Rett syndrome.4 The approval of this new formulation was informed by the results of a bioequivalence study, which demonstrated that both original DAYBUE oral solution and the new DAYBUE STIX for oral solution powder formulation provide comparable exposure.1
Families interested in exploring this new option should speak with their healthcare provider. Acadia also offers families access to Acadia Connect®, a multi-faceted support program that offers a dedicated, experienced support team assisting with financial resources and prescription support to patients and caregivers throughout the DAYBUE treatment journey. The original oral solution formulation approved by the U.S. Food and Drug Administration in 2023 will remain available.
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and occurs in approximately one of every 10,000 to 15,000 female births worldwide.5-7 In the U.S., 6,000 to 9,000 patients are affected.8 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration, which can last the rest of the patient’s life.6 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.9 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.9-11
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.12 Most Rett patients typically live into adulthood and require round-the-clock care.5,13
About DAYBUE® (trofinetide) and DAYBUE® STIX (trofinetide)
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor-1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.14
Indication and Important Safety Information for DAYBUE® (trofinetide) and DAYBUE® STIX (trofinetide)
Indication
DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
Important Safety Information
Warnings and Precautions
Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was mild or moderate in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE.
Advise patients to stop laxatives before starting DAYBUE or DAYBUE STIX. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if severe diarrhea occurs or if dehydration is suspected.
Vomiting: In a 12-week study, vomiting occurred in 29% of patients treated with DAYBUE and in 12% of patients who received placebo.
Patients with Rett syndrome are at risk for aspiration and aspiration pneumonia. Aspiration and aspiration pneumonia have been reported following vomiting in patients being treated with DAYBUE. Interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if vomiting is severe or occurs despite medical management.
Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE or DAYBUE STIX if significant weight loss occurs.
Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).
Drug Interactions: Effect of DAYBUE and DAYBUE STIX on other Drugs
Trofinetide, a weak inhibitor of CYP3A and an inhibitor of P-gp, can increase the plasma concentrations of CYP3A and/or P-gp substrates (e.g., loperamide), which may increase the risk of adverse reactions associated with these substrates.
Closely monitor patients when DAYBUE or DAYBUE STIX is administered concomitantly with sensitive CYP3A and/or P-gp substrates for which a minimal increase in substrate plasma concentration (i.e., drugs with a narrow therapeutic index) may lead to serious adverse reactions.
Use in Specific Population: Renal Impairment
DAYBUE and DAYBUE STIX are not recommended for patients with severe renal impairment.
DAYBUE is available as an oral solution (200 mg/mL).
DAYBUE STIX for oral solution powder is available in 5,000 mg, 6,000 mg, and 8,000 mg packets.
Please read the full Prescribing Information also available at DAYBUEhcp.com.
About Acadia Pharmaceuticals
Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “expects,” “anticipates,” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) the efficacy and safety profile of DAYBUE and DAYBUE STIX and anticipated Rett syndrome symptom improvements, (ii) the flexibility in administration and allowance for customized care provided by DAYBUE STIX, (iii) the use of DAYBUE and DAYBUE STIX as the standard of care for patients with Rett syndrome and (iv) potential future use of DAYBUE and DAYBUE STIX. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to: our ability to continue to successfully commercialize DAYBUE and DAYBUE STIX and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2025 filed with the Securities and Exchange Commission on February 26, 2026, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.
References
1
Mona D, Yamamoto A, Adegbenle Y, et al. A Phase 1, Randomized, Open-Label Study to Assess the Bioequivalence of Trofinetide as a Ready-to-Use Oral Solution and Constituted Powder for Oral Solution in Healthy Adults. Adv Ther. 2026.
2
Acadia Pharmaceuticals Inc., Data on file.
3
Prange EO, Beisang A, Pehlivan D, et al. Expert Consensus on Real-World Use of Trofinetide for Rett Syndrome Using a Modified Delphi Method. Ann Child Neurol. 2026; 4:38-51
4
Acadia Pharmaceuticals Inc. DAYBUE® [package insert]. San Diego, CA; 2025
5
Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020; 4:1-14.
6
Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216.
7
May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J of Med Econ. 26(1), 1570–1580.
8
Acadia Pharmaceuticals Inc., Data on file. RTT US Prevalence. March 2022.
9
Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188.
10
Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6):537-544.
11
Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1):217-227.
12
Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6):944-950.
13
Tarquinio DO, Hou W, Neul JL, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5):402-411.
14
Acadia Pharmaceuticals Inc., Data on file. Study Report 2566-026. 2010.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260407618433/en/
Investor Contact:
Acadia Pharmaceuticals Inc.
Al Kildani
(858) 261-2872
ir@acadia-pharm.com
Acadia Pharmaceuticals Inc.
Jessica Tieszen
(858) 261-2950
ir@acadia-pharm.com
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Original: Acadia Pharmaceuticals Announces DAYBUE® STIX (trofinetide) is Now Broadly Available in the United States for the Treatment of Rett Syndrome
US Market News
3月前
Acadia Pharmaceuticals Appoints Jonathan M. Poole to its Board of DirectorsMarch 3, 2026 4:05 PM
Business Wire
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Jonathan M. Poole to its Board of Directors, effective March 3, 2026. Mr. Poole will serve on the Company’s Audit Committee and brings deep biopharmaceutical finance leadership experience in supporting global growth and innovation across multiple therapeutic areas and modalities.
“Jonathan brings extensive biotechnology corporate leadership experience and a proven track record of supporting significant business growth across complex, global organizations,” said Stephen R. Biggar, M.D., Ph.D., Chairman of Acadia’s Board of Directors. “His appointment further strengthens the Board’s financial and operational expertise at a pivotal time as Acadia continues to execute its growth strategy, advance its commercial portfolio and pipeline, and deliver sustained, long-term value for shareholders.”
“I am honored to join Acadia’s Board of Directors,” said Mr. Poole. “I look forward to working with the Board and management team to support the Company’s mission of developing and delivering innovative therapies for patients with significant unmet needs.”
Since March 2020, Mr. Poole has served as Senior Vice President, Finance at Vertex Pharmaceuticals, Inc., where he has led the global finance team during a period of significant R&D portfolio and global commercial growth and diversification as well as the execution and integration of multiple acquisitions. Previously, from March 2018 to March 2020, Mr. Poole served as Chief Financial Officer of Evelo Biosciences, Inc., a biotechnology company developing orally delivered product candidates for inflammation and cancer. Prior to that, from April 2014 to March 2018, he served as Chief Financial Officer of Genocea Biosciences, Inc., a biotechnology company focused on developing infectious disease and neoantigen cancer vaccines. From July 2018 through March 2020, Mr. Poole also served as a director of Codiak Biosciences, Inc., where he was Chair of the Audit Committee.
Mr. Poole received a B.Sc. in Biological Sciences from Durham University in the United Kingdom and an M.B.A. from London Business School.
About Acadia Pharmaceuticals
Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260303056836/en/
Investor Contact:
Acadia Pharmaceuticals Inc.
Al Kildani
(858) 261-2872
ir@acadia-pharm.com
Acadia Pharmaceuticals Inc.
Jessica Tieszen
(858) 261-2950
ir@acadia-pharm.com
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Original: Acadia Pharmaceuticals Appoints Jonathan M. Poole to its Board of Directors
US Market News
3月前
Acadia Pharmaceuticals Announces Plan to Request Re-Examination Following Negative CHMP Opinion for Trofinetide for the Treatment of Rett SyndromeMarch 2, 2026 4:05 PM
Business Wire
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) informed the company it has formally adopted a negative opinion regarding the Marketing Authorization Application for trofinetide for the treatment of Rett syndrome in patients two years of age and older. Acadia has reviewed the CHMP grounds for refusal in detail and intends to request a re-examination of the opinion.
While the pivotal LAVENDERTM trial successfully met its co-primary and key secondary endpoints, the CHMP issued a refusal based on perceived deficits including: the treatment effect observed with trofinetide after 12 weeks, while measurable, was viewed as limited in magnitude; the study did not capture all core symptoms of Rett syndrome; and that assessment of longer-term outcomes was influenced by patient discontinuations over time. Acadia believes this feedback provides important information as it considers the intended re-examination.
“While we are disappointed by the CHMP’s recommendation to refuse approval, we continue to be encouraged by the meaningful benefits trofinetide has demonstrated for people living with Rett syndrome,” said Catherine Owen Adams, Acadia’s Chief Executive Officer. “The strong engagement and positive feedback we have seen from patients, caregivers, and clinicians in the Rett community reinforce our belief in the treatment’s clinical value. We remain committed to working constructively with EU regulators to explore next steps and to bring this therapy to patients.”
“Our family and others who play an important role in the delivery of care know first-hand the challenges that individuals living with Rett syndrome face every day,” said Markus Schulze, caregiver and member of the Rett Syndrome Society Nordrhein-Westfalen from Germany. “It is our hope that this important therapy will be approved to help the EU Rett community better navigate life with Rett syndrome.”
Trofinetide is approved in the United States, Canada and Israel, where it represents the first and only treatment approved for Rett syndrome.
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder and occurs in approximately one of every 10,000 to 15,000 female births worldwide.1-3 A child with Rett syndrome generally exhibits an early period of apparently normal development until six to 18 months, when many of their skills seem to slow down or stagnate. This is typically followed by a regression phase when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they could show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those individuals living with Rett may continue to experience a stage of motor deterioration, which can last the rest of the patient’s life.2 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.4 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication and brain plasticity, and the deficits in synaptic function may be associated with Rett manifestations.4-6
Features of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.7 Most individuals living with Rett syndrome typically live into adulthood and require intense round-the-clock care.1,8
About Trofinetide
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.9
About Acadia Pharmaceuticals
Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “could,” “would,” “intends”, “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential,” “guidance,” “continue” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about our plan to pursue the re-examination process, our beliefs about the benefits of trofinetide, and our commitment to making trofinetide available in the European Union. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to, the inherent uncertainty regarding development of product candidates, including the outcome or results of any re-examination of the CHMP’s formal opinion; our dependency on the continued successful commercialization of our products and our ability to maintain or increase sales of our products; our ability to obtain necessary regulatory approvals to commercialize our products and product candidates; if and when approved, market acceptance of our products and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2025 as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.
References
1 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:e000717.
2 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216.
3 May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J Med Econ. 2023;26(1):1570–1580.
4 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188.
5 Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6):537-544.
6 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1):217-227.
7 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6):944-950.
8 Tarquinio DO, Hou W, Neul JL, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5):402-411.
9 Acadia Pharmaceuticals Inc., Data on file. Study Report 2566-026. 2010.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260302383420/en/
Investor Contact:
Acadia Pharmaceuticals Inc.
Al Kildani
(858) 261-2872
ir@acadia-pharm.com
Acadia Pharmaceuticals Inc.
Jessica Tieszen
(858) 261-2950
ir@acadia-pharm.com
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Original: Acadia Pharmaceuticals Announces Plan to Request Re-Examination Following Negative CHMP Opinion for Trofinetide for the Treatment of Rett Syndrome
US Market News
4月前
Acadia Pharmaceuticals Provides Update on Regulatory Submission for Trofinetide for the Treatment of Rett Syndrome in the European UnionFebruary 2, 2026 4:05 PM
Business Wire
- Company expects to receive a negative opinion following oral explanation feedback and intends to request re-examination upon review of formal adoption.
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Company was informed by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) of a negative trend vote on its Marketing Authorization Application (MAA) for trofinetide for the treatment of Rett syndrome, following its recent CHMP oral explanation. Subject to the outcome of the CHMP vote in February, Acadia intends to request a re-examination of the opinion by the CHMP upon its formal adoption.
“While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver,” said Catherine Owen Adams, Chief Executive Officer. “We now have more than 1,000 patients on active treatment globally, from newly diagnosed 2-year-olds to adults who have lived with their disease for decades. Our ongoing real-world experience study in the U.S. continues to show outcomes that closely mirror the impact observed in rigorous randomized clinical trials conducted across a broad age range. We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU. Our commitment to the Rett syndrome community in the EU remains steadfast, and we are fully dedicated to making trofinetide available to individuals and families who urgently need a new therapeutic option.”
Pursuant to EU legislation, an applicant has the right to request a re-examination of a CHMP opinion within 15 calendar days of receipt of the opinion, followed by submission of the grounds for the request for re-examination within 60 calendar days of receipt of the opinion. The CHMP has up to 60 days after receipt of these grounds to re-examine its opinion.
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder and occurs in approximately one of every 10,000 to 15,000 female births worldwide.1-3 A child with Rett syndrome generally exhibits an early period of apparently normal development until six to 18 months, when many of their skills seem to slow down or stagnate. This is typically followed by a regression phase (general duration few weeks) when the child loses for example acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they could show mild recovery in cognitive interests, but body movements and other disease features may remain severely diminished. As they age, those individuals living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient’s life.2 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.4 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.4-6 Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.7 Most individuals living with Rett syndrome typically live into adulthood and require intense round-the-clock care.1,8
About Trofinetide
Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor.1 The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.9
About Acadia Pharmaceuticals
Acadia is committed to turning scientific promise into meaningful innovation that makes the difference for underserved neurological and rare disease communities around the world. Our commercial portfolio includes the first and only FDA-approved treatments for Parkinson’s disease psychosis and Rett syndrome. We are developing the next wave of therapeutic advancements with a robust and diverse pipeline that includes mid- to late-stage programs in Alzheimer’s disease psychosis and Lewy body dementia psychosis, along with earlier-stage programs that address other underserved patient needs. At Acadia, we’re here to be their difference. For more information, visit us at acadia.com and follow us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “may,” “will,” “should,” “could,” “would,” “intends”, “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential,” “guidance,” “continue” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about our plan to pursue the re-examination process, our beliefs about the benefits of trofinetide, and our commitment to making trofinetide available in the European Union. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to, the inherent uncertainty regarding development of product candidates, including the outcome of the CHMP vote and the results of any re-examination of the CHMP’s formal opinion; our dependency on the continued successful commercialization of our products and our ability to maintain or increase sales of our products; our ability obtain necessary regulatory approvals to commercialize our products and product candidates; if and when approved, market acceptance of our products and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2024 as well as our subsequent filings with the Securities and Exchange Commission from time to time, including our quarterly report on Form 10-Q for the quarter ended September 30, 2025. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.
References
1
Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020; 4:1-14.
2
Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216.
3
May DM, Neul JL, Satija A, et al. Real-world clinical management of individuals with Rett syndrome: a physician survey. J of Med Econ. 26(1), 1570–1580.
4
Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2):185-188.
5
Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6):537-544.
6
Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1):217-227.
7
Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6):944-950.
8
Tarquinio DO, Hou W, Neul JL, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5):402-411.
9
Acadia Pharmaceuticals Inc., Data on file. Study Report 2566-026. 2010.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260202406586/en/
Investor Contact:
Acadia Pharmaceuticals Inc.
Al Kildani
(858) 261-2872
ir@acadia-pharm.com
Acadia Pharmaceuticals Inc.
Jessica Tieszen
(858) 261-2950
ir@acadia-pharm.com
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Original: Acadia Pharmaceuticals Provides Update on Regulatory Submission for Trofinetide for the Treatment of Rett Syndrome in the European Union
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