Genfit SA (GNFT) ニュース

Volume: 620
avg daily vol (30): 7.8k

GNFT under $4

Compared to Cymabays Seladelpar, Genfits Elafibranor is the better option for chronic itch as their ppar alpha activation seems to be more effective in reducing bile acid levels.

GNFT under $6

good PBC science

GNFT under $5

GNFT under $5

$GNFT FDA approval will make a decision on whether new drug application can be approved for the disease primary bilary cholangitis which is a liver disease the treatment is called Elafibranor.

~~Phase 2~~
Efficacy, safety and tolerability of vs-01 in adult patients with acute-on-chronic liver failure and acetitis (UNVEIL IT) Completion expected 08/01 (53 days)

~~ Phase 2~~
Combination of GNS561 and trametinib of patients with advanced KRASS mutation Cholangiocarcinoma, expected 06/01 (357 days remain)
--- this is a long term hold ---

This company will be worth more than $4 billion dollars upon P2 success of acute liver failure trial ready for interim readout in Aug 24

The acquisition of Swiss Versantis AG expands Genfit’s pipeline with three programmes: the Phase II-ready candidate VS-01-ACLF designed to treat acute-on-chronic liver failure, VS-01-UCD, a pediatric program focused on urea cycle disorder, and VS-02-HE, an early-stage program focused on hepatic encephalopathy. Genfit will pay €40m upfront, €65m in potential milestones and 33% of the net proceeds from the sale of a FDA-Priority Review Voucher worth $100m.

With this acquisition of all shares and voting rights from the Zurich-based R&D specialist, Genfit consolidates its position in ACLF via the integration of VS-01-ACLF. Genfit's main asset, VS-01, is a first-in-class innovative liposomal-based therapeutic product candidate currently in clinical development as a potential first-line therapy for the timely recovery of ACLF and UCD. ACLF is an underserved medical condition associated with short-term mortality (23% to 74% mortality at 28 days, depending on severity grade) and a significant cost of care. ACLF syndrome is characterized by an abrupt life-threatening worsening of a pre-existing advanced chronic liver disease resulting in liver and extrahepatic organ failure. The cascade of brain, kidney, cardiovascular and respiratory failure, include the development of a neuropsychiatric condition called hepatic encephalopathy (HE).

Its main asset, VS-01, is a first-in-class innovative liposomal-based therapeutic product candidate currently in clinical development as a potential first-line therapy for the timely recovery of ACLF and UCD. If approved, it would be the first drug to use the intraperitoneal route to simultaneously support the liver, kidney and brain, the organs that most often fail in cirrhotic patients. VS-01 operates to clear toxic metabolites from the body following paracentesis, by extracting them from the blood into the peritoneal (abdominal) cavity, where they are captured by proprietary scavenging liposomes which are then drained from the body. A planned 60-patient, randomized and controlled Phase 2 Proof-of-concept trial of VS-01 in ACLF is expected to launch in the fourth quarter 2022. Efficacy and safety interim data are expected as early as the first half of 2024. The US Food and Drug Administration (FDA) granted VS-01 with the Orphan Drug Designation (ODD) in ACLF and in UCD and with the Rare Pediatric Diseases Designation (RPDD) for the acute treatment of UCD. The European Medicines Agency (EMA) also granted VS-01 with ODD in acute liver failure. Given the unmet medical need and the current standard of care, GENFIT intends to seek approval of these candidates via expedited regulatory pathways.

A planned 60-patient, randomized and controlled Phase 2 Proof-of-concept trial of VS-01 in ACLF is expected to launch in the fourth quarter 2022. Efficacy and safety interim data are expected as early as the first half of 2024. The US Food and Drug Administration (FDA) granted VS-01 with the Orphan Drug Designation (ODD) in ACLF and in UCD and with the Rare Pediatric Diseases Designation (RPDD) for the acute treatment of UCD. The European Medicines Agency (EMA) also granted VS-01 with ODD in acute liver failure. Given the unmet medical need and the current standard of care, GENFIT intends to seek approval of these candidates via expedited regulatory pathways.

VS-02 is a pre-clinical oral, small molecule drug candidate being developed for the chronic management of HE, considered an endemic disease worldwide. HE is a nervous system disorder brought on by advanced chronic liver disease. VS-02 will be developed as a unique colon-active formulation designed to minimize systemic absorption of ammonia and act where ammonia is primarily produced, while reducing glutamine levels in the brain.

Be patient
The drug was approved by FDA and P2 for acute liver trial is complete on 08/01
This will be bought out first $4 billion dollars soon

an new down 6% for GNFT

Agreement gives Ipsen global1 rights to develop and commercialize GENFIT’s late-stage, first-in-class PPAR alpha and delta agonist elafibranor in Primary Biliary Cholangitis (PBC)
Investigational treatment elafibranor being evaluated in the global Phase III trial, ELATIVETM, with topline data expected early 2023
GENFIT receives €120m upfront and is eligible to receive up to €360m in milestone payments as well as tiered double-digit royalties of up to 20%
Ipsen becomes 8% shareholder of GENFIT via an equity investment of €28m
Paris (France), 17 December 2021 – Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT (Nasdaq and Euronext: GNFT) have entered into a long-term strategic partnership for global collaboration between the two companies. The agreement gives Ipsen exclusive worldwide* license to develop, manufacture and commercialize GENFIT’s investigational treatment elafibranor, for people living with Primary Biliary Cholangitis (PBC). The partnership also gives Ipsen access to future clinical programs led by GENFIT and combines GENFIT’s scientific expertise and proprietary technologies in liver disease with Ipsen’s development and commercialization capabilities. To underscore the long-term commitment represented by this partnership, Ipsen will also purchase newly issued GENFIT equity representing 8% post-issuance through a €28m investment in GENFIT, becoming one of the largest shareholders.
The ongoing, pivotal Phase III global trial, ELATIVETM, 1 is evaluating the safety and efficacy of elafibranor in 150 people living with PBC who have an inadequate response or intolerance to ursodeoxycholic acid (UDCA). Global recruitment is well underway. There is significant unmet medical need for people with PBC and, following positive Phase II data, 2 elafibranor was granted Breakthrough Therapy Designation by the U.S Food and Drug Administration (FDA) and Orphan Drug Designation by the U.S. FDA and European Medicines Agency (EMA). 3,4 Results from the Phase II randomized double-blind, placebo controlled trial found that after 12 weeks of dosing with elafibranor, patients with PBC unresponsive to UDCA experienced significantly reduced levels of disease-activity markers including alkaline phosphatase (ALP) and composite endpoints with bilirubin as well as other markers of disease activity when compared to placebo.2
David Loew, Chief Executive Officer, Ipsen, said “Today’s announcement marks an exciting new stage in Ipsen’s ambitions to expand our portfolio to support more people living with rare diseases around the world. We are excited by elafibranor’s data package, demonstrating the potential benefit of this first-in-class, innovative treatment option to help the PBC community. We look forward to the results of the ongoing Phase III program and regulatory submissions around the world to bring this potential new treatment option to patients. Ipsen is pleased to partner with GENFIT, a company that shares our common values and goals of bringing to market first-in-class treatments to improve the lives of people living with rare conditions like PBC.”

Pascal Prigent, Chief Executive Officer of GENFIT added: “We are excited to partner with Ipsen and launch this long-term strategic collaboration, with the goal to accelerate our growth and generate value for our shareholders. Ipsen’s world-class development capabilities, well-established global commercial footprint and excellent track record in delivering therapies to patient populations with unmet medical need makes it the ideal partner for GENFIT. Today’s landmark agreement demonstrates our ability to advance highly promising assets into late-stage development in-house and derive significant value. While we hope, above all, that this partnership with Ipsen will be a significant step towards having a positive impact on the lives of millions of patients suffering from life-threatening liver diseases, we also believe our shareholders will recognize the benefit offered by this collaboration model. The transaction proceeds indeed reinforce GENFIT’s long-term financial visibility, including further funding for GENFIT to expand its pipeline, and they also provide opportunities for targeted business development, as exemplified by today’s other announcement regarding our in-licensing of a new molecule.”

PBC is a rare, progressive, chronic autoimmune disease of the liver.5 Bile is a liquid produced inside the liver that is used to help digest fats and remove waste products from the body.6 PBC leads to a slow, progressive destruction of the small bile ducts of the liver, causing bile and other toxins to build up in the liver (known as cholestasis).5 Further damage can lead to scarring, fibrosis and eventually cirrhosis of the liver.5 Common symptoms of PBC include fatigue and pruritus’ (itching) which can be debilitating and, in more advanced cases, jaundice.5 Untreated, PBC can lead to liver failure, or in some cases death. PBC is more common in women with nine women diagnosed for every man; it is also a leading cause of liver transplantation.5

GENFIT remains responsible for the Phase III ELATIVETM trial until the completion of the double-blind period. Ipsen will assume responsibility for all additional clinical development, including completion of the long-term extension period of the ELATIVETM trial, and global* commercialization. This newly established strategic partnership will also provide Ipsen with access to GENFIT’s research capabilities and other clinical programs through rights to first negotiation.
Under the agreement, Ipsen will pay GENFIT up to €480m, comprising upfront cash payment of €120m, as well as regulatory, commercial, and sales-based milestone payments up to €360m, plus tiered double-digit royalties of up to 20%. Ipsen also becomes a shareholder of GENFIT through the purchase of 3,985,239 newly issued shares representing 8% of GENFIT S.A after issuance, via a €28m investment. The new shares will be issued pursuant to the twentieth resolution of GENFIT’s 30 June 2021 shareholders’ meeting and will be subject, upon issuance, to a lock-up period ending, in the event of positive ELATIVE TM results, on the earlier of the date on which the EMA makes a formal recommendation to the European Commission for the marketing authorisation of elafibranor in PBC or the date on which the U.S. FDA grants approval of elafibranor in PBC. Issuance of the new shares is expected to take place on or about December 22, 2021. In addition, the Board of Directors of GENFIT will propose at the next shareholders’ meeting that Ipsen becomes a board member.
The transaction is expected to be dilutive to Ipsen’s profitability over the near term, primarily reflecting R&D and launch-preparation expenses. This is in line with Ipsen’s medium-term outlook regarding its strategic focus on building a high-value and

$GNFT Ipsen’s Iqirvo® receives U.S. FDA accelerated approval as a first-in-class PPAR treatment for primary biliary cholangitis.

Iqirvo® (elafibranor) 80 mg tablets is the first new medicine approved in nearly a decade for the treatment of the rare liver disease called primary biliary cholangitis

Approval based on positive Phase III ELATIVE trial data

Primary biliary cholangitis impacts approximately 100,000 people in the US and is growing in global prevalence. If inadequately treated, it can cause liver failure

U.S. approval of Iqirvo establishes Ipsen as a leader in the treatment of rare cholestatic liver diseases

Get ready for 300% rise upon FDA approval on next Monday
Only $260 mil MC now
Similar drug got bought out for $4.3 billion
This drug outperform the current approval drug
Super excited next Monday

This is why this drug will be approved on 06/10

Ocaliva, the only approved second-line treatment for PBC, can exacerbate rather than ease itching. That suggests even the numerical improvement seen in the Ipsen and Genfit trial could give elafibranor an edge over Ocaliva.

Primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis, is the most common cholestatic disease among middle-aged women in the US. 1 The autoimmune disorder is characterized by progressive degeneration of intrahepatic bile ducts, culminating in periportal inflammation and cholestasis. Prolonged hepatic cholestasis precipitates the development of cirrhosis and portal hypertension, posing significant clinical challenges.


Ursodeoxycholic acid (UDCA) is the first treatment authorized by the US Food and Drug Administration (FDA) for managing PBC. It has steadfastly retained its status as the first-line therapeutic modality. Its mechanism of action is multifaceted: shielding cholangiocytes from the deleterious effects of hydrophobic bile acids, augmenting hepatobiliary bile acid excretion, enhancing the hydrophilicity index of the circulating bile pool, and purportedly exerting immunomodulatory and anti-inflammatory responses.2

UDCA can diminish progression to cirrhosis and reduce the incidence of liver transplants. However, approximately 40% of patients with PBC are refractory to it, and see their disease advance despite therapy.3 There is a paucity of approved second-line options for UDCA-resistant PBC, with obeticholic acid, a selective farnesoid X receptor agonist, as the standalone drug approved in this category.4


Reducing Bile Acid Toxicity and Uptake

Peroxisome proliferator-activated receptors (PPARs), including PPAR-a, PPAR-d, and PPAR-?, are a subset of the nuclear receptor family of ligand-activated factors, which play a pivotal role in cellular regulation. PPAR agonists such as bezafibrate are used off-label as second-line treatments in PBC.5 These agents have garnered considerable attention as potential therapeutic tools for PBC and various cholestatic liver disorders.6

Upon ligand binding, PPARs engage in heterodimerization with the retinoid X receptor and orchestrate the transcriptional activation of genes involved in diverse metabolic pathways, including fatty-acid metabolism, glucose metabolism, and modulation of inflammatory responses. Additionally, PPARs exert inhibitory effects on the transcriptional activity of pro-inflammatory genes, thereby contributing to the regulation of inflammatory processes.6

Specifically, PPAR-a exhibits preferential expression within hepatocytes of the liver, while PPAR-d displays a broader expression profile encompassing hepatocytes, Kupffer cells (hepatic macrophages), stellate cells, and cholangiocytes within the hepatic milieu.

Saroglitazar was one of the first PPAR agonists studied in patients with treatment-refractory PBC; however, trials have been hindered due to a lack of enrollment.7 Saroglitazar has been overshadowed by the positive results of other PPAR agonists—elafibranor and seladelpar, which have recently been studied as second-line agents for UDCA-resistant PBC. Elafibranor is a dual PPAR-a and PPAR-d agonist, while seladelpar is a selective PPAR-d agonist.


Read more about PBC experimental therapies

PPAR-a and PPAR-d agonists modulate several complementary pathways. A key mechanism for both experimental therapies in treating PBC is the suppression of bile-acid synthesis, as evidenced by lower levels of the bile acid precursor C4 and total serum bile acids.6 In addition, PPAR-a agonism is also known to reduce bile acid toxicity and the uptake of bile acids into hepatocytes. These remarkable anticholestatic characteristics are critical to the efficacy of these medications.

In the current trials, elafibranor and seladelpar improved lipid profiles, which might be mediated by their known induction of fibroblast growth factor. Although PPAR-a agonism reduced liver fibrosis in preclinical models, only seladelpar reduced liver stiffness and fibrosis in patients with PBC.6

A Dual-Pronged Approach

The efficacy and safety of elafibranor, which is administered orally, was evaluated in the ELATIVE trial. This was a multinational, phase 3, double-blind, placebo-controlled trial with an open-label long-term extension. Patients were enrolled and randomized 2:1 to receive 80 mg of elafibranor once daily or placebo.9

The double-blind period of the study comprised 2 sequential parts. In the initial phase, patients were allocated randomly to either elafibranor or placebo groups, with treatment administered in a double-blind manner for a minimum of 52 weeks. Subsequently, in the second phase, patients continued their assigned treatment regimen beyond the initial 52 weeks until all participants had completed their week 52 assessments or until reaching a maximum duration of 104 weeks, whichever came first.9

The primary endpoint of the trial was a biochemical response at week 52 (defined as an alkaline phosphatase [ALP] level of less than 1.67 times the upper limit of normal (ULN), plus a reduction of 15% or more from baseline, and normal total bilirubin levels). At week 52, a significant biochemical response was noted in 51% of the elafibranor group, compared to 4% of patients in the placebo group (95% CI, 32-57; P

Ipsen and Genfit have shown off the data they believe can finally secure approval of their liver disease candidate elafibranor. The publication puts meat on the bones of an earlier data drop, providing a closer look at how the therapy holds up against Intercept Pharmaceuticals’ Ocaliva and CymaBay Therapeutics’ own challenger.

Elafibranor, a dual peroxisome activated receptor, is no longer the potential cash cow it looked to be as Genfit was racing Intercept for the nonalcoholic steatohepatitis market. But the promise of the molecule in the rare liver disease primary biliary cholangitis (PBC), a setting in which Intercept’s Ocaliva is already approved, was compelling enough to persuade Ipsen to pay 120 million euros ($129 million) to partner on the project in 2021. Ipsen has gained a clearer look at what it bought in recent months.




In June, Ipsen and Genfit reported 51% of people on the 80-mg dose of elafibranor achieved the primary endpoint response, compared to 4% of people in the placebo group. Yet, the molecule’s failure to cause a statistically significant improvement in itching, a potential point of differentiation versus Ocaliva, raised questions about the competitive positioning of elafibranor.


The publication of full phase 3 results in The New England Journal of Medicine provides more fuel for the debate. In patients with moderate to severe pruritus, scores on the 10-point Worst Itch Numeric Rating Scale improved by 1.93 in the elafibranor group and 1.15 in the placebo arm by Week 52. The numerical difference fell short of statistical significance.

RELATED

Ipsen, Genfit report phase 3 rare liver disease success, setting up showdown with Intercept
Ipsen and Genfit also assessed itching using two other measures. The 52-week data on a quality-of-life questionnaire and another itch scale “appeared to favor elafibranor over placebo,” the authors of the NEJM paper wrote.



Ocaliva, the only approved second-line treatment for PBC, can exacerbate rather than ease itching. That suggests even the numerical improvement seen in the Ipsen and Genfit trial could give elafibranor an edge over Ocaliva. The problem, for the two French drugmakers, is that Ocaliva is unlikely to be the only other game in town.

CymaBay shared top-line phase 3 results on its PBC prospect seladelpar in September and followed up with a closer look at the data Monday. In the CymaBay trial, 61.7% of patients on 10 mg of seladelpar met the primary composite endpoint, but the placebo-response rate, 20%, was much higher than in the elafibranor trial. Differences between the baseline characteristics complicate the cross-trial comparison, but the placebo-adjusted analysis factors elafibranor.

Seladelpar looks to have an edge over elafibranor in pruritus, achieving a statistically significant result on the worst itch scale and a clear separation from placebo on another measure that tied (PDF) the changes to improved sleep. Ipsen and Genfit have submitted their data to regulators, while CymaBay is looking “toward moving into the regulatory filing process.” The biotechs are targeting a market in which Ocaliva is forecast to generate $320 million to $340 million this year.

This will be trading over $20 next week
FDA approval is given

Results from Ipsen’s ELATIVE® pivotal Phase III trial of elafibranor in PBC presented as late breaking data at AASLD congress and published in New England Journal of Medicine
- 14 November 2023 - 11 mins read
ELATIVE® Phase III trial confirms potential for investigational elafibranor as a novel, first-in-class, dual PPAR a,d agonist for patients with primary biliary cholangitis.
Elafibranor demonstrates significant improvements in biomarkers of disease progression versus placebo, including significant treatment benefit with improvement in biochemical response and alkaline phosphatase (ALP) normalization, along with patient-reported outcomes data suggesting a possible improvement in pruritus.
Elafibranor was generally well-tolerated with a well-documented safety profile consistent with previous trials.
PARIS, FRANCE, 13 November, 2023 – Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT (Nasdaq and Euronext: GNFT) today announced full results from the pivotal Phase III ELATIVE® trial, which are being presented in a late-breaking oral session (Abstract #484, Monday, 13 November at 16.45 EST)at the American Association for the Study of Liver Disease (AASLD) and simultaneously published in the New England Journal of Medicine (NEJM). This trial evaluated the efficacy and safety of investigational elafibranor, an oral, dual PPAR a,d agonist, as a potential novel class of treatment for patients with the rare, autoimmune cholestatic liver disease, primary biliary cholangitis (PBC).

Results show statistically significant improvements in biomarkers of disease progression across key endpoints with a significant treatment benefit achieved in the primary composite endpoint, demonstrating a 47% placebo-adjusted difference (P

February 12, 2024

Gilead Sciences Expands Liver Portfolio With Acquisition of CymaBay Therapeutics
-- Gilead Adds Seladelpar to Portfolio, a PPARd Agonist for the Treatment of Primary Biliary Cholangitis (PBC) with FDA Priority Review and Anticipated U.S. Approval in Third Quarter of 2024 --

-- Seladelpar Phase 3 Data Demonstrates a Best-in-Disease Profile for Second-Line PBC --

-- Acquisition Expands Gilead’s Long-Standing Commitment to Patients with Liver Diseases --
FOSTER CITY, Calif. & NEWARK, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion. The addition of CymaBay’s investigational lead product candidate, seladelpar for the treatment of primary biliary cholangitis (PBC) including pruritus, complements Gilead’s existing liver portfolio and aligns with its long-standing commitment to bringing transformational medicines to patients.

“We are looking forward to advancing seladelpar by leveraging Gilead’s long-standing expertise in treating and curing liver diseases,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “Building on the strong research and development work by the CymaBay team to date, we have the potential to address a significant unmet need for people living with PBC and expand on our existing broad range of transformational therapies.”

PBC is a rare, chronic, cholestatic liver disease mainly affecting women (1 in 1,000 women over the age of 40 or about 130,000 total people in the U.S.) that impairs liver function and quality of life. The most common early symptoms of PBC are pruritus (itching) and fatigue, which can be debilitating for some patients. Progression of PBC is associated with an increased risk of liver-related mortality.

Seladelpar is an investigational, oral, selective peroxisome proliferator-activated receptor delta (PPARd) agonist, shown to regulate critical metabolic and liver disease pathways. The United States Food and Drug Administration (FDA) has completed its filing review and accepted a New Drug Application for seladelpar and granted priority review with a Prescription Drug User Fee Act target action date of August 14, 2024.

Seladelpar received FDA Breakthrough Therapy Designation for use in the treatment of PBC including pruritus in patients without cirrhosis or with compensated cirrhosis and PRIME status (EMA), as well as Orphan Drug Designation in the U.S. and Europe for the treatment of patients with PBC.

In the pivotal Phase 3 RESPONSE trial, seladelpar achieved statistical significance over placebo across primary composite endpoints of biochemical response (61.7% for patients on seladelpar vs 20.0% for placebo), normalization of alkaline phosphatase at 12 months (25.0% for patients on seladelpar vs 0.0% for placebo) and statistically significant improvement in pruritus at six months among people living with moderate-to-severe itch that was sustained through 12 months.

“Today’s agreement with Gilead is the culmination of years of focus and determination at CymaBay to advance seladelpar and bring new hope to people living with PBC and their families,” said Sujal Shah, President, and CEO at CymaBay Therapeutics. “Now that seladelpar has achieved priority review with the FDA, we are excited that Gilead, with its long-standing commitment to patients with liver disease, can apply its regulatory and commercial expertise to bring seladelpar as quickly as possible to people with PBC.”

similar drug Seladelpar was bought by Gilead for 4.3bln.
This stock will explode over $20-$40 upon FDA approval next Monday

This could explode over $20 upon FDA approval on 06/10

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

December 7, 2023
PDF Version
New Drug Application granted priority review with PDUFA date set for June 10, 2024
European Medicines Agency (EMA) has also validated the Marketing Authorization Application (MAA) for elafibranor
Investigational elafibranor is the first novel second-line treatment for primary biliary cholangitis (PBC) to be filed in E.U. and U.S. in nearly a decade

Paris (France), December 07, 2023 – Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT (Nasdaq and Euronext: GNFT) today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for investigational elafibranor. An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR a,d) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade. The target FDA PDUFA date under priority review is June 10, 2024.

The European Medicines Agency (EMA) has also validated Ipsen’s Marketing Authorization Application (MAA) for elafibranor and the review of the submission to the EMA’s Committee for Medicinal Products for Human Use (CHMP) began on 26 October 2023. Furthermore, a third simultaneous regulatory filing of elafibranor has been validated for review by the UK Medicines and Healthcare products Regulatory Agency (MHRA).

“We are delighted to have achieved simultaneous filings for elafibranor, which is in line with our ambition to be able to bring a new and much needed medicine to as many people living with PBC as rapidly as possible,” said Christelle Huguet, EVP and Head of Research & Development, Ipsen. “This is a condition where many patients are living with worsening disease and debilitating symptoms despite being on treatment. Elafibranor, if approved, has the potential to change the management of this challenging condition for people living with PBC, offering a new second line treatment choice, where the number of effective options are currently limited.”

PBC is a rare, progressive, autoimmune cholestatic liver disease1 in which bile ducts in the liver are gradually destroyed.2 The damage to bile ducts can inhibit the liver’s ability to rid the body of toxins, and can lead to scarring of liver tissue, known as cirrhosis.1,2,3 Common symptoms of PBC include fatigue and pruritus (itch), which can be severely debilitating.4 Untreated, PBC can lead to liver failure, or in some cases death.1 It primarily affects women, with nine women diagnosed for every man.3 A significant proportion of people living with PBC do not benefit from existing
therapies.5, 67

“These simultaneous regulatory submission acceptances are another important step in the elafibranor journey. We are pleased to be partnering with Ipsen, who we know has a good understanding of the rare-disease regulatory process,” said Pascal Prigent, Chief Executive Officer of GENFIT. “We know they share the same goal as GENFIT, to bring a new, much needed treatment option to people living with PBC as fast as possible; we look forward to elafibranor’s progress through the regulatory review processes.”

NOBODY??
GNFT https://schrts.co/dZtaIjyN GNFT mcap 250mln.
small cousin of Cymabay (acquired by Gilead for 4.3bn in Feb 2024), think frontrun of PDUFA on June10 2024

PDUFA action date for GNFTs Elafibranor (PPARd in PBC) is June10. Cymabay was only Seladelpar, also a PPARd in PBC indication. Elafibranor looks as great as Seladelpar. GNFT also has other programs. I bought shares when the Cymabay buyout was announced.
Please note that Seladelpars PDUFA is in Aug 24. Gilead BO validates the MOA.

Seladelpar was sold for 4.3bln.