Pegozafermin
- Generic Name
- Pegozafermin
GSK Acquires Efimosfermin Alfa from Boston Pharmaceuticals in $2 Billion Deal for Liver Disease Treatment
• GSK has agreed to acquire efimosfermin alfa, a phase III-ready liver disease drug, from Boston Pharmaceuticals in a deal worth up to $2 billion.
• The acquisition includes an upfront payment of $1.2 billion, with potential for additional success-based milestone payments totaling $800 million.
• This strategic move strengthens GSK's pharmaceutical portfolio in the liver disease therapeutic area, addressing significant unmet medical needs.
NASH Pipeline Shows Promise with 80+ Emerging Therapies in Development
• Over 80 companies are actively developing treatments for Non-Alcoholic Steatohepatitis (NASH), with Inventiva Pharma and Cirius Therapeutics leading with Phase III candidates lanifibranor and MSDC-0602K respectively.
• Lanifibranor, a triple PPAR agonist with FDA Breakthrough Therapy designation, demonstrates significant anti-inflammatory and antifibrotic properties, while MSDC-0602K selectively modulates the mitochondrial pyruvate carrier to improve metabolic parameters.
• Novel therapeutic approaches include Terns Pharmaceuticals' THR-β agonist TERN-501 and HighTide Biopharma's HTD1801, both showing promising results in reducing liver fat and improving metabolic markers in Phase II trials.
Akero's Efruxifermin Shows Promise in Reversing Cirrhosis Due to MASH in Phase 2b Trial
• Akero Therapeutics' Efruxifermin (EFX) demonstrated statistically significant cirrhosis reversal in patients with MASH in a Phase 2b trial.
• In patients with baseline and week 96 biopsies, 39% treated with 50mg EFX showed cirrhosis reversal without worsening MASH, compared to 15% with placebo.
• The SYMMETRY study underscores the benefit of longer EFX treatment, showing a doubling of effect size from weeks 36 to 96 in the 50mg group.
• Akero is continuing to evaluate 50mg EFX in the Phase 3 SYNCHRONY Outcomes study for patients with compensated cirrhosis due to MASH.
89bio's Pegozafermin Advances in Phase 3 Trials for MASH and Severe Hypertriglyceridemia
• 89bio completed enrollment in the Phase 3 ENTRUST trial for severe hypertriglyceridemia (SHTG), with topline data expected in the second half of 2025.
• The Phase 3 ENLIGHTEN program, targeting metabolic dysfunction-associated steatohepatitis (MASH), continues to enroll patients in both non-cirrhotic and cirrhotic arms.
• 89bio's financial position is strong, with approximately $440 million in cash, cash equivalents, and marketable securities as of December 31, 2024.
NASH Treatment Market to Reach Significant Growth by 2032, Driven by Emerging Therapies
• The non-alcoholic steatohepatitis (NASH) market is poised for substantial growth, projected to expand significantly by 2032 across the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
• This growth is fueled by the introduction of novel therapies targeting NASH, with several drugs in Phase II and Phase III clinical trials showing promise for improving treatment outcomes.
• Key players such as Madrigal Pharmaceuticals, Intercept Pharmaceuticals, and Novo Nordisk are actively developing innovative treatments, contributing to a dynamic and competitive market landscape.
• The increasing prevalence of NASH and the growing understanding of its pathophysiology are driving the demand for effective therapies, creating opportunities for pharmaceutical companies.
Efimosfermin Alfa Shows Promise in Phase II MASH Trial with Monthly Dosing
• Boston Pharmaceuticals' efimosfermin alfa demonstrated statistically significant fibrosis improvement in MASH patients, with 45.2% showing at least a one-stage improvement compared to 20.6% in the placebo group.
• The Phase II trial also revealed that 67.7% of patients treated with efimosfermin alfa achieved MASH resolution without fibrosis worsening, versus 29.4% in the placebo group, indicating a significant treatment effect.
• Efimosfermin alfa, a long-acting FGF21 analog administered monthly, offers a potentially more convenient dosing schedule compared to other FGF21 analogs in development for MASH.
• The drug exhibited a good tolerability profile over 24 weeks, with a low incidence of injection-site reactions and gastrointestinal toxicities, supporting its further development.
89bio's Pegozafermin Advances Through Phase 3 Trials for MASH and SHTG
• 89bio is progressing pegozafermin, a FGF21 analog, through Phase 3 trials for metabolic dysfunction-associated steatohepatitis (MASH) and severe hypertriglyceridemia (SHTG).
• Pegozafermin received Breakthrough Therapy Designation from the FDA for MASH and PRIME designation from the EMA, based on positive Phase 2b ENLIVEN trial data.
• The company initiated Phase 3 ENLIGHTEN trials for MASH and the ENTRUST trial for SHTG, with topline SHTG results expected in 2025.
• 89bio's current funds of $423.8 million are expected to support operations for at least one year, as the company expands its clinical and commercial capabilities.
Madrigal's Resmetirom Nears Full Approval for NASH Cirrhosis Treatment Following Phase 3 Enrollment Completion
• Madrigal Pharmaceuticals completed patient enrollment for its MAESTRO-NASH OUTCOMES trial, evaluating resmetirom for compensated NASH cirrhosis.
• The Phase 3 trial involves 845 patients and aims to measure the progression to liver decompensation events over two to three years.
• Positive outcomes from the trial could support full approval of Rezdiffra for noncirrhotic NASH and expand its indication to cirrhotic patients.
• Resmetirom, a liver-directed THR-β agonist, has already received accelerated approval for NASH with moderate to advanced liver fibrosis.
FDA Approves Rezdiffra (Resmetirom) as First Treatment for NASH with Liver Fibrosis
• The FDA has granted accelerated approval to Rezdiffra (resmetirom) for adults with noncirrhotic NASH and moderate to advanced liver fibrosis.
• Rezdiffra, a THR-β agonist, is the first FDA-approved medication for NASH, also known as MASH, marking a significant milestone.
• Approval was based on Phase 3 MAESTRO-NASH trial data, which demonstrated improved liver fibrosis and NASH resolution compared to placebo.
• Madrigal plans to launch Rezdiffra in the U.S. in April, with a patient support program to aid access and affordability.
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