Clinical Trials
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- GSK initiated two Phase 3 trials (NEBULA-1 and NEBULA-2) for efimosfermin alfa, an FGF21 analog, targeting compensated cirrhosis (fibrosis stage F4) due to MASH. - NEBULA-1 plans to enroll 1,740 patients with a primary endpoint of time to first liver-related clinical composite outcome, while NEBULA-2 plans 380 patients assessing fibrosis improvement without MASH worsening at 96 weeks. - The move signals a competitive shift in the MASH field from intermediate fibrosis stages toward cirrhosis, a population with no currently approved pharmacological treatment options. - Efimosfermin holds FDA Breakthrough Therapy and EMA PRIME designations, with Phase 2 data showing 45.2% fibrosis improvement and 67.7% MASH resolution without worsening fibrosis.
- Variant Bio, a genomics-driven AI drug discovery company, has appointed Craig T. Basson, MD, PhD, as Chief Medical Officer and President of R&D to advance clinical programs. - Dr. Basson brings over 25 years of leadership experience, including successful development of multiple therapies at Novartis such as Entresto, Leqvio, and Ilaris. - The appointment positions Variant Bio to accelerate translation of genetic discoveries into differentiated clinical programs using their proprietary Inference AI platform. - Dr. Basson will direct research and lead clinical strategy as the company moves therapeutics informed by large-scale human genomics studies into clinical development.
- 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.
- GSK has agreed to acquire efimosfermin alfa from Boston Pharmaceuticals for $1.2 billion upfront, with potential additional milestone payments of $800 million, strengthening its hepatology pipeline. - Efimosfermin, a phase III-ready FGF21 analog, has shown promising results in reversing liver fibrosis in patients with metabolic dysfunction-associated steatohepatitis (MASH), with a convenient once-monthly dosing regimen. - Steatotic liver disease affects approximately 5% of the global population with limited treatment options, and interventions that reduce fibrosis could save the US healthcare system between $40-100 billion over the next two decades.
• 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.
• Efimosfermin alfa demonstrated statistically significant improvement in liver fibrosis without worsening of MASH compared to placebo over 24 weeks. • Two-thirds of patients treated with efimosfermin achieved MASH resolution without worsening of fibrosis, showing a significant benefit over placebo. • The study also revealed significant and rapid improvements in cardiometabolic parameters, including liver fat reduction and glycemic control. • Boston Pharmaceuticals plans to advance efimosfermin to late-stage clinical development in 2025 based on these promising Phase 2 results.
- GSK has agreed to acquire Boston Pharmaceuticals' phase 3-ready liver disease candidate efimosfermin alfa for $1.2 billion upfront, with potential milestone payments bringing the total to $2 billion. - Efimosfermin, a once-monthly FGF21 analog therapeutic, has demonstrated significant ability to reverse liver fibrosis and halt disease progression in patients with moderate-to-advanced MASH in phase 2 trials. - The acquisition expands GSK's hepatology pipeline and offers potential combination therapy options with GSK's siRNA therapeutic GSK'990, targeting steatotic liver disease which affects approximately 5% of the global population.
