Sagimet's Denifanstat Receives FDA Breakthrough Therapy Designation for MASH
核心洞察
Sagimet Biosciences' denifanstat has been granted Breakthrough Therapy designation by the FDA for the treatment of metabolic dysfunction-associated steatohepatitis (MASH (搜索)).
Phase 2b FASCINATE-2 study results, published in The Lancet Gastroenterology & Hepatology, demonstrated significant improvements in disease activity, MASH (搜索) resolution, and fibrosis with denifanstat.
Sagimet has completed end-of-Phase 2 interactions with the FDA and plans to initiate a Phase 3 program for denifanstat by the end of 2024.
Sagimet Biosciences Inc. (Nasdaq: SGMT) is set to advance its lead drug candidate, denifanstat, into Phase 3 development for metabolic dysfunction-associated steatohepatitis (MASH (搜索)) following successful end-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). The FDA has granted Breakthrough Therapy designation to denifanstat for the treatment of non-cirrhotic MASH with moderate to advanced liver fibrosis (F2-F3). This designation underscores the potential of denifanstat to address the significant unmet need for new therapies for this serious liver disease.
Phase 2b FASCINATE-2 Study
The Phase 2b FASCINATE-2 clinical trial results of denifanstat were published in The Lancet Gastroenterology & Hepatology. The study, titled "Denifanstat for the treatment of Metabolic-dysfunction Associated Steatohepatitis: a multicentre, double-blind, randomised, placebo-controlled, ph2b trial," highlighted that denifanstat treatment achieved statistically significant and clinically meaningful improvements in disease activity, MASH (搜索) resolution, and fibrosis.
Planned Phase 3 Program
Sagimet's Phase 3 program for denifanstat in MASH (搜索) is expected to commence by the end of 2024. The program will consist of two double-blind, placebo-controlled, multicenter registrational trials:
- FASCINATE-3: This trial will evaluate the efficacy and safety of denifanstat in patients with F2/F3 (non-cirrhotic) MASH. The primary endpoints will be liver biopsy assessments at 52 weeks, with Sagimet planning to seek accelerated approval in the US and Europe at this time. The trial will continue until the required number of clinical outcomes is reached, estimated at 3.5 years.
- FASCINIT: This trial will evaluate the efficacy and safety of denifanstat in patients with suspected or confirmed metabolic dysfunction-associated steatotic liver disease (MASLD)/MASH. The primary endpoints will be safety and tolerability at 52 weeks. Non-invasive biomarkers will be assessed as part of the secondary endpoints, with no liver biopsy endpoint.
The Phase 3 program is designed to include a minimum of 1,800 patients exposed to denifanstat.
Financial Position
As of September 30, 2024, Sagimet reported $170.0 million in cash, cash equivalents, and marketable securities, which is expected to fund operations through 2025. Research and development expenses for the three and nine months ended September 30, 2024, were $12.7 million and $24.2 million, respectively. The net loss for the three and nine months ended September 30, 2024, was $14.6 million and $29.4 million, respectively.
About Denifanstat
Denifanstat is an oral, once-daily, selective fatty acid synthase (FASN (搜索)) inhibitor in development for the treatment of MASH (搜索). It is designed to target dysfunctional metabolic and fibrotic pathways in diseases resulting from the overproduction of the fatty acid, palmitate.
MASH: A Growing Concern
Metabolic-dysfunction associated steatohepatitis (MASH (搜索)) is a progressive and severe liver disease estimated to impact over 115 million people worldwide. There is only one recently approved treatment in the United States and no currently approved treatments in Europe, highlighting the urgent need for new therapeutic options.