Friday, September 22, 2023
89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company specializing in innovative therapies for liver and cardiometabolic diseases, has achieved Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for pegozafermin's application in treating nonalcoholic steatohepatitis (NASH).
Rohan Palekar, the CEO of 89bio, expressed excitement over the FDA's recognition of pegozafermin with Breakthrough Therapy Designation. He expects that this designation will streamline the finalization of the Phase 3 development strategy for NASH, with discussions planned with regulatory agencies in the fourth quarter of 2023. Pegozafermin is positioned as a leading FGF21 analog treatment option, offering compelling histology data, excellent tolerability, and convenient dosing. It holds promise for addressing the treatment needs of F2/3 patients as well as compensated cirrhotic (F4) patients. The benefits of this designation encompass more comprehensive guidance from the FDA on the development program, the opportunity to explore innovative development plan options, access to a scientific liaison, and potential eligibility for Priority Review.
The BTD designation is grounded in data from the ENLIVEN Phase 2b trial of pegozafermin in NASH patients. In this study, both the 44mg every-two-week (Q2W) and 30mg weekly (QW) doses met the primary histology endpoints outlined in the FDA's guidance on endpoints and statistical analysis, achieving high statistical significance. Additionally, these dose groups demonstrated significant and clinically meaningful improvements in liver fat, non-invasive markers of liver fibrosis and inflammation, as well as other metabolic and lipid markers. The ENLIVEN trial also included biopsy-confirmed F4 patients, and in a descriptive analysis, 45% of pegozafermin-treated patients experienced at least one-stage improvement in liver fibrosis with no worsening of NASH by week 24. Overall, pegozafermin exhibited favorable tolerability and a safety profile consistent with previous studies.
BTD is an FDA program designed to expedite the development and review of product candidates intended for serious or life-threatening conditions. To qualify for this designation, preliminary clinical evidence must suggest that the product candidate may demonstrate substantial improvement over currently available options on at least one clinically significant endpoint.
Pegozafermin is a specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21) developed for the treatment of non-alcoholic steatohepatitis (NASH) and severe hypertriglyceridemia (SHTG). FGF21 is a hormone that plays a role in modulating key factors in lipid metabolism and NASH, such as triglyceride reduction, glycemic control, steatosis, inflammation, and fibrosis. Pegozafermin has been engineered using glycoPEGylation technology to extend its half-life while maintaining its potency.