SAN FRANCISCO, Calif. — 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to pegozafermin in patients with MASH. The PRIME status was supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin in patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).
“The EMA PRIME status further supports pegozafermin’s positioning as a leading FGF21 analog for the treatment of MASH, which has demonstrated robust anti-fibrotic and metabolic benefits as seen in our Phase 2b ENLIVEN trial,” said Rohan Palekar, Chief Executive Officer of 89bio. “This status recognizes the urgent need for effective treatment options for MASH patients with advanced fibrosis and underscores pegozafermin’s potential to address the targeted unmet medical need. We look forward to working closely with regulatory agencies as we continue to advance our Phase 3 clinical development program, ENLIGHTEN, aimed at potentially benefiting advanced MASH patients and MASH patients with compensated cirrhosis.”
The PRIME status is granted by the EMA to provide early and proactive support to developers of promising medicines that, based on clinical data, may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to provide multiple benefits so that these medicines can reach patients earlier including, enhanced interaction and early dialogue with EMA, guidance on the overall development plan and regulatory strategy, and the potential for accelerated assessment of the time of marketing authorization application.
About Metabolic dysfunction-associated steatohepatitis (MASH)
MASH, formerly known as nonalcoholic steatohepatitis (NASH), is a more advanced form of metabolic dysfunction-associated steatotic liver disease (MASLD) which is a chronic, progressive disease in which fat accumulates in the liver, ultimately leading to scarring or fibrosis. Fibrosis damages the liver and can lead to more severe liver-related complications, including cirrhosis, liver failure, and hepatocellular cancer (HCC) and is associated with increased risk for cardiovascular disease. In later stages, MASH can cause cirrhosis which increases the risk for serious intervention such as a liver transplant, with MASH being a leading cause of liver transplants among adults. Most people with MASH experience few or no symptoms until they’ve progressed to an advanced stage, and as a result, the disease often goes undetected for years, or even decades.
About The ENLIGHTEN Program
The ENLIGHTEN program is comprised of two Phase 3 global, multi-center, randomized, double-blind, placebo-controlled trials, evaluating the efficacy and safety of pegozafermin in patients with MASH. The ENLIGHTEN-Fibrosis trial, the first of two Phase 3 trials in the program, will enroll approximately 1,000 patients with non-cirrhotic MASH (fibrosis stage F2-F3) to evaluate the efficacy and safety of pegozafermin. The co-primary endpoints, for which demonstration of an effect on each is needed to support regulatory approval, measured at week 52 are a one-point improvement in fibrosis with no worsening of MASH and MASH resolution with no worsening of fibrosis, assessed at week 52. ENLIGHTEN-Cirrhosis, the second of the two Phase 3 trials in the program, will evaluate the efficacy and safety of pegozafermin in MASH patients with compensated cirrhosis (F4).
About ENLIVEN
ENLIVEN was a multicenter, randomized, double-blind, placebo-controlled Phase 2b trial designed to evaluate the safety and efficacy of weekly or every-two-week dosing of pegozafermin for the treatment of patients with biopsy confirmed MASH and NAS ≥ 4 for 48 weeks. In the trial, 192 patients were dosed with pegozafermin 15mg QW, 30mg QW and 44mg Q2W, or placebo. Primary outcomes measured were proportion of participants with resolution of MASH without worsening of fibrosis and proportion of participants with ≥1 stage decrease in fibrosis stage with no worsening of MASH at week 24. Secondary measures included change from baseline in liver fat, liver enzymes, noninvasive markers of liver fibrosis, glycemic control, lipoproteins, and body weight as well as safety and tolerability measures. Patients who entered the blinded extension phase were subsequently treated for an additional 24 weeks for a total treatment period of 48 weeks. Some patients who were on placebo (n=19) were re-randomized to receive pegozafermin in the extension phase. Key endpoints in the extension phase include liver fat and non-invasive markers of liver fibrosis and inflammation. ENLIVEN achieved high statistical significance on primary histology endpoints with 30mg QW and 44mg Q2W dosing at week 24 and the results were published in the New England Journal of Medicine. To learn more about the clinical trial, visit clinicaltrials.gov: NCT04929483.
About pegozafermin
Pegozafermin is a specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21) being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH) and severe hypertriglyceridemia (SHTG). FGF21 is an endogenous hormone that has broad effects such as regulating energy expenditure, glucose and lipid metabolism. In clinical trials, pegozafermin has demonstrated direct anti-fibrotic and anti-inflammatory effects on the liver, as well as reduced triglyceride levels, improved insulin resistance and glycemic control, and continued to demonstrate a favorable safety and tolerability profile. The FDA granted pegozafermin Breakthrough Therapy designation (BTD) for the treatment of MASH with fibrosis. Pegozafermin is advancing into the Phase 3 ENLIGHTEN trial program for MASH and is being studied in the Phase 3 ENTRUST trial for SHTG.
About 89bio
89bio is a clinical-stage biopharmaceutical company dedicated to the development of best-in-class therapies for patients with liver and cardiometabolic diseases who lack optimal treatment options. The company is focused on rapidly advancing its lead candidate, pegozafermin, through clinical development for the treatment of metabolic dysfunction-associated steatohepatitis (MASH) and severe hypertriglyceridemia (SHTG). Pegozafermin is a specifically engineered, potentially best-in-class fibroblast growth factor 21 (FGF21) analog with unique glycoPEGylated technology that optimizes biological activity through an extended half-life. The company is headquartered in San Francisco.
Investor Contacts
Annie Chang
89bio, Inc.
[email protected]
PJ Kelleher
LifeSci Advisors, LLC
+1-617-430-7579
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Media Contact
Sheryl Seapy
Real Chemistry
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