The promising but challenging indication of nonalcoholic steatohepatitis (NASH) hit headlines following two major developments.
Firstly, the frontrunner in the NASH space, Intercept Pharmaceuticals, Inc. ICPT, broke positive news at the start of this week. The company recently announced that the FDA accepted its New Drug Application (NDA) for obeticholic acid (OCA) seeking accelerated approval for the treatment of fibrosis due to NASH. The regulatory agency also granted Priority Review to the same. The authority assigned a Prescription Drug User Fee Act (PDUFA) target action date of Mar 26, 2020 for the NDA. It indicated to hold an advisory committee meeting for discussing the application. However, the conference date is yet to be finalized and the timeline for the NDA review is subject to change.
A potential approval of OCA for NASH will significantly boost Intercept’s prospects, given the market opportunity for patients with fibrosis due to NASH.
Concurrently, CymaBay Therapeutics, Inc. CBAY announced the termination of mid-stage study of seladelpar for NASH. The company stopped its phase IIb study of seladelpar in patients with NASH and its recently-initiated phase II study of the drug in patients with primary sclerosing cholangitis (PSC). Moreover, it is putting a hold on all studies of seladelpar in patients with primary biliary cholangitis (PBC). The decision to discontinue these studies was based on initial histological findings observed in the phase IIb study of seladelpar for NASH after data showed some patients experiencing liver damage. These planned, blinded, histological assessments of the first tranche of liver biopsies in the study demonstrated atypical histological outcomes including histology characterized as an interface hepatitis presentation, with or without biliary injury.
In June 2019, Conatus CNAT announced that top-line results from its ENCORE-LF clinical study of emricasan failed to meet its primary endpoint. Consequently, Conatus and partner Novartis mutually agreed to cease the collaboration agreement for the global development and commercialization of emricasan.
As a result, the spotlight is back on this space. The market for NASH holds promises and is expected to be tapped by most pharma/biotech bigwigs in the near future. It is poised to witness rapid growth unlike other lucrative yet saturated markets like cancer.
More on NASH
A chronic liver disease, NASH is caused by excessive fat accumulation in the liver or steatosis. Per statistics, it affected up to 15 million people in the United States causing inflammation, hepatocellular injury, progressive fibrosis and cirrhosis. The ailment is anticipated to be the leading reason for liver transplantation by 2020. Currently, NASH is the primary reason for liver transplants in people under 50 across the United States. Such patients also suffer obesity and type II diabetes. With no treatments presently approved to address this disease, the market scope is substantially huge and many companies are investing a major chunk of their R&D budget in the same.
Pharma Players With an Edge in NASH
The latest news from industry players in the NASH area underscores the complexity of treatments being developed. The winner so far in the race seems to be Intercept Pharmaceuticals, courtesy of the aforementioned news.
Let us take a look at some other players with promising candidates for the indication:
France-based Genfit’s lead candidate elafibranor, which is in late-stage studies, also buoys hope in this regard. The phase III study RESOLVE-IT is evaluating the efficacy and safety of elafibranor 120 mg versus placebo in patients with NASH and fibrosis. Most recently, the Data Safety Monitoring Board (DSMB) issued a positive recommendation for the continuation of the study evaluating elafibranor for NASH without any modifications. The 42-month DSMB safety review supports Genfit’s continuation of the RESOLVE-IT study. This DSMB sanction marks the seventh review with no safety signals that would justify modification or shelving of the study including review of safety data from patients enrolled in the study, who have received elafibranor for three years. An NDA is expected to be filed by 2020 end.
Allergan plc AGN too has an interesting candidate in its pipeline, cenicriviroc, for the treatment of NASH. The 2016 Tobira acquisition added the candidate to its profile. The candidate is being evaluated in a phase III study, AURORA.
Clinical-stage biopharmaceutical company Galmed Pharmaceuticals Ltd. GLMD is firmly eyeing the development of Aramchol, a liver targeted, oral SCD1 modulator. The company recently began a phase III/IV ARMOR, a double-blind, placebo-controlled, global study, to evaluate the efficacy and safety of Aramchol in patients with NASH and fibrosis. The company targets to complete enrollment by the second quarter of 2021 and report top-line results by the fourth quarter of 2022. Galmed currently carries a Zacks Rank #2 (Buy).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Madrigal Pharmaceuticals, Inc. MDGL, a clinical-stage biopharmaceutical company, is evaluating its lead candidate MGL-3196, (resmetirom), a first-in-class, orally administered, small-molecule, liver-directed, thyroid hormone receptor (THR) β-selective agonist, for NASH. Earlier in the year, the company commenced a phase III study on MGL-3196 for NASH.
Given the fact that NASH has a profitable market, quite a few companies inked deals to enter this space. Evidently, approval of any of the candidates will attract more players and investments in this space under healthcare.
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