ARTICLE | Regulation

May 17 Quick Takes: Intercept falls on negative tone of FDA briefing docs

Plus: Siren, Sania debut and updates from Aitia, PTC, enGene, Ardelyx, Akero and Satellos   

May 17, 2023 11:42 PM UTC

Shares of Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) fell $2.38 (15%) to $13.83 on Thursday on concerns of both safety and efficacy of non-alcoholic steatohepatitis (NASH) candidate obeticholic acid (OCA) raised by FDA in briefing documents released ahead of Friday’s meeting of the Gastrointestinal Drugs Advisory Committee (GIDAC). The documents highlight the fact that OCA met only one of two primary endpoints in the Phase III study, raised questions about the histological markers as surrogate endpoints in the indication, and provide a list of adverse events — including a worsening of co-morbidities that exist in the indication and the introduction of new co-morbidities such as excess risk of diabetes, dyslipidemia, acute kidney injury, and hepatotoxicity. The committee will vote on whether the benefits of a 25 mg dose outweigh the risks for Stage 2 and 3 NASH patients, and whether accelerated approval or traditional approval is appropriate.

Siren Biotechnology Inc. debuted with preclinical data at the American Society of Gene and Cell Therapy conference, suggesting that AAV-mediated delivery of cytokines could help treat high-grade gliomas. Intratumoral injection of the gene therapy vectors, which avoids the delivery challenges of achieving high blood brain barrier penetration with systemic delivery and the inability to frequently dose directly into the tumor, reduced tumor size in mice. The San Francisco-based biotech has not disclosed funding information...