Sept. 13 Quick Takes: Madrigal gets March PDUFA date for NASH therapy
Plus: Rocket parlays gene therapy update into $175M raise and updates from DalCor, Mironid, Crinetics, Shionogi, Hasten, Dewpoint and Orchid
FDA granted priority review to resmetirom from Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL), which is seeking accelerated approval of the THRB agonist to treat non-alcoholic steatohepatitis with liver fibrosis. The company expects a decision by its PDUFA date of March 14, 2024, and said FDA currently has no plans to convene an advisory committee during the review. With strong data from a Phase III trial, the company could be first to market in NASH; FXR agonist obeticholic acid from Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) received a complete response letter in June.
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) raised $175 million in a follow-on priced early Wednesday, hours after the biotech said it had reached alignment with FDA on the design of a pivotal Phase II trial of gene therapy RP-A501 to treat Danon disease, a cardiomyopathy. The company expects to dose 12 patients to assess the single-infusion, adeno-associated virus serotype 9 (AAV9)-based therapy; the study’s dual primary endpoint will measure improvements in LAMP2 expression and reductions in left ventricular mass. Moments after releasing its news after market hours Tuesday, Rocket proposed to raise $150 million; it priced 7.8 million shares at $16, plus 3.1 million warrants at $15.99, representing a 5% premium over its closing price of $15.29. Rocket was up 39% Wednesday to close at $21.23...