Shares of Applied Therapeutics Inc. (NASDAQ:APLT) dropped 36% Jan. 5 on news that its oral aldose reductase inhibitor, AT-001 (caficrestat), failed to reach statistical significance in a phase III study in patients with diabetic cardiomyopathy. The New York-based firm, however, pointed to positive trends in the Arise-HF study that could offer a path forward, albeit via a partner.
After discussions with the FDA, Applied Therapeutics Inc. said it will again delay an NDA filing for its galactosemia treatment, AT-007, until it has more time to discuss the filing with the agency.
Jaguar Gene Therapy LLC, a startup reuniting former Avexis Inc. executives to develop a portfolio of potential treatments for severe genetic diseases, announced its public debut Feb. 25 with more than $40 million in series A financing from co-creator Deerfield Management.
Applied Therapeutics Inc. (ATI) CEO Shoshana Shendelman said positive top-line findings with aldose reductase inhibitor AT-007 in galactosemia were “the data we needed, and we’re going to move as quickly as we can to get in and speak to the FDA about this, not only to firm up our NDA filing but also to get into the pediatric study.”