FDA Endorses Ammonia as Key Pediatric Endpoint; Arcturus Adds ARCT-2601 to Phase 2 Trial
ARCT has more than doubled off its 52-week low of $5.5.
Summary
Arcturus will add ARCT-2601 to its Phase 2 ARCT-810 trial after an FDA meeting, with dosing of 3-6 OTCD patients planned near year-end to assess safety, PK, and biomarkers. The FDA indicated ammonia is a key pediatric endpoint while glutamine may be acceptable for adults, prompting longer metabolic stability studies. The company also unveiled liver-focused preclinical programs for PKU and gout, with PKU data showing phenylalanine control in mice and primates. This follows the September 23 interim Phase 2 data for ARCT-810 and the myNEO acquisition, reinforcing the pipeline's momentum. The FDA's endpoint guidance de-risks the pediatric development path, a meaningful regulatory clarity for a company with a $394M market cap.
At the time of this announcement, ARCT was trading at $13.80 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $393.7M. The 52-week trading range was $5.50 to $24.17. This news item was assessed with positive market sentiment and an importance score of 7 out of 10. Source: Wiseek News.