Jan. 7 Quick Takes: Sarepta tumbles after hours on DMD miss; plus InnoCare, AstraZeneca, Astellas and Autolus
Sarepta microdystrophin gene therapy misses endpoint
Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell 50% to $84.45 in after-hours trading after microdystrophin gene therapy SRP-9001 missed the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment in 41 patients with Duchenne muscular dystrophy. The study did meet the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment.
China’s NMPA approves InnoCare’s orelabrutinib
InnoCare Pharma Ltd. (HKEX:09969) said Wednesday that China National Medical Products Administration approved two NDAs for Btk inhibitor orelabrutinib: to treat patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL); and to treat patients with relapsed/refractory mantle cell lymphoma (MCL).
Priority review for AZ’s Farxiga for CKD, Astellas’ mirabegron
AstraZeneca plc (LSE:AZN; NASDAQ:AZN) said FDA granted priority review to an sNDA for SGLT2 inhibitor Farxiga dapagliflozin to treat chronic kidney disease, with a PDUFA date in 2Q21. Separately, Astellas Pharma Inc. (Tokyo:4503) said the agency granted priority review to two applications for mirabegron: an NDA for mirabegron for oral suspension and an sNDA for Myrbetriq mirabegron tablets, both to treat neurogenic detrusor overactivity in pediatric patients three years or older. Both applications have a PDUFA date of March 28...