Nov. 10 Quick Takes: BrainStorm falls on refusal-to-file letter from FDA

New data and analyses for stem cell therapy NurOwn from BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) failed to persuade FDA, which issued a refusal-to-file letter regarding the company’s BLA. Shares in the company fell $1.67 (42%) to $1.22 Thursday. After correcting a vendor’s error in the statistical analysis of NurOwn’s Phase III trial, BrainStorm said in August that it planned to resubmit its data package to FDA with a single statistically significant analysis in a prespecified subgroup, plus new biomarker data. FDA had previously sought to head off optimism for NurOwn’s approval in March 2021, when it took the unusual step of commenting on the results of a Phase III trial before taking any regulatory action. The next chance in ALS will come from tofersen; the Biogen Inc. (NASDAQ:BIIB) therapy is under FDA review with an April 25 PDUFA date.

AstraZeneca plc (LSE:AZN; NASDAQ:AZN) will continue to pursue the availability of Vaxzevria globally except in U.S., where it has abandoned its plans to submit a BLA to FDA for the COVID-19 vaccine. The U.S. market is already well supplied and demand for vaccines is declining, said CEO Pascal Soriot. Vaxzevria received conditional marketing authorization in the U.K. in December 2020 and the EU in January 2021; EMA converted the EU’s to a standard marketing authorization last month. Global revenue for the vaccine fell 83% to $173 million in 3Q22 compared with the prior year’s quarter. AZ’s COVID-19 antibody therapy Evusheld tixagevimab/cilgavimab brought in $537 million, bringing its total to $1.5 billion so far this year...