The FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outline ambitious plans in second quarter presentations; Sarepta taps Michael Severino as its new CEO; and Amgen launches its official defense of Tavneos.
It’s all about advisory committee meetings and Q2 earnings this week in biopharma.
The FDA published briefing documents regarding upcoming advisory committee meetings for Capricor Therapeutics’ Duchenne muscular dystrophy therapy and Replimune’s advanced melanoma drug—and it appears that approvals are far from certain for both. The FDA disagrees with Capricor’s claim that its cell therapy met the primary endpoints in a Phase 3 trial, while the agency continues to have major qualms with Replimune’s use of a single-arm study to support its candidate.
More generally, some regulatory experts are surprised that the FDA has resumed adcomms—which were largely paused under former Commissioner Marty Makary—while the agency is still staffing back up, while others have questioned the rationale behind the meetings.
Meanwhile, second quarter earnings continue to roll in. GSK’s stock popped Tuesday as the U.K.-based company unveiled a $2.5 billion cost-cutting plan. And on Monday, AstraZeneca CEO Pascal Soriot said the company is “very much on track” to hit its $80 billion by 2030 revenue target despite the recent Phase 3 failure of Wainua in ATTR-CM. Still to report this week are Sanofi, BMS, Moderna and more. And while the focus usually lands on the Big Pharmas, several plucky biotechs are also beginning to report—with some potentially with big launches ahead.
Also on the business front, Sarepta has a new leader. Michael Severino, who most recently served as CEO of Flagship Pioneering’s gene writing biotech Tessera, took the reins of the muscular dystrophy-focused company on Tuesday. Severino has a tough job ahead of him as Sarepta tries to secure full FDA approval for its exon skippers Amondys 45 and Vyondys 53 after both failed their confirmatory trials.
Finally, Amgen has launched its official defense of Tavneos, the rare disease drug the FDA has recommended for withdrawal.
