Rare diseases
Seven months after launching with $16 million in seed financing, Jennifer Doudna’s Aurora Therapeutics is pivoting away from its lead indication, phenylketonuria, owing to growing competition in the field.
The industry has been abuzz since the weekend when talks of a potential mega-merger between Bristol Myers Squibb and AstraZeneca were reported. Meanwhile, Pfizer, Merck and others release second quarter earnings, with some disclosing pipeline discontinuations and cost-cutting measures. Finally, all eyes turn to the FDA in the wake of heated advisory committee meetings for Capricor Therapeutics and Replimune.
After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior motive” and “biased intentions.”
Alnylam’s revenue cut “may create a near term credibility overhang and could “raise questions on peak sales potential, even if there’s tailwinds in the competitive landscape,” according to Stifel.
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Replimune and Capricor.
During a perplexing advisory committee meeting in which FDA reviewers focused largely on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, experts pointed to “very fragile” data. The committee voted 9-3 against approval of deramiocel.
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.
Oak Hill Bio is developing the antisense therapy rugonersen, which it licensed from Roche in 2025, for Angelman syndrome.
In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor’s assertion that a Phase 3 trial met its primary outcome measures.
After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
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