ClinicaSpace

Many of the FDA’s decisions this quarter involve applications that have previously been delayed, declined or outright rejected, including one for an mRNA vaccine that became the center of controversy earlier this year.

A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.

Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.

The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.

When the variance can’t be modeled, even disciplined biotech investors stop deploying. Here’s the cheapest fix for biotech’s investability problem.

The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven.

A new report from RBC Capital Markets lists Biotechnology Innovation Organization chief executive John Crowley and military physician Heidi Overton as potential candidates for the role of FDA commissioner—in addition to the agency’s current acting leader, top food regulator Kyle Diamantas.

Among the health department’s efforts is an expedited investigational new drug pilot program that would leverage collaborations with U.S. research institutions to reduce early trial timelines by as much as 12 months.

Results for Definium Therapeutics psychedelic candidate for major depressive disorder “exceeded expectations,” according to Stifel, while Jefferies called the efficacy data “profound.”

Nura Bio will use the series B financing to carry two neuroprotective drug candidates through early- to mid-stage clinical studies, one targeting ALS and the other for a broader neurological profile.