ClinicaSpace

The FDA’s new investigational new drug pilot program—one part of HHS’s broader clinical trial modernization initiatives—has lofty goals to expedite first-in-human trials of novel drugs, but experts say it won’t tip the scales much on its own.

The Phase 3 failure of AstraZeneca and Ionis Pharmaceuticals’ antisense therapy in transthyretin amyloid cardiomyopathy last month left the space reeling—and readjusting. BioSpace looks at five contenders and where they currently stand.

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.”

The FDA’s Cellular, Tissue and Gene Therapies advisory committee voted 10 to 3 in favor of Replimune’s immunotherapy for advanced melanoma in combination with Bristol Myers Squibb’s Opdivo, but trial design was a major sticking point as the panel deliberated.

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 will hold advisory committee meetings this week for previously rejected investigational therapies from Replimune and Capricor Therapeutics. Some industry leaders were surprised the meetings were called at all, while many have questioned the rationale behind them and other adcomms on deck.

Having started to publish CRLs without consulting the public, the FDA is under pressure to change course amid concerns it failed to openly and fully evaluate a complex issue.

After the trailblazing approval of Sanofi’s Tzield for stage 3 type 1 diabetes, several companies, including SAB Biotherapeutics and Eledon Pharmaceuticals, are vying for a spot in the landscape with innovative approaches—and experts say there’s room for all of them.

New mid-stage data from Biogen’s Ionis-partnered therapy diranersen served to both validate the tau hypothesis in Alzheimer’s disease and raise questions that could soon be elucidated by anti-tau candidates from Denali Therapeutics, Eisai and Eli Lilly.

Biogen touted an “unprecedented” drop in tau in a Phase 2 trial, backing the company’s decision to take diranersen to Phase 3 despite a missed primary endpoint and seemingly supporting the anti-tau approach.