FDA Investigates Elevidys Safety as Industry Sees Mixed Developments

Sarepta's Gene Therapy Under Scrutiny Following Patient Deaths

The Food and Drug Administration (FDA) has launched an investigation into two patient deaths associated with Sarepta Therapeutics' gene therapy, Elevidys, for Duchenne muscular dystrophy. Both patients, who were unable to walk due to disease progression, died of acute liver failure this year after receiving the treatment. The FDA stated that the deaths appear to be treatment-related and will evaluate the need for further regulatory action.

Sarepta has treated over 900 patients with Elevidys in clinical and commercial settings. The company previously announced plans to convene an expert committee to develop an improved immunosuppressive drug regimen to enhance safety for non-ambulatory patients.

Capricor's Deramiocel Faces Uncertain Future as FDA Cancels Advisory Meeting

Capricor Therapeutics confirmed that the FDA has cancelled a planned advisory committee meeting to discuss the approval application for deramiocel, another Duchenne muscular dystrophy treatment. The decision comes amid reports of skepticism from Vinay Prasad, the new head of the FDA office overseeing such therapies.

Despite the cancellation, Capricor remains confident in its submission, citing successful regulatory milestones to date, including a pre-license inspection and a mid-cycle review without major issues. The FDA's decision on deramiocel is expected by August 31.

Nektar and AstraZeneca See Positive Developments in Atopic Dermatitis and Lung Cancer

Nektar Therapeutics' shares surged over 150% following positive mid-stage trial results for rezpegaldesleukin in atopic dermatitis. The study, involving 393 patients with moderate-to-severe eczema, met its primary and secondary endpoints, demonstrating significant improvements in skin clearance and itch reduction compared to placebo.

Meanwhile, AstraZeneca and Daiichi Sankyo received FDA approval for Datroway, an antibody-drug conjugate, to treat certain patients with locally advanced or metastatic non-small cell lung cancer. The approval is limited to patients with EGFR mutations who have previously received EGFR-targeted treatment and chemotherapy. Datroway is the first TROP2-targeted ADC approved for lung cancer.

Industry Deals and Mergers Shape Pharmaceutical Landscape

Revolution Medicines secured up to $2 billion in funding through a rights deal with Royalty Pharma and a term loan. The agreement provides Revolution with immediate access to $250 million in exchange for single-digit royalties on potential future sales of its cancer drug daraxonrasib, which targets RAS mutations and is currently in Phase 3 testing for pancreatic and lung tumors.

In a strategic shift, Carisma Therapeutics announced a merger with OrthoCellix after facing challenges in developing cell therapies for cancer and immune diseases. The newly formed company will focus on regenerative cell therapies, including OrthoCellix's knee cartilage implant technology slated for Phase 3 testing later this year. Carisma stockholders will retain a 10% ownership in the merged entity and receive contingent value rights for potential payments from the sale of Carisma's cell therapy assets.