Cell therapy

The FDA held a public forum on Monday to discuss the future of psychedelic therapeutics, followed by a meeting on Wednesday regarding recommendations for reauthorizing the Prescription Drug User Fee Act the day after launching its TrialBlazer initiative. Meanwhile, Definium Therapeutics notched another late-stage trial win for its LSD drug, and Novartis continues to reel from the company’s triple asset stumble.

Blood centers have the donors, resources and community footprint drug sponsors need to solve supply chain issues around cell and gene therapies.

In this episode of Denatured, sponsored by SK pharmteco, Jennifer Smith-Parker speaks to Arvind Singh, vice president and global head of supply chain. In this episode, we explore how CDMOs are reshaping supply‑chain strategy for a new generation of complex modalities.

September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.

Bristol Myers Squibb is positioning arlo-cel for quadruple-class exposed patients, which could help reduce competition with an upcoming multiple myeloma CAR T therapy from Gilead Sciences, according to BMO Capital Markets.

Novartis hits pause on several CAR T trials after three patient deaths, followed by Bristol Myers Squibb after separate safety signals; Revolution Medicines scores early FDA approval for groundbreaking pancreatic cancer drug; BioNTech suspends a mid-stage trial of its mRNA cancer immunotherapy after a futility analysis; and nine mid-sized biopharma companies join their big pharma peers in President Donald Trump’s Most Favored Nation drug pricing scheme.

After downsizing by 50% last year, Arsenal Biosciences is again laying off employees. This time, 99 people are getting cut as part of a strategic shift to focus the company’s R&D efforts on in vivo CAR T therapies.

Novartis suspended multiple studies of a CAR T candidate after three patients who received the therapy suffered fatal cases of immune effector cell-associated hemophagocytic syndrome. BMS, meanwhile, paused some CAR T trials after separately observing “transient and reversible inflammatory events” with its therapy.

Thanks in part to the revolution cell therapy brought to treating blood cancers, the modality is now showing promise in some autoimmune applications. This expansion is poised to yield even wider benefits.

Biopharmas developing CAR T therapies for autoimmune indications face major barriers to maximizing the market. Advances in the provider ecosystem and clinical infrastructure, as well as access and education, will be key to launching at scale.