Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA panel backs unapproved peptides despite safety concerns
An FDA advisory committee has voted to recommend adding six unapproved peptides to the agency's 503A bulk compounding list, a move that could eventually allow compounding pharmacies to produce the substances for patient-specific prescriptions despite ongoing concerns over their safety and efficacy. In closely split votes, the panel backed BPC-157, KPV, TB-500, MOTS-c, epitalon, and semax for a range of proposed uses including wound healing, ulcerative colitis, weight loss, migraines, pain, and insomnia, while rejecting emideltide for opioid withdrawal and sleep disorders. FDA scientific reviewers had advised against adding any of the peptides, citing a lack of robust clinical evidence and unanswered safety questions, but supporters argued that regulated compounding would provide a safer alternative to the growing grey market, where the substances are widely sold as research-grade products despite not being approved for human use. The recommendation does not immediately legalize compounded versions of the peptides, with the FDA now needing to decide whether to initiate the rulemaking process to add them to the 503A bulk compounding list, a step that could take a year or more. – Bree Foster
J&J partners with Sail Biomedicines on in vivo CAR T
Johnson & Johnson announced a strategic collaboration with Sail Biomedicines to advance in vivo CAR T therapies for immune-mediated diseases, alongside an exclusive option to acquire the company for $2.58 billion. Unlike traditional cell therapies that require harvesting and re-engineering a patient's cells outside the body, Sail's platform is designed to reprogram immune cells directly inside the body, an approach the companies say could make CAR T manufacturing simpler and more scalable while delivering durable, potentially curative results. J&J will make initial payments of $785 million, including a $465 million equity investment, plus up to $140 million in milestone payments, with Sail contributing its endless RNA (eRNA) and targeted nanoparticle delivery technology. For drug developers, the deal signals growing pharma investment in overcoming the manufacturing and cost bottlenecks that have limited traditional ex vivo cell therapies, potentially widening patient access to CAR T approaches beyond oncology. — Andrea Corona
Altimmune’s GLP-1 drug shows positive results for alcohol use disorder
On Tuesday, Altimmune announced topline data from their Phase 2 RECLAIM trial showing that pemvidutide, an investigational balanced glucagon/GLP-1 dual receptor agonist, significantly reduced the number of heavy drinking days compared to placebo in patients with moderate to severe alcohol use disorder. Specifically, patients on the drug showed an average of 4.2 fewer heavy drinking days over 24 weeks. The drug was generally well-tolerated with mild to moderate adverse events. “As someone who has dedicated his career to understanding and treating alcohol use disorder, I recognize the significance of these findings, particularly given the current attention on the adverse health impact of heavy drinking,” said Henry Kranzler, Professor of Psychiatry at the University of Pennsylvania Perelman School of Medicine and Principal Investigator of the RECLAIM trial. Pemvidutude works by activating glucagon receptors and GLP-1 receptors in a 1:1 balance, and was developed to treat metabolic dysfunction-associated steatohepatitis along with alcohol use disorder and alcohol-associated liver disease. However, the drug raises hopes that the GLP-1 class of drugs more generally could help treat alcohol use disorder, which aligns well with recent data released in May from the Copenhagen University Hospital investigating semaglutide. – Allison Whitten
Pfizer's LITFULO meets primary endpoints in Phase 3 vitiligo trials
Pfizer reported topline results from two Phase 3 trials of LITFULO (ritlecitinib) in nonsegmental vitiligo, with both the 50 and 100 milligram once-daily doses meeting co-primary endpoints for facial and total body repigmentation over placebo at Week 52. TRANQUILLO and TRANQUILLO 2 together enrolled 2,174 patients across 271 sites worldwide, the largest Phase 3 program to date for an oral systemic therapy in this indication. LITFULO, a JAK3 and TEC family kinase inhibitor already approved for severe alopecia areata, showed a safety profile consistent with prior alopecia areata data, with no new signals reported. Pfizer plans to submit global regulatory filings seeking approval in adults with nonsegmental vitiligo, a condition that currently has no approved oral systemic treatment. For drug developers, the results illustrate how an approved kinase inhibitor's mechanism can be extended into adjacent autoimmune skin conditions, adding to a broader industry push toward oral systemic options in dermatology. – Andrea Corona
MapLight reports mixed results in Schizophrenia trial
In their Phase 2 ZEPHR trial, MapLight’s novel oral drug for schizophrenia, the Ml/M4 muscarinic agonist ML-007C-MA, successfully hit its primary endpoint — but only for the twice-daily version, not the once-daily dose. The primary endpoint was a meaningful reduction in symptoms based on the Positive and Negative Syndrome Scale at five weeks. Given the disappointing result for the once-daily version, shares of MapLight fell by more than 60 percent on Monday following the announcement, as it means that the drug is unlikely to out compete Bristol Myers Squibb’s twice-daily oral Cobenfy approved in 2024. However, MapLight highlighted in the press release that the once-daily dose did show a clinically meaningful improvement in cognitive performance that could be investigated further. “We observed a robust signal on a pre-specified secondary cognition endpoint that appears independent of antipsychotic effect. Cognitive impairment affects the majority of people living with schizophrenia and remains an area where no therapy has yet been approved,” said Chris Kroeger, cofounder and CEO of MapLight in the statement. – Allison Whitten
AstraZeneca reports mixed Phase 3 results across oncology and rare disease
AstraZeneca reported mixed late-stage clinical results, with its Claudin 18.2-targeting antibody-drug conjugate sonesitatug vedotin meeting its primary endpoint in patients with previously treated advanced gastric cancer, while rare disease therapy Ultomiris missed the primary endpoint in a Phase 3 trial for thrombotic microangiopathy following haematopoietic stem cell transplantation (HSCT-TMA). Top-line data from the CLARITY-Gastric01 study showed sonesitatug vedotin significantly extended overall survival in patients who had received at least two prior lines of therapy and also met a key secondary endpoint in an earlier-line population, although the trial failed to achieve statistical significance for progression-free survival. AstraZeneca said the antibody-drug conjugate, acquired through its 2023 licensing deal with KYM Biosciences, has blockbuster potential with projected peak sales of $3 billion to $5 billion. In contrast, Ultomiris failed to significantly improve event-free survival over placebo in adults and adolescents with HSCT-TMA, marking the latest setback for AstraZeneca's pipeline after a failed heart disease trial earlier this month and an FDA advisory committee voted against approving its breast cancer therapy in May. However, a separate Phase 3 study in paediatric patients showed clinically meaningful overall survival, prompting AstraZeneca to proceed with regulatory submissions for the paediatric indication while continuing discussions with health authorities regarding the adult program. – Bree Foster
