Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Moderna and Merck say personalized melanoma cancer vaccine worked in late-stage trial
Moderna’s mRNA cancer vaccine for melanoma coupled with Merck’s prized immunotherapy, Keytruda, successfully reduced the risk of recurrence and metastasis, according to press releases from the companies. No data was shared yet, and the statement says that the results will be presented at an upcoming international meeting. The announcement alone was enough to send shares of Moderna’s stock soaring up to 177 percent on Wednesday as the news ushered in the first time that an mRNA-based cancer vaccine has shown positive results, and the first time that a combination treatment with Keytruda was more efficacious than Keytruda alone. “These Phase 3 findings represent a pivotal moment for the field of cancer research. For many years, the idea of creating an mRNA treatment designed specifically for an individual patient's cancer was aspirational. We are now helping turn that vision into a reality,” said Stéphane Bancel, CEO of Moderna, in the statement. – Allison Whitten
Trump nominates Heidi Overton as next FDA commissioner
President Trump nominated Heidi Overton, currently Deputy Assistant to the President for Domestic Policy at the White House, to lead the FDA, filling the vacancy left when Marty Makary resigned in May after 13 months in the role. Overton, a trained surgeon, previously worked at the conservative America First Policy Institute, where she wrote papers opposing gender-affirming care for minors and questioned the safety of medication abortion, including its availability via telehealth. At an August 10 press conference, she called Trump's executive order pushing to separate the combined measles, mumps, and rubella vaccine into individual shots "a historic action" and said parents should have the option to space them out, a position public health experts, including former FDA Principal Deputy Commissioner Joshua Sharfstein, say lacks scientific support and could raise manufacturing costs and increase missed doses. Overton still needs Senate confirmation; Senator Patty Murray has said she will vote no, while Senator Bill Cassidy, a physician, voiced concerns without committing to a position. Whoever runs the FDA sets the tone for every review, inspection, and guidance document a drug developer touches. Overton's record suggests an agency chief more willing to bend toward political conviction than scientific consensus, which is precisely the tension Sharfstein says defines the job: smart enough to understand the science, tough enough to say no anyway. – Andrea Corona
New gene therapy approval marks first treatment for glycogen storage disease
On Wednesday, the FDA approved Ultragenyx Pharmaceuticals’ gene therapy, Genclycos, for the treatment of glycogen storage disease type Ia (GSDIa). The rare disease is caused by a mutation in the gene that encodes for the glucose-6-phosphate enzyme, which turns stored glycogen into glucose. Ultragenyx’ new therapy employs an AAV to deliver the mutated gene. Results from their Phase 3 trial showed that the therapy reduced daily cornstarch intake — which patients usually eat to manage disease — and maintained glycemic control. “The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. … As our first gene therapy approval, GENGLYCOS represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases,” said Eric Crombez, Chief Medical Officer at Ultragenyx, in the press release. – Allison Whitten
Sanofi cuts 229 jobs in first major move under new CEO Garijo
Sanofi is laying off 229 employees from Blueprint Medicines, the Cambridge, Massachusetts rare disease biotech it acquired for $9.1 billion dollars in 2025, marking the first major workforce reduction disclosed under new CEO Belén Garijo. The cuts, revealed in a state WARN notice, will happen between October 2026 and June 2027 and affect roughly a third of Blueprint's pre-acquisition headcount of 682 full-time employees. Alongside the layoffs, Sanofi is closing two legacy Blueprint sites in Cambridge, including the biotech's former global headquarters, with remaining staff relocating to Sanofi's Cambridge Crossing campus. A company spokesperson framed the changes as organizational decisions to align structure with long-term business priorities, adding that many Blueprint employees are joining Sanofi directly. The deal gave Sanofi rights to Ayvakit (avapritinib), the only approved treatment for advanced systemic mastocytosis, along with a pipeline of additional KIT inhibitor candidates. Several former Blueprint leaders, including its previous CEO and Chief Scientific Officer, had already departed before this round of cuts. Well-regarded rare disease acquisitions often lead to workforce and site consolidation once integration begins. Since 2025, at least six major pharma acquisitions — Gilead/Arcellx, BioMarin/Amicus, Novartis/Tourmaline Bio, BioNTech/CureVac, Biogen/Apellis, and Sanofi/Blueprint Medicines — have been followed by disclosed layoffs at the acquired biotech. – Andrea Corona
FDA approves Regeneron’s drug for rare bone disorder
Twenty years after Regeneron scientists discovered that they could target the Activin A protein to treat the rare bone disorder fibrodysplasia ossificans progressiva (FOP), the FDA approved their monoclonal antibody, Pasatru, for the condition. The condition causes non-bone tissues — like ligament and muscles — to gradually turn into bone. The approval was based on positive results from the Phase 3 OPTIMA trial that showed both doses led to a 90 and 94 percent reduction, respectively, in new bone lesions. “The approval of Pasatru is the culmination of decades of pioneering research that Regeneron has pursued alongside the FOP community, rooted in our discovery of the role that Activin A plays in driving this disease,” said George Yancopoulos, President and Chief Scientific Officer at Regeneron. – Allison Whitten
FDA issues fourth CRL to Xspray's leukemia drug Dasynoc
Xspray Pharma received a Complete Response Letter (CRL) from the FDA for Dasynoc, its optimized dasatinib formulation for chronic myeloid leukemia and acute lymphoblastic leukemia, marking the fourth rejection for the candidate since 2023. The letter centers entirely on manufacturing rather than the drug itself: The FDA flagged previously communicated GMP observations at Xspray's Italian contract manufacturer, NerPharMa, and requested additional consecutive batch data at commercial scale, while explicitly raising no questions about Dasynoc's clinical data, bioequivalence, or stability. Xspray also noted that the medication-error risk cited in earlier CRLs has now been resolved. NerPharMa has told the FDA its remediation work at the facility is complete, though the agency has not yet decided whether a reinspection is needed to close out its assessment. Xspray plans to prioritize production of the requested commercial-scale batches and resubmit the application before the end of 2026 to secure a new PDUFA date. The repeated setback is a reminder of how heavily a novel drug candidate's approval timeline can hinge on a third-party manufacturer's compliance record, even when the sponsor's own clinical package is not in question. – Andrea Corona
