Pfizer exits Sangamo pact; Roche, Ideaya strike ADC deals

Happy New Year! We hope you had a restful holiday; If you took a break from keeping up with biotech news, we’ve got you covered with a brief rundown of the most important updates from the past week and a half.

Pfizer won’t move forward with asking regulators to approve a gene therapy for hemophilia A and instead will hand back rights to development partner Sangamo Therapeutics in a blow to the California-based biotechnology company. In a Dec. 30 statement, Sangamo CEO Sandy Macrae said his company was “surprised and extremely disappointed” by Pfizer’s decision, which comes some five months after the treatment met its goal in a Phase 3 study of people with the inherited bleeding disorder. Sangamo plans to search for an “optimal path” forward, which could include partnering with another company. Pfizer has steadily retreated from gene therapy, although it sells a hemophilia B gene therapy called Beqvez that it obtained from Spark Therapeutics. — Ned Pagliarulo

Roche and Ideaya Biosciences acquired rights to similar, experimental lung cancer drugs in separate deals with China-based biotechnology companies. First, Ideaya on Monday paid Hengrui Pharma $75 million upfront for an antibody-drug conjugate that targets the protein delta-like ligand 3, or DLL3, and is currently in preclinical testing. Roche followed on Thursday by paying Innovent Biologics $80 million for a DLL3-targeting ADC that began a Phase 1 trial last month. Both drugs are being evaluated against small-cell lung cancer, a more aggressive and less common form of the disease. — Ben Fidler

After many years of effort, Novartis has obtained positive results from a Phase 3 study for a form of its spinal muscular atrophy gene therapy Zolgensma that’s delivered via spinal injection rather than intravenous infusion. Data from the study, called Steer, showed treatment improved motor abilities in people with the neuromuscular disease who were two years of age or older and could still sit but had never walked independently. U.S. regulators suspended testing of the intrathecal formulation in late 2019 over safety concerns, but in August 2021 permitted Novartis to begin the Steer study. Novartis plans to share the new data with health authorities to support approval of the gene therapy in a broader range of people with spinal muscular atrophy. — Ned Pagliarulo

BioNTech will pay the National Institutes of Health $792 million to settle disputes over use of patented technology to develop the mRNA COVID-19 vaccine Comirnaty, marketed by Pfizer, according to a securities filing.The payment will consist of $750 million worth of claimed royalties for the years 2020 through 2023 and $42 million for an amended license agreement that sets new royalty terms. Separately, BioNTech said it has settled with the University of Pennsylvania over patents related to technology used to develop Comirnaty, paying up to $467 million to Penn. Pfizer will reimburse BioNTech $365 million related to the NIH settlement and up to $170 million related to the Penn settlement. — Jonathan Gardner

Axsome Therapeutics intends to ask regulators for approval of a combination drug it’s developed to treat agitation caused by Alzheimer’s disease, despite mixed results from the company’s latest late-stage trials. In one Phase 3 study, Axsome’s drug, called AXS-05, lowered the risk of relapse in agitation versus placebo. However, AXS-05 missed its goal in another trial. Drawing on positive data from two prior studies, Axsome said it will submit a U.S. approval application in the second half of the year, which analysts expect may still be granted. — Ned Pagliarulo

Sanofi and partner SK Bioscience are expanding their partnership as they begin Phase 3 testing of a next-generation pneumococcal conjugate vaccine in children. The shot is designed to protect against 21 serotypes of Streptococcus pneumoniae and, according to Sanofi, is the first of its type targeting more than 20 to enter a Phase 3 trial in infants. The planned trial program will include over 7,700 infants, children and adolescents. If successful, the companies’ shot could eventually compete with Pfizer’s Prevnar 20, which in 2023 received a green light from the Food and Drug Administration for use in infants and children. Sanofi and SK Bioscience will co-fund research and development costs, while SK Bioscience will receive a 50 million euros upfront payment from Sanofi. — Delilah Alvarado

The Food and Drug Administration has approved Hikma Pharmaceuticals’ generic version of Novo Nordisk’s daily GLP-1 shot for diabetes, Victoza. First approved in 2010, Victoza once was the biggest selling drug in the class. But by 2023 its sales had declined to 8.7 billion kroner, or about $1.3 billion, while revenue from the more potent weekly GLP-1 shot Ozempic has grown since its 2017 launch. Teva Pharmaceutical launched an authorized generic of Victoza in June 2024. — Jonathan Gardner

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