Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
New TrumpRx direct-to-consumer website announced with Pfizer on board
On Tuesday, President Trump announced a new initiative that aims to bring down prices of drugs for Americans, referred to as “most-favored-nation” pricing. The new plans also revealed a new website, TrumpRx, that will sell drugs directly to American consumers at discounted prices. Pfizer became the first drugmaker to agree to sell their drugs to Medicaid at the lowest price available to peer countries, and on TrumpRx at a discounted rate with savings that average 50 percent. Yet, because most Americans fill their prescriptions through insurance, health policy expert Stacie Dusetzina of Vanderbilt University told The New York Times of the direct-to-consumer route, “I don’t think that they are going to help many people.” As part of the deal, Pfizer also receives a three-year grace period in which the company won’t be subjected to tariffs, contingent upon further manufacturing investments in the US. – Allison Whitten
FDA issues CRL for Sentynl’s CUTX-101 NDA over manufacturing issues
Sentynl Therapeutics, a Zydus Lifesciences company, reported this week that the FDA has issued a Complete Response Letter (CRL) for its NDA of CUTX-101, a copper histidinate injection for Menkes disease in pediatric patients. Menkes disease is a rare, X-linked genetic disorder caused by mutations in the ATP7A gene, which disrupts copper absorption and transport in the body. It leads to severe neurological impairment, connective tissue abnormalities, and often early death in childhood if untreated. The letter focused on current good manufacturing practice (cGMP) inspection findings at the manufacturing facility but raised no concerns about the therapy’s safety or efficacy, which has shown survival benefits when used early. Zydus has responded to the FDA’s re-inspection and is awaiting the Establishment Inspection Report, while Sentynl plans to meet with the agency to discuss resubmission. The company stressed its commitment to advancing CUTX-101 as a potential treatment option for children living with this rare and life-threatening disorder. – Andrea Corona
Trump moves to expand AI use in pediatric cancer research amid broader cuts
Donald Trump signed an executive order on Tuesday to expand the use of artificial intelligence (AI) in childhood cancer research, pledging an additional $50 million in grants to accelerate new diagnostics and treatments. The order doubles the National Institutes of Health’s investment in the Childhood Cancer Data Initiative, a 10-year, $500 million program launched during Trump’s first term to collect and analyze data on every child, adolescent, and young adult diagnosed with cancer in the US. The funding will support competitive research grants and encourage the application of AI to improve diagnostics, treatments, and prevention strategies. Cancer rates among children in the US have been increasing since the 1970s, and advocates note that pediatric cancers have historically received less than 5 percent of the federal cancer research budget. While the new investment is seen as a step forward, critics argue it is modest compared with broader proposals under the Trump administration to cut billions from the National Cancer Institute’s overall funding. – Bree Foster
Takeda to discontinue cell therapy programs
Takeda announced it will discontinue its internal cell therapy research as part of a broader portfolio prioritization and seek external partners to carry forward its platform technologies and clinic-ready programs. The company said it will concentrate near-term investment on other modalities such as small molecules, biologics, and antibody-drug conjugates, while incorporating insights gained from its cell therapy work into preclinical efforts. Takeda expects to record an impairment loss of about 58 billion Japanese yen (JPY; $0.4 billion) in the second quarter of FY2025, largely tied to intangible assets from its gamma delta T cell therapy platform, and will update financial guidance as needed in its October 30 results announcement. – Andrea Corona
New technique lets scientists see the earliest signs of Parkinson’s
For the first time, scientists have directly visualized the protein clusters believed to trigger Parkinson’s disease, marking a major breakthrough in the study of the world’s fastest-growing neurological disorder. Researchers from the University of Cambridge, University College London, the Francis Crick Institute, and Polytechnique Montréal used a new imaging technique to detect and quantify tiny alpha-synuclein oligomers in post-mortem human brain tissue. These nanoclusters have long been considered the likely culprits for Parkinson’s disease, but until now, they have evaded direct detection in human brain tissue. The team found that oligomers in Parkinson’s patients were larger, brighter, and more numerous than in healthy brains, and identified a subclass present only in patients, potentially representing the earliest visible markers of the disease. The discovery could pave the way for better diagnostics and treatments, and the researchers suggest the technique could be applied to other neurodegenerative diseases, including Alzheimer’s disease and Huntington’s.– Bree Foster
Genmab will buy out Merus for $8B, acquire cancer drug
The antibody-focused oncology company, Genmab, announced plans to buy Merus at a value of approximately $8 billion. The buyout gives Genmab the bispecific antibody drug, petosemtamab, which has received two breakthrough therapy designations from the FDA to treat head and neck cancers. The drug is currently in two Phase 3 trials run by Merus, and Genmab noted that after its anticipated approval, they expect that the drug will lead to at least $1 billion annual sales potential by 2029. “Petosemtamab has the potential to be a transformational therapy for patients living with head and neck cancer. With our proven track record of success, both in clinical development and in commercialization, we are confident that we will be able to unlock the promise of petosemtamab,” said Jan van de Winkel, President and CEO of Genmab in the press release. – Allison Whitten
