Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Novo Nordisk taps Anthropic's Claude to speed drug discovery
Novo Nordisk is partnering with Anthropic to use Claude, including Claude Science, in its drug discovery and development work, part of a broader wave of drugmakers turning to AI to accelerate R&D and cut costs across their pipelines. The company will initially test the models on targeted R&D problems to strengthen its AI-driven software development, building on its existing use of Claude to automate trial report generation, cutting patient documentation timelines from months to minutes. In the press release, Novo CEO Mike Doustdar said AI can aid reasoning and understanding of human biology and drug mechanics; Anthropic CEO Dario Amodei said the partnership aims to shorten research timelines and improve outcomes. It comes as AI industry leaders remain divided on how fast the technology should be developed. – Andrea Corona
Vyriad begins first-in-human trial of in vivo CAR T therapy
Vyriad has opened a Phase 1 trial of VV169, an investigational in vivo CAR T therapy designed to generate BCMA-targeting T cells directly inside patients with relapsed or refractory multiple myeloma. The single-infusion therapy uses a retargeted lentiviral vector to deliver an anti-BCMA CAR to T cells in the body, avoiding the leukapheresis, ex vivo cell manufacturing, and lymphodepleting chemotherapy required for conventional CAR T treatments. VV169 also uses a promoter designed to activate CAR production specifically in T cells, helping prevent unintended CAR expression in other cell types. The trial will enroll up to 40 patients and primarily assess safety, dose-limiting toxicities, and a recommended Phase 2 dose, while also measuring CAR T cell expansion, persistence, and preliminary antitumor activity. VV169 is following other in vivo CAR-T approaches that have already reached the clinic, including BCMA-targeted therapies from EsoBiotec and Kelonia Therapeutics, adding to the emerging clinical evidence for generating engineered immune cells directly inside patients and potentially simplifying CAR T manufacturing and treatment. – Bree Foster
FDA announces program to accelerate Phase 1 trials
The FDA is launching a new program designed to hasten the time between finding a new drug and testing it in first-in-human Phase 1 trials, termed the Expedited Investigational New Drug (IND) Pilot. The key lies in pairing drug companies with qualified research institutions (QRI) that will aid in the efficient development of IND applications, according to the press release. As part of the US Department of Health and Human Services Operation Trailblazer, the new initiative will seek to cut down on the nearly two-year time period that first-in-human trials currently take in the US and come closer to the much quicker trials occurring in countries like China and Australia. “The FDA is doing our part to ensure American patients continue to receive access to therapies first, while keeping scientific innovation and investment in America,” said Karim Mikhail, Director of the Center for Biologics Evaluation and Research (CBER), in the release. The statement also noted that the FDA will be accepting new applications for the program up until October 30th. – Allison Whitten
Definium reports second Phase 3 win for LSD-based anxiety therapy
Definium Therapeutics has reported a second positive Phase 3 result for its LSD-based therapy DT120 Orally Disintegrating Tablet (ODT) in generalized anxiety disorder (GAD), strengthening the case for the psychedelic drug as the company moves toward an FDA filing. The PANORAMA study enrolled 245 patients and met its primary endpoint, demonstrating that the 100μg dose reduced Hamilton Anxiety Rating Scale scores by 9.8 points from baseline at week 12, compared with 4.7 points for placebo, a statistically significant and clinically meaningful improvement of 5.1 points. The therapy also met all three key secondary endpoints, with improvements over placebo evident as early as day two. DT120 ODT was generally well tolerated, with most adverse events mild to moderate and occurring on the day of dosing, although patients were monitored for at least eight hours following treatment. The result follows positive Phase 3 data from the company's VOYAGE trial in GAD last month and the EMERGE trial in major depressive disorder in June. Definium plans to meet with the FDA later this year and expects to submit a New Drug Application in the first half of 2027. – Bree Foster
SK And 1ST Biotherapeutices link up in Parkinson’s deal
Two South Korean drug developers, SK Biopharmaceuticals and 1ST Biotherapeutics, announced on Thursday that they will collaborate to advance 1STBIO’s orall small molecule dual inhibitor or LRRK2 (leucine-rich repeat kinase 2) and c-Abl (cellular Abelson tyrosine kinase) to treat Parkinson’s disease. The deal is worth up to $314.8 millon, and the statement outlined that 1STBIO will first receive an upfront payment of $1.8 million. The novel drug works by blocking LRRK2 — which contributes to Parkinson’s pathogenesis when the protein becomes overactive — in a different way from other drugs, namely a Type 2 binding mode, and the drug also simultaneously blocks c-Abl to reduce toxic alpha-synuclein buildup. “We will work to translate this candidate into successful clinical development, opening up new possibilities for the treatment of Parkinson's disease,” said Donghoon Lee, CEO of Sky Biopharmaceuticals, in the press release. – Allison Whitten
