Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Lilly and NVIDIA to build pharma industry’s most powerful AI supercomputer
Eli Lilly and Company has teamed up with NVIDIA to build what it calls the most powerful supercomputer ever owned by a pharmaceutical company, aiming to transform how medicines are discovered, developed, and delivered. The new system will serve as the backbone of Lilly’s “AI factory,” a dedicated computing infrastructure that manages the full AI lifecycle, from data ingestion and model training to fine-tuning and large-scale inference. Powered by more than 1,000 of NVIDIA’s latest B300 GPUs, the system will enable Lilly scientists to train AI models on millions of experiments, accelerating the identification, optimization, and validation of new drug candidates. Beyond discovery, Lilly plans to use the supercomputer to streamline development, manufacturing, and imaging workflows. Researchers will be able to deploy advanced AI agents for reasoning and planning, create digital twins to enhance production efficiency, and apply deep learning models to medical imaging to better track disease progression. Some of these proprietary tools will be made available through Lilly’s TuneLab platform, a federated AI and machine learning hub designed to foster collaboration across the biopharma sector. According to Lilly, the supercomputer will run entirely on renewable energy as part of the company’s carbon-neutrality goals for 2030. – Andrea Corona
BioMarin joins industry retreat from AAV gene therapies
BioMarin is preparing to divest its struggling hemophilia A gene therapy Roctavian after the treatment failed to gain meaningful commercial traction. The company reported third-quarter Roctavian sales of just $3 million — a 57 percent drop year-over-year — despite earlier expectations that the one-time gene therapy could reshape care. Uptake has been limited amid high costs, strict eligibility requirements, and uncertainty around long-term durability, especially as Roche’s Hemlibra and newer factor therapies continue to dominate the market. The move comes as other major drugmakers step back from AAV-based gene therapies, with Biogen, Vertex, Pfizer, and Takeda all discontinuing or scaling down their AAV programs. BioMarin says it will maintain access and patient support during the transition as it evaluates out-licensing or sale options, while shifting strategic focus toward its core enzyme therapy and skeletal disease portfolio, led by growth drivers Voxzogo and Palynziq. – Bree Foster
New antibiotic hiding in plain sight shows promise against drug-resistant bacteria
Chemists from the University of Warwick and Monash University have uncovered a potent new antibiotic hiding in plain sight, offering hope against drug-resistant bacterial infections including methicillin-resistant Staphylococcus aureus (MRSA) and vancomycin-resistant Enterococcus. The discovery, published in the Journal of the American Chemical Society, identifies pre-methylenomycin C lactone, a previously overlooked intermediate in the biosynthesis of the 50-year-old antibiotic methylenomycin A, as being over 100 times more active against Gram-positive bacteria. Importantly, the team observed no emergence of resistance to pre-methylenomycin C lactone under laboratory conditions where vancomycin resistance typically appears, a promising sign for tackling vancomycin-resistant Enterococcus, a WHO high-priority pathogen. With a scalable synthetic route now established, researchers are planning pre-clinical testing and development of analogues, suggesting a new strategy for antibiotic discovery by exploring biosynthetic intermediates in well-known natural compounds. – Bree Foster
Intellia CRISPR trials on pause after liver injury
On Monday, Intellia Therapeutics announced that they had paused two of their Phase 3 trials testing the CRISPR gene therapy, nex-z, to treat patients with transthyretin amyloidosis, a rare disease caused by misfolded proteins that accumulate in organs. The decision came after a patient was hospitalized with Grade 4 liver transaminases and an increase in total bilirubin, indicative of drug-induced liver injury. After the announcement, Intellia’s stock went down by 50 percent. On Wednesday, the FDA officially placed the trials under clinical hold. The company said in the release that they estimate more than 450 patients across the trials were given nex-z. – Allison Whitten
FDA approves Bayer’s non-hormonal menopause drug
Bayer’s non-hormonal pill to treat hot flashes, Lynkuet, received FDA approval based on data from the Phase 3 OASIS clinical trials. The drug becomes the first and only dual neurokinin drug (targeting both neurokinin 1 and 3) to treat menopause-related hot flashes, and has already been approved in Australia, Canada, the UK, and Switzerland. Bayer reported that they expect Lynkuet to be available in the US starting this November, which will compete with the already-approved, non-hormonal neurokinin 3 receptor agonist, Veozah, from Astellas Pharma. Yet, Veozah has seen lower than expected demand so far, and last December, the FDA placed a black box warning on the drug due to the risk of serious liver injury. – Allison Whitten
FDA aims to speed biosimilar approvals and lower drug costs
The FDA has introduced new draft guidance aimed at boosting biosimilar competition and lowering the cost of biologic medicines once their patents expire. Under the proposed measures, all biosimilars would automatically be designated as interchangeable with their branded counterparts, allowing pharmacists to substitute them without additional approvals. The agency also plans to eliminate switching studies and ease clinical testing requirements, moves which are expected to cut development time and costs roughly in half. While biologics represent just 5 percent of US prescriptions, they account for more than half of total drug spending, and the FDA noted that Europe has approved more than twice as many biosimilars. Officials said the reforms are intended to close this gap and make affordable alternatives more accessible to patients, addressing long-standing regulatory and industry barriers that have limited biosimilar availability in the US. – Andrea Corona
