Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
First FDA Priority Voucher goes to antibiotic
On Tuesday, the FDA announced its first approval under the Commissioner’s National Priority Voucher program, which was rolled out in June of this year. The approval — which was completed in just two months over the usual 10–12 months — went to Augmentin XR, an antibiotic made of amoxicillin and clavulanate potassium produced by USAntibiotics. Augmentin XR was originally developed by GSK and approved by the FDA back in 2002, with the new approval bringing the drug into a US manufacturing plant. "This first drug approval under the CNPV pilot program will strengthen domestic manufacturing and increase our national security,” said FDA commissioner Marty Makary in the press release. The release also noted that the approval will help address antibiotic shortages in the US. – Allison Whitten
Zealand Pharma, OTR Therapeutics launch multi-program metabolic disease partnership worth up to $2.5 billion
Zealand Pharma and OTR Therapeutics have entered a multi-program collaboration and licensing agreement to co-discover and co-develop oral small-molecule therapeutics for multiple metabolic disease targets, pairing Zealand’s expertise in obesity and metabolic health with OTR’s proprietary discovery platform. OTR will lead research and preclinical work, while Zealand will oversee clinical development, regulatory activities, and global commercialization. OTR will receive an upfront payment of $20 million, potentially rising to $30 million, and is eligible for up to roughly $2.5 billion in preclinical, development, regulatory, and commercial milestones, plus tiered royalties on net sales. The partnership is intended to expand Zealand’s metabolic pipeline into next-generation oral therapeutics and accelerate the development of novel treatment options for metabolic diseases. – Andrea Corona
Phase 1 trial shows TERN 701 effective in treating resistant CML
Terns Pharmaceuticals reported new Phase 1 data for its experimental chronic myeloid leukemia (CML) therapy, TERN‑701, at the American Society of Hematology meeting, showing strong responses in patients who had become resistant to other therapies. In the updated CARDINAL study results, 74 percent of efficacy-evaluable participants achieved a major molecular response by 24 weeks, with higher response rates and more frequent deep molecular responses observed at doses of 320mg or above. The therapy was well tolerated across all doses, with no dose‑limiting toxicities and only one discontinuation due to side effects. The findings add to growing interest in TERN-701, an oral allosteric BCR-ABL1 inhibitor that could eventually challenge Novartis’ asciminib in later-line CML treatment, though no head-to-head trials have been conducted. Shares of Terns surged 35 percent to $39.42 following the announcement, hitting an intraday high of $45 and continuing a nearly eightfold increase for the year. – Bree Foster
AIM-NASH becomes first AI tool cleared by FDA and EMA
The FDA has qualified the first AI tool designed to help doctors assess severe fatty liver disease in drug trials, a move that is expected to streamline the development of treatments for metabolic dysfunction-associated steatohepatitis (MASH). The cloud-based system, called AIM-NASH, analyzes liver biopsy images to identify fat buildup, inflammation, and scarring, providing standardized scores for doctors to interpret. Qualification means the tool can now be used in any drug development program for MASH, supporting regulatory submissions without additional review. AIM-NASH, developed by PathAI, has shown results comparable or superior to traditional expert assessments, reducing variability and the time needed for biopsy evaluation. The AI tool follows similar endorsement from the European Medicines Agency and could significantly accelerate trials for a disease that affects millions worldwide and can lead to cirrhosis, liver failure, or cancer. With only two drugs currently approved for MASH, AIM-NASH is expected to aid the growing pipeline of experimental therapies while enabling more accurate, scalable, and reproducible evaluation of biopsy endpoints. – Bree Foster
Apple Tree Partners files for Chapter 11 to reorganize operations
Apple Tree Partners (ATP) has filed voluntary Chapter 11 petitions for several of its affiliates in the US Bankruptcy Court for the District of Delaware as part of a plan to restructure operations while continuing to support its life sciences portfolio. The firm, which has invested $2.5 billion over more than two decades, said it intends to maintain funding for companies developing therapies in oncology, neurological disorders, infectious diseases, and other areas. ATP has been one of the few venture firms that routinely incubates companies in-house, often developing programs internally rather than sourcing them from external startups. Several of its portfolio companies, such as Akero have advanced into late-stage clinical development or public markets, giving the firm an outsized role in shepherding early biomedical research toward commercialization. – Andrea Corona
FDA ups the ante on approvals for CAR T therapies
In a new perspective article in JAMA Network, Vinay Prasad, FDA Chief Medical and Scientific Officer and Director of the Center for Biologics Evaluation and Research, and his colleagues outlined the FDA’s new regulatory framework for the approval of CAR T cell cancer therapies. The plan includes the preference for randomized control trials with a survival or acceptable time-to-event end point, the selection of a control group that considers the available standard treatments and approved CAR T cell therapies, and evidence that demonstrates superiority of the therapy compared to the control. However, the authors noted that it may not always be feasible or ethical to conduct a randomized control trial, so sponsors may consider a one-trial approach that can be analyzed early to support an accelerated approval. Given that there is only one CAR T cell therapy approved for pediatric tumors, the authors also emphasized flexibility in that space by noting that “the FDA is committed to maximum regulatory flexibility to expedite additional products to children with terminal or life-limiting cancers.” – Allison Whitten
