Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
GLP-1 gene therapy enters first human trial
Fractyl Health received clinical trial authorization in the Netherlands this week to begin a first-in-human study of RJVA-001. The therapy takes a fundamentally different approach to GLP-1 treatment: Rather than delivering the hormone systemically through chronic injectable or oral drugs, RJVA-001 is designed as a one-time, endoscopically delivered infusion directly into the pancreas, where it reprograms beta cells to produce GLP-1 naturally in response to meals. The goal is physiologic hormone signaling at low circulating levels, potentially sidestepping the nausea, tolerability issues, and chronic dosing burden that cause many patients to discontinue existing GLP-1 therapies. The Phase 1/2 study will enroll adults with inadequately controlled type 2 diabetes who are already on GLP-1 receptor agonists and multiple oral agents, with first patient dosing and preliminary data expected in the second half of 2026. Fractyl also has a clinical trial application pending in Australia. For drug developers watching the GLP-1 space, RJVA-001 represents an early but notable test of whether gene therapy can move from managing metabolic disease to potentially treating it once. This emerging modality is one thread in the wider story of GLP-1 and metabolic disease, spanning the receptor biology, drug discovery, and therapeutic pipeline behind the class. – Andrea Corona
FDA commissioner Marty Makary resigns after months of political clashes
Marty Makary has resigned as commissioner of the FDA, ending a 13-month tenure marked by repeated clashes with the White House, Congress, industry, and public health experts. The departure follows weeks of turmoil at the agency and comes after President Trump had earlier signed off on a plan to fire Makary after he resisted White House pressure to approve fruit-flavoured vaping products. Makary’s acting replacement will be Kyle Diamantas, previously the agency’s top food official, whom Trump described as a “very talented person” in a Truth Social post. In a resignation message shared publicly by the president, Makary defended his record, citing what he described as dozens of major reforms, including sharply shortened drug review timelines, new guidance for psychedelic therapies, a plausible mechanism pathway for rare disease drugs, and changes to estrogen labelling. Trump praised Makary as “a great doctor” and thanked him for his service, even as the former commissioner’s tenure was dogged by controversy over vaccine policy, including reports that the agency halted or delayed publication of research supporting the safety of COVID-19 and shingles vaccines, and by criticism from anti-abortion Republicans keen on having the FDA restrict telehealth prescription of the abortion pill mifepristone. Former FDA officials and public health advocates have warned that the turmoil and political interference risk undermining trust in the regulator, particularly as the administration has sought to avoid public debate over unpopular vaccine policy changes ahead of the midterm elections. – Bree Foster
Bristol Myers Squibb links up with Hengrui in $15B deal
In a partnership worth $15.2 billion that aims to advance 13 assets, Bristol Myers Squibb (BMS) and Hengrui Pharma announced that they have agreed to combine BMS’ strengths including global clinical development capabilities, regulatory expertise, and commercial scale with Hengrui’s discovery engine and platform technologies. The assets are all early-stage programs and include four oncology/hematology assets from Hengrui, four immunology assets from BMS, and five “innovative assets to be jointly discovered and developed by both companies,” according to the statement. BMS will obtain exclusive worldwide rights to the Hengrui-originated assets outside of mainland China, Hong Kong, and Macau. As per the agreement, Hengrui will take on full responsibility for early clinical development. For BMS, the deal signals their aspiration to take advantage of China’s speedier timelines for early-stage programs. "By leveraging complementary capabilities across geographies, we aim to accelerate early clinical learning and make informed decisions that support driving top tier growth in the next decade and, ultimately, our mission to deliver medicines that help patients prevail over serious diseases,” said Robert Plenge, Executive Vice President and Chief Research Officer at BMS. – Allison Whitten
AI-driven platform boosts potency of experimental antibiotics
Researchers at the University of Pennsylvania have developed ApexGO, an AI-powered platform designed to rapidly optimize existing antimicrobial peptides rather than simply search for new ones, addressing a key bottleneck in antibiotic discovery as resistance rises worldwide. Reported in Nature Machine Intelligence, the approach iteratively refines imperfect peptide templates using predictive modeling and Bayesian optimization to propose targeted sequence edits under practical design constraints. In laboratory tests, 85 percent of AI-designed peptides showed antimicrobial activity and 72 percent outperformed their starting molecules against Gram-negative pathogens. Two candidates reduced bacterial loads in mouse models of Acinetobacter baumannii infection at levels comparable to polymyxin B, a last-resort antibiotic. Led by César de la Fuente and Jacob Gardner, the study positions ApexGO as a more systematic and scalable route to improving antibiotic candidates, shifting AI’s role from broad screening toward precision molecular design. – Bree Foster
FDA approves first drug for rare bile duct cancer
Last Friday, the FDA granted its seventh National Priority Voucher to Bizengri from Partner Therapeutics to treat NRG1 (neuregulin 1) fusion-positive cholangiocarcinoma — a rare but extremely life-threatening cancer of the bile ducts. Just three days later, the FDA approved the drug for adults with disease progression on or after prior systemic therapy. Bizengri thus became the first drug approved to treat the rare cancer after previously receiving the Breakthrough Therapy designation and Orphan Drug designation. The approval was based on the eNRGy Phase 2 trial in 22 patients, which showed an overall response rate of 36.8 percent and a duration of response range from 2.8 to 12.9 months. "Today's FDA approval of BIZENGRI for NRG1 fusion-positive cholangiocarcinoma is a historic milestone for patients who have had no approved targeted therapy. … We are grateful that the FDA's Commissioner's National Priority Voucher pilot program greatly reduced the review time, helping bring this treatment to patients more quickly,” said Pritesh Gandhi, Chief Development Officer of Partner Therapeutics, in the press release. – Allison Whitten
Biogen's Alzheimer's tau drug misses its primary endpoint, pushes forward
Biogen shared topline results from the Phase 2 CELIA study of diranersen (BIIB080), a tau-targeting antisense oligonucleotide discovered by Ionis Pharmaceuticals and licensed to Biogen in 2019. The trial, which enrolled 416 participants with early Alzheimer's disease, did not meet its primary endpoint assessing dose response on the Clinical Dementia Rating–Sum of Boxes at week 76. Despite that miss, Biogen says it will advance diranersen to registrational development, pointing to robust reductions in cerebrospinal fluid tau and tau positron emission tomography signals across all studied doses, as well as pre-specified secondary analyses showing slowing of cognitive decline, particularly at the lowest dose of 60mg every 24 weeks. Full results are expected at the Alzheimer's Association International Conference in July 2026. Still, the data mark the first time a randomized Phase 2 study of a tau-directed therapy has shown both biomarker impact and signals of cognitive benefit, a meaningful step for a field that has long struggled to move beyond amyloid-targeting approaches. — Andrea Corona
