Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Novartis pauses autoimmune CAR T trials following three deaths
Novartis has hit the brakes across eight early- and mid-stage trials of its experimental CD19-directed CAR T therapy rapcabtagene autoleucel (rap-cel). The company made the move after three patients died from immune effector cell-associated hemophagocytic syndrome (IEC-HS), a rare but potentially fatal hyperinflammatory complication of CAR T treatment. The pause affects the therapy’s immunology and neuroscience programs, including Phase 2 AUTOGRAPH studies in systemic lupus erythematosus, lupus nephritis, systemic sclerosis, ANCA-associated vasculitis, and idiopathic inflammatory myopathies, as well as Phase 1/2 trials in rheumatoid arthritis, Sjögren’s disease, myasthenia gravis, and multiple sclerosis. Novartis’ oncology studies of rap-cel remain ongoing. The move comes as drugmakers increasingly explore CAR T beyond cancer, raising new questions about how the therapy’s potentially serious toxicities can be managed in autoimmune diseases. Bristol Myers Squibb has also paused enrollment in its autoimmune studies of zolacabtagene autoleucel (zola-cel) after observing transient, reversible inflammatory events, although the company is still conducting the trials. – Bree Foster
GSK bets big on Hutchmed’s antibody-targeted therapy conjugate
On Thursday, Hutchmed announced a new licensing agreement with GSK for their KRAS-EGFR-antibody conjugate therapy, HMPL-A830. The deal includes a $110 million upfront payment with potential development, regulatory, and commercial milestone payments that could reach up to $1.295 billion. The drug has a unique dual mechanism that pairs a monoclonal antibody targeting EGFR with a small molecule inhibitor targeting KRAS, and avoids the harsh chemotherapy agents in typical ADCs. In the agreement, Hutchmed will develop the Phase 1 program, which is expected to begin later this year, and GSK will be responsible for all clinical development and commercialization after that, excluding Mainland China, Hong Kong, Macau and Taiwan. “HMPL-A830 is our third drug candidate from these novel payload platforms and the first from our platform to be licensed to a global partner, following two candidates that have entered clinical development. This collaboration marks a significant step in maximizing its potential as a treatment for patients, unlocking an entirely new class of precision oncology medicines,” said Johnny Cheng, Acting CEO and Chief Financial Officer at Hutchmed. For GSK, the deal marks their first asset targeting the once-undruggable KRAS pathway and signals a push into next-generation drug conjugates. – Allison Whitten
uniQure files BLA for first gene therapy targeting Huntington's disease
uniQure submitted a Biologics License Application (BLA) to the FDA and a Marketing Authorization Application to the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for ifezuntirgene inilparvovec (AMT-130), a one-time gene therapy that silences the huntingtin gene via direct neurosurgical delivery into the brain, based on three-year Phase 2/3 data showing the high dose slowed disease progression by 75 percent versus an external control from the Enroll-HD natural history database. The path here wasn't straightforward: The FDA reversed course in November 2025 on whether that external-control comparison could support a BLA, then reaffirmed it could this June, sending shares up roughly 70 percent after the news. Huntington's disease still has no approved disease-modifying therapy, and recent efforts from Roche and Wave Life Sciences have stumbled on the same underlying problem uniQure is trying to solve with an external control instead of a placebo arm, namely how hard it is to run a conventional sham-controlled trial in a small, slowly progressive, neurosurgically invasive indication. For drug developers, the filing is as much a regulatory case study as a clinical one, testing how far the FDA's accelerated approval pathway can stretch to accommodate external controls in ultra-rare neurodegenerative disease, a precedent that could shape the next wave of gene therapies chasing conditions where a traditional trial design isn't feasible. – Andrea Corona
Trump adds nine drugmakers to MFN pricing agreements
Nine additional pharmaceutical companies have signed most-favored-nation (MFN) drug pricing agreements with the Trump administration, bringing the total number of drugmakers participating in the initiative to 26 and expanding coverage to roughly 90 percent of the US branded drug market. The latest agreements were signed by Alcon, Astellas, BeOne Medicines, BridgeBio, CSL, Kyowa Kirin, Sun Pharma, Teva Pharmaceuticals, and UCB, nearly a year after Pfizer became the first company to sign an MFN deal. Under the agreements, the companies will offer certain medicines to state Medicaid programs at prices aligned with the lowest prices paid by other developed nations, covering treatments for conditions including hemophilia, Parkinson’s disease, macular degeneration, glaucoma, liver disease, and cancer. Unlike earlier deals with large pharmaceutical companies, which included commitments to sell medicines through the TrumpRx direct-to-consumer platform and offered relief from certain pharmaceutical tariffs, the latest agreements do not appear to include TrumpRx or disclosed tariff exemptions. The nine companies have instead pledged a combined $19.6 billion in US manufacturing and R&D investments, while four will also contribute active pharmaceutical ingredients to the Strategic Active Pharmaceutical Ingredients Reserve. – Bree Foster
Teva announces positive Phase 2 results in celiac disease
TEV ‘408, Teva Pharmaceuticals’ investigational anti-interleukin-15 monoclonal antibody drug, was shown to prevent gluten-induced intestinal damage in a Phase 2a study of 50 adults with celiac disease, according to the press release. The trial followed patients adhering to a strict gluten-free diet — currently the only option to treat the disease — who received one dose of TEV ‘408 and then two weeks later began a six-week daily gluten challenge. The drug met its primary endpoint in preventing gluten-induced intestinal damage compared to placebo at week eight, and the company noted that it was well tolerated with no safety signals seen yet. “These results underscore the potential to move beyond managing gluten exposure and address celiac disease at its biological source. They also strengthen our confidence in targeting the IL-15 pathway as an approach to reducing immune-driven intestinal damage,” said Eric Hughes, Executive Vice President, Global R&D and Chief Medical Officer at Teva, in the statement. Teva is also investigating TEV ‘408 to treat the skin condition vitiligo, suggesting the potential for a broad application across autoimmune diseases. – Allison Whitten
GSK advances dual-target mRNA flu vaccine to phase 3
GSK presented positive Phase 2 data for its mRNA seasonal flu vaccine candidate, showing higher immune responses across all tested strains than licensed standard-dose and high-dose vaccines in a trial with 971 adults. Unlike current vaccines, which target only hemagglutinin (HA), GSK's shot also targets neuraminidase (NA), the other major surface antigen flu viruses use to spread, an approach the company says could improve protection and reduce transmission. Based on the results, GSK will start a Phase 3 trial this month, the first late-stage test of a dual HA/NA mRNA flu vaccine, and the program already holds FDA Fast Track designation. The advance puts GSK in direct competition with Moderna, whose mRNA flu shot mFLUVISA won FDA approval last month, at a moment when GSK's traditional flu vaccine sales fell roughly 25 percent last year. For drug developers, it's an early signal that the mRNA flu vaccine race is shifting from single-target designs to multi-antigen ones, with NA-targeting data set to shape how competitors design their next candidates. – Andrea Corona
