TN-201

Plus, news about Tenaya, AtaiBeckley, Galecto, Damora, A2 Biotherapeutics, AstraZeneca and Evommune: 📉 AnaptysBio’s lead drug fails ulcerative colitis study: The company said the T cell depleter called rosnilimab showed a “lack of adequate efficacy,” and the Phase 2 trial will be discontinued. In September, AnaptysBio said it was weighing a split of its pipeline from its royalty business. Anaptys’ stock $ANAB fell 16% on Monday. — Lei Lei Wu 🤝 Lyell Immunopharma licenses CAR-T cell candidate: The company will pay Innovative Cellular Therapeutics $40 million upfront to take the global rights to LYL273 (formerly codenamed GCC19CART) except for mainland China, Hong Kong, Macau and Taiwan. ICT will also receive 1.9 million shares of Lyell’s common stock, with further cash and equity consideration on the table if the asset reaches certain milestones. LYL273 is under investigation for cancers with guanylyl cyclase C (GCC) protein receptors; it has Phase 1 data in metastatic colorectal cancer. – Reynald Castaneda 🛑 FDA slaps hold on Tenaya heart drug trial: Tenaya said regulators had requested the company take measures to “standardize” patient monitoring activities across sites in a Phase 1b/2a study. The trial is looking at TN-201 in MYBPC3-associated hypertrophic cardiomyopathy. Tenaya said the hold won’t impact the drug’s development timelines. — Max Gelman 📊 AtaiBeckley debuts psychedelic redosing data : The recently-merged psychedelic biotech found that a second dose of the company’s intranasal 5-MeO-DMT psychedelic helped further reduce patients’ depression symptoms. The data were collected in an open-label portion of the Phase 2b study that included more than 100 patients. There was a drug-related serious adverse event reported in one patient eight days after a second dose was administered. The company did not detail the case, but said it resolved after “additional in-patient monitoring and support.” AtaiBeckley expects to launch a Phase 3 study in the second quarter of 2026. — Max Bayer 💰 Galecto acquires Damora, raises $285M: Galecto bought out its counterpart for an undisclosed sum and said the deal will boost development of drugs for blood cancers. The $285 million raise came in the form of a private placement from investors who participated in Galecto’s Series C, giving the company runway into 2029. — Max Gelman 🫁 Cell therapy clears lung cancer patient’s disease completely : A cell therapy from A2 Biotherapeutics cleared all signs of cancer down to the molecular level in a lung cancer patient, marking a rare case in which a cell therapy has resulted in a complete response in a solid tumor patient. It was a surprising result, given the patient’s lung cancer had high-risk markers, according to investigator Salman Punekar of New York University. Last year, Stanford researchers also showed that a CAR-T therapy led to a complete response in a brain cancer patient. A2 Biotherapeutics’ trial was complex, as patients needed to have a certain HLA signature to enroll in the early-stage study. The cell therapy uses “logic-gating,” which is meant to help the treatment target cancer cells while sparing healthy ones. — Lei Lei Wu 🫀AstraZeneca touts more data for potential heart disease blockbuster: The company said its drug baxdrostat reduced average blood pressure by a statistically significant margin compared to placebo after 12 weeks. Researchers were evaluating baxdrostat, acquired from the 2023 CinCor buyout, in 218 patients with resistant hypertension. The data build on two successful pivotal studies for baxdrostat announced in July and October , respectively. — Max Gelman 💵 Evommune closes IPO with $172.5M raise: The biotech had planned a $150 million raise, but those with options fully exercised them , bringing the total to $172.5 million. Evommune began trading on the New York Stock Exchange last Thursday at a starting share price of $16. — Max Gelman

Tenaya Therapeutics shared data from a phase 1b/2a trial of the gene therapy TN-201 over the weekend.\n The FDA has put a clinical hold on a Tenaya Therapeutics gene therapy study while the biotech works to standardize activities related to the immunosuppression regimen across trial sites.Tenaya said the FDA asked it to change the protocol for a phase 1b/2a trial of the gene therapy TN-201 in MYBPC3-associated hypertrophic cardiomyopathy. The request followed proactive correspondence with the FDA about the next steps for TN-201, the biotech said, and was mainly based on data the trial’s data safety monitoring board (DSMB) reviewed in the summer.While the DSMB signed off on enrollment in expansion cohorts at two doses in July, the FDA has identified a need to standardize immunosuppression patient monitoring and management activities across the trial sites. Tenaya attributed the clinical hold to the FDA’s request for a protocol amendment.The biotech said TN-201 has been generally well tolerated so far, adding that no new meaningful safety events associated with the gene therapy have emerged since the DSMB review. Tenaya plans to resume dosing once the protocol changes have been implemented at trial sites and doesn’t expect the FDA hold to affect data milestones or development timelines for TN-201.  The biotech shared data from the phase 1b/2a trial over the weekend at the American Heart Association’s 2025 Scientific Sessions. The company said shortening the course of immunosuppression resulted in the second cohort having faster tapers and lower cumulative corticosteroid doses, despite patients receiving more of TN-201 than their peers in the first cohort.Initial results for the participants in the second cohort showed early dose-responsive increases in TN-201 transduction and MyBP-C protein expression. MyBP-C expression increased by 14% after 12 weeks in the first evaluable patient in the second cohort. Tenaya reported an average 4% increase from the first biopsy to Week 52 in the first cohort. Cardiac troponin I levels declined 48% to 74% to normal or near-normal levels in all patients in the first cohort. TD Cowen analysts highlighted troponin data when they saw initial data from the trial 11 months ago, telling investors the normalization of levels of the biomarker in one patient was a particularly encouraging sign.