Marnetegragene autotemcel

Welcome back to Endpoints Weekly! Our team tracked a handful of deals announced this week as the first quarter comes to a close. Check below for a summary of those moves, including Merck’s $6.7 billion deal to acquire Terns. Meanwhile, Sanofi unveiled a new partnership with a California startup focused on T cell engagers. Our reporters also covered two FDA approvals for rare disease treatments and a disappointing readout for Wave’s obesity candidate.  — Nicole DeFeudis 💰A triumvirate of large pharma companies pulled off acquisitions this week, apparently treating the end of the first quarter as a self-imposed deadline. The biggest comes from Merck, which scooped up Terns Pharmaceuticals off the public market for $6.7 billion — $53 per share, a 31% premium to Terns’ 60-day average stock price. The centerpiece of the Terns deal is a drug designed to treat chronic myeloid leukemia, a slow-progressing blood cancer. Last December, Terns outlined data for the drug that justified its pivot away from obesity. Merck was evidently impressed: TERN-701 will likely compete with Novartis’ Scemblix, a newly minted blockbuster, should it win approval. Terns had planned to kick off a pivotal study later this year or in early 2027. Novartis also got in on the action, buying out immunology biotech Excellergy for up to $2 billion . It’s a quick exit for a company that only debuted with a $70 million Series A in October . The focus on this deal is Excellergy’s “effector cell response inhibitors,” designed to interfere with overactive immune responses related to immunoglobulin E, or IgE. Excellergy has said its lead drug could have use in food allergy, chronic hives and asthma. Gilead rounds out this week’s deal flurry with a deal for Ouro Medicines, which is developing T cell engagers for immunologic diseases. Gilead will pay about $1.68 billion upfront , while roping in longtime partner Galapagos to conduct early work on the lead drug. That program, called gamgertamig, is a bispecific targeting BCMA and CD3. 🌊Trying to beat GLP-1s in obesity is a tough road. Wave Life Sciences underlined that reality this week when its obesity candidate yielded weight loss of just 1% more than placebo in an early-stage study. Patients received a single 240 mg dose of WVE-007, a small interfering RNA drug. The FDA’s approvability bar for obesity meds has been set by the FDA at 5%. Wave’s stock price fell nearly 50% in response. The biotech has argued that its drug prioritizes fat loss and retains lean mass. That thesis remained relatively intact after the readout. There was a placebo-adjusted reduction in visceral fat of 14%, which just grazed significance at a p-value below 0.05. At the same dose level, total fat was reduced by 5%, lean mass increased 2% and patients’ average waist circumference fell by 3%, all on a placebo-adjusted basis. Executives still see a path forward. The second part of the study, functioning as a Phase 2, will start by mid-year. Data could come this year or next. The company is also planning to study WVE-007 as an add-on to a GLP-1 drug, and in the post-GLP-1 maintenance setting, this year. 🤝Sanofi is back in on T cell engagers. After a couple years of fluctuating interest in the space, the French pharma giant is teaming up with a California startup in a licensing deal worth $180 million in upfront and near-term payments. The pact focuses on KT501, a trispecific TCE that goes after the well-known targets of CD19, BCMA and CD3. Kali CEO Weihao Xu told Endpoints that the deal was inked in December, but the partners waited until KT501 was in the clinic to announce it. Kali said last week that it began dosing the first cohort of adults with rheumatoid arthritis. The partnership suggests Sanofi is recommitting to TCEs. The company had made a $1 billion bet on TCEs with the purchase of Amunix in 2021, only to then divest those assets to Vir Biotechnology in 2024. Sanofi has dropped other TCEs along the way, including an anti-GPC3 nanobody-based candidate. The Sanofi-partnered asset isn’t all Kali has in the works — it’s just one of multiple medicines the startup hopes to bring into the clinic, including two more that are expected to enter human testing this year. Read more here from Kyle. ✅The FDA approved two rare disease treatments this week, bucking a recent trend of rejections in the space. First came a greenlight for Denali Therapeutics’ Avlayah in Hunter syndrome . The drug became the first to win accelerated approval using heparan sulfate as a surrogate biomarker, a path that eluded two rivals pursuing the same strategy: Ultragenyx and Regenxbio. Both are developing gene therapies for rare diseases under the same genetic umbrella. Then, the FDA’s biologics division approved a gene therapy from Rocket Pharmaceuticals for an ultra-rare immune disease. It’ll be sold as Kresladi and is approved to treat leukocyte adhesion deficiency-I (LAD-I), where patients face serious and potentially deadly bacterial and fungal infections. The nod comes after the FDA rejected the therapy in 2024, leading to layoffs and pipeline cuts at Rocket. ✉️In a warning letter released this week, the FDA raised concerns with California-based ImmunityBio for a TV advertisement and podcast that it said gave the “misleading impression” that Anktiva, the company’s treatment for a type of bladder cancer, can cure or prevent all cancers. The ad and podcast featured billionaire entrepreneur and ImmunityBio founder Patrick Soon-Shiong. In a separate warning letter for a Daman, India-based drug manufacturing facility, the agency made the rare move of posting photographs of dilapidated buildings. The photographs are the latest sign that FDA Commissioner Marty Makary is taking a tougher stance on foreign drug manufacturers. DON’T MISS:

Rocket is not counting on Kresladi as a major money-maker, with CEO Gaurav Shah, M.D., noting on a Friday investor call that his company will execute a “minimal viable launch” for the therapy. With an end-of-week green light from the FDA, Rocket Pharmaceuticals has officially broken into commercial orbit. Still, the company is taking a more measured approach for the launch of its new gene therapy Kresladi, as it conserves fuel for a commercial push into future cardiovascular frontiers. Friday morning, Rocket announced that the FDA signed off on an accelerated approval for its hematopoietic stem cell-based gene therapy Kresladi, also known as marnetegragene autotemcel, to treat certain children with severe leukocyte adhesion deficiency-1 (LAD-1). Given the accelerated nature of the nod, the clinical benefit of Kresladi will need to be confirmed through evaluation of longer-term follow-up data of treated patients in both an ongoing trial and through a post-marketing registry, Rocket said in a March 27 press release. The FDA’s current thumbs-up was based on an increase in neutrophil CD18 and CD11a surface expression in Rocket’s Kresladi data, per the company. Meanwhile, Rocket has also secured a rare pediatric disease priority review voucher from the regulator in tandem with its approval, which the company plans to sell to support its R&D ambitions.  The FDA nod marks the first for a gene therapy for kids with severe LAD-1 due to biallelic variants in the ITGB2 gene and blesses Rocket with its first commercial product. But Rocket is not counting on Kresladi as a major money-maker, with CEO Gaurav Shah, M.D., noting on a Friday investor call that his company will execute a “minimal viable launch” for the therapy. By that, he meant that Rocket will make Kresladi available to patients and doctors seeking it out, while eschewing a more concentrated marketing push or big investments in commercialization, instead saving that commercial muscle for the gene therapy developer’s newer cardiovascular focus. LAD-1 is an ultra-rare genetic disease caused by mutations in the ITGB2 gene encoding for the protein CD18, Rocket explained in its release. Patients with the severe form of the condition often show “very diminished” CD11a expression and infants with the disease suffer from recurrent, life-threatening bacterial and fungal infections, according to the company. Up until now, standard treatment for severe LAD-1 relied on allogeneic hematopoietic stem cell transplantation, which itself comes with some significant risks, such as graft-versus-host disease, Shah said.In terms of the addressable market for the gene therapy, roughly 25 children are estimated to be born with LAD-1 in the U.S. each year, and approximately two-thirds of that group typically manifest with the severe form of the disease, Sarbani Chaudhuri, Rocket’s chief commercial and medical affairs officer, added on the call. Nevertheless, given the inherent complexities of delivering a gene therapy like Kresladi, Chaudhuri said that Rocket expects the number of patients treated annually with the drug to “remain in the single digit range, including in the years following launch.” In terms of launch timing, Rocket expects patients to be able to enroll in Kresladi treatment in the fourth quarter of this year, giving the company several months to shore up “product supply readiness” with external manufacturers, coordinate its commercial treatment process and onboard “a limited number of highly experienced, qualified treatment centers,” Chaudhuri explained. “Given the complexity of treatment and the vulnerability of the pediatric patient population, Kresladi will initially be available at a limited number of specialized centers to support operational excellence and patient safety during early commercialization,” she added. The company did not speculate on pricing or access for now, with Chaudhuri noting that the company will divulge those details closer to the gene therapy’s planned fourth-quarter rollout. Rocket anticipates the first patient infusion with Kresladi, “and therefore initial product revenue,” to start pouring in by 2027, she added. As of the end of 2025, Rocket had cash, cash equivalents and investments on hand equivalent to some $188.9 million, which Shah said he expects to fuel his company’s operations into the second quarter of 2027, and potentially into 2028 upon a successful sale of the priority review voucher it’s obtained. While Rocket may not be making Kresladi a commercial priority, others in the field appeared enthusiastic about the gene therapy’s green light, especially against a backdrop of recent regulatory uncertainty at the FDA. “This is welcome news for both the rare disease patient community and the cell and gene therapy sector in general,” Tim Hunt, CEO of the Alliance for Regenerative Medicine, said in a Friday statement. “It is our hope that this approval, combined with the search for a new head of CBER, will usher in a new, patient-centric chapter at the FDA,” he continued, referring to the recent controversy around the FDA’s soon-to-depart biologics and gene therapy czar Vinay Prasad, M.D.He added: “For the currently stalled rare disease CGT programs, FDA should honor its past alignment with companies, use its Congressionally authorized regulatory flexibility, and grant Advisory Committee meetings to companies that request one.”For its part, Rocket initially sought out FDA review for Kresladi in its LAD-1 indication back in late 2023, but the regulator rebuffed the gene therapy on the need for additional information about its manufacturing.  Meanwhile, Rocket charted a major trajectory change last summer following a patient death in one of its gene therapy trials earlier in 2025. In late July, the company revealed that it would reduce its headcount by 30% and narrow its development focus around cardiovascular indications, sidelining much of its previous attention on hematology. The move was part of a broader prioritization in its adeno-associated virus platform. On Kresladi, analysts at Jefferies wrote in a note published Friday morning that while the launch itself provides just a “small” commercial opportunity for Rocket, the approval more broadly de-risks the company’s gene therapy platform and positions it well for future product approvals.