Sangamo Therapeutics, Inc.

Astellas has ended work on a cell therapy that it was exploring for CD20-positive B cell lymphomas, the Japan-based pharma confirmed to Endpoints News on Wednesday. The pharma giant ended a Phase 1 of ASP2802 after a continual evaluation of its resources, a spokesperson said in an emailed statement to Endpoints. “As a result of our prioritization efforts, we have made the difficult decision to terminate ASP2802,” the spokesperson said. “Through this work, we made important progress and have acquired drug development knowledge in key scientific fields, which will serve us well in the future as we evaluate other disease areas.” Astellas was creating an autologous cell therapy, or a treatment derived from a patient’s own cells. Biopharmas are looking to expand the field’s scope with allogeneic approaches that use cells from donors in a bid to make them more scalable and limit manufacturing hurdles. The therapy also contained what Astellas calls a MicAbody, which comprises an antibody-ligand fusion protein. “Arming T cells with MicAbodies enables control of their activity, so in comparison with conventional CAR-T therapy, we believe ASP2802 may offer less long-term toxicity and a more prolonged response,” Astellas has said in previous presentations. The cell therapy was created using technology from its $665 million buyout of CAR-T platform company Xyphos Biosciences in December 2019 . The Astellas spokesperson said the company “remain[s] committed to the Xyphos technology.” “Combining this technology with our capabilities in cell therapy that we have been working on so far, we can create next-generation high-function cells and maximize the value of our technology,” Astellas’ then-CEO Kenji Yasukawa said at the time. Xyphos, which is a subsidiary of Astellas, was active on the dealmaking front last year. It inked an in vivo cell therapy cancer deal with Kelonia Therapeutics and an off-the-shelf cell therapy tie-up for solid tumors with Poseida Therapeutics. Roche is expected to complete its acquisition of Poseida on Wednesday. Astellas didn’t submit an offer as part of Poseida’s M&A process, Endpoints reported last month. Astellas’ cell therapy pipeline also includes ASP7317, which is being explored for the blinding condition geographic atrophy secondary to age-related macular degeneration. The drugmaker has placed an emphasis on the gene therapy side over the years. It has multiple programs in the works and collaborations with Kate Therapeutics , AviadoBio and, as of last month, Sangamo .

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here . Our team is busy gearing up for this year’s JP Morgan Healthcare Conference in San Francisco. You can register here for our two-day event featuring conversations with gene editing pioneer Jennifer Doudna, former FDA leader Janet Woodcock, ARCH Venture Partners’ Robert Nelsen, and many more . — Nicole DeFeudis Neumora , ARCH’s “Really Big Neuroscience Company,” raised a ton of money on the promise of doing things differently in hard-to-treat indications. But its first test came back negative, with a Phase 3 depression study showing no statistical difference between the experimental drug and placebo. Neumora is running two additional identical pivotal trials, and some analysts believe the company only needs to succeed in two of the three studies. But Wall Street hammered the stock price, with Neumora shares falling 80% on Thursday. Nine-figure private financings dominated biotech in 2024, writes Senior Reporter Kyle LaHucik in a new analysis . There were 96 such funding rounds last year, according to LaHucik’s tally, approaching the single-year record of 106 from 2021. Most of the megarounds went to Series A and B financings, a sign that company launches could be getting more expensive, or investors want their initial dollars to support clinical trials. Most were US-based, and about one-third (33 companies) are fully focused on, or have a foot in, oncology. Pfizer is pulling back from its hemophilia A gene therapy partnership with Sangamo , the biotech announced this week. Pfizer touted positive Phase 3 results from the program this summer. But after getting two other hemophilia drugs approved this year, the pharma giant said there’s “limited interest in an additional gene therapy option in this patient population at this time.” The news sent Sangamo shares spiraling on Tuesday morning. The struggling biotech said it will seek out “all options” to commercialize its gene therapy, including a new partner. SPOTLIGHT CagriSema’s weight loss data disappointed. What does Novo do next? Regeneron buys London-based ocular biotech MORE IN R&D MORE IN PHARMA DON’T MISS

Sangamo Therapeutics said it “intends to explore all options to advance the program\" on which it was working with Pfizer.\n Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones.The Big Pharma paid $70 million to license the candidate in 2017 and handed over a further $55 million in milestones as it took the program through to a phase 3 readout last year. The pivotal trial met its primary endpoint, but, with BioMarin’s Roctavian showing clinical wins are no guarantee of commercial success in the space, Pfizer has decided to cut its losses rather than seek approval as previously planned.Pfizer’s decision sent Sangamo’s share price plummeting 56% to $1.02 on Dec. 31. Investors had flagged the progression of the partnered gene therapy, giroctocogene fitelparvovec, as a potential source of cash for Sangamo.The biotech ended September with around $39 million to its name. After factoring in a subsequent $10 million payment from Genentech, Sangamo said it only had the cash to fund operations into the first quarter of 2025. A deal with Astellas gave Sangamo a further $20 million last month, but the biotech is still racing toward the end of its cash runway. Milestones from Pfizer could have helped Sangamo navigate the cash crisis. Sangamo CEO Sandy Macrae, Ph.D., told investors on an earnings call in November that Pfizer could pay up to $220 million in regulatory and commercial milestones over the next two years.With Pfizer returning the rights to the gene therapy, Sangamo said it “intends to explore all options to advance the program, including seeking a potential new collaboration partner.” A new deal could secure Sangamo an upfront cash injection and an ongoing infusion of financial milestones, but Pfizer’s decision to pass on the program raises doubts about the attractiveness of the gene therapy to other drugmakers.The travails of BioMarin’s Roctavian have potentially weakened Sangamo’s hand. BioMarin broke new ground by winning FDA approval for the gene therapy in 2023 but has found few takers for the product. The biotech posted Roctavian sales of $16 million over the first nine months of 2024 and is now focused on cutting costs and growing revenues to the extent that the product is profitable.Roctavian’s commercial troubles suggest patients are content to stick with existing drugs such as Roche’s once-weekly treatment Hemlibra rather than join the vanguard of people trying gene therapies. Better gene therapies or commercialization strategies could tip the balance, but Pfizer has concluded that its dollars and attention are better spent on assets other than giroctocogene fitelparvovec.