The stars have aligned for Bristol Myers Squibb and Orbital Therapeutics. Pushing deeper into in vivo cell therapies, BMS has agreed to pay $1.5 billion to buy the biotech for a pipeline led by a preclinical CD19 autoimmune program.
BMS established itself as a major player in the cell therapy market by acquiring Celgene in 2019. The deal covered autologous CAR-T cell therapies that originated at bluebird bio and Juno Therapeutics. Since then, the idea of triggering the production of CAR-T cells in patients, rather than engineering cells outside of the body, has gained more and more traction.
Orbital launched in 2022 with plans to advance RNA medicines and raised $270 million from Arch Venture Partners and other backers in 2023. Led by former Spark Therapeutics CEO Ron Philip, the biotech has identified in vivo CAR-T therapies as the first application of its RNA capabilities.
While Orbital plans to move into oncology, the traditional use case for CAR-T therapies, the company has made an autoimmune candidate its lead asset. Early evidence that ex vivo CD19 CAR-T cell therapies can transform outcomes in patients with hard-to-treat lupus has triggered a surge in interest in applying the modality to autoimmune diseases.
Orbital has put a twist on that idea by developing an in vivo CD19 CAR-T cell therapy. The candidate, OTX-201, consists of circular RNA encoding a CD19-targeted CAR delivered via targeted lipid nanoparticles. Once in the body, OTX-201 could initiate the production of CAR-T cells, eliminating the logistical complexity and conditioning regimens associated with ex vivo approaches.
Orbital is a long way from showing its approach works. The biotech is currently running IND-enabling studies with a view to having OTX-201 ready for clinical development in the first half of next year.
BMS has seen enough promise in the technology to pay $1.5 billion to expand in an area that has been on the radar of its cell therapy team. Lynelle Hoch, who leads BMS’ cross-functional cell therapy business, commented on in vivo technology at a Bernstein event last month. Hoch told analysts that her team has been investing in what it sees as a promising but early-stage modality.
“I think in vivo is an interesting platform. Obviously, it's much, much earlier and further out [than ex vivo], but I think that is another great concept of can you potentially give a patient almost like a vaccine shot and provide them that same kind of CAR expression,” Hoch said. “Early data looks super intriguing and interesting, and so we've been watching that field quite closely.”
BMS isn't alone in using M&A dollars to bulk up in this field. In June, AbbVie inked a $2.1 billion purchase of Capstan Therapeutics to add an early-phase autoimmune in vivo CAR-T drug to its pipeline. In addition, Gilead Sciences' Kite Pharma forked over $350 million for in vivo CAR-T player Interius BioTherapeutics in August, while AstraZeneca paid $1 billion for EsoBiotec and its in vivo lentiviral vector platform back in March.