Johnson & Johnson (J&J) is venturing further into the increasingly crowded in vivo CAR-T market, this time through an autoimmune drug development and potential acquisition deal with Sail Biomedicines.
Together, the duo will fare the seas of development for Sail’s lead immune-mediated disease programme, SAIL-0839, which is designed to CD4+ and CD8+ T-cells inside the body. According to John Reed, EVP of innovative medicine R&D at J&J, in vivo CAR-Ts could “fundamentally transform how immune-mediated diseases are treated” by potentially offering a simpler and more scalable solution than their autologous or allogeneic predecessors.
Through this deal, J&J has pledged to contribute $785m, including a $465m equity investment, plus a $140m additional payment, provided Sail can reach certain development milestones. If J&J so chooses, it now also holds the rights to acquire the Massachusetts-based biotech for $2.58bn.
J&J’s agreement sees the pharma giant double down in the in vivo cell therapy approach, which is currently experiencing a strong surge in interest within the wider pharma sector.
Recently, big names like AstraZeneca, Eli Lilly, AbbVie, Bristol Myers Squibb (BMS) and Gilead-owned Kite have all inked high-profile deals in the space, as companies hedge their bets on different delivery mechanisms that may offer the greatest safety and efficacy profile. Generally, companies hope the modality will overcome the manufacturing and logistical challenges linked to ex vivo approaches – thus potentially improving access to such therapies on a wider scale.
Before in vivo specialist Kelonia Therapeutics was bought out by fellow big pharma company Eli Lilly, J&J had entered into a collaboration with the biotech to harness its platform to develop next-generation in vivo CAR-Ts focused on J&J’s targets of interest.
Early data for in vivo approach looks promising
As many in the big pharma realm place their hopes on in vivo approaches, the industry must answer several questions around the safety and efficacy of this drug type in the clinic.
With the field progressing at a rapid pace, there are now several in vivo CAR-Ts in clinical development. This includes AstraZeneca-owned EsoBiotec’s multiple myeloma (MM) therapy, ESOT-01, which displayed promising efficacy but potentially concerning safety signals in a recent Phase I readout involving five patients.
Another company to achieve a positive outcome from its in vivo MM programme was Kelonia Therapeutics, which shared data from the Phase I inMMyCAR study (NCT07075185) on KLN-1010 revealing that the overall response rate (ORR) was 100% across all 18 evaluable patients, with all these individuals testing negative for minimal residual disease (MRD) one month after treatment. According to GlobalData analysts, the early positive safety results indicate that KLN-1010 could be administered in an outpatient setting in the future.
As in vivo CAR-Ts make their way through the clinical pipeline, it is yet to be seen which delivery mechanism will come out on top. Previously, GlobalData senior oncology analyst, Jack Cuthbertson, told Pharmaceutical Technology that lentiviral systems can effectively deliver complete responses (CRs) with good prognoses, but immune-related safety concerns could cloud their efficacy. Meanwhile, lipid nanoparticle (LNP)-based approaches may be more tolerable based on early data, but they also may not be able to deliver the sustained CRs ex vivo CAR-Ts are capable of.
