Eight years after Novartis AG gained U.S. FDA approval of the first CAR T therapy, Kymriah (tisagenlecleucel), for B-cell acute lymphoblastic leukemia, developers are advancing prospects that could significantly impact another disease space outside of cancer – autoimmunity. The efforts are getting a swirl of attention, with Bristol Myers Squibb Co. (BMS) announcing Oct. 10 that it would offer $1.5 billion in cash to buy three-year-old privately held Orbital Therapeutics Inc., including its lead, next-generation CAR T-cell therapy OTX-201, which is designed to reprogram cells in vivo for autoimmune diseases.
Coming off a presentation at the European Alliance of Associations for Rheumatology meeting, where a single report of disease recurrence in a lupus patient overshadowed promising early data for Kyverna Therapeutics Inc.’s autoimmune candidate, KYV-101, the Emeryville, Calif.-based company rallied on news of a case report describing the first use of the CD19-targeting CAR T-cell therapy in a patient with stiff-person syndrome, a rare, progressive neurological autoimmune disorder for which there is limited treatment.
CAR T-cell therapies have worked well at curing blood malignancies, but a group out of the University Hospital of Erlangen have repurposed the technology as a treatment for autoimmune diseases. The expansion into new diseases has required cooperation between multiple departments, with CAR T experts taking the lead on treatment and potential side effects, and rheumatologists measuring the outcomes of the treatment.
Cabaletta Bio Inc. has announced CABA-201, a newly designed, fully human CD19 chimeric antigen receptor (CAR) containing a 4-1BB co-stimulatory domain.
Sanofi SA’s high-profile phase III blow-up Sept. 9 with the oral BTK inhibitor rilzabrutinib in pemphigus brought fresh attention to the group of rare diseases – which cause blisters on the skin and mucous membranes throughout the body – and to players pushing for a new treatment.
Roughly 40 years after Bio-Response launched the first-ever biotech IPO, three companies priced IPOs on Friday, riding a wave of momentum that has put 2019 into second place for the most IPO money raised in a single year. Only 2018's record $10.7 billion is beyond this year's total: 54 global IPOs raising $7.98 billion.
