CLINICAL AND REGULATORY
BMS’s lisocabtagene maraleucel meets mark in CLL study
BMS said CAR T therapy Breyanzi lisocabtagene maraleucel met the primary endpoint of complete response rate compared with historical controls in the Phase 1/2 TRANSCEND CLL 004 study to treat relapsed or refractory chronic lymphocytic leukemia or small lymphocytic leukemia. BMS said it will discuss the results with health authorities. The treatment, which targets CD19 with a 4-1BB costimulatory domain, is approved to treat large B cell lymphoma. BMS believes it could reach $3 billion in annual peak sales, pending label expansions; it delivered $127 million in revenue across the first nine months of 2022.
Allogene gains on ALLO-715 data in multiple myeloma
Shares of Allogene Therapeutics gained 16 % on news that the company’s allogeneic CAR T cell therapy targeting BCMA, ALLO-715, may lead to durable responses – an outcome that has evaded most allogeneic cell therapies. In a Phase 1 study, a 320 x 106 dose of cells led to 17 (70.8%) responses among 24 multiple myeloma patients, with 11 (45.8%) achieving a very good partial response (VGPR) or better and six (26%) achieving a complete response. One patient in the study remained in complete response at nearly 12 months, and another remained in VGPR after 11 months.
Chinese NMPA reviewing Legend’s CAR-T therapy cilta-cel
Carvykti ciltacabtagene autoleucel (cilta-cel) from Legend Biotech and partner Janssen is a step closer to approval in China to treat multiple myeloma after NMPA accepted the company’s NDA application. No date for action by the agency was disclosed. Last February, Carvykti became the first Chinese CAR T therapy to be granted an FDA approval.
New data open door for Carvykti’s use in earlier multiple myeloma settings
Results from the Phase 3 CARTITUDE4 study suggest CAR T therapy Carvykti cilta-cel from Legend and Janssen could be used in earlier settings in the treatment paradigm for multiple myeloma. At the study’s first interim analysis, Carvykti showed a progression-free survival benefit, meeting its primary endpoint. CARTITUDE4 studied Carvykti plus combinations of Pomalyst pomalidomide, Velcade bortezomib and dexamethasone; or Darzalex daratumumab, Pomalyst and dexamethasone, in relapsed and lenalidomide-refractory multiple myeloma patients who had received one to three prior lines of therapy. Carvykti is approved in a fifth-line or later setting.
Cabaletta’s CAR T shows positive signals in myasthenia gravis
Cabaletta Bio and University of Pennsylvania researchers presented preclinical data on a Phase 1 CAR T cell candidate for the autoimmune disorder myasthenia gravis, dubbed MUSK-CAART, that targets B cells expressing antibodies against the autoantigen MUSK. The CAR, which contains 4-1BB–CD3z signaling domains, induced MUSK-specific B cell depletion in an experimental autoimmune myasthenia gravis mouse model, with no off-target interactions detected in vivo, in human cell screens or in membrane proteome arrays.