BMS’ Breayanzi met its primary endpoint in Phase 1/2 in multiple myeloma

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.

DEALS AND FINANCING

IASO Biotherapeutics raises $75 million in Series C1 round to fund CAR T launch

Cell therapy and antibody company Nanjing IASO Biotherapeutics raised a series C1 round of nearly $75 million to support its pipeline and the commercial launch of its first product, multiple myeloma therapy equecabtagene autoleucel. Shanghai Guoxin Investment & Development, Efung Capital, Shanghai Waigaoqiao Free Trade Zone Group, Nanjing Jiangbei New Area State-owned Asset Management, Housen Care Brothers and Hongcheng Investment participated in the financing. IASO said it is the first company to have an NDA accepted by China’s NMPA for a CAR T therapy that was fully developed in-house, and that the agency has also accepted an IND for the CAR T to treat neuromyelitis optica spectrum disorder.

CAR T cell durability company MPC raises CHF1.5M seed round

University of Geneva spinout MPC Therapeutics is targeting 2024 for the entry of its small molecule T cell therapy adjuvant program to the clinic, with a CHF1.5 million ($1.6 million) seed round led by London-based family office Plutus Investment Group. MPC’s small molecules improve the performance and durability of CAR T cell therapy by modulating mitochondrial function. They are also being studied to treat alopecia.