Gene editing giant CRISPR Therapeutics is moving away from multiple myeloma candidate CTX120, while a small, separate dataset released yesterday sent the company’s stock plummeting.
Held Tuesday, the Swiss company’s Innovation Day was supposed to showcase early research and development in its pipeline. But instead, a few headlines sent investors reeling.
At the beginning of the event, CRISPR’s newly appointed chief medical officer Phuong Khanh Morrow, M.D., announced a pivot from multiple myeloma candidate CTX120, an anti-B-cell maturation antigen (BCMA) therapy.
CTX120 did produce some dose-dependent encouraging responses in a phase 1 trial that launched in January 2020, said Morrow, who moved to CRISPR Tx from Amgen in May. The 80-patient study was looking for the incidence of adverse events as the main goal, according to the clinical trial registry.
She cited the high threshold for efficacy within the BCMA myeloma landscape as the main reason for moving away from the program, especially considering the FDA’s recent approval of Johnson & Johnson's Carvykti for adults with refractory or relapsed multiple myeloma.
The ultimate plan for CTX120 remains murky, with Morrow abstaining from any mention of continuing the trial, instead stating that the company will submit trial data to future scientific publications. Efforts will now be redirected to newly developed CTX121, which CRISPR Tx hopes will have extra potent edits and is engineered to improve efficacy and durability of response, according to Morrow.
She dubbed CTX121 a “next generation” program and revealed that the allogeneic CAR-T therapy is also being developed for multiple myeloma patients, though not much else about the therapy was shared. The new candidate has yet to be listed on CRISPR Tx’s site as part of the pipeline.
However, this pivot wasn’t what stole the show—instead, it was a small dataset for phase 1 program CTX130 that sent CRISPR Tx’s stock tumbling Tuesday afternoon, falling from $74.83 per share at 1 p.m. ET Tuesday to $64.67 at close. The trial, called COBALT-RCC, evaluated the efficacy of gene-edited allogeneic CAR-T therapy CTX130 in patients with relapsed or refractory renal cell carcinoma finding that only one of 14 patients demonstrated an overall response, meaning there was evidence that the tumor was destroyed or significantly reduced.
CTX130 demonstrated a 79% overall disease control rate across the 14 patients. The stable events are quite meaningful, especially since the patients consist of a heavily pretreated population, according to Sumanta Pal, M.D., principal investigator of the COLBALT-RCC trial, as well as professor of medical oncology and therapeutics research and co-director of the kidney cancer program at City of Hope, who spoke during a Q&A following the presentation.
During the Q&A, CRISPR leaders said it was encouraging and significant that even one person with a solid tumor experienced a complete response to an allogeneic CAR-T therapy, touting the feat as nearly unimaginable just three years ago.
As of 10:45 a.m. ET Wednesday morning, CRISPR Tx's stock stood at $59.21 per share.
The fall interrupted a four-day hot streak that began when CRISPR Tx presented data at the European Hematology Association Congress regarding exa-cel, previously known as CTX001. CRISPR Tx is developing the therapy with Vertex Pharmaceuticals for patients with transfusion-dependent beta thalassemia or severe sickle cell disease. The new data from 75 patients found that exa-cel is safe and effective up to three years after treatment.