Positive pivotal cell graft data bring Novartis-backed Boston biotech step closer to finish line
Gamida Cell — the Novartis-backed Israeli biotech developing a cell graft to help patients with blood cancer addressing limitations associated with bone marrow transplants — has its first taste of late-stage data, paving the way for US approval.
The therapy, called omidubicel, is comprised of stem cells, progenitor cells as well as dendritic cells extracted from the umbilical cord. It was tested in 125 patients aged 12-65 years suffering from high-risk lethal blood cancers. One group of patients undergoing a bone marrow transplant received omidubicel, while the comparator group was given a standard umbilical cord blood transplant.
The study was designed this way because many patients in need of bone marrow transplants do not get matched donors — and while unrelated donor umbilical cord blood can provide a sufficiently matched donor to practically all patients, such transplants are associated with a high risk of graft failure.
The main goal of the study was time to neutrophil engraftment: Neutrophils are the most common type of white blood cell, and engraftment refers to a process by which cells begin to grow and reproduce new blood cells. Typically, engraftment begins about 20 days post a bone marrow transplant, according to the NIH.
The median time to neutrophil engraftment was statistically significantly shorter (p<0.001) for patients who received omidubicel at 12 days, versus 22 days in the comparator group, the company said, adding that among patients who were transplanted per protocol, 96% of patients who received omidubicel achieved successful neutrophil engraftment, compared to 88% in the comparator group.
The graft was well-tolerated, and detailed data will be disclosed at a later date.
An application to market omidubicel, which has been granted the FDA’s breakthrough therapy status, is expected to be submitted on a rolling basis beginning the fourth quarter of this year, said CEO Julian Adams, a well-known scientist whose biotech experience spans stints at Millennium and Infinity.
Shares of the company, which went public in a $50 million IPO in 2018, shot up more than 46% to $6.64 in morning trading.