A phase 3 trial of Gamida Cell’s omidubicel in blood cancer patients has met its primary endpoint, sending the biotech’s stock up as much as 86% in premarket trading. The study found Gamida’s cells cut the time to neutrophil engraftment, suggesting the drug has benefits over umbilical cord blood.
Patients with hematological cancers such as leukemia and lymphoma undergo hematopoietic stem cell transplantation to restore bone marrow function. However, physicians are unable to source donor-matched cells for all patients, forcing them to rely on alternatives such as umbilical cord blood. As umbilical cord contains fewer stem and progenitor cells, engraftment can take longer, resulting in longer hospital stays and increased risk of infection.
Gamida thinks omidubicel, formerly known as NiCord, can cut the time to neutrophil engraftment. Now, the U.S.-Israeli biotech, which lists Novartis as its second biggest shareholder, has top-line phase 3 data to back up that hypothesis
The phase 3 enrolled 125 people with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma and randomized them to receive either omidubicel or standard umbilical cord blood.
Neutrophil engraftment took 12 days in patients who received omidubicel, as compared to 22 days in the control cohort, resulting in the trial hitting its primary endpoint with a p-value of 0.001. Gamida has previously said shortening engraftment by one week would “make a difference in the value proposition” of omidubicel.
In beating its seven-day target, Gamida has delivered data that hew closely to results from its phase 1/2 trial, which found neutrophil engraftment happened in people who took omidubicel in 11.5 days. The historical control used in the early phase trial put time to engraftment in patients who receive umbilical cord blood at 21 days.
Gamida is yet to share a look at the full phase 3 data and, given engraftment is a fast measure of efficacy, is likely still gathering results for other endpoints. The only other information shared so far is that the cell therapy was generally well tolerated and, on a per protocol basis, associated with a numerically higher rate of transplant success than umbilical cord blood, beating the control by 96% to 88%.
Having generated the results, Gamida plans to begin a rolling submission to the FDA in the fourth quarter. The results are also likely to trigger other activities, with Gamida Chief Financial Officer Shai Lankry telling investors in February that a positive phase 3 outcome would prompt the biotech to start additional clinical trials.
Bringing omidubicel to market in the U.S. would mark a high point for a biotech that has been through ups and downs in recent years. In 2014, Novartis was lined up to buy Gamida for $170 million upfront. That deal fell through, only for Novartis to quickly put together a revised agreement that saw it invest $35 million in Gamida and secure an option to buy the biotech. Less than one year later, Novartis turned down the option, despite not needing to make a decision at that time.