Israeli-American Gamida hits goal in phase 3 blood cancer trial
The company uses umbilical cord blood, which like bone marrow and peripheral blood, is a rich source of stem cells for transplantation.
It is the first bone marrow transplant product to receive “Breakthrough Therapy Designation” from the US Food and Drug Administration, and according to its CEO Dr. Julian Adams, it has the potential to be the first FDA-approved bone marrow transplant graft.
Adams said that the company expects to begin submitting its biologics license application for Omidubicel to the FDA on a rolling basis in the fourth quarter of this year.
“We are very pleased with the results of the Phase 3 data reported, which moves us one step closer toward bringing potentially curative therapies to patients,” he said. “By the second half of 2021, Omidubicel could actually be available.”
The study specifically showed that the median time to neutrophil engraftment was 12 days for patients randomized to Omidubicel compared to 22 days for the comparator group. Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells.
Adams said that rapid neutrophil engraftment is associated with reconstituting the immune system more rapidly and allowing patients to leave the hospital sooner. He said every day immunocompromised patients are in the hospital they are at greater risk for infection and other complications.
The company uses umbilical cord blood, which like bone marrow and peripheral blood, is a rich source of stem cells for transplantation. For some patients, there may be advantages to having a cord blood stem cell transplant instead of a transplant with marrow or peripheral blood stem cells. However, Adams said that even though for 30 years the value of this cord blood was known, many times there were simply not enough donor cells. The Omidubicel technology expands the cells 30 to 50 fold to make a dose that can be administered to adults. This, he said, “solves the ability to reach those patients who cannot find adult genetically matched donors.”
Stem cell transplants are in high demand, with over 30,000 individuals in line for the procedure each year. It is estimated that between 40% and 70% of patients cannot find a matching donor and in some cases this lack of treatment can be fatal.
Cord blood banking helps to alleviate this issue, as their storage facilities make cord blood readily available for those in need. Moreover, in cord blood stem cell transplants, the donor and recipient can be mismatched making it a lot easier to find a donor.
Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of stem cells while preserving the cells’ functional therapeutic characteristics.
The international, multi-center, randomized Phase 3 study was designed to evaluate the safety and efficacy of Omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. The primary endpoint was time to neutrophil engraftment. Some 125 people with various forms of blood cancer between the ages of 12 and 65 participated.
The study showed that patients treated with omidubicel demonstrated more rapid neutrophil engraftment.
“These results have the potential to substantially move the field forward,” said Dr. Mitchell Horwitz, principal investigator and professor of medicine at the Duke Cancer Institute, “and represent an important step toward making stem cell transplantation more accessible and more successful for patients with lethal blood cancers.”