Omidubicel Shows Further Trial Benefits for Marrow Transplants in AML, Other Cancers

Omidubicel Shows Further Trial Benefits for Marrow Transplants in AML, Other Cancers

Omidubicel, an experimental cell therapy for blood cancer patients needing a bone marrow transplant, is superior to standard umbilical cord blood transplant at increasing the proportion of patients achieving normal platelet counts within 42 days, according to new top-line data from a Phase 3 trial.

The study (NCT02730299) also showed omidubicel to be better than conventional cord blood transplants at lowering the number of patients who developed moderate to severe bacterial or fungal infections following the procedure, as well as the time they remained hospitalized. All three measures were secondary trial goals.

Based on these data, which will be more fully presented at a meeting later this year, Gamida Cell is planning to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) requesting omidubicel’s approval.

“These additional data reinforce the potential of omidubicel and move us another step closer toward bringing potentially curative therapies to patients,” Julian Adams, PhD, CEO of Gamida Cell, the therapy’s developer, said in a press release.

“[W]e are continuing our work to enable the submission of our biologics license application for omidubicel to the FDA on a rolling basis, both expected in the fourth quarter” of this year, Adams added.

Patients with different types of blood cancers being treated with high doses of chemo or radiation therapy often require bone marrow transplants to restore production of healthy blood cells, including red and white blood cells and platelets. This happens because these treatments can damage blood cell progenitors, called hematopoietic stem cells, which live in the bone marrow.

Statistics indicate that such transplants are not given to about 40% of eligible patients in the U.S. for reasons that include the lack of a matched donor. In others, a marrow transplant is not always effective, and can cause complications affecting quality of life, Gamida Cell stated in a press release.

Omidubicel, formerly known as NiCord, is an investigational cell therapy that aims to overcome the limitations of conventional bone marrow transplants by providing patients with a therapeutic dose of blood cell progenitors, while preserving their functional and therapeutic characteristics.

The Phase 3 trial is evaluating omidubicel’s safety and efficacy, compared with standard umbilical cord blood, in transplants given 125 patients with different types of blood cancers, including acute myeloid leukemia (AML). It is due to conclude in December.

Gamida Cell announced earlier this year that the trial met its primary goal: omidubicel’s superiority to umbilical cord blood at shortening the time, post-transplant, in which patients start producing and having normal levels of neutrophils (a type of white blood cell).

Those given omidubicel achieved these levels within a median of 12 days, while those given an umbilical cord blood transplant did so after 22 days, top-line data showed. Omidubicel was also found to be safe and well-tolerated in patients.

“Shortening the time to [neutrophil] engraftment is clinically meaningful, as it can reduce a patient’s time in the hospital and decrease likelihood of infection,” Mitchell Horwitz, MD, a professor of medicine at Duke Cancer Institute, and the trial’s principal investigator, said in the release.

New trial data reinforce these findings, by showing omidubicel’s superiority at increasing the number of patients achieving normal platelet counts within 42 days, lowering the number of those developing infections in the first 100 days after the transplant, and reducing the length of a hospital stay.

“These data, obtained in a global, randomized, multi-institutional setting could represent an important step forward in the field. In addition to more rapid platelet engraftment, a key step toward recovery, reducing infections and hospitalizations are considered meaningful patient outcomes and have the potential to provide substantial value for patients, their families and the healthcare system,” Horwitz said.

“The totality of these data strengthen my belief that omidubicel has the potential to be a graft source for any patient who does not have access to a matched related donor and could help make stem cell transplantation more accessible and more successful for patients with lethal blood cancers,” he added.

This Phase 3 trial built on previous data from a Phase 1/2 trial (NCT01816230), which showed omidubicel led to a faster neutrophil engraftment in 36 patients with different blood cancers, again compared with conventional umbilical cord blood transplant.

Omidubicel was the first bone marrow transplant product to be designated a breakthrough therapy by the FDA. It has also received orphan drug designation by U.S. and European regulatory authorities.

In addition to these trials, omidubicel is being tested in a Phase 1/2 trial (NCT03173937) in patients with severe aplastic anemia, a bone marrow disease in which the body becomes unable to produce new blood cells.