Kalytera Therapeutics (TSX-V:KALY) has received encouraging results from a Phase 2a study evaluating the safety and efficacy of cannabidiol (CBD), a primary constituent of the marijuana plant, for the treatment of acute (grades 3 and 4) graft-versus-host disease (GvHD).
In the present study, 10 patients with acute (Grade 3-4) GvHD that was refractory to standard treatment with high-dose steroids, were administered daily doses of CBD for up to three months. Nine of the 10 patients enrolled in the study responded to treatment; seven achieved complete remission, and two achieved a near-complete response.
Six patients are still alive with a median follow-up period of 13 months (range five-to-30 months). No patient deaths were determined to be associated with CBD treatment.
The preliminary study results compare favorably with the results of a historical control group of 29 patients with steroid-refractory Grade 3-4 GvHD, among which 26 patients died from GvHD and its complications.
Talent Biotechs, a privately held, Israeli-based developer of CBD therapeutics, recently acquired by Kalytera, conducted the latest study.
The ability to treat GvHD is a major unmet need. GvHD remains a major cause of morbidity and mortality after allogeneic hematopoietic cell transplantation. Typically, only 60% of patients respond to first-line therapy with high-dose steroids. The 12-month mortality rate among patients with steroid-refractory Grade 3 and 4 GvHD exceeds 60% and 80%, respectively.
The FDA has recommended that the sponsor of the study apply for both breakthrough therapy and fast track designations, each of which could accelerate the approval process.
"We are very excited about these results and the benefits to patients with no other treatment options," Dr. Andrew L. Salzman, CEO, said in a statement. "These results are significant in this disease setting, and we look forward to starting a comparator-controlled, randomized, multicenter Phase 2b study in the near-future."