The European Medicines Agency granted an orphan drug designation to devimistat for the treatment of patients with relapsed or refractory Burkitt lymphoma. The novel compound targets mitochondria in tumor cells.
The European Medicines Agency has granted an orphan drug designation to devimistat (CPI-613), a novel compound targeting mitochondria, for the treatment of patients with relapsed or refractory Burkitt lymphoma.1
The intravenous lipoate analog was developed to shut down the mitochondria, which creates energy and can result in the growth of tumor cells. These cells are known to be more susceptible to the turn-off process compared with healthy cells.
The agent is under investigation in patients with relapsed or refractory Burkitt lymphoma or leukemia or high-grade B-cell lymphoma with high-risk translocations as part of a multicenter, open-label, single-arm, phase 2 trial (NCT03793140).2
“Relapsed or refractory Burkitt lymphoma can be a devastating form of cancer that is in need of effective treatment options,” Sanjeev Luther, president and chief executive officer of Rafael Pharmaceuticals, stated in a press release. “Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to.”
The trial enrolled previously treated patients into the following 2 cohorts: those with Burkitt lymphoma and those with high-grade B-cell lymphoma who harbor MYC and BCL-2 (DHL) and/or BCL-6 (THL) rearrangements.3
To participate, patients needed to be at least 18 years of age, have had at least 1 prior line of therapy fail, have had a prior bone marrow transplant fail or have been ineligible for one, and have had an ECOG performance status of 0 to 3. Patients also needed to have measurable disease or isolated bone marrow involvement and undergone stem cell transplant for at least 3 months before enrollment.
If patients had received a chemotherapy regimen with stem cell support in the 2 months prior to the trial, had any medical condition that was clinically unstable despite present therapy, or had platelet counts that were less than 50,000/mm3, unless attributable to marrow based, they were excluded. Patients with central nervous system parenchymal disease were excluded, but if patients had cerebrospinal fluid cleared for more than 4 weeks and they were receiving maintenance intrathecal therapy, they were permitted.
In the trial, patients received induction treatment with intravenous devimistat at a daily dose of 2500 mg/m2 on days 1 through 5 for the first 2 treatment cycles, followed by devimistat maintenance treatment, which was given on days 1 through 5 for all cycles thereafter.
The primary end point of the trial is overall response rate, and important secondary end points include duration of response, progression-free survival, and overall survival. Investigators are also seeking to understand whether the regimen could serve as a bridge to autologous stem cell transplant or allogeneic stem cell transplant.
Previously, in June 2018, devimistat was granted an orphan drug designation for use in the treatment of patients with Burkitt lymphoma.4