Minovia Therapeutics recently announced dosing of the first patient in a Phase I/II clinical trial of the company’s Mitochondrial Augmentation Therapy (MAT) for the treatment of Pearson syndrome. According to Minovia, the first patient in the clinical trial for this pediatric mitochondrial disease was dosed at the Sheba Medical Center Hospital in Tel Aviv, Israel.
What does this mean for treatment of Pearson syndrome here in the U.S.? This investigational treatment has been granted Fast-Track, Orphan Drug and Rare Pediatric Disease designations by the FDA. Minovia is opening a U.S. operation in Cambridge, MA. The company intends to use its U.S. presence to expand its clinical and research collaborations with leading medical and academic institutions across North America, as well as with biotech and pharmaceutical companies focused on improving care for patients living with mitochondrial diseases.
According to Natalie Yivgi Ohana, Ph.D., Co-founder and CEO of Minovia Therapeutics, the expected outcome for the trial will be to enroll a total of seven patients by invitation and the primary outcome will be safety as determined by the number of participants with treatment-related adverse events at one year, as well as improvement in quality of life as measured by the International Pediatric Mitochondrial DiseaseScore (IPMDS) questionnaire. Secondary endpoints will include cognitive function, changes in brain MRI, changes in physical activity and muscle function, changes in weight and height, changes in blood normal mitochondrial DNA levels, number of metabolic crisis events or hospitalizations, and changes in peripheral blood lactate levels.
The announcement also states that the clinical and lab improvement evidenced in the first three patients treated by the team under compassionate use encourages us to continue with the current clinical study and in the future hopefully to implement this technique in the treatment of other mitochondrial diseases. Minovia has received approval from the IRB in Sheba Medical Center, as well as from the Israeli Ministry of Health to treat patients under compassionate use program. “The compassionate use program allowed patients access to our MAT in cases where there are no treatments available and the condition has a poor prognosis,” explains Ohana. “Each case is evaluated individually to ensure any use is ethical, in compliance with all regulations and has a strong scientific basis before proceeding. This was granted before the formal clinical trial started.”
Ohana explains that this is a small study — only 7 patients — in a very rare indication, but the company believes it will enroll rapidly because of the need and the positive responses seen in all four patients treated to date under the compassionate use program. “We view this as a proof-of-concept study which, if positive, would allow us to expand our clinical program to other mitochondrial diseases,” Ohana states. “We are also working to establish a collaboration with a leading clinical center in the U.S. to allow expanded access for more patients in need.”
As stem cell transplantations and cellular therapies continue to gain momentum rapidly, this is an example of trial that could pave the way for the development of treatments for all mitochondrial diseases.