CAMBRIDGE, Mass. & HAIFA, Israel- Minovia Therapeutics today 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. The first patient in the clinical trial for this pediatric mitochondrial disease was dosed at the Sheba Medical Center Hospital in Tel Aviv, Israel.
The Pearson syndrome clinical study is the first ever mitochondrial cell therapy trial undertaken to treat a mitochondrial disease. This investigational treatment has been granted Fast-Track, Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration. Under the study protocol, autologous CD34+ cells enriched with blood-derived mitochondria manufactured by Minovia’s proprietary MAT platform will be transplanted via a single dose into pediatric patients with Pearson syndrome to increase the levels of normal mitochondrial DNA.
“This trial of MAT in Pearson syndrome is an important step toward addressing mutations and deletions of mitochondrial DNA, because there are many other mitochondrial diseases with no available therapies,” said Natalie Yivgi Ohana, Ph.D., Co-founder and Chief Executive Officer of Minovia. “If it is successful, it will not only provide improved quality of life for those living with Pearson syndrome; it could pave the way for the development of treatments for all mitochondrial diseases.”
Minovia will present case studies of three children with Pearson syndrome and one patient with Kearns-Sayre syndrome who have been treated to date. The presentation will take place at the Mitochondrial Medicine 2019 meeting of the United Mitochondrial Medicine Foundation at the Hilton Alexandria Mark Center in Arlington, Virginia, June 26-29. Treatment under the compassionate use program, which was provided at the Sheba Medical Center, was the basis for the Phase I/II clinical trial that was just launched.
“Although our main expertise is treating children with cancer, our vast clinical and lab experience with stem cell transplantations and cellular therapies enabled a smooth and uneventful entrance to the project,” said Professor Amos Toren M.D, Ph.D., MHA, head of the Division of Pediatric Hemato-oncology and Principal Investigator of the study, and Elad Jacobi, M.D., head of pediatric immunotherapy center co-PI. “The clinical and lab improvement evidenced in the first three patients treated by Dr Jacoby and 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 is opening a U.S. operation in Cambridge, Massachusetts. The company will 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.
Minovia has appointed John Cox as chairman of the board. Cox was previously the CEO of a global rare disease hematology company called Bioverativ which was recently sold to Sanofi for $11.6 billion.
“We look forward to establishing capabilities throughout the world, and the Boston biotech hub is an excellent place to start,” said Dr. Yivgi Ohana. “We also are fortunate that John Cox has assumed the chairmanship of Minovia. As we progress through clinical research and build international capabilities, his global experience in drug development, biotech manufacturing and commercial business management will be invaluable to our mission to help those with mitochondrial diseases.”
About the Phase I/II Study
The Pearson syndrome trial is a Phase I/II, open label, single dose clinical study to evaluate the safety and therapeutic effects of transplantation of MNV-BM-BLD, Minovia’s autologous CD34+ cells enriched with blood-derived mitochondria, in pediatric patients. The trial will 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 IPMDS questionnaire. Other outcomes measured at one year will include cognitive status, changes in brain MRI, changes in physical activity and muscle function, changes in weight, and changes in peripheral blood lactate levels. For more information: https://clinicaltrials.gov/ct2/show/NCT03384420