CAMBRIDGE, Mass. & HAIFA, Israel--(BUSINESS WIRE)--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
About Pearson Syndrome
Pearson syndrome is caused by a deletion in the mitochondrial DNA and affects approximately 100 children globally. These deletions lead to poor energy production by the cells in the body. Pearson syndrome affects many parts of the body but especially the bone marrow and the pancreas. Pearson syndrome affects the cells in the bone marrow (hematopoietic stem cells) that produce red blood cells, white blood cells, and platelets. Pearson syndrome also affects the pancreas, which can cause frequent diarrhea and stomach pain, trouble gaining weight, and diabetes. Some children with Pearson syndrome may also have problems with liver, kidney, heart, eye, ear, and/or brain function. Many children with Pearson syndrome die during infancy. Some children may survive into later childhood, but may go on to develop Kearns-Sayre syndrome.1
About Mitochondrial Diseases
Mitochondrial diseases are chronic, genetic, often inherited disorders that occur when mitochondria fail to produce enough energy for the body to function properly. Mitochondrial diseases can be present at birth but can also occur at any age.
Mitochondrial diseases can affect almost any part of the body, including the cells of the brain, nerves, muscles, kidneys, heart, liver, eyes, ears or pancreas.
Mitochondrial dysfunction occurs when the mitochondria do not work as well as they should due to another disease or condition. Many conditions are related to mitochondrial dysfunction, including Alzheimer’s disease, muscular dystrophy, Lou Gehrig’s disease and diabetes.2
Minovia Therapeutics, a clinical-stage international biotechnology research company, is the first company to use mitochondrial cell therapy to treat mitochondrial diseases through our Mitochondrial Augmentation Therapy (MAT) platform. MAT increases the level of normal mitochondrial DNA by using autologous stem cells enriched with blood-derived mitochondria, with the goal of extending and enhancing human lives. Our initial clinical focus is on rare mitochondrial diseases for which there are no approved treatments, such as Pearson syndrome, a fatal pediatric disease, as well as Kearns Sayre syndrome, MELAS and Leigh syndrome. Following a compassionate use program using MAT, Minovia is now conducting a clinical trial in Pearson syndrome. We are committed to exploring the full potential of our MAT platform to address a range of disorders associated with mitochondrial dysfunction. Minovia is headquartered in Haifa, Israel, with operations in Cambridge, Massachusetts. For more information, visit http://minovia.com/