Minovia Therapeutics Announces FDA Clearance of Second IND Application, for a Phase II Clinical Trial of Lead Product MNV-201 in Pearson Syndrome
Friday, April 04, 2025
Minovia Therapeutics Ltd, a clinical stage biopharmaceutical company advancing mitochondrial therapies for primary and secondary mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its second Investigational New Drug (IND) application for MNV-201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. The IND supports the initiation of a Phase II clinical trial of MNV-201 in pediatric patients with Pearson Syndrome, a primary mitochondrial disease.
Based on previous clinical experience from the 1st generation product, MNV-101 (autologous hematopoietic stem cell product augmented with syngeneic maternal mitochondria), Minovia designed this phase II study with change in growth (height SDS) as primary endpoint. According to the natural history study recently published by Dr. Rebecca Ganetzky from CHOP, all patients with Pearson Syndrome suffer from failure to thrive and do not respond to growth hormone. Natural history shows an annual reduction of 0.5 units in height SDS, while MNV-101 treated patients showed stabilization or improvement, with no decline of height SDS at the 6 and 12 month follow up time points in a comparable subset of patients. This change in growth correlated with an improved International Pediatric Mitochondrial Disease Scale (IPMDS), which measures how the patient feels and functions (R2=0.9; p=0.0036). Linear growth was also suggested as an objective and clinically meaningful endpoint for a pivotal trial in Pearson by the FDA in early interactions.
“The FDA’s clearance of our IND marks an important achievement for Minovia, allowing us to clinically evaluate our allogeneic mitochondrial cell therapy approach and proceed with the Phase II clinical program for this first-in-class allogeneic mitochondrial therapy for Pearson Syndrome patients,” said Natalie Yivgi Ohana, PhD, CEO of Minovia. “We are pleased to have safely dosed three Pearson patients enrolled in an ongoing study under the Israeli Ministry of Health. We look forward to treating additional patients under this IND, as well as to learning about the potential of MNV-201 to improve growth in this patient population.”
“We are pleased that our cumulative interactions with the FDA enabled alignment on requirements for the entire MNV-201 program, including preclinical, CMC, and clinical aspects,” said Noa Sher, PhD, CSO of Minovia. “Early clinical and regulatory experience with MNV-101 shaped the current program and enabled a successful IND submission.”
The Phase II clinical trial is an open-label, single dose study to evaluate the safety and efficacy of MNV-201 in pediatric subjects diagnosed with Pearson Syndrome. The trial will also enable assessment of efficacy in improving growth and quality of life. The study is expected to enroll three additional patients up to a total of 6 patients.
Source: globenewswire.com
