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Minovia Therapeutics Announces FDA Clearance of Second IND Application, for a Phase II Clinical Trial of Lead Product MNV-201 in Pearson Syndrome

1. FDA clears IND application for Minovia's MNV-201 therapy. 2. MNV-201 targets Pearson Syndrome, aiming to improve patient growth outcomes. 3. Phase II trial enrolls pediatric patients with promising early data. 4. No effective therapies available for Pearson Syndrome; MNV-201 could be transformative. 5. Minovia aims to expand indications based on successful MNV-201 data.

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Why Bullish?

FDA clearance often leads to increased investor confidence and stock price. Historical data shows similar FDA approvals boost biotech firms temporarily but significantly.

How important is it?

FDA approvals directly correlate with future revenue potential; this could significantly affect Minovia's valuation.

Why Long Term?

Successful trials and potential market entry can lead to sustained growth. Prior cases in the biotech space indicate strong long-term market responses to promising therapies.

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MNV-201 is Minovia’s second generation mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria Rare Pediatric Designation granted MNV-201 is also being studied in a Phase Ib for low-risk Myelodysplastic Syndrome; Preliminary clinical data demonstrate safety and efficacy HAIFA, Israel, April 03, 2025 (GLOBE NEWSWIRE) -- 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. For more information visit clinicaltirals.gov About MNV-201MNV-201 is an autologous hematopoietic stem cell product enriched with allogeneic mitochondria. MNV-201 aims to restore mitochondrial function in patient hematopoietic stem cells, resulting in improved differentiation and function. Preclinical research suggests the potential for safe dosing with low immunogenicity risk and scalable manufacturing to address the significant number of patients who are potentially eligible for MNV-201 therapy. About Pearson SyndromePearson Syndrome is a multisystem progressive pediatric mitochondrial disease caused by single large-scale mitochondrial deletions (SLSMDS) of mitochondrial DNA (mtDNA), with consequent defects in the mitochondrial respiratory chain function. Pearson Syndrome classically presents in the first year of life with bone marrow failure and exocrine pancreatic dysfunction. Patients have macrocytic sideroblastic anemia that is frequently transfusion-dependent and may be accompanied by thrombocytopenia and neutropenia. Pancreatic dysfunction occurs secondary to fibrosis and leads to chronic diarrhea, malabsorption, and failure to thrive. Pearson Syndrome is marked by accumulating organ system involvement and worsening disease: variable other organ involvement can occur, including renal tubulopathy, liver cholestasis and/or fibrosis, adrenal insufficiency, diabetes mellitus, cardiomegaly, and/or cardiac conduction defects. Pearson Syndrome is universally fatal and since there is no effective therapy, the diagnosis of Pearson Syndrome is one of the worst diagnoses that a caregiver must deliver to parents of an affected infant. MNV-201 aims to reduce disease-associated symptoms and the risk of disease progression and death, thereby improving both lifespan and quality of life. About Minovia TherapeuticsMinovia Therapeutics Ltd. is a clinical stage biotechnology company advancing mitochondrial therapies for primary-genetic and age-related mitochondrial diseases. Minovia's clinical stage product candidate, MNV-201, is composed of mobilized peripheral blood, autologous CD34+ cells enriched with allogeneic, cryopreserved placental derived mitochondria, produced by Minovia's proprietary Mitochondrial Augmentation Technology (MAT). The enrichment of hematopoietic stem cells with healthy and functional mitochondria aims to restore stem cells function of patients suffering mitochondrial dysfunction, caused both by mtDNA mutations or deletions in pediatric patients suffering from primary mitochondrial diseases, or in adults with age-related diseases. MNV-201 is currently in clinical studies for pediatric patients with single-large scale mtDNA deletion syndromes (Pearson Syndrome and Kearn Sayre Syndrome) with five patients successfully dosed; and in Low Risk Myelodysplastic Syndrome. For more information, please visit www.minoviatx.com or follow the Company LinkedIn. Contact Information: Natalie Yivgi Ohana, Co-Founder and CEO Phone: +972-74-7039954 Email: info@minoviatx.com

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