Minovia Therapeutics מודיעה על אישור ה- FDA לבקשת IND לניסוי קליני שלב Ib של MNV-201 בתסמונת מיאלודיספלסטית בסיכון נמוך

MNV-201 הוא מוצר לטיפול בתאים מיטוכונדריאליים המורכב מתאי גזע המטופויטיים אוטולוגיים המועשרים במיטוכונדריה אלוגנית

במחקרים פרה-קליניים, MNV-201 הדגים פוטנציאל משופר לקליטה מחדש ולבנייה מחדש של מח עצם של תאי גזע המטופויטיים שמקורם במטופלים

נתוני מבחנה הדגימו גם יכולת משופרת להתמיין לתאים אריתרואידים, מה שתומך בפוטנציאל לשיפור בסמנים ביולוגיים של אנמיה

חיפה, 26 בספטמבר 2024 (GLOBE NEWSWIRE) –

Minovia Therapeutics Ltd, חברת ביו-פרמצבטיקה בשלב הקליני המקדמת טיפולים בתאים מיטוכונדריאליים למחלות מיטוכונדריאליות ראשוניות ומשניות, הודיעה היום כי מנהל המזון והתרופות האמריקני (FDA) אישר את בקשתה לתרופה ניסיונית חדשה (IND) עבור MNV-201, מוצר תאי גזע אוטולוגיים המטופויטיים המוגברים במיטוכונדריה אלוגנית. ה- IND תומך בייזום ניסוי קליני בחקר מינון שלב Ib של MNV-201 בחולים עם תסמונת מיאלודיספלסטית בסיכון נמוך (MDS).

Minovia Therapeutics Announces FDA Clearance of IND Application for a Phase Ib Clinical Trial of MNV-201 in Low Risk Myelodysplastic Syndrome

MNV-201 is a mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria

In pre-clinical studies, MNV-201 demonstrated improved engraftment and bone marrow reconstitution potential of patient derived hematopoietic stem cells

In vitro data also demonstrated improved ability to differentiate to erythroid cells, supporting potential for improvement in biomarkers of anemia

HAIFA, Israel, Sept. 26, 2024 (GLOBE NEWSWIRE) –

Minovia Therapeutics Ltd, a clinical stage biopharmaceutical company advancing mitochondrial cell therapies for primary and secondary mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its 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 Ib dose exploration clinical trial of MNV-201 in patients with Low Risk Myelodysplastic Syndrome (MDS).

Anemia is a common and serious symptom in patients with low-risk myelodysplastic syndrome (LR-MDS), affecting almost 90% of cases and is often the primary characteristic of the disease. Anemia in MDS can have a negative impact on quality of life and may correlate with decreased progression-free survival and overall survival.

“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 Ib clinical program for this first-in-class allogeneic mitochondrial therapy for low risk MDS patients,” said Natalie Yivgi Ohana, PhD, CEO of Minovia. “We are pleased to have safely dosed two MDS 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 MAT to improve anemia in this patient population.”

The Phase Ib clinical trial is an open-label, dose exploration study to evaluate the safety and efficacy of MNV-201 in subjects with low risk MDS. This trial will continue our campaign to evaluate dose exploration and safety of single or repeat dosing of MNV-201. The trial will also enable assessment of efficacy in improving anemia and durability of response. The study is expected to enroll at least three patients each in the low, medium and high dose cohorts, and up to a total of 15 patients in total. For more information visit clinicaltrials.gov.

About MNV-201

MNV-201 is an autologous hematopoietic stem cell product enriched with allogeneic mitochondria. MNV-201 aims to restore mitochondrial function in low risk MDS patient hematopoietic stem cells, resulting in improved differentiation to erythroid lineage with the potential to improve anemia. 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 Minovia Therapeutics

Minovia Therapeutics Ltd. is a clinical stage biotechnology company advancing mitochondrial cell 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 bone marrow failure disorders. MNV-201 is currently in clinical studies for pediatric patients with single-large scale mtDNA deletion syndromes (Pearson Syndrome and Kearn Sayre Syndrome) with four patients successfully dosed; and in Low Risk Myelodysplastic Syndrome. For more information, please visit www.minoviatx.com or follow the Company LinkedIn.

About Low Risk Myelodysplastic Syndrome

Myelodysplastic syndromes (MDS) are a group of bone marrow failures that occur when the blood-forming cells in the bone marrow become abnormal leading to an abnormal differentiation and production of one or more blood cell types. Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenias or progression to acute myeloid leukemia (AML). MDS is generally a disease that develops with aging; the median age at diagnosis of MDS is ~70 years.

Mitochondrial Dysfunction in MDS: Scientific literature shows a correlation between mitochondrial dysfunction and MDS progression. It is known that ineffective hematopoiesis in MDS results from increased susceptibility of clonal myeloid progenitors to apoptosis. This may be triggered by intrinsic factors, such as mitochondrial polarization due to iron retention in ringed sideroblasts. A subset of MDS patients present with sideroblastic anemia, a phenotype common in Pearson Syndrome patients and which implicates mitochondrial dysfunction of HSPCs as part of the pathology of MDS.

Contact Information: Natalie Yivgi Ohana, Co-Founder and CEO

Phone: +972-74-7039954

Email: [email protected]

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