Minovia Therapeutics Announces Interim Data from Phase 2 Trial in Pearson Syndrome Demonstrating No Treatment-Related Serious Adverse Events and Preliminary Signal for Efficacy Measured by Growth
Minovia Therapeutics (NASDAQ:LPAA) has reported positive interim results from its Phase 2 trial of MNV-201 for Pearson Syndrome, a fatal pediatric mitochondrial disease. The trial demonstrated no treatment-related serious adverse events and showed preliminary efficacy signals in growth parameters.
Key findings include improved growth parameters in two out of three initial patients at six-month follow-up, and enhanced quality of life scores. The company also reported favorable safety data from two compassionate use patients with Kearns-Sayre syndrome.
MNV-201 has received both Fast Track and Rare Pediatric Disease Designation from the FDA. Minovia plans to initiate registrational studies in 2026 and is merging with Launch One Acquisition Corp., with the combined entity expected to trade on Nasdaq by late 2025.
Minovia Therapeutics (NASDAQ:LPAA) ha riportato risultati intermedi positivi dal suo studio di Fase 2 su MNV-201 per la Sindrome di Pearson, una malattia mitocondriale pediatrica fatale. Lo studio ha evidenziato assenza di eventi avversi gravi correlati al trattamento e ha mostrato segnali preliminari di efficacia nei parametri di crescita.
I dati chiave includono un miglioramento dei parametri di crescita in due dei tre pazienti iniziali a sei mesi di follow-up, oltre a un aumento dei punteggi relativi alla qualità della vita. L'azienda ha inoltre riportato dati di sicurezza favorevoli da due pazienti trattati in uso compassionevole affetti da sindrome di Kearns-Sayre.
MNV-201 ha ottenuto sia la designazione Fast Track che quella per Malattie Pediatriche Rare dalla FDA. Minovia prevede di avviare studi registrativi nel 2026 e sta procedendo alla fusione con Launch One Acquisition Corp., con la società combinata che dovrebbe essere quotata al Nasdaq entro la fine del 2025.
Minovia Therapeutics (NASDAQ:LPAA) ha informado resultados interinos positivos de su ensayo de Fase 2 con MNV-201 para el Síndrome de Pearson, una enfermedad mitocondrial pediátrica fatal. El ensayo demostró ausencia de eventos adversos graves relacionados con el tratamiento y mostró señales preliminares de eficacia en parámetros de crecimiento.
Los hallazgos clave incluyen mejoras en los parámetros de crecimiento en dos de los tres pacientes iniciales a los seis meses de seguimiento, así como una mejora en las puntuaciones de calidad de vida. La compañía también informó datos de seguridad favorables de dos pacientes tratados bajo uso compasivo con síndrome de Kearns-Sayre.
MNV-201 ha recibido las designaciones de Fast Track y Enfermedad Pediátrica Rara por parte de la FDA. Minovia planea iniciar estudios registracionales en 2026 y está fusionándose con Launch One Acquisition Corp., con la entidad combinada que se espera cotice en Nasdaq a finales de 2025.
Minovia Therapeutics (NASDAQ:LPAA)는 치명적인 소아 미토콘드리아 질환인 피어슨 증후군을 대상으로 한 MNV-201의 2상 임상시험 중간 결과를 긍정적으로 보고했습니다. 이번 임상시험에서는 치료 관련 중대한 이상반응이 없었으며, 성장 지표에서 초기 효능 신호가 나타났습니다.
주요 결과로는 6개월 추적 관찰에서 초기 환자 3명 중 2명에서 성장 지표가 개선되었고, 삶의 질 점수도 향상되었습니다. 또한, 회사는 키언스-세이어 증후군 환자 2명에 대한 동정적 사용 사례에서 안전성 데이터도 긍정적으로 보고했습니다.
MNV-201은 FDA로부터 패스트 트랙 및 희귀 소아 질환 지정을 받았습니다. Minovia는 2026년에 등록 임상시험을 시작할 계획이며, Launch One Acquisition Corp.와 합병 중이며, 합병 법인은 2025년 말까지 나스닥에 상장될 예정입니다.
Minovia Therapeutics (NASDAQ:LPAA) a annoncé des résultats intermédiaires positifs de son essai de phase 2 sur MNV-201 pour le syndrome de Pearson, une maladie mitochondriale pédiatrique fatale. L'essai a démontré l'absence d'événements indésirables graves liés au traitement et a montré des signaux préliminaires d'efficacité sur les paramètres de croissance.
Les résultats clés incluent une amélioration des paramètres de croissance chez deux des trois premiers patients lors du suivi à six mois, ainsi qu'une amélioration des scores de qualité de vie. La société a également rapporté des données de sécurité favorables provenant de deux patients en usage compassionnel atteints du syndrome de Kearns-Sayre.
MNV-201 a reçu à la fois la désignation Fast Track et la désignation Maladie Pédiatrique Rare de la FDA. Minovia prévoit de lancer des études d'enregistrement en 2026 et est en cours de fusion avec Launch One Acquisition Corp., l'entité combinée devant être cotée au Nasdaq d'ici la fin 2025.
Minovia Therapeutics (NASDAQ:LPAA) hat positive Zwischenergebnisse aus seiner Phase-2-Studie mit MNV-201 bei Pearson-Syndrom, einer tödlichen pädiatrischen mitochondriale Erkrankung, berichtet. Die Studie zeigte keine behandlungsbedingten schwerwiegenden unerwünschten Ereignisse und erste Hinweise auf Wirksamkeit bei Wachstumsparametern.
Wichtige Ergebnisse umfassen verbesserte Wachstumsparameter bei zwei von drei Anfangspatienten nach sechs Monaten Nachbeobachtung sowie verbesserte Lebensqualitätswerte. Das Unternehmen berichtete zudem über günstige Sicherheitsdaten von zwei Patienten mit Kearns-Sayre-Syndrom, die eine Behandlung im Rahmen einer Ausnahmegenehmigung erhielten.
MNV-201 erhielt sowohl die Fast Track- als auch die Rare Pediatric Disease Designation von der FDA. Minovia plant, 2026 Zulassungsstudien zu starten und fusioniert mit Launch One Acquisition Corp., wobei das kombinierte Unternehmen voraussichtlich Ende 2025 an der Nasdaq gehandelt wird.
- No treatment-related serious adverse events (SAEs) reported in Phase 2 trial
- Two of three patients showed improved growth parameters at 6-month follow-up
- FDA granted both Fast Track and Rare Pediatric Disease Designation
- Potential eligibility for valuable pediatric priority review voucher (PRV)
- Upcoming Nasdaq listing through SPAC merger with Launch One
- Small patient sample size in trial (only three patients)
- Pediatric Review Voucher program currently on hold pending Congressional reauthorization
- Most adverse events associated with required apheresis procedure
Insights
Minovia's Phase 2 trial shows promising safety and preliminary efficacy for a fatal pediatric mitochondrial disease with FDA fast-track designation.
The interim data from Minovia's Phase 2 trial of MNV-201 for Pearson Syndrome represents a significant development in addressing an ultra-rare, fatal pediatric condition with no approved treatments. The trial has successfully met its primary safety endpoint with no treatment-related serious adverse events reported across the initial three patients and two compassionate use cases.
Most encouraging is the preliminary efficacy signal observed in two of three patients showing improved growth parameters at six-month follow-up, measured by height standard deviation score. This endpoint was specifically developed through a natural history study at CHOP, showing methodological rigor in trial design for this heterogeneous condition.
The dual regulatory designations from FDA (Fast Track and Rare Pediatric Disease) significantly enhance the development pathway. Fast Track status enables increased FDA interactions and potential rolling BLA submission, while the Rare Pediatric Disease designation positions Minovia for a potential priority review voucher upon approval - historically valued at
From a development timeline perspective, the company plans to complete enrollment by end of 2025 and is preparing for pivotal registrational studies in 2026. The parallel business combination with Launch One Acquisition Corp. (NASDAQ: LPAA) should provide public market access and potentially additional capital to support these advanced clinical stages.
For rare mitochondrial disorders like Pearson Syndrome, which manifests with sideroblastic anemia, growth failure, and pancreatic dysfunction, even modest clinical improvements can represent meaningful patient benefit. The quality of life improvements noted in two of three trial patients and both compassionate use Kearns-Sayre syndrome patients suggest potential broader applicability across mitochondrial deletion syndromes.
Data presented at the UMDF meeting highlighting first three of expected six subjects and two compassionate use patients provide encouraging results supporting continuation of trial
HAIFA, Israel, July 24, 2025 (GLOBE NEWSWIRE) -- Minovia Therapeutics Ltd. (“Minovia” or the “Company”), a clinical-stage biotechnology company developing novel therapies to treat mitochondrial diseases and combat age-related decline, announces positive interim results of the ongoing Phase 2 trial of the Company’s lead investigational compound MNV-201 in Pearson Syndrome, a fatal pediatric mitochondrial disease characterized by sideroblastic anemia (a disorder causing abnormal red blood cell development), failure to thrive and exocrine pancreas dysfunction. The interim analysis was presented at the United Mitochondrial Disease Foundation (UMDF) Annual Meeting on June 20, 2025. The analysis showed that MNV-201 has a positive safety profile even in this fragile patient population, currently meeting the Phase 2 trial’s primary safety endpoint.
Minovia Co-founder and CEO, Natalie Yivgi-Ohana, Ph.D., stated, “These Phase 2 data show extremely encouraging results, even in such a small patient sample. Pearson Syndrome is a devastating condition seen in young children. The clinical experience from our first-generation product helped identify a novel primary endpoint for this heterogenic group of patients and resulted in positive interactions with the FDA that could be leveraged towards MNV-201 development, enabling the current clinical trial. MAT’s unique mechanism of action provides a novel approach to addressing these devastating mitochondrial deletion syndromes. We look forward to finalizing patient recruitment and to providing the mitochondrial disease community with future updates on trial progress.”
In the presentation, Minovia’s Chief Scientific Officer Noa Sher reported no treatment related severe adverse events (SAEs) and no anti-mitochondrial antibodies were detected. In addition, all adverse events were transient in nature and the majority resolved within four days. Most adverse events were associated with the apheresis procedure necessary to procure patient-derived cells and conducted prior to the drug’s administration. In addition to safety data, Minovia is collecting preliminary efficacy data based on a novel endpoint identified through a natural history study performed at the Children’s Hospital of Philadelphia (CHOP) and supported by Minovia funding. Results indicate that at the six-month follow-up, two of the first three patients experienced improved growth parameters relative to those prior to treatment, as measured by height standard deviation score. Quality of life scores also showed improvement for two of the first three patients. The Company continues to enroll patients in the trial, which is expected to conclude before the end of 2025.
In addition to the three patients in the trial, the Company gathered data from two compassionate use patients affected by Kearns-Sayre syndrome (KSS), another mitochondrial deletion syndrome. In addition to a favorable safety profile, early data indicates improved quality of life scores for both patients.
The FDA has granted both Fast Track and Rare Pediatric Disease Designation to MNV-201, which is in Phase 2 clinical trials for the treatment of Pearson Syndrome, an ultra-rare and life-threatening mitochondrial disorder affecting children. The Company is advancing interactions with the FDA to align on a pivotal trial design and expects to initiate registrational studies in 2026.
FDA’s Fast Track Designation is designed to accelerate the development and review of therapies for serious or life-threatening conditions with unmet medical need. The designation provides Minovia with the opportunity for increased FDA interactions, potential eligibility for priority review, and the opportunity for a rolling submission of a future Biologics License Application (BLA) for MNV-201. Concurrently, Rare Pediatric Disease Designation (RPD) is granted to drugs which are under development for rare childhood diseases and provides the Company with the potential to receive a pediatric priority review voucher (PRV) if the drug is initially approved for that rare childhood disease. A PRV grants the holder an expedited six-month review of a new drug application. PRVs are tradeable and have historically commanded prices in excess of US
The Company also recently announced entry into a definitive business combination agreement (the “Business Combination Agreement”) with Launch One Acquisition Corp. (Nasdaq: LPAA, “Launch One”), a publicly traded special purpose acquisition company. Following the expected closing of the transaction contemplated by this Business Combination Agreement (the "Business Combination"), projected for late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq under a new ticker symbol.
About MNV-201
MNV-201 is a first-in-class cell therapy that uses Minovia’s proprietary Mitochondrial Augmentation Technology (MAT) to add healthy, energy-producing mitochondria into a patient’s own stem cells — aiming to restore organ function and improve health. In early-stage clinical studies, MAT has demonstrated a strong safety profile and signs of multi-system benefit in patients with Pearson Syndrome, including improvements in growth, muscle function, hematologic stability, and improved quality of life.
About Pearson Syndrome
Pearson Syndrome is caused by large-scale deletions in mitochondrial DNA (mtDNA) that impair the energy-generating function of cells, leading to bone marrow failure, metabolic crises, and organ dysfunction. With no approved therapies, current care is purely supportive, and patients die during childhood.
About Minovia Therapeutics
Minovia Therapeutics, chaired by John Cox, is a clinical-state biotechnology company working on treatments to replace dead or defective mitochondria with new healthy mitochondria, helping people with mitochondrial diseases and fighting aging. Its main treatment, MNV-201, is already being tested for Pearson Syndrome and Myelodysplastic Syndrome. Minovia is also developing ways to help people live longer, healthier lives. Based in Haifa, Israel, where it operates a GMP facility for mitochondrial drug substance and drug product manufacturing for clinical trials related to its therapy, Minovia is planning to expand to the U.S. For more information, visit www.minoviatx.com.
About Launch One Acquisition Corp.
Launch One Acquisition Corp. is a company set up to merge with and take public an exciting business in healthcare or technology. Listed on Nasdaq under the ticker LPAA, Launch One is led by experienced leaders who want to support game-changing solutions. For more information, contact Jurgen van de Vyver at jurgen@launchpad.vc.
Additional Information and Where to Find It
In connection with the Business Combination and the Business Combination Agreement, among Launch One, Minovia and Mito US One Ltd., a newly formed Israeli company limited by shares (“Pubco”), and certain other parties named therein. Launch One and Minovia intend to file relevant materials with the U.S. Securities and Exchange Commission ("SEC"), including a Registration Statement on Form F-4 of Pubco (the "Registration Statement"), which will include a proxy statement/prospectus of Launch One, and will file other documents regarding the proposed Business Combination with the SEC. This communication is not intended to be, and is not, a substitute for the proxy statement/prospectus or any other document that Launch One has filed or may file with the SEC in connection with the proposed Business Combination. The Registration Statement has not been filed or declared effective by the SEC. Following such filing and upon such declaration of effectiveness, the definitive proxy statement/prospectus contained within the Registration Statement and other relevant materials for the proposed Business Combination will be mailed or made available to stockholders of Launch One as of a record date to be established for voting on the proposed Business Combination.
Before making any voting or investment decision, investors and stockholders of Launch One are urged to carefully read, when they become available, the entire Registration Statement, the proxy statement/prospectus, and any other relevant documents filed with the SEC, as well as any amendments or supplements to these documents, and the documents incorporated by reference therein, because they will contain important information about Launch One, Minovia, Pubco and the proposed Business Combination. Launch One’s investors and stockholders and other interested persons will also be able to obtain copies of the Registration Statement, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, other documents filed with the SEC that will be incorporated by reference therein, and all other relevant documents filed with the SEC by Launch One and/or Pubco in connection with the Business Combination, without charge, once available, at the SEC’s website at www.sec.gov, or by directing a request to Launch One or Minovia at the addresses set forth below.
Participants In the Solicitation
Launch One, Minovia, Pubco and their respective directors, executive officers, other members of management and employees may be deemed participants in the solicitation of proxies from Launch One’s stockholders with respect to the Business Combination. Investors and security holders may obtain more detailed information regarding the names, and interests in the Business Combination, of Launch One’s directors and officers in Pubco's and Launch One’s filings with the SEC, including, when filed with the SEC, the preliminary proxy statement/prospectus, the definitive proxy statement/prospectus, amendments and supplements thereto, and other documents filed with the SEC. Such information with respect to Minovia’s directors and executive officers will also be included in the proxy statement/prospectus. You may obtain free copies of these documents as described above under the heading "Additional Information and Where to Find It."
Non-Solicitation
This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the potential transaction and shall not constitute an offer to sell or a solicitation of an offer to buy the securities of Launch One, Pubco, or Minovia, nor shall there be any sale of any such securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of the Securities Act of 1933, as amended.
Forward-Looking Statements
This press release includes certain statements that may be considered forward-looking statements within the meaning of the federal securities laws. Forward-looking statements include, without limitation, statements about future events or Minovia’s, Launch One's, or Pubco's future financial or operating performance. For example, statements regarding the development and regulatory approval of MNV-201, the implications of Fast Track Designation, RPD and PRVs and the timing of future clinical trials or potential applications are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may,” “should,” “could,” “might,” “plan,” “possible,” “project,” “strive,” “budget,” “forecast,” “expect,” “intend,” “will,” “estimate,” “anticipate,” “believe,” “predict,” “potential” or “continue,” or the negatives of these terms or variations of them or similar terminology.
These forward-looking statements regarding future events and the future results of Minovia or Launch One are based on current expectations, estimates, forecasts, and projections about the industry in which Minovia or Launch One operates, as well as the beliefs and assumptions of Minovia’s and Launch One's management. These forward-looking statements are only predictions and are subject to, without limitation, (i) known and unknown risks, including the risks and uncertainties indicated from time to time in the final prospectus of Launch One relating to its initial public offering filed with the SEC, including those under “Risk Factors” therein, and other documents filed or to be filed with the SEC by Launch One or Pubco; (ii) uncertainties; (iii) assumptions; and (v) other factors beyond Minovia’s or Launch One's control that are difficult to predict because they relate to events and depend on circumstances that will occur in the future. They are neither statements of historical fact nor promises or guarantees of future performance. Therefore, Minovia’s actual results may differ materially and adversely from those expressed or implied in any forward-looking statements and Minovia and Launch One therefore caution against relying on any of these forward-looking statements.
These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Minovia and its management, as the case may be, are inherently uncertain and are inherently subject to risks, variability and contingencies, many of which are beyond Minovia’s or Launch One's control. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (i) the occurrence of any event, change or other circumstances that could give rise to the termination of the Business Combination Agreement and any subsequent definitive agreements with respect to the Business Combination; (ii) the outcome of any legal proceedings that may be instituted against Launch One, Minovia, Pubco, or others following the announcement of the Business Combination and any definitive agreements with respect thereto; (iii) the inability to complete the Business Combination due to the failure to obtain consents and approvals of the shareholders of Launch One and Minovia, to obtain financing to complete the Business Combination or to satisfy other conditions to closing, or delays in obtaining, adverse conditions contained in, or the inability to obtain necessary regulatory approvals required to complete the transactions contemplated by the Business Combination Agreement; (iv) changes to the proposed structure of the Business Combination that may be required or appropriate as a result of applicable laws or regulations or as a condition to obtaining regulatory approval of the Business Combination; (v) projections, estimates and forecasts of revenue and other financial and performance metrics, projections of market opportunity and expectations, and the estimated implied enterprise value of Minovia; (vi) Minovia’s ability to scale and grow its business, and the advantages and expected growth of Minovia; (vii) Minovia’s ability to source and retain talent, and the cash position of Minovia following closing of the Business Combination; (viii) the ability to meet stock exchange listing standards in connection with, and following, the consummation of the Business Combination; (ix) the risk that the Business Combination disrupts current plans and operations of Minovia as a result of the announcement and consummation of the Business Combination; (x) the ability to recognize the anticipated benefits of the Business Combination, which may be affected by, among other things, competition, the ability of Minovia to grow and manage growth profitably, maintain key relationships and retain its management and key employees; (xi) costs related to the Business Combination; (xii) changes in applicable laws, regulations, political and economic developments; (xiii) the possibility that Minovia may be adversely affected by other economic, business and/or competitive factors; (xiv) Minovia’s estimates of expenses and profitability; (xv) the failure to realize estimated shareholder redemptions, purchase price and other adjustments; and (xvi) other risks and uncertainties set forth in the filings by Launch One and Minovia with the SEC. There may be additional risks that neither Launch One nor Minovia presently know or that Launch One and Minovia currently believe are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. Any forward-looking statements made by or on behalf of Launch One or Minovia speak only as of the date they are made. Neither Launch One nor Minovia undertakes any obligation to update any forward-looking statements to reflect any changes in their respective expectations with regard thereto or any changes in events, conditions or circumstances on which any such statements are based.
Contacts:
Minovia Therapeutics Ltd.
Natalie Yivgi Ohana, Co-Founder and CEO
+972-74-7039954
info@minoviatx.com
Launch One Acquisition Corp.
Jurgen van de Vyver
jurgen@launchpad.vc
+1-510-692-9600
Investor Relations
Dave Gentry, CEO
RedChip Companies
+1-407-644-4256
LPAA@redchip.com
Investor Relations
Jules Abraham
Managing Director, Communications
CORE IR
1-212-655-0924
Julesa@coreir.com
FAQ
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