Minovia Therapeutics Announces FDA Fast Track and Rare Pediatric Disease Designations for MNV-201 in Pearson Syndrome
Minovia Therapeutics (NASDAQ:LPAA) has received two significant FDA designations for its lead compound MNV-201: Fast Track Designation and Rare Pediatric Disease Designation. The therapy is currently in Phase 2 clinical trials for treating Pearson Syndrome, an ultra-rare and life-threatening mitochondrial disorder affecting children.
The Fast Track Designation enables accelerated development and review processes, increased FDA interactions, potential priority review eligibility, and rolling BLA submission opportunities. The Rare Pediatric Disease Designation could qualify Minovia for a pediatric priority review voucher (PRV) upon initial approval, historically valued at over $100 million.
The company plans to initiate registrational studies in 2026 and has entered a business combination agreement with Launch One Acquisition Corp. Following the expected closing in late 2025, Minovia will trade on Nasdaq under a new ticker symbol.
Minovia Therapeutics (NASDAQ:LPAA) ha ottenuto due importanti designazioni dalla FDA per il suo composto principale MNV-201: la Designazione Fast Track e la Designazione per Malattie Pediatriche Rare. La terapia è attualmente in fase 2 di sperimentazione clinica per il trattamento della Sindrome di Pearson, una malattia mitocondriale ultra-rara e potenzialmente letale che colpisce i bambini.
La Designazione Fast Track consente uno sviluppo e una revisione accelerati, maggiori interazioni con la FDA, la possibilità di una revisione prioritaria e la presentazione scorrevole della domanda di autorizzazione biologica (BLA). La Designazione per Malattie Pediatriche Rare potrebbe permettere a Minovia di ottenere un voucher per la revisione prioritaria pediatrica (PRV) al momento della prima approvazione, storicamente valutato oltre 100 milioni di dollari.
L'azienda prevede di avviare studi registrativi nel 2026 ed ha stipulato un accordo di fusione con Launch One Acquisition Corp. Dopo la prevista chiusura a fine 2025, Minovia sarà quotata al Nasdaq con un nuovo simbolo di borsa.
Minovia Therapeutics (NASDAQ:LPAA) ha recibido dos designaciones importantes de la FDA para su compuesto principal MNV-201: Designación Fast Track y Designación para Enfermedades Pediátricas Raras. La terapia se encuentra actualmente en ensayos clínicos de fase 2 para tratar el Síndrome de Pearson, un trastorno mitocondrial ultra-raro y potencialmente mortal que afecta a niños.
La Designación Fast Track permite acelerar los procesos de desarrollo y revisión, aumentar las interacciones con la FDA, la posible elegibilidad para revisión prioritaria y la presentación continua de la solicitud BLA. La Designación para Enfermedades Pediátricas Raras podría calificar a Minovia para un vale de revisión prioritaria pediátrica (PRV) tras la aprobación inicial, valorado históricamente en más de 100 millones de dólares.
La compañía planea iniciar estudios registracionales en 2026 y ha firmado un acuerdo de combinación empresarial con Launch One Acquisition Corp. Tras el cierre previsto a finales de 2025, Minovia cotizará en Nasdaq bajo un nuevo símbolo bursátil.
Minovia Therapeutics (NASDAQ:LPAA)는 주력 화합물 MNV-201에 대해 두 가지 중요한 FDA 지정인 패스트 트랙 지정과 희귀 소아 질환 지정을 받았습니다. 이 치료제는 현재 어린이에게 영향을 미치는 극희귀 및 생명을 위협하는 미토콘드리아 질환인 피어슨 증후군 치료를 위한 2상 임상 시험 중입니다.
패스트 트랙 지정은 개발 및 심사 과정을 가속화하고, FDA와의 상호작용을 증가시키며, 우선 심사 자격 및 순차적 생물학적 제품 허가 신청(BLA) 기회를 제공합니다. 희귀 소아 질환 지정은 초기 승인 시 미노비아가 소아 우선 심사 바우처(PRV)를 받을 수 있게 하며, 이는 역사적으로 1억 달러 이상의 가치가 있습니다.
회사는 2026년에 등록 연구를 시작할 계획이며 Launch One Acquisition Corp.와 사업 결합 계약을 체결했습니다. 2025년 말 예상 마감 후 Minovia는 새로운 티커 심볼로 나스닥에 상장될 예정입니다.
Minovia Therapeutics (NASDAQ:LPAA) a obtenu deux désignations importantes de la FDA pour son composé principal MNV-201 : la désignation Fast Track et la désignation pour maladies pédiatriques rares. Le traitement est actuellement en essais cliniques de phase 2 pour traiter le syndrome de Pearson, une maladie mitochondriale ultra-rare et potentiellement mortelle qui touche les enfants.
La désignation Fast Track permet d'accélérer les processus de développement et d'examen, d'augmenter les interactions avec la FDA, d'éventuellement bénéficier d'une révision prioritaire et de soumettre la demande de licence biologique (BLA) de manière progressive. La désignation pour maladies pédiatriques rares pourrait permettre à Minovia d'obtenir un bon de révision prioritaire pédiatrique (PRV) lors de l'approbation initiale, historiquement valorisé à plus de 100 millions de dollars.
L'entreprise prévoit de lancer des études d'enregistrement en 2026 et a conclu un accord de fusion avec Launch One Acquisition Corp. Après la clôture prévue fin 2025, Minovia sera cotée au Nasdaq sous un nouveau symbole boursier.
Minovia Therapeutics (NASDAQ:LPAA) hat für seinen führenden Wirkstoff MNV-201 zwei bedeutende FDA-Designationen erhalten: Fast Track Designation und Rare Pediatric Disease Designation. Die Therapie befindet sich derzeit in Phase-2-Studien zur Behandlung des Pearson-Syndroms, einer ultra- seltenen und lebensbedrohlichen mitochondrialen Erkrankung bei Kindern.
Die Fast Track Designation ermöglicht beschleunigte Entwicklungs- und Überprüfungsprozesse, verstärkte FDA-Interaktionen, potenzielle Prioritätsprüfung und rollierende Einreichung des Biologics License Application (BLA). Die Rare Pediatric Disease Designation könnte Minovia bei der Erstzulassung einen pädiatrischen Prioritätsprüfungs-Gutschein (PRV) einbringen, der historisch mit über 100 Millionen US-Dollar bewertet wird.
Das Unternehmen plant, im Jahr 2026 registrierende Studien zu starten und hat eine Unternehmenszusammenschlussvereinbarung mit Launch One Acquisition Corp. abgeschlossen. Nach dem erwarteten Abschluss Ende 2025 wird Minovia unter einem neuen Börsenticker an der Nasdaq gehandelt werden.
- Received FDA Fast Track Designation for MNV-201, enabling accelerated development and review process
- Granted Rare Pediatric Disease Designation, potentially qualifying for valuable priority review voucher
- Successfully advancing Phase 2 clinical trials for Pearson Syndrome treatment
- Planned business combination with Launch One Acquisition Corp for Nasdaq listing
- Pediatric Priority Review Voucher program currently on hold pending Congressional reauthorization
- Registrational studies not expected to begin until 2026
Insights
FDA's Fast Track and Rare Pediatric Disease designations significantly enhance MNV-201's development prospects and potential market value.
The FDA's granting of Fast Track Designation for Minovia's MNV-201 represents a significant regulatory advantage that could accelerate the development timeline for this first-in-class mitochondrial cell therapy. This designation acknowledges both the serious nature of Pearson Syndrome and the lack of approved treatments, potentially allowing for more frequent FDA interactions, rolling review submissions, and priority review eligibility—all of which can reduce time-to-market by several months to years.
Equally important is the Rare Pediatric Disease Designation, which positions Minovia to potentially receive a pediatric priority review voucher (PRV) upon approval. These vouchers are tremendously valuable assets in the pharmaceutical industry, having historically sold for
The company is strategically advancing from their current Phase 2 clinical trials toward pivotal studies, with plans to finalize trial design through FDA interactions and initiate registrational studies in 2026. This regulatory momentum comes at a pivotal time as Minovia progresses toward becoming publicly traded through its business combination with Launch One Acquisition Corp.
For a clinical-stage biotech developing therapies for ultra-rare diseases, these FDA designations substantially de-risk the regulatory pathway while potentially compressing the timeline to commercialization. The validation from these designations may also strengthen Minovia's position in attracting partnerships or additional funding as they advance their mitochondrial medicine platform.
Regulatory Milestone Underscores Urgency and Promise of First-in-Class Mitochondrial Cell Therapy
Haifa, ISRAEL, June 30, 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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to the Company’s lead investigational compound, MNV-201. The FDA has also granted 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.
“Both Fast Track Designation and Pediatric Rare Disease Designation are critical milestones for Minovia, as they strongly validate the clinical approach for our science, while also acknowledging the urgent need for new treatment options for Pearson Syndrome. Importantly, these FDA designations help us to decrease the potential time to market and provide additional benefits across the FDA process that will prove both medically and financially valuable,” said Minovia Co-founder and CEO, Natalie Yivgi-Ohana, Ph.D.
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
Minovia is currently conducting an IND-enabled Phase 2 clinical trial of MNV-201 in Pearson Syndrome. The Company is advancing interactions with the FDA to finalize a pivotal trial design and expects to initiate registrational studies in 2026.
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 expanding 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.
Contact
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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