Thiogenesis Announces Presentation on UMDF Webcast Featuring Clinical Trials in Mitochondrial Disease
Thiogenesis Therapeutics presented updates on two clinical programs in mitochondrial disease at the UMDF Webcast. The company received acceptance of its Clinical Trial Application Part I for MELAS in Europe and submitted Part II in December 2024, planning a Phase 2 trial with 12 patients in Netherlands and France by Q1 2025.
For Leigh Syndrome (LS), following a pre-IND meeting with the FDA in November 2024, the company is filing an IND and plans to initiate a Phase 2a trial in Q1 2025. The company's lead product, TTI-0102, targets oxidative stress in both conditions. MELAS affects approximately 15,000 patients in the US and 20,000 in the EU, while LS occurs in about 1/40,000 births.
Thiogenesis Therapeutics ha presentato aggiornamenti su due programmi clinici per le malattie mitocondriali durante il Webcast dell'UMDF. L'azienda ha ricevuto l'accettazione della sua domanda di sperimentazione clinica Parte I per MELAS in Europa e ha presentato la Parte II nel dicembre 2024, pianificando una sperimentazione di Fase 2 con 12 pazienti nei Paesi Bassi e in Francia entro il primo trimestre del 2025.
Per la Sindrome di Leigh (LS), dopo un incontro pre-IND con la FDA nel novembre 2024, l'azienda sta presentando una richiesta IND e prevede di avviare una sperimentazione di Fase 2a nel primo trimestre del 2025. Il prodotto principale dell'azienda, TTI-0102, mira a ridurre lo stress ossidativo in entrambe le condizioni. MELAS colpisce circa 15.000 pazienti negli Stati Uniti e 20.000 nell'UE, mentre la LS si verifica in circa 1 ogni 40.000 nascite.
Thiogenesis Therapeutics presentó actualizaciones sobre dos programas clínicos en enfermedades mitocondriales durante el Webcast de UMDF. La empresa recibió la aceptación de su Solicitud de Ensayo Clínico Parte I para MELAS en Europa y envió la Parte II en diciembre de 2024, planeando un ensayo de Fase 2 con 12 pacientes en los Países Bajos y Francia para el primer trimestre de 2025.
Para el Síndrome de Leigh (LS), tras una reunión pre-IND con la FDA en noviembre de 2024, la empresa está presentando un IND y planea iniciar un ensayo de Fase 2a en el primer trimestre de 2025. El producto principal de la empresa, TTI-0102, se dirige al estrés oxidativo en ambas condiciones. MELAS afecta a aproximadamente 15,000 pacientes en EE. UU. y 20,000 en la UE, mientras que LS ocurre en alrededor de 1 de cada 40,000 nacimientos.
Thiogenesis Therapeutics는 UMDF 웹캐스트에서 미토콘드리아 질환에 대한 두 가지 임상 프로그램 업데이트를 발표했습니다. 이 회사는 유럽에서 MELAS에 대한 임상 시험 신청서 1부를 승인받았으며 2024년 12월에 2부를 제출하여 2025년 1분기까지 네덜란드와 프랑스에서 12명의 환자를 대상으로 한 2상 시험을 계획하고 있습니다.
리 증후군(LS)에 대해서는 2024년 11월 FDA와의 사전 IND 회의 후 이 회사는 IND를 제출하며 2025년 1분기에 2a상 시험을 시작할 계획입니다. 회사의 주요 제품인 TTI-0102는 두 조건 모두에서 산화 스트레스를 목표로 합니다. MELAS는 미국에서 약 15,000명의 환자와 유럽에서 20,000명의 환자에게 영향을 미치며, LS는 약 40,000명 출생당 1명에게 발생합니다.
Thiogenesis Therapeutics a présenté des mises à jour sur deux programmes cliniques concernant les maladies mitochondriales lors du Webcast de l'UMDF. L'entreprise a reçu l'acceptation de sa demande d'essai clinique Partie I pour MELAS en Europe et a soumis la Partie II en décembre 2024, prévoyant un essai de Phase 2 avec 12 patients aux Pays-Bas et en France d'ici le premier trimestre de 2025.
Pour le Syndrome de Leigh (LS), suite à une réunion pré-IND avec la FDA en novembre 2024, l'entreprise dépose une demande d'IND et prévoit de commencer un essai de Phase 2a au premier trimestre 2025. Le produit phare de l'entreprise, TTI-0102, cible le stress oxydatif dans les deux conditions. MELAS concerne environ 15 000 patients aux États-Unis et 20 000 en UE, tandis que le LS survient chez environ 1 naissance sur 40 000.
Thiogenesis Therapeutics hat im UMDF-Webcast Aktualisierungen zu zwei klinischen Programmen bei mitochondrialen Erkrankungen vorgestellt. Das Unternehmen erhielt die Annahme seines Antrags auf klinische Prüfung Teil I für MELAS in Europa und reichte Teil II im Dezember 2024 ein, mit dem Plan, eine Phase-2-Studie mit 12 Patienten in den Niederlanden und Frankreich bis zum ersten Quartal 2025 durchzuführen.
Für das Leigh-Syndrom (LS) reicht das Unternehmen nach einem Pre-IND-Meeting mit der FDA im November 2024 einen IND-Antrag ein und plant die Initiierung einer Phase-2a-Studie im ersten Quartal 2025. Das führende Produkt des Unternehmens, TTI-0102, zielt auf oxidativen Stress bei beiden Erkrankungen ab. MELAS betrifft etwa 15.000 Patienten in den USA und 20.000 in der EU, während LS in etwa 1 von 40.000 Geburten auftritt.
- Clinical Trial Application Part I accepted for MELAS in Europe
- Phase 2 trials for both MELAS and Leigh Syndrome planned for Q1 2025
- Addressing significant market opportunities with 35,000 MELAS patients in US/EU combined
- None.
San Diego, California--(Newsfile Corp. - December 10, 2024) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing novel thiol compounds, including precursors to cysteamine, that have potent antioxidant and anti-inflammatory properties for mitochondrial diseases and related metabolic conditions is pleased to announce that Patrice Rioux, MD, Ph.D., Thiogenesis' Chief Executive Officer, presented an update on the Company's two clinical programs in mitochondrial disease at the United Mitochondrial Disease Foundation's ("UMDF") Webcast entitled, "Bench-to-Bedside" on December 9, 2024.
Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes ("MELAS")
On March 25, 2024, the Company announced the acceptance of its Clinical Trial Application Part I in Europe in MELAS. In December 2024, Thiogenesis submitted its Clinical Trial Application Part II which, when cleared, will allow the Company to commence a planned Phase 2 clinical trial in the Netherlands and France the first quarter of 2025. The randomized, placebo-controlled Phase 2 clinical trial will enroll 12 patients in total, 8 to be treated with Thiogenesis' lead product candidate, TTI-0102, and 4 with placebo. The primary endpoint will be a 12-minute walk test, with the measurement of relevant biomarkers as secondary endpoints.
MELAS is a rare, inherited mitochondrial disorder caused by mutations in mitochondrial DNA. Symptoms include seizures, muscle weakness, fatigue, loss of motor skills and intellectual disability. Oxidative stress plays an important role in the mitochondria and is a potential pathological mechanism of mitochondrial disease, that could be antagonized with TTI-0102, for the treatment of MELAS and other mitochondrial diseases. The prevalence of MELAS is estimated to be approximately 15,000 patients in the US and 20,000 in the EU.
Leigh Syndrome ("LS")
On July 18, 2024, the Company announced a collaboration with a leading US children's hospital to treat LS. In November 2024, the Company and the children's hospital jointly conducted a pre-investigational new drug ("pre-IND") meeting via conference call with the US Food and Drug Administration ("FDA") to discuss their proposed protocol for a Phase 2a clinical trial of TTI-0102 in LS. Incorporating feedback from the FDA, the Company is in the process of filing its IND for LS, and if cleared, anticipates initiating a Phase 2a clinical trial in the first quarter of 2025.
LS is a rare inherited genetic disease that results from mutations in both mitochondrial and nuclear DNA. It is one of the most debilitating of the mitochondrial diseases, usually diagnosed in infancy and occurs in an estimated 1/40,000 births. Initial symptoms for LS include impaired sucking/breastfeeding capability, loss of motor and communication skills, respiratory issues, poor muscle development, loss of appetite and seizures. Oxidative stress is a distinguishing feature of the disease, and there is currently no approved treatment for LS.
"It is a privilege to get the opportunity to support UMDF whenever possible as they provide invaluable services in increasing the profile of mitochondrial disease, patient and family support, and industry advocacy," said Patrice Rioux, MD, Ph.D., Thiogenesis' Chief Executive Officer. "Therefore, I was thrilled to get the opportunity to present updates on our MELAS and Leigh syndrome clinical programs on the UMDF Webcast."
About UMDF
The United Mitochondrial Disease Foundation's ("UMDF") mission is to promote research and education for the diagnosis, treatment and cure of mitochondrial disorders and to provide support to affected individuals and families. For more than 25 years, UMDF has built a network of the top clinicians, hospitals and researchers dedicated to fighting mitochondrial disease. It is driven by a nationwide community of ambassadors solely focused on supporting patients and families affected by mitochondrial disease. UMDF is committed to making a difference by funding the best science no matter where it is found in the world and providing critical programs and services to patients and their families.
About Thiogenesis
Thiogenesis Therapeutics, Corp. (TSXV: TTI) is a clinical-stage biopharmaceutical company operating through its wholly owned subsidiary based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Prodrugs are drugs that contain previously approved active ingredients and are modified so that they only become active when metabolized. For regulatory purposes prodrugs can use existing third-party safety data in regulatory submissions in the streamlined 505 (b)(2) regulatory pathway in the U.S., and its equivalent hybrid system in Europe, to proceed into human efficacy trials with regulatory clearance. Prodrugs may enhance the profile of the active ingredient to increase its bioavailability and reduce side effects. The Company's initial target indications include MELAS, Leigh syndrome, pediatric MASH and Rett syndrome.
For further information, please contact:
Brook Riggins, Director, and CFO
Email: info@thiogenesis.com
Tel.: (888) 223-9165
Forward Looking Statements
This news release contains certain forward-looking statements and forward-looking information (collectively referred to herein as "forward- looking statements") within the meaning of Canadian securities laws including, without limitation, statements with respect to the future investments by the Company. All statements other than statements of historical fact are forward-looking statements. Undue reliance should not be placed on forward-looking statements, which are inherently uncertain, are based on estimates and assumptions, and are subject to known and unknown risks and uncertainties (both general and specific) that contribute to the possibility that the future events or circumstances contemplated by the forward-looking statements will not occur. Although the Company believes that the expectations reflected in the forward-looking statements contained in this press release, and the assumptions on which such forward-looking statements are made, are reasonable, there can be no assurance that such expectations will prove to be correct. Readers are cautioned not to place undue reliance on forward-looking statements included in this document, as there can be no assurance that the plans, intentions, or expectations upon which the forward-looking statements are based will occur. By their nature, forward-looking statements involve numerous assumptions, known and unknown risks and uncertainties that contribute to the possibility that the predictions, forecasts, projections and other forward-looking statements will not occur, which may cause the Company's actual performance and results in future periods to differ materially from any estimates or projections of future performance or results expressed or implied by such forward-looking statements. The forward-looking statements contained in this news release are made as of the date hereof and the Company does not undertake any obligation to update publicly or to revise any of the included forward-looking statements, except as required by applicable law. The forward-looking statements contained herein are expressly qualified by this cautionary statement.
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this news release.
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/233143
FAQ
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