OS Therapies Provides Regulatory Update on Rare Pediatric Cancer Immunotherapy Candidate OST-HER2 for Human Osteosarcoma
OS Therapies (NYSE-A: OSTX) provided a regulatory update for its OST-HER2 immunotherapy candidate targeting osteosarcoma. The company outlined its regulatory timeline for 2025-2026, including planned interactions with FDA, MHRA, and EMA.
Key milestones include:
- FDA: Q1/25 regulatory communication, Q2/25 End of Phase 2 Meeting, Q3/25 rolling BLA submission, Q4/25 Conditional BLA
- MHRA: Scientific Advice Meetings and Conditional Marketing Authorization application in Q4/25
- EMA: Scientific Advice Meetings and Conditional Marketing Authorization application in Q1/26
The company has strengthened its regulatory infrastructure and confirmed sufficient cash through 2026. Additionally, OSTX reported progress in identifying biomarkers from its canine osteosarcoma program, which may predict treatment response in humans given the 96% genetic homology between human and canine osteosarcoma.
OS Therapies (NYSE-A: OSTX) ha fornito un aggiornamento normativo per il suo candidato immunoterapico OST-HER2, mirato all'osteosarcoma. L'azienda ha delineato la sua tempistica regolatoria per il 2025-2026, inclusi gli incontri pianificati con la FDA, la MHRA e l'EMA.
I principali traguardi includono:
- FDA: comunicazione regolatoria Q1/25, incontro di fine fase 2 Q2/25, presentazione della BLA rolling Q3/25, BLA condizionale Q4/25
- MHRA: incontri di consulenza scientifica e richiesta di autorizzazione all'immissione in commercio condizionale nel Q4/25
- EMA: incontri di consulenza scientifica e richiesta di autorizzazione all'immissione in commercio condizionale nel Q1/26
L'azienda ha rafforzato la sua infrastruttura regolatoria e ha confermato di avere liquidità sufficiente fino al 2026. Inoltre, OSTX ha riportato progressi nell'identificazione dei biomarcatori dal suo programma di osteosarcoma canino, che potrebbero prevedere la risposta al trattamento negli esseri umani, data la somiglianza genetica del 96% tra osteosarcoma umano e canino.
OS Therapies (NYSE-A: OSTX) proporcionó una actualización regulatoria para su candidato de inmunoterapia OST-HER2 dirigido al osteosarcoma. La empresa delineó su cronograma regulatorio para 2025-2026, incluyendo interacciones planificadas con la FDA, la MHRA y la EMA.
Los hitos clave incluyen:
- FDA: comunicación regulatoria en Q1/25, reunión de fin de fase 2 en Q2/25, presentación de BLA continua en Q3/25, BLA condicional en Q4/25
- MHRA: reuniones de asesoramiento científico y solicitud de autorización de comercialización condicional en Q4/25
- EMA: reuniones de asesoramiento científico y solicitud de autorización de comercialización condicional en Q1/26
La empresa ha fortalecido su infraestructura regulatoria y ha confirmado que tiene suficiente liquidez hasta 2026. Además, OSTX informó sobre avances en la identificación de biomarcadores de su programa de osteosarcoma canino, que podrían predecir la respuesta al tratamiento en humanos, dado el 96% de homología genética entre el osteosarcoma humano y canino.
OS Therapies (NYSE-A: OSTX)는 골육종을 표적으로 하는 OST-HER2 면역 요법 후보에 대한 규제 업데이트를 제공했습니다. 이 회사는 FDA, MHRA 및 EMA와의 계획된 상호작용을 포함하여 2025-2026년 규제 일정 개요를 설명했습니다.
주요 이정표는 다음과 같습니다:
- FDA: Q1/25 규제 소통, Q2/25 2상 종료 회의, Q3/25 순차적 BLA 제출, Q4/25 조건부 BLA
- MHRA: Q4/25 과학 자문 회의 및 조건부 마케팅 승인 신청
- EMA: Q1/26 과학 자문 회의 및 조건부 마케팅 승인 신청
회사는 규제 인프라를 강화하고 2026년까지 충분한 자금을 확보했다고 확인했습니다. 또한 OSTX는 개 골육종 프로그램에서 치료 반응을 예측할 수 있는 바이오마커를 식별하는 데 진전을 보였으며, 이는 인간과 개 골육종 간의 96% 유전적 동질성과 관련이 있습니다.
OS Therapies (NYSE-A: OSTX) a fourni une mise à jour réglementaire pour son candidat en immunothérapie OST-HER2 ciblant l'ostéosarcome. L'entreprise a décrit son calendrier réglementaire pour 2025-2026, y compris les interactions prévues avec la FDA, la MHRA et l'EMA.
Les principales étapes incluent:
- FDA : communication réglementaire au T1/25, réunion de fin de phase 2 au T2/25, soumission de BLA en continu au T3/25, BLA conditionnelle au T4/25
- MHRA : réunions de conseils scientifiques et demande d'autorisation de mise sur le marché conditionnelle au T4/25
- EMA : réunions de conseils scientifiques et demande d'autorisation de mise sur le marché conditionnelle au T1/26
L'entreprise a renforcé son infrastructure réglementaire et a confirmé avoir des liquidités suffisantes jusqu'en 2026. De plus, OSTX a signalé des progrès dans l'identification des biomarqueurs de son programme d'ostéosarcome canin, qui pourraient prédire la réponse au traitement chez les humains, étant donné l'homologie génétique de 96 % entre l'ostéosarcome humain et canin.
OS Therapies (NYSE-A: OSTX) hat ein regulatorisches Update für seinen Immuntherapie-Kandidaten OST-HER2 gegeben, der auf Osteosarkome abzielt. Das Unternehmen skizzierte seinen regulatorischen Zeitplan für 2025-2026, einschließlich geplanter Interaktionen mit der FDA, MHRA und EMA.
Wichtige Meilensteine sind:
- FDA: regulatorische Kommunikation Q1/25, Ende der Phase-2-Sitzung Q2/25, rollierende BLA-Einreichung Q3/25, bedingte BLA Q4/25
- MHRA: wissenschaftliche Beratungsgespräche und Antrag auf bedingte Marktzulassung im Q4/25
- EMA: wissenschaftliche Beratungsgespräche und Antrag auf bedingte Marktzulassung im Q1/26
Das Unternehmen hat seine regulatorische Infrastruktur gestärkt und bestätigt, dass es bis 2026 über ausreichende Mittel verfügt. Darüber hinaus berichtete OSTX über Fortschritte bei der Identifizierung von Biomarkern aus seinem Programm für osteosarkom bei Hunden, die möglicherweise die Behandlungsergebnisse bei Menschen vorhersagen können, da eine genetische Homologie von 96 % zwischen menschlichem und canine Osteosarkom besteht.
- Potential Priority Review Voucher eligibility through September 2026
- Sufficient cash runway into 2026
- Identification of new biomarkers that could expand treatment applications
- 96% genetic homology between human and canine osteosarcoma supporting biomarker strategy
- No immediate revenue generation as product still in development phase
- Multiple regulatory approvals still pending across different jurisdictions
Insights
OS Therapies has outlined an ambitious regulatory strategy for OST-HER2, their immunotherapeutic candidate for osteosarcoma. The company has initiated a multi-pronged approach simultaneously engaging with FDA, MHRA, and soon the EMA, signaling confidence in their clinical package.
The timelines presented are aggressive but structured, targeting potential FDA conditional approval via the Accelerated Approval Program by Q4 2025. This expedited pathway is often reserved for treatments addressing serious conditions with options – precisely the case for pediatric osteosarcoma. The company's eligibility for a Priority Review Voucher represents significant potential value (historically
Particularly noteworthy is the company's statement of having "sufficient cash on hand to operate into 2026," which theoretically covers their regulatory timeline. This removes immediate financing concerns that typically plague small biotechs during the approval process.
The parallel regulatory strategy across multiple jurisdictions is sophisticated for a company of this size (
The most scientifically significant aspect of this update is OS Therapies' discovery of potential treatment response biomarkers from their canine osteosarcoma program. This represents a compelling application of comparative oncology, leveraging the remarkable
These biomarkers could prove transformative by potentially:
- Expanding OST-HER2's application beyond the current focus on prevention/delay of recurrent lung metastatic osteosarcoma
- Enabling treatment of unresectable lung metastases
- Supporting limb-sparing approaches prior to surgical resection
The validation of canine-derived biomarkers as predictors of human response could significantly enhance patient selection, potentially improving efficacy signals in clinical trials. Regulatory bodies have previously accepted such translational biomarkers as surrogate endpoints for rare diseases where traditional clinical trials face recruitment challenges.
OST-HER2's mechanism as a listeria monocytogenes-based immunotherapy targeting HER2 represents an innovative approach to a pediatric cancer that has seen minimal therapeutic advancement in decades. The immunotherapy approach aims to prevent metastatic recurrence – addressing the primary cause of mortality in osteosarcoma patients who initially respond to standard therapy but later develop metastatic disease.
The recent patent allowance covering manufacturing improvements suggests process optimization that could enhance consistency and scalability – critical factors for biologics manufacturing.
- Dialogue open with FDA in preparation for End of Phase 2 Meeting request
- New canine elite responder biomarkers added to human biomarker strategy
“We are making rapid progress in putting together an appropriate data package to achieve accelerated approval for OST-HER2 given the feedback we’ve received to date from the FDA,” said Paul Romness, MHP, Chairman & CEO of OS Therapies. “We know one of the US government’s stated priorities is to treat deadly childhood cancers, and we believe that OST-HER2 for osteosarcoma aligns well with that mission.”
Regulatory Timelines
The Company initiated regulatory correspondence with the US Food & Drug Administration (FDA) and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) in the first quarter of 2025. The Company intends to initiate regulatory interaction with the European Medicines Agency (EMA) and EMA National Competent Authorities in the second quarter of 2025.
FDA
- Q1/25: Regulatory communication regarding endpoints for accelerated approval
- Q2/25: End of Phase 2 Meeting
- Q3/25: Initiation of rolling BLA submission
- Q4/25: Conditional BLA via Accelerated Approval Program
MHRA
- Q1/25: Scientific Advice Meeting requested and granted
- Q3/25: Scientific Advice Meeting with MHRA and ILAP application submission
- Q4/25: Application for joint Scientific Advice Meeting with MHRA and National Institute for Health and Care Excellence (NICE)
- Q4 25: MHRA Conditional Marketing Authorisation application.
EMA
-
Q2/25: EMA National Competent Authority Scientific Advice Meeting Request (Medicines Evaluation Board,
Netherlands ) - Q3/25: EMA PRIME and EMA Orphan Designation applications
- Q4/25: EMA-FDA Parallel Scientific Advice application
- Q1 26: EMA Conditional Marketing Authorisation application.
The Company has recently buttressed its regulatory and clinical strategy & operations infrastructure with the addition of key consulting agencies with significant track records in rare pediatric cancer, Priority Review Voucher (PRV) program approvals, and worldwide Conditional Market Access Applications including in the EU and
The Company reiterates its commitment to seeking to obtain FDA approval for OST-HER2 in the rare pediatric cancer osteosarcoma in 2025. The Company is eligible to receive a Priority Review Voucher (PRV) if OST-HER2 is approved under its Rare Pediatric Disease Designation (RPDD) by September 30, 2026.
The Company has sufficient cash on hand to operate into 2026.
Potential Treatment Response Biomarkers from Canine Osteosarcoma Program
The Company’s canine osteosarcoma program recently made significant progress in understanding which biomarkers likely drive anti-tumor activity in prevention of recurrence of metastases, metastasis treatment (lung resection is not standard of care in canines) and primary tumor treatment to achieve limb sparing in dogs. The data generated creates the potential for the eventual expansion of the uses of OST-HER2 in osteosarcoma into treatment of unresectable lung metastases and limb sparing prior to surgical resection.
The Company intends to evaluate these biomarkers as potentially predictive of treatment response in humans in preparation for regulatory discussions with FDA, MHRA and EMA. Canine equivalent biomarkers have previously been used as surrogate endpoints for rare diseases in Comparative Oncology, which is the study of diseases that occur in humans and animals and have significant genetic homology. Human and Canine osteosarcoma have
The Company is preparing to initiate regulatory correspondence with the United States Department of Agriculture (USDA) with the Company’s updated manufacturing process which incorporates key improvements covered under a patent that was recently granted a notice of allowance by the United States Patent & Trademark Office (USPTO).
Shelter Me is a film that was produced to highlight the important role of human and canine Comparative Oncology disease research. The movie will premiere on April 3, 2025 at AMC Century City in
About Shelter Me
Shelter Me: The Cancer Pioneers also features leading scientists at the National Cancer Institute/National Institutes of Health, the University of
A trailer of the movie can be viewed here.
About OS Therapies
OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other solid tumors. OST-HER2, the Company's lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the US Food & Drug Administration and Fast-Track and Orphan Drug designations from the US FDA and European Medicines Agency. The Company positive data in its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma demonstrating statistically significant benefit in the 12-month event free survival (EFS) primary endpoint of the study. The Company anticipates submitting a Biologics Licensing Application (BLA) to the US FDA for OST-HER2 in osteosarcoma in 2025 and, if approved, would become eligible to receive a Priority Review Voucher that it could then sell. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the
In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) and Drug Conjugates (DC), known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone Si-Linker and Conditionally Active Payload (CAP) technology, enabling the delivery of multiple payloads per linker. For more information, please visit www.ostherapies.com.
Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of the federal securities laws. These forward-looking statements and terms such as "anticipate," "expect," "intend," "may," "will," "should" or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. OS Therapies cautions readers that forward-looking statements are based on management’s expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially, including, but not limited to the approval of OST-HER2 by the US FDA and grant of a priority review voucher and other risks and uncertainties described in “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in the Company’s registration statement on Form S-1 filed with the Securities and Exchange Commission (the “SEC”) on November 12, 2024, as amended on November 27, 2024, and other subsequent documents we file with the SEC, including but not limited to our Quarterly Reports on Form 10-Q. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250331351474/en/
OS Therapies Contact Information:
Jack Doll
571.243.9455
Irpr@ostherapies.com
https://x.com/OSTherapies
https://www.instagram.com/ostherapies/
https://www.facebook.com/OSTherapies/
https://www.linkedin.com/company/os-therapies/
Source: OS Therapies
FAQ
What are the key regulatory milestones for OSTX's OST-HER2 osteosarcoma treatment in 2025?
How long does OSTX's current cash runway extend?
What is the significance of OSTX's canine osteosarcoma biomarker findings?
When is OSTX eligible to receive the Priority Review Voucher for OST-HER2?
