Theriva™ Biologics Announces U.S. FDA Guidance on Design of Phase 3 Study of VCN-01 for the Treatment of Metastatic Pancreatic Cancer
Theriva Biologics (NYSE American: TOVX) announced outcomes from a Type D meeting with the FDA regarding the Phase 3 study design of VCN-01 for metastatic pancreatic cancer treatment. The FDA advised against expanding the ongoing VIRAGE Phase 2b study into Phase 3, recommending instead a standalone Phase 3 study of VCN-01 with gemcitabine/nab-paclitaxel.
The FDA agreed with Theriva's proposed Phase 3 design and indicated that including additional standard-of-care chemotherapy was unnecessary. The meeting also addressed statistical elements for confirmatory clinical studies. The company recently completed target enrollment for the VIRAGE Phase 2b study and plans to request another FDA meeting after its completion to discuss Phase 3 protocol details.
Theriva Biologics (NYSE American: TOVX) ha annunciato i risultati di un incontro di Tipo D con la FDA riguardo al design dello studio di Fase 3 per il trattamento del cancro pancreatico metastatico con VCN-01. La FDA ha sconsigliato l'espansione dello studio VIRAGE di Fase 2b in Fase 3, raccomandando invece uno studio di Fase 3 autonomo per VCN-01 con gemcitabina/nab-paclitaxel.
La FDA ha concordato con il design proposto da Theriva per la Fase 3 e ha indicato che l'inclusione di chemioterapie standard aggiuntive non era necessaria. L'incontro ha anche affrontato elementi statistici per studi clinici di conferma. L'azienda ha recentemente completato l'arruolamento dell'obiettivo per lo studio VIRAGE di Fase 2b e prevede di richiedere un altro incontro con la FDA dopo il suo completamento per discutere i dettagli del protocollo di Fase 3.
Theriva Biologics (NYSE American: TOVX) anunció los resultados de una reunión de Tipo D con la FDA sobre el diseño del estudio de Fase 3 del VCN-01 para el tratamiento del cáncer de páncreas metastásico. La FDA desaconsejó ampliar el estudio VIRAGE de Fase 2b a Fase 3, recomendando en su lugar un estudio independiente de Fase 3 de VCN-01 con gemcitabina/nab-paclitaxel.
La FDA estuvo de acuerdo con el diseño de Fase 3 propuesto por Theriva e indicó que no era necesaria la inclusión de quimioterapia estándar adicional. La reunión también abordó elementos estadísticos para los estudios clínicos confirmatorios. La compañía completó recientemente la inscripción objetivo para el estudio VIRAGE de Fase 2b y planea solicitar otra reunión con la FDA después de su finalización para discutir los detalles del protocolo de Fase 3.
Theriva Biologics (NYSE American: TOVX)는 전이성 췌장암 치료를 위한 VCN-01의 3상 연구 설계와 관련하여 FDA와의 타입 D 회의 결과를 발표했습니다. FDA는 진행 중인 VIRAGE 2b상 연구를 3상으로 확대하는 것을 권장하지 않았으며, 대신 gemcitabin/nab-paclitaxel과 함께 VCN-01의 독립적인 3상 연구를 권장했습니다.
FDA는 Theriva가 제안한 3상 설계에 동의했으며, 추가 표준 치료 화학요법의 포함은 불필요하다고 밝혔습니다. 회의에서는 확증 임상 연구를 위한 통계적 요소에 대해서도 논의되었습니다. 이 회사는 최근 VIRAGE 2b상 연구의 목표 등록을 완료했으며, 완료 후 3상 프로토콜 세부 사항을 논의하기 위해 FDA에 또 다른 회의를 요청할 계획입니다.
Theriva Biologics (NYSE American: TOVX) a annoncé les résultats d'une réunion de type D avec la FDA concernant la conception de l'étude de Phase 3 de VCN-01 pour le traitement du cancer du pancréas métastatique. La FDA a déconseillé d'étendre l'étude VIRAGE de Phase 2b en Phase 3, recommandant à la place une étude autonome de Phase 3 de VCN-01 avec gemcitabine/nab-paclitaxel.
La FDA a accepté le design proposé par Theriva pour la Phase 3 et a indiqué que l'inclusion de chimiothérapies standards supplémentaires n'était pas nécessaire. La réunion a également abordé des éléments statistiques pour les études cliniques de confirmation. L'entreprise a récemment terminé l'inscription cible pour l'étude VIRAGE de Phase 2b et prévoit de demander une autre réunion avec la FDA après son achèvement pour discuter des détails du protocole de Phase 3.
Theriva Biologics (NYSE American: TOVX) gab die Ergebnisse eines Type-D-Meetings mit der FDA bekannt, das das Studiendesign der Phase 3 für die Behandlung von metastasierendem Pankreaskrebs mit VCN-01 betrifft. Die FDA riet davon ab, die laufende VIRAGE-Studie der Phase 2b in die Phase 3 zu erweitern und empfahl stattdessen eine eigenständige Phase-3-Studie von VCN-01 in Kombination mit Gemcitabin/Nab-Paclitaxel.
Die FDA stimmte dem von Theriva vorgeschlagenen Design für die Phase 3 zu und erklärte, dass die Einbeziehung zusätzlicher Chemotherapien im Standardbehandlungsregime nicht notwendig sei. Das Treffen behandelte auch statistische Aspekte für die Bestätigungsstudien. Das Unternehmen hat kürzlich die Zielanmeldung für die VIRAGE-Studie der Phase 2b abgeschlossen und plant, nach deren Abschluss ein weiteres Treffen mit der FDA anzufordern, um Einzelheiten zum Protokoll der Phase 3 zu besprechen.
- FDA provided general agreement with proposed Phase 3 study design
- Successfully completed target enrollment in VIRAGE Phase 2b clinical study
- Clear regulatory pathway established for Phase 3 trial
- FDA rejected expansion of current Phase 2b into Phase 3, requiring new standalone study
- Additional regulatory meetings needed before Phase 3 can begin
Insights
The FDA's guidance on Theriva's Phase 3 study design for VCN-01 represents a significant milestone in their pancreatic cancer program. The regulator's preference for a standalone Phase 3 trial, rather than expanding the current VIRAGE Phase 2b study, provides clear direction for the development pathway. The FDA's endorsement of combining VCN-01 with gemcitabine/nab-paclitaxel without additional chemotherapy agents streamlines the trial design and potentially reduces complexity in data interpretation.
The completion of target enrollment in the VIRAGE study positions Theriva to gather important efficacy data that will inform the Phase 3 protocol. For a micro-cap company with a market cap of just
The FDA's recommendation for a standalone Phase 3 trial, while potentially extending the development timeline, actually strengthens the program's scientific rigor. The guidance on statistical considerations, including sample size estimation and population analysis, will be important for generating robust, registration-worthy data. The regulator's support for the simpler combination with just gemcitabine/nab-paclitaxel, rather than multiple standard-of-care options, should result in cleaner data and more straightforward statistical analysis.
The planned follow-up meeting after VIRAGE study completion indicates a well-structured regulatory strategy. This stepwise approach allows for incorporation of Phase 2b results into the Phase 3 design, potentially optimizing trial parameters and increasing chances of success in this challenging indication.
ROCKVILLE, Md., Dec. 05, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced the outcomes of a recent Type D meeting with the U.S. Food and Drug Administration (FDA) to obtain guidance on the design of a Phase 3 clinical study of lead clinical candidate VCN-01 in combination with standard-of-care chemotherapy for the treatment of metastatic pancreatic adenocarcinoma (PDAC). The Company recently announced the completion of target enrollment into the multinational VIRAGE Phase 2b clinical study evaluating intravenous VCN-01 in combination with gemcitabine/nab-paclitaxel as a first line therapy for PDAC patients.
Type D meetings are focused on a narrow set of issues that are used to discuss issues at key decision points to provide timely feedback critical to moving a drug development program forward. The FDA advised that the on-going VIRAGE Phase 2b study should not be expanded into a Phase 3 study; rather, the optimal path forward for the VCN-01 PDAC program is to conduct a stand-alone Phase 3 study of VCN-01 with gemcitabine/nab-paclitaxel. The FDA provided general agreement with Theriva’s proposed design for a Phase 3 clinical study and indicated that inclusion of additional standard-of-care chemotherapy for PDAC was not necessary as it would complicate the study design and analysis. The FDA meeting also highlighted the FDA’s preferences regarding certain statistical elements of confirmatory clinical studies, including methods for sample size estimation and the study population(s) used for data analysis.
“The FDA’s advice on key elements of a potential confirmatory Phase 3 study evaluating VCN-01 plus gemcitabine/nab-paclitaxel as a first-line treatment for metastatic PDAC patients is critical as the VIRAGE study has entered final patient follow-up and we are actively planning the next steps in VCN-01 development,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “The feedback from the FDA and European regulatory agencies will facilitate the design of a Phase 3 study protocol that is expected to maximize our ability to provide a new therapeutic option to patients suffering this terrible disease.”
An additional meeting with the FDA will be requested after the completion of the VIRAGE study to discuss the details of the proposed confirmatory Phase 3 study protocol.
About Pancreatic Ductal Adenocarcinoma
Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma (“PDAC”) accounts for more than
About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in Company- and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.
About Theriva™ Biologics, Inc.
Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com.
Forward-Looking Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding the feedback from the FDA and European regulatory agencies facilitating the design of a Phase 3 study protocol; the protocol maximizing the Company’s ability to provide a new therapeutic option to patients suffering pancreatic cancer. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to effectively design the Phase 3 study protocol; the Company’s and VCN’s ability to reach clinical milestones when anticipated, including the ability to continue to enroll patients as planned, generating positive clinical data that establishes VCN-01 may lead to improved clinical outcomes for patients with PDAC and other solid cancers; the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s and VCN’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s and VCN’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s and VCN’s ability to maintain license agreements, the continued maintenance and growth of the Company’s and VCN’s patent estate, the ability to continue to remain well financed, and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
For further information, please contact:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.
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