Theriva™ Biologics Announces Presentation of Data from VCN-01 Retinoblastoma Phase 1 Clinical Trial at ASCO 2025 and Investigator Meeting to Review Topline Data from the VIRAGE Phase 2b Trial of VCN-01 in Metastatic Pancreatic Cancer
- None.
- None.
Insights
Theriva's dual cancer trial updates show promising progress for VCN-01 across pediatric retinoblastoma and pancreatic cancer programs.
Theriva Biologics is making significant progress with their lead candidate VCN-01 (zabilugene almadenorepvec) across two critical cancer indications. The company will present final data from their Phase 1 trial in retinoblastoma at ASCO 2025, focusing on patients with refractory disease who had limited treatment options beyond eye removal (enucleation). The Study Monitoring Committee has already deemed these results positive, with investigator Dr. Guillermo Chantada highlighting the drug's "tolerable toxicity profile and encouraging response in refractory vitreous seeds" - a major cause of treatment failure.
What makes this approach particularly valuable for pediatric patients is that VCN-01, as an oncolytic virus, operates through a different mechanism than standard chemotherapy or radiation. This is especially important for retinoblastoma patients who face elevated risks of treatment-induced secondary cancers.
Additionally, Theriva management will meet with investigators to discuss recently reported topline results from their VIRAGE Phase 2b trial in metastatic pancreatic ductal adenocarcinoma (PDAC), with the specific purpose of gathering insights for designing a potential Phase 3 trial. This suggests positive momentum for their pancreatic cancer program, though specific efficacy data isn't disclosed.
This dual-program progress demonstrates Theriva's advancing pipeline in both rare pediatric cancers and aggressive adult malignancies, potentially expanding treatment options in areas of significant unmet need. The advancement toward Phase 3 planning for the pancreatic cancer program is particularly noteworthy given the historically difficult treatment landscape for this indication.
- Safety and clinical outcomes of Phase 1 study of VCN-01 (zabilugene almadenorepvec) in refractory retinoblastoma patients to be presented in a poster session at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago on Saturday, May 31st, 2025 –
- Theriva senior management to attend the ASCO conference and participate in an off-site investigator meeting to review the topline data from the VIRAGE Phase 2b clinical trial of VCN-01 in first-line metastatic pancreatic ductal adenocarcinoma (PDAC) -
ROCKVILLE, Md., May 27, 2025 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced the upcoming presentation of final clinical outcomes and safety data from the investigator sponsored Phase 1 clinical study conducted at Sant Joan de Déu Barcelona Children’s Hospital evaluating the safety and tolerability of two intravitreal injections of VCN-01 (zabilugene almadenorepvec) in patients with intraocular retinoblastoma that was refractory to systemic, intra-arterial, or intravitreal chemotherapy, and for whom enucleation was the only recommended treatment (NCT03284268). These data will be featured in a poster at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place in Chicago, IL from May 30-June 03, 2025.
Details on Poster number 161 (abstract number 10046) can be found below.
- Presenting Author: Dr. Jaume Català-Mora, Pediatric Ophthalmologist, Sant Joan de Déu-Barcelona Children’s Hospital
- Title: A Phase I dose-escalation study to assess the oncolytic virus VCN-01 safety and efficacy in refractory retinoblastoma patients.
- Poster Session: Pediatric Oncology
- Date & Time: Saturday May 31st, 2025, at 0900-1200 US CDT
- Location: online at the conference portal and in person at Hall A, McCormick Place, Chicago, IL
“We are very pleased that the important work by our collaborators at Sant Joan de Déu-Barcelona Children’s Hospital is being presented at the premier international oncology conference” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We previously reported that the Study Monitoring Committee determined the trial results to be positive. This poster presentation provides the investigators with the opportunity to discuss the detailed results on VCN-01 safety and long-term efficacy in this population with a broader clinical oncology audience and obtain feedback that may assist in refining our clinical strategy for VCN-01 in this underserved pediatric cancer population.”
Guillermo Chantada, world-recognized expert in retinoblastoma and one of the investigators in the trial indicated “The oncolytic virus VCN-01 is a promising new player for the treatment of retinoblastoma. In our study, it has shown a tolerable toxicity profile and encouraging response in refractory vitreous seeds which are still the major cause of conservative therapy failure. Through its mechanism of action, we found that VCN-01 specifically targets the tumor cells and by being a non-chemotherapeutic or radiotherapeutic agent, it is of additional interest in this population with higher risk of treatment-induced malignancies”.
Theriva senior management will also attend the ASCO conference and participate in an off-site meeting on Monday June2nd, 2025 to review the recently reported topline results of the VIRAGE Phase 2b clinical trial in first-line metastatic pancreatic ductal adenocarcinoma (PDAC) with investigators and garner additional insights relevant to the design and execution of a Phase 3 clinical trial in this indication.
About VCN-01
VCN-01 (zabilugene almadenorepvec) 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 over 140 patients to date in 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. VCN-01 has Orphan Drug designation from the EMA and both Orphan Drug designation and Fast Track designation from the FDA for the treatment of pancreatic cancer. VCN-01 also has Orphan Drug designation and Rare Pediatric Diseases designation from the FDA for the treatment of retinoblastoma.
About Retinoblastoma
Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 - 1/18,000 live newborns and accounts for
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’s subsidiary Theriva Biologics, S.L. , has been developing 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 clinical-stage candidates are: (1) VCN-01 (zabilugene almadenorepvec), 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 VCN-01 being a promising new player for the treatment of retinoblastoma.. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, VCN-01’s ability to have positive results in future trials for the treatment of retinoblastoma and other therapeutic benefits; the Company’s ability to reach clinical milestones when anticipated including enrolling the expected number of patients in each trial; the Company’s product candidates, including VCN-01, 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, continuing clinical trial enrollment as expected; 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 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 products, developments by competitors that render such products obsolete or non-competitive, the Company’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, 2024 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:
Kevin Gardner
LifeSci Advisors, LLC
kgardner@lifesciadvisors.com
Source: Theriva Biologics, Inc.
FAQ
What are the key findings from Theriva's (TOVX) VCN-01 Phase 1 trial in retinoblastoma?
What is the current development stage of Theriva's (TOVX) VCN-01 for pancreatic cancer?
When will Theriva (TOVX) present the VCN-01 retinoblastoma data at ASCO 2025?
What is VCN-01's potential impact on retinoblastoma treatment?
