Theriva™ Biologics Announces Upcoming Presentation of Data from VCN-01 Retinoblastoma Phase 1 Clinical Trial at APAO 2026
- Safety and clinical outcomes of Phase 1 study of VCN-01 (zabilugene almadenorepvec) in refractory retinoblastoma patients to be presented in an invited session at the 41st Asia-Pacific Academy of Ophthalmology (APAO) Congress in Hong Kong on Saturday, 07 February 2026 –
- Preclinical data will highlight synergistic antitumor activity for the combination of topotecan and VCN-01 that may improve outcomes in refractory retinoblastoma patients with difficult-to-treat vitreous seeds -
ROCKVILLE, Md. and BARCELONA, Spain, Feb. 03, 2026 (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 an invited presentation of clinical outcomes and safety data from the investigator sponsored Phase 1 clinical study (NCT03284268) evaluating the safety and tolerability of two intravitreal injections of VCN-01 (zabilugene almadenorepvec) in pediatric patients with intraocular retinoblastoma that was refractory to systemic, intra-arterial, or intravitreal chemotherapy, and for whom enucleation was the only recommended treatment. Preclinical data describing the synergistic antitumor effects observed when topotecan (a standard of care chemotherapy for retinoblastoma) is coadministered with VCN-01 will also be presented. These data will be presented during a retinoblastoma focused session at the upcoming Asia-Pacific Academy of Ophthalmology (APAO) Congress (held in conjunction with the 37th Annual Scientific Meeting Hong Kong Ophthalmological Symposium) taking place in Hong Kong, China, 5-8 February 2026.
Details of the presentation can be found online and below.
- Presenting Author: Dr. Jaume Català-Mora, Pediatric Ophthalmologist, Sant Joan de Déu-Barcelona Children’s Hospital
- Title: Update on Oncolytic Adenovirus VCN-01 Trial in Retinoblastoma
- Session: 0211 Retinoblastoma in Association With Asian Retinoblastoma Group
- Date & Time: Saturday 07 February, 2026, at 4:46 PM HKT (3:46 AM US EDT)
- Location: Rooms S224-225, Level 2, Hong Kong Convention and Exhibition Centre (HKCEC)
“We are very pleased with the on-going international interest in the novel clinical and preclinical findings by our collaborators treating retinoblastoma patients at Sant Joan de Déu-Barcelona Children’s Hospital,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “Effective treatment of refractory retinoblastoma with vitreous seeds remains a significant unmet medical need for pediatric patients with this condition worldwide. Based on our previously reported Phase 1 clinical data, and the emerging preclinical findings, we believe that the intravitreal combination of VCN-01 and topotecan provides an exciting new opportunity to address this challenge and help preserve the eyes and quality-of-life of children with this rare but devastating cancer. VCN-01 has Orphan Drug and Rare Pediatric Disease designations for the treatment of retinoblastoma, and we are currently refining a potential pivotal clinical trial design in this indication for discussion with regulatory agencies.”
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 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In Europe, retinoblastoma has an estimated incidence rate of 1 per 13,844 live births (14.1 per million children under the age of 5) with approximately 300 children diagnosed per year (Stacey et al. 2021). Preserving life and preventing the loss of an eye, blindness, and other serious effects of treatment that reduce the patient’s life span or the quality of life remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.
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 preclinical data highlighting synergistic antitumor activity for the combination of topotecan and VCN-01 that may improve outcomes in refractory retinoblastoma patients with difficult-to-treat vitreous seeds; the intravitreal combination of VCN-01 and topotecan providing an exciting new opportunity to address the challenge of effective treatment of refractory retinoblastoma with vitreous seeds retinoblastoma and a potential pivotal clinical trial design in 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, the ability to design a pivotal clinical trial in retinoblastoma for discussion with the FDA; 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.
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