On May 27, 2025 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, reported 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) (Press release, Theriva Biologics, MAY 27, 2025, View Source [SID1234653408]). These data will be featured in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago, IL from May 30-June 03, 2025.
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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 (Free ASCO Whitepaper) 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 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.