On March 12, 2026 Theriva Biologics, Inc. (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the full year ended December 31, 2025, and provided a corporate update.

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"We are very pleased to have completed the licensing of our versatile Phase 2-ready asset SYN-020 to Rasayana Therapeutics, executing on our plan to derive value from our GI portfolio while we remain focused on the advancement of our oncology pipeline," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We continue to advance our lead oncolytic virus candidate VCN-01 towards pivotal clinical development in multiple indications of high unmet need. With the recent feedback from the EMA, we have further clarity on dosing regimen, protocol and overall design for our proposed Phase 3 trial in pancreatic ductal adenocarcinoma (PDAC). An End-of-Phase 2 meeting with the FDA is planned for the first half of 2026 to finalize our design for a multinational pivotal Phase 3 trial in PDAC, aiming to provide patients with a novel therapy for this difficult to treat solid tumor that has a high mortality rate. Additional interactions with the FDA and EMA are planned for 2026 to refine the design of a potential Phase 2/3 trial for retinoblastoma, for which VCN-01 has received Orphan Drug and Rare Pediatric Disease designation. We continue to engage in potential partnership discussions for the additional innovative drug candidates in our portfolio."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (PDAC):

As recently announced, Theriva received positive scientific advice from the EMA on the design of a Phase 3 trial in PDAC. The EMA provided overall agreement with the proposed Phase 3 clinical trial of VCN-01 in combination with gemcitabine/nab-paclitaxel for the first-line treatment of metastatic PDAC, including sample size, repeated dosing of VCN-01, and an adaptive design to potentially optimize trial timelines and outcomes. Specific advice included agreement on proposed inclusion/exclusion criteria, primary endpoint (overall survival), secondary endpoints (including progression free survival, duration of response, and patient reported outcomes).
An End-of-Phase 2 meeting with the FDA is planned for 1H 2026, aiming to finalize the design of a pivotal multinational Phase 3 clinical trial in PDAC.
Additional analysis from the VIRAGE Phase 2b study has been accepted for a poster presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) meeting in San Diego, CA, April 17-22, 2026.
Retinoblastoma:

Further discussions are planned with both the FDA and EMA in 2026 to align on the protocol design, including target population and endpoints, for a Phase 2/3 trial in retinoblastoma, an indication for which VCN-01 has been granted Orphan Drug and Rare Pediatric Disease designation.
Safety and clinical outcomes from the Phase 1 study of VCN-01 in refractory retinoblastoma patients were recently presented at the 41st Asia-Pacific Academy of Ophthalmology (APAO) Congress.
SYN-020

On February 18, 2026, Theriva announced that it granted Rasayana Therapeutics, Inc. an exclusive, worldwide license, with the right to sublicense, to develop and commercialize SYN-020 (recombinant bovine intestinal alkaline phosphatase) for therapeutic and diagnostic use. Theriva received a $300,000 up-front payment at signing and is eligible for up to $16M in development and regulatory milestones, tiered single digit royalties on net product sales, and up to $22M in milestones payable upon achievement of certain annual aggregate net sales.
SYN-020 was well tolerated in Phase 1 clinical studies and is now poised to enter Phase 2 clinical testing. Rasayana will assume responsibility and costs for future clinical development and commercialization.
Full-Year Ended December 31, 2025 Financial Results

General and administrative expenses increased to $15.4 million for the year ended December 31, 2025, from $7.4 million for the year ended December 31, 2024. This increase of 109% is primarily comprised of the contingent consideration adjustment of $9.0 million due to the VIRAGE Phase 2b clinical trial of VCN-01 (zabilugene almadenorepvec) in PDAC achieving its primary survival and safety endpoints, offset by a decrease in compensation costs, investor relations costs, consulting fees, and lower director and officer insurance. The charge relating to stock-based compensation expense was $379,000 for the year ended December 31, 2025, compared to $438,000 for the year ended December 31, 2024.

Research and development expenses decreased to $8.6 million for the year ended December 31, 2025, from $12.0 million for the year ended December 31, 2024. This decrease of 28% is primarily the result of lower clinical trial expenses related to the completion of our VIRAGE Phase 2b clinical trial of VCN-01 (zabilugene almadenorepvec) in PDAC, lower clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients and lower indirect cost related to compensation, offset by higher patent expenses related to SYN-020. We anticipate research and development expense to decrease in 2026 as a result of the workforce reduction plan that was implemented on September 30, 2025, the completion of our VIRAGE Phase 2b clinical trial of VCN-01 and our focus on regulatory interactions around potential pivotal clinical trials of VCN-01 in PDAC and retinoblastoma, the planning for VCN-01 manufacturing scale-up activities, a potential Phase 2a study evaluating VCN-01 dosing frequency, and continuing to support our other preclinical and discovery initiatives. In addition, pursuant to the terms of the Rasayana License Agreement that we entered into in February 2026, we will not continue to conduct research and development activities with respect to SYN-020, and do not expect to incur material expenditures in connection therewith since Rasayana is now responsible for all such expenditures including patent expenses. Research and development expenses also include a charge relating to non-cash stock-based compensation expense of $281,000 for the year ended December 31, 2025, compared to $233,000 for the year ended December 31, 2024.

Cash and cash equivalents totaled $13.1 million as of December 31, 2025, compared to $11.6 million as of December 31, 2024. The Company’s current cash of approximately $15.2 million at February 26, 2026 will allow it to fund operations into the first quarter of 2027, including overhead costs, close out of the VIRAGE Phase 2b clinical trial, a potential Phase 2a study evaluating VCN-01 dosing frequency, exploratory VCN-01 (zabilugene almadenorepvec) manufacturing scale-up activities, regulatory interactions regarding proposed VCN-01 clinical trials in PDAC and retinoblastoma, and preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program.

The audited financial statements for the year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K contain an audit opinion from the Company’s independent registered public accounting firm that includes an explanatory paragraph related to the Company’s ability to continue as a going concern.

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 142 patients to date in clinical trials of different cancers, including pancreatic ductal adenocarcinoma (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 SYN-020

SYN-020 is a recombinant bovine intestinal alkaline phosphatase (IAP) produced under cGMP conditions and formulated for oral delivery to the small intestine. SYN-020 is designed to reduce fat absorption and intestinal inflammation, tighten the gut barrier to mitigate leaky gut, and promote a healthy microbiome. These complementary modes of action mean SYN-020 has the potential to address multiple metabolic and inflammatory disorders and diseases associated with aging. Despite its broad therapeutic potential, a key hurdle to commercialization has been the high cost of IAP manufacture. Theriva has overcome this hurdle and has developed a process to produce SYN-020 at a scale and cost viable for clinical and commercial development. In February 2026, Theriva granted to Rasayana Therapeutics, Inc. an exclusive, worldwide license, with the right to sublicense, to develop and commercialize SYN-020 (recombinant bovine intestinal alkaline phosphatase) for therapeutic and diagnostic use.

(Press release, Theriva Biologics, MAR 12, 2026, View Source [SID1234663510])