Full Press Release Details
Theriva Biologics Reports First Quarter
2025 Operational Highlights
and Financial Results
- VCN-01 Achieves Primary Efficacy and Safety Endpoints
for Pancreatic Ductal Adenocarcinoma in VIRAGE Phase 2b Clinical Trial -
- Closed a public offering on May 8, 2025, raising
the Company's cash balance and extending its cash runway into the first quarter of 2026 -
Rockville, MD, May 14, 2025 - 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 reported financial results for the first quarter ended March 31, 2025, and provided a corporate update.
"We have started 2025 with outstanding clinical
progress," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "The VIRAGE Phase 2b clinical trial of
VCN-01 (zabilugene almadenorepvec) with gemcitabine/nab-paclitaxel in newly diagnosed metastatic pancreatic cancer patients achieved its
primary survival and safety endpoints, highlighting the potential therapeutic benefits of our oncolytic virus platform. We are working
to scale up manufacturing and finalize the design of a Phase 3 trial of VCN-01 with gemcitabine/nab-paclitaxel which if successful, may
allow us to deliver this innovative treatment option to patients suffering this fatal disease."
Recent Highlights and Anticipated Milestones
Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC):
Allogeneic hematopoietic cell transplant (HCT):
First Quarter Ended March 31, 2025 Financial
General and administrative expenses decreased
to $1.4 million for the three months ended March 31, 2025, from $1.9 million for the three months ended March 31, 2024. This decrease
of 25% is primarily comprised of the decrease in salary costs, travel, lower director and officer insurance, and a decrease in fair value
of the contingent consideration adjustment. The charge related to stock-based compensation expense was $54,000 for the three months ended
March 31, 2025, compared to $101,000 for the three months ended March 31, 2024.
Research and development expenses decreased to
$3.0 million for the three months ended March 31, 2025, from approximately $3.5 million for the three months ended March 31, 2024. This
decrease of 14% is primarily the result of lower indirect cost related to decreased VCN-01 manufacturing costs and lower clinical trial
expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by slightly higher clinical
trial expenses related to our VIRAGE Phase 2b clinical trial of VCN-01 in PDAC and higher patent expenses related to SYN-020. We anticipate
research and development expense to increase as we complete our VIRAGE Phase 2b clinical trial of VCN-01and plan for our Phase 3 clinical
trial of VCN-01 in PDAC, advance our VCN-01 program in retinoblastoma, expand GMP scale-up manufacturing activities for VCN-01, and continue
supporting our other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $46,000 for the
three months ended March 31, 2025, compared to $58,000 related to stock-based compensation expense for the three months ended March 31,
Other income was $93,000 for the three months
ended March 31, 2025 compared to other income of $227,000 for the three months ended March 31, 2024. Other income for the three months
ended March 31, 2025 is primarily comprised of interest income of $96,000 and an exchange loss of $3,000. Other income for the three months
ended March 31, 2024 is primarily comprised of interest income of $228,000 and exchange loss of $1,000.
Cash and cash equivalents totaled $10 million
as of March 31, 2025, compared to $11.6 million as of December 31, 2024. Subsequent to closing of the public offering on May 8 2025, the
Company's cash balance was $14.1 million.
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 90% of all pancreatic tumors. It can be
located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other
less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in
any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease)
when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable
at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.
VIRAGE was a two-arm, Phase 2b open-label, randomized,
controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled
at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received
gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment
arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel
treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability.
Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and
immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials
Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).
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 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. 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. These forward-looking statements include, but are not
limited to, statements regarding having a cashrunway into the first quarter of 2026; the potential therapeutic benefits of the Company's
oncolytic virus platform; the design of a Phase 3 trial of VCN-01 with gemcitabine/nab-paclitaxel if successful, allowing the Company
to deliver this innovative treatment option to patients suffering this fatal disease; the intended use of net proceeds from the offering;
the suggestion that the second dose of VCN-01 (administered 3 months after the first dose) provides a meaningful additional benefit in
the treatment subgroup; a potential Phase 3 confirmatory trial for VCN-01 in PDAC; and research and development expense increasing as
the Company completes its VIRAGE Phase 2b clinical trial of VCN-01and plans for its Phase 3 clinical trial of VCN-01 in PDAC, advances
its VCN-01 program in retinoblastoma, expands GMP scale-up manufacturing activities for VCN-01, and continues supporting its other preclinical
and discovery initiatives. The Company undertakes no obligation to publicly update any forward-looking
statement, whether as a result of new information, future events or otherwise, except as required under applicable law. Important
factors that could cause actual results to differ materially from those in the forward-looking statements include the ability
of VCN-01 to have therapeutic benefits and continue to do so in future trials; the