Theriva™ Biologics Reports Full-Year 2024 Operational Highlights and Financial Results
Rhea-AI Summary
Theriva Biologics (TOVX) has reported its full-year 2024 operational highlights and financial results. The company completed enrollment in the VIRAGE Phase 2b clinical trial of VCN-01 for metastatic pancreatic ductal adenocarcinoma (PDAC), with topline data expected in Q2 2025. Key achievements include FDA Fast Track designation for PDAC treatment and Rare Pediatric Disease designation for retinoblastoma.
Financial highlights show cash position of $11.6 million as of December 31, 2024, providing runway into Q3 2025. Research and development expenses decreased 16% to $12.0 million, while general and administrative expenses increased 4% to $7.4 million. The company received a Spanish government manufacturing funding award of €2.28 million and recognized a $1.8 million tax credit for R&D efforts in Spain.
Positive
- FDA Fast Track designation received for VCN-01 in PDAC treatment
- Completed enrollment of 92 patients in VIRAGE Phase 2b trial
- Secured €2.28M Spanish government manufacturing funding
- R&D expenses decreased 16% to $12.0M
- Received $1.8M Spanish R&D tax credit
Negative
- Cash decreased from $23.2M to $11.6M year-over-year
- Going concern warning from auditors
- General and administrative expenses increased 4% to $7.4M
- SYN-004 development halted without additional funding or partnership
Insights
Theriva's latest report shows mixed signals for investors tracking their oncology pipeline. The completion of enrollment in their VIRAGE Phase 2b trial for VCN-01 in pancreatic cancer (PDAC) marks a important milestone, with topline data expected in Q2 2025. The FDA Fast Track designation for this indication and Rare Pediatric Disease designation for retinoblastoma are significant regulatory validations potentially accelerating approval pathways and offering marketing exclusivity advantages.
The regulatory guidance from both FDA and EMA on Phase 3 trial design demonstrates agency engagement for their lead asset. However, these positive clinical developments must be viewed alongside concerning financial realities. The $11.6 million cash position provides runway only into Q3 2025 - barely past their upcoming data readout. More concerning is the auditor's going concern warning, indicating substantial doubt about the company's ability to continue operations without additional funding.
The deprioritization of their SYN-004 program for graft-versus-host disease prevention, despite positive safety data allowing progression to final cohort, reflects their financial constraints. This strategic narrowing to focus resources on VCN-01 is sensible but highlights their capital limitations. The upcoming VIRAGE data represents a pivotal binary event that will likely determine both the company's clinical and financial trajectory.
Theriva's financial position warrants investor caution amid promising clinical progress. Their cash runway into Q3 2025 creates a tight window for completing multiple critical objectives, including delivering VIRAGE trial data (
The cash burn rate shows concerning trajectory - declining from
The Spanish government manufacturing funding (
Investors should recognize that even with positive VIRAGE data, Theriva would need substantial additional capital for Phase 3 execution. Given their current market capitalization (
– Enrollment completed in VIRAGE Phase 2b clinical trial of VCN-01 in metastatic pancreatic ductal adenocarcinoma (PDAC); topline data expected in Q2 2025 –
– VCN-01 development bolstered by FDA award of Fast Track designation for the treatment of PDAC and Rare Pediatric Disease designation for retinoblastoma –
– Received guidance from the FDA and EMA on Phase 3 pivotal trial design for VCN-01 in combination with standard-of-care chemotherapy for metastatic PDAC –
–– As of December 31, 2024, Theriva Biologics reports
ROCKVILLE, Md., March 06, 2025 (GLOBE NEWSWIRE) -- 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 full-year ended December 31, 2024, and provided a corporate update.
“We are very pleased with our clinical and regulatory achievements in 2024,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “The VIRAGE Phase 2b clinical trial of our unique, stroma-degrading, oncolytic virus VCN-01in metastatic PDAC is nearing a topline data readout. Should this trial be successful, our interactions with regulatory agencies in the USA and Europe have provided us with clear direction on the path forward to a potential pivotal trial for VCN-01 in this indication. As PDAC incidence increases worldwide, the need for new therapies to treat this deadly disease is even more urgent.”
Recent Program Highlights and Anticipated Milestones:
VCN-01
Pancreatic Ductal Adenocarcinoma (PDAC):
- In May 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to lead clinical candidate VCN-01 in combination with gemcitabine and nab-paclitaxel to improve progression-free survival and overall survival in patients with metastatic pancreatic adenocarcinoma.
- In September 2024 we reported that our target enrollment of 92 evaluable patients was achieved in VIRAGE, the randomized, controlled, multicenter, open-label Phase 2b trial of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy in newly diagnosed metastatic PDAC patients. Topline data from the VIRAGE study, and the outcomes of a second Data Monitoring Committee review of safety data, are expected in Q2 2025.
- In December 2024 we received minutes from a Type D meeting with the FDA and in February 2025 we received scientific advice from the Committee for Human Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), both providing guidance on the design of a potential pivotal Phase 3 clinical study of lead clinical candidate VCN-01 in combination with standard-of-care chemotherapy gemcitabine/nab-paclitaxel for the treatment of metastatic PDAC. Feedback from the FDA and EMA is being incorporated into the design of a Phase 3 study protocol and an End-of-Phase 2 meeting with the FDA to discuss the proposed Phase 3 study will be requested before the end of 2025.
Retinoblastoma:
- In April 2024, we announced positive topline data from the investigator sponsored Phase 1 trial of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma conducted at Sant Joan de Déu-Barcelona Children’s Hospital. The database for this study has been locked and the clinical study report is being prepared.
- In July 2024, VCN-01 was granted Rare Pediatric Drug Designation by the FDA and in October 2024 VCN-01 received Orphan Medicinal Product Designation from the European Commission, both for the treatment of retinoblastoma. We continue discussions with key opinion leaders to develop a potential pivotal trial protocol for discussion with regulatory agencies.
SYN-004
- In October 2024 we reported a positive outcome from the Data and Safety Monitoring Committee (DSMC) review of results from the second Cohort of our Phase 1b/2a randomized, double-blinded, placebo-controlled clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease (aGVHD). Based on a review of the safety and pharmacokinetic data, the DSMC recommended that the study proceed to enroll the final Cohort 3. Based upon our current available funding and our focus on our clinical development of VCN-01 we do not anticipate that enrollment for the Cohort 3 will commence unless we obtain grant funding, or find a licensee or partner for the SYN-004 development program.
Business Updates
In September 2024, we were awarded manufacturing funding from the Spanish government’s National Knowledge Transfer Program. Theriva Biologics and the Universitat Autònoma de Barcelona (UAB) were awarded a total of
In 2024, we recognized a tax credit receivable and offsetting deferred R&D tax credit of
Full-Year Ended December 31, 2024 Financial Results
General and administrative expenses increased to
Research and development expenses decreased to
Other income was
Cash and cash equivalents totaled
The audited financial statements for the year ended December 31, 2024 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 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, and include statements regarding topline data expected in Q2 2025; cash runway into Q3 2025;the outcomes of a second Data Monitoring Committee review of safety data from the VIRAGE Phase 2b clinical trial of VCN-01 in metastatic pancreatic ductal adenocarcinoma expected in Q2 2025 ; having clear direction on the path forward to a potential pivotal trial for VCN-01 in PDAC should the VIRAGE Phase 2b trial be successful; the potential pivotal Phase 3 clinical study of lead clinical candidate VCN-01 in combination with standard-of-care chemotherapy gemcitabine/nab-paclitaxel for the treatment of metastatic PDAC; an End-of-Phase 2 meeting with the FDA to discuss the proposed Phase 3 study being requested before the end of 2025; preparing the clinical study report from the investigator sponsored Phase 1 trial of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma conducted at Sant Joan de Déu-Barcelona Children’s Hospital; continuing discussions with key opinion leaders for developing a potential pivotal trial protocol for retinoblastoma; obtaining grant funding or finding a licensee or partner for the SYN-004 development program; the THERICEL project establishing the viability of using Theriva’s proprietary A549 suspension cells for the clinical manufacture of adenoviral and AAV therapies; anticipated increase in research and development expense as the Company completes its VIRAGE Phase 2 clinical trial of VCN-01 and 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. Important factors that could cause actual results to differ materially from current expectations include, among others, the topline data being positive for the VIRAGE Phase 2b clinical trial of VCN-01 in metastatic pancreatic ductal adenocarcinoma; Company’s ability to effectively design the pivotol trial for VCN-01 in PDAC; the Company’s and VCN’s ability to reach clinical milestones when anticipated, including the ability to continue to enroll patients as planned, generating positive clinical data when anticipated (including from the VIRAGE Phase 2b clinical trial of VCN-01 in metastatic pancreatic ductal adenocarcinoma) that establishes VCN-01 may lead to improved clinical outcomes for patients with PDAC and other solid cancers; the Company’s and VCN’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the Company’s ability to complete clinical trials on time and achieve the desired results and benefits; the Company’s ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements; regulatory limitations relating to the Company’s and VCN’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 and VCN’s products; developments by competitors that render such products obsolete or non-competitive; the Company’s and VCN’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; the ability to find licensees and grant funding for the SYN-004 development program 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.
Investor Relations Contact:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
| Condensed Consolidated Balance Sheet | ||||||||
| (In thousands except share and par value amounts) | ||||||||
| (Unaudited) | ||||||||
| December 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Assets | ||||||||
| Current Assets | ||||||||
| Cash and cash equivalents | $ | 11,609 | $ | 23,177 | ||||
| Tax credit receivable | 3,228 | 1,812 | ||||||
| Prepaid expenses and other current assets | 1,444 | 2,414 | ||||||
| Total Current Assets | 16,281 | 27,403 | ||||||
| Non-Current Assets | ||||||||
| Property and equipment, net | 270 | 422 | ||||||
| Restricted cash | 96 | 102 | ||||||
| Right of use asset | 1,272 | 1,759 | ||||||
| In-process research and development | 17,358 | 19,755 | ||||||
| Goodwill | — | 5,700 | ||||||
| Deposits and other assets | 75 | 78 | ||||||
| Total Assets | $ | 35,352 | $ | 55,219 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current Liabilities: | ||||||||
| Accounts payable | $ | 859 | $ | 770 | ||||
| Accrued expenses | 3,368 | 2,995 | ||||||
| Accrued employee benefits | 1,144 | 1,517 | ||||||
| Deferred research and development tax credit-current portion | 1,614 | 906 | ||||||
| Loans payable-current | 61 | 63 | ||||||
| Operating lease liability-current portion | 539 | 487 | ||||||
| Total Current Liabilities | 7,585 | 6,738 | ||||||
| Non-current Liabilities | ||||||||
| Non-current contingent consideration | 6,973 | 6,274 | ||||||
| Loan Payable – non-current | 92 | 162 | ||||||
| Non-current deferred research and development tax credit | 762 | 906 | ||||||
| Non-current operating lease liability | 873 | 1,442 | ||||||
| Total Liabilities | 16,285 | 15,522 | ||||||
| Commitments and Contingencies | — | — | ||||||
| Temporary Equity; 10,000,000 authorized | ||||||||
| Series C convertible preferred stock, | — | 2,006 | ||||||
| Series D convertible preferred stock, | — | 728 | ||||||
| Stockholders’ Equity: | ||||||||
| Common stock, | 3 | 1 | ||||||
| Additional paid-in capital | 355,501 | 346,536 | ||||||
| Treasury stock at cost, 28,809 shares at December 31, 2024 and at December 31, 2023 | (288 | ) | (288 | ) | ||||
| Accumulated other comprehensive (loss) income | (1,178 | ) | 32 | |||||
| Accumulated deficit | (334,971 | ) | (309,318 | ) | ||||
| Total Stockholders‘ Equity | 19,067 | 36,963 | ||||||
| Total Liabilities and Stockholders’ Equity | $ | 35,352 | $ | 55,219 | ||||
| Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||||||
| (In thousands, except share and per share amounts) | ||||||||
| (Unaudited) | ||||||||
| For the year ended | ||||||||
| December 31, | ||||||||
| 2024 | 2023 | |||||||
| Operating Costs and Expenses: | ||||||||
| General and administrative | $ | 7,396 | $ | 7,120 | ||||
| Research and development | 12,031 | 14,311 | ||||||
| In-process research and development impairment | 1,325 | — | ||||||
| Goodwill impairment | 5,594 | — | ||||||
| Total Operating Costs and Expenses | 26,346 | 21,431 | ||||||
| Loss from Operations | (26,346 | ) | (21,431 | ) | ||||
| Other Income: | ||||||||
| Foreign currency exchange (loss) gain | (4 | ) | 3 | |||||
| Interest income | 697 | 1,439 | ||||||
| Total Other Income | 693 | 1,442 | ||||||
| Net Loss before income taxes | (25,653 | ) | (19,989 | ) | ||||
| Income tax benefit | — | 1,640 | ||||||
| Net Loss Attributable to Common Stockholders | $ | (25,653 | ) | $ | (18,349 | ) | ||
| Net Loss Per Share - Basic and Dilutive | $ | (19.03 | ) | $ | (28.48 | ) | ||
| Weighted average number of shares outstanding during the period - basic and dilutive | 1,348,126 | 644,282 | ||||||
| Net Loss | (25,653 | ) | (18,349 | ) | ||||
| (Loss) gain (loss) on foreign currency translation | (1,210 | ) | 711 | |||||
| Total comprehensive loss | $ | (26,863 | ) | $ | (17,638 | ) | ||
FAQ
When will Theriva Biologics (TOVX) release VIRAGE Phase 2b trial results for VCN-01?
How much cash does TOVX have and how long will it last?
What regulatory designations has TOVX received for VCN-01 in 2024?
How much funding did TOVX receive from the Spanish government?
What was TOVX's R&D expense reduction in 2024?
