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Theriva Biologics Reports First Quarter 2024 Operational Highlights and Financial Results - Reported topline data from the investigator sponsored Phase 1 trial of intravitreal VCN-01 in pediatric patients with refractory

Key Takeaway: Theriva Biologics has reported positive topline results from its Phase 1 trial of the oncolytic adenovirus VCN-01 for pediatric patients with refractory retinoblastoma. Additionally, the company has presented preclinical data highlighting the potential synergy of VCN-01 with liposomal irinotecan in pancreatic cancer treatments. The ongoing Phase 2b clinical trial, VIRAGE, is advancing as planned, with expected completion of enrollment in the third quarter of 2024. Financial results indicate a decrease in administrative expenses, alongside an increase in research and development costs, attributed to expanding clinical trials and initiatives.

Market Sentiment Analysis

POSITIVE FACTORS

  • Positive topline data from the Phase 1 trial of VCN-01 in pediatric retinoblastoma.
  • Preclinical data indicating potential synergy of VCN-01 with other chemotherapeutics for enhanced efficacy.
  • Solid financial position with $18.3 million cash, providing runway into 2025.
  • Progress in enrollment for the Phase 2b VIRAGE trial for metastatic PDAC.

Full Press Release Details

Theriva Biologics Reports First Quarter
2024 Operational Highlights and Financial Results
- Reported topline data from the investigator
sponsored Phase 1 trial of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma; trial results were determined to
be positive by the study Monitoring Committee-
-Presented preclinical data demonstrating the
potential synergy between VCN-01 and liposomal irinotecan in a human pancreatic mouse xenograft at the ASGCT Annual Meeting; emphasizes
VCN-01's potential in diverse chemotherapy combinations for improved efficacy in pancreatic cancer-
- VIRAGE, the Phase 2b clinical trial of VCN-01
in combination with chemotherapy for metastatic Pancreatic Ductal Adenocarcinoma (PDAC), is expected to complete enrollment in the third
- As of March 31, 2024, Theriva Biologics reports
$18.3 million in cash, which is expected to provide runway into the first quarter of 2025-
Rockville, MD, May 7, 2024 - 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, 2024, and provided a corporate update.
"In the first quarter we reported positive topline data from
the investigator sponsored Phase 1 trial in patients with refractory retinoblastoma, further validating VCN-01's unique mechanism
of action and therapeutic potential to improve treatment outcomes as an adjunct to chemotherapy in this area of high unmet need,"
said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "In parallel, we continue to build a portfolio of potentially
improved therapeutic combinations for PDAC patients. VIRAGE, our Phase 2b trial evaluating VCN-01 in metastatic PDAC is progressing and
enrolling patients across sites in the U.S. and Spain. Further, the recently presented preclinical data at ASGCT support the potential
antitumor synergy of VCN-01 with topoisomerase 1 inhibitors, an important class of chemotherapeutic agents used in a number of different
cancers. We look forward to identifying opportunities to evaluate the combination of VCN-01 with additional first-line pancreatic cancer
chemotherapy regimens (FOLFIRINOX or NALIRIFOX) that incorporate the topoisomerase 1 inhibitor irinotecan. Building on this momentum,
we are well positioned to achieve several important milestones that will continue to drive forward our clinical development program."
Recent Program Highlights and Anticipated
Pancreatic Ductal Adenocarcinoma (PDAC):
SYN-004 (ribaxamase):
First Quarter Ended March 31, 2024 Financial Results
General and administrative expenses decreased
to $1.9 million for the three months ended March 31, 2024, from $2.2 million for the three months ended March 31, 2023. This decrease
of 12% is primarily comprised of the decrease in salary costs, consulting, legal fees, and lower director and officer insurance, offset
by an increase in fair value of the contingent consideration adjustment. The charge related to stock-based compensation expense was $101,000
for the three months ended March 31, 2024, compared to $87,000 for the three months ended March 31, 2023.
Research and development expenses increased to
$3.5 million for the three months ended March 31, 2024, from approximately $3.0 million for the three months ended March 31, 2023. This
increase of 16% is primarily the result of higher clinical trial expenses related to our VIRAGE Phase 2 clinical trial of VCN-01 in PDAC,
increased expenses related to the Phase 1 Trial of intravitreal VCN-01 in patients with retinoblastoma, and increased expenses related
to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by lower expenses related to our Phase
1a clinical trial of SYN-020. We anticipate research and development expense to increase as we continue enrollment in our VIRAGE Phase
2 clinical trial of VCN-01 in PDAC, advance our VCN-01 program in retinoblastoma, expand GMP manufacturing activities for VCN-01, and
continue supporting our VCN-11 and other preclinical and discovery initiatives. The charge related to stock-based compensation expense
was $58,000 for the three months ended March 31, 2024, compared to $39,000 related to stock-based compensation expense for the three months
ended March 31, 2023.
income for the three months ended March 31, 2024 is primarily comprised of interest income of $228,000 and an exchange loss of $1,000.
Other income for the three months ended March 31, 2023 is primarily comprised of interest income of $364,000 and exchange gain of $6,000.
Cash and cash equivalents totaled $18.3 million as of March 31, 2024,
compared to $23.2 million as of December 31, 2023.
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 the potential synergy between VCN-01 and liposomal irinotecan in a human pancreatic mouse xenograft,
VCN-01's potential in diverse chemotherapy combinations for improved efficacy in pancreatic cancer VIRAGE expecting to complete
enrollment in the the third quarter of 2024, enrolling 92 evaluable patients and continuing to enroll patients across seven sites in the
U.S. and ten sites in Spain, cash being expected to provide runway into the first quarter of 2025, the therapeutic potential of VCN-01
in retinoblastoma and VCN-01's potential for use in diverse cancer indications, the SYN-004 trial expecting to announce a data readout
in the third quarter of 2024, the potential synergy of VCN-01 and additional first-line pancreatic cancer chemotherapy, continuing to
build a portfolio of potentially improved therapeutic combinations for PDAC patients, identifying opportunities to evaluate the combination
of VCN-01 with additional first-line pancreatic cancer chemotherapy regimens, being well positioned to achieve several important milestones
that will continue to drive forward the clinical development program and anticipated research and development expense to increase . Important
factors that could cause actual results to differ materially from current expectations include, among others, the Company's and
VCN's ability to reach clinical milestones when anticipated, including the ability to continue to enroll patients as planned and
the completion of enrollment in VIRAGE in the third quarter of 2024, and announcing a data readout for the SYN-004 second cohort in the
third quarter of 2024, generating clinical data 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 ability to complete clinical trials on time and achieve the desired results and benefits,;
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 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 and the cash providing a runway into the first quarter of 2025, and other factors
described in the Company's Annual Report on Form 10-K for the year ended December 31, 2023 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:
LifeSci Advisors, LLC
Theriva Biologics, Inc. and Subsidiaries
Consolidated Balance Sheets
(In thousands except share and par value amounts)
March 31, 2024 December 31, 2023
Assets
Current Assets
Cash and cash equivalents $ 18,261 $ 23,177
Tax credit receivable 1,772 1,812
Prepaid expenses and other current assets 1,623 2,414
Total Current Assets 21,656 27,403
Non-Current Assets
Property and equipment, net 375 422
Restricted cash 99 102
Right of use asset 1,634 1,759
In-process research and development 19,316 19,755
Goodwill 5,573 5,700
Deposits and other assets 77 78
Total Assets $ 48,730 $ 55,219
Liabilities and Stockholders Equity
Current Liabilities:
Accounts payable $ 571 $ 770
Accrued expenses 3,327 2,995
Accrued employee benefits 665 1,517
Deferred research and development tax credit-current portion 886 906
Loans payable-current 62 63
Operating lease liability-current portion 498 487
Total Current Liabilities 6,009 6,738
Non-current Liabilities
Non-current contingent consideration 6,476 6,274
Loan Payable - non-current 159 162
Non-current deferred research and development tax credit 664 906
Non-current operating lease liability 1,299 1,442
Total Liabilities 14,607 15,522
Commitments and Contingencies (Note 13) - -
Temporary Equity; 10,000,000 authorized
Series C convertible preferred stock, $0.001 par value; 275,000 issued and outstanding 2,006 2,006
Series D convertible preferred stock, $0.001 par value; 100,000 issued and outstanding 728 728
Stockholders' Equity:
Common stock, $0.001 par value; 350,000,000 shares authorized, 17,868,282 issued and 17,148,049 outstanding at March 31, 2024 and 17,868,282 issued and 17,148,049 outstanding at December 31, 2023 18 18
Additional paid-in capital 346,679 346,519
Treasury stock at cost, 720,233 shares at March 31, 2024 and at December 31, 2023 (288 ) (288 )
Accumulated other comprehensive (loss) income (537 ) 32
Accumulated deficit (314,483 ) (309,318 )
Total Stockholders' Equity 31,389 36,963
Total Liabilities and Stockholders' Equity $ 48,730 $ 55,219
Theriva Biologics, Inc. and Subsidiaries
Consolidated Statements of Operations and Comprehensive

Frequently Asked Questions

What were the key findings of Theriva's Phase 1 trial?

The Phase 1 trial of VCN-01 in pediatric retinoblastoma showed positive topline data.

What financial position does Theriva Biologics hold?

As of March 31, 2024, Theriva Biologics reported $18.3 million in cash.

What is the VIRAGE trial focusing on?

The VIRAGE trial evaluates VCN-01 with chemotherapy for metastatic PDAC.

How did the R&D expenses change in Q1 2024?

R&D expenses increased to $3.5 million, up from $3.0 million in Q1 2023.

What potential does VCN-01 have in cancer treatment?

VCN-01 may enhance efficacy when combined with various chemotherapy regimens.

Last updated: May 7, 2024