Theriva™ Biologics Receives Rare Pediatric Drug Designation by the U.S. FDA for VCN-01 for the Treatment of Retinoblastoma

Rhea-AI Summary

Theriva™ Biologics (NYSE American: TOVX) has received Rare Pediatric Drug Designation (RPDD) from the U.S. FDA for VCN-01 in treating retinoblastoma. VCN-01, Theriva's lead candidate, is a systemic, selective, stroma-degrading oncolytic adenovirus. This follows a previous orphan drug designation for the same indication.

The designation highlights the urgent need for new treatments for pediatric retinoblastoma patients. Theriva is working with physicians and regulators to refine its clinical strategy for VCN-01 as an adjunct to chemotherapy. Recent Phase 1 trial results for intravitreal VCN-01 in refractory retinoblastoma were deemed positive by the study Monitoring Committee.

If VCN-01 receives FDA approval for retinoblastoma, Theriva may be eligible for a Priority Review Voucher, which can be used or sold.

Positive

• Received Rare Pediatric Drug Designation (RPDD) from FDA for VCN-01
• Previously granted orphan drug designation for VCN-01
• Positive Phase 1 trial results for intravitreal VCN-01 in refractory retinoblastoma
• Potential eligibility for Priority Review Voucher if VCN-01 is approved

Negative

• None.

Oncology Specialist

The FDA's granting of Rare Pediatric Drug Designation (RPDD) for VCN-01 in treating retinoblastoma is a significant development for Theriva Biologics. As an oncologist specializing in pediatric cancers, I can attest to the critical need for new treatments in this area. Retinoblastoma, a rare eye cancer affecting young children, has treatment options, especially in advanced cases.

The RPDD status brings several benefits:

• It acknowledges the urgent need for innovative therapies in this underserved patient population.
• It may expedite the drug's development and review process.
• If approved, it could lead to a Priority Review Voucher, a valuable asset for the company.

The positive results from the Phase 1 trial evaluating intravitreal VCN-01 in refractory retinoblastoma patients are encouraging. This suggests that VCN-01, as a stroma-degrading oncolytic adenovirus, may offer a novel approach to treating this challenging cancer. The potential use of VCN-01 as an adjunct to chemotherapy could significantly improve outcomes for these young patients.

However, it's important to note that we are still in early stages. More extensive clinical trials will be necessary to fully establish the safety and efficacy of VCN-01 in retinoblastoma treatment. The road from RPDD to FDA approval can be long and complex, especially for novel therapies like oncolytic viruses.

Financial Analyst

The Rare Pediatric Drug Designation (RPDD) for VCN-01 is a positive development for Theriva Biologics from a financial perspective. Here's why:

• Market Potential: While retinoblastoma is rare, affecting fewer than 200,000 persons in the US, successful treatments for rare diseases can command premium pricing, potentially leading to significant revenue streams if approved.
• Priority Review Voucher: If VCN-01 receives FDA approval, Theriva could obtain a Priority Review Voucher. These vouchers are highly valuable and can be sold for substantial sums, often in the range of $100 million to 200 million.
• Competitive Advantage: The RPDD, combined with the previous orphan drug designation, provides Theriva with market exclusivity benefits, potentially deterring competitors and allowing for a stronger market position.

However, investors should be cautious. Theriva is still a clinical-stage company and VCN-01 is not yet approved. The path to commercialization can be long and costly, with no guarantee of success. The company's financial health and ability to fund ongoing research and potential commercialization will be important factors to monitor.

Additionally, while the RPDD is positive news, it doesn't significantly change the company's near-term financial outlook. Theriva will likely continue to operate at a loss as it advances its pipeline. Investors should closely watch for updates on clinical progress, partnerships and the company's cash position in upcoming financial reports.

ROCKVILLE, Md., July 31, 2024 (GLOBE NEWSWIRE) -- 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, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Drug Designation (RPDD) for VCN-01 for the treatment of retinoblastoma. VCN-01, Theriva’s lead product candidate, is a systemic, selective, stroma-degrading oncolytic adenovirus. Previously, the FDA granted orphan drug designation to VCN-01 for treatment of retinoblastoma.

“The FDA’s decision to grant rare pediatric drug designation to VCN-01 highlights the urgent need for new treatment options for pediatric patients with retinoblastoma,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We are encouraged by this important step forward and, in parallel, continue to work closely with leading physicians and regulatory agencies to refine our clinical strategy for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma. Most recently, results from the investigator sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma were determined to be positive by the study Monitoring Committee. Data from this study will further inform our clinical development pathway in this area of high unmet need.”

The FDA grants RPDD for rare diseases (fewer than 200,000 affected persons in the United States) that are serious and life-threatening and primarily affect children ages 18 years or younger. If a Biologics License Application for VCN-01 for the treatment of retinoblastoma is approved by the FDA, Theriva may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred.

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 the U.S., retinoblastoma shows an incidence rate of 3.3 per 1,000,000 with only about 200 to 300 children diagnosed per year according to the American Cancer Society. 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.

About VCN-01

VCN-01 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 80 patients in Phase 1 and investigator-sponsored 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, and include statements regarding continuing to work closely with leading physicians and regulatory agencies to refine the Company’s clinical strategy for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma, the data from the investigator sponsored Phase 1 trial at Hospital Sant Joan de Déu in Barcelona evaluating intravitreal VCN-01 in pediatric patients with refractory retinoblastoma informing the Company’s clinical development pathway and being eligible to receive a Priority Review Voucher. 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 receive a Priority Review Voucher, 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:
Investor Relations:
Chris Calabrese
LifeSci Advisors, LLC
ccalabrese@lifesciadvisors.com
917-680-5608
Source: Theriva Biologics, Inc.

FAQ

What is the Rare Pediatric Drug Designation (RPDD) granted to Theriva Biologics (TOVX) for?

The FDA granted Rare Pediatric Drug Designation (RPDD) to Theriva Biologics (TOVX) for VCN-01, their lead product candidate, for the treatment of retinoblastoma in pediatric patients.

What is VCN-01 and how does it work in treating retinoblastoma?

VCN-01 is a systemic, selective, stroma-degrading oncolytic adenovirus. It is being developed as an adjunct to chemotherapy for pediatric patients with advanced retinoblastoma, targeting the urgent need for new treatment options in this area.

What were the results of the Phase 1 trial for VCN-01 in retinoblastoma patients?

The Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma showed positive results, as determined by the study Monitoring Committee. These results will inform further clinical development.

What potential benefits could Theriva Biologics (TOVX) receive if VCN-01 is approved for retinoblastoma?

If the FDA approves a Biologics License Application for VCN-01 in treating retinoblastoma, Theriva Biologics (TOVX) may be eligible to receive a Priority Review Voucher. This voucher can be used for priority review of a subsequent marketing application or can be sold or transferred.