Theriva™ Biologics Receives Rare Pediatric Drug Designation by the U.S. FDA for VCN-01 for the Treatment of Retinoblastoma
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.
Theriva™ Biologics (NYSE American: TOVX) ha ricevuto la Designazione di Farmaco Pediatrico Raro (RPDD) dalla FDA degli Stati Uniti per VCN-01 nel trattamento del retinoblastoma. VCN-01, il candidato principale di Theriva, è un adenovirus oncolitico sistemico e selettivo che degrada il stroma. Questa designazione segue una precedente designazione di farmaco orfano per la stessa indicazione.
La designazione evidenzia l'urgenza di nuovi trattamenti per i pazienti pediatrici affetti da retinoblastoma. Theriva sta collaborando con medici e regolatori per perfezionare la sua strategia clinica per VCN-01 come aggiunta alla chemioterapia. I recenti risultati della sperimentazione di Fase 1 per VCN-01 intravitreale nel retinoblastoma refrattario sono stati ritenuti positivi dal Comitato di Monitoraggio dello studio.
Se VCN-01 riceve l'approvazione della FDA per il retinoblastoma, Theriva potrebbe essere idonea per un Voucher di Revisione Prioritaria, che può essere utilizzato o venduto.
Theriva™ Biologics (NYSE American: TOVX) ha recibido la Designación de Medicamento Pediátrico Raro (RPDD) de la FDA de EE.UU. para VCN-01 en el tratamiento del retinoblastoma. VCN-01, el candidato principal de Theriva, es un adenovirus oncolítico sistémico y selectivo que degrada el estroma. Esta designación sigue a una anterior designación de medicamento huérfano para la misma indicación.
La designación destaca la urgente necesidad de nuevos tratamientos para los pacientes pediátricos con retinoblastoma. Theriva está colaborando con médicos y reguladores para refinar su estrategia clínica para VCN-01 como complemento a la quimioterapia. Los resultados del ensayo de Fase 1 recientes para VCN-01 intravítreo en retinoblastoma refractario fueron considerados positivos por el Comité de Monitoreo del estudio.
Si VCN-01 recibe la aprobación de la FDA para el retinoblastoma, Theriva podría ser elegible para un Voucher de Revisión Prioritaria, que puede ser utilizado o vendido.
Theriva™ Biologics (NYSE American: TOVX)는 미국 FDA로부터 VCN-01에 대해 희귀 소아약물 지정(RPDD)을 받았습니다. VCN-01은 Theriva의 주요 후보 물질로, 전신 선택적 간섭 조직 분해 종양 바이러스입니다. 이는 동일한 적응증에 대해 이전에 부여된 오르판 약물 지정에 이어진 것입니다.
이 지정은 소아 망막모세포종 환자들에게 새로운 치료법의 긴급한 필요성을 강조합니다. Theriva는 VCN-01을 항암화학요법의 보조요법으로 삼기 위해 의사 및 규제 기관과 협력하고 있습니다. 최근 1상 임상시험 결과는 난치성 망막모세포종에서의 VCN-01의 안구 주사에 대해 연구 모니터링 위원회에 의해 긍정적으로 평가되었습니다.
VCN-01이 망막모세포종에 대해 FDA 승인을 받을 경우, Theriva는 사용할 수 있거나 판매할 수 있는 우선심사 권한 바우처를 받을 수 있습니다.
Theriva™ Biologics (NYSE American: TOVX) a reçu la désignation de médicament pédiatrique rare (RPDD) de la FDA américaine pour VCN-01 dans le traitement du rétinoblastome. VCN-01, le candidat principal de Theriva, est un adénovirus oncolytique systémique et sélectif qui dégrade le stroma. Cette désignation fait suite à une précédente désignation de médicament orphelin pour la même indication.
Cette désignation souligne le besoin urgent de nouveaux traitements pour les patients pédiatriques atteints de rétinoblastome. Theriva collabore avec des médecins et des régulateurs pour affiner sa stratégie clinique pour VCN-01 en tant que complément à la chimiothérapie. Les récents résultats des essais de phase 1 pour VCN-01 intravitréen dans le rétinoblastome réfractaire ont été jugés positifs par le Comité de surveillance de l'étude.
Si VCN-01 reçoit l'approbation de la FDA pour le rétinoblastome, Theriva pourrait être éligible à un bon de révision prioritaire, qui peut être utilisé ou vendu.
Theriva™ Biologics (NYSE American: TOVX) hat von der U.S. FDA die Rare Pediatric Drug Designation (RPDD) für VCN-01 zur Behandlung von Retinoblastom erhalten. VCN-01, der Hauptkandidat von Theriva, ist ein systemisches, selektives, stromadernes onkolytisches Adenovirus. Diese Auszeichnung folgt einer vorherigen Orphan Drug Designation für dieselbe Indikation.
Die Auszeichnung hebt den dringenden Bedarf an neuen Behandlungen für pädiatrische Retinoblastom-Patienten hervor. Theriva arbeitet mit Ärzten und Aufsichtsbehörden zusammen, um ihre klinische Strategie für VCN-01 als Ergänzung zur Chemotherapie zu verfeinern. Die aktuellen Phase-1-Studienergebnisse zu intravitrealem VCN-01 bei refraktärem Retinoblastom wurden vom Studienüberwachungsausschuss als positiv bewertet.
Wenn VCN-01 von der FDA für Retinoblastom genehmigt wird, könnte Theriva für einen Priority Review Voucher in Frage kommen, der verwendet oder verkauft werden kann.
- 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
- None.
Insights
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.
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 to200 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
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?
What is VCN-01 and how does it work in treating retinoblastoma?
What were the results of the Phase 1 trial for VCN-01 in retinoblastoma patients?
What potential benefits could Theriva Biologics (TOVX) receive if VCN-01 is approved for retinoblastoma?
