ROCKVILLE, Md. - Theriva Biologics (NYSE American: TOVX), a clinical-stage biopharmaceutical company, announced today that the European Commission has endorsed the European Medicines Agency's (EMA) recommendation to grant orphan medicinal product designation for its clinical candidate VCN-01. The designation is for the treatment of retinoblastoma, a rare form of eye cancer primarily affecting children.
The orphan medicinal product designation in the European Union (EU) is reserved for treatments targeting conditions that are life-threatening or chronically debilitating, with a prevalence not exceeding 5 in 10,000 or where the treatment would not be profitable without incentives. VCN-01 has also received orphan drug designation and rare pediatric disease designation from the United States Food and Drug Administration (FDA).
Theriva's CEO Steven A. Shallcross expressed satisfaction with the EU's decision, highlighting the need for new treatments for retinoblastoma. The company has previously reported positive outcomes from a Phase 1 trial assessing VCN-01's safety and activity in pediatric patients with refractory retinoblastoma. Theriva is collaborating with medical experts and regulatory bodies to refine VCN-01's clinical approach as a supplement to chemotherapy for children with this disease.
Retinoblastoma, which originates in the retina, is the most prevalent eye cancer in children, with an estimated incidence rate in Europe of 1 per 13,844 live births. The condition poses significant challenges, including the risk of blindness and other severe side effects from current treatments.
VCN-01 is a systemically administered oncolytic adenovirus, designed to selectively replicate within tumor cells and degrade tumor stroma, which acts as a barrier to cancer treatment. This approach aims to enhance the effectiveness of co-administered chemotherapy and immunotherapy. To date, VCN-01 has been administered to over 140 patients in various cancer clinical trials.
The orphan designation provides several benefits, including 10 years of market exclusivity upon marketing authorization in the EU, protocol assistance, and reduced regulatory fees.
Theriva Biologics focuses on developing therapies for cancer and related diseases with unmet medical needs. Alongside VCN-01, the company's portfolio includes other clinical-stage candidates targeting different conditions.
This news is based on a press release statement from Theriva Biologics, Inc.
In other recent news, Theriva Biologics has made significant strides in the clinical trial of its drug SYN-004, with positive outcomes from the second cohort of its phase 1b/2a clinical trial. The trial aims to prevent acute graft-versus-host-disease in recipients of allogeneic hematopoietic cell transplants. The study remains blinded, but initial findings suggest that adverse events were typical for allo-HCT patients and not related to the study drug.
Simultaneously, Theriva Biologics has announced a reverse stock split of its common stock at a 1-for-25 ratio, aiming to meet the NYSE American's per-share price requirements. This decision will reduce the number of outstanding common shares from approximately 25.1 million to around 1 million.
Theriva Biologics has also been granted Rare Pediatric Drug Designation by the U.S. Food and Drug Administration for VCN-01, its leading product candidate for treating retinoblastoma, a rare type of eye cancer in children. In addition, the FDA has granted Fast Track Designation to Theriva's VCN-01 for treating metastatic pancreatic adenocarcinoma in combination with chemotherapy drugs. The VIRAGE Phase 2b trial, investigating the efficacy of VCN-01 as a first-line therapy for patients with pancreatic ductal adenocarcinoma, is expected to complete patient enrollment by the third quarter of 2024. These are recent developments in Theriva Biologics' ongoing commitment to address unmet medical needs in oncology.
InvestingPro Insights
While Theriva Biologics (NYSE American: TOVX) has made significant strides in its clinical development, particularly with the EU orphan medicinal product designation for VCN-01, the company's financial health presents a mixed picture. According to InvestingPro data, Theriva's market capitalization stands at a modest $3.09 million, reflecting the early-stage nature of its pipeline.
InvestingPro Tips highlight that Theriva "holds more cash than debt on its balance sheet," which could provide some financial flexibility as it advances its clinical programs. This is particularly important for a company developing treatments for rare diseases like retinoblastoma, where development costs can be substantial.
However, another InvestingPro Tip notes that the company is "quickly burning through cash," which is not uncommon for clinical-stage biopharmaceutical companies but warrants attention from investors. This cash burn rate aligns with the company's focus on advancing its pipeline, including VCN-01, through costly clinical trials.
The stock's recent performance has been challenging, with InvestingPro data showing a 1-month price total return of -63.9% and a 1-year return of -87.91%. This volatility is typical in the biotech sector, where stock prices can fluctuate dramatically based on clinical trial results and regulatory decisions.
For investors seeking a more comprehensive analysis, InvestingPro offers 13 additional tips for Theriva Biologics, providing a deeper understanding of the company's financial position and market performance.
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