8-K: Theriva Biologics Announces Positive Phase 2b Trial Results for VCN-01 in Metastatic Pancreatic Cancer

Theriva Biologics reports positive topline outcomes from its VIRAGE Phase 2b clinical trial, showing improved survival rates for patients with metastatic pancreatic cancer treated with VCN-01 plus standard chemotherapy.

Summary

• Theriva Biologics announced positive results from the VIRAGE Phase 2b clinical trial of VCN-01 in combination with gemcitabine/nab-paclitaxel for first-line treatment of metastatic pancreatic ductal adenocarcinoma (PDAC).
• The trial included 96 newly-diagnosed metastatic PDAC patients.
• Patients treated with VCN-01 plus standard-of-care (SoC) chemotherapy had a median overall survival (OS) of 10.8 months, compared to 8.6 months for those treated with SoC alone [Hazard Ratio (HR) = 0.57, 95% CI 0.34-0.96, p=0.0546].
• Progression-free survival (PFS) also improved with VCN-01, with a median PFS of 7.0 months compared to 4.6 months for SoC alone [HR = 0.55, 95% CI 0.34-0.88, p= 0.0105].
• The median duration of response (DoR) doubled in the VCN-01+SoC arm to 11.2 months, compared to 5.4 months in the SoC arm [HR = 0.22, 95% CI 0.08-0.62, p=0.0035].
• Patients who received two doses of VCN-01 and at least four cycles of chemotherapy had a median OS of 14.8 months, compared to 11.6 months for those who received at least four cycles of chemotherapy alone [HR=0.44, 95% CI: 0.21-0.92, p=0.046].
• The adverse event profile of VCN-01 was consistent with prior trials, with transient and reversible AEs like pyrexia, flu-like illness, vomiting, nausea, and elevated transaminases.
• The company intends to discuss the presentation which is furnished as Exhibit 99.2 to this Current Report on Form 8-K.

Sentiment

Score: 8

Explanation: The document presents positive clinical trial results, suggesting a potential breakthrough in pancreatic cancer treatment. While risks remain, the overall tone is optimistic and suggests a positive outlook for the company.

Positives

• VCN-01 in combination with standard chemotherapy showed statistically significant improvements in overall survival, progression-free survival, and duration of response in patients with metastatic pancreatic cancer.
• The hazard ratios for survival parameters in the VCN-01 treatment group were significantly reduced.
• The adverse event profile of VCN-01 was manageable and consistent with previous trials.
• Regulatory advice from the FDA and EMA is expected to facilitate the design of a Phase 3 confirmatory trial.
• The company has Orphan Drug Designation and Fast Track Designation from the FDA for VCN-01.

Negatives

• The p-value for overall survival (p=0.0546) was slightly above the conventional threshold of 0.05 for statistical significance, although the hazard ratio was favorable.
• The most common VCN-01 related AEs (pyrexia, flu-like illness, vomiting, nausea, and elevated transaminases) were transient and reversible.

Risks

• The success of a Phase 3 confirmatory trial is not guaranteed.
• Regulatory approval for VCN-01 is not assured.
• The company's ability to engage with industry partners and secure funding for further development is subject to market conditions and other factors.
• Developments by competitors could render VCN-01 obsolete or non-competitive.

Future Outlook

The company expects the positive data and recent advice from the FDA and EMA to facilitate engagement with industry partners and enable the design of a Phase 3 confirmatory trial, which, if successful, could lead to a new therapeutic option for metastatic PDAC patients.

Management Comments

• Steven A. Shallcross, Chief Executive Officer of Theriva Biologics, stated that the data demonstrates the potential opportunity for VCN-01 to benefit metastatic PDAC patients treated with gemcitabine/nab-paclitaxel SoC chemotherapy.
• Shallcross noted that the significantly reduced hazard ratios for survival parameters provide compelling evidence that VCN-01 in combination with gemcitabine/nab-paclitaxel may extend the lives of metastatic PDAC patients.

Industry Context

Pancreatic cancer is a highly fatal disease with limited treatment options, making the positive results of the VIRAGE trial a potentially significant advancement in the field. The company is targeting a high unmet need with its novel oncolytic adenovirus platform.

Comparison to Industry Standards

• The hazard ratios for OS (0.57), PFS (0.55), and DoR (0.22) in the VIRAGE trial compare favorably to values reported for a Phase 3 trial comparing NALIRIFOX with gemcitabine/nab-paclitaxel (0.83, 0.69, and 0.67 respectively).
• The NALIRIFOX trial, as reported by Wainberg et al. in The Lancet (2023), had a median age of 65 years and an ECOG score of 1 in 57% of the NALIRIFOX group, providing a benchmark for comparison.

Stakeholder Impact

• Positive results could lead to improved treatment options and outcomes for patients with metastatic pancreatic cancer.
• Successful development and commercialization of VCN-01 could increase shareholder value.
• The company's employees may benefit from the company's success and growth.

Next Steps

• The company intends to engage with industry partners to facilitate the design of a Phase 3 confirmatory trial.
• The company will continue to develop VCN-01 and other oncolytic virus therapeutics.
• The company will host a live virtual event to review and discuss the data from the VIRAGE trial of VCN-01 on Wednesday, May 7th 2025, 8:00 AM EDT.

Key Dates

Keywords

VCN-01, pancreatic cancer, metastatic PDAC, clinical trial, VIRAGE, oncolytic adenovirus, Theriva Biologics, chemotherapy, survival, FDA, EMA