Phio Pharmaceuticals (PHIO) Life Sciences Virtual Investor Forum summary

Market opportunity and technology overview

• Focus on cutaneous squamous cell carcinoma (cSCC), a cancer with 1.8 million annual cases and a $20 billion addressable market.

• INTASYL, a patented short interfering RNA technology, selectively silences genes to enhance T-cell tumor killing.

• The technology is protected by 54 patents through 2044, covering chemistry, gene targets, compounds, and indications.

• Ultra-lean team structure with eight employees and four subject matter experts, all with extensive dermatology and drug development experience.

• Originates from Nobel Prize-winning RNA interference research, enabling precise gene silencing.

Clinical development and trial results

• PH-762 targets the PD-1 gene and is administered via direct intratumoral injection, avoiding systemic side effects.

• Phase 1b trial in cSCC showed 85% pathological response in the highest dose cohort, with 4 of 6 patients achieving complete tumor clearance.

• No immune-related or treatment-related toxicities observed across a 20-fold dose escalation.

• Treatment is office-based, convenient, and economically favorable for physicians, with no need for infusion centers or capital outlays.

• PH-762 offers a tissue-sparing alternative to surgery, minimizing recovery time and surgical fatigue.

Pipeline, next steps, and financials

• PH-894, targeting the BRD4 gene, has completed IND-enabling studies and shows a clean toxicology profile.

• Next clinical study for PH-762 is being designed, with FDA guidance expected in late Q2 and trial start targeted for Q1 2027.

• Manufacturing and toxicology studies are underway to support regulatory submissions.

• Cash runway extends into H1 2027, with no debt and 11.6 million shares outstanding.

• Out-licensing and partnership discussions are ongoing for non-core programs and broader INTASYL applications.