Phio Pharmaceuticals (PHIO) investor relations material

Company overview and technology

• Focuses on immunotherapy for skin cancers using INTASYL, a short interfering RNA technology with broad patents extending to 2044.

• Operates as a lean virtual organization with eight employees and four subject matter experts, all with prior drug approval experience.

• INTASYL selectively targets and silences specific genes, notably PD-1, to reactivate T cells and enhance tumor cell killing.

• Technology is protected by 54 issued patents, covering composition, gene targets, and therapeutic indications.

Clinical development and results

• Lead program PH-762 targets PD-1 and completed a phase I-B trial in January, showing strong efficacy and safety.

• In the highest dose cohort, 85% response rate was observed, with 100% tumor clearance in four of six patients.

• No dose-limiting toxicities or immune-related adverse events were reported, even at 20-fold higher doses.

• Treatment is administered intratumorally, offering convenience and minimizing off-target effects.

Market opportunity and strategy

• Addressable market for cutaneous squamous cell carcinoma is estimated at $20 billion, with rising incidence due to aging population.

• No FDA-approved drugs exist for early-stage disease; current standard is surgical intervention.

• Plans to monetize non-strategic assets through out-licensing, while focusing internal resources on two main programs.

• Secondary program PH-894 targets BRD4 and is IND-ready, with future development pending FDA feedback on PH-762.