Fortress Biotech (FBIO) Corporate presentation summary

Investment highlights

• Triplex vaccine shows positive Phase 2 data, achieving a 50% reduction in CMV events in HSCT patients and demonstrating strong safety and immunogenicity profiles.

• Addresses a significant unmet need in CMV prevention for transplant recipients, with no approved immunotherapy and limitations in current antiviral treatments.

• Large market opportunity with over 100,000 organ transplants performed globally each year and potential peak sales exceeding $1 billion in the US/EU SOT market alone.

• Robust IP and regulatory exclusivity, including 7–10 years orphan exclusivity and 12 years biologic exclusivity, with patents extending to 2033.

• Multiple ongoing and planned trials in liver, kidney, HSCT, and HIV/CMV co-infection, with Phase 2 studies in liver and kidney transplants commencing in 2024 and 2025.

Clinical development and efficacy

• Four completed clinical trials confirm Triplex’s safety, immunogenicity, and efficacy, including a 50% reduction in CMV events versus placebo in Phase 2 HSCT.

• Triplex induces rapid, robust, and durable CD4 and CD8 T-cell responses, critical for CMV control post-transplant.

• Demonstrated comparable or superior efficacy to standard antivirals, with more convenient dosing and no observed resistance.

• Ongoing studies include combinations with CAR-T therapies and trials in HIV/CMV co-infection, expanding the clinical dataset.

• Planned Phase 2 and 3 trials target broader transplant populations, including donor vaccination and pediatric cohorts.

Market opportunity and competitive landscape

• SOT and HSCT represent well-defined orphan markets with high unmet need and favorable reimbursement dynamics.

• Over 40,000 solid organ transplants and 10,000+ allogeneic HSCTs occur annually in the US, with similar numbers in the EU.

• Current antivirals have significant toxicity, resistance, and dosing limitations; Triplex offers a differentiated profile with no drug resistance and durable immune reconstitution.

• Competitor HB101 failed to show robust CD4 T-cell responses and efficacy in kidney transplant, highlighting Triplex’s superior immunogenicity.

• Commercialization requires a modest salesforce targeting specialized transplant centers, with potential for rapid uptake.