Apellis Pharmaceuticals (APLS) Citi's 2024 Global Healthcare Conference summary

Clinical development and trial results

• VALIANT phase 3 study in C3G and IC-MPGN showed strong efficacy across diverse patient groups, including adolescents, adults, pre- and post-transplant, and both C3 depleted and competent patients.

• Key endpoints such as proteinuria, eGFR stabilization, and C3c deposition clearance were met, with 70% of patients showing complete C3c disappearance after six months.

• The need for concomitant immunosuppressive medications may be significantly reduced, improving patient safety and quality of life.

• The U.S. prevalence for C3G and IC-MPGN is estimated at 5,000 patients, split evenly, with 20% being transplant recipients.

• The company’s approach included broader patient inclusion than competitors, covering both phenotypes and all transplant statuses.

Regulatory and commercialization plans

• Supplemental NDA filing for C3G/IC-MPGN is on track for early next year, with potential commercialization by year-end depending on review timelines.

• Commercial infrastructure leverages existing PNH resources, with a targeted sales force of 40–50 focused on nephrologists.

• International rights for the kidney indication are held by Sobi (Aspaveli) outside the U.S. and Asia.

• Long-term extension studies (VALE) are planned to monitor ongoing patient outcomes post-VALIANT.

Competitive landscape and differentiation

• Competitors have narrower clinical data, focusing only on C3G, adults, pre-transplant, and C3 depleted patients.

• Efficacy, especially in C3c deposition and broader patient coverage, is seen as a key differentiator.

• The company expects to be first to market in IC-MPGN and competitive in C3G, with unique positioning in post-transplant and pediatric segments.