APL-3007 / Biogen - LARVOL DELTA

Biogen ends Apellis research programs, cuts ‘small number’ of roles after $5.6B buyout (FierceBiotech) - "As part of the move, 'a small number of roles within the research organization have been eliminated,” the spokesperson added. Biogen continues to assess Apellis’ clinical and preclinical portfolio to “further evaluate strategic fit,' the company representative said...In cutting these research efforts, Biogen is making explicit its desire to focus on Apellis’ Empaveli and Syfovre, marketed drugs which share the active ingredient pegcetacoplan and which served as the centerpiece of the March deal....Besides the approved Apellis meds, the biotech had been conducting research on clinical-stage asset APL-3007 before the merger, according to its pipeline."

Licensing / partnership • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria

A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio (clinicaltrials.gov) - P2 | N=240 | Recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Trial completion date: Mar 2028 ➔ Nov 2027 | Initiation date: Sep 2025 ➔ Jun 2025 | Trial primary completion date: Sep 2027 ➔ Jun 2027

Trial completion date • Trial initiation date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio (clinicaltrials.gov) - P2 | N=240 | Recruiting | Sponsor: Apellis Pharmaceuticals, Inc. | Trial completion date: Nov 2027 ➔ Mar 2028 | Initiation date: Jun 2025 ➔ Sep 2025 | Trial primary completion date: Jun 2027 ➔ Sep 2027

Trial completion date • Trial initiation date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

First time phase 1 results of APL-3007: A novel systemic C3 siRNA for geographic atrophy secondary to AMD (ARVO 2026) - "APL-3007 is a novel systemic C3 siRNA evaluated in phase 1 monotherapy and in phase 2 combination trials with intravitreal pegcetacoplan, targeting a comprehensive C3/C3b blockade in both the retina and choroid. Conclusions APL-3007 was safe and well tolerated and resulted in on-target PD activity. A single dose phase 1b and multi-dose phase 2 clinical study are ongoing to evaluate safety and efficacy of combined local C3/C3b inhibition and systemic C3 silencing in GA patients."

First-in-human • P1 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio (clinicaltrials.gov) - P2 | N=240 | Recruiting | Sponsor: Apellis Pharmaceuticals, Inc.

New P2 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders