ALN-CFB / Regeneron, Alnylam - LARVOL DELTA

FIRST-IN-HUMAN, FIRST-IN-PATIENT STUDY OF ALN-CFB, AN INVESTIGATIONAL SIRNA TARGETING HEPATIC COMPLEMENT FACTOR B: TREATMENT OF PERSISTENT ANEMIA IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (EHA 2026) - P1/2 | "Results Part A of the study is actively recruiting in the UK, Canada, and South Korea. . Summary/Conclusion This FIH study will provide the first clinical evaluation of ALN-CFB in participants with PNH and persistent anemia on C5 inhibitors, generating safety, PK, PD and preliminary efficacy data and informing dose selection for future studies."

Clinical • First-in-human • P1 data • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

CHARACTERIZATION OF ALN-CFB, AN INVESTIGATIONAL SIRNA TARGETING HEPATIC COMPLEMENT FACTOR B, IN NON-HUMAN PRIMATES AND CFB-HUMANIZED MICE (EHA 2026) - P1/2 | "If translated to humans, these data suggest the potential for robust AP inhibition with convenient, infrequent SC dosing. ALN-CFB is under investigation in a phase 1/2 study for safety and efficacy in participants with PNH and persistent anemia on a C5 inhibitor (NCT07187401)."

Preclinical • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Dry Age-related Macular Degeneration • Hematological Disorders • Macular Degeneration • Nephrology • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Retinal Disorders • CFB

A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Jan 2030 ➔ Jul 2031

First-in-human • Trial completion date • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases