A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy
Latest Information Update: 11 Jul 2026
At a glance
- Drugs ALN CFB (Primary)
- Indications Anaemia; Paroxysmal nocturnal haemoglobinuria
- Focus Adverse reactions; First in man
- Sponsors Regeneron Pharmaceuticals
Most Recent Events
- 14 Jan 2026 Planned End Date changed from 4 Jan 2030 to 15 Jul 2031.
- 14 Jan 2026 Planned primary completion date changed from 11 Oct 2029 to 29 Nov 2029.
- 14 Jan 2026 Planned initiation date changed from 28 Nov 2025 to 16 Jan 2026.