ALN-CFB

PreclinicalSmall molecule

ALN-CFB is one of 3 small molecule programs targeting Paroxysmal Nocturnal Hemoglobinuria (PNH). It is currently in Preclinical.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Paroxysmal Nocturnal Hemoglobinuria (PNH)Recruiting

Paroxysmal Nocturnal Hemoglobinuria (PNH)Preclinical

Recruiting

Competition

Competitive Landscape

2 competitors in Paroxysmal Nocturnal Hemoglobinuria (PNH)

#DrugCompanyPhaseTrials

LNP023

Novartis

Phase 3

1 2

Empaveli

Apellis Pharmaceuticals

Phase 2

1 3

ALN-CFB

Regeneron

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2031

PreclinicalRecruiting

Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT07187401

Clinical

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria (PNH)1 trial

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

Recruiting· Regeneron logo

Regeneron·Preclinical·NCT07187401·24 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for ALN-CFB. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — ALN-CFB compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for ALN-CFB. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Regeneron company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Preclinical[1]
Company: Regeneron

ALN-CFB

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2031

Trials by Indication[1]

Paroxysmal Nocturnal Hemoglobinuria (PNH)1 trial

References

Drug Info

Quick Facts

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