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ALN-CFB · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| Single-Ascending Dose Escalation | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ALN-CFB | DRUG | Administered as defined in the protocol |
| Placebo | DRUG | Administered as defined in the protocol |
Key Inclusion Criteria: 1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing 2. Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as describ...
ALN-CFB is an investigational small interfering RNA (siRNA) being developed for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have persistent anemia despite treatment with a C5 inhibitor. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
ALN-CFB targets complement factor B, a protein involved in the complement system. By silencing complement factor B, the siRNA is designed to address persistent anemia in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are already on a C5 inhibitor.
ALN-CFB is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. The drug is currently in Phase 1 clinical development for Paroxysmal Nocturnal Hemoglobinuria (PNH).
ALN-CFB is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is recruiting participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) and persistent anemia.
ALN-CFB is being studied in a single Phase 1 trial, NCT07187401, which is currently recruiting. This first-in-human study evaluates the safety and efficacy of ALN-CFB in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) with persistent anemia on a C5 inhibitor. The trial is randomized, double-blind, and placebo-controlled, with an enrollment target of 24 participants.
ALN-CFB is the sole name provided for this investigational drug; no alternative names have been listed. It is a small interfering RNA (siRNA) targeting complement factor B, developed by Regeneron Pharmaceuticals for Paroxysmal Nocturnal Hemoglobinuria (PNH).