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Also known as ALN-PRNP
ALN-6457 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Dose Escalation | EXPERIMENTAL | - |
| Cohort Expansion | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| ALN-6457 | DRUG | Administered per the protocol |
Key Inclusion Criteria: 1. Participant must meet all of the criteria under either: 1. sporadic CJD as described in the protocol or 2. genetic PrD, as described in the protocol 2. Has a Body Mass Index (BMI) \<35 kg/m2 (inclusive) at the screening visit 3. Able to understand and complete tria...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE2 | ION717 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | ALN-6457 |
ALN-6457 is an investigational small molecule being developed by Regeneron Pharmaceuticals, Inc. for the treatment of prion disease, a neurological condition. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is designed for adult patients with symptomatic prion disease.
ALN-6457 is being studied for the treatment of prion disease in adults with symptomatic disease. Prion disease is a rare, progressive neurological disorder. The drug is investigational and has not been approved for any indication. Its only clinical trial enrolls adults aged 18 years and older with symptomatic prion disease.
ALN-6457 is being developed by Regeneron Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol REGN. Regeneron is the sponsor of the Phase 1 trial evaluating the drug in adult patients with symptomatic prion disease.
ALN-6457 is in Phase 1 clinical development. It is an investigational small molecule and has not received FDA approval. The single Phase 1 trial is currently not yet recruiting and is designed to enroll 60 adult participants with symptomatic prion disease.
ALN-6457 is being evaluated in one Phase 1 trial, NCT07832370, titled A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD). The trial has a controlled design, is not yet recruiting, and plans to enroll 60 participants aged 18 years and older of all sexes.