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ALN-6457

Phase 1

Prion Disease | Small molecule | Other |Regeneron Pharmaceuticals, Inc.|Last Updated: Sep 22, 2026

Development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Sep 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

ALN-6457 · 1 trial · 1 indication

Phase 1 1
NCT07832370A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD)Prion Disease
NOT YET_RECRUITING60 Analytics
PHASE1NOT YET_RECRUITING
A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD)
Prion DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Occurrence of Treatment Emergent Adverse Events (TEAEs)
Through Week 156
Severity of TEAEs
Through Week 156
Percentage change in concentration of Neurofilament Light chain (NfL) in plasma from baseline
Through Week 130

Secondary Endpoints

Concentration of Prion Protein (PrP) in CSF
Through Week 130
Change from baseline in concentration of PrP in CSF
Through Week 130
Concentration of ALN-6457 and its major metabolites in plasma
Through Week 130
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose EscalationEXPERIMENTAL -
Cohort ExpansionEXPERIMENTAL -

Interventions

NameTypeDescription
ALN-6457DRUGAdministered per the protocol
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

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...

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Recent Changes (Last 90 Days)

LOWSep 22, 2026NCT07832370NEW_TRIAL: changed
LOWSep 22, 2026NCT07832370NEW_TRIAL: changed

Frequently asked questions about ALN-6457

What is ALN-6457?

ALN-6457 is an investigational small molecule being developed by Regeneron Pharmaceuticals, Inc. (ticker: REGN) for the treatment of prion disease. It is currently in Phase 1 clinical development and has not been approved by the FDA. The program is focused on adult participants with symptomatic prion disease.

What is ALN-6457 used for?

ALN-6457 is being studied for the treatment of prion disease, a rare and progressive neurodegenerative condition. The only registered trial enrolls adult participants with symptomatic prion disease. Because the drug remains in Phase 1 testing, it is investigational and is not available as an approved therapy for prion disease or any other indication.

Who makes ALN-6457?

Regeneron Pharmaceuticals, Inc., which trades under the ticker REGN, is the developer of ALN-6457. The company is running the Phase 1 program for the asset in prion disease. Regeneron is the sponsor of record for the trial listed on ClinicalTrials.gov.

What phase is ALN-6457 in?

ALN-6457 is in Phase 1 clinical development. It is an investigational small molecule and has not received FDA approval. The program currently consists of a single Phase 1 trial in adult participants with symptomatic prion disease, and that trial has not yet begun recruiting.

What clinical trials is ALN-6457 in?

ALN-6457 is being evaluated in one registered clinical trial, NCT07832370, titled A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD). The Phase 1 study is controlled, is not yet recruiting, and plans to enroll 60 participants aged 18 years and older, with no healthy volunteer enrollment.

How many patients are enrolled in the ALN-6457 trial?

The Phase 1 trial of ALN-6457, NCT07832370, plans to enroll 60 adult participants with symptomatic prion disease. Enrollment has not started because the study status is not yet recruiting. The trial is controlled and enrolls participants aged 18 years and older of any sex.