Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 20/100

Failure Rate

14.3%

1 terminated/withdrawn out of 7 trials

Success Rate

66.7%

-19.8% vs industry average

Late-Stage Pipeline

43%

3 trials in Phase 3/4

Results Transparency

150%

3 of 2 completed trials have results

Key Signals

1 recruiting3 with results

Enrollment Performance

Analytics

Phase 2

3(50.0%)

Phase 3

3(50.0%)

6Total

Phase 2(3)

Phase 3(3)

Activity Timeline

Global Presence

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Clinical Trials (7)

Showing 7 of 7 trials

NCT07221292Phase 3Not Yet Recruiting

Pivotal Study of N-acetyl-L-leucine for CACNA1A

Role: lead

NCT03759678Phase 2Terminated

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Role: lead

NCT07380165Unknown

Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)

Role: lead

NCT06673056Phase 3Active Not Recruiting

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

Role: lead

NCT05163288Phase 3Recruiting

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Role: lead

NCT03759665Phase 2Completed

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

Role: lead

NCT03759639Phase 2Completed

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Role: lead

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