Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 5 trials

Late-Stage Pipeline

20%

1 trials in Phase 3/4

Results Transparency

0%

0 of 0 completed trials have results

Key Signals

4 recruiting

Enrollment Performance

Analytics

Phase 1
3(75.0%)
Phase 3
1(25.0%)
4Total
Phase 1(3)
Phase 3(1)

Activity Timeline

Global Presence

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Clinical Trials (5)

Showing 5 of 5 trials
NCT06692712Phase 3Recruiting

Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

Role: lead

NCT07447557Phase 1Not Yet Recruiting

Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)

Role: lead

NCT06151600Recruiting

A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J

Role: lead

NCT06609889Phase 1Recruiting

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

Role: collaborator

NCT05518188Phase 1Recruiting

Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

Role: lead

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