Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

High Risk

Score: 65/100

Failure Rate

25.0%

4 terminated/withdrawn out of 16 trials

Success Rate

73.3%

-13.2% vs industry average

Late-Stage Pipeline

19%

3 trials in Phase 3/4

Results Transparency

45%

5 of 11 completed trials have results

Key Signals

5 with results

Enrollment Performance

Analytics

Phase 2

5(33.3%)

Phase 1

4(26.7%)

Phase 3

3(20.0%)

N/A

2(13.3%)

Early Phase 1

1(6.7%)

15Total

Phase 2(5)

Phase 1(4)

Phase 3(3)

N/A(2)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (16)

Showing 16 of 16 trials

NCT04231266Phase 2Active Not Recruiting

Multi-Center Study of ManNAc for GNE Myopathy

Role: lead

NCT00227266Phase 2Completed

Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

Role: collaborator

NCT06260748Phase 3Withdrawn

A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)

Role: lead

NCT00374075Phase 1Completed

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

Role: collaborator

NCT02043587Phase 2Terminated

Chemotherapy With Liposomal Cytarabine CNS Prophylaxis for Adult Acute Lymphoblastic Leukemia & Lymphoblastic Lymphoma

Role: collaborator

NCT01420627Phase 3Completed

EZN-2279 in Patients With ADA-SCID

Role: lead

NCT01954017Phase 1Completed

STP206 for the Prevention of Necrotizing Enterocolitis (NEC)

Role: lead

NCT01878708Early Phase 1Terminated

A Pilot Study of Oncaspar® + Dexamethasone in Patients With Relapsed or Refractory T-Cell Lymphoma

Role: collaborator

NCT00905034Phase 2Completed

Methotrexate, Vincristine, Pegylated L-Asparaginase and Dexamethasone (MOAD) in Acute Lymphoblastic Leukemia (ALL) Salvage

Role: collaborator

NCT00661453Phase 1Completed

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

Role: collaborator

NCT00010426Not ApplicableCompleted

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

Role: collaborator

NCT02229721Phase 2Completed

Effect of Chronic Intranasal Oxytocin Administration on Sexual Function in Pre- and Postmenopausal Women

Role: collaborator

NCT00922324Phase 1Completed

Safety and Tolerability of STP206 in Healthy Adult Subjects

Role: lead

NCT00984750Phase 3Completed

Acetyl-L-Carnitine in Type 2 Diabetes

Role: collaborator

NCT00351234Completed

Carnitine Levels and Carnitine Supplementation in Type I Diabetes

Role: collaborator

NCT00892632Not ApplicableTerminated

Confocal Endomicroscopy for Biliary Strictures -Phase I

Role: collaborator

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