Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 55/100

Failure Rate

17.8%

30 terminated/withdrawn out of 169 trials

Success Rate

76.2%

-10.4% vs industry average

Late-Stage Pipeline

20%

34 trials in Phase 3/4

Results Transparency

64%

61 of 96 completed trials have results

Key Signals

14 recruiting61 with results25 terminated5 withdrawn

Enrollment Performance

Analytics

Phase 2

37(34.9%)

Phase 1

28(26.4%)

Phase 3

27(25.5%)

Phase 4

7(6.6%)

N/A

7(6.6%)

106Total

Phase 2(37)

Phase 1(28)

Phase 3(27)

Phase 4(7)

+1 more

Activity Timeline

Global Presence

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Clinical Trials (169)

Showing 20 of 169 trials

NCT05734196Phase 1Active Not Recruiting

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Role: collaborator

NCT07473973Phase 3Active Not Recruiting

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Role: collaborator

NCT05368038Enrolling By Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Role: collaborator

NCT07573631Phase 2Enrolling By Invitation

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

Role: lead

NCT07441876Phase 2Recruiting

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Role: lead

NCT05270837Phase 3Active Not Recruiting

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

Role: lead

NCT04684940Phase 1Completed

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Role: lead

NCT04476862Active Not Recruiting

Cerliponase Alfa Observational Study in the US

Role: lead

NCT06168201Recruiting

VIrtual STudy in Achondroplasia for the US (VISTA)

Role: lead

NCT06280209Phase 1Active Not Recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

Role: lead

NCT06309979Recruiting

A Study to Assess Growth in Children With Idiopathic Short Stature

Role: lead

NCT07477691Phase 4Recruiting

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

Role: lead

NCT03856203Completed

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Role: collaborator

NCT06382155Phase 2Recruiting

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Role: lead

NCT06668805Phase 2Recruiting

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Role: lead

NCT06738017Phase 1Active Not Recruiting

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Role: lead

NCT04800692Phase 1Active Not Recruiting

The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication

Role: collaborator

NCT02597881Recruiting

Achondroplasia Natural History Multicenter Clinical Study

Role: collaborator

NCT07126262Phase 2Recruiting

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Role: lead

NCT05813678Recruiting

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

Role: lead