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ALXN1101

Phase 1

Molybdenum Cofactor Deficiency (MoCD) | Small molecule | Rare Disease |BridgeBio Pharma, Inc.|Last Updated: Mar 19, 2019

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01894165Phase 1 Single Dose Study of ALXN1101 in Healthy VolunteersPHASE1 COMPLETED 24Jun 1, 2013Sep 1, 2013Mar 19, 20191 United States
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Study Endpoints
Primary Endpoints
Safety and tolerability of single dose of ALXN1101 in healthy adult subjects
following the Day 30 visit for the last study subject

Physical examination, vital signs, ECGs, laboratory evaluations, and Adverse Events.

Secondary Endpoints
PK parameters of ALXN1101
following the Day 5 visit for the last study subject
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALXN1101EXPERIMENTALFour cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV.
PlaceboPLACEBO_COMPARATORFour cohorts planned. Within each cohort, healthy volunteers are randomized to ALXN1101 IV single dose or placebo IV single dose. Each subsequent cohort is testing an increased dose of ALXN1101 IV or placebo IV.
Interventions
NameTypeDescription
ALXN1101DRUGRandomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
PlaceboDRUGRandomized to receive a single dose of ALXN1101 or placebo as per assigned cohort dose level.
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: 1. Male or female subjects ≥ 18 and ≤ 60 years of age and weight ≥ 55 kg and ≤ 100 kg 2. Willing and able to give written informed consent 3. Female subjects of child bearing potential must have a negative serum pregnancy test or must be practicing an approved contraceptive ...

Countries:United States
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