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BIIB074

Phase 2

Primary Inherited Erythromelalgia | Small molecule | Rare Disease |Biogen Inc.|Last Updated: Jan 10, 2018

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-BlindCONTROLLED
Total Trials1
Total Enrollment8
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02917187A Phase 2a Study of BIIB074 in the Treatment of ErythromelalgiaPHASE2 COMPLETED 8Sep 9, 2016Jan 5, 2017Jan 10, 20185 United States
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Study Endpoints
Primary Endpoints
Weekly average severity of paroxysms
Day 1 to Week 12

11-point Pain Intensity Numerical Rating Scale (PI-NRS) is used to assess EM paroxysmal pain. PI-NRS is an 11-point pain intensity numerical rating scale, where 0=no pain and 10=worst possible pain. Weekly average is defined as the total of severity scores during a week divided by the total number of paroxysms during that week.

Secondary Endpoints
Weekly maximum severity of paroxysms
Day 1 to Week 12
Weekly average and maximum number of paroxysms
Day 1 to Week 12
Weekly average and weekly maximum duration of paroxysms
Day 1 to Week 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Randomized Group 1EXPERIMENTALAfter two week run-in, BIIB074 three times a day (TID) followed by placebo (TID) after two week washout period
Randomized Group 2EXPERIMENTALAfter two week run-in, Placebo three times a day (TID) followed by BIIB074 (TID) after two week washout period
Interventions
NameTypeDescription
BIIB074DRUGAdministered as specified in the treatment arm
PlaceboDRUGMatched placebo
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites5

Key Inclusion Criteria: * A diagnosis of primary inherited EM with family history of EM made at least 3 months from initial diagnosis. * Failed at least one prior treatment for EM (defined as an inadequate response or intolerance to treatment). * Approved concomitant medications must have been stab...

Countries:United States
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