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REN001

Phase 1

McArdle Disease | Small molecule | Other |OnKure Therapeutics, Inc.|Last Updated: Jan 12, 2022

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04226274A Study of the Safety of REN001 in Patients With McArdle DiseasePHASE1 COMPLETED 19Dec 10, 2019Oct 11, 2021Jan 12, 20222 Spain, United Kingdom
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Study Endpoints
Primary Endpoints
Adverse Events
up to Week 12

Number of participants with Adverse Events (AEs) as a measure of safety and tolerability

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
REN001EXPERIMENTALOral
Interventions
NameTypeDescription
REN001DRUGOnce daily for 12 weeks
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Subjects must give written, signed and dated informed consent * Confirmed diagnosis of McArdle Disease * Subjects must be able to remain on stable medication through the study and specifically must not commence or have changes to agents that affect metabolism such as medicatio...

Countries:SpainUnited Kingdom
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