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ATR-101

Phase 2

Congenital Adrenal Hyperplasia | Small molecule | Rare Disease |Tempest Therapeutics, Inc.|Last Updated: Mar 10, 2021

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 Enrollment10
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02804178A Study of ATR-101 for the Treatment of Congenital Adrenal HyperplasiaPHASE2 COMPLETED 10 — — May 18, 2016Aug 17, 2017Mar 10, 20216 United States
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Study Endpoints
Primary Endpoints
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.

17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ATR-101EXPERIMENTALAscending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
Interventions
NameTypeDescription
ATR-101DRUG125-1000 mg twice per week
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency based on: Documented genetic mutation in the CYP21A2 enzyme consistent with a diagnosis of classic CAH, or historical documentation of elevated 17-hydroxyprogesterone * Biochemical marker of diseas...

Countries:United States
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