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RGLS4326

Phase 1

Polycystic Kidney Disease, Autosomal Dominant | Small molecule | Nephrology |Regulus Therapeutics Inc.|Last Updated: Dec 15, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04536688A Study of RGLS4326 in Patients With Autosomal Dominant Polycystic Kidney DiseasePHASE1 COMPLETED 19Oct 13, 2020Nov 12, 2021Dec 15, 202112 United States
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Study Endpoints
Primary Endpoints
Changes in primary biomarker levels from baseline
Baseline to Day 44

Changes in polycystin-1 (PC-1) and polycystin-2 (PC-2) protein levels in urinary exosomes from baseline to Day 44

Secondary Endpoints
Changes in secondary biomarker levels from baseline
Baseline to Day 44
Pharmacokinetics (Cmax)
Baseline to Day 44
Pharmacokinetics (Tmax)
Baseline to Day 71
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RGLS4326 1 mg/kg Q2WEXPERIMENTALEligible participants will receive subcutaneous injection of 1 mg/kg of RGLS4326 every other week for 4 doses
RGLS4326 0.3 mg/kg Q2WEXPERIMENTALEligible participants will receive subcutaneous injection of 0.3 mg/kg of RGLS4326 every other week for 4 doses
RGLS4326 0.1 or 0.5 mg/kg Q2WEXPERIMENTALEligible participants will receive subcutaneous injection of 0.1 or 0.5 mg/kg of RGLS4326 every other week for 4 doses
Interventions
NameTypeDescription
RGLS4326DRUGSolution for subcutaneous injection
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Male or female ADPKD patients 18 to 70 years old * Class 1C, 1D, or 1E Mayo Imaging Classification of ADPKD (based upon prior MRI or CT Scan or MRI obtained during screening) * Estimated GFR at Screening between 30 to 90 mL/min/1.73 m\^2 calculated by the investigator using th...

Countries:United States
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