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RGLS8429

Phase 1

Autosomal Dominant Polycystic Kidney Disease | Small molecule | Nephrology |Regulus Therapeutics Inc.|Last Updated: Jun 3, 2025

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-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment68
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05521191A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney DiseasePHASE1 COMPLETED 68Oct 6, 2022Mar 27, 2025Jun 3, 202521 United States
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Study Endpoints
Primary Endpoints
Safety and tolerability of RGLS4829: Incidence of AEs
Baseline to Day 113

Incidence of adverse events over time

Impact of RGLS8429 on ADPKD biomarkers
Baseline to Day 113

Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers

Secondary Endpoints
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Baseline to Day 113
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RGLS8429EXPERIMENTALThe randomized,double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). * Cohort 1: first dose level of RGLS8429 or placebo * Cohort 2: second dose level of RGLS8429 or placebo * Cohort 3: third dose level of RGLS8429 or placebo
PlaceboEXPERIMENTALThe randomized double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). * Cohort 1: first dose level of RGLS8429 or placebo * Cohort 2: second dose level of RGLS8429 or placebo * Cohort 3: third dose level of RGLS8429 or placebo
Open Label Fixed Dose RGLS8429EXPERIMENTALThe open-label fixed dose part of the study (Part B and Cohort 4) will consist of a single cohort of up to 30 subjects each receiving 300 mg RGLS8429.
Interventions
NameTypeDescription
RGLS8429DRUGSolution for subcutaneous injection
PlaceboDRUGSolution for subcutaneous injection
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites21

Key Inclusion Criteria: 1. Male or female ADPKD patients, 18 to 70 years old 2. Class 1C, 1D, or 1E Mayo Imaging Classification of ADPKD (based upon either the MRI obtained during screening, or a prior MRI obtained within 5 years of screening with documented Mayo classification) 3. eGFR between 30 ...

Countries:United States
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