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RG012

Phase 1

Alport Syndrome | Small molecule | Other |Sanofi|Last Updated: Apr 25, 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
Double-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment4
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03373786A Study of RG-012 in Subjects With Alport SyndromePHASE1 COMPLETED 4Dec 22, 2017May 20, 2019Apr 25, 20227 United States
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Study Endpoints
Primary Endpoints
Safety - Adverse Events
8 weeks

Incidence and severity of adverse events

Effect of RG-012 on renal microRNA-21 (miR-21)
8 weeks

Change in miR-21 expression in renal tissue

Secondary Endpoints
Pharmacokinetic (PK) parameter - Cmax
8 weeks
Pharmacokinetic (PK) parameter - Tmax
8 weeks
Pharmacokinetic (PK) parameter - AUC
8 weeks
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RG-012 Single DoseEXPERIMENTAL1.5 mg/kg RG012 subcutaneous injection
RG012 Every Other WeekEXPERIMENTAL1.5 mg/kg RG012 subcutaneous injections every other week
Interventions
NameTypeDescription
RG012DRUGRG012 in 0.3% sodium chloride
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: 1. Males or females, ages 18 to 65 years 2. Confirmed diagnosis of Alport syndrome 3. eGFR between 40 and 90 mL/min/1.73m2 4. Proteinuria of at least 300 mg protein/g creatinine 5. For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 3...

Countries:United States
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