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PRN1008

Phase 2

Pemphigus Vulgaris | Small molecule | Dermatology |Sanofi|Last Updated: Feb 13, 2023

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment42
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02704429A Study of PRN1008 in Adult Patients With Pemphigus VulgarisPHASE2 COMPLETED 42 — — Jan 22, 2016Jan 10, 2020Feb 13, 202313 Australia, Croatia +3
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Study Endpoints
Primary Endpoints
Percentage of Participants With Treatment-emergent Adverse Events
Part A: until 24 weeks and Part B: until 28 weeks

Treatment-emergent adverse events (TEAEs) including clinically significant changes in physical examination, laboratory tests, and vital signs. An AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. TEAEs were defined as AEs that developed or worsened or became serious during on-treatment phase that was defined as the time from the start of study drug up to study completion.

Percentage of Participants Who Are Able to Achieve Control of Disease Activity (CDA) Within 4 Weeks of Starting PRN1008 Treatment Without the Need for Doses of Prednisone or Prednisolone >0.5 mg/kg
4 weeks

CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.

Secondary Endpoints
Percentage of Participants Able to Achieve Control of Disease Activity (CDA) Without Corticosteroids Within 4 Weeks
4 weeks
Percentage of Participants Able to Achieve a Complete Response (CR) Without Corticosteroids
Part A: 12 weeks treatment and Part B: 24 weeks treatment
Percentage of Participants Able to Achieve Complete Remission (CR) Without the Need for Doses of Prednisone or Prednisolone of Greater Than 0.5mg/kg
Part A: 12 weeks treatment and Part B: 24 weeks treatment
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PRN1008EXPERIMENTALPart A: Open-label PRN1008, 12 weeks; 12 weeks follow-up; Part B: Open-label PRN1008, 24 weeks; 4 weeks follow-up
Interventions
NameTypeDescription
PRN1008DRUGPart A dosing was initiated administering 400 mg BID. Intrapatient dose adjustments (reductions and increases) were permitted based upon tolerability and clinical response with the maximum dose allowed up to 600 mg BID for 12 weeks. Part B initial dosing was 400 mg QD for 2 weeks with dose escalation to 400 mg BID at the discretion of the Investigator for the purposes of investigating dose response and identifying the minimal efficacious dose of rilzabrutinib for 24 weeks.
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: * Male or female patients, aged 18 to 80 years old, with biopsy-proven, mild-moderate PV (PDAI 8 to 45) in Part A and mild to severe PV in Part B (PDAI 8 to 60) that are either: * newly diagnosed patients (i.e. naïve to an effective induction treatment regimen) for whom an in...

Countries:AustraliaCroatiaFranceGreeceIsrael
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