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REGN3918

Phase 2

Paroxysmal Nocturnal Hemoglobinuria (PNH) | Small molecule | Other |Regeneron Pharmaceuticals, Inc.|Last Updated: Jun 26, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03946748Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)PHASE2 COMPLETED 24May 16, 2019Jun 10, 2021Jun 26, 202312 Hong Kong, Hungary +3
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Study Endpoints
Primary Endpoints
Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis
Week 4 through Week 26

Participants were considered to have had adequate control of intravascular hemolysis if all of their lactose dehydrogenase (LDH) readings from Week 4 through Week 26 inclusive had values less than or equal to ≤ 1.5 × upper limit of normal (ULN). Participants must have greater than or equal to (≥) 50 percent (%) of scheduled LDH measures in those weeks, must not have had more than (\>) 2 consecutive visits without LDH measures, must not have experienced breakthrough hemolysis, and must not have discontinued study treatment early. Participants were considered not to have had adequate control of intravascular hemolysis if they failed any of these criteria.

Percentage of Participants Who Achieved Transfusion Avoidance
Up to 26 Weeks

Transfusion avoidance was defined as not having received red blood cell (RBC) transfusion during the first 26 weeks. A transfusion was counted only if it was per-protocol, that is, it followed the predefined transfusion algorithm: RBC transfusion due to a post-baseline hemoglobin level \< 9 grams per deciliter (g/dL) (with anemia symptoms) or a post-baseline hemoglobin level \< 7 g/dL (without anemia symptoms).

Secondary Endpoints
Percentage of Participants Who Had Breakthrough Hemolysis (BTH)
Baseline up to 26 Weeks
Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis
Week 4 through Week 26
Time to First Lactate Dehydrogenase (LDH) ≤1.5 x ULN
Up to Week 26
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
REGN3918EXPERIMENTALCohort A (Dose Confirmation) If a decision is made to expand Cohort A, patients will be assigned to Cohort A. Cohort B (Dose Expansion) If a decision is made to progress to Cohort B, patients will be assigned to Cohort B.
Interventions
NameTypeDescription
REGN3918DRUGSingle intravenous (IV) dose, then a subcutaneous (SC) dose once weekly (QW).
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites12

Key Inclusion Criteria: * Diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) confirmed by high-sensitivity flow cytometry * PNH granulocytes \> 10% at screening visit * Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms (eg, fatigue, hemoglobinuria, abdominal ...

Countries:Hong KongHungaryMalaysiaSouth KoreaUnited Kingdom
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