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APL2

Phase 2

FSGS | Small molecule | Other |Apellis Pharmaceuticals, Inc.|Last Updated: Oct 9, 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-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment270
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07213960A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental GlomerulosclerosisPHASE2 NOT YET_RECRUITING 270Dec 1, 2025Dec 1, 2029Oct 9, 20252 United States
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Study Endpoints
Primary Endpoints
Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
Baseline to Week 12

Change from Baseline in Log-Transformed uPCR will be based on triplicate first morning urine (FMU)

Phase 3: Change from baseline in log-transformed urine protein to creatinine ratio (uPCR)
Baseline to Week 52

Change from baseline in log-transformed uPCR will be based on triplicate first morning urine (FMU)

Secondary Endpoints
Phase 2: Evaluate the efficacy of APL2 in terms of change from baseline in log-transformed urine albumin to creatinine ratio (uACR)
Baseline to Week 12
Phase 3: Change from Baseline in log-transformed urine protein to creatinine ratio (uPCR)
Baseline to Week 104
Phase 3: Proportion of participants achieving Complete Remission
Week 104
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Phase 2 - APL2EXPERIMENTALSub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly
Phase 3 - APL2EXPERIMENTALAdults: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly Adolescents: ≥50 Kg: Sub-cutaneous infusions of APL2 (1080 mg / 20mL) twice weekly 35 to \<50kg: First sub-cutaneous infusion of 648mg (12 mL) followed by 810mg (15 mL) every infusion thereafter (i.e. twice weekly) 30 to \<35kg: First \& Second sub-cutaneous infusion of 540mg (10mL) followed by 648mg (12 mL) every infusion thereafter (twice weekly)
Phase 3 - PlaceboPLACEBO_COMPARATORSubcutaneous infusions of a sterile solution, twice-weekly, and equivalent in volume to the active arm based on participant's age and weight
Interventions
NameTypeDescription
APL2DRUGComplement (C3) Inhibitor
PlaceboOTHERSterile solution of equal volume to active arm
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Age * Phase 2: adults aged ≥18 years * Phase 3: adults aged ≥18 years; if and where approved, adolescents (aged 12--17 years) at the time of signing the informed consent and assent form * Weight ≥30 kg and ≤100 kg at screening * FSGS diagnosis * Phase 2: primary, genet...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07213960primaryCompletionDate: changed
LOWMay 24, 2026NCT07213960studyFirstPostDate: changed