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rVA576

Phase 3

Paroxysmal Nocturnal Hemoglobinuria (PNH) | Small molecule | Other |Akari Therapeutics Plc|Last Updated: Apr 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
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment9
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03588026Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576PHASE3 COMPLETED 9Jun 7, 2018Sep 3, 2020Apr 9, 20253 Kazakhstan, Lithuania +1
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Study Endpoints
Primary Endpoints
HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions
9 months

Haemoglobin stabilisation rate defined as haemoglobin greater than the set point for each patient during the pre-study randomisation period and the avoidance of PRBC transfusions during the treatment period.

Secondary Endpoints
Number of units of packed red blood cells (PRBC) transfused
Day 1 to Day 180
Percentage of patients who achieve transfusion avoidance
Day 1 to Day 180
Change in (QOl) Quality of Life score
Day 1 to Day 180
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm 1 - 9 months of treatment (rVA576 plus SOC)EXPERIMENTAL6 months (SOC plus rVA576), Followed by a further 3 months of (SOC plus rVA576).
Arm 2 - 6 months on SOCEXPERIMENTAL6 months on SOC only. Followed by 3 months (SOC plus rVA576).
Interventions
NameTypeDescription
rVA576DRUG6 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC.
Standard of care (SOC)OTHER6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Willing to give informed consent to treatment with rVA576 2. Diagnosed with paroxysmal nocturnal haemoglobinuria (PNH) 3. Have not received any complement inhibitor within the 4 months prior to screening 4. ≥ 18 years of age at the time of screening 5. Weight ≥50kg 6. Complet...

Countries:KazakhstanLithuaniaSri Lanka
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