Recent Updates
Recently added Catalysts

ALN-CC5

Phase 1

Paroxysmal Nocturnal Hemoglobinuria (PNH) | Small molecule | Other |Alnylam Pharmaceuticals, Inc.|Last Updated: Mar 30, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment62
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02352493A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHPHASE1 COMPLETED 62Jan 1, 2015Aug 1, 2017Mar 30, 20204 Spain, United Kingdom
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Number of Participants With Adverse Events
Part A: through day 658; Part B: through day 532; Part C: through day 280

Adverse events were reported for single-ascending doses (SAD) or multiple ascending doses (MAD) of ALN-CC5 when administered to healthy adult subjects and of multiple doses (MD) in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Secondary Endpoints
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Alternative Pathway (CAP)
Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in Complement Classical Pathway (CCP)
Part A: through day 70; Part B: through day 140; Part C: through day 140
Pharmacodynamic (PD) Effect of ALN-CC5: Percentage Reduction From Baseline in C5 Protein Levels
Part A: through day 70; Part B: through day 140; Part C: through day 140
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-CC5ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-CC5DRUGSingle or multiple doses of ALN-CC5 by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)DRUGcalculated volume to match active comparator
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersYes
Study Sites4

Inclusion Criteria: * Adequate complete blood counts, liver and renal function * 12-lead electrocardiogram (ECG) within normal limits * Female subjects of child bearing potential agreeing to use a protocol specified method of contraception * Male subjects agreeing to use protocol specified methods ...

Countries:SpainUnited Kingdom
Unlock Eligibility Criteria