• A Phase 3, Randomized, Multicenter, Double-Blind, Placebo Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis [NEOD001-301]
  • Status, Active, enrolling

• A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02 (NCT01961921)
  • Status: Complete
• APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP) (NCT01960348)
  • Status: Complete
• A MULTICENTER, INTERNATIONAL, PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF DAILY ORAL DOSING OF TAFAMIDIS MEGLUMINE (PF-06291826) 20 MG OR 80 MG IN COMPARISON TO PLACEBO IN SUBJECTS DIAGNOSED WITH TRANSTHYRETIN CARDIOMYOPATHY (TTR-CM) (NCT01994889)
  • Status: Complete
• APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) (NCT03997383)
  • Status: Active, not recruiting
• A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (NCT04136184)
  • Status: Active, not recruiting
• HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) (NCT03759379)
  • Status: Active, not recruiting
• HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) (NCT04153149)
  • Status: Active, not recruiting

Overview: This research project investigates targeting the PKG/Chip signaling nexus to attenuate transthyretin cardiac amyloidosis.

Significance: We identified a pathway that is uniquely reduced in transthyretin cardiac amyloidosis and we hope by stimulating PKG and Chip that we will improve upon current therapeutic strategies and the outcomes for cardiac amyloidosis patients.

Innovation: This program explores a novel mechanism to increase the degradation of transthyretin once it is taken up by the myocardium thereby reducing the burden on the heart while enhancing the function of the heart.

Impact: Increasing the removal of transthyretin in the heart is not achievable with current pharmacological strategies. Our study addresses this need by providing a new therapeutic approach to better treatment for cardiac amyloidosis patients.