Prothena Update. Opening PRONTO TRIAL for previously treated AL with Cardiac Involvement. Contact Prothena for locations (for sure at Mayo, BU, Tufts, UPenn, Stanford, Karmanos, Sloan Kettering)
- Recently, announced plans to initiate PRONTO, a global trial of NEOD001 in patients with AL Amyloidosis with primary endpoint of cardiac functional biomarker NT-proBNP best response
- Completed enrollment in expansion cohort of Phase 1/2 trial of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction; data expected in second quarter of 2016
Recent Highlights and Program Updates:
- In October, announced plans to initiate PRONTO, a randomized, double-blind global registration-directed Phase 2b trial of NEOD001 in previously-treated patients with AL amyloidosis and persistent cardiac dysfunction. The primary endpoint is the cardiac functional biomarker NT-proBNP as measured by best response over a 12-month period. Secondary endpoints include evaluations of Short Form 36 (SF-36, quality of life measure), six-minute walk test (6MWT), and renal function as assessed by proteinuria. When combined with data from the ongoing NEOD001 Phase 1/2 trial, the PRONTO trial is intended to provide a foundation for discussions with European regulatory agencies around the potential for conditional approval.
- In October, announced the completion of enrollment for the expansion cohort of the Phase 1/2 study of NEOD001 to treat patients with AL amyloidosis and persistent organ dysfunction. Based on strong interest from patients and physicians, enrollment in this trial was increased to 42 from the originally planned 25 and topline results are expected in the second quarter of 2016.
- On November 2, presented preclinical results from a series of novel, conformation-specific protein immunotherapy antibodies that selectively bind to amyloidogenic (diseased) forms of the transthyretin (ATTR) protein at the First European Congress on Hereditary TTR Amyloidosis in Paris, France. These data suggest that Prothena’s antibodies can prevent deposition, and enhance clearance of, ATTR in patients with wild type and hereditary TTR-mediated amyloidosis.
Upcoming Research and Development Milestones
Prothena’s clinical development pipeline includes three lead protein immunotherapy programs.
NEOD001 is a monoclonal antibody for the potential treatment of AL amyloidosis:
- Expect to provide an update on enrollment timeline for The VITAL Amyloidosis Study in February 2016
- Expect topline results from the expansion cohort of the Phase 1/2 study in the second quarter of 2016
PRX002 is a monoclonal antibody for the potential treatment of Parkinson's disease and other related synucleinopathies, and is the primary focus of Prothena's worldwide collaboration with Roche:
- Expect topline data from Phase 1 multiple ascending dose study in patients with Parkinson's disease in the first half of 2016
PRX003 is a monoclonal antibody for the potential treatment of psoriasis and other inflammatory diseases:
- Expect topline data from Phase 1 single ascending dose study in healthy volunteers in the first half of 2016
- Expect to initiate Phase 1 multiple ascending dose, proof-of-biology study in patients with psoriasis in 2016