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Prothena Corp: Prothena To Initiate Pronto, A Global Trial Of Neod001 In Patients With

The following excerpt is from the company's SEC filing.

AL Amyloidosis with Primary Endpoint of Cardiac Response

Accelerates Development Timeline and Potential Path to Patients

DUBLIN, Ireland - October 15, 2015 - Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapies, today announced it will initiate PRONTO, a global trial of NEOD001 in previously-treated patients with AL amyloidosis and persistent cardiac dysfunction. The primary endpoint of the trial is a cardiac functional biomarker, defined by NT-proBNP best response over 12 months.

“In addition to the VITAL Amyloidosis Study, which remains on-track to provide data for full registration, the PRONTO trial enables us to explore an accelerated path forward to address the high unmet need for therapies that improve organ function in patients with AL amyloidosis,” said Dale Schenk, PhD, President and Chief Executive Officer of Prothena. “NT-proBNP response is a widely validated functional biomarker that predicts survival in patients with AL amyloidosis. Demonstrating significant improvements in this key cardiac functional biomarker, along with supporting trends in key secondary endpoints, will expedite our development timeline and provide an additional opportunity to engage with European regulators on a dialogue around conditional approval for NEOD001.”

The PRONTO trial was designed to align with feedback from the European Medicines Agency (EMA) related to The VITAL Amyloidosis Study, a global Phase 3 registrational trial. When combined with data from the ongoing NEOD001 Phase 1/2 trial, the PRONTO trial has the potential to expedite patient access.

In addition to the ongoing Phase 1/2 clinical trial, The VITAL Amyloidosis Study is evaluating NEOD001 in newly-diagnosed, treatment-naïve patients with AL amyloidosis with cardiac dysfunction, and will assess a composite of all-cause mortality or cardiac hospitalizations in addition to biomarker, functional and quality of life endpoints. The PRONTO trial will evaluate NEOD001 in patients with AL amyloidosis and persistent cardiac dysfunction who have been previously-treated and are not eligible for The VITAL Amyloidosis Study.

“We are encouraged with the continued consensus of leading physicians regarding NT-proBNP as a surrogate endpoint for clinical trials as exemplified by the recently announced Amyloid Research Consortium (ARC) white paper,” said Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development of Prothena. “We believe an ongoing dialogue, such as the Amyloidosis Patient Forum organized by ARC on November 16 with U.S. Food and Drug Administration, may accelerate further alignment on important topics such as the use of functional biomarkers as surrogate endpoints to expedite new therapeutic options for patients.”

PRONTO Registration-Directed Trial Design

The global, multi-center, randomized, double-blind, placebo-controlled Phase 2b trial further exemplifies Prothena's commitment to provide disease-modifying therapeutic alternatives for patients...


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