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Prothena to Start Phase IIb Study on AL Amyloidosis Drug

Prothena Corporation plcPRTA announced that it will commence a multi-center, randomized, double-blind, placebo-controlled phase IIb study (PRONTO) on NEOD001 for treatment-experienced patients suffering from AL amyloidosis and persistent cardiac dysfunction.

The company stated that the PRONTO study will be conducted according to feedback received from the European Medicines Agency (EMA) on an ongoing phase III registration study (the VITAL Amyloidosis study) evaluating NEOD001 in newly-diagnosed, treatment-naive AL amyloidosis patients with cardiac dysfunction. Patients who are not eligible for The VITAL Amyloidosis Study will be evaluated in the PRONTO study. The primary endpoint of the study is NT-proBNP best response as measured over 12 months.

The PRONTO study should help expedite the development path for NEOD001, in turn providing an additional opportunity to the company to discuss the regulatory process with the European regulatory authority for a conditional approval of the candidate.

Prothena intends to combine data from the PRONTO study with that from an ongoing phase I/II study on NEOD001. Based on an increased interest from both physicians and patients, Prothena increased enrollment in the expansion cohort of the phase I/II study to 42 patients from 25 patients planned initially. Top-line results from the expansion cohort should be out in the second quarter of 2016, with detailed results expected by mid 2016.

NEOD001 enjoys orphan drug status in the U.S. for the treatment of AL and AA amyloidosis and in the EU for the treatment of AL amyloidosis.

According to the company, nearly 30,000 to 45,000 patients in the U.S. and Europe are estimated to suffer from AL amyloidosis. No treatment has yet been approved for AL amyloidosis. Hence, if developed and commercialized successfully, NEOD001 can grab a large market share.

Prothena carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the health care sector are UCB S.A. UCBJF , Lannett Company, Inc. LCI and Anacor Pharmaceuticals, Inc. ANAC . While UCB and Lannett carry a Zacks Rank #1 (Strong Buy), Anacor holds a Zacks Rank #2 (Buy).

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UCB SA (UCBJF): Free Stock Analysis Report

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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