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Raptor Crushed by Liver Disease Data, Hits 52-Week Low

Novato, CA-based biotech company, Raptor Pharmaceuticals Corp. 's RPTP shares touched a 52-week low of $6.82 on Sep 14 with the company announcing data from a mid-stage study. Shares were down 37.5%.

What Led to the Share Price Slump?

Raptor announced disappointing top-line data from a phase IIb study (CyNCh - Cy steamine Bitartrate Delayed-Release for the Treatment of N onalcoholic Fatty Liver Disease in Ch ildren) on its pipeline candidate, RP103.

RP103, which was being evaluated for the improvement of nonalcoholic steatohepatitis (NASH) in children, failed to meet the primary endpoint of the study.

The study was sponsored and conducted by the National Institute of Diabetes and Digestive and Kidney Diseases (a part of the National Institutes of Health) under a Cooperative Research and Development Agreement with Raptor.

Raptor said that it does not intend to proceed with the development of the candidate for the NASH indication. Data from the study will be presented at an upcoming medical meeting this November.

However, Raptor plans to continue with the development of RP103 for other orphan disease indications like Huntington's disease (phase II/III) and mitochondrial diseases including Leigh syndrome (phase II). Meanwhile, Raptor is preparing for the launch of Quinsair in Europe and Canada slated for the first half of 2016. In addition, the company plans to begin studies on Quinsair either for the treatment for non-tuberculous mycobacteria or bronchiectasis.

We remind investors that Raptor had signed an agreement last month with Tripex Pharmaceuticals to acquire Quinsair.

Our Take

Considering that there are no approved treatments for NASH, we find the phase IIb study results discouraging. The successful development of RP103 for NASH would have allowed the company to enter a market which holds significant commercial potential.

NASH, a common, serious chronic liver disease, affects about 10-20% of people in the developed world and could lead to more serious conditions like progressive fibrosis, cirrhosis and liver failure. Several companies including Gilead Sciences Inc. GILD and Intercept Pharmaceuticals, Inc. ICPT are working on bringing NASH treatments to market.

Raptor is a Zacks Rank #3 (Hold) stock. Gilead and Regeneron Pharmaceuticals, Inc. REGN are better-ranked stocks in the health care sector, each carrying a Zacks Rank #1 (Strong Buy).

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REGENERON PHARM (REGN): Free Stock Analysis Report

GILEAD SCIENCES (GILD): Free Stock Analysis Report

RAPTOR PHARMACT (RPTP): Free Stock Analysis Report

INTERCEPT PHARM (ICPT): 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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