NIDDK Provides Additional Data From CyNCh Study Evaluating RP103 in Pediatric NASH at AASLD

NOVATO, Calif., Nov. 16, 2015 (GLOBE NEWSWIRE) — Raptor Pharmaceutical Corp. (NASDAQ:RPTP) today announced researchers have provided additional results from the CyNCh study of RP103 for the treatment of children 8 to 17 years with biopsy-confirmed moderate-to-severe nonalcoholic fatty liver disease, or NAFLD.

The data will be presented today at the American Association for the Study of Liver Diseases (AASLD) Annual Meeting (The Liver Meeting®) at the Moscone Center in San Francisco in a late-breaking poster (#LB-31) entitled “Cysteamine Bitartrate Delayed-Release (DR) for the Treatment of Nonalcoholic Fatty Liver Disease (NAFLD) in Children (CyNCh Trial).” Joel Lavine, M.D., Ph.D. will discuss the data at a CME Symposium entitled “An Urgent Update on Pediatric Nonalcoholic Steatohepatitis (NASH): Management of Pediatric NASH: Current and Emerging Approaches” at 7:40 p.m. PT at the Marriott Marquis, Yerba Buena Salon 9 in San Francisco.

As previously reported by the company on September 14, 2015, an evaluation of the primary endpoint of change in baseline liver histology as measured by a NAS score decrease of ≥2 and no worsening of fibrosis, there was no difference in the response rate between RP103 and placebo (28% versus 22%, respectively; p=0.34).

Based upon additional analyses of the CyNCh study conducted by NIDDK researchers, RP103 demonstrated a significant effect on the NAFLD Activity Score, or NAS, in a post-hoc analysis of patients weighing less than or equal to 65kg at enrollment.

The following data will be highlighted in an abstract and poster at AASLD:

  • No statistically significant difference was seen in the components of the NAS score or fibrosis; however an improvement in the number of subjects with lobular inflammation was observed (36% and 21% in RP103 and placebo groups, respectively; p=0.03), though not statistically significant when adjusted for multiple comparisons.
  • A post-hoc analysis evaluating response by baseline weight category of ≤ 65kg versus >65kg demonstrated a statistically significant difference in the primary endpoint of improvement in liver histology in the lower weight category, favoring RP103 versus placebo (p=0.01). This cohort represented approximately 28% of the population of subjects enrolled and received the lowest assigned dose of 600mg per day. An improvement in liver histology was not observed in subjects >65kg.
  • A statistically significant improvement in liver transaminsases, ALT and AST (p=0.02 and p=0.008, respectively), and GGT* (p=0.02) between RP103 and placebo was seen as measured by the mean change from baseline after 52 weeks of treatment. * Data on GGT was not included in the abstract or poster presentation but submitted separately to Raptor by NIDDK.
  • As was previously reported, there were no differences in adverse events observed in children on RP103 compared to placebo.

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health, sponsored and conducted the CyNCh study through a Cooperative Research and Development Agreement (CRADA) with Raptor.

“While the primary endpoint is unchanged from top-line data released in September, the data support a potentially strong anti-inflammatory effect of RP103 as demonstrated by statistically significant improvement in serum biomarkers of liver injury and an effect on lobular inflammation on histology. While this didn’t translate into an improvement in the overall primary endpoint, there were intriguing data on liver histology in a subset of patients that warrant further discussions with the NIDDK,” said Julie Anne Smith, Raptor’s President and CEO. “The NIDDK is responsible for publication of the manuscript and release of the full data set to us, which we expect sometime next year and which should provide additional clarity. While we await additional data, our decision not to advance this program remains unchanged.”

About CyNCh

CyNCh (Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease [NAFLD] in Children) has been sponsored and conducted by the NIDDK. CyNCh was a multi-center, randomized, placebo-controlled clinical trial of 169 children ages eight to 17 years with biopsy-confirmed moderate-to-severe NAFLD. Patients were randomized to receive placebo or a specified fixed dose of RP103 based on weight: 600 mg/day (≤ 65kg), 750 mg/day (65-80kg) or 900 mg/day (≥ 65kg) for 52 weeks. The primary objective was to evaluate whether 52 weeks of treatment with RP103 would result in improvement in liver disease severity defined as: (1) a decrease in NAFLD Activity Score of two or more points, and; (2) no worsening of fibrosis. Secondary endpoints included: reduction in serum aminotransferase and gamma-glutamyl transpeptidase; reduction in MRI-determined hepatic fat fraction; changes to markers of oxidation and anti-oxidant status; changes in fasting insulin and glucose; changes in weight, height, body mass index (BMI) and waist circumference; changes in the Pediatric Quality of Life score; changes to any symptoms that patient may have experienced; proportion with a change from a histological diagnosis of definite NASH or indeterminate for NASH to not NASH at end of treatment; individual histological characteristics at end of treatment compared to baseline such as steatosis (fatty liver), lobular inflammation, portal chronic inflammation, ballooning, fibrosis score and stage 1a versus 1b fibrosis; and, change in mean NAS. End of study biopsies were conducted in patients after the 52-week treatment period, with all biopsies centrally scored in a blinded fashion. Subjects not completing 52 weeks of treatment or subjects completing 52 weeks of treatment but refusing a biopsy were consider non-responders for the purpose of the primary analyses. Further details can be found at

About RP103 (cysteamine bitartrate)

RP103 is Raptor’s proprietary delayed and extended release oral medication designed to treat the underlying metabolic cause of several rare diseases and disorders including cystinosis and  Huntington’s disease. RP103 is in clinical development for Huntington’s disease and mitochondrial diseases based on a number of proteostatic and antioxidative properties demonstrated in multiple animal models. Clinical data were reported from the ongoing Phase 2 CYST-HD study in Huntington’s disease in 2014.

About PROCYSBI® (cysteamine bitartrate) delayed-release capsules

PROCYSBI is a cystine depleting agent that is approved in the U.S. for the management of nephropathic cystinosis in adults and children ages 2 years and older. It is contraindicated in patients with a hypersensitivity to penicillamine. The most commonly reported side effects are vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash. For additional information on PROCYSBI, including full prescribing information, please visit

About Raptor Pharmaceutical

Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of transformative therapeutics for rare, debilitating and often fatal diseases. With the acquisition of QUINSAIR, the company plans to develop MP-376, the pharmaceutical product known commercially as QUINSAIR, in cystic fibrosis (CF) and at least one of bronchiectasis (BE) or lung infections associated with nontuberculous mycobacteria (NTM). In addition, Raptor is developing RP103, known commercially as PROCYSBI, in multiple therapeutic areas such as nephropathic cystinosis, Huntington’s disease (HD) and mitochondrial diseases including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for nephropathic cystinosis in the U.S. and EU, and orphan drug designation for HD in the U.S. and EU. For additional information, please visit

Forward-Looking Statements

This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are indicated by words or phrases such as “believes,” “expects,” “anticipates,” “estimates,” “plans,” “continuing,” “ongoing,” “projected” and similar words or phrases and relate to future events or our future results of operations or future financial performance, including, but not limited to, statements regarding: ongoing development of Raptor’s product candidates; Raptor’s plans and timing for regulatory submissions; and anticipated clinical and other milestones. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause the company’s actual results to be materially different from these forward-looking statements. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Factors which may contribute to differences in actual results include, among others: Raptor’s ability to market and sell QUINSAIR; continued market acceptance and sales of PROCYSBI in the U.S. and other territories; Raptor’s ability to expand the use of RP103 and MP-376 and to receive regulatory approval for other indications; Raptor’s reliance on single active pharmaceutical ingredient suppliers for PROCYSBI and QUINSAIR and other third parties in connection with drug product development; compliance with healthcare regulations, ongoing regulatory requirements and potential penalties; any serious adverse side effects associated with PROCYSBI, QUINSAIR or any other future products, any product liability claims; third-party payor coverage, reimbursement and pricing; enacted and future healthcare legislation; Raptor’s ability to obtain and maintain orphan drug or other regulatory exclusivity for PROCYSBI, QUINSAIR or any other future products; the integration of European operations with U.S. operations; relationships with key scientific and medical collaborators; intellectual property protection and claims and continued license rights; and Raptor’s ability to fund its operations and make required payments on its debt. Certain of these risks, uncertainties and other factors are described in greater detail in the company’s filings from time to time with the Securities and Exchange Commission (the “SEC”), which Raptor strongly urges you to read and consider, including: Raptor’s annual report for the twelve months ended December 31, 2014 on Form 10-K filed with the SEC on March 2, 2015, Raptor’s quarterly reports on Form 10-Q for the quarterly periods ended March 31, 2015 and June 30, 2015 filed with the SEC on May 7, 2015 and August 6, 2015, respectively, Raptor’s current report on Form 8-K filed with the SEC on September 9, 2015 and other periodic reports filed with SEC, all of which are available free of charge on the SEC’s web site at Subsequent written and oral forward-looking statements attributable to Raptor or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in Raptor’s reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements except as may be required by law.

         Kimberly Lee, D.O.
         Vice President, Corporate Strategy and Communications
         Raptor Pharmaceutical Corp.
         (415) 408-6351
         Westwicke Partners, LLC
         Robert H. Uhl
         Managing Director
         (858) 356-5932
         Cammy Duong
         Canale Communications
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