Raptor Pharmaceutical Reports Positive Interim Phase 2a Clinical Data in Non-Alcoholic Steatohepatitis (NASH)

Monday, October 12, 2009 General News
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NOVATO, California, October 12

- Primary Endpoint Achieved in Completed Treatment Phase

Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTPD),announced positive findings from the completed treatment phase of itsopen-label Phase 2a clinical trial of delayed-release cysteamine bitartrate("DR Cysteamine") in adolescent patients with non-alcoholic steatohepatitis("NASH"), a progressive form of liver disease believed to affect 2% to 5% ofthe U.S. population. At the completion of the initial six-month treatmentphase, the study achieved the primary endpoint: mean blood levels of alanineaminotransferase ("ALT"), a common biomarker for NASH, were reduced by over50%. Additionally, over half of the study participants had achievednormalized ALT levels by the end of the treatment phase.

(Logo: http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )

There are no currently approved drug therapies for NASH, and patients arelimited to lifestyle changes such as diet, exercise and weight reduction tomanage the disease. DR Cysteamine represents an important potential treatmentoption for patients with NASH. Although NASH is most common ininsulin-resistant obese adults with diabetes and abnormal serum lipidprofiles, its prevalence is increasing among juveniles as obesity rates risewithin this patient population. Although most patients are asymptomatic andfeel healthy, NASH causes decreased liver function and can lead to cirrhosis,liver failure and end-stage liver disease.

Under a collaboration agreement between Raptor and the University ofCalifornia, San Diego ("UC San Diego"), the open-label Phase 2a trial of aprototype formulation of DR Cysteamine is being conducted at UC San Diego'sGeneral Clinical Research Center and entails six months of treatment followedby a six-month post-treatment monitoring period. Eligible patients withbaseline ALT and aspartate aminotransferase ("AST") measurements at leasttwice that of normal levels were enrolled to receive twice-daily, escalatingoral doses of up to 1,000 mg of DR Cysteamine. The trial currently hasenrolled eleven NASH patients between 11-18 years old. No major adverseevents were reported during the six-month treatment phase. Trial subjectscontinue to be monitored during the six-month post-treatment period currentlyunderway. Full results are being submitted for peer review by Raptor and UCSan Diego, and are expected to be presented in 2010.

Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San Diegoand principal investigator for the NASH study, stated, "We were encouraged bythe results of this study. The degree of ALT and AST reductions areindicative of likely improvements in severity of fatty liver damage. Thetrial results are consistent with ALT and AST reductions normally seen inpatients that achieve at least 10% weight loss, even though studyparticipants did not show a significant change in body mass index. DRCysteamine appears to be a promising candidate for NASH and we look forwardto further analyzing these patients during the post-treatment phase."

Raptor's chief medical officer, Patrice Rioux, M.D., Ph.D., said, "Theseinterim results have established proof-of-concept and support furtherclinical development of DR Cysteamine in NASH. This is an area of significantunmet need, especially with growing numbers of obese children diagnosed withthe disorder. While the clinical hurdle is usually high for studies inchildren and adolescents, we are satisfied with the long-term safetydemonstrated in this age group by the currently-marketed immediate-releasecysteamine bitartrate formulation. This safety track record, coupled with ourinterim Phase 2a efficacy data, gives us a great sense of encouragement as weadvance DR Cysteamine through the clinic."

Under a license with UC San Diego, Raptor is developing DR Cysteamine forcystinosis, NASH and other potential therapeutic indications. Cysteamine isknown to be a scavenger of reactive oxygen species and potent antioxidant,most likely through its ability to increase intracellular glutathione levels.Cysteamine has also demonstrated potential efficacy in preclinical andclinical studies in Huntington's Disease, Batten Disease and otherindications.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq: RPTPD) ("Raptor") is dedicated tospeeding the delivery of new treatment options to patients by working toimprove existing therapeutics through the application of highly specializeddrug targeting platforms and formulation expertise. Raptor focuses onunderserved patient populations where it can have the greatest potentialimpact. Raptor currently has product candidates in clinical developmentdesigned to potentially treat nephropathic cystinosis, non-alcoholicsteatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase("ALDH2") deficiency, and a non-opioid solution designed to potentially treatchronic pain and thrombotic disorder.

Raptor's preclinical programs are based upon bioengineered novel drugcandidates and drug-targeting platforms derived from the humanreceptor-associated protein ("RAP") and related proteins that are designed totarget cancer, neurodegenerative disorders and infectious diseases.

For additional information, please visit www.raptorpharma.com.


This document contains forward-looking statements as that term is definedin the Private Securities Litigation Reform Act of 1995. These statementsrelate to future events or our future results of operation or futurefinancial performance, including, but not limited to the followingstatements: Raptor's and UC San Diego's ability to complete the clinicaltrial in NASH patients, DR Cysteamine's ability to treat NASH, ALT and AST asa biomarker to determine the efficacy of a treatment for NASH, Raptor'sability to further develop DR Cysteamine in NASH and other indications. Thesestatements are only predictions and involve known and unknown risks,uncertainties and other factors, which may cause the Company's actual resultsto be materially different from these forward-looking statements. Factorswhich may significantly change or prevent the Company's forward lookingstatements from fruition include that Raptor may be unsuccessful indeveloping any products or acquiring products; that Raptor's technology maynot be validated as it progresses further and its methods may not be acceptedby the scientific community; that Raptor is unable to retain or attract keyemployees whose knowledge is essential to the development of its products;that unforeseen scientific difficulties develop with the Company's process;that Raptor's patents are not sufficient to protect essential aspects of itstechnology; that competitors may invent better technology; that Raptor'sproducts may not work as well as hoped or worse, that the Company's productsmay harm recipients; and that Raptor may not be able to raise sufficientfunds for development or working capital. As well, Raptor's products maynever develop into useful products and even if they do, they may not beapproved for sale to the public. Raptor cautions readers not to place unduereliance on any such forward-looking statements, which speak only as of thedate they were made. Certain of these risks, uncertainties, and other factorsare described in greater detail in the Company's filings from time to timewith the Securities and Exchange Commission (the "SEC"), which Raptorstrongly urges you to read and consider, including the joint proxystatement/prospectus on Form S-4 filed with the SEC on August 19, 2009;Raptor's annual report on Form 10-K filed with the SEC on March 27, 2009;Raptor's quarterly report on Form 10-Q filed with the SEC on August 11, 2009;Raptor's wholly-owned subsidiary's, Raptor Pharmaceuticals Corp. ("RPC")Registration Statement on Form S-1, as amended, that was declared effectiveon August 7, 2008; RPC's annual report on Form 10-K filed with the SEC onOctober 30, 2008, as amended by that Form 10-K/A filed with the SEC onDecember 23, 2008; and RPC's quarterly report on Form 10-Q filed with the SECon July 15, 2009, all of which are available free of charge on the SEC's website at http://www.sec.gov. Subsequent written and oral forward-lookingstatements attributable to Raptor or to persons acting on its behalf areexpressly qualified in their entirety by the cautionary statements set forthin Raptor's reports filed with the SEC. Raptor expressly disclaims any intentor obligation to update any forward-looking statements.

For more information, please contact: Karl Cahill, Investor Relations +1-858-531-6100 kcahill@raptorpharma.com The Ruth Group Sara Ephraim Pellegrino (investors) +1-646-536-7002 spellegrino@theruthgroup.com Janine McCargo (media) +1-646-536-7033 jmccargo@theruthgroup.com

Karl Cahill, Investor Relations, +1-858-531-6100, kcahill@raptorpharma.com; or Sara Ephraim Pellegrino (investors), +1-646-536-7002, spellegrino@theruthgroup.com or Janine McCargo (media), +1-646-536-7033, jmccargo@theruthgroup.com, both of The Ruth Group / Logo: http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO

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