NOVATO, Calif., Dec. 21, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP) announced today a program update for its lead investigational compound, RP103 for the potential treatment of nephropathic cystinosis. RP103 is Raptor's proprietary, enteric-coated, microbead delayed-release oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and reduce gastrointestinal side effects associated with the currently approved immediate-release cysteamine bitartrate.
Following the successful completion of Raptor's pivotal Phase 3 clinical trial of RP103, which was announced in July 2011, the Company has been conducting a planned voluntary extension study to monitor white blood cell ("WBC") cystine levels and collect long-term safety and quality of life data. Of the 40 patients who entered the safety study after completing the Phase 3 clinical trial, 38 are currently still enrolled. All of these 38 patients have now been taking RP103 in the extension study for at least 6 months, with some patients having been in the extension study for as long as 15 months. Raptor intends to include at least 6 months of safety data for all Phase 3 completers who remain in the extension study, with its New Drug Application ("NDA") and Marketing Authorization Application ("MAA") filings that are expected to be filed in the first calendar quarter of 2012. The Company plans to keep the extension study open to all enrolled patients until RP103 becomes locally commercially available.
Based on the positive results of Raptor's Phase 3 clinical trial and on the findings of its bioequivalence study, which demonstrated similar drug exposure whether administered in whole capsule or sprinkled onto applesauce, US and European regulatory agencies approved the Company's expanded enrollment in the extension study to include patients who did not qualify for the Phase 3 clinical trial. These patients include children 1-6 years old and patients who have undergone a kidney transplant. Ten of an anticipated 18 additional patients have already enrolled in the expanded extension study. The Company also plans to study RP103 in cystinosis patients who have either stopped taking or have not been adequately controlled with the currently-marketed, immediate-release cysteamine bitartrate. While the data for these additional patient groups are not required to be included in Raptor's NDA and MAA filings, the Company expects to include them in subsequent updates to these marketing applications.
"The data from these additional study patients will be helpful for physicians in determining optimal treatments in a broader population of patients with nephropathic cystinosis than we were able to study in our Phase 3 clinical trial," said Patrice P. Rioux, M.D. Ph.D., Raptor's Chief Medical Officer.
Raptor has completed pre-submission meetings with the US Food and Drug Administration ("FDA") and the European Medicines Agency ("EMA") and is on track to submit marketing applications in both markets in the first calendar quarter of 2012. Raptor plans to file for "Priority Review" in the US and "Accelerated Approval" in the EU. If granted, the Company could potentially receive approval for the sale of RP103 for the treatment of nephropathic cystinosis in both the US and the EU in the fourth calendar quarter of 2012. In anticipation of the potential approval in 2012, Raptor is actively building its US and European commercial infrastructure and pre-commercial programs to market RP103.
About Nephropathic Cystinosis
Nephropathic cystinosis is a rare disease resulting from an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of lysosomes. Poor compliance with current treatments for nephropathic cystinosis can cause serious health consequences, including: renal failure and resultant need for a kidney transplant; growth failure; rickets and fractures; and photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes.
About Cysteamine and RP103
RP103 is Raptor's proprietary enteric-coated, delayed-release, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and reduce gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat nephropathic cystinosis. Raptor has been granted orphan product designation for RP103 by both regulatory agencies.
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development RP103 for nephropathic cystinosis and cysteamine for other potential indications including Huntington's Disease, and Non-alcoholic Steatohepatitis ("NASH").
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ("Raptor") seeks to research, produce, and deliver medicines that improve life for patients with severe, rare disorders. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, Non-alcoholic Steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase deficiency ("ALDH2"), and thrombotic disorder.
Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein and related proteins that are designed to target cancer and infectious diseases.
For additional information, please visit www.raptorpharma.com.
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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 relate to future events or our future results of operation or future financial performance, including, but not limited to the following statements: that Raptor will file six months safety data from the extension study with the FDA and EMA; that Raptor will be able to enroll new patients in its extension study; that Raptor will file its NDA and MAA in the first calendar quarter of 2012, if at all; that Raptor will use additional extension study data in its NDA and MAA updates; that the data from these additional extension study patients will be helpful for physicians in determining optimal treatments in a broader population of patients with nephropathic cystinosis; that Raptor will file for "Priority Review" in the US and "Accelerated Approval" in the EU; that Raptor will be granted Priority Review in the US and/or Accelerated Approval in the EU; that Raptor will receive marketing approval in 2012, if at all; that Raptor will successfully commercialize RP103, if approved; that Raptor will be able to build its US and European commercial infrastructure and pre-commercial programs to market RP103; and that Raptor will be able to successfully develop RP103 or any of its other product candidates. 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. Factors which may significantly change or prevent the Company's forward looking statements from fruition include: that Raptor may be unsuccessful in developing any products or acquiring products; that Raptor's technology may not be validated as it progresses further and its methods may not be accepted by the scientific community; that Raptor is unable to retain or attract key employees 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 its technology; that competitors may invent better technology; that Raptor's products may not work as well as hoped or worse, that the Company's products may harm recipients; and that Raptor may not be able to raise sufficient funds for development or working capital. As well, Raptor's products may never develop into useful products and even if they do, they may not be approved for sale to the public. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. 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 on Form 10-K and Amendment No. 1 to its Annual Report on Form 10-K filed with the SEC on November 14, 2011 and December 19, 2011; which are available free of charge on the SEC's web site at http://www.sec.gov. 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.