Cystinosis News and Research

Latest Cystinosis News and Research

New 'magic bullet' can eradicate side effects of drug used to treat cystinosis

A 'magic bullet' designed by pharmaceutical scientists at an English university could eradicate the side effects of a drug used to treat a rare genetic disease. Researchers at the University of Sunderland have chemically modified a drug used to treat cystinosis a disease which occurs when the body's mechanism to remove excess cystine (an amino acid) breaks down, leading to kidney problems and eventually affecting other organs to remove unpleasant side effects such as nausea, vomiting, severe halitosis, body odour and a noxious taste.

11 Nov 2010

Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

Raptor Pharmaceutical Corp., today announced that the first patient has been dosed in its Phase 2 clinical trial of DR Cysteamine for the potential treatment of Huntington's Disease. The Phase 2 clinical trial is being conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers"), which has commenced enrolling patients at eight clinical sites throughout France.

8 Nov 2010

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical Corp.,provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

24 Aug 2010

Raptor Pharmaceutical to raise $15 million through PIPE financing

Raptor Pharmaceutical Corp., announced it has entered into a securities purchase agreement in connection with a private placement, or PIPE financing. Upon the closing of the PIPE financing, Raptor will receive gross proceeds of approximately $15 million resulting from the sale of 4,897,614 units at a purchase price of $3.075 per unit.

10 Aug 2010

Raptor partners with Uni Pharma to commercialize Convivia in Taiwan

Raptor Pharmaceutical Corp., announced that it has entered into an exclusive agreement with Uni Pharma Co., Ltd. to commercialize Convivia™, Raptor's proprietary oral formulation of 4-methylpyrazole, in Taiwan.

30 Jun 2010

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

Raptor Pharmaceutical Corp. announced that it has enrolled the first patient in a pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

28 Jun 2010

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis

Raptor Pharmaceutical Corp., announced that the European Medicines Agency's ("EMA") Committee for Orphan Medicinal Products ("COMP") has issued a positive opinion recommending orphan medicinal product designation for DR Cysteamine for the potential treatment of cystinosis.

15 Jun 2010

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Raptor Pharmaceutical Corp., announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

2 Jun 2010

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Sigma-Tau Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the filing of the company's new drug application (NDA) for its cysteamine hydrochloride ophthalmic solution, an investigational therapy for the treatment of corneal cystine crystals in patients with cystinosis. The FDA also granted the NDA Priority Review status, a designation given to drugs that may provide major advances or a treatment where no adequate therapy exists.

18 May 2010

Raptor Pharmaceutical presents positive results from Phase 2a trial of DR cysteamine bitartrate for NASH

Raptor Pharmaceutical Corp., announced positive Phase 2a clinical trial results from its pilot study of delayed-release cysteamine bitartrate in 11 adolescent patients with non-alcoholic steatohepatitis, a progressive form of liver disease believed to affect 5% to 11% of the U.S. population. The results were presented at the Digestive Disease Week 2010 conference in New Orleans, LA on May 2, 2010.

3 May 2010

Raptor Pharmaceutical engages The Trout Group for strategic advisory and investor relations services

Raptor Pharmaceutical Corp., today announced the engagement of The Trout Group LLC, a leading New York-based investor relations and strategic advisory firm. The Trout Group will work with Raptor to facilitate the Company's exposure to the international capital markets and coordinate their communications with investors worldwide.

7 Apr 2010

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Raptor Pharmaceutical Corp., today announced the presentation of cystinosis data at the Annual Lysosomal Disease Network WORLD Symposium 2010, being held February 10-12 in Miami, Florida. Biomarker data from a Phase IIb pilot study of Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") will be the subject of a poster titled, "Correlation of Plasma Cysteamine and WBC Cystine Levels at Steady State in Patients Treated with Cysteamine Bitartrate." The results will also be published in an upcoming issue of the journal Molecular Genetics and Metabolism.

10 Feb 2010

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

Raptor Pharmaceutical Corp. ("Raptor" or the "Company"), today announced the publication of results from a Phase 2a clinical trial of a prototype formulation of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

11 Jan 2010

CRF awards $1.75 million in 11 new grants to researchers to find a cure for cystinosis

The Cystinosis Research Foundation of Irvine, Calif., announced it awarded $1.75 million in 11 new grants to researchers in the United States, Canada, Belgium and Italy who are working to find a cure for cystinosis, a rare, deadly metabolic and genetic disease that afflicts about 500 children and young adults in the U.S. and 2,000 worldwide.

7 Jan 2010

Raptor Pharmaceutical completes $7.5 million registered direct offering

Raptor Pharmaceutical Corp. ("Raptor" or the "Company"), today announced that on December 22, 2009, it completed its registered direct offering with institutional investors, raising an aggregate amount of approximately $7.5 million of gross proceeds (before placement agent fees and offering expenses) through the sale of 3,747,558 units.

24 Dec 2009

Raptor Pharmaceutical enters into definitive agreements with institutional investors to purchase 3.7M units

Raptor Pharmaceutical Corp., today announced that it has entered into definitive agreements with institutional investors to purchase 3,747,558 units for gross proceeds of approximately $7.5 million, before placement agent fees and offering expenses.

19 Dec 2009

Raptor Pharmaceutical announces Phase 2b results of DR Cysteamine trial

Raptor Pharmaceutical Corp., today announced results from a Phase 2b clinical trial of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis"). The trial, conducted at the University of California, San Diego ("UCSD") General Clinical Research Center, evaluated safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in nine cystinosis patients.

23 Nov 2009

Raptor Pharmaceutical announces results of NGX426 clinical trial

Raptor Pharmaceutical Corp. today announced the presentation of clinical trial data on NGX426, the Company's orally administered, non-opioid, AMPA/kainate receptor antagonist, at the 12th International Conference on the Mechanisms and Treatment of Neuropathic Pain, held on November 20-21 in San Francisco.

23 Nov 2009

Raptor Pharmaceutical to present oral AMPA-kainate antagonist data at the neuropathic pain conference

Raptor Pharmaceutical Corp., today announced that data from a clinical trial of NGX426, the Company's orally administered non-opioid, AMPA/kainate antagonist, will be presented at the 12th International Conference on the Mechanisms and Treatment of Neuropathic Pain, to be held November 20-21, 2009 in San Francisco.

5 Nov 2009

Results of Phase 2a clinical trial of DR Cysteamine announced by Raptor Pharmaceutical

Raptor Pharmaceutical Corp., announced positive findings from the completed treatment phase of its open-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% of the U.S. population.

12 Oct 2009

Cystinosis News and Research

Latest Cystinosis News and Research

New 'magic bullet' can eradicate side effects of drug used to treat cystinosis

Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical to raise $15 million through PIPE financing

Raptor partners with Uni Pharma to commercialize Convivia in Taiwan

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

EMA COMP grants orphan medicinal product designation for Raptor's DR Cysteamine for cystinosis

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

FDA accepts for filing Sigma-Tau Pharmaceuticals' cysteamine hydrochloride ophthalmic solution NDA

Raptor Pharmaceutical presents positive results from Phase 2a trial of DR cysteamine bitartrate for NASH

Raptor Pharmaceutical engages The Trout Group for strategic advisory and investor relations services

Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Results from Phase 2a clinical trial of Raptor Pharmaceutical's cysteamine bitartrate published

CRF awards $1.75 million in 11 new grants to researchers to find a cure for cystinosis

Raptor Pharmaceutical completes $7.5 million registered direct offering

Raptor Pharmaceutical enters into definitive agreements with institutional investors to purchase 3.7M units

Raptor Pharmaceutical announces Phase 2b results of DR Cysteamine trial

Raptor Pharmaceutical announces results of NGX426 clinical trial

Raptor Pharmaceutical to present oral AMPA-kainate antagonist data at the neuropathic pain conference

Results of Phase 2a clinical trial of DR Cysteamine announced by Raptor Pharmaceutical

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Cystinosis News and Research

Latest Cystinosis News and Research

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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