Enrollment in Pivotal Phase 3 Clinical Trial in Cystinosis on Schedule for  Completion in Q4 2010 with Results Expected Q1 2011

Phase 2 Clinical Trial in Huntington's Disease to Commence Enrollment in September

NOVATO, Calif., Aug. 24, 2010 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), 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").

Financial Updates and Guidance

As of August 24, 2010, the Company has approximately $17.2 million in cash and cash equivalents taking into account approximately $14 million of proceeds, net of placement agent fees and expenses, the Company received in a private placement financing which closed on August 12, 2010. The Company anticipates that its cash and cash equivalents will support planned operations and its planned clinical development programs, as described below, into December 2011. 


Raptor's cystinosis program remains the highest priority for the Company.  "Over the next 18 months, the majority of the Company's resources will be devoted to completing what we believe will be the final development phase of this program," said Christopher M. Starr, Ph.D., CEO of Raptor.  "Anticipating the successful completion of our ongoing Phase 3 clinical trial in 2010 and NDA submission in 2011, we are building the commercial infrastructure in our anticipation of a commercial launch in 2012."  

In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine in cystinosis.  DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.

On June 28, 2010, Raptor enrolled the first patient in its pivotal Phase 3 clinical trial in cystinosis.  This trial is designed as a randomized, crossover, outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine compared to immediate-release cysteamine bitartrate in cystinosis patients.  The study is being conducted at nine centers: four in the U.S., including Emory University, Children's Memorial Hospital at Northwestern University, Stanford University, and Texas Children's Hospital at Baylor University; and five in the EU including in Hopital Necker-Enfants Malades and Hopital Robert Debré in Paris, Hospices Civils de Lyon in Lyon, France, Centre Hospitalier Universitaire in Montpellier, France and Radboud University Medical Center in Nijmegen, The Netherlands.