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
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.
"We are very excited to have begun this study and are pleased with the patient support and initial rate of enrollment we are seeing at our sites," said Craig Langman, M.D., the Isaac A. Abt, M.D., Professor of Kidney Diseases at Feinberg School of Medicine, Northwestern University in Chicago and Principal Investigator of the Phase 3 clinical trial. "We currently have three U.S. sites enrolling patients and a fourth to commence early next month. Our European sites will begin enrolling patients by mid-September".
The Company expects to complete patient enrollment in the next few months and anticipates that all patients will have completed the treatment period by the end of the year. The treatment period for each of the anticipated 30 patients in the clinical trial is a total of 9 weeks and all patients will continue on DR Cysteamine in an extension study following the treatment period.
If the results from this trial are successful, Raptor anticipates filing an NDA for DR Cysteamine in cystinosis in mid-2011 and is planning a concurrent filing with the EMA for registration in the EU.
Raptor anticipates that the Phase 2 study using DR Cysteamine in 96 Huntington's Disease patients will start in the third quarter of 2010. The Phase 2 clinical trial will be conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") of Angers, France. Clinical expenses of the study will be covered by a grant from the French government.
Under the collaboration agreement between the Company and CHU d'Angers, Raptor will supply the DR Cysteamine study drug and will retain commercial rights to the clinical trial results. This Phase 2 trial was contemplated based on encouraging preclinical results reported by Institut Curie, CNRS and Inserm scientists that cysteamine prevents death of neurons and increases levels of a brain growth factor called brain-derived neurotrophic factor ("BDNF") in Huntington's Disease models. Reduced levels of BDNF in the brain have been widely reported to be important in the clinical development of Huntington's Disease.
In addition to intellectual property licensed from University of California, San Diego ("UCSD") related to its DR Cysteamine programs, Raptor holds exclusive worldwide licenses to Huntington's Disease related intellectual property from the Weizmann Institute of Science in Israel and Japan's Niigata University.
Non-Alcoholic Steatohepatitis ("NASH")
Raptor's management views its DR Cysteamine development program in NASH as an exciting opportunity based on both its potential market size and the possibility of creating partnering relationships. Following encouraging results from its Phase 2a clinical trial of DR Cysteamine in juvenile NASH patients, reported earlier this year, Raptor is moving its NASH program forward in anticipation of a future Phase 2b clinical trial.
In the next six months, the Company will be developing a commercial-ready formulation for this potential indication, designed as a delayed-release, coated compressed tablet dosage form of cysteamine bitartrate, which will be appropriate for the expected dose levels for NASH patients, anticipated to be significantly lower than the DR Cysteamine dosage for cystinosis patients. The Company plans to file an IND with the FDA by mid-2011 for a Phase 2b clinical trial to start when funding for this trial becomes available. In this regard, the Company is exploring partnering opportunities and potential grant support for the Phase 2b clinical trial.
The Company completed its first out-licensing agreement of ConviviaTM with Uni Pharma Co., Ltd in Taiwan in June 2010. Raptor has ongoing discussions with other Asian companies to potentially develop ConviviaTM further in various Asian markets.
Source : Raptor Pharmaceutical