More progress in the pipeline:
ACR16 study underway in the U.S.; cysteamine trial to begin in France
Two drugs are moving through the research pipeline. Neurosearch has started the U.S. Phase IIB clinical trial of its dopamine stabilizer, ACR16. Recruitment is still ongoing for the ACR16 study so there is still time to sign up. Contact information can be found at the Huntington Study Group site: HSG/HART
Raptor Pharmaceuticals has announced a collaboration with Centre Hospitalier Universitaire d'Angers (CHU d'Angers) of France to conduct a Phase II trial of cysteamine, a drug which boosts BDNF (brain derived neurotrophic factor). Those in France who are interested in the trial, can find more information here: CHU d'Angers
Here is the Neurosearch press release:
NeuroSearch announces the dosing of the first patients in the US HART study, a part of the ongoing ACR16 pivotal programme in Huntington's disease
NeuroSearch is pleased to announce that the first patients have now been treated in the US HART (Huntington's disease ACR16 Randomised Trial) study with ACR16, a dopaminergic stabiliser and the company's unique and novel compound for the treatment of Huntington's disease. The HART study is a randomised, placebo-controlled, double-blinded study expected to include a total of 220 patients with Huntington's disease. The patients will be randomised to three months treatment with one of three doses of ACR16 (10 mg BID, 22.5 mg BID and 45 mg BID) or placebo. The study will be conducted in a number of centres in the United States and Canada.
The ongoing European MermaiHD (Multinational EuRopean Multi-centre ACR16 study In Huntington's Disease) Phase III study with ACR16 in Huntington's disease, in which the first patients were dosed in April 2008, is progressing satisfactorily. Almost all the centres participating in the study are now enrolling patients, and also the first patients that have finalised the six months blinded treatment period are now entering into the six months open-label extension to the study.
The primary efficacy endpoint for both HART and MermaiHD is the effect of ACR16 on Huntington patients' motor function (such as gait/balance, hand functionality and parkinsonism) measured by the modified Motor Score, mMS - a subscale of the Unified Huntington's Disease Rating Scale (UHDRS). Secondary endpoints include the overall clinical impression of the patients, their cognitive function and the severity of neuropsychiatric symptoms such as depression and anxiety.
Flemming Pedersen, CEO of NeuroSearch commented, "The initiation of the HART study represents a very important milestone for us in our efforts to get ACR16 on the market and make this promising drug available for the patients suffering from Huntington's disease. We now have a full pivotal programme running and with the European MermaiHD study progressing according to plan, we remain confident that we will have the first key data in the second half of 2009 and be able to apply for market registration as soon as possible thereafter."
Previously, ACR16 has been evaluated in four clinical Phase I/II studies with patients suffering from Huntington's disease, Parkinson's disease and psychoses, demonstrating a good safety and tolerability profile. In a Phase II study in Huntington's disease, 28 days' treatment with ACR16 resulted in a statistically significant improvement in the patients' voluntary movements including parkinsonism and gait function.
Here is the Raptor press release:
Raptor Pharmaceuticals to Collaborate with Centre Hospitalier Universitaire d'Angers for Phase II Clinical Trial in Huntington's Disease
Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP), today announced that the Company has entered into an agreement with the Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") of France to evaluate Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in a Phase II clinical trial in patients with Huntington's Disease ("Huntington's"). CHU d'Angers has received a grant from the Programme Hospitalier de Recherche Clinique - National, a program under the French Ministry of Health, to fund the two-year, multi-center Phase II clinical trial in Huntington's patients. Under the terms of the agreement, Raptor will provide clinical supplies of DR Cysteamine for the trial.
In May 2008, the Office of Orphan Product Development at the U.S. Food and Drug Administration ("FDA") granted orphan drug designation to Raptor for cysteamine-based drugs, including DR Cysteamine, for the treatment of Huntington's.
Ted Daley, President of Raptor's Clinical Division, stated, "We look forward to collaborating with CHU d'Angers to evaluate DR Cysteamine in patients with Huntington's Disease. Our formation work with DR Cysteamine in conjunction with the CHU d'Angers studies in Huntington's fits our strategic plan to explore multiple potential indications for cysteamine and DR Cysteamine. Cysteamine has demonstrated neuroprotective effects in preclinical studies, and the planned Phase II study led by CHU d'Angers could confirm existing data regarding cysteamine's safety and potential efficacy in Huntington's."
Professor Dominique Bonneau, M.D., Ph.D. of CHU d'Angers, commented, "We are encouraged by the existing studies that indicate cysteamine's potential as a neuroprotectant for treating Huntington's, and we believe that Raptor's formulation could offer a better tolerated drug for this patient population. We are confident that this trial will enhance our understanding of cysteamine's effects in Huntington's patients and we hope it may lead to a new treatment for this serious, debilitating disease."
The primary endpoint of the Phase II trial will be evaluated using the Unified Hungtington's Disease Rating Scale ("UHDRS"). The secondary endpoint will measure brain-derived neurotrophic factor ("BDNF") levels, known to be deficient in Huntington's patients. A preclinical study published by Drs. Sandrine Humbert and Frédéric Saudou from the Curie Institute demonstrated a potential mechanism for cysteamine in an in vivo preclinical Huntington's model that showed increased brain and blood levels of BDNF. The Curie Institute will participate in the clinical trial by determining the BDNF levels in the patients.
Marsha L. Miller, Ph.D., November 10, 2008