NeuroSearch (NEUR) reported positive top-line results from the MermaiHD study, the European phase-III study with Huntexil (pridopidine) in Huntington's disease.
The MermaiHD study met the primary endpoint to show an effect on voluntary motor function. In addition, data from the 437 Huntington patients, who participated in the study (= ITT population) show that six months' (26 weeks) treatment with Huntexil results in significant improvements in a broader range of voluntary and involuntary motor symptoms associated with the disease. The study was conducted in 32 centres across Europe, and showed a very high compliance with 92 per cent of the patients completing the study and 82 per cent in full compliance with the study.
Treatment with Huntexil 45 mg BID (twice daily) demonstrated statistically and clinically significant improvements compared to placebo in the ITT population on the following measures of motor symptoms in Huntington's disease:
Treatment with Huntexil 45 mg QD (once daily) showed some improvements on these motor function domains, however did not reach statistical significance.
The improvements in motor function observed with Huntexil 45 mg twice daily in the MermaiHD study appear very robust, as they remain consistent across assessments and analyses; show increasing separation from placebo over time; and are consistent also across the two pre-stratified study groups of patients on use/non-use of antipsychotic medication; approximately 40 per cent of patients were on antipsychotics. The importance of studying both patient groups has been emphasized by experts and regulators to demonstrate that Huntexil improves the symptoms of Huntington's disease per se, and that it is also safe in patients treated with antipsychotics. The use of antipsychotics showed no influence on the positive treatment effects of Huntexil.
In the study, Huntexil was generally very well tolerated with an adverse event profile similar to placebo, and the results show no indication that treatment with Huntexil would be associated with worsening of disease signs and symptoms.
Following the results, NeuroSearch is now initiating interactions with scientific advisors and regulatory agencies (EMEA and FDA) to discuss the MermaiHD study outcome and the plans for submissions for market authorisation for Huntexil as a novel treatment for Huntington's disease.
Principal investigator, Prof. Justo García de Yébenes, Hospital Ramon y Cajal, Madrid, Spain, commented, “Huntington’s disease is a progressive disorder with motor, cognitive, and behavioral symptoms. Huntexil is the first medication to have demonstrated an overall improvement of the motor impairment in Huntington patients with no worsening of other signs or symptoms and without compromising patient safety. Overall, the MermaiHD study results show that Huntexil has a favorable clinical risk/benefit profile, and I believe it could be useful to many of my patients."
Chairman of the European Huntington's Disease Network, EHDN, professor G B Landwehrmeyer, commented, "The EHDN is very proud to have been part of making the MermaiHD study a success both in terms of quality and time. With more than 400 patients participating, the trial is one of the largest so far conducted in Huntington's disease in Europe, and recruitment was completed within one year. I would like to thank all participants and their caregivers, all investigators and their staff as well as the employees at the EHDN for their dedicated contribution to this achievement."
Flemming Pedersen, CEO of NeuroSearch, commented, “The phase-III results from the MermaiHD study are very encouraging, demonstrating that Huntexil can provide significant benefits to Huntington patients on symptoms not reached by any current treatment, and thiswithout any "trade-offs" in terms of safety or worsening of any other disease symptoms. We remain determined to bring Huntexil forward as a new medication to patients with Huntington's disease and we will work closely with physicians and regulatory authorities to make this happen as soon as possible.”
NeuroSearch is also evaluating Huntexil in a second randomised and placebo-controlled study, the HART study, conducted in the USA and Canada and in approximately 220 patients with Huntington's disease. Patient recruitment in the HART study is still ongoing, and study results are expected in the second half of 2010.
Also, the MermaiHD study is followed by an open-label treatment period, which is still ongoing. Patients, who completed the six months' randomised study treatment, have been offered to continue open-label treatment with up to 45 mg Huntexil twice daily for six months. Close to 90 per cent of the patients have chosen to continue treatment in the open-label phase, and the last patient is expected to complete the full 12 months treatment period in May 2010. Results from the open-label treatment period are also expected to be available in the second half of 2010.
NeuroSearch will give financial guidance for 2010 in connection with release of the 2009 Annual Report on 10 March 2010.