Positive results from Neuraltus NP002 Phase 1/2 study for levodopa-induced dyskinesias in Parkinson's disease

Dec 4 2010

Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet medical needs, primarily for the treatment of neurodegenerative diseases, announced today top-line results from the Company's Phase 1/2 clinical study of NP002 for the treatment of dyskinesias (muscle movement disorders) resulting from levodopa therapy for patients with Parkinson's disease.  Of the 500,000-1.5 million Parkinson's patients in the United States, about 50% experience incidence of levodopa-induced dyskinesias (LIDs), for which there are no approved treatments.  NP002 is a small molecule, orally available nicotinic receptor agonist that has been shown in preclinical studies to reduce LIDs without affecting Parkinsonian symptoms.

A double blinded, placebo controlled, Phase 1/2 study was performed to assess the safety and tolerability of NP002 in a total of 65 individuals with Parkinson's disease and documented LIDs.  The primary objective of the study was the establishment of safety and tolerability of NP002, with patients being assessed over a 14-week treatment and monitoring period.  The secondary objective was to investigate the effects of NP002 on a set of Parkinson's disease and dyskinesia assessment scales, including The Unified Parkinson's Disease Rating Scale (UPDRS), the Unified Dyskinesia Rating Scale (UDRS), the Lang-Fahn Activities of Daily Living Scale (LF-ADL), and the clinician and patient global impressions of change scales (CGIC and PGIC).

NP002 administered concurrently with levodopa treatment was found to be generally safe and well-tolerated in Parkinson's patients with LIDs.  In addition, NP002 was not associated with any impulsivity or withdrawal issues when compared to placebo.  Although the trial was not powered as an efficacy study, clinically relevant trends and, in two cases, statistical superiority of NP002 over placebo were observed in a variety of physician- and patient-rated PD efficacy outcome measures relating to dyskinesias.

Abraham Lieberman, MD, the study's principal investigator and Director of the Muhammad Ali Parkinson's Center, commented, "NP002 represents an innovative approach to a common side effect in treating Parkinson's disease and has opened up new ideas on how to approach dyskinesias.  The early study results of NP002 in patients with Parkinson's show promising trends in reducing dyskinesias and in improving gait.  I am encouraged by these results and believe they provide a key piece in understanding and addressing the mechanism of dyskinesias in many of our patients with Parkinson's disease."

J. William Langston, MD, Founder, CEO and Scientific Director of the Parkinson's Institute, stated, "It's impressive that the NP002 study data were encouraging across several scales used to measure dyskinesias.  In particular, the study showed a significant trend in improvement in the objective UDRS scale and statistically significant results on the PGIC scale, which measures how patients feel overall, suggesting an improvement in quality of life.  We are very excited to see this work progress to Phase 2 trials based on these encouraging data."

Carrolee Barlow, MD, PhD, CSO and CMO of BrainCells, Inc., and scientific advisor to Neuraltus on the NP002 program, stated, "Patients with Parkinson's disease currently have no therapeutic options to address the dyskinesias caused by levodopa treatment.  The clinical study demonstrated a clinically important improvement in dyskinesia symptoms that could be detected by both clinicians and patients.  The data are compelling and provide a breakthrough for this condition."

The Phase 1/2 data are the second clinical program results reported by Neuraltus this week, following announcement of clinical results for NP001, which showed safety, tolerability and statistically significant effects on a biomarker of disease progression in ALS.  Neuraltus is committed to accelerating the development of new drug molecules based on novel biology and representing first-in-class approaches to target indications in neurodegenerative disease where there is substantial unmet medical need.