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April 8, 2013
Omeros reported results from a phase I trial of OMS824 for Huntington’s disease, schizophrenia and other central nervous system disorders. This randomized, double-blind, single- and multiple-dose escalation study enrolled 64 male patients. Subjects received a single dose of OMS824, daily doses of OMS824 for seven to 10 days. Results showed the pharmacokinetic parameters (Cmax and AUC) increased linearly with the dose and OMS824 had a long half-life that is consistent with once daily. OMS824 was detected in the cerebrospinal fluid at the expected concentration relative to that in the blood. The drug concentration in the cerebrospinal fluid is predicted to achieve near-complete inhibition of the PDE10 target in the brain. OMS824 was well tolerated. The only apparent drug-related adverse event was mild somnolence at the highest dose evaluated. No subject had extrapyramidal symptoms (involuntary muscle movements), which are associated with marketed antipsychotic medications and have been seen with other companies’ PDE10 inhibitors at levels of approximately 50% engagement of the PDE10 enzyme in the brain. Based on these data, Omeros is advancing OMS824 toward a phase II clinical program.
February 8, 2010
NeuroSearch released positive results from a phase III trial of Huntexil for the treatment of Huntingtons disease. This European-based, randomized, double-blind, placebo-controlled study, MermaiHD (Multinational EuRopean Multi-centre ACR16 study In Huntingtons Disease), enrolled 420 subjects. The subjects received either 45 mg Huntexil once daily (QD), 45 mg Huntexil twice daily (BID) or placebo over 26 weeks of treatment. The primary efficacy endpoint was the effect of Huntexil on motor function measured by the modified Motor Score, mMS - a subscale of the Unified Huntingtons Disease Rating Scale. Treatment with Huntexil 45 mg BID demonstrated a statistically significant improvement compared to placebo (p<0.02). Other measures of motor symptoms were also reached with statistical significance in the 45 mg arm versus placebo, including Total Motor Score (p<0.001), Eye Movements (p<0.002) and Dystonia (p <0.001). Treatment with Huntexil 45 mg QD showed some improvements on these motor function domains, but did not reach statistical significance. Huntexil was generally very well tolerated with an adverse event profile similar to placebo.
August 4, 2008
Medivation released positive results from a phase II trial of Dimebon for the treatment of Huntingtons disease. This randomized, double-blinded, placebo-controlled study enrolled 90 subjects in the United States and United Kingdom. The subjects received Dimebon or placebo for a three-month dosing period. The primary endpoint was safety and tolerability. The secondary endpoint was efficacy, as measured by the Mini-Mental State Examination (MMSE) and the United Huntington's Disease Rating Scale (UHDRS). Dimebon was very well tolerated, and the overall incidence of adverse events was lower in the Dimebon group than in the placebo group. In addition, the subjects treated with Dimebon had fewer falls (9%) compared to placebo (16%). Cognitive function was significantly improved over placebo (p=0.03) as measured by the MMSE. An improvement over placebo was also observed on the behavioral component of the UHDRS, but these results did not reach statistical significance. Based on the results, Medivation plans to move forward with the development of Dimebon for Huntington's disease.
April 30, 2007
Addex issued positive results from a phase IIa trial of ADX10059 for the treatment of migraine headaches. This double-blind, placebo-controlled comparison trial enrolled 129 subjects in the United Kingdom and Germany. Subjects received a single dose of ADX10059 or placebo to treat a single moderate or severe (IHS Grade 2 or 3) migraine, in an outpatient setting. The primary endpoint was the proportion of subjects pain-free (IHS 0) two hours after dosing. This endpoint reached statistical significance, with 16.1% of the subjects taking ADX10059 pain-free compared to 4.5% of those taking placebo (p = 0.039). Improvement in migraine pain could be seen from 1 hour after dosing, with the compound being numerically superior to placebo at 1.0 and 1.5 hours post-dose. In addition, there were trends to superiority for ADX10059 over placebo for migraine pain improvement (mild or no pain) at all time points up to two hours post-dosing. Based on the results Addex plans to initiate phase IIb trials shortly.
Amarin reported negative results from two phase III trials of Miraxion for the treatment of Huntington's disease. These randomized, double-blind, placebo-controlled trials enrolled 600 subjects who received Miraxion 2g (1g twice daily) or placebo for six months. The primary endpoint was a change in the Total Motor Score 4 (TMS-4) component of the Unified Huntington's Disease Rating Scale (UHDRS). Secondary endpoints included cognition, behavioral and Total Functional Capacity outcomes. Data showed no statistically significant difference in either study between Miraxion and placebo with regard to the primary and secondary endpoints. Amarin plans to fully evaluate the data in order to determine a future course of action.
UCB announced positive top-line results from a phase III trial of Keppra for adjunctive therapy in the treatment of partial onset seizures in children. This double-blind, randomized, placebo-controlled study enrolled 116 children aged one month to four years. Subjects received Keppra (20-50 mg/kg/day) or placebo for five days. The primary endpoint was efficacy based on a 48 hour video EEG performed at baseline and at the end of the evaluation period. Results revealed 43.1% of Keppra-treated subjects experienced at least a 50% reduction in seizure frequency during the evaluation period compared with 19.6% of placebo-treated subjects. Treatment was generally well tolerated, with the most common adverse events somnolence and irritability. Based on the results UCB plans to file a sNDA with the FDA for Keppra as therapy in this population.
January 30, 2006
Avicena announced results of a phase I/II trial of HD-02, for the treatment of Huntington's disease (HD), in the journal Neurology. Results from the study indicated that serum levels of 8-hydroxy-2'-deoxyguanosine (8OH2'dG), an HD disease biomarker, were significantly reduced in patients receiving the drug. Safety and tolerability results were generally positive, and after drug-free washout, serum creatine levels had returned to baseline. This multi-center, double-blind, placebo-controlled study enrolled 64 patients, who were randomized to receive 8 g HD-02 or placebo daily for 16 weeks, followed by an 8 week drug washout.
Axonyx has announced positive results of a phase I trial of Posiphen for the prevention of Alzheimer's disease (AD) progression. The drug yielded a positive safety profile, with no serious adverse events reported and a good overall tolerability profile. Peak serum drug concentrations exceeded those predicted in animal models to be efficacious in affecting beta-amyloid metabolism. This double-blind, placebo controlled study enrolled 60 healthy volunteers, who received 1 of 5 single doses of Posiphen (10 mg, 20 mg, 40 mg, 80 mg or 160 mg) or placebo.
Javelin Pharmaceuticals issued preliminary results of a phase IIb trial of Dyloject (diclofenac sodium injection) for the treatment of pain. Trial data met their primary efficacy endpoint, producing significant, linear-dose-response pain relief over 6 hours, as measured on the Visual Analog Scale. This response was superior to placebo and non inferior to approved therapy with Ketorolac. The drug experienced superior onset of pain relief after 5 minutes, compared to Ketorolac, on both the Visual Analog and Categorical scales. This randomized study enrolled 353 patients with moderate-to-severe post-surgical pain, who received single administrations of 1 of 5 doses of the drug (up to 75 mg), an approved regimen of IV Ketorolac (30 mg), or placebo.
October 11, 2004
Prestwick Pharmaceuticals reported positive results of a phase III trial of tetrabenazine, a dopamine depleter for the treatment of chorea associated with Huntington’s disease. The drug is still investigational in the US, but already approved in Europe, Australia and Canada. Trial data showed that the drug produced significant improvements in chorea severity, as measured on the UHDRS, a standardized self-diagnostic scale (p<0.0001). The trial also achieved its secondary endpoint, with tetrabenazine producing significant improvements in clinician-rated symptom severity (p=0.007). This randomized, double-blind, placebo-controlled study enrolled a total of 84 Huntington’s disease patients with chorea, who received tetrabenazine (n=54 or placebo (n=30) for 12 weeks. Prestwick announced that based upon this data, they expected to file a NDA for tetrabenazine in the near future.
August 30, 2004
Amarin announced positive results of a gene-variant analysis of data from their phase III study of Miraxion (LAX-101), for the treatment of Huntington’s disease. Original results from the study, announced October 2002, indicated that Miraxion was not significantly more efficacious than placebo in the intent-to-treat-population, despite a positive trend towards efficacy. Post hoc analysis has shown that a subset of patients with a specific variation in the gene thought to be responsible for causing Huntington’s disease demonstrated statistically significant improvement in symptom severity scores. Amarin estimates that this polymorphic variation is present in roughly 65% of Huntington’s patients. The company has announced plans to initiate additional studies of the drug to investigate this gene-dependent efficacy.<
Avanir has reported positive results of their pivotal phase III study of Neurodex, for the treatment of pseudobulbar affect (uncontrollable laughing or crying; PBA), in patients with multiple sclerosis (MS). Study data show that the study met its primary endpoint, with patients receiving the drug achieving significant reduction in symptom severity score on the CNS-LS diagnostic scale, compared with placebo. The study also met all four of its secondary endpoints, significantly improving quality of life and quality of relationships, and significantly reducing PBA episode frequency and pain. The drug was well tolerated, with only dizziness occurring more frequently on Neurodex than placebo. The study enrolled a total of 150 MS patients across 22 sites, all of whom met pre-determined PBA-frequency inclusion criteria.
November 4, 2002
Amarin reported some favorable results from a phase III trial of LAX-101, a drug being investigated for the treatment of Huntington's disease (HD). Preliminary data shows the primary end point, change in the Total Motor Score 4 (TMS-4) over a one-year period, did not reach statistical significance. One analytical method specified in the protocol reached statistical significance in subjects in the per protocol group. Lax-101 was found to be well tolerated, with all incidences of adverse events similar to placebo. The results are from a multi-center, double-blind, randomized, placebo-controlled study of LAX-101, which enrolled 135 subjects with HD at six sites, located in the U.S., Canada, U.K. and Australia.
January 24, 2002
Phase II trial results suggest that Amarin's LAX-101 produces improvement in subjects with advanced Huntington's disease (HD). In the six-month randomized, double-blind and placebo-controlled trial, three HD subjects received LAX-101 and four received placebo. After six months of treatment, all three subjects receiving LAX-101 showed significant improvement on the orofacial component of the Unified Huntington's Disease Rating Scale (UHDRS). A mean 34% improvement was observed for subjects receiving LAX-101, compared to a mean 23% decline for placebo-treated subjects. Improvement was also observed in the total movement score of the UHDRS, with a mean 16% improvement for the LAX-101 group, compared to a mean 38% decline for the subjects receiving placebo.