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Neuropathy

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May 7, 2012

Baxter International issued results from a phase III trial of Gammagard Liquid 10% for the treatment of multifocal motor neuropathy (MMN). This randomized, double-blind, placebo-controlled, cross-over, multi-center study enrolled 44 patients for 12 weeks. Subjects received monthly doses of 1.2g/kg of body weight of Gammagard Liquid 10% or placebo. The trial met its primary endpoint, demonstrating a 3.75% increase in mean grip strength of the more affected hand during treatment, as compared to a 31.38% decrease in mean grip strength with placebo administration. If subjects experienced intolerable interference with daily activities, they were allowed to switch from placebo to Gammagard Liquid 10% (69% of subjects required an accelerated switch before 12 weeks). The drug was well tolerated, with most commonly reported adverse events—headaches and muscular weakness—reported in only 5% of subjects. If approved, Gammagard Liquid 10% will be the first immunoglobulin treatment available for patients with MMN in the U.S.

April 18, 2011

Pfizer issued results from a phase II/III trial of tafamidis for the treatment of Transthyretin Familial Amyloid Polyneuropathy. This open-label, 12-month extension study (Fx-006) was conducted to evaluate long-term safety and efficacy in 86 subjects who completed an 18-month pivotal study. All subjects received tafamidis 20 mg orally once-daily for 12 months; the subjects randomized to placebo in the previous study crossed over to active drug. Data showed that earlier treatment with tafamidis resulted in better outcomes. Subjects treated with tafamidis for 30 months had less neurologic deterioration than those who began tafamidis 18 months later, showing a 55.9% preservation of function as measured by the Neuropathy Impairment Score-Lower Limb (NIS-LL), or a mean change from baseline of 3.0 for those treated for 30 months versus 6.8 for those initiating treatment 18 months later (p≡0.04). In addition, subjects treated with tafamidis over 30 months showed preservation in large nerve fiber function (66% preservation or 1.6 versus 4.7 for those treated 18 months later, p≡0.007) and small nerve fiber function (45.5% or 1.2 versus 2.2 for those treated 18 months later.

February 14, 2011

Epicept reported results from a phase IIb trial evaluating NP-1, a topical analgesic cream for chemotherapy-induced peripheral neuropathy (CPN) This multi-center, double-blind, randomized, placebo-controlled study enrolled 460 subjects who received treatment for six weeks. Data from the intent to treat (ITT) population showed that the change in average daily neuropathy intensity scores in the NP-1 group achieved a statistically significant reduction in CPN intensity versus placebo (p<0.001). In a pre-specified subgroup of the ITT population, subjects who had previously received taxane chemotherapy, data also showed a statistically significant reduction in average daily neuropathy intensity scores (p≡0.034). This subgroup constituted more than 50% of the ITT population. Secondary efficacy endpoints confirmed the superiority of NP-1 vs. placebo.

July 27, 2009

FoldRx released positive results from a phase II/III trial of tafamidis for the treatment of TTR amyloid polyneuropathy (ATTR-PN), also known as Familial Amyloid Polyneuropathy (FAP). This international, randomized, double-blind, placebo-controlled trial enrolled 128 subjects with FAP and a confirmed V30M TTR mutation. The subjects received tafamidis or placebo once a day for 18 months. The co-primary endpoints were response to treatment at 18 months via the Neuropathy Impairment Score - Lower Limb (NIS-LL) and quality of life, as measured by the Norfolk QOL-DN. Statistical significance was achieved for both primary endpoints. No disease progression was observed in 60% of subjects in the tafamidis arm compared to 38% in the placebo arm after 18 months treatment (p≡0.041). There was a significant deterioration in QOL in the placebo arm compared to the tafamidis arm after 18 months (p≡0.045). In the intent to treat population, where liver transplant patients were treated as non-responders, there was a clinically meaningful difference between the treatment groups as measured by the NIS-LL (p≡0.068) and Norfolk (p≡0.12). In addition, a number of secondary endpoints, including objective measures of disease severity and nerve function were reached with statistical significance over placebo.

May 12, 2008

Schwarz Pharma released positive results from a phase III trial of lacosamide for the treatment of diabetic neuropathic pain. This placebo-controlled, withdrawal study was a sub-trial of an open- label, follow-on to a phase III, double-blind trial. Subjects had been followed for a mean of twenty months before entering the sub-trial. Prior to any lacosamide treatment, subjects had an average daily pain score of 6.5. After more than a year of open-label treatment, the mean pain score was 2.5, the baseline score of the withdrawal sub-trial. A total of 106 subjects treated with lacosamide (100-400 mg/day) participated in the withdrawal study. In a blinded fashion, lacosamide was withdrawn (less than or equal to twenty-eight days) and reintroduced. The primary endpoint was the change in average daily pain score from baseline to the last seven days of each period, using the 11-point Likert pain scale. The difference in average daily pain scores was statistically significant in favor of lacosamide over placebo (p= 0.007). Average daily pain scores increased during placebo treatment and improved to values similar to sub-trial baseline (2.5 on the pain scale) when lacosamide was reintroduced. A key secondary endpoint, worsening of pain when lacosamide was withdrawn, was also reached. The intra-individual change in average daily pain score from open-label lacosamide treatment at baseline to the end of the placebo period was statistically significant (p<0.001). More subjects receiving placebo (39%) versus lacosamide (29%) experienced sustained worsening of pain during the withdrawal periods. Treatment was safe and well tolerated. An MAA and NDA are currently under review by the EMEA and FDA, respectively.

March 10, 2008

NeurogesX issued negative preliminary results from a phase III trial of NGX-4010 for the treatment of HIV-distal sensory polyneuropathy (HIV-DSP). This multi-center, randomized, double-blind, controlled study, dubbed C119, enrolled 494 subjects in Australia, Canada, the UK and the US. The subjects received a single 30- or 60-minute treatment with NGX-4010 or low concentration control patch, applied directly to the site of pain. After 30 to 60 minutes, the patch was removed and the response levels were evaluated during a subsequent 12-week study period. The pre-specified primary endpoint, comparing all subjects treated with NGX-4010 to all subjects treated with the control patch, did not meet statistical significance. The 30-minute NGX-4010 treatment arm achieved a 26.1% reduction in pain from baseline compared to 19.1% for the control group (p=0.1). The 60-minute treatment arm achieved a 32.8% reduction in pain from baseline compared to 30.1% for the control group (p=0.5). Secondary endpoints also did not achieve statistical significance. NeurogesX plans to fully analyze the data in order to determine the best path towards regulatory approval.

July 23, 2007

Newron announced positive results from a phase II trial of ralfinamide for the treatment of neuropathic pain. This double-blind, randomized, placebo controlled trial enrolled 272 subjects internationally. Subjects received ralfinamide at doses ranging from 80 to 320 mg/day or placebo for eight weeks. The primary endpoint was efficacy measured via a patient rated visual analogue scale (VAS) of the severity of pain compared to baseline. Treatment was generally safe and well tolerated at the highest dose tested. Ralfinamide reached statistical significance in reduction in pain severity when compared to placebo on the VAS scale, with a score of -18.1 compared to a placebo score of -12.5 (p=0.075). In addition, ralfinamide showed significant improvement in the mean ratings of the patient rated Daily Pain Diary: (p=0.003), significant improvement in the quality of sleep (p=0.002) and significant improvement in daily activities (p=0.033). Several phase II trials are currently underway and an IND was recently approved for the commencement of US based trials.

February 12, 2007

Avigen posted positive interim results from a phase IIa trial of AV411 for the treatment of neuropathic pain. This randomized, double blind, placebo-controlled trial enrolled 18 healthy subjects in Australia who received either 60 mg/day of AV411 or placebo in a 3:1 ratio, in single-dose and two-week repeat dose phases. Treatment was well tolerated, with all adverse events mid to moderate in nature and pharmacokinetic data support a twice daily dosing profile. This trial was expected to be complete later in 2007 with a United States IND to follow shortly thereafter.

August 14, 2006

Evotec AG announced positive results from a phase I trial of EVT 101, for the treatment of Alzheimer's disease and neuropathic pain. This trial enrolled 90 healthy, young and elderly subjects who received EVT101 in single or multiple ascending oral doses. Tolerability profiles were positive, with no significant adverse events reported. Pharmacokinetic data revealed a positive profile, with good drug absorption and a half-life of 11 hours, a time frame is consistent with a once or twice daily oral dosing regimen.

Vectura Group issued positive results from a phase II study of VR004 for the treatment of "off episodes" related to Parkinson's disease. This randomized, double-blind, placebo-controlled, four parallel group, ascending dose study enrolled 24 subjects. Subjects were taken off their conventional treatment to induce an "off" episode, than given one or two fine particle doses of VR004 or placebo at 200 mcg, 300 mcg, 500 mcg, 750 mcg, 800 mcg or 1,200 mcg. Safety data was positive, with no serious adverse events reported. Pharmacokinetic data demonstrated that maximum VR004 plasma concentrations occurred 1-3 minutes after dose inhalation. Efficacy data revealed that the median onset of response was 10 minutes and the median duration of response after inhalation was 25 minutes. Based on these results, Vectura planned to further advance VR1004 in future clinical trials.

May 22, 2006

Corgentech issued positive results of a phase III trial of 3268, their fast acting local anesthetic for the prevention of pain associated with venipuncture and intravenous (IV) line placement procedures, at the Society of Academic Emergency Medicine Annual Meeting in San Francisco. Trial data demonstrated significant efficacy in the trial's primary endpoint, reducing mean score on the FACES pain scale prior to needle insertion (p=0.007). A significant reduction was also noted in mean parental observational pain assessment score during venipuncture, vs. placebo (p<0.001). No clinically significant safety concerns were noted. This randomized, double-blind, placebo-controlled study enrolled 574 pediatric patients at 6 sites in the US; subjects received the drug or placebo 1 to 3 minutes before venipuncture procedures.

Targacept and AstraZeneca have issued positive results of a phase II trial of TC-1734 (AZD3480) for the treatment of age-associated memory impairment (AAMI). Results from the study indicated significant improvements for the higher trial dose in three efficacy measures in both the per-protocol population (Atten p=0.010, Mem p=0.030, SGI p=0.008) and the intent to treat population (Atten p=0.014, Mem p=0.029, SGI p=0.015). The lower trial dose achieved significant efficacy in the power of attention endpoint (p=0.023, p=0.025 for the two populations, respectively). This multi-center, randomized, double-blind, placebo-controlled, dose-finding study enrolled 193 patients AAMI without evidence of additional disease at 16 US sites, who received one of two doses of the drug (25 mg or 50 mg) or placebo once daily for 16 weeks.

April 17, 2006

NeuroVax announced positive results of a clinical trial of NeuroVax for the treatment of multiple sclerosis (MS) at the AAN Annual Meeting. This open-label study enrolled 25 subjects, 17 of whom had not previously received NeuroVax. The untreated patient subset showed significantly lower levels of FOXP3+ regulatory T-cells (as measured by FOXP3+ mRNA (p=0.03) and protein expression (p=0.02)). Trial data indicated that NeuroVax significantly increased FOXP3 expression in 14 of 17 subjects, including levels of both FOXP3+ mRNA (p=0.01) and FOXP3 protein (p=0.02). Based on these results, the company confirmed plans to initiate a phase II trial of NeuroVax for the treatment of MS across sites in the US and Eastern Europe later in 2006; this study was designed to investigate the effect of the drug on MRI measures and relapse rates.

Sangamo announced positive results of a phase I trial of SB-509, for the treatment of diabetic neuropathy, at the 58th Annual Meeting of the American Academy of Neurology (AAN) in San Diego. Primary safety data were positive, with no serious adverse events reported and mild injection site reactions the only noted all-severity adverse events. Dose limiting toxicities were not observed. Single administrations of the drug produced improvements in severity of pain, numbness and neurological symptoms in roughly 50% of subjects. Additional preliminary efficacy was noted in neurologic exam scores and electrophysiological testing. This single blind, single-dose dose-escalation study enrolled 12 subjects with mild to moderate disease, who received intramuscular injections of the drug. Based on these results, the company announced plans to initiate a phase II trial of SB-509 in the second half of 2006.

Somaxon reported positive results of their first phase III trial of Silenor (doxepin HCl), for the treatment of chronic insomnia. The drug was shown to significantly reduce 8-hour Wake After Sleep Onset (WASO), with a mean improvement of 26 minutes for the low dose and 31 minutes for the high dose groups, vs. placebo (p<0.0001). Improvement in secondary measures was also noted: Total Sleep Time was 415 min. and 421 min. for the two Silenor doses (respectively), vs. 374 min. for placebo (p<0.0001); Latency to Persistent Sleep during the initial treatment period was 27 minutes for both doses, vs. 45 minutes for placebo (p=0.011, p=0.0018); these improvements were not maintained at subsequent timepoints (due in part to improvements in the placebo group). This randomized, double-blind, placebo-controlled, parallel-group, multi-center study enrolled 229 patients, who received one of two doses of the drug (3 mg or 6 mg) or placebo nightly for 35 days.

February 13, 2006

NeurogesX announced positive results of a phase III trial, dubbed C107, of Transacin (NGX-4010), for the treatment of HIV-associated sensory neuropathy. Trial data met their primary efficacy endpoint, producing a significant decrease in daily patient reported pain score (visual analog scale) over 12 weeks, vs. control (-22.8% for all subjects, vs. -10.7%; p=0.0025). Further, 34% of subjects receiving the drug experienced a decrease in pain of at least 30%, compared to 18% for placebo (p=0.0093). The greatest reductions in pain score were seen in the short- and long-duration treatment groups (-27.7%, p=0.0007; -24.7%, p=0.0046, respectively); the reduction for the middle-duration treatment was not significant (-15.9%, p=0.257). No drug-related systemic adverse events were noted. This double-blind, randomized, controlled study enrolled 307 subjects, who received a single application of the drug (30, 60 or 90 minutes) with a 12 week evaluation follow-up and a 40 week open-label extension.

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.

January 9, 2006

Depomed announced positive results of a phase II trial of gabapentin GR, for the treatment of post-herpetic neuralgia. Results indicated that a twice-daily regimen of the drug significantly reduced average daily pain scores on the Likert diagnostic scale from baseline, vs. placebo (-2.24 vs. -1.29; p=0.014). Secondary measure of reduction in Sleep Interference score from baseline was also improved (-2.28 vs. -1.29; p=0.006). A once-daily regimen significantly improved Sleep Interference scores (-1.94 vs. -1.16; p=0.048), and a trend was noted in improving daily pain score (-1.93 vs. -1.29; p=0.089). No serious adverse events were reported. This randomized, double-blind, placebo-controlled study enrolled 158 patients, who received one of two regimens of the drug (1800 mg once- or twice-daily) or placebo.

September 5, 2005

Altea Therapeutics reported positive results of a phase II trial of their hydromorphone transdermal patch, for the treatment of acute pain. Pharmacokinetic data indicated steady-state drug levels within 4 hours of treatment initiation; these levels were maintained for 3 days, and there was no evidence of drug accumulation. Minimum effective dose levels were also established. This open-label study enrolled 14 opioid-naïve patients, who received a single patch daily for up to 3 days. Based on these results, the company announced plans to initiate additional phase II studies of the drug in the near future.

Renovis has issued negative results of a phase II trial of REN-1654, for the treatment of sciatica, a form of neuropathic pain. Data from the study failed to reach statistical significance in the primary endpoint, a change in average daily spontaneous pain ratings, though a positive trend was noted. Significance was reached in a secondary endpoint, reduction in maximum leg pain, at interim analyses after 7 and 14 days, but not at the end of treatment. This double-blinded, placebo-controlled, multi-center study enrolled 74 sciatica patients, who received single daily doses of 100 mg REN-1654 or placebo for 21 days. Based on these results, Renovis announced that they would not pursue further development of the oral formulation of REN-1654.

June 13, 2005

CeNeS Pharmaceuticals has announced positive results of a phase IIa trial of CNS 5161, their NMDA receptor antagonist under investigation for the treatment of neuropathic pain. Trial data met primary safety and tolerability endpoints, establishing a maximum tolerated dose (MTD) of 500 mcg. Incidence of hypertension occurred in the highest cohort (750 mcg), and enrollment in this cohort was not completed. No psychotomimetic effects were observed. Treatment at the MTD yielded trends towards improvement in pain levels a 2, 6 and 12 hours after the start of infusion, especially in patients with diabetic neuropathy. This multi-centre, double blind, cross-over, dose escalating proof- of-concept study enrolled 48 patients with etiologically varied neuropathic pain across sites in Europe. Subjects received one of four escalating regimens of the drug (125 mcg, 250 mcg, 500 mcg and 750 mcg) via intravenous infusion over 6 hours.

PharmaFrontiers has reported interim results of a pair of phase I/II trials of Tovaxin, their trivalent formulation of attenuated myelin-peptide reactive T cells (MRTCs) for the treatment of multiple sclerosis (MS). Preliminary results of the companys dose-escalation study indicated that administration of the drug produced reductions in MRTC levels, along with improvements in disability scores on the Kurtzke Expanded Disability Status Scale (EDSS) and disability neurological assessments for psychological scores Multiple Sclerosis 29 point Impact Scale (MSIS-29). Disease exacerbation was reported by 1 subject during the 6 month evaluable period of the study. Results of the company's extension study indicated a maintained a mean reduction in all categories of MRTCs at 3 and 6 months. This included a greater than 60% reduction in proteolipid protein T-cells. The dose- escalation study had treated 6 MS patients to date, who received a low- (6-9 million MTRCs) or mid-dose (30-45 million MRTCs) during a 6 month evaluation period. The extension study had enrolled 9 patients, who received one of two mid-dose-level doses of the drug during a 6 month evaluation. Based on these results, the company announced plans to initiate phase IIb/III trials by the end of 2005 or early 2006.

November 1, 2004

Avanir Pharmaceuticals reported positive results of its first phase III trial of their drug Neurodex (dextromethorphan/quinidine), for the treatment of pseudobulbar affect in patients with amyotrophic lateral sclerosis. Trial data indicated that the drug met its primary efficacy endpoint of emotional lability, producing significant improvement in CNS-LS diagnostic scale symptom severity score, compared with either of its component drugs (3.3 points greater vs. dextromethorphan, p=0.001; 3.7 points greater vs. quinidine, p<0.001). The drug also met all secondary endpoints, including reduction in affect episode incidence and improving quality of life and quality of relationship score significantly vs. the component drugs (p<0.01). Discontinuation due to adverse events with Neurodex occurred at four times the rate of dextromethorphan and three times the rate of quinidine, though adverse events were generally mild to moderate. The multicenter, randomized, double-blind, controlled, parallel, three-arm study enrolled a total of 140 ALS patients demonstrating pseudobulbar affect across 17 American sites.

Newron Pharmaceuticals has reported preliminary results of a phase II trial of ralfinamide, their non-opioid sodium-channel blocker for the treatment of neuropathic pain. Study results indicated that the drug was successful in meeting its efficacy primary endpoint, a significant improvement in score on the Visual Analogue Scale, a standard diagnostic tool, versus baseline. Secondary efficacy measures, including provoked allodynia, pin-prick test, clinical global impression and patient global impression, supported the primary finding. The primary safety endpoint was also met, with no consistent pattern of clinically relevant serious adverse events at any dosing regimen. This observer-blinded tolerability and efficacy pilot study enrolled a total of 18 patients with neuropathic pain, who received ascending oral doses of ralfinamide for 4 weeks; the trial was conducted at a single center in Austria. Newron announced plans to use these results to support future trials.

October 13, 2003

Indevus Pharmaceuticals reported positive results from a phase II trial investigating IP 751 (CT-3), an analgesic cannabinoid for the treatment of neuropathic pain. Results showed that IP 751 significantly reduced the degree of neuropathic pain without causing psychoactive adverse events. Data demonstrated that the degree of pain experienced by subjects decreased significantly during periods of treatment with IP 751. In addition, no significant differences were observed between treatments of measurements of cognitive function and prototypic subjective experiences. The two-week, randomized, crossover study enrolled 21 subjects with chronic neuropathic pain from spinal nerve injuries. Subjects received IP 751, (20 mg-40mg given 2x a day) or placebo. The study was conducted at the Hannover Medical School in Germany. Results were reported in the Journal of the American Medical Association.

June 16, 2003

AVANIR Pharmaceuticals reported positive results from a phase II trial investigating Neurodex, a neuroprotective for the treatment of neuropathic pain. Results showed the drug was well tolerated and subjects reported a significantly decreased pain from baseline. Data revealed that by day eight, 91% of the subjects reported pain relief compared to baseline, and 97% reported pain relief by Day 15. The Pain Relief Rating Scale, a 6-point Likert, administered on Days 8, 15, 22 and 29 showed a statistically significant difference at all time points. In addition, a Peripheral Neuropathy Quality of Life Scale showed statistically significant improvement at Day 29 compared to Day one. The study enrolled 36 subjects with diabetic neuropathy who experience pain on a daily basis in the lower extremities and was designed to evaluate the safety and tolerability of escalating doses. The trial was conducted at six sites in the U.S. Commonly reported adverse events included nausea, constipation, diarrhea, dry mouth, fatigue, dizziness, insomnia, headache, upper respiratory tract infection, and somnolence.

February 10, 2003

EpiCept reported positive results from a phase II trial investigating EpiCept NP-1, an amitriptyline plus ketamine containing cream for the treatment of neuropathic pain. Data indicated strong pain relief effects in all treatment groups with study results being comparable to those reported with alternative drugs such as gabapentin. Treatment effect was shown in subjects regardless of the cause of the neuropathy. The multi-center, randomized, placebo controlled trial enrolled 80 subjects with chronic neuropathic pain in a double blind fashion. Subjects were randomized into four groups: amitriptyline alone, ketamine alone, amitriptyline plus ketamine, or placebo. EpiCept NP-1 is expected to be a C III controlled substance.