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P.3.D.030 Evenamide, a Voltage-Gated Sodium Channel Blocker in the Treatment of Schizophrenia

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P.3.D.030 Evenamide, a Voltage-Gated Sodium Channel Blocker in the Treatment of Schizophrenia P.3 .d .0 3 0 Evenamide, a voltage-gated sodium channel blocker in the treatment of schizophrenia: results from the placebo-controlled Study 002 3 1 4 2 1 Ravi Anand , Emma Forrest , Richard Hartman , Stephen M. Graham , Laura Faravelli 1 Newron Pharmaceuticals SpA, Bresso (MI), Italy, 2 Newron Pharmaceuticals US, Inc., Morristown, NJ, USA, 3 APC, AG, St. Moritz, Switzerland, 4 NeurWrite LLC, Morristown, NJ, USA Introduction Currently available antipsychotics that block dopaminergic, serotoninergic, and histaminergic transmission have failed to provide effective control of symptoms of psychosis (CATIE, CUTLASS, and EUFEST). Evenamide (NW-3509A), a potent anti-epileptic is a totally selective inhibitor of voltage- gated sodium channels and attenuates the stimulated release of glutamate, as shown in in-vivo micro-dialysis. It is effective in animal models of psychosis, mood disorders, aggression, negative symptoms, and cognitive dysfunction, both as monotherapy and as add-on treatment. A study in healthy male volunteers evaluating single doses of 1 to 30 mg of evenamide indicated that its Tmax was 1.5-2.0 hr, half-life (t1/2) was 1.1-4.7 hrs; its Cmax and AUC(0-t) increased proportionately with increasing dose. Cmax values of >20 ng/ml, exceeding projected effective exposures (based on PBPK modelling of results of in-vitro/ in-vivo studies), were noted at doses of 20 mg and higher. These results were followed by the conduct of Study 002, a placebo-controlled, randomized study, with a dose range of 15-25 mg BID evenamide as an add-on to patients with schizophrenia who were on stable doses of risperidone or aripiprazole. Study Objectives Results Primary: to explore safety and tolerability of a dose range of evenamide Disposition: Premature discontinuations: placebo, 1 (2.6%); evenamide (15-20-25 mg BID) in patients with schizophrenia who showed signs of 8 (16.0%) [2 (4.0%) due to AEs: seizure and atrial fibrillation] worsening, despite treatment with a therapeutic dose of risperidone or Safety: Most frequent AEs aripiprazole for at least 4 weeks at a stable dose. Secondary: to assess efficacy using the PANSS positive subscale, Preferred Term NW-3509A (N=50) Placebo (N=39) Total (N=89) PANSS total score, Clinical Global Impression of Change (CGI-C), CGI- n (%) Mod. n (%) Mod. n (%) At least one TEAE Severity (CGI-S), as well as functioning, using the Strauss-Carpenter 23 (46.0) 12 (30.8) 35 (39.3) Somnolence Level of Functioning Scale (LOF). 8 (16.0) 5 (12.8) 13 (14.6) Insomnia 5 (10.0) 1 1 (2.6) 6 (6.7) Study Design Headache 3 (6.0) 2 0 3 (3.4) This 27-day, randomized, double-blind, placebo-controlled, multicenter Overdose 3 (6.0) 1 (2.6) 4 (4.5) study (2 sites in US, 3 in India) enrolled schizophrenic patients Dry mouth 3 (6.0) 2 (5.1) 5 (5.6) experiencing worsening of psychotic symptoms. After a 3-21 day Diarrhoea 0 2 (5.1) 2 (2.2) screening period, eligible patients were randomized to receive either Pain in extremity 0 3 (7.7) 3 (3.4) evenamide (starting dose 15 mg BID) or placebo; the 3:1 initial ratio was Cold sweat/hyperhidrosis 2 (4.0) 0 2 (2.2) later amended to 1:1. Patients were hospitalized overnight after the Mod. = AEs of moderate severity starting dose and weekly dose increases to 20 and 25 mg BID (based on Ø There were no reports of EPS, sedation, weight gain, cardiac tolerability). Safety and efficacy evaluations were performed weekly. changes, or sexual dysfunction. Patient Population Efficacy: Selection Criteria PANSS Positive Scale Total Score: Statistical Analysis of Change Ø Male or female (non-fecund), 18-65 yrs of age, with a DSM-5 diagnosis from Baseline using MMRM+ by visit (mITT population) of schizophrenia; diagnosed ≥ 2 years ago, with current symptoms present for at least one month, receiving risperidone (>2 mg/day or Change from Baseline Difference: NW-3509A vs. Placebo aripiprazole (>10 mg/day) for ≥ 4 weeks at a stable dose. NW-3509A (N=48) Placebo (N=39) Ø Total PANSS <80; Clinical Global Impression - Severity (CGI-S) rating Visit n LS Mean (SE) n LS Mean (SE) LS Mean (95% CI) p-value of mildly, moderately, or moderately severely ill (score of 3, 4 or 5). Day 8 46 -1.262 (0.245) 38 -0.043 (0.263) -1.22 (0.350) (-1.917, -0.523) 0.0008 Ø Patients with 1 or more core positive symptoms (hallucinations, Day 15 44 -1.469 (0.310) 38 -0.592 (0.331) -0.88 (0.446) (-1.765, 0.011) 0.0528 delusions, excitement, suspiciousness/persecution and hostility) rated Day 22 42 -2.273 (0.404) 38 -1.299 (0.430) -0.97 (0.584) (-2.137, 0.188) 0.0993 moderately severe or higher, or rating of moderate on more than 2 of Day 28 47 -2.062 (0.439) 39 -0.872 (0.478) -1.19 (0.643) (-2.47, 0.09) 0.0678 these items, were excluded. +Mixed Model Repeated Measures (MMRM) model: Change from + Region + Baseline value; MMRM model for change from baseline in PANSS Positive total score includes treatment Demographics and Baseline Characteristics group, region, visit, treatment-by-visit interaction as fixed effects and baseline PANSS NW-3509 Placebo Total Positive total score value as covariates. Statistic N=50 N=39 N=89 Summary of Other Efficacy Results Age (Years) Mean (SD) 43.5 (11.93) 44.4 (10.38) 43.9 (11.22) Gender Male [n (%)] 42 (84.0) 35 (89.7) 77 (86.5) Baseline Value and Mean Change from Baseline at Day 28 Baseline Value Change from Baseline to Day 28 Weight (kg) Mean (SD) 83.5 (17.37) 83.8 (19.76) 83.7 (18.35) NW-3509 (N=48) Placebo (N=39) NW-3509 (N=48) Placebo (N=39) BMI (kg/m2) Mean (SD) 27.8 (5.27) 27.9 (5.12) 27.8 (5.17) Scale n Mean (SD) n Mean (SD) n Mean (SD) n Mean (SD) Duration of Schizophrenia Mean (SD) 197.6 (130.6) 243.3 (129.5) 217.6 (131.31) PANSS Total 48 62.7 (6.51) 39 63.1 (8.60) 47 -5.1 (9.67) 39 -3.7 (9.65) (Months) CGI-S 48 3.5 (0.50) 39 3.4 (0.50) 47 -0.3 (0.60) 39 -0.2 (0.74) Number of Hospitalizations Mean (SD) 3.3 (6.34) 2.5 (4.15) 2.9 (5.48) for Schizophrenia LOF Total 48 22.04 (3.608) 39 20.64 (4.533) 47 0.72 (3.321) 39 0.31 (3.130) PANSS Total Score Mean (SD) 62.7 (6.51) 63.1 (8.60) 62.9 (7.42) Proportion of Responders [n/n (%)] at Day 28 Scale Responder Criterion N NW-3509 N Placebo CGI-Severity Mean (SD) 3.5 (0.50) 3.4 (0.50) 3.4 (0.50) PANSS Positive Change from baseline less than 0 (reduction in 47 35/47 (74.5)* 39 17/39 (43.6) Concomitant Antipsychotic: score = improvement) ( ) Risperidone n (%) 41 (82.0) 29 (74.4) 70 (78.7) CGI-C Rating oF 1, 2 or 3 (very much, much or 47 26/47 (55.3) * 39 14/39 (35.9) minimally improved, respectively) Aripiprazole n (%) 9 (18.0) 10 (25.6) 19 (21.3) *p < 0.05 vs. placebo, Fisher’s Exact chi-square test; (*) p<0.1 vs. placebo Conclusions Evenamide, a glutamate modulator, demonstrated evidence of antipsychotic efficacy as an add-on to risperidone or aripiprazole, while devoid of any activity on dopaminergic and serotoninergic pathways. Benefits of evenamide were greater in younger patients. Additional, larger, longer studies are planned to conclusively demonstrate its efficacy in patients with schizophrenia. References [1] Lodge and Grace, 2011. Hippocampal dysregulation of dopamine system function and the pathophysiology of schizophrenia. Trends in Pharmacological Sciences, 32 (9): 507-513. [2] Schwartz et al., 2012. Glutamate neurocircuitry: theoretical underpinnings in schizophrenia. Front Pharmacol. 3(195): 1-11. [3] Tregellas et al., 2014. Intrinsic Hippocampal Activity as a Biomarker for Cognition and Symptoms in Schizophrenia. Am J Psychiatry 171(5): 549–556. [4] Faravelli, et al., 2014 NW-3509A Targets new Mechanisms, and represents a new Approach to the Treatment of Schizophrenia. SIRS conference poster presentation, Florence, Italy 4-9 April 2014. [5] Anand et al., 2017. Evenamide, a Putative Antipsychotic, Targets Abnormal Electrical Activity and Glutamatergic Abnormalities to Improve Psychotic Symptoms in Patients with Schizophrenia: Results from a Phase II, Placebo-Controlled Trial. ICOSR San Diego, USA, 24-28 March 2017. [6] Lieberman JA, Stroup TS, McEvoy JP, et al. Effectiveness of antipsychotic drugs in patients with chronic schizophrenia. N Engl J Med 2005;353:1209-1223. [7] Jones PB, Barnes TR, Davies L, et al. Randomized controlled trial of the effect on Quality of Life of second- vs. first generation antipsychotic drugs in schizophrenia: cost Utility of the Latest Antipsychotic Drugs in Schizophrenia Study (CUtLASS). Arch Gen Psychiatry. 2006;63:1079-1087 . [8] Kahn RS, Fleischhacker WW, Boter H, et al. Effectiveness of antipsychotic drugs in first-episode schizophrenia and schizophreniform disorder: an open randomised clinical trial. Lancet 2008;371:1085-1097. L Faravelli, E Forrest, and S Graham are employees of Newron Pharmaceuticals; R Anand and R Hartman are consultants to Newron.
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