Cingulate Therapeutics LLC, of Kansas City, said it started dosing in its proof-of-concept trial for CTX-1301, one of two first-line stimulant medications in development to treat attention deficit hyperactivity disorder. The four-week, randomized, three-arm, open-label crossover study is being conducted in healthy volunteers to establish the in vivo pharmacokinetic behavior of CTX-1301 using both gamma scintigraphic imaging and traditional assays to evaluate the body’s absorption, distribution, metabolism and excretion of CTX-1301. It is being conducted by partner Bio-Images Drug Delivery Ltd., of Glasgow, U.K.
Eli Lilly and Co., of Indianapolis, said its phase III RANGE study of Cyramza (ramucirumab) met its primary endpoint of progression-free survival, demonstrating a statistically significant improvement. The global, randomized, double-blind, placebo-controlled trial is evaluating ramucirumab in combination with docetaxel in patients with locally advanced or unresectable or metastatic urothelial carcinoma whose disease progressed on or after platinum-based chemotherapy. Although the primary endpoint has been met, Lilly said it anticipates that overall survival (OS) results are likely to be required for global regulatory submissions. Final OS results are currently expected in mid-2018.
Glycomimetics Inc., of Rockville, Md., said the second of two patient cohorts in the phase II portion of its ongoing phase I/II trial of GMI-1271 in patients with acute myeloid leukemia (AML) has completed enrollment. The second cohort is composed of 66 participants with relapsed/refractory AML. The study is designed to evaluate the potential of GMI-1271, an E-selectin antagonist drug candidate, in combination with chemotherapy, as a treatment for individuals with either newly diagnosed or relapsed/refractory AML.
M Pharmaceutical Inc., of Cincinnati, along with Camargo Pharmaceutical Services, said it received an FDA response for its clinical study plan through the 505(b)(2) pathway for obesity management drug C-103, a reformulated version of orlistat. Trials are set to start this year.
Medical Prognosis Institute A/S (MPI), of Hoersholm, Denmark, said the first patient entered the Oncology Venture APO-010 phase I/II trial in multiple myeloma. In total, 150 evaluable patients will be screened using MPI’s DRP (Drug Response Predictor) technology with the aim to identify those patients with the highest likelihood to benefit from treatment with APO-010, which targets the FAS-ligand.
Mei Pharma Inc., of San Diego, said an independent safety review committee completed its pre-specified review of the first cohort of six evaluable patients in a phase Ib, open-label, dose-escalation study of ME-401, a selective, oral PI3K delta inhibitor, in relapsed/refractory chronic lymphocytic leukemia (CLL) and follicular lymphoma, and declared a minimum biologically effective dose for ME-401 at the starting dose of 60 mg and recommended escalation to a 120-mg dose cohort. To date, all six patients have been on study for a minimum of 10 weeks (ranging 10 to 28 weeks), with no reports of ALT/AST elevations, colitis or pneumonitis, events commonly reported with other drugs in that class. One patient in the study experienced grade 3 neutropenia that was considered related to study drug. The trial is set to enroll up to 84 patients.
Novartis AG, of Basel, Switzerland, said findings from a pilot study of CTL-119, a humanized CD19-directed CAR T-cell therapy, in combination with BTK inhibitor Imbruvica (ibrutinib, Abbvie Inc./Johnson & Johnson) in patients with relapsed/refractory chronic lymphocytic leukemia (CLL), who had been taking ibrutinib for at least six months and who were not in complete remission, showed that eight of nine evaluable patients had no signs of CLL in their bone marrow at three months. One of those patients had a partial response. All study patients had to have failed at least one prior regimen before ibrutinib or carried high-risk cytogenetics or mutations. The results will be presented at the upcoming American Society of Clinical Oncology meeting in Chicago.
Nymox Pharmaceutical Corp., of Hasbrouck Heights, N.J., reported phase III results showing that prostate enlargement and prostate cancer drug fexapotide triflutate produced clinically important improvements in sexual function in first-line patients who received fexapotide in its U.S. long-term clinical trials. Data demonstrated several lines of prospective randomized double-blind evidence indicating sexual function improvement. At 12 months after treatment in the first-line previous treatment-naive patients (n=370), there was a statistically significant improvement from baseline in fexapotide-treated subjects (p<0.0001), while placebo showed no improvement from baseline. In first-line previous treatment-naive subjects with SFQ long-term assessments (12 months to 51 months after treatment, n=156), the fexapotide-treated subjects had statistically significant improvement compared to placebo. Placebo-treated patients at long-term showed worsening of sexual function (mean -0.88 points) while fexapotide treated patients showed improvement (+0.64 points, p=0.0049). The percentage of first-line previous treatment-naive patients in the study with pre-existent sexual dysfunction (baseline SFQ ≤3 points) who ended the study improved and no longer with self-reported sexual dysfunction (SFQ ≥4 points) was statistically significant (p<0.05) while there was no change in the placebo group. Nymox filed for marketing approval for fexapotide in Europe earlier this month for benign prostatic hyperplasia.
Ralexar Therapeutics Inc., of Malvern, Pa., said it started patient enrollment in a phase IIb trial of ALX-101 topical gel as a potential treatment for atopic dermatitis. The multicenter, randomized, double-blind, vehicle-controlled study will enroll adults and adolescents with moderate atopic dermatitis. Two concentrations of ALX-101 will be tested (ALX-101 gel 1.5 percent and ALX-101 gel 5 percent) along with placebo gel. Gel will be applied topically twice daily for six weeks. The primary objective is to evaluate the mean change in Physician’s Global Assessment score from baseline to day 43. ALX-101 is an LXR agonist.
Regenxbio Inc., of Rockville, Md., said the first patient was dosed in a phase I trial testing gene therapy RGX-314 in patients with wet age-related macular degeneration (AMD). The multicenter, open-label, multiple-cohort, dose-escalation trial will assess the safety and tolerability of RGX-314 as a one-time therapy in about 18 patients with previously treated wet AMD. Primary endpoints include adverse events, certain laboratory measures (including immunological parameters) and ocular examinations and imaging. An interim trial update is expected by the end of the year.
Teva Pharmaceutical Industries Ltd., of Jerusalem, reported results from the phase III HALO study of fremanezumab, a monoclonal antibody targeting the CGRP ligand. In the chronic migraine study, patients treated with fremanezumab experienced statistically significant reduction in the number of monthly headache days of at least moderate severity vs. placebo (-2.5 days) during the 12-week period after first dose, for both monthly (-4.6 days, p<0.0001) and quarterly (-4.3 days, p<0.0001) dosing regimens. Similar to the phase II trials, both patients that were on monotherapy and stable doses of prophylactic medications were included in the trial. Patients on fremenezumab also experienced significant improvement compared to placebo on all secondary endpoints for both monthly and quarterly dosing regimens, including response rate, onset of efficacy, efficacy as a monotherapy and disability. The results were positive, and of 13 hierarchical comparisons, the “p” value was <0.0001 in 12 of them, being 0.0004 in the remaining. The most commonly reported adverse event in the study was injection site pain, with similar rates in the placebo and active groups. Teva anticipates submitting a BLA this year.
Tonix Pharmaceuticals Holding Corp., of New York, presented data at the American Society of Clinical Psychopharmacology meeting in Miami Beach, including results from a retrospective analysis of the phase II AtEase trial, which indicated that a study entry CAPS-5 severity score of ≥33 is more aligned with the entry criteria of previous post traumatic stress disorder (PTSD) pharmacotherapy registration trials using prior CAPS versions. In the AtEase baseline CAPS-5 ≥33 subgroup, the effect size of TNX-102 SL 5.6 mg is about 0.5 on total CAPS-5 and also about 0.5 on cluster B (intrusion) and cluster E (arousal and reactivity) scores. The effect of TNX-102 SL was studied on remission, which was defined by CAPS-5 < 11 at week 12, and sustained remission, which was CAPS-5 < 11 at both weeks eight and 12. TNX-102 SL 5.6 mg showed a statistically significant increase in sustained remission relative to placebo, and was well-tolerated with a high completion rate in AtEase. TNX-102 SL is a sublingual transmucosal formulation of cyclobenzaprine, currently in phase III development for PTSD.