Acadia Pharmaceuticals Inc., of San Diego, started ADVANCE, a phase II study to evaluate pimavanserin for adjunctive treatment in patients with negative symptoms of schizophrenia. Studies show that about 40 to 50 percent of schizophrenia patients suffer from prominent negative symptoms. There is currently no drug approved by the FDA for the symptoms. As a selective serotonin inverse agonist, pimavanserin is a new class of antipsychotic medication with a distinct mechanism of action targeting serotonergic 5-HT2A receptors while avoiding activity at dopamine and other receptors commonly targeted by other antipsychotics, the company said. ADVANCE is a 26-week, randomized, double-blind, placebo-controlled, multicenter study. About 380 patients will be randomized to receive either pimavanserin or placebo, orally, once daily, in addition to their ongoing antipsychotic in a flexible dosing regimen. The starting daily dose of 20 mg of pimavanserin at baseline may be adjusted to 34 mg or 10 mg during the first eight weeks of treatment. The primary endpoint of the study is the change from baseline to week 26 on the Negative Symptom Assessment-16 total score. Following participation in ADVANCE, patients will be eligible to enroll in a 52-week open-label extension study.
Bayer AG, of Basel, Switzerland, disclosed a phase III study evaluating the efficacy and safety of a newly developed formulation of investigational medication nifurtimox (Lampit) in children of all age groups who have been diagnosed with Chagas disease. Nifurtimox is not approved by the FDA and there are no other FDA-approved treatments for children with Chagas disease, a potentially life-threatening illness caused by the protozoan parasite Trypanosoma cruzi. The pathogen is often transmitted by an infected mother to her newborn during pregnancy or childbirth. Nifurtimox is on the World Health Organization’s (WHO) list of essential medicines for both adults and children, and WHO is distributing nifurtimox in its efforts to control Chagas disease by 2020. Since 2004, Bayer has given WHO a permanent supply guarantee for nifurtimox and provided financial assistance to support logistics and drug distribution.
Cellectar Biosciences Inc., of Madison, Wis., said it selected CRO INC Research to oversee its National Cancer Institute (NCI)-supported phase II trial testing CLR 131 in patients with multiple myeloma and select hematologic malignancies. The 80-patient study will include relapsed/refractory patients with multiple myeloma, chronic lymphocytic leukemia/small lymphocytic lymphoma, lymphoplasmacytic lymphoma, marginal zone lymphoma, mantle cell lymphoma and potentially diffuse large B-cell lymphoma, who have been treated with standard therapy for their underlying malignancies. It is set to start in the first quarter of next year. The company anticipates that its $2 million NCI grant will cover about 50 percent of the study’s cost, and the terms of the grant allow Cellectar to pursue an additional $3 million for a pivotal phase III trial of the company’s lead radiotherapeutic compound.
Clearside Biomedical Inc., of Alpharetta, Ga., enrolled the first patient in a phase I/II trial called HULK of Zuprata, its suspension formulation of the corticosteroid triamcinolone acetonide, for the treatment of diabetic macular edema. The HULK trial is an open-label, multicenter study designed to assess the safety and efficacy of the administration of Zuprata to the suprachoroidal space concomitant with intravitreal Eylea (aflibercept, Regeneron Pharmaceuticals Inc.), as well as suprachoroidal Zuprata monotherapy. The trial targets enrollment of about 20 patients, with about equal numbers in each of the two arms. Anatomical and functional data and safety information will be collected at each monthly visit during the six-month evaluation period.
Mallinckrodt plc, of Chesterfield, U.K., confirmed enrollment of the first patients in its phase IV study testing H.P. Acthar gel (repository corticotropin injection) in systemic lupus erythematosus (SLE) patients with persistently active disease. The randomized, double-blind, placebo-controlled trial is designed to build on data from a pilot study in patients with active SLE, recently published in Lupus Science & Medicine. H.P. Acthar gel is approved by the FDA for use during an exacerbation or as a maintenance therapy in select patients with SLE.
Modus Therapeutics AB, of Stockholm, a Karolinska Development AB company, said the independent data safety monitoring board, after a planned safety review, said adolescents, between the ages of 12 and 18, will now be enrolled in the ongoing phase II trial testing sevuparin for sickle cell disease (SCD). Modus said it also decided to increase the sample size of the study to about 150 patients. The trial is designed to demonstrate a reduced time to resolution of vaso-occlusive crises (VOC) in hospitalized SCD patients treated with sevuparin in comparison to those treated with placebo and is targeting a potential 30 percent reduction in the time to resolution of the VOC.
Nantkwest Inc., of Culver City, Calif., presented early analysis from its ongoing phase II Merkel cell carcinoma study at the Society for Immunotherapy of Cancer meeting in National Harbor, Md., showing that its activated natural killer, or aNK, cell therapy showed evidence of a radiological complete response following single-agent infusion in a patient with recurrent disease after multiple lines of therapy, including relapse after checkpoint inhibitor therapy.
Nivalis Therapeutics Inc., of Boulder, Colo., said it completed enrollment in the second of two phase II trials of cavosonstat (N91115), a stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is being evaluated as an add-on therapy to Kalydeco (ivacaftor, Vertex Pharmaceuticals Inc.) in adult patients who have one copy of the F508del mutation and a second mutation that results in a gating defect in the CFTR protein. A total of 19 patients have been enrolled in the U.S., and top-line results are expected in the first quarter of next year.
OWC Pharmaceutical Research Corp., of Petach Tikva, Israel, is submitting the final safety protocol for a planned early stage trial of its medical-grade cannabis cream to the Israeli national IRB committee at the office of the Health Minister. The 12-week trial will test multiple escalating doses of a topical creme infused with active cannabinoids to determine safety and tolerability in healthy volunteers.
Repros Therapeutics Inc., of The Woodlands, Texas, reported top-line results for both its pooled oral and vaginal delivery phase II studies in the treatment of uterine fibroids, both of which enrolled women with confirmed fibroids by MRI at baseline and who were experiencing more than 80 mL of blood loss during menses as confirmed by alkaline hematin assessment. Proellex at doses of both 6 mg and 12 mg, delivered by either route, substantially and significantly reduced excessive menstrual bleeding. Pooled oral and vaginal delivery vs. placebo met the primary endpoint of induction of amenorrhea (p<0.0001 and p=0.0071, respectively), and a statistically significant reduction in fibroid size from baseline was achieved by the combined active arms for the pooled oral dosage form compared to placebo (p=0.0004). The company said it will propose an oral route of administration for phase III development and plans to submit a request to the FDA before the end of this year to discuss the phase III program.