Phase I
Celularity Inc., of Florham Park, N.J. CYNK-001 Human placental hematopoietic stem cell-derived natural killer cells Glioblastoma First participant dosed in study to determine maximum tolerated dose to treat recurrent disease
City of Hope, of Duarte, Calif. COH-04S1 Synthetic modified vaccinia ankara-based vaccine COVID-19 infection Trial expected to enroll at least 119 healthy volunteers to determine safe and tolerable dose and assess whether single dose may be sufficient for immune protection
Reithera Srl, of Rome GRAd-COV2 COVID-19 Spike glycoprotein modulator COVID-19 infection Trial advancing on schedule, with dosing complete on first cohort of healthy adults divided into 3 study arms of 15 participants; preliminary results showed vaccine candidate generated spike-binding antibodies and T-cell responses at tested doses; trial advancing to second set of 3 cohorts of healthy older adults ages 65 to 85
Targovax ASA, of Oslo, Norway ONCOS-102 GM-CSF receptor agonist Mesothelioma 18-month follow-up data from phase I/II combination trial with standard-of-care (SOC) chemotherapy in 31 people with malignant pleural disease showed more than half in first-line treatment group (n=8) were still alive, with median overall survival (mOS) not reached and expected mOS of 18.2 months or longer based on current data vs. first-line SOC (n=6), with fewer than half alive and mOS of 14.2 months or less; next survival analysis planned in first half of 2021
Phase II
Apnimed Inc., of Cambridge, Mass. AD-109 (atomoxetine + oxybutynin) Norepinephrine reuptake inhibitor + muscarinic receptor antagonist Obstructive sleep apnea First of 48 participants dosed in crossover factorial study; primary endpoint is change in apnea-hypopnea index for treatment vs. each individual component of study drug and placebo; data expected in first half of 2021
Capricor Therapeutics Inc., of Los Angeles CAP-1002  Allogeneic cardiosphere-derived cell therapy COVID-19 infection First 2 of 60 participants with severe infection requiring supplemental oxygen dosed in Inspire study
Kubota Vision Inc., of Seattle, a subsidiary of Kubota Pharmaceutical Holdings Co. Ltd. Emixustat hydrochloride Inhibits RPE65 Macular atrophy secondary to Stargardt disease Phase IIa data, published in the British Journal of Ophthalmology, showed subjects randomized to 10 mg demonstrated near complete suppression of recovery rate of the rod photoreceptor b-wave on electroretinography, indicating robust RPE65 inhibition, while those in the 5-mg group demonstrated moderate suppression; no effects were seen in the 2.5-mg group
Phase III
Bausch Health Cos. Inc., of Laval, Quebec NOV-03 (perfluorohexyloctane) Ophthalmic liquid formulation using Eyesol technology Dry eye disease Second pivotal study initiated to treat DED associated with Meibomian gland dysfunction; initial phase III study 85% enrolled
Glaxosmithkline plc, of London GSK-3888550A Vaccine Respiratory syncytial virus Started patient dosing in Grace trial to test maternal immunization 
Oramed Pharmaceuticals Inc., of New York ORMD-0801 Insulin ligand; insulin receptor agonist Type 2 diabetes First of 675 participants screened in ORA-D-013-1 trial to treat inadequate glycemic control; primary endpoint is efficacy vs. placebo in improving glycemic control assessed by A1c, with secondary endpoint of change from baseline in fasting plasma glucose at 26 weeks
Principia Biopharma Inc., of South San Francisco, unit of Sanofi SA, of Paris Rilzabrutinib BTK inhibitor Pemphigus Pegasus trial enrolled ahead of schedule with 120 participants with moderate to severe disease who are newly diagnosed or relapsing with chronic disease; primary efficacy endpoint is achievement of durable complete remission at 37 weeks of treatment
National Research Center for Epidemiology and Microbiology and Russian Direct Investment Fund (RDIF), of Moscow Sputnik V  COVID-19 Spike glycoprotein modulator COVID-19 infection Second interim analysis showed 91.4% efficacy for vaccine on day 28 after first dose; efficacy is >95% 42 days after initial dose, based on analysis of data on volunteers (n=18,794) who received both doses or placebo
Strongbridge Biopharma plc, of Dublin Recorlev (levoketoconazole) Cortisol synthesis inhibitor Cushing's syndrome Analyses from Sonics study, published online in Pituitary, showed mean improvements from baseline at end of maintenance phase (month 6) for acne, hirsutism (females) and peripheral edema; improvements seen as early as day 1 of maintenance for hirsutism, end of month 1 for acne and month 4 for peripheral edema; by month 3 of maintenance, mean improvements were seen in patient-reported quality of life outcomes and, by month 6, symptoms of depression were improved
Tolmar Pharmaceuticals Inc., of Buffalo Grove, Ill. Fensolvi (leuprolide acetate) GNRH receptor agonist Central precocious puberty Data published in Journal of Clinical Endocrinology and Metabolism showed study drug suppressed reproductive hormones and stopped or caused regression of pubertal progression


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