Company Product Description Indication Status
Phase I
Aivita Biomedical Inc., of Irvine, Calif. AV-COVID-19 COVID-19 vaccine COVID-19 infection Well-tolerated, with no serious adverse events; treated individuals produced antibodies
Altimmune Inc., of Gaithersburg, Md. AdCOVID COVID-19 vaccine COVID-19 infection Trial begun
Celsion Corp., of Lawrenceville, N.J. GEN-1 IL-12 immunotherapy Ovarian cancer One-third of 110 patients enrolled; 27 have had debulking surgery; 12 of 15, or 80%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed
Compugen Ltd., of Holon, Israel COM-701 PVRG immune checkpoint inhibitor Ovarian, breast, endometrial and microsatellite-stable colorectal cancers In 15 patients with a median of 5 prior anticancer therapies (range of 2-10), COM-701 in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) was well-tolerated with no reported dose-limiting toxicities up to the fifth and final dose cohort of 20 mg/kg and Opdivo 480 mg, both I.V. Q4 weeks
Kronos Bio Inc., of San Mateo, Calif. KB-0742 Cyclin dependent kinase 9 inhibitor Myc-amplified solid tumors First patient dosed
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2) COVID-19 vaccine COVID-19 infection Trial begun in order to understand the effect of a booster on immunity against COVID-19 caused by the circulating and potential newly emerging SARS-CoV-2 variants 
Phase II
Bioage Labs Inc., of Richmond, Calif. BGE-117  HIF prolyl hydroxylase inhibitor Anemia Initiated phase IIa study in Australia for unexplained anemia of aging in 166 participants 65 and older; primary efficacy endpoints are hemoglobin levels and patient-reported scores on FACIT-F scale; top-line results expected in first half of 2022
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 (dexmedetomidine, sublingual) Alpha-2a receptor agonist  Agitation Initiated Placidity trial in adults in ICU with delirium-related agitation, expecting to randomize 20 into each ascending-dose cohort (starting doses of 120 ug, 180 ug, 240 ug or 300 ug) or matching placebos to determine optimal starting dose; primary endpoint is reduction in agitation measured by at least 2-point drop in RASS 2 hours post administration; top-line results expected in first quarter of 2022
Bluebird Bio Inc., of Cambridge, Mass., and Bristol Myers Squibb Co., of New York Idecabtagene vicleucel (BB-2121) BCMA-directed CAR T-cell immunotherapy Multiple myeloma The New England Journal of Medicine published data from pivotal Karmma study, which met primary endpoint of overall response and key secondary endpoint of complete response in adults with relapsed/refractory disease following at least 3 prior therapies
Chimerix Inc., of Durham, N.C. DSTAT (dociparstat) Glycosaminoglycan derivative of heparin COVID-19 infection In first cohort in phase II/III study of 12 people hospitalized with infection and acute lung injury, with primary endpoint of survival without need for mechanical ventilation through day 28, 1 person on study drug was ventilated and recovered but no deaths were reported in DSTAT arm; 2 on placebo progressed to ventilation and died
Clene Inc., of Salt Lake City CNM-Au8 Bioenergetic nanocatalyst Multiple sclerosis Wholly owned subsidiary Clene Nanomedicine Inc. presented blinded interim data from Visionary-MS study showing increasing mean improvements in overall MSFC scores and in 4 MSFC subscales (low contrast letter acuity, symbol digit modalities test, 9-hole peg test and timed 25-foot walk) vs. comparator group (mixed-effects model; p<0.0001 vs. baseline) and updated interim data from ongoing Repair-MS study showing CNS target engagement, with catalytic bioenergetic improvements in NAD+/NADH ratio and adenosine triphosphate levels
CNS Pharmaceuticals Inc., of Houston Berubicin Topoisomerase II inhibitor Glioblastoma Adaptive, potentially pivotal trial on schedule to begin enrollment in March 2021; primary endpoint is overall survival
Entera Bio Ltd., of Jerusalem EB-612 (teriparatide) Parathyroid hormone receptor agonist Hypoparathyroidism Results of previously completed phase IIa study, which met primary and secondary endpoints, published in Journal of Bone and Mineral Research 
Glaxosmithkline plc, of London Otilimab GM-CSF receptor modulator COVID-19 infection On primary endpoint of proportion of participants of all ages (n=806) alive and free of respiratory failure 28 days after treatment with study drug + standard of care (SOC) vs. SOC alone, Oscar study showed treatment difference of 5.3% but missed statistical significance; pre-planned efficacy analysis in those 70 and older (n=180) showed 65.1% on study drug met primary endpoint vs. 45.9% on SOC alone (nominal p=0.009); mortality analysis up to day 60 showed treatment difference of 14.4% favoring otilimab in people 70 and older (nominal p=0.040); GSK amending study to expand older cohort
Karuna Therapeutics Inc. of Boston Karxt (xanomeline + trospium chloride) Dual muscarinic M1/M4 receptor agonist Schizophrenia The New England Journal of Medicine published data from Emergent-1 trial that showed statistically significant improvements in primary and key secondary efficacy measures vs. placebo
Roche Holding AG, of Basel, Switzerland Evrysdi (risdiplam) SMN2 gene modulator Spinal muscular atrophy The New England Journal of Medicine published Firefish part 1 data showing treatment at 12 months helped 90% of infants with SMA survive without permanent ventilation and 33% sit without support
Springworks Therapeutics Inc., of Stamford, Conn.,  Mirdametinib Dual MEK-1/MEK-2 protein kinase inhibitor NF1-associated plexiform neurofibroma Interim data from first 20 adults in phase IIb Reneu trial showed 10/20 achieved objective response, 16/20 remained on study and median time on treatment was 10.1 cycles (about 10 months); trial reached about 70% of target enrollment of 100 participants, with full enrollment expected in second half of 2021
Stero Biotechs Ltd., of Tel Aviv, Israel Cannabidiol-steroid formulation Small molecule COVID-19 infection 11 of planned enrollment of up to 20 participants recruited to date to assess benefits in people with infection resulting in severe acute respiratory distress syndrome
Viela Bio Inc., of Gaithersburg, Md. Uplizna (inebilizumab-cdon) B-lymphocyte antigen CD19 inhibitor Neuromyelitis optica spectrum disorder Data from open-label extension (n=216) of pivotal N-Momentum trial showed reduction of attacks was sustained for duration of 4-year observation period, with 2 participants on therapy for >4 years
Phase III
Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 (repurposed ondansetron) 5-HT 3 receptor antagonist Alcohol use disorder Pivotal Onward trial reached 50% enrollment, with 86% retention rate 
Aimmune Therapeutics, a unit of Société des Produits Nestlé SA, of Vevey, Switzerland Palforzia Peanut (arachis hypogaea) allergen powder-dnfp Peanut allergy Pooled data from the Palisade, Ramses and Artemis and 3 open-label extension studies showed 3 out of 4 patients were able to achieve the 300-mg maintenance dose; over an approximately 3.5-year treatment period, 14 participants (1.2%) experienced a treatment-related severe systemic allergic reaction
Centre for Probe Development and Commercialization, of Hamilton, Ontario [18F]PSMA-1007  Positron-emitting tomography imaging agent targeting prostate-specific membrane antigen Prostate cancer First patient dosed in the study that will enroll 100 men with suspected persistent or recurrent prostate cancer based on detectable levels of prostate serum antigen and a negative or equivocal detection of disease by conventional imaging
I-Mab, of Shanghai Eftansomatropin alfa (TJ-101) Long-acting recombinant human growth hormone Pediatric growth hormone deficiency Treated first of 165 patients in the noninferiority Taller study comparing eftansomatropin alfa to Norditropin (somatropin, Novo Nordisk A/S)
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Regen-Cov (casirivimab/imdevimab)  Monoclonal antibodies targeting SARS-CoV-2 Non-hospitalized COVID-19 Independent data monitoring committee recommended stopping enrollment into the placebo group due to clear clinical efficacy on reducing the rate of hospitalization and death with both the 1,200-mg and 2,400-mg doses; study will continue to enroll patients in both dose groups
Phase IV
EMD Serono, a unit of Merck KGaA, of Darmstadt, Germany Mavenclad (cladribine) Synthetic purine nucleoside  Relapsing multiple sclerosis In a retrospective analysis, 12 patients treated with the seasonal influenza vaccine and 3 patients given the varicella zoster virus vaccine while taking Mavenclad developed protective antibodies; in the Clock-MS study, protective antibodies increased 4 weeks after administration of an influenza vaccine in 3 patients studied

Notes

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