Company Product Description Indication Status
Phase I
Aileron Therapeutics Inc., of Watertown, Mass. ALRN-6924 Chemoprotectant Small-cell lung cancer Interim data from phase Ib dose-optimization part of phase Ib/II trial in chemotherapy-induced toxicity showed protective effect against severe chemotherapy-induced anemia and thrombocytopenia across all dose levels vs. historical controls; patients in 0.3-mg/kg group met protocol-defined criteria for reduction of NCI CTC grade 3/4 neutropenia to 50% in first treatment cycle; study enrolled patients with advanced p53-mutated disease treated with second-line topotecan
Belx Bio-Pharmaceutical (Taiwan) Corp., of Taiwan Bel-X-HG Small molecule Advanced cancer Terminated in light of ongoing COVID-19 pandemic, which resulted in difficulty with patient enrollment
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Valoctocogene roxaparvovec F8 gene stimulator Hemophilia A 4-year results for 6e13-vg/kg and 3-year update for-4e13 vg/kg cohorts in phase I/II study showed all participants remained off prophylactic factor VIII treatment following initial single dose of study therapy; cumulative mean annualized bleed rates remained < 1 in both cohorts and below pretreatment baseline; in past year, 6 of 7 in 6e13-vg/kg cohort and 5 of 6 in 4e13-vg/kg cohort were free of spontaneous bleeds
Histogen Inc., of San Diego HST-001 FGF-7/VEGF/WNT 7A ligand; follistatin agonist Alopecia First of about 36 participants with male pattern hair loss dosed in phase I/II study; efficacy measures include non-vellus hair count, total hair count and hair thickness density
Lineage Cell Therapeutics Inc., of Carlsbad, Calif. Opregen Human embryonic stem cell-derived retinal pigmented epithelial cells  Dry age-related macular degeneration Restoration of retinal tissue observed in participant in phase I/IIa study; area of geographic atrophy assessed at 9 months was about 25% smaller than patient’s pretreatment baseline and grew about 50% slower than historical rate during subsequent 6 months
Phase II
Antibe Therapeutics Inc., of Toronto ATB-346 Cyclooxygenase 1/2 inhibitor Osteoarthritis Phase IIb study met primary endpoint, with study drug showing superiority to placebo at 250 mg (p=0.01) and 200 mg (p=0.007) and 150-mg dose also showing efficacy; 250-mg and 200-mg doses showed highly statistically significant reduction in WOMAC stiffness subscale score (p<0.001 for both) and were superior to placebo in WOMAC difficulty performing daily activities (DPDA) subscale score (p=0.004 and 0.001, respectively); 150-mg dose showed statistically significant improvement in stiffness vs. placebo (p= 0.03) and response in DPDA
Glaxosmithkline plc, of London GSK-3772847  Anti-interleukin-33 receptor monoclonal antibody Moderate to severe asthma with allergic fungal airway disease Recruitment terminated early due to high screen failure rate and feasibility of completing study in timely way
Mesoblast Ltd., of Melbourne, Australia Ryoncil (remestemcel-L) Allogeneic mesenchymal stem cells Chronic obstructive pulmonary disease Improvements seen in each of prespecified endpoints of forced expiratory volume, forced vital capacity and 6-minute walk test distance (all p<0.01), with maximal effects at 4 months
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID19 spike glycoprotein modulator COVID-19 infection First participants dosed in 2 age cohorts (18-55 years, n=300; 55 and above, n=300) of healthy individuals; study assessing 50-μg and 100-μg dose at 2 vaccinations 28 days apart vs. placebo
Novus Therapeutics Inc., of Irvine, Calif. OP-0201 Combination surfactant/spreading agent Otitis media Phase IIa trial missed statistical significance in resolution of bulging tympanic membrane vs. placebo (51% vs. 47.3%, respectively, p=0.62) and middle ear effusion (55.9% vs. 37.9%, p=0.07)
Taiwan Liposome Co. (TLC), of Taipei, Taiwan TLC-590 (sustained release ropivacaine) Sodium channel inhibitor Pain Study drug reduced pain intensity more than placebo and bupivacaine at every time interval from 0 hours through end of study at 168 hours and achieved statistically significant pain relief vs. placebo (p<0.001) and bupivacaine (p=0.0188) from 0 to 24 hours; differences in AUC 0 to 72 hours did not reach statistical significance
Phase III
CTI Biopharma Corp., of Seattle Pacritinib  CSF-1 antagonist; FLT3/IRAK-1 kinase inhibitor COVID-19 infection First of about 358 participants hospitalized with severe infection enrolled in Pre-Vent trial; primary endpoint is proportion who progress to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or die by day 28; top-line data expected by year-end 2020
Gilead Sciences Inc., of Foster City, Calif. Remdesivir  COVID19 non-structural protein 8 modulator/replicase polyprotein 1AB inhibitor; RNA polymerase COVID-19 infection In second Simple trial, in 600 hospitalized patients with moderate COVID-19 pneumonia, those in 5-day treatment group were 65% more likely to have clinical improvement at day 11 vs. standard of care (p=0.017); 10-day treatment group vs. SOC trended toward but did not reach statistical significance (p=0.18)
Iterum Therapeutics plc, of Dublin Sulopenem Penem beta-lactam antibiotic Urinary tract infection In Sure 2 trial, study drug missed statistical noninferiority vs. ertapenem in primary endpoint of overall clinical and microbiologic response on day 21 in micro-MITT population, with responder rates of 67.8% vs. 73.9%, respectively
Merck KGaA, of Darmstadt, Germany, and its EMD Serono unit Evobrutinib BTK inhibitor Relapsing multiple sclerosis Study comparing drug to Avonex (interferon-beta-1a) terminated due to sponsor decision
Novartis AG, of Basel, Switzerland Brolucizumab VEGF inhibitor Macular edema secondary to retinal vein occlusion Trial temporarily paused due to COVID-19 and safety measure
Ocugen Inc., of Malvern, Pa. OCU-300 (repurposed brimonidine) Alpha 2 adrenoceptor agonist Ocular graft-vs.-host disease  Trial halted for futility following pre-planned interim sample size analysis by independent data monitoring committee
Pfizer Inc., of New York Ibrance (palbociclib) Cyclin-dependent kinase-4/6 inhibitor Breast cancer In preplanned analysis, independent data monitoring committee determined Pallas trial unlikely to show statistically significant improvement in primary endpoint of invasive disease-free survival in HR+, HER2- early disease


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