Company Product Description Indication Status
Phase I
Applied Genetic Technologies Corp., of Gainesville, Fla. AAV-CNGB3 gene therapy Gene therapy Achromatopsia Completed enrollment of adults in phase I/II trials; pediatric enrollment continuing; interim data of all adult dose groups expected in second half of 2020
Aptevo Therapeutics Inc., of Seattle APVO-436 Anti-CD123, anti-CD3 targeted bispecific antibody Acute myeloid leukemia; myelodysplastic syndrome Trial advancing on schedule with dosing in cohort 6 set to begin shortly; dose levels now entering potential therapeutic range; no evidence of dose-limiting toxicities observed in cohort 5; no evidence of drug-induced anti-drug antibodies observed to date
Genetx Biotherapeutics LLC, of Sarasota, Fla., and Ultragenyx Pharmaceutical Inc., of Novato, Calif. GTX-102 Antisense oligonucleotide Angelman syndrome Dosed the first patient in open-label phase I/II KIK-AS trial
Moderna Inc., of Cambridge, Mass. mRNA-1273 mRNA vaccine SARS-CoV-2  Dosed first participant in study being conducted by NIH
Phase II
Can-Fite Biopharma Ltd., of Petah-Tikva, Israel Namodenoson Adenosine A3 receptor agonist; phosphatidylinositol 3 kinase subunit 3 inhibitor Hepatocellular cancer Enrolled 7 patients for the compassionate use program in HCC; no severe adverse events reported; 2 patients from former phase II study still undergoing treatment with namodenoson, each with an overall survival of >2.5 years
Cortexyme Inc., of South San Francisco COR-388 Gingipain inhibitor Alzheimer’s disease Phase II/III Gain trial enrollment has reached 300 of target 570 patients; top-line results expected in Q4 2021
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris  Kevzara (sarilumab) IL-6 receptor antagonist COVID-19 U.S. phase II/III trial initiated and will begin enrolling patients hospitalized with severe COVID-19 infection
Phase III
Aimmune Therapeutics Inc., of Brisbane, Calif. Palforzia (peanut allergen powder-dnfp) Oral immunotherapy Peanut allergy New data from ARC004 follow-on trial to 52-week Palisade study showed patients tolerated more peanut protein, experienced fewer adverse events and continued immunomodulation as evidenced by reductions in peanut-specific immunoglobulin blood levels after an additional 56 weeks of daily treatment
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. Topical roflumilast PDE4 inhibitor Plaque psoriasis Submitted protocol amendment to FDA to include children, ages 2-11, in ongoing Dermis-1 and Dermis-2 pivotal trials
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat (BCX-7353) Oral plasma kallikrein antagonist Prophylaxis of hereditary angioedema attacks Data from Apex-2 trial showed 150-mg once-daily dose reduced patients’ monthly use of standard-of-care (SoC) on-demand medicine by 53.6% (p<0.001) vs. placebo, and reduced number of HAE attacks requiring acute SoC treatment by 49.2% (p<0.001) vs. placebo
Novo Nordisk A/S, of Bagsvaerd, Denmark Concizumab Inhibits tissue factor pathway inhibitor Hemophilia A and B Studies explorer7 and 8 were paused as a result of occurrence of nonfatal thrombotic events in 3 patients enrolled; company and independent data monitoring committee are assessing the events; phase II program, explorer5, also paused

Notes

For more information about individual companies and/or products, see Cortellis.

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