Company Product Description Indication Status
Phase I
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C.   BCX-9930 Factor D inhibitor Paroxysmal nocturnal hemoglobinuria  First patients, naïve to eculizumab and ravulizumab, dosed in a proof-of-concept trial; company expects to report data in the second quarter
JSC Grindeks, of Riva, Latvia Methyl-γ-butyrobetaine (metyl-GBB) Inhibitor of l-carnitine biosynthesis and transport Cardiovascular disease Completed study 
Kancera AB, of Stockholm KAND-567 Fractalkine blocker Myocardial infarction Final part of phase Ib program showed desired tolerability profile for intravenous administration complements previous phase I studies that show the same desired characteristics after oral treatment for 7 days
Phase II
Hansa Biopharma AB, of Lund, Sweden Imlifidase Recombinant cysteine protease Kidney transplant of crossmatch-positive patients Of the 46 transplanted patients treated with the drug, 96% had a functioning graft after 6 months; 2-year death censored graft survival was 89% for 27 patients; overall graft survival was 80% for 30 patients
Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 Free fatty acid formulation of omega-3 pentaenoic acid Elevated triglycerides Started Enhance-IT study testing drug against Vascepa (icosapent ethyl, Amarin Corp. plc) in reducing triglyceride levels and other lipid markers; trial also will gather data on bioavailability and blood levels of eicosapentaenoic acid and other omega-3 fatty acids
Palvella Therapeutics Inc., of Wayne, Pa. PTX-022 (Qtorin 3.9% rapamycin anhydrous gel) mTOR inhibitor Pachyonychia congenita Completed enrollment in phase II/III Valo study in adults; top-line results expected in fourth quarter of 2020
Phase III
Astrazeneca plc, of Cambridge, U.K. Imfinzi (durvalumab) PD-L1 inhibitor Unresectable, stage IV bladder cancer Danube trial did not meet primary endpoints of improving overall survival vs. standard-of-care chemotherapy for Imfinzi monotherapy in patients whose tumor cells and/or tumor-infiltrating immune cells express high levels of PD-L1 or for Imfinzi plus tremelimumab in patients regardless of PD-L1 expression
Idera Pharmaceuticals Inc., of Exton, Pa. Tilsotolimod (IMO-2125) Synthetic Toll-like receptor 9 agonist Anti-PD-1-refractory advanced melanoma Completed enrollment in Illuminate-301 trial testing combination with Yervoy (ipilimumab, Bristol-Myers Squibb Co.); top-line overall response rate and other preliminary data expected in the first quarter of 2021

Notes

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