Company Product Description Indication Status
Phase I
Apollomics Inc., of Foster City, Calif. APL-106 (uproleselan sodium) E selectin antagonist Acute myeloid leukemia First adult with relapsed/refractory disease enrolled in trial in China, part of phase I and phase III bridging study in combination with chemotherapy
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo Cholesterol binding agent; lipid metabolism modulator Niemann-Pick disease type C Last participant completed last visit in phase I/II study; top-line data expected in March 2021, with pivotal phase III study expected to open enrollment in second quarter of 2021
Dnatrix Inc., of Houston DNX-2440 (Delta-24-RGDOX) OX40 co-stimulator Metastatic liver cancer First of 24 to 30 participants with resectable disease dosed in 2-part study; efficacy measures include evaluation of tumor cell killing of injected and uninjected tumors, of viral replication in injected tumor and of local and systemic antitumor immune responses
Landos Biopharma Inc., of Blacksburg, Va. NX-13 NLRX1 stimulator Inflammatory bowel disease Trial met primary and secondary endpoints, with maximum tolerated dose identified as 10-fold greater than expected therapeutic dose
Ridgeback Biotherapeutics LP, of Miami Molnupiravir (EIDD-2801/MK-4482) RNA polymerase inhibitor COVID-19-related severe acute respiratory syndrome Study in healthy volunteers, which met primary safety, tolerability and pharmacokinetics measures, published online in Antimicrobial Agents and Chemotherapy 
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole (thin film freezing/powder formulation/inhalant) Lanosterol-14 demethylase inhibitor Invasive pulmonary aspergillosis Trial showed that doses of 10, 20, 40 and 80 mg could be delivered twice daily using dry powder inhaler device without significant adverse events; mean peak plasma drug levels reached concentrations of 227 ng/mL following repeated dosing at pivotal trial selected dose of 80 mg, twice daily for 7 days
Ysopia Bioscience SA, of Bordeaux, France Xla1 Christensenella bacteria-based microbiome therapy Obesity First arm of Causality trial in 8 healthy volunteers confirmed safety profile; dosing initiated in fully enrolled second arm of 30 obese people with metabolic disorders; results expected in third quarter of 2021
Phase II
Abivax SA, of Paris ABX-464 Rev protein modulator Ulcerative colitis Gastroenterology published induction and long-term maintenance follow-up data from phase IIa study that confirmed clinical data and safety in moderate to severe disease
Allarity Therapeutics A/S, of Hørsholm, Denmark Ixempra (ixabepilone) Microtubulin inhibitor Breast cancer First of 60 participants enrolled in European trial to treat third-line metastatic disease
Dicerna Pharmaceuticals Inc., of Lexington, Mass., and Roche Holding AG, of Basel, Switzerland RG-6346 GalXC RNAi targeting HBsAg mRNA Chronic hepatitis B virus infection Started testing RG-6346 in a platform study; drug will be tested in combination with standard-of-care nucleos(t)ide therapy and in triple combinations with Pegasys (pegylated interferon alfa-2a, Roche), Roche’s CpAM inhibitor or Roche’s TLR7 agonist; primary endpoint is the percentage of participants with hepatitis B surface antigen loss 24 weeks after the end of the 48-week treatment period
Galapagos NV, of Mechelen, Belgium Jyseleca (filgotinib) JAK1 inhibitor Inflammatory bowel disease or rheumatic conditions In the Manta and Manta-Ray studies, 8.3% patients on placebo and 6.7% patients on filgotinib had a 50% or more decline in sperm concentration at week 13
Provention Bio Inc., of Red Bank, N.J. Teplizumab (PRV-031) Monoclonal antibody targeting CD3 Type 1 diabetes Data from the At-Risk study published in Science Translational Medicine showed teplizumab delayed the onset of clinical disease and insulin dependence by a median of 32.5 months; after 3.5 years of follow-up, 50% of patients taking teplizumab remained free of clinical type 1 diabetes compared to 22% of patients who received placebo (HR=0.457 p=0.01)
Phase III
Diamyd Medical AB, of Stockholm Diamyd Diabetes vaccine Newly diagnosed type 1 diabetes with HLA DR3-DQ2 haplotype Plans to run a study in 2021 with the co-primary endpoints of change in endogenous insulin production (measured as stimulated C-peptide) and change in HbA1c, both measured at 24 months from baseline
Eli Lilly and Co., of Indianapolis Tirzepatide Dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist Type 2 diabetes In the Surpass-2 study, 5-mg and 15-mg doses of tirzepatide reduced A1C by 2.09% and 2.46%, respectively, compared to a reduction of 1.86% for semaglutide; 5-mg and 15-mg doses of tirzepatide reduced body weight by 7.8 kg (17.2 lb., 8.5%) and 12.4 kg (27.3 lb., 13.1%), respectively, compared to 6.2 kg (13.7 lb., 6.7%) for semaglutide; 51% of patients taking the 15-mg dose achieved an A1C of less than 5.7%, compared to 20% of patients taking semaglutide
Mallinckrodt plc, of Dublin Terlipressin  Vasopressin analogue selective for V1 receptors Hepatorenal syndrome (HRS) type 1  Data from the Confirm study published in The New England Journal of Medicine showed 32% of patients who received terlipressin had verified reversal of HRS, compared to 17% of the placebo group (p=0.006); HRS reversal occurred in 39% of the terlipressin group and 18% of the placebo group (p<0.001); HRS reversal without renal replacement therapy by day 30 occurred in 34% of the terlipressin group and 17% of the placebo group (p=0.001); HRS reversal among patients with systemic inflammatory response syndrome occurred in 37% of the terlipressin group and 6% of the placebo group (p<0.001)
Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel Ofranergene obadenovec (VB-111) CD95 modulator; TNF receptor modulator Recurrent platinum-resistant ovarian cancer Based on data from the first 60 patients in the Oval study, authors of an article published in Gynecologic Oncology calculated the CA-125 GCIG response rate was 58% or higher in evaluable patients treated with the drug

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