Company Product Description Indication Status
Phase I
Bolt Biotherapeutics Inc., of Redwood City, Calif. BDC-1001 Immune-stimulating antibody conjugate   HER2-expressed solid tumors Patient dosing begun in an open-label, dose-escalation and dose-expansion study of BDC-1001 monotherapy
Decibel Therapeutics Inc., of Boston DB-020 Sodium thiosulfate Cisplatin-induced hearing loss Phase Ia results demonstrating safety and tolerability in healthy volunteers published in Investigational New Drugs; administration resulted in only nominal systemic increases of sodium thiosulfate, suggesting that DB-020 should have no impact on the efficacy of cisplatin; phase Ib trial in patients initiated 
Imcheck Therapeutics SAS, of Marseille, France ICT-01 Gamma delta T cell-activating monoclonal antibody Solid and hematologic tumors Dosed first patient in a phase I/IIa Eviction 2-part, open-label trial to assess the safety, tolerability and activity of ICT-01 as monotherapy and in combination with an immune checkpoint inhibitor in patients with advanced, relapsed/refractory disease
Inflazome Ltd., of Dublin Inzomelid  Small-molecule inhibitor of NLRP3 inflammasome Autoinflammatory disease Completed study, with results supporting progression into phase II
Tolero Pharmaceuticals Inc., of Salt Lake City Dubermatinib (TP-0903) AXL receptor tyrosine kinase inhibitor Acute myeloid leukemia Joined The Leukemia & Lymphoma Society in Beat AML master clinical trial for newly diagnosed patients 60 or older; dubermatinib selected for a new arm in the trial for patients with TP53 mutations and/or complex karyotype; primary objectives are to determine the safety and maximum tolerated dose and evaluate the composite complete response rate
Uniqure NV, of Amsterdam, the Netherlands AMT-130 AAV-based gene therapy Huntington’s disease First 2 patients in phase I/II trial enrolled, but due to the expanding impact of the COVID-19 pandemic, their procedures have been temporarily postponed
Phase II
Healios K.K., of Tokyo HLCM-051 Stem cells Acute respiratory distress syndrome Continues to make enrollment progress in Japan; in One-Bridge study, inclusion criteria allow patients who develop pneumonia-induced ARDS caused by COVID-19
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. VGX-3100 DNA medicine Anal dysplasia Interim results from open-label study showed VGX-3100 to be safe and effective; of 20 subjects who had results at the time of data review, 50% showed clearance of HPV-16/18-associated precancerous lesions and 75% demonstrated an overall decrease in the number of lesions 6 months after the start of treatment; no cases of anal cancer have been observed in the trial
Oncopeptides AB, of Stockholm Melphalan flufenamide Peptide-drug conjugate  Multiple myeloma Drug attained an ORR of 30% in patients with relapsed refractory MM, as assessed by the independent review committee, in the intent-to-treat analysis of the pivotal open-label Horizon study
Qurient Co. Ltd., of Seongnam, South Korea Telacebec Ubiquinol cytochrome C reductase 14 kDa inhibitor Tuberculosis Data from trial, now published in The New England Journal of Medicine, support continued development of telacebec, which could become the first universal regimen to overcome tuberculosis regardless of drug resistance status
Uniqure NV, of Amsterdam, the Netherlands AMT-130 HTT gene inhibitor Huntington's chorea First 2 patients are enrolled, but procedures have been temporarily postponed due to the expanding impact of the COVID-19 pandemic
Phase III
Bristol Myers Squibb Co., of Princeton, N.J., and and Acceleron Pharma Inc., of Cambridge, Mass. Reblozyl (luspatercept-aamt) TGF beta receptor antagonist; TGF beta ligand inhibitor; GDF modulator Anemia; beta-thalassemia Published results from pivotal Believe trial in The New England Journal of Medicine following the therapy's November 2019 approval
Ipsen SA, of Paris Palovarotene  Retinoic acid receptor gamma agonist  Fibrodysplasia ossificans progressiva Paused dosing in the global Move trial as well as the ongoing phase II extension studies based on results of a futility analysis as part of the prespecified interim analysis; reinitiating dosing in patients 14 years and older
Ironwood Pharmaceuticals Inc., of Boston IW-3718 (colesevelam) Gastric-retentive formulation of bile acid sequestrant colesevelam Gastroesophageal reflux No longer guiding for top-line data in the second half of 2020 due to certain clinical sites suspending enrollment and lower patient participation; trial is 70% enrolled
Otsuka Pharmaceutical Co. Ltd., of Tokyo, and Medimetriks Pharmaceuticals Inc., of Fairfield, N.J. Difamilast PDE4 inhibitor Atopic dermatitis In both the adult and pediatric trials, IGA success rates were higher in the active drug treatment groups than in the vehicle groups; no major adverse reactions
Uniqure NV, of Amsterdam, the Netherlands Etranacogene dezaparvovec (AMT-061) AAV5-based gene therapy Hemophilia B Achieved targeted dosing of patients in the Hope-B pivotal trial 
Vascular Biogenics Ltd. (VBL Therapeutics), of Tel Aviv, Israel VB-111 (ofranergene obadenovec) Dual CD95/TNF receptor modulator Ovarian  cancer Data safety monitoring committee reviewed unblinded data and determined study has met the interim prespecified criterion, of an absolute percentage advantage of 10% or higher in CA-125 response in the VB-111 treated arm vs. control; recommended study proceed without modification

Notes

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