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