Company Product Description Indication Status
Phase I
Allena Pharmaceuticals Inc., of Newton, Mass. ALLN-346 Oral urate-degrading enzyme Hyperuricemia in gout and chronic kidney disease Data from healthy volunteer study showed drug well-tolerated with no clinically significant safety signals and no dose-limiting toxicities in any cohort up to highest dose; assay of serum samples by ELISA immunoassay showed drug was not absorbed systemically, supporting that its mechanism of action appears to be restricted to gastrointestinal tract
Autolus Therapeutics plc, of London AUTO-6 Second-generation GD2-targeting CAR T candidate Solid tumors Data published in Science Translational Medicine show drug can induce rapid regression of bulky disease in solid tumor setting without inducing on-target, off-tumor toxicity, despite dose-dependent CAR T expansion; 3 of 6 patients showed evidence of transient CAR T-cell activity, including cytokine release syndrome, and regression of soft tissue and bone marrow disease activity
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda KPL-404 Monoclonal antibody inhibitor of CD40-CD40 ligand interaction Autoimmune disease Preliminary data from healthy volunteer study showed all 6 subjects dosed with 3 mg/kg I.V. had full receptor occupancy through day 29, which corresponded with complete suppression of the T-cell dependent antibody response to keyhole limpet hemocyanin through day 29; consistent dose relatedness shown in lower dose level cohorts, including 0.03 mg/kg, 0.3 mg/kg, 1 mg/kg I.V. and 1 mg/kg SC; data collection for higher dose cohorts, 10 mg/kg I.V. and 5 mg/kg SC, is ongoing
Marinomed Biotech AG, of Vienna Carragelose Iota-/kappa-carrageenan nasal spray COVID-19 Swansea University Medical School is planning clinical study testing prophylaxis in health care workers; objective is to assess efficacy in reducing the rate, severity and duration of infections
Neoimmunetech Inc., of Rockville, Md. NT-17 (efineptakin alfa) Long-acting human IL-7 COVID-19 First patient dosed in study enrolling adults with mild COVID-19
University of Oxford, in Oxford, U.K. Inhaled budesonide Corticosteroid COVID-19 Drug to be included in Principle trial testing treatments in patients ages 50 and older
Valirx plc, of London VAL-201 Short peptide designed to prevent binding of androgen receptor to SRC kinase Prostate cancer Full dataset from phase I/II study in locally advanced or metastatic disease includes PSA results showing 2 patients with decrease in PSA during treatment period, 1 of which maintained decrease throughout trial; analysis of doubling time of patients shows statistically significant (p<0.05, two-tailed Wilcoxon Signed-Rank test for paired samples) increase in PSA doubling time comparing pretreatment doubling time to post-treatment doubling time; tumor response data demonstrate patients did not have increase in metastatic lesions during treatment period, and that metastatic lesions already present did not grow at all in 2 of 3 patients
Phase II
Abivax SAS, of Paris ABX-464 Rev protein modulator Moderate to severe ulcerative colitis Completed recruitment in phase IIb induction study; top-line results expected in second quarter of 2021
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil)  NMDA receptor antagonist COVID-19 Final patient enrolled in phase IIb/III trial, for total of 168; interim data from 75 patients from day 15 expected in first week of December 2020
Azurrx Biopharma Inc., of Delray Beach, Fla. MS-1819 Recombinant lipase enzyme; derived from Yarrowia lipolytica yeast lipase Severe exocrine pancreatic insufficiency in cystic fibrosis First 2 patients dosed in Turkey in study testing combination with current standard of care, porcine-derived pancreatic enzyme replacement therapy
CTI Biopharma Corp., of Seattle Pacritinib JAK2, FLT3, IRAK1 and CSF1R kinase inhibitor Advanced myelofibrosis Data published in Blood Advances showed 200 mg twice daily (BID) had favorable benefit-risk profile; spleen volume response (SVR) rates were highest among patients treated with pacritinib 200 mg BID who had a baseline platelet count of less than 50 x 109/L; data supported selection of 200 mg BID for use in ongoing phase III Pacifica study
Curevac NV, of Tübingen, Germany Cvncov (SARS-CoV-2 mRNA vaccine) COVID-19 spike glycoprotein modulator COVID-19 infection Contingent on regulatory clearance, pivotal phase IIb/III trial set to test 35,000+ individuals using 12-µg dose, stratified according to age (>18 and >60 years old), initially at sites in Europe and South America; interim analysis could be conducted in first quarter of 2021
Edesa Biotech Inc., of Toronto EB-05 (NI-0101) TLR4 antagonist COVID-19 infection First participant hospitalized with infection enrolled in phase II/III study to assess monoclonal antibody's effect in regulating immune response associated with acute respiratory distress syndrome; study expected to enroll about 316 people in phase II portion
Galecto Inc., of Copenhagen GB-0139 Galectin-3 inhibitor Idiopathic pulmonary fibrosis Phase IIa findings, published in European Respiratory Journal, showed plasma levels of disease biomarkers, including YKL-40, CCL-18,PDGF-B, PAI-1 and Galectin-3, were reduced in dose-dependent fashion from baseline with statistical significance vs. placebo, with strongest effects in 10-mg dose group
Hookipa Pharma Inc., of New York HB-101 Non-replicating arenavirus vaccine Cytomegalovirus infection Early interim data showed CMV-negative kidney transplant recipients (n=8) vaccinated with 3 doses had 48% reduction in CMV viremia, 42% reduction in use of antiviral therapy and no CMV disease
Imago Biosciences Inc., of South San Francisco Bomedemstat (IMG-7289)  Lysine specific histone demethylase 1 inhibitor Myelofibrosis Phase IIb trial in people with advanced disease expanded enrollment into and dosed first participant in Hong Kong
Istari Oncology Inc., of Durham, N.C. PVS-RIPO  Viral immunotherapy based on live attenuated Sabin type 1 polio vaccine Recurrent glioblastoma First participant dosed in Luminos-101 combination trial with checkpoint inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.)
Novavax Inc., of Gaithersburg, Md. NVX‑CoV2373 COVID-19 spike glycoprotein modulator COVID-19 infection Phase IIb trial in South Africa fully enrolled 4,422 people, including 245 medically stable, HIV-positive participants; interim efficacy data expected as soon as early first quarter of 2021
Verastem Oncology Inc., of Boston VS-6766 + defactinib RAF/MEK inhibitor + FAK inhibitor Ovarian cancer Adaptive, 2-part, registration-directed trial initiated in people with recurrent low-grade serous disease; first part will determine optimal regimen of VS-6766 monotherapy or combination with defactinib, with determination of which regimen to advance into expansion phase based on objective response rate data
Phase III
Agile Therapeutics Inc., of Princeton, N.J. Twirla (ethinylestradiol + levonorgestrel) Estrogen receptor agonist + progesterone receptor agonist Female contraception Data from Secure study published online in Contraception
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat Kallikrein inhibitor Hereditary angioedema Allergy published data from Apex-J trial in Japan assessing prevention of HAE attacks
Citius Pharmaceuticals Inc., of Cranford, N.J. Mino-Lok (minocycline + edetate + ethyl alcohol antibiotic lock solution) 30S ribosomal protein inhibitor Staphylococcus aureus infection Boston Analytical study showed 3 components of study drug (30 mg/mL EDTA, 19.5% ethanol and 1 mg/mL minocycline) were superior to 30 mg/mL EDTA + 19.5% ethanol against 2 strains of S. aureus at exposure time of 60 minutes (p=0.0598) 
Deciphera Pharmaceuticals LLC, of Waltham, Mass. Qinlock (ripretinib) KIT/PDGFRA tyrosine kinase inhibitor Gastrointestinal stromal tumor Intrigue study completed target enrollment of about 426 people with second-line disease; primary efficacy endpoint is median progression-free survival vs. sunitinib; top-line results due in second half of 2021
Fergene Inc., of Cambridge, Mass. Nadofaragene firadenovec IFNA2 gene stimulator Bladder cancer The Lancet Oncology published data from study that met primary endpoint in 157 people with high-grade, Bacillus Calmette-Guérin unresponsive non-muscle invasive disease
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID-19 spike glycoprotein modulator COVID-19 infection Primary efficacy analysis conducted on 196 cases confirmed efficacy seen at first interim analysis, indicating efficacy of 94.1%, consistent across age, race and ethnicity and gender; 100% efficacy seen in severe infection
Montreal Heart Institute, of Montreal Colchicine Microtubule polymerization inhibitor COVID-19 infection After second interim analysis, independent data monitoring committee recommended Colcorona trial continue to recruit non-hospitalized adults with COVID-19 to assess efficacy in reducing complications
Novavax Inc., of Gaithersburg, Md. NVX‑CoV2373 COVID-19 spike glycoprotein modulator COVID-19 infection Pivotal trial in U.K. fully enrolled 15,000 participants, with interim efficacy data expected as soon as early first quarter of 2021
PTC Therapeutics Inc., of South Plainfield, N.J. Vatiquinone (PTC-743) 15-lipoxygenase inhibitor Friedreich ataxia Registration-directed Move-FA study initiated; 18-month trial expected to enroll about 110 children and young adults, with primary endpoint of change from baseline in modified FA rating scale and key secondary endpoints assessing ambulation and activities of daily living


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