Company Product Description Indication Status
Phase I
Lineage Cell Therapeutics Inc., of Carlsbad, Calif., and Cancer Research UK, of London VAC-2 Allogeneic, off-the-shelf cancer vaccine Non-small-cell lung cancer Preliminary results from ongoing study showed potent induction of immune responses in all patients dosed to date, with high levels of peripheral antigen-specific immunogenicity observed at multiple time points and confirmed by multimer staining; based on those findings and following completion of study, Lineage will seek to evaluate VAC-2 in combination regimens
Moleculin Biotech Inc., of Houston WP-1066 JAK tyrosine kinase inhibitor; C-myc binding protein inhibitor; STAT-3 inhibitor; hypoxia inducible factor-1 alpha inhibitor Glioblastoma Additional preliminary data support progression of trial to fourth and final dose-escalation cohort; 3 patients completed treatment in third cohort at dose level of 8 mg/kg with no adverse events
Nanobiotix SA, of Paris NBTXR-3 Designed to destroy tumors when activated by radiotherapy Pancreatic cancer First patient injected; objectives are the determination of dose-limiting toxicity, the maximum tolerated dose and the recommended phase II dose
Nasus Pharma, of Tel Aviv, Israel Taffix  Nasal powder inhaler COVID-19 prophylaxis Rate of infection was 10% for non-users compared to 2.4% for participants that were assigned to used Taffix (p=0.37)
Targovax ASA, of Oslo, Norway ONCOS-102 GM-CSF receptor agonist Colorectal and platinum-resistant ovarian cancer Colorectal cancer cohort in part 1 of phase I/II trial testing drug plus durvalumab met predefined efficacy threshold of patients without progression at end of week 24; second part of colorectal expansion cohort is open for recruitment
Uniqure NV, of Amsterdam AMT-130 HTT gene inhibitor Huntington’s disease 2 additional patient procedures have been completed in phase I/II trial 
Vaxart Inc., of South San Francisco VXA-CoV2-1 Oral tablet vaccine candidate COVID-19 Dosed first subject; trial to test safety and immunogenicity of 2 doses in up to 48 healthy adult volunteers ages 18 to 54; enrollment is expected to be completed by early November 2020
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) NMDA receptor antagonist Idiopathic pulmonary fibrosis and chronic cough Reached 25% enrollment
Apogenix AG, of Heidelberg, Germany Asunercept Extracellular domain of CD95 receptor and Fc domain of IgG1 antibody fusion protein  COVID-19 First patient enrolled in Asunctis trial to test 3 doses in 4 treatment arms vs. standard of care in about 400 patients with severe disease; enrolling patients in Spain and Russia
Cytrx Corp., of Los Angeles Aldoxorubicin  Doxorubicin with an acid-sensitive linker Locally advanced or metastatic pancreatic cancer Immunity Bio Inc. and Nantkwest Inc. added aldoxorubicin to third cohort of ongoing combination immunotherapy study; cohort will involve patients who have failed all approved standards of care
Evelo Biosciences Inc., of Cambridge, Mass. EDP-1815 Oral biologic; strain of Prevotella histicola Mild to moderate psoriasis Dosed first patients
Gossamer Bio Inc., of San Diego GB-001 G-protein coupled receptor-44 antagonist; PGD2 antagonist Moderate to severe eosinophilic asthma Phase IIb Leda study did not meet primary endpoint of asthma worsening; however, consistent numeric reductions ranging from 32% to 35% were observed across all 3 GB-001 groups; statistically significant improvements in key secondary endpoint of time to first asthma worsening of 28% and 30% observed for 20-mg and 60-mg doses, respectively; 23% improvement observed in 40-mg group
Gossamer Bio Inc., of San Diego GB-001 G-protein coupled receptor-44 antagonist; PGD2 antagonist Chronic rhinosinusitis Titan study did not meet primary or secondary endpoints
Humanigen Inc., of Burlingame, Calif., Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Abbvie Inc., of North Chicago Lenzilumab and risankizumab Monoclonal antibodies targeting granulocyte-macrophage colony stimulating factor and interleukin 23A  COVID-19 The U.S. National Institute of Allergy and Infectious Diseases launched the Big Effect Trial testing the drugs in combination with remdesivir
Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holding AG, of Geneva RLF-100 (aviptadil) Formulation of vasoactive intestinal polypeptide Critical COVID-19 and respiratory failure Survival rate beyond 60 days was 81% for patients taking RLF-100 and 17% for control patients treated with standard of care in the same setting
Nicox SA, of Sophia Antipolis, France NCX-4251 0.1% Ophthalmic suspension of fluticasone propionate nanocrystals Blepharitis Upcoming Mississippi trial will focus on acute exacerbations of blepharitis; if successful, trial could be first of 2 pivotal studies needed to support NDA; study set to start in December 2020, with top-line data expected in the fourth quarter of 2021
Phase III
Johnson & Johnson, of New Brunswick, N.J. JNJ-78436735 Vaccine COVID-19 Temporarily paused further dosing in vaccine trials, including Ensemble trial, due to unexplained illness in a study participant; participant’s illness is being reviewed and evaluated by data safety monitoring board 
Kyowa Kirin International plc, of Galashiels, U.K. Poteligeo (mogamulizumab) Defucosylated anti-CC chemokine receptor 4 antibody Mycosis fungoides and Sézary syndrome Post-hoc analysis of the Mavoric study showed progression-free survival was significantly greater for mogamulizumab compared to vorinostat in patients with higher levels of blood involvement (B1 and B2 blood classifications); patients with B2 classification also has significantly greater overall response rates for
mogamulizumab compared to vorinostat; in patients with B1 or B2, time to next treatment was 13.7 months for mogamulizumab  and 3.3 months for vorinostat (p<0.0001)
Mesoblast Ltd., of Melbourne, Australia Remestemcel-L Culture-expanded mesenchymal stem cells Acute respiratory distress syndrome due to COVID-19 Study testing drug on top of maximal care in ventilator-dependent patients surpassed 50% enrollment
Nabriva Therapeutics plc, of Dublin Xenleta (lefamulin) Semi-synthetic pleuromutilin antibiotic Community-acquired bacterial pneumonia Post-hoc analysis from 1,289 patients in Leap 1 and 2 trials by age showed consistently high efficacy and similar safety and tolerability profiles across all patient groups, including adults over 65 who are at higher risk of morbidity and mortality
Outlook Therapeutics Inc., of Monmouth Junction, N.J. ONS-5010/Lytenava (ophthalmic formulation of bevacizumab-vikg) VEGF inhibitor Wet age-related macular degeneration Initiated enrollment of first patients in its supplemental open-label safety study to support BLA filing
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) Monoclonal antibody targeting interleukin-4 Uncontrolled moderate to severe asthma Met primary and all key secondary endpoints in children, ages 6-11; in broad type 2 inflammatory asthma population, defined as having elevated eosinophils or elevated fractional exhaled nitric oxide, addition to standard of care significantly reduced asthma attacks and improved lung function as early as 2 weeks after first dose vs. standard of care alone
Shionogi & Co. Ltd., of Osaka, Japan Cefiderocol Cephalosporin antibiotic Hospital-acquired, ventilator-associated, or health care-associated pneumonia caused by gram-negative bacteria Apeks-NP trial met primary endpoint of noninferiority in all-cause mortality (ACM) at day 14 with 12.4% for cefiderocol arm (18/145) and 11.6% for meropenem arm (17/146; adjusted treatment difference 0.8%) in modified intention-to treat-population; data published in The Lancet Infectious Diseases
Shionogi & Co. Ltd., of Osaka, Japan Cefiderocol Cephalosporin antibiotic Carbapenem-resistant infections Credible-CR trial showed microbiological outcomes were generally similar between cefiderocol and best available treatment (BAT), except for metallo-beta-lactamases infections where cefiderocol was substantially better (75% and 44% vs. 29% and 14%); observed mortality difference between treatment arms in the subset of patients with Acinetobacter spp. infections, likely linked to imbalances in risk factors at baseline; no mortality difference observed in P. aeruginosa or Enterobacterales without Acinetobacter spp. co-infection; data published in The Lancet Infectious Diseases


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