Company Product Description Indication Status
Phase I
Adagene Inc., of San Francisco ADG-116 Fully human, anti-CTLA4 monoclonal antibody Advanced/metastatic solid tumors Interim dose-escalation data from Australia trial showed drug well-tolerated; dose-dependent change in CD8+ and CD4+ TEM/Treg ratios, pharmacodynamic biomarkers indicating immune activation, was observed; clinical proof of mechanism consistent with preclinical observations of potency
Celldex Therapeutics Inc., of Hampton, N.J. CDX-0159 Monoclonal antibody binding KIT receptor Antihistamine-refractory cold contact urticaria and symptomatic dermographism Data from ongoing study showed, in all patients treated and assessed for at least 15 days after treatment (n=10), 8 of 10 (80%) experienced a complete response to provocation testing post-treatment and 1 patient experienced a partial response; based on data, the study will be expanded to include 10 patients with cholinergic urticaria
Cohbar Inc., of Menlo Park, Calif. CB-4211 Mitochondria-based therapeutic Nonalcoholic steatohepatitis and obesity Completed enrollment in phase Ib stage; top-line data expected by end of second quarter of 2021
Cynata Therapeutics Ltd., of Melbourne, Australia Cymerus MSCs Mesenchymal stem cell  COVID-19; respiratory failure Received ethics approval to expand recruitment in phase I/II Mend study to patients in intensive care with respiratory failure from other causes beyond COVID-19
Gritstone Oncology Inc., of Emeryville, Calif. COVID-19 vaccine candidate mRNA vaccine COVID-19 First person dosed under Coral program; both heterologous and homologous prime-boost vaccinations of the adenoviral vector and/or self-amplifying mRNA vector expressing either SARS-CoV-2 spike alone or spike plus additional SARS-CoV-2 T-cell epitopes are being studied in a parallel design
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Trike NK cell engager High-risk myelodysplastic syndromes and relapsed/refractory acute myeloid leukemia Interim phase I/II data showed up to 63.7% reduction in bone marrow blast levels; treatment restored patient’s endogenous NK cell function, proliferation and immune surveillance; no cytokine release syndrome observed; 3 of last 5 patients treated responded to therapy
Hutchison China Meditech Ltd., of Hong Kong HMPL-306 Dual IDH1/2 inhibitor Advanced solid tumors; hematological malignancies Trials initiated in U.S. and Europe both in solid tumors and in relapsed/refractory or resistant hematological malignancies that harbor IDH1 or IDH2 mutations
Immunitybio Inc., of Culver City, Calif. Human adenovirus immunotherapy vaccine hAd5 vector-delivered tumor-associated antigen Metastatic prostate cancer Journal of Immunotherapy of Cancer published data showing 1 participant achieved partial response and 5 had confirmed stable disease for > 6 months; median progression-free survival was 22 weeks and median overall survival was not reached
Phase II
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Zavegepant CGRP receptor antagonist Migraine First of about 2,900 participants enrolled in phase II/III trial assessing 100-mg and 200-mg doses for preventive treatment
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of Cambridge, U.K. Enhertu (trastuzumab deruxtecan) HER2-directed antibody-drug conjugate Non-small-cell lung cancer First of about 150 participants with HER2-mutated metastatic disease that recurred or progressed during or after at least 1 platinum-containing regimen dosed in Destiny-Lung02; primary endpoint is objective response rate
Destiny Pharma plc, of Brighton, U.K. XF-73 (exeporfinium chloride nasal gel) Antibacterial Staphylococcus aureus infection Phase IIb study met primary endpoint of reduction in mean nasal burden of S. aureus in people undergoing open heart surgery by 2.5 log (CFU/ml) in 24 hours prior to surgery in micro intent to treat population (p<0.0001)
Eli Lilly and Co., of Indianapolis, Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London Bamlanivimab + VIR-7831 Angiotensin converting enzyme 2 inhibitor + COVID-19 spike glycoprotein modulator COVID-19 infection Blaze-4 trial in low-risk adults with mild to moderate infection met primary endpoint, showing 70% (p<0.001) relative reduction in persistently high viral load (> 5.27; cycle threshold value < 27.5) at day 7 vs. placebo
Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan FP-025 MMP-12 inhibitor COVID-19 infection First of about 99 adults with severe to critical infection with associated acute respiratory distress syndrome dosed in phase II portion of phase II/III trial; primary outcome measures include proportion alive and not requiring non-invasive or invasive ventilation at day 28; interim analysis at end of phase II portion expected in fourth quarter of 2021
Gradalis Inc., of Dallas Vigil (gemogenovatucel-T) Bi-shRNA furin plus DNA expressing human GM-CSF Ovarian cancer Gynecologic Oncology published subgroup analysis of stage III/IV newly diagnosed patients with BRCA wild-type profile and homologous recombination proficiency showing improved overall survival (p=0.019) and recurrence-free survival (p=0.007) vs. placebo (n=20)
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. IONIS-PKK-LRx KLKB1 inhibitor; plasma kallikrein inhibitor Hereditary angioedema Study showed mean reduction of 90% in number of monthly attacks in weeks 1 to 17 (p <0.001) and of 97% in number of monthly attacks in weeks 5 to 17 (p=0.003); in weeks 5 to 17, 92% treated with study therapy were attack-free vs. 0% for placebo group (p <0.001)
Medsenic SAS, of Strasbourg, France Arscimed (arsenic trioxide) Immunosuppressant Graft-vs.-host disease At 6-month follow-up, primary endpoint of complete or partial disease remission was met in 15/21 patients for 75% clinical efficacy rate; sustained response seen in all participants at 12 months post-treatment
Neurorx Inc., of Radnor, Pa. Zyesami (intravenous aviptadil)  VIP receptor agonist COVID-19 infection Phase II/III trial met primary endpoint of recovery from respiratory failure at days 28 (p=0.014) and 60 (p=0.013) and showed survival benefit (p=<0.001) after controlling for ventilation status and treatment site; immediate EUA application to FDA expected, followed by NDA submission
Orphazyme A/S, of Copenhagen Arimoclomol Chaperonin stimulator; Hsp70 stimulator Inclusion body myositis  Phase II/III trial missed primary endpoint of effect on disease progression measured by inclusion body myositis functional rating scale and secondary endpoints
Oryzon Genomics SA, of Madrid Vafidemstat LSD1 inhibitor Borderline personality disorder First of 156 participants enrolled in adaptive design phase IIb Portico trial; co-primary endpoints are reduction of aggression and agitation and overall improvement in BPD
Shanghai Henlius Biotech Inc., of Shanghai HLX-10 PD-1 inhibitor Solid tumors Study in people with unresectable/metastatic microsatellite instability-high or mismatch repair-deficient disease met primary endpoint of objective response rate
Phase III
Adamas Pharmaceuticals Inc., of Emeryville, Calif. Gocovri (amantadine) Reduces the amount of glutamate hyperactivity Parkinson’s disease Data published in Frontiers in Neurology showed Gocovri increased the "on" time without dyskinesia by 2.9 hours compared to placebo 
Humanigen Inc., of Burlingame, Calif. Lenzilumab  Monoclonal antibody targeting granulocyte macrophage-colony stimulating factor Hospitalized COVID-19 Lenzilumab plus standard of care improved the likelihood of survival without need for invasive mechanical ventilation by 54% compared to standard of care alone (p=0.0365); mortality was 9.6% for lenzilumab and 13.9% for placebo (p=0.2287)
Uniqure NV, of Amsterdam Etranacogene dezaparvovec (AMT-061)  F9 gene stimulator; factor IX modulator Hemophilia B Independent investigation of hepatocellular carcinoma in participant in pivotal Hope-B trial showed no evidence that AAV vector delivered in study played pathogenic role; participant had abnormalities on chromosomes 1 and 8, commonly associated with HCC, and mutations in other potentially oncogenic genes along with multiple risk factors for HCC; data shared with FDA with respect to status of clinical hold
Phase IV
Bayer AG, of Leverkusen, Germany, and Merck & Co. Inc., of Kenilworth, N.J. Adempas (riociguat)  Soluble guanylate cyclase stimulator Pulmonary arterial hypertension Data from the Replace study published in The Lancet Respiratory Medicine showed 41% of patients treated with Adempas achieved the composite primary endpoint of clinical improvement in the absence of clinical worsening, compared with 20% of patients who received  phosphodiesterase-5 inhibitor therapy (p=0.0007)

Notes

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