Company Product Description Indication Status
Phase I
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Trike Single-chain, trispecific scFv recombinant fusion protein conjugate  High-risk myelodysplastic syndromes and refractory/relapsed acute myeloid leukemia Enrolled patient 10 in phase I/II trial; data from first 9 patients show up to 63.7% reduction in bone marrow blast levels, with treatment restoring endogenous NK cell function, proliferation and immune surveillance; no cytokine release syndrome observed
Immunitybio Inc., of Culver City, Calif. COVID-19 vaccine candidate Single prime hAd5 vaccine COVID-19 Initial data indicate single subcutaneous injection in healthy participants stimulates generation of T cells reactive to the spike (S) and nucleocapsid (N) protein antigens delivered by vaccine; 14-16 days after single dose, mean level of T cells were 10 times higher for N specific T cells; by day 21, both S and N T-cell responses achieved levels 10 times higher vs. pre-vaccination levels; data published on preprint server medRxiv
Portage Biotech Inc., of Westport, Conn. PORT-3 Nanoparticle co-formulation of invariant natural killer T-cell agonist IMM60 and NY-ESO-1 immunogenic peptides NY-ESO-1-positive solid tumors First patient dosed in phase I portion of Precious-01 open-label, dose-escalation and expansion trial
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) NMDA receptor antagonist COVID-19 Completed initial review of full dataset following completion of phase IIb part of phase IIb/III trial; plans to file end-of-phase II meeting request with FDA
Beigene Co. Ltd., of Cambridge, Mass. Brukinsa (zanubrutinib) Small-molecule BTK inhibitor COVID-19 Study in patients hospitalized with respiratory symptoms, requiring supplemental oxygen without mechanical ventilation, did not meet the co-primary efficacy endpoints of respiratory failure-free survival or reduction in days on oxygen vs. placebo; no new or additional safety signals were identified
Connect Biopharma Holdings Ltd., of San Diego CBP-201 IL-4Rα monoclonal antibody Moderate to severe atopic dermatitis Completed full enrollment
Galderma SA, of Lausanne, Switzerland Nemolizumab Humanized monoclonal antibody directed against IL-31 receptor Moderate to severe atopic dermatitis Post-hoc analysis of phase IIb trial, published in the Journal of the European Academy of Dermatology and Venereology, show drug led to rapid and sustained improvements over placebo in itch (-22.8% vs. -12.3%; p=0.005), sleep (-26.6% vs. -9%; p<0.001) and skin lesions (75% EASI observed at week 16 in 50% of nemolizumab patients vs. 15.9% placebo; p<0.001) in adults with uncontrolled disease 
Laurent Pharmaceuticals Inc., of Montreal LAU-7b (fenretinide) Nuclear factor kappa B gene modulator COVID-19 infection Data safety monitoring board unanimously recommended Resolution study in people hospitalized with infection continue without modification following pre-scheduled safety review and interim futility analysis of unblinded efficacy data from first 102 participants; top-line data expected in second half of 2021
Laurent Pharmaceuticals Inc., of Montreal LAU-7b (fenretinide) Nuclear factor kappa B gene modulator Cystic fibrosis Applaud study fully enrolled 166 adults; top-line data expected in second half of 2021
Ocuphire Pharma Inc., of Farmington Hills, Mich. APX-3330 Reduction-oxidation effector factor-1 inhibitor Diabetic retinopathy First participant screened in Zeta-1 trial in those with moderately severe to severe non-proliferative and mild proliferative disease; primary endpoint is percentage of participants with ≥ 2 step improvement on Diabetic Retinopathy Severity Scale score
Secura Bio Inc., of Summerlin, Nev. Copiktra (duvelisib) Phosphoinositide-3 kinase delta/gamma inhibitor Peripheral T-cell lymphoma Primo study fully enrolled 101 participants with relapsed/refractory disease; interim data expected by year-end 2021
Phase III
Aurinia Pharmaceuticals Inc., of Victoria, British Columbia Lupkynis (voclosporin) Calcineurin inhibitor Lupus nephritis Pooled data on 532 participants in Aura-LV and Aurora 1 studies showed Lupkynis in combination with standard-of-care (SOC) mycophenolate mofetil and low-dose corticosteroids led to treatment benefits across biopsy class subgroups vs. SOC alone, with odds ratio of 4.26 for pure class III (p=0.0054), 2.59 for pure class IV (p=0.0005), 1.5 for pure class V (p=0.4090) and 2.68 for mixed class III/IV and V (p=0.0166)
Bristol Myers Squibb Co., of New York Opdivo (nivolumab) + Yervoy (ipilimumab)  PD-1 inhibitor + CTLA4 inhibitor Esophageal squamous cell carcinoma In CheckMate -648 trial in people with unresectable advanced/metastatic disease, combination met primary and secondary endpoints, showing statistically significant improvement in overall survival (OS) in tumors expressing PD-L1 and in randomized population but missed co-primary endpoint of progression-free survival (PFS) by blinded independent central review in tumors expressing PD-L1; Opdivo + chemotherapy also showed statistically significant benefit for primary and secondary endpoints of OS in tumors expressing PD-L1 and in randomized population at prespecified interim analysis and showed statistically significant improvement in PFS
Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. Olumiant (baricitinib) JAK1/JAK2 inhibitor COVID-19 infection COV-Barrier trial of study drug + standard of care (SOC) vs. placebo + SOC in 1,525 people hospitalized with infection missed statistical significance (p=0.18) on primary endpoint of difference in proportion who progressed to non-invasive or invasive mechanical ventilation or death by day 28; trial showed reduction (nominal p=0.0018) in death from any cause by day 28
Global Blood Therapeutics Inc., of South San Francisco Oxbryta (voxelotor) Hemoglobin alpha subunit modulator Sickle cell disease The Lancet Haematology published analysis of 72-week data from Hope study showing sustained improvement in hemoglobin levels, reduction in hemolysis and improved overall health status in treated participants; about 90% achieved hemoglobin improvement of >1 g/dL from baseline at 1 or more time points vs. placebo (25%) and about 59% achieved hemoglobin increase of >2 g/dL and 20% achieved >3 g/dL at 1 or more time points vs. about 3% and none for placebo, respectively
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) PD-1 inhibitor  Renal cell carcinoma Pivotal Keynote-564 trial met primary endpoint of statistically significant disease-free survival vs. placebo as adjuvant treatment following nephrectomy with or without resection of metastatic lesions

Notes

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