Company Product Description Indication Status
Phase I
Equillium Inc., of La Jolla, Calif. Itolizumab  Targets the CD6-ALCAM pathway Acute graft-vs.-host disease Of the 3 patients treated in the third cohort of the Equate study, 2 patients had a complete response and 1 had a very good partial response; independent data monitoring committee recommended expanding cohort 3
Homology Medicines Inc., of Bedford, Mass. HMI-102  Gene therapy expressing PAH Phenylketonuria  Well-tolerated and resulted in marked reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Tyr) ratio at 2 doses; Homology is progressing to the randomized, concurrently controlled expansion phase of the trial, which has the potential to be converted to a registrational trial
Seres Therapeutics Inc., of Cambridge, Mass. SER-301 Microbiome therapeutic Mild-to-moderate ulcerative colitis Treated first of approximately 65 patients in the study; safety and pharmacokinetics (PK) will be measured in first 15 patients; the placebo-controlled cohort of the final 50 patients will measure safety and PK, clinical remission and other measures of drug pharmacology and efficacy
Viela Bio Inc., of Gaithersburg, Md. VIB-7734  Monoclonal antibody targeting plasmacytoid dendritic cells Cutaneous lupus erythematosus Median change in the type I interferons signature score in peripheral blood at month 3 was -54% for the 50-mg dose, -83% for the 150-mg dose and +8% for placebo; 75% of patients taking the 150-mg dose had a ≥50% improvement in CLASI-A compared to 29% of patients taking placebo
Phase II
Anavex Life Sciences Corp., of New York Anavex 2-73 (blarcamesine)  Oral small-molecule activator of the sigma-1 receptor Parkinson’s disease dementia (PDD) Well-tolerated in oral doses up to 50 mg once daily; results showed clinically meaningful, dose-dependent and statistically significant improvements in the Cognitive Drug Research computerized assessment system analysis; study validated the precision medicine approach of targeting SIGMAR1 as a genetic biomarker of response, confirming it acts through SIGMAR1 activation; results support continued development in PDD / PD as well as the currently ongoing phase 2 and phase 2/3 clinical studies in Rett syndrome and Alzheimer’s disease
Assembly Biosciences Inc., of South San Francisco Vebicorvir  Core inhibitor Chronic HBV infection After withdraw from vebicorvir plus a nucleos(t)ide analogue reverse transcriptase inhibitor, 39 of 41 patients relapsed
Puma Biotechnology Inc., of Los Angeles Neratinib  Kinase inhibitor Metastatic non-small-cell lung cancer with EGFR exon 18 mutations after treatment with an EGFR targeted tyrosine kinase inhibitor  In 10 evaluable patients there were 6 partial responses; clinical benefit was 80%; enrollment continues
Phase III
Boehringer Ingelheim GmbH, of Ingelheim, Germany Ofev (nintedanib)  Tyrosine kinase inhibitor Interstitial lung disease  Interim analysis showed 347 patients with systemic sclerosis-associated disease in Senscis-On extension study who received Ofev had average change in FVC of -51.3 mL from baseline to week 52, similar to change from baseline to week 52 of -42.7 mL for those who received drug in phase III Senscis trial 
Humanigen Inc., of Burlingame, Calif. Lenzilumab  GM-CSF ligand inhibitor  Hospitalized COVID-19 Interim data showed 37% more recoveries in the lenzilumab arm compared to the placebo arm
Janssen Pharmaceutical Inc., of Spring House, Pa., a unit of New Brunswick, N.J.-based Johnson & Johnson Tremfya (guselkumab) Monoclonal antibody targeting the p19 subunit of IL-23 Psoriatic arthritis In the Discover-1 and -2 studies, 54%-63% of patients taking Tremfya had a ≥4 point improvement in FACIT-Fatigue compared to 35%-46% of patients taking placebo (unadjusted p≤0.003)
Novartis AG, of Basel, Switzerland Canakinumab  Monoclonal antibody targeting interleukin-1 beta Hospitalized COVID-19  pneumonia and cytokine release syndrome Study didn't meet its primary endpoint of improved survival without the need for invasive mechanical ventilation; data to be submitted for publication
Pfizer Inc., of New York Tofacitinib  Jak inhibitor Ankylosing spondylitis At week 16, 56.4% of patients taking tofacitinib achieved an ASAS20 response compared to 29.4% of patients taking placebo (p<0.0001); ASAS40 response rate was 40.6% for tofacitinib and 12.5% for placebo (p<0.0001)


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