Company Product Description Indication Status
Phase I
Aligos Therapeutics Inc., of South San Francisco ALG-000184 Class II capsid assembly modulator Hepatitis B virus infection Dosed first subject in the study testing the safety and pharmacokinetics of single and multiple ascending doses of ALG-000184 in healthy volunteers and antiviral activity in patients
Axovant Gene Therapies Ltd., of New York AXO-Lenti-PD Gene therapy Parkinson’s disease At 6 months post-treatment, all 4 patients in the second dose cohort had improvement in Hauser diary “Good on time” and “off time” changes from baseline; 2 evaluable patients had improvement in UPDRS Part II and Part III “OFF” score; first patient had a 23-point improvement from baseline to 12 months in UPDRS Part III “off” score at 12 months
Bellicum Pharmaceuticals Inc., of Houston BPX-601  GoCAR-T candidate  Pancreas tumor In cohort 5C, 3 patients had stable disease and 1 patient had progressive disease; there was evidence of rimiducid-mediated CAR-T cell activation
Phase II
Eli Lilly and Co., of Indianapolis Olumiant (baricitinib) JAK inhibitor  Alopecia areata After 36 weeks of treatment in the BRAVE-AA1 study, 33.3% and 51.9% of patients taking 2 mg and 4 mg of the drug, respectively, had 20% or less of scalp hair loss, compared to 3.6% of patients taking placebo (p=0.016 and p=0.001, respectively)
Phase III
Biolinerx Ltd., of Tel Aviv, Israel Motixafortide  CXCR4 inhibitor Hematopoietic stem cell transplantation Independent data monitoring committee recommended stopping enrollment based on statistically significant evidence favoring treatment with motixafortide; data from the 122 enrolled patients are expected in the first half of 2021
Cerevel Therapeutics LLC, of Boston Tavapadon  Dopamine D1/D5 receptor partial agonist Parkinson’s disease First patients dosed in 3 27-week studies; Tempo-1 is testing fixed doses of the drug; Tempo-2 tests flexible doses; Tempo-3 tests drug plus levodopa in late stage patients; primary endpoint of  TEMPO-1 and -2 is the change from baseline in the MDS-UPDRS Part II and Part III combined score; primary endpoint of TEMPO-3 is the change from baseline in total daily “on” time without troublesome dyskinesia


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