Company Product Description Indication Status
Phase I
Chondrial Therapeutics Inc., of Bala Cynwyd, Pa. CTI-1601 Frataxin fusion protein prodrug Friedreich’s ataxia Dosed first patient in the study testing the safety, tolerability and pharmacokinetics of single ascending doses; top-line data expected by the end of 2020
Eleusis Ltd., of London Lysergic acid diethylamide Psychoactive drug Healthy older volunteers (eventually Alzheimer’s disease) Data published in Psychopharmacology showed low doses of the drug produced no deviations from baseline or abnormalities on safety or cognitive outcome measures
Rezolute Inc. AB-101 Ultra-long acting basal insulin  Type 1 and type 2 diabetes AB101 was administered subcutaneously in 3 ascending dosing cohorts, which resulted in slow onset and sustained insulin levels for more than 7 days; at the higher doses, the drug volume was greater than anticipated, as was variability in onset time between patients
Zentalis Pharmaceuticals Inc., of New York  ZN-c3 Small molecule DNA damage response candidate targeting protein tyrosine kinase WEE1 Solid tumor Dosed the first patient in a phase I/II trial evaluating ZN-c3 in patients with advanced solid tumors
Homology Medicines Inc., of Bedford, Mass. HMI-102 Gene therapy expressing PAH Phenylketonuria  First patient in cohort 2 (mid-dose) had reductions in Phe of 35% and 48% and increases in Tyr levels of 72% and 85% from baseline to week 1 and week 4, respectively
Immix Biopharma Inc., of Los Angeles Imx-110 NF-kB/Stat3/pan-kinase inhibitor curcumin plus doxorubicin encased in a nano-sized delivery system Advanced solid tumors Synergy Hematology Oncology institutional review board approved dosing patients
Modag GmbH, of Wendelsheim, Germany Anle-138b  Binds oligomeric structures of alpha-synuclein Healthy volunteers (eventually Parkinsonian disorders) Started study testing the safety, tolerability and pharmacokinetic profile of single and multiple ascending doses of the drug
Neuclone Pharmaceuticals Ltd., of Sydney Neulara  Biosimilar of Stelara (ustekinumab) Healthy volunteers (eventually plaque psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis) Completed dosing of subjects in study comparing a single dose of Neulara to U.S. and EU sourced Stelara; data expected in the third quarter of 2020
Poxel SA, of Lyon, France PXL-065 Deuterium-stabilized R-pioglitazone Healthy volunteers (eventually nonalcoholic steatohepatitis) Pharmacokinetic assessment showed plasma exposure (Cmax and area under the curve) increased in a dose-proportional manner up to the maximum dose tested; dosing fed vs. fasted didn't produce a clinically meaningful difference; plans to start a 120-patient phase II study in the second quarter of 2020
Regenxbio Inc., of Rockville, Md. RGX-121 Gene therapy expressing iduronate-2-sulfatase Mucopolysaccharidosis Type II In 3 patients in cohort 1, heparan sulfate in the cerebral spinal fluid was reduced by a mean of 33.3% from baseline to week 8; first patient treated in cohort 2
Topas Therapeutics GmbH, of Hamburg, Germany TPM-203 Small peptide-loaded nanoparticles Pemphigus vulgaris First of 24 patients enrolled in the study testing single ascending doses, followed by multiple doses
Phase II
Diamedica Therapeutics Inc., of Minneapolis DM-199 Recombinant human KLK1 Chronic kidney disease First of 60 patients treated in the study testing 2 dose levels twice weekly for 95 days; primary endpoints include safety, tolerability, blood pressure, albuminuria and kidney function
Medicure Inc., of Winnipeg, Manitoba Aggrastat (tirofiban hydrochloride) Antagonist of platelet glycoprotein IIb/IIIa receptor Percutaneous coronary intervention Completed SAVI-PCI study comparing shortened infusions of Aggrastat to long infusions of Aggrastat and to standard infusion of Integrilin (eptifibatide); top-line data expected in the first quarter of 2020
Inventiva, of Daix, France Odiparcil  Orally available small molecule Mucopolysaccharidosis type VI The 26-week study of 20 patients 16 years or older met its safety primary objective; the majority of AEs were mild or moderate; 1 death occurred in the placebo group and three SAEs were assessed as treatment-related; improvements were observed with regards to corneal clouding, cardiac and respiratory functions though no clear difference was observed in locomotor functions
Phase III
MC2 Therapeutics A/S, of Copenhagen Wynzora Calcipotriene and betamethasone dipropionate Plaque psoriasis Treatment success, defined as a minimum 2-point decrease in the Physician Global Assessment score to clear or almost clear disease at week 8, was 52% for Wynzora (p<0.05 compared to Daivobet); Wynzora improved mPASI from baseline to week 8 greater than Daivobet (p<0.05); change in mPASI at week 4 was 68% for Wynzora and better than Daivobet (p<0.001)
Mycovia Pharmaceuticals Inc., of Durham, N.C. VT-1161  Antifungal  Recurrent vulvovaginal candidiasis Completed enrollment of more than 200 patients in the Ultraviolet study; top-line data expected in the second half of 2020
Xenikos BV, of Nijmegen, the Netherlands T-Guard  Toxin-conjugated monoclonal antibodies that target CD3 and CD7 Steroid-refractory acute graft-vs.-host disease First of 47 patients treated in the single-arm study; primary endpoint is complete response rate on day 28; secondary endpoints include duration of complete response, overall response rate and overall survival rate six months after treatment
Phase IV
Eli Lilly and Co., of Indianapolis Emgality (galcanezumab-gnlm) Monoclonal antibody targeting calcitonin gene-related peptide Migraine prevention Started the 2,850-patient Triumph study comparing the use of Emgality to other treatments for patients switching or beginning a new prescription over 2 years; study will measure patient-reported outcomes related to migraine burden, quality of life and overall treatment satisfaction, as well as reduction in monthly migraine headache days, acute medication outcomes and other patterns of migraine treatment


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