Company Product Description Indication Status
Phase I
Denali Therapeutics Inc., of South San Francisco, and Sanofi SA, of Paris DNL-758 Peripherally restricted small-molecule inhibitor of RIPK1 Severe COVID-19 Sanofi began dosing in phase Ib study in hospitalized adults with severe COVID-19 lung disease
Repare Therapeutics Inc., of Cambridge, Mass. RP-3500 Oral small-molecule inhibitor of ATR Solid tumors  Dosed first patient in phase I/II trial; study to enroll about 230 patients with advanced solid tumors of any histology that harbor gene alterations that suggest sensitivity to ATR inhibition 
Samus Therapeutics Inc., of Boston PU-AD Oral, small-molecule epichaperome inhibitor Alzheimer’s disease Results from healthy volunteer study showed drug was well-tolerated at all doses, with adverse event profile comparable to placebo; pharmacokinetic profile favorable and dose-proportional; PU-AD detected in cerebrospinal fluid samples provided evidence of blood-brain barrier penetration
Phase II
Alector Inc., of South San Francisco AL-001 Human monoclonal antibody designed to modulate progranulin Frontotemporal dementia Preliminary data study in patients whose disease has progranulin gene mutation (FTD-GRN) showed drug generally safe and well-tolerated; treatment led to sustained restoration of plasma progranulin levels in all FTD-GRN participants back to the normal range; preliminary data from symptomatic patients showed decrease in plasma NfL levels from baseline in the majority at last measured time point; in case study of longest treated patients (28+ weeks), there was sustained decrease in plasma NfL by 29% from baseline
Auris Medical Holding Ltd., of Hamilton, Bermuda AM-125 (betahistine hydrochloride, intranasal) Histamine H1 receptor partial agonist; histamine H3 receptor antagonist Vertigo Enrollment completed in part A of Travers study; interim data expected in third quarter of 2020
Constant Therapeutics LLC, of Boston TXA-127 Angiotensin II receptor modulator COVID-19 infection Trial initiated, seeking to recruit 100 people hospitalized with infection who require oxygen but are not in ICU; endpoints include incidence of renal failure and of respiratory failure
Dimerix Ltd., of Melbourne, Australia DMX-200 (irbesartan + propagermanium) Angiotensin II AT-1 receptor antagonist; CCR2 chemokine antagonist Focal segmental glomerulosclerosis Phase IIa study met primary and secondary endpoints; 86% on study drug showed reduction of proteinuria vs. placebo; 29% reduction observed across those who received DMX-200 vs. placebo, with 29% achieving >40% reduction vs. placebo
Phase III
Abbvie Inc., of North Chicago Atogepant CGRP receptor antagonist Migraine Advance trial met primary endpoint of statistically significant reduction from baseline in mean monthly migraine days vs. placebo for all doses evaluated across 12-week treatment period (p=<0.0001); trial also showed statistically significant improvements in all 6 secondary endpoints in 30-mg and 60-mg once-daily arms 
Genentech, unit of Roche Holding AG, of Basel, Switzerland Actemra (tocilizumab) IL-6 receptor antagonist COVID-19 infection Covacta trial missed primary endpoint of improved clinical status in those with COVID-19 associated pneumonia and key secondary endpoint of reduced mortality
Krystal Biotech Inc., of Pittsburgh, Pa. Beremagene geperpavec COL7A1 gene stimulator Epidermolysis bullosa dystrophica Placebo-controlled Gem-3 study initiated, aiming to enroll about 30 participants 6 months or older; up to 3 investigator-identified wound pairs in each person will be treated once weekly for 6 months; primary outcome measure is complete wound healing compared to baseline vs. placebo at weeks 20, 22 and 24


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

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