Company Product Description Indication Status
Phase I
Amolyt Pharma SAS, of Lyon, France AZP-3601 Parathyroid hormone analogue Hypoparathyroidism Dosed first patient; study will test single and multiple ascending doses in up to about 130 healthy subjects and patients 
Basilea Pharmaceutical Ltd., of Basel, Switzerland Derazantinib Fibroblast growth factor receptor Advanced urothelial cancer and FGFR genetic aberrations Testing when combined with Tecentriq (atezolizumab, Roche Holding AG); independent committee determined that derazantinib and atezolizumab can be safely combined at doses of 300 mg of daily oral derazantinib and 1,200 mg atezolizumab, administered intravenously once every 3 weeks
Celldex Therapeutics Inc., of Hampton, N.J. CDX-0159 KIT receptor binder Chronic spontaneous urticaria Trial begun
Cyclerion Therapeutics Inc., of Cambridge, Mass. IW-6463 Soluble guanylate stimulator CNS disorders Results showed treatment in 15-day 24-subject crossover study confirmed and extended results seen in earlier studies: once-daily oral treatment demonstrated blood-brain barrier penetration, desired CNS exposure levels and target engagement; subjects showed meaningful improvements in certain neurophysiological and objective performance measures associated with age-related cognitive decline and neurodegenerative diseases
Cytoagents Inc., of Pittsburgh GP-1681 Inhibitor of cytokine release Cytokine storm Enrollment begun
Evelo Biosciences Inc., of Cambridge, Mass. EDP-1815 Oral biologic; strain of Prevotella histicola Mild to moderate atopic dermatitis Completed enrollment of 24 patients in phase Ib trial cohort in atopic dermatitis; data expected in 2021
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. IONIS-ENAC-2.5Rx Antisense medicine designed to reduce  expression of ENaC in the lung Cystic fibrosis Study showed a mean 55.6% decrease (p<0.05) in ENaC mRNA expression at the 75-mg dose in the multidose segment of the trial
Linnaeus Therapeutics Inc., of Haddonfield, N.J. LNS-8801 GPER agonist Advanced cancers Dosed the first patient with drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.)
Noxxon Pharma NV, of Berlin NOX-A12 CXCL12 inhibitor Brain cancer All 3 patients in second dose cohort enrolled; phase I/II study is testing 3 dose regimens, each combined with external beam radiotherapy in newly diagnosed patients
Shasqi Inc., of San Francisco SQ-3370 Activates non-toxic protodrug into doxorubicin Advanced solid tumors First 2 patients dosed in dose-escalation trial enrolling subjects with locally advanced or metastatic disease ineligible for standard of care; trial expected to be completed in 2021
Taiwan Liposome Co., of Taipei, Taiwan TLC-19 Liposomal formulation of small amount of hydroxychloroquine COVID-19 Enrolled first subject for treatment or prophylaxis; trial to enroll 30 healthy volunteers and test 3 dose levels 
Phase II
Anaptysbio Inc., of San Diego Imsidolimab (ANB-019) Antibody inhibiting IL-36R Moderate to severe generalized pustular psoriasis Interim analysis from Gallop trial showed 6 of 8 patients achieved primary endpoint of improvement in clinical global impression scale (CGI) on day 29, with rapid reduction of skin pustules by 60% on day 8 and 94% clearance on day 29; FDA end-of-phase II meeting anticipated in fourth quarter of 2020
Auris Medical Holding Ltd., of Hamilton, Bermuda AM-125 (intranasal betahistine) Histamine H3 receptor antagonist; histamine H1 receptor partial agonist Acute vertigo Randomized first patient in part B of Travers trial following positive readout from interim analysis on part A, completion of enrollment in oral treatment group and receipt of regulatory clearance for part B in first 3 participating countries
Azurrx Biopharma Inc., of New York MS-1819 Recombinant lipase enzyme Cystic fibrosis with severe exocrine pancreatic insufficiency Activated 2 trial sites in Turkey for trial testing combination with porcine pancreatic enzyme replacement therapy
Bone Therapeutics SA, of Gosselies, Belgium Allob Allogeneic cell therapy Lumbar spinal fusion procedures 24-month follow-up results from phase IIa study showed high percentage of successful lumbar vertebrae fusion of 90%; patients continued to experience clinical improvements in function and pain, from as early as 6 months after treatment, up to the 24-month follow-up period
Cyclerion Therapeutics Inc., of Cambridge, Mass. Olinciguat Oral, once-daily vascular sGC stimulator Sickle cell disease Results from Strong-SCD study did not demonstrate adequate activity to support further internal clinical development
Horizon Therapeutics plc, of Dublin Tepezza (teprotumumab-trbw) Insulin-like growth factor 1 receptor antagonist Thyroid eye disease Long-term follow-up data showed sustained response up to 1 year following completion of treatment
Phase III
Mesoblast Ltd., of Melbourne, Australia, and New York Remestemcel-L Mesenchymal stem cell-based therapy Acute respiratory distress due to COVID-19 Trial testing drug on top of maximal care in ventilator-dependent patients surpassed 50% enrollment
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA for injection Acetylcholine receptor antagonist; botulinum toxin A stimulator Cervical dystonia Top-line results showed Aspen-1 study met primary endpoint at both doses, demonstrating clinically meaningful improvements in signs and symptoms at the average weeks of 4 and 6; subjects treated with either 125 units or 250 units showed statistically significant greater change from baseline vs. placebo (12.7 and 10.9 respectively vs. 4.3; p<0.0001 and p=0.0006) as measured on Toronto Western Spasmodic Torticollis Rating Scale Total Score; median duration of effect was 24 and 20.3 weeks, for the 125-unit and 250-unit dose groups, respectively, based on median time to loss of 80% of the peak treatment effect


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