Company Product Description Indication Status
Phase I
Denali Therapeutics Inc., of South San Francisco DNL-151   LRRK2 inhibitors Parkinson’s disease Enrollment in phase I and phase Ib studies paused due to COVID-19; analyzing data from studies; plans to select DNL-151 or DNL-201 for phase II/III development by mid-2020
Denali Therapeutics Inc., of South San Francisco DNL-310   Recombinant iduronate 2-sulfatase enzyme Hunter syndrome Study remains on track to start in the second quarter; enrollment in observational biomarker study paused due to COVID-19
Denali Therapeutics Inc., of South San Francisco DNL-343  Restores function of EIF2B Healthy volunteers (eventually amyotrophic lateral sclerosis/frontotemporal degeneration) Enrollment paused due to COVID-19
Novan Inc., of Morrisville, N.C. SB-206 (berdazimer sodium) Nitric oxide-releasing macromolecule Molluscum contagiosum infection Partner Sato Pharmaceutical Co. Ltd. said it plans to advance development program in Japan
Restorbio Inc., of Boston RTB-101 Inhibits target of rapamycin complex 1 Parkinson’s disease  Postponing enrollment of fifth cohort due to COVID-19
Phase II
Axon Neuroscience SE, of Vienna AADvac-1 Tau vaccine Mild Alzheimer's disease AADvac-1 reduced neurofilament light (NfL) chain in the blood by 58% compared to placebo (p=0.004); in patients with young onset of Alzheimer's disease, drug reduced rate of neuronal NfL accumulation in the blood by 73% in comparison to placebo (p=0.033); in those patients, drug reduced clinical decline measured by CDR-SB by 42% compared to placebo (p=0.062), MMSE (reduced by 31%) and ADCS-MCI-ADL (reduced by 26%)
Neurocrine Biosciences Inc., of San Diego Crinecerfont  Corticotropin-releasing factor type 1 receptor antagonist Congenital adrenal hyperplasia Enrollment paused due to COVID-19
Neurocrine Biosciences Inc., of San Diego, and Voyager Therapeutics Inc., of Cambridge, Mass. NBIb-1817 Gene therapy expressing AADC Parkinson's disease Enrollment in the Restore-1 study paused due to COVID-19
Oryzon Genomics SA, of Madrid Vafidemstat  LSD1 inhibitor Alzheimer’s disease After 6 months of treatment in the Reimagine-AD study of moderate and severe patients, there was a reduction of aggression as measured by the Clinical Global Impression of Improvement, the Cohen-Mansfield Agitation Inventory and the Neuropsychiatric Inventory (NPI) 4-item Agitation/Aggression subscale; NPI total score improved; caregiver burden as measured by the Zarit Caregiver Burden Interview improved (p<0.05 for all measurements); in the Etheral study of mild to moderate patients, drug reduced YKL40 compared to placebo (p=0.007)
Umecrine Cognition AB, of Stockholm, a Karolinska Development portfolio company Golexanolone (GR-3027) GABA-A receptor modulating steroid antagonist Hepatic encephalopathy Top-line data from a phase IIa study in patients at risk of developing HE showed safety and tolerability, but no indications of clinical effects could be observed; results will be further analyzed to determine whether further development is merited
Urogen Pharma Ltd., of Princeton, N.J. UGN-102 Sustained-release mitomycin Low-grade intermediate-risk non-muscle invasive bladder cancer Data from 63 patients in the Optima study published in The Journal of Urology showed the drug produced a complete response rate after 3 months of 65%; 97% and 85% of those patients were disease-free after at 6 and 9 months of follow-up, respectively
Phase III
Neurocrine Biosciences Inc., of San Diego Ingrezza (valbenazine ) Vesicular monoamine transporter 2 inhibitor Chorea in Huntington disease Enrollment paused due to COVID-19
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris Dupixent (dupilumab) IL-4/IL-13 receptor antagonist Atopic dermatitis In children ages 6 to 11 with uncontrolled severe disease, study drug combined with standard-of-care topical corticosteroids improved disease signs, symptoms and health-related quality of life
Vanda Pharmaceuticals Inc., of Washington Tradipitant  NK1 receptor antagonist COVID-19 acute respiratory distress syndrome Initiated Odyssey VLY-686-3501 study expected to enroll 300 individuals ages 18 to 90 hospitalized with COVID-19, initially in New York, to treat and prevent inflammatory lung injury associated with severe infection; primary endpoint is proportion of those with normalization of fever and oxygen saturation by day 14 


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