Company Product Description Indication Status
Phase I
Arrowhead Pharmaceuticals, Inc., of Pasadena, Calif. ARO-HSD RNAi targeting HSD17B13 Nonalcoholic steatohepatitis Treated first patient in the AROHSD1001 study testing safety, tolerability, pharmacokinetics and pharmacodynamic effects
CNS Pharmaceuticals Inc., of Houston Berubicin Anthracycline Glioblastoma multiforme The 1 patient out of 25 who had a durable complete response in the 2006 study remains cancer free as of February 2020; company plans to start a phase II study in the second half of 2020
Mina Therapeutics Ltd., of London MTL-CEBPA Up-regulates CCAAT/enhancer binding protein alpha Advanced solid tumors Treated the first patient in the Timepoint study testing the drug in combination with Keytruda (pembrolizumab, Merck & Co. Inc.); study includes dose-escalation phase followed by a dose-expansion phase
PDC*Line Pharma SA, of Liege, Belgium  PDC*lung-01 Irradiated human plasmacytoïd dendritic cells loaded with 7 tumor antigens Non-small-cell lung cancer First patient treated in the study designed to to assess the safety, tolerability, immunogenicity and preliminary clinical activity of the drug
Prevail Therapeutics Inc., of New York PR-001 Gene therapy expressing GBA1 Parkinson’s disease with GBA1 mutations Enrollment continues in the Propel study putting the company on track to report interim data in the second half of 2020
Puretech Health plc, of Boston LYT-100 Deuterated pirfenidone Healthy volunteers (eventually lymphedema) First of 100 volunteers dosed in the multiple ascending-dose and food effect study designed to test the safety, tolerability and pharmacokinetic profile of the drug; data expected later in 2020
Targovax ASA, of Oslo, Norway ONCOS-102 Adenovirus-based cancer therapy  Advanced anti-PD1 refractory melanoma Completed enrollment of 12 patients into part 2 of the study testing 12 injections of ONCOS-102 and treatment with Keytruda (pembrolizumab, Merck & Co. Inc.); data expected in the second half of 2020
VBI Vaccines Inc., of Cambridge, Mass., VBI-1901 GM-CSF receptor agonist  Recurrent glioblastoma Updated data from patients in part A of the study showed the 12-month overall survival rate was 83% for 6 patients who responded to the vaccine, compared to 33% for 9 vaccine nonresponders; median OS for responders was 14 months, compared to 7.75 months for nonresponders
Phase II
Neuraly Inc., of Germantown, Md. NYL-01 Glucagon-like peptide 1 receptor agonist Parkinson’s disease Dosed first of about 240 patients in the study to test the safety, tolerability and efficacy of the drug
Phase III
Avita Medical Ltd., of Valencia, Calif. Recell System Autologous skin cell suspension Soft tissue reconstruction of skin defects Enrolled first of at least 65 patients in the study comparing the Recell System to conventional skin grafting; primary endpoints are superior donor skin sparing and noninferior incidence of healing by 8 weeks post-treatment
Eisai Co. Ltd., of Tokyo, and Bioarctic AB, of Stockholm BAN-2401 Amyloid beta-targeting monoclonal antibody Alzheimer’s disease Study expands sites to 4 Swedish clinics; data expected in 2022


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

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