Company Product Description Indication Status
Phase I
4D Molecular Therapeutics, of Emeryville, Calif. 4D-125 Gene therapy expressing RPGR X-linked retinitis pigmentos Treated first of approximately 18 patients in study testing the  safety, tolerability and biological activity of a single intravitreal injection of 4D-12
Acelrx Pharmaceuticals Inc., of Redwood City, Calif. Dsuvia (sufentanil) Opioid analgesic Acute pain Data published in the Journal of Clinical Anesthesia and Pain Management showed discharge from the post anesthesia care unit was 36.3 minutes for Dsuvia compared to 54.9 minutes for historical controls who received intravenous opioid; patients taking Dsuvia required mean opioid dose of 10.9 mg morphine equivalents (MME), compared to 20.0 mg MME for the controls (p < 0.001)
PCI Biotech AS, of Oslo, Norway Fimaporfin  Light-activated benzenesulfonic acid isomers Inoperable extrahepatic bile duct cancer Case report series will be published in Endoscopy International Open
Pluristem Therapeutics Inc., of Haifa, Israel PLX-R18 Cell therapy Incomplete hematopoietic recovery following hematopoietic cell transplantation Completed enrollment in the study; data expected in the first quarter of 2021
Trefoil Therapeutics Inc., of San Diego TTHX-1114 Fibroblast growth factor-1 Corneal endothelial dystrophies Started Intrepid study of up to 71 patients; primary endpoint is the change in corneal endothelial cell count from baseline to day 56; secondary endpoints include measures of visual acuity, corneal edema and corneal endothelial cell growth
Phase II
Albireo Pharma Inc., of Boston Elobixibat Ileal bile acid transporter inhibitor  Nonalcoholic fatty liver disease and nonalcoholic steatohepatitis Drug lowered low-density lipoprotein cholesterol by 20.5 mg/dL in serum compared to an 11.1 mg/dL decline for placebo (p<0.022); drug reduced liver fat by 2.6%; company discontinuing further development
Epitopoietic Research Corp., of Isnes, Belgium Gliovac (ERC-1671) Immunotherapy based on freshly extracted tumor cells and lysates  Recurrent glioblastoma Interim results from first 10 patients treated with Gliovac, GM CSF, cyclophosphamide and bevacizumab showed a 6-month overall survival rate of 100%, a 12-month OS rate of 40% and a median OS of 10.5 months
Jaguar Health Inc., of San Francisco Mytesi (crofelemer) Non-opiate chloride ion channel modulator antidiarrheal drug  Functional diarrhea Plans to start enrollment in the fourth quarter of 2020 with top-line results expected in the fourth quarter of 2021
Qu Biologics Inc., of Vancouver, British Columbia QBECO SSI Enteropathic E. coli Colorectal cancer Israeli Ministry of Health approved the start of the study
Turning Point Therapeutics Inc., of San Diego Repotrectinib Kinase inhibitor Non-small-cell lung cancer and solid tumors Objective response rate was 86% for 7 patients with ROS1-positive tyrosine kinase inhibitor (TKI)-naïve tumors, 40% for 5 patients with ROS1-positive tumors pretreated with 1 prior TKI with prior chemotherapy, 67% for 6 patients with ROS1-positive tumors pretreated with one prior TKI without prior chemotherapy, 0% for 10 patients with ROS1-positive tumors pretreated with 2 prior TKIs with prior chemotherapy, 40% for 5 patients with ROS1-positive tumors treated with 2 prior TKIs without prior chemotherapy and 50% for 6 patients with NTRK-positive TKI-pretreated solid tumors
Verona Pharma plc, of London Pressurized metered-dose inhaler formulation of ensifentrine Phosphodiesterase 3 and 4 inhibitor Moderate to severe chronic obstructive pulmonary disease Started multiple dose part B of of study; primary endpoint is peak FEV1 after 7 days of treatment; secondary endpoints include safety and tolerability, trough FEV1, average FEV1 over 4 and 12 hours and steady state pharmacokinetic profile of ensifentrine
Phase III
Biolinerx Ltd., of Tel Aviv, Israel Motixafortide (BL-8040) CXCR4 antagonist  Mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma Enrolled approximately 65% of planned sample size in the Genesis study; interim analysis will be done in second half of 2020
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 First-line locally advanced or metastatic esophageal carcinoma Keynote-590 met its primary endpoints of overall survival and progression-free survival; data submitted for presentation at the European Society for Medical Oncology Virtual Congress 2020
Northwest Biotherapeutics Inc., of Bethesda, Md. Dcvax-L Personalized immune therapy  Glioblastoma   Final quality control checking and confirmation are underway to enable data lock
Phase IV
Jaguar Health Inc., of San Francisco Mytesi (crofelemer) Non-opiate chloride ion channel modulator antidiarrheal drug  Chronic idiopathic diarrhea Started enrollment, which is expected to be complete by the end of 2020
Jaguar Health Inc., of San Francisco Mytesi (crofelemer) Non-opiate chloride ion channel modulator antidiarrheal drug  Diarrhea prophylaxis is HER2-positive breast cancer Nearing completion of enrollment; data expected in mid-2021
Kamada Ltd., of Rehovot, Israel, and Kedrion Biopharma, of Fort Lee, N.J. Kedrab (rabies immune globulin) Human rabies immunoglobulin Post-exposure prophylaxis treatment of rabies infection No incidence of rabies disease or deaths for the 30 pediatric patients throughout the 84-day study period


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

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