Company Product Description Indication Status
Phase I
Celon Pharma S.A., of Kielpin, Poland CPL-116 JAK/ROCK kinases dual inhibitor Autoimmune diseases Trial successfully completed
Galecto Inc., of Boston GB-1211 Oral, small-molecule galectin-3 inhibitor Nonalcoholic steatohepatitis and liver fibrosis Phase I/II trial withdrawn due to COVID-19 pandemic and change in clinical development strategy for compound
Regulus Therapeutics Inc., of La Jolla, Calif. RGLS-4326 Oligonucleotide designed to inhibit miR-17 Autosomal dominant polycystic kidney disease First cohort enrolled
Tessa Therapeutics Ltd., of Bedminster, N.J., and Singapore TT-11X CD30 modulator; allogeneic cell therapy CD30+ lymphoma Completed dosing of first patient cohort
Yumanity Therapeutics Inc., of Boston YTX-7739 Small molecule targeting stearoyl-CoA desaturase Parkinson’s disease Results from single ascending-dose study in healthy volunteers showed no safety concerns identified and drug was well-tolerated; half-life of drug combined with favorable dose-proportional pharmacokinetic profile, in the fed state, supports that low daily doses administered with food will sustain the target range of exposure
Yumanity Therapeutics Inc., of Boston YTX-7739 Small molecule targeting stearoyl-CoA desaturase Parkinson’s disease Phase Ib study initiated to enroll 30 patients and collect safety, tolerability, pharmacokinetic and pharmacodynamic parameters, including potential biomarkers of SCD activity and target engagement in the CSF, plasma and other fluids or tissues; preliminary results expected by midyear 2021
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia Ifenprodil  Noncompetitive NMDA antagonist COVID-19 Company is reviewing protocol for phase II/III trial to consider adding lung scarring as additional endpoint if sufficient data are available from significant number of patients
Basilea Pharmaceutica Ltd., of Basel, Switzerland Derazantinib Oral small-molecule FGFR inhibitor  Intrahepatic cholangiocarcinoma Top-line results from Fides-01 trial in patients with inoperable or advanced disease and confirmed FGFR2 gene fusions showed objective response rate of 20.4% based on 21 patients with partial response; disease control rate was 72.8%; median progression-free survival was 6.6 months
Gemini Therapeutics Inc., of Cambridge, Mass. GEM-103  Recombinant human complement factor H Dry age-related macular degeneration Completed enrollment in phase IIa Regatta study in patients with CFH loss-of-function gene variants; top-line data expected in first half of 2021
Genfit SA, of Lille, France Elafibranor PPAR alpha agonist; Insulin sensitizer; PPAR delta agonist Primary biliary cholangitis Results from study in patients with incomplete response to ursodeoxycholic acid, published in the Journal of Hepatology, show clinically relevant improvement in primary and composite biochemical endpoints, positive trend on pruritus improvement, while maintaining favorable tolerability profile; results support longer-term, pivotal Elative phase III study
Phase III
Galapagos NV, of Mechelen, Belgium, and Gilead Sciences Inc., of Foster City, Calif. Ziritaxestat Autotaxin inhibitor Idiopathic pulmonary fibrosis Companies decided to halt Isabela studies based on recommendation of independent data monitoring committee, which concluded after regular review of unblinded data that risk-benefit profile no longer supported continuing these studies
Gamida Cell Ltd., of Boston Omidubicel Cell therapy Patients with high-risk hematologic malignancies undergoing bone marrow transplant  Results showed faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital vs. standard umbilical cord transplant; median time to neutrophil engraftment was 12 days for patients randomized to omidubicel vs. 22 days for the comparator group (p<0.001), meeting primary endpoint
Infant Bacterial Therapeutics AB, of Stockholm IBP-9414 Contains active substance Lactobacillus reuteri Prevention of necrotizing enterocolitis and improvement of feeding tolerance in premature infants Reached milestone after recruiting 300 premature infants to ongoing study
Protalix Biotherapeutics Inc., of Carmiel, Israel Pegunigalsidase alfa (PRX–102) Alpha-galactosidase stimulator Fabry disease Final results from Bridge trial showed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR), in both male and female patients who were switched from agalsidase alfa to pegunigalsidase alfa; mean annualized eGFR slope of participants improved from -5.90 mL/min/1.73m2/year while on agalsidase alfa to -1.19 mL/min/1.73m2/year on pegunigalsidase alfa in all patients
University of Oxford, of Oxford, U.K. Almitrine  Respiratory stimulant COVID-19 Started study to test whether oral drug, administered over a 7-day period, is effective in reducing the need for other forms of ventilatory support


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