Company Product Description Indication Status
Phase I
Aprea Therapeutics Inc., of Boston APR-246 Small molecule designed to reactivate mutant and inactivated p53 protein  TP53-mutated myelodysplastic syndromes and acute myeloid leukemia  In the French trial, updated data for 24 evaluable patients enrolled before June 2019 showed an overall response rate of 71%, including complete remission for 54%; including 3 additional patients enrolled after the cutoff changed ORR to 74% and CR rate to 59%; median overall survival hasn’t been reached for the 53 enrolled patients
Beigene Ltd., of Beijing Tislelizumab Monoclonal antibody targeting PD-1 Platinum-resistant ovarian cancer Treatment with tislelizumab plus sitravatinib produced an overall response rate of 23.5% in 7 patients, all of which were partial responses; median progression-free survival was 18 weeks
Kaleido Biosciences Inc., of Lexington, Mass. KB-174 Mixture of oligosaccharides Well-compensated cirrhosis In a 15N-labeled tracer test, treatment with drug for 28 days reduced urinary 15N excretion by a median of 26%, compared to a 3% median reduction for patients taking the negative control maltodextrin; plans to start a study in patients with hepatic encephalopathy in the second half of 2020
Trevena Inc., of Chesterbrook, Pa. TRV-250 G protein-selective delta-receptor agonist Acute migraine Drug was well-tolerated with a pharmacokinetics profile appropriate for an acute migraine therapy
Phase II
Biogen Inc., of Cambridge, Mass. Gosuranemab (BIIB-092) Monoclonal antibody targeting N-terminal tau Progressive supranuclear palsy In the Passport study, primary endpoint of changes in the Progressive Supranuclear Palsy Rating Scale at week 52 was not met; key clinical secondary endpoints also not met; plans to discontinue development for progressive supranuclear palsy and other primary tauopathies
Biolinerx Ltd., of Tel Aviv, Israel BL-8040 CXCR4 inhibitor Metastatic pancreatic adenocarcinoma In the Combat/Keynote-202 study, BL-8040 plus Keytruda (pembrolizumab, Merck & Co. Inc.) and chemotherapy produced an overall response rate of 32% in 22 patients, all of which were partial responses; disease control rate was 77%; data on progression-free survival and overall survival expected in mid-2020
Innate Pharma SA, of Marseille, France Lacutamab (IPH-4102) Monoclonal antibody targeting KIR3DL2  T-cell lymphoma Due to quality issues at a manufacturing subcontractor, drug will not be administered to new patients; currently enrolled patients will continue to be treated except in Italy where the study has been suspended
Oncosec Medical Inc., of San Diego Tavo Plasmid expressing IL-12 Metastatic, chemotherapy-refractory triple-negative breast cancer Tavo plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced a 28.5% objective response rate in 14 evaluable patients; stable disease was observed in an additional 21.4% of patients
Umecrine Cognition AB, of Stockholm, a Karolinska Development Co. Golexanolone  GABAA receptor antagonist Liver cirrhosis and hepatic encephalopathy Completed enrollment in study testing multiple ascending doses for safety, pharmacokinetics and changes in cognitive function
Phase III
Adial Pharmaceuticals Inc., of Charlottesville, Va. AD-04 Genetically targeted therapeutic agent Alcohol use disorder Completed validation of the genetic biomarker test to be used in the study; in process of obtaining regulatory approvals in Europe to start study
Athenex Inc., of Buffalo, N.Y. Oral paclitaxel Chemotherapy Metastatic breast cancer In the 402-patient study, 35.8% of patients taking oral paclitaxel plus encequidar (Pac+E) had a confirmed tumor response compared to 23.4% of patients taking intravenous paclitaxel (IV Pac) (p=0.011); for population with evaluable post-baseline scan, confirmed response rates were 50.3% for Pac+E vs. 29.6% for IV Pac (p=0.0005)
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) Sodium glucose co-transporter-2 inhibitor Chronic heart failure with reduced and preserved ejection fraction In the Emperial-Reduced and Emperial-Preserved studies there were no significant changes in 6-minute walk test over 12 weeks between Jardiance and placebo; data to be presented in 2020
Intra-Cellular Therapies Inc., of New York Lumateperone  Serotonin 5-HT2A receptor antagonist Bipolar depression In Study 404, compared to placebo, lumateperone improved Montgomery-Åsberg Depression Rating Scale at week 6 by an effect size (ES) of 0.56 (p<0.0001); compared to placebo, drug also improved Clinical Global Impression Scale for Bipolar for Severity of Illness (CGI-BP-S) Total Score (p<0.001; ES=0.46) and CGI-BP-S Depression Score (p<0.001; ES=0.50)
Roche Holding AG, of Basel, Switzerland, and Exelixis Inc., of Alameda, Calif. Tecentriq (atezolizumab) Monoclonal antibody targeting PD-L1 Previously untreated BRAF V600 mutation-positive advanced melanoma In the Imspire150 study, Tecentriq (atezolizumab) plus Cotellic (cobimetinib) and Zelboraf (vemurafenib) improved progression-free survival compared to placebo plus Cotellic and Zelboraf; data to be presented at an upcoming medical meeting


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