Company Product Description Indication Status
Phase I
Delta-Fly Pharma Inc., of Tokushima, Japan DFP-14927 Pegylated-DFP-10917 (nucleoside analogue) Advanced solid tumors Study initiated at MD Anderson Cancer Center
Imbrium Therapeutics LP, subsidiary of Purdue Pharma LP, of Stamford, Conn., and Spinethera Inc., of Plymouth, Minn. SX-600 (reformulated dexamethasone acetate, epidural/sustained release) Glucocorticoid receptor agonist Pain First dose administered in phase I/II Salient study evaluating efficacy and safety of 12.5-mg and 25-mg doses to treat sciatica
Phase II
Bio-Path Holdings Inc., of Houston Prexigebersen (BP-1001) GRB2 gene inhibitor Acute myeloid leukemia;  myelodysplastic syndrome Safety segment of stage 2 of trial, which comprised 6 evaluable participants who received drug in combination with decitabine, confirmed safety profile; 2 participants showed complete responses with incomplete hematologic recovery and 1 showed partial response
Phase III
Acadia Pharmaceuticals Inc., of San Diego Pimavanserin (Nuplazid) 5-HT 2a receptor inverse agonist Schizophrenia In Advance study in 403 participants, drug showed statistically significant improvement on primary endpoint of change from baseline to week 26 on Negative Symptom Assessment-16 (NSA-16) total score vs. placebo (-10.4 vs. -8.5; p=0.043; effect size=0.21); greater improvement in NSA-16 total score vs. placebo seen in 53.8% of those (n=107) who received highest dose of 34 mg (-11.6 vs. -8.5; unadjusted p=0.0065, effect size=0.34); pimavanserin did not separate from placebo on key secondary endpoint of Personal and Social Performance scale; second pivotal study with 34-mg dose expected to begin in first half of 2020
Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid derived from krill oil Hypertriglyceridemia Final participant completed final visit in pivotal Trilogy 1 trial, with top-line results expected as planned in December
Allergy Therapeutics plc, of Worthing, U.K.
Grass MATA MPL 
TLR4 agonist Allergy Upcoming Grass study will take stepwise approach, with 2 stages covering both 2020/21 and 2021/22 pollen seasons rather than single large trial originally planned for 2020/21 season
Delta-Fly Pharma Inc., of Tokushima, Japan DFP-10917 Nucleoside analogue Acute myelogenous leukemia First participants registered in U.S. study enrolling individuals with relapsed/refractory disease
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Nonalcoholic steatohepatitis Data safety monitoring board recommended continuing Resolve-IT study without modification; top-line interim results expected in first quarter 2020
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) IL-17 antagonist Axial spondylarthritis In Coast-X study in 303 adults with non-radiographic disease, at week 16, 35% who received drug every 4 weeks and 40% who received it every 2 weeks achieved ASAS40 response vs. 19% who received placebo; at week 52, 30% treated with drug every 4 weeks and 31% treated every 2 weeks achieved ASAS40 response vs. 13% for placebo; Taltz also met major secondary endpoints at weeks 16 and 52 
Marinomed Biotech AG, of Vienna Budesolv (budesonide) Synthetic steroid Hay fever In a 75-patient study, Budesolv reduced the total nasal symptom score by 1.2 points after 4 hours; Rhinocort Aqua showed no therapeutic effect at 4 hours; after 8 days drugs had comparable efficacy
Poxel SA, of Lyon, France Imeglimin  Targets mitochondrial bioenergetics Type 2 diabetes In 208-patient open-label extension of Times3 study, imeglimin and insulin reduced HbA1c by 0.64% vs. baseline over 52 weeks; patients who took placebo and insulin for the first 16 weeks and imeglimin and insulin for the final 36 weeks had a 0.54% reduction in HbA1c from baseline
Vifor Fresenius Medical Care Renal Pharma Ltd., of St. Gallen, Switzerland, and Chemocentryx Inc., of Mountain View, Calif. Avacopan Complement 5a receptor inhibitor Anti-neutrophil cytoplasmic antibody-associated vasculitis In 331-patient Advocate study, at week 26, 72.3% of patients treated with avacopan and 70.1% of patients treated with glucocorticoid standard of care were responders, defined as a Birmingham Vasculitis Activity Score of 0 and off glucocorticoid treatment for ANCA vasculitis for at least the preceding 4 weeks (p<0.0001 for noninferiority); at week 52, 65.7% of the avacopan-treated patients and 54.9% of glucocorticoid-treated patients had sustained remission (p=0.0066 for superiority of avacopan)

Notes

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

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