Company Product Description Indication Status
Phase I
Actinium Pharmaceuticals Inc., of New York Actimab-A (225Ac-lintuzumab) CD33 antagonist Acute myeloid leukemia Additional sites activated in phase I/II combination trial with Venclexta (venetoclax, Abbvie Inc.) enrolling adults with relapsed/refractory disease
Affimed NV, of Heidelberg, Germany AFM-13 CD30/CD16A ROCK-derived bispecific antibody CD30-positive lymphomas First of about 30 participants with relapsed/refractory disease was dosed in investigator-initiated study with allogeneic cord blood-derived natural killer cells preloaded with AFM-13 and moved into AFM-13 monotherapy phase; efficacy measures include overall, complete and partial response rates
Immunicum AB, of Stockholm Ilixadencel Off-the-shelf cell-based immune primer Solid tumors 15 of 21 people now enrolled in phase Ib portion of ongoing Iliad combination trial with Keytruda (pembrolizumab, Merck & Co. Inc.)
Neximmune Inc., of Gaithersburg, Md. NEXI-002 T-cell therapy Multiple myeloma First of 22 to 28 participants enrolled in phase I/II study; efficacy measures include signals of antitumor activity as well progression-free and overall survival
NMD Pharma A/S, of Aarhus, Denmark NMD-670 Voltage gated chloride channel 1 inhibitor Myasthenia gravis First of about 79 participants dosed in phase I/IIa trial in Leiden, the Netherlands
Phase II
Amgen Inc., of Thousand Oaks, Calif. Sotorasib (AMG-510) K-Ras GTPase inhibitor Advanced non-small-cell lung cancer Top-line data from CodeBreak 100 study in 126 people with KRAS G12C-mutant advanced disease showed primary endpoint of objective response rate was consistent with phase I data at 960-mg daily dose
Axovant Gene Therapies Ltd., of New York AXO-Lenti-PD Gene therapy Parkinson's disease In 6-month follow-up from second cohort of Sunrise-PD trial, 2 evaluable patients showed 21-point mean improvement in UPDRS part III (motor function) “off” score, representing 40% improvement from their baseline average score of 52, and 14-point mean improvement in the UPDRS part II (activities of daily living) “off” score, representing 71% improvement from baseline
Cerevel Therapeutics LLC, of Boston CVL-865 GABA A receptor 2/3 agonist Epilepsy First of about 150 participants dosed in Realize trial; primary outcome is measure of focal onset seizure frequency rate per week; data expected in second half of 2022
Chromadex Corp., of Los Angeles Niagen (nicotinamide riboside) Dietary supplement Mild to moderate COVID-19 infection Research conducted in partnership with Scandibio Therapeutics AB showed 29% reduction in recovery time in 100 outpatients who received standard of care and nutritional protocol that included agent, reducing average recovery time to 6.6 days vs. 9.3 days for placebo
EIP Pharma Inc., of Boston Neflamapimod p38 MAP kinase alpha inhibitor Mild to moderate Lewy body dementia Ascend-LB study met primary endpoint, showing improved cognition measured by Neuropsychological Test Battery in those who received study drug 3 times daily vs. twice daily or placebo (p=0.015, effect size [Cohen's d]=0.52)
Index Pharmaceuticals Holding AB, of Stockholm Cobitolimod TLR9 agonist Moderate to severe ulcerative colitis Findings from Conduct study, published in The Lancet Gastroenterology and Hepatology, showed phase IIb study met primary endpoint, with 21.4% of participants achieving clinical remission at week 6 in highest dose group (250 mg x 2) vs. 6.8% in placebo group (p=0.0247)
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab GM-CSFR-alpha antagonist Giant cell arteritis Primary efficacy endpoint of time-to-first adjudicated GCA flare by week 26 in treated patients was met with statistical significance (p=0.0263)
Miragen Therapeutics Inc., of Boulder, Colo. Cobomarsen miR-155 inhibitor Cutaneous T-cell lymphoma Internal review of preliminary top-line data from 37 participants suggested Solar trial would not achieve primary endpoint of objective skin response of at least 4 months duration vs. vorinostat control; company halting development
Neurotrope Inc., of New York Bryostatin-1 Protein kinase C agonist  Moderately severe and moderate Alzheimer's disease First patient treated in the 100-patient study
Tarsus Pharmaceuticals Inc., of Irvine, Calif. TP-03 Topical ophthalmic drug Demodex blepharitis In the lo study, 72% of patients treated with TP-03 had a collarette cure and 78% had mite eradication at day 42; in the Europa study, at day 42, collarette cure rate was 80% for TP-03 compared to 16% for vehicle (p<0.001) and mite eradication rate was 73% for TP-03 and 21% for vehicle (p=0.003); enrollment in the Saturn-1 trial began in September 2020
Zai Lab Ltd., of Shanghai Margetuximab Fc-optimized monoclonal antibody targeting HER2 HER2-positive gastric cancer or gastroesophageal junction cancer First patient in greater China treated in the Mahogany study testing margetuximab plus the anti-PD-1 antibody retifanlimab; study will also test margetuximab plus a checkpoint inhibitor and chemotherapy; primary efficacy endpoint is overall survival
Phase III
Amgen Inc., of Thousand Oaks, Calif. Sotorasib (AMG-510) K-Ras GTPase inhibitor Advanced non-small-cell lung cancer Recruitment initiated in confirmatory CodeBreak 200 study of sotorasib vs. comparator docetaxel in people with KRAS G12C-mutant disease
Brickell Biotech Inc., of Boulder, Colo. Sofpironium bromide gel Anticholinergic Primary axillary hyperhidrosis Stated the pivotal Cardigan 1 study of up to 350 patients; co-primary efficacy endpoints are the proportion of subjects achieving at least a 2-point improvement on the Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) scale and change in gravimetric sweat production
Can-Fite Biopharma Ltd., of Petach Tikva Piclidenoson  A3 adenosine receptor agonist Moderate to severe plaque psoriasis After an interim analysis, the independent data monitoring committee recommended the Comfort study continue with the original sample size and to drop 1 dose group
Can-Fite Biopharma Ltd., of Petach Tikva Piclidenoson  A3 adenosine receptor agonist Rheumatoid arthritis Independent data monitoring committee recommended not continuing the Acrobat study after an interim analysis
Eli Lilly and Co., of Indianapolis Reyvow (lasmiditan) Binds to 5-HT1F receptors Migraine In the Centurion study, patients taking 100 mg or 200 mg had 3.8 and 7.2 times greater odds, respectively, of achieving superior pain freedom at 2 hours post treatment compared to those taking placebo in at least 2 out of the 3 treated migraines
Gamida Cell Ltd., of Boston Omidubicel Stem/progenitor cell therapy Bone marrow transplant In addition to meeting its primary endpoint, as previously announced, the study met secondary endpoints related to platelet engraftment, infections and hospitalizations and clinical measures in bone marrow transplant; plans to submit a BLA in the fourth quarter of 2020
Phasebio Pharmaceuticals Inc., of Malvern, Pa. Bentracimab Monoclonal antibody fragment targeting ticagrelor Reversal of the antiplatelet effects of Brilinta (ticagrelor) Expanded the pivotal Reverse-It study into Canada; first patient enrolled and treated
Puma Biotechnology Inc., of Los Angeles Neratinib  EGFR antagonist; Erbb2/Erbb4 tyrosine kinase receptor inhibitor  HER2-positive, hormone receptor-positive, early stage breast cancer Data from the 2,840-patient Extenet study published in Clinical Breast Cancer showed HR+ patients who initiated treatment within a year of completing an adjuvant trastuzumab-containing treatment had an absolute 5-year invasive disease-free survival benefit vs. placebo of 5.1% and absolute 8-year overall survival benefit of 2.1% 
Santhera Pharmaceuticals Holding AG, of Pratteln, Switzerland Puldysa (idebenone) Oxidoreductase inhibitor Duchenne muscular dystrophy Discontinuing the Sideros study after an interim analysis by the independent data and safety monitoring board concluded that the study was unlikely to meet its primary endpoint; plans to withdraw the European marketing authorization application and end the global development program for Puldysa
Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London VIR-7831 Monoclonal antibody SARS-CoV-2 COVID-19 After reviewing phase II data, the independent data monitoring committee recommended the COMET-ICE study testing the early treatment of COVID-19 in patients who are at high risk of hospitalization move to phase III portion of the study

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