Company Product Description Indication Status
Phase I
Abivax SA, of Paris ABX-196 Invariant natural killer T-cell agonist Hepatocellular carcinoma Dosed first patient in U.S. phase I/II study testing combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.)
Actinium Pharmaceuticals Inc., of New York Actimab-A (225Ac-lintuzumab) CD33 antagonist Acute myeloid leukemia Dose-escalation combination trial with chemotherapy regimen 7+3 will be initiated in individuals with newly diagnosed disease who have intermediate or high-risk cytogenetic or molecular markers
Apeiron Biologics AG, of Vienna  APN-01 Angiotensin-converting enzyme 2 stimulator Coronavirus (COVID-19) infection Pilot investigator-initiated trial opened in China and expected to enroll 24 participants with severe infection for 7 days to assess impact on biological, physiologic and clinical outcomes, along with safety in those with severe SARS-CoV-2 infection, to determine whether phase IIb trial in larger patient population is warranted
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo Endotoxin-controlled hydroxy-propyl-beta-cyclodextrin Niemann-Pick disease type C1 Enrollment completed in extension protocol to phase I trial to evaluate safety and tolerability
GT Biopharma Inc., of Tampa, Fla. GTB-3550  Anti-CD16/IL-15/anti-CD33 NK cell engager Myelodysplastic syndromes; acute myeloid leukemia; advanced systemic mastocytosis First participant dosed in phase I portion of phase I/II study enrolling individuals with CD33-expressing, high-risk myelodysplastic syndromes, refractory/relapsed AML or advanced systemic mastocytosis
Ionctura SA, of Geneva IOA-244 PI3K-delta inhibitor Solid tumors First of about 60 participants with solid tumors that overexpress PI3K-delta dosed in dose-escalation and expansion study evaluating safety and preliminary efficacy of lead program
Seelos Therapeutics Inc., of New York SLS-002 (intranasal ketamine) NMDA receptor antagonist Suicidal ideation Additional interim data from pharmacokinetic/pharmacodynamic studies showed doses of 30 mg, 60 mg, 75 mg and 90 mg were generally safe and well-tolerated, with no discontinuations due to drug-related adverse events (AEs) and no serious AEs
Sellas Life Sciences Group Inc., of New York Galinpepimut-S Wilms tumor protein modulator; zinc finger protein transcription factor modulator Acute myeloid leukemia Final analysis of phase I/II data showed statistically significant median overall survival of 21 months, at median follow-up of 30.8 months, for study drug vs. previously reported 5.4 months in control arm of best standard care (p<0.02)
Phase II
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod Facioscapulohumeral muscular dystrophy Dual p38 MAP kinase alpha/beta inhibitor Enrollment completed in phase IIb Redux4 trial, with top-line data on primary endpoint of change from baseline on DUX4 activity in affected skeletal muscle expected in third quarter of 2020; open-label extension opened for those who completed 24 weeks of dosing 
Inflarx NV, of Jena, Germany IFX-1 Complement C5a factor inhibitor Pyoderma gangrenosum Initial data from ongoing open-label phase IIa study showed 2 of first 5 participants achieved complete remission; dose escalation warranted by pharmacodynamic analysis and approved by relevant authorities for expected enrollment of 18 people in 3 dose cohorts
Menlo Therapeutics Inc., of Redwood City, Calif. Serlopitant NK1 receptor antagonist Chronic pruritus Trial that enrolled 233 participants missed primary endpoint of statistically significant reduction in pruritus vs. placebo; 37.9% of those in serlopitant group (N=116) achieved 4-point or > improvement on worst-itch numeric rating scale at week 10 compared to baseline vs. 39.3% in placebo arm (N=117); no meaningful differences in secondary endpoints seen between study drug and placebo
Oramed Pharmaceuticals Inc., of Jerusalem ORMD-0801 (oral insulin) Insulin ligand; insulin receptor agonist Diabetes Top-line data from second and final cohort of phase IIb trial showed primary endpoints met and clinical benefits of lower dose confirmed; 0.95% (0.81% placebo adjusted) A1C reduction seen in those treated once daily at 8 mg; those with A1C readings > 9% at baseline who received 8 mg once daily experienced 1.26% reduction in A1C by week 12
Phase III
Akcea Therapeutics Inc., of Boston, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. AKCEA-TTR-LRx Antisense drug Transthyretin-mediated amyloid cardiomyopathy Initiated Cardio-TTRansform cardiovascular outcomes study; primary composite endpoint is cardiovascular (CV) mortality and frequency of CV clinical events comparing 2 study arms at week 120.
Amgen Inc., of Thousand Oaks, Calif.; Cytokinetics Inc., of South San Francisco; and Servier SAS, of Suresnes, France Omecamtiv mecarbil  Myosin stimulator Cardiac failure After second and final planned interim analysis, data monitoring committee recommended Galactic-HF trial that enrolled > 8,200 participants continue without changes; top-line results expected in fourth quarter of 2020
Bayer AG, of Leverkusen, Germany Vitrakvi (larotrectinib) TRK inhibitor Solid tumors Updated data published in The Lancet Oncology showed overall response rate (ORR) of 79%, including ORRs of 73% in adults, 92% in pediatric patients and 75% in those with brain metastases; median progression-free survival was 28.3 months
Exelixis Inc., of Alameda, Calif.   Cabometyx (cabozantinib )   Kinase inhibitor Thyroid cancer Reported enrollment of the first 100 patients in Cosmic-311 pivotal trial in patients with radioactive iodine-refractory differentiated disease who have progressed after up to 2 VEGF receptor-targeted therapies; analysis of first 100 patients for the co-primary endpoint of objective response rate, and an interim analysis of progression-free survival expected in the second half of 2020
Vanda Pharmaceuticals Inc., of Washington Tradipitant Neurokinin-1 receptor antagonist Pruritus in atopic dermatitis Epione trial in adults did not meet its primary endpoint in reduction of pruritus across overall study population; antipruritic effect of tradipitant was robust in  mild AD study population
Zynerba Pharmaceuticals Inc., of Devon, Pa. Zygel CBD Gel Cannabidiol Fragile X syndrome Completed enrollment in 14-week pivotal Connect-FX trial testing efficacy and safety for treating behavioral symptoms of fragile X in 210 children, ages 3-17, with full mutation FXS; top-line results expected in the second quarter of 2020; primary endpoint is change from baseline to the end of treatment period in the Aberrant Behavior Checklist-Community FXS Specific Social Avoidance subscale


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