Company Product Description Indication Status
Phase I
Auris Medical Holding Ltd., of Hamilton, Bermuda  AM-201 (betahistine, intranasal) Histamine H1 receptor partial agonist; histamine H3 receptor antagonist Weight gain Phase Ib trial unaffected by COVID-19 pandemic; last participant received last visit in early March 2020 and top-line data expected in early May 2020
Bryn Pharma LLC, of Raleigh, N.C. Epinephrine (bi-dose nasal formulation) Adrenergic receptor agonist Anaphylaxis Intranasal (IN) delivery was comparable to intramuscular (IM) injection in pharmacokinetics, pharmacodynamics and safety in study that enrolled 95 healthy participants; 2 IN doses at 6.6 mg in same or opposite nostrils led to greater absorption vs. single IM dose at 0.3 mg; 2 IN doses at 6.6 mg in same nostril led to increased absorption vs. 2 IM doses at 0.3 mg in opposite legs
Compugen Ltd., of Holon, Israel COM-902  T cell immunoreceptor Ig ITIM protein inhibitor Advanced malignancies First participant dosed in dose-escalation trial in U.S. evaluating study drug as monotherapy
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. INO-4800 DNA vaccine COVID-19 infection Initial dosing in up to 40 healthy adults expected to begin on April 6, 2020, following FDA acceptance of IND application; participants will receive 2 doses 4 weeks apart, with initial immune responses and safety data expected in third quarter of 2020
Sun Biopharma Inc., of Minneapolis SBP-101  Caspase-3 stimulator Pancreatic cancer Enrollment paused due to COVID-19 pandemic in ongoing dose-escalation and expansion combination study with nab-paclitaxel and gemcitabine; currently enrolled participants will remain on treatment
Phase II
Aeterna Zentaris Inc., of Charleston, S.C. Macimorelin Ghrelin receptor agonist Growth hormone deficiency First of 2 studies in pediatric investigation plan in concurrence with EMA identified dose of 1 mg/kg for evaluation in second study to assess efficacy and safety as GHD diagnostic
American Brivision Holding Corp., of Fremont, Calif. ABV-1505 (BLI-1008) Noradrenaline transporter inhibitor Adult attention-deficit hyperactivity disorder  Site monitoring visit for ongoing part I, conducted at University of California, San Francisco Medical Center on March 10 and 11, 2020, confirmed 4 qualified adults were enrolled and dosed; first 2 expected to complete therapy on April 8 and April 11, 2020
Auris Medical Holding Ltd., of Hamilton, Bermuda AM-125 (betahistine, intranasal) Histamine H1 receptor partial agonist; histamine H3 receptor antagonist Acute peripheral vertigo Enrollment in Travers trial halted in March 2020 after sites reduced or suspended research activities due to COVID-19 pandemic; interim analysis following part A of trial now expected no sooner than third quarter of 2020
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 infection First 2 patients with mild to moderate disease dosed; 15 patients with severe disease also treated under FDA emergency IND application, with 2 extubated and 7-day results for first 10 expected by April 10, 2020
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 infection Phase IIb/III trial expected to enroll 342 participants with severe disease expected to begin dosing week of April 6, 2020, in 2-to-1 drug-to-placebo ratio; primary endpoint is mortality rate at 14 days; interim analysis expected on first 50 patients following 2 weeks of therapy
Millendo Therapeutics Inc., of Ann Arbor, Mich. Livoletide Ghrelin analogue Prader-Willi syndrome Phase IIb Zephyr study missed statistical significance in primary endpoint of change in hyperphagia and food-related behaviors; study showed improvements from baseline in HQ-CT scores of -4.7 (p=0.13) and -3.8 (p=0.45) for treated groups (60 µg/kg or 120 µg/kg, respectively) at 12 weeks vs. -2.8 for placebo; no positive trends seen for secondary endpoints of fat mass, body weight or waist circumference; company terminating program
Windtree Therapeutics Inc., of Warrington, Pa. Istaroxime Na+ K+ ATPase inhibitor Acute heart failure Post-hoc analysis of phase IIb data showed that, dosed at 0.5 µg/kg/min, study drug produced similar response on primary endpoint of E/e' (-5.32±4.21 vs. -3.90±5.19; p=0.373) and on stroke volume index (4.78±7.15 ml/beat/m2 vs. 5.83±6.37 ml/beat/m2; p=0.653) in Caucasian and Asian participants, respectively
Yiviva Inc., of New York YIV-906 IL-6/NF-kappaB/COX2/iNOS mediator Hepatocellular carcinoma First of 125 participants dosed in phase IIb combination study with sorafenib as first-line therapy in hepatitis B-positive disease; primary endpoint is progression-free survival, with secondary efficacy endpoints of time to progression, overall survival, objective response rate and disease control rate
Phase III
Axsome Therapeutics Inc., of New York AXS-07 (meloxicam + rizatriptan, fixed dose) Dual 5-HT 1b/1d receptor agonist; cyclooxygenase 2 inhibitor Migraine Intercept trial achieved co-primary endpoints of freedom from migraine pain (33% vs. 16% for placebo, p=0.002) and from most bothersome symptom (44% vs. 27% for placebo, p=0.003), both at 2 hours; study drug prevented progression of migraine pain beyond mild intensity from 2 to 24 hours in 74% of treated patients vs. 47% for placebo (p<0.001); return to normal functioning at 24 hours achieved in 74% of treated patients vs. 47% for placebo (p<0.001)
Catalyst Biosciences Inc., of South San Francisco Marzaa (marzeptacog alfa) Factor VIIa agonist Hemophilia A or B with inhibitors In accord with FDA and EMA feedback, open-label Crimson-1 study will enroll about 75 individuals who experience episodic bleeding to evaluate effectiveness of up to 3 doses in about 230 bleeding episodes compared with prior standard of care in similar number of episodes; primary endpoint will be hemostatic efficacy using standard 4-point assessment scale; trial expected to begin by year-end 2020
Immunomedics Inc., of Morris Plains, N.J. Sacituzumab govitecan Antibody-drug conjugate Triple-negative breast cancer Confirmatory Ascent study will be halted due to compelling evidence of efficacy, based on unanimous recommendation of independent data safety monitoring committee
Incyte Corp., of Wilmington, Del. Ruxolitinib JAK inhibitor Atopic dermatitis Data from both TruE-AD studies showed treatment with ruxolitinib cream has rapid, substantial and sustained impact on itch, a key quality of life measure, with significantly more patients vs. vehicle showing reduction in itch (NRS4) at week 8; in TruE-AD1, 40.4% in ruxolitinib 0.75% BID arm and 52.2% in 1.5% BID arm achieved NRS4 vs. 15.4% for vehicle (p<0.001 and p<0.0001, respectively); in TRuE-AD2, 42.7% in 0.75% BID arm and 50.7% in 1.5% BID arm achieved NRS4 vs. 16.3% for vehicle (p<0.0001)
Janssen Pharmaceutical Cos., of Beerse, Belgium, part of Johnson & Johnson Tremfya (guselkumab) Monoclonal antibody inhibiting IL-23 receptor Active psoriatic arthritis Data from Discover-1 and -2 studies published in The Lancet show that, at week 24, the primary endpoints achieved statistical significance in both studies (p<0.001 in Discover-1 for both 100 mg given every 4 weeks (q4w) and 100 mg given every 8 weeks (q8w); p<0.0001 in Discover-2 for both q4w and q8w groups); secondary endpoint data showed improvements with guselkumab vs. placebo on joint, skin, soft-tissue inflammation and composite measures of disease activity as well as patient-reported outcomes
Menlo Therapeutics Inc., of Bridgewater, N.J. Serlopitant Acts as an NK1 receptor antagonist Pruritus associated with prurigo nodularis Top-line results showed MTI-105 and MTI-106 studies did not meet respective primary endpoint of statistically significant reduction in pruritus vs. placebo based upon 4-point improvement responder analysis; in MTI-105, 26.45% in serlopitant group achieved 4-point or greater improvement on the worst-itch numeric rating scale, or WI-NRS, at week 10 compared to baseline vs. 20.31% on placebo (p=0.229); in MTI-106, 25.90% in serlopitant group achieved 4-point or greater improvement on WI-NRS at week 10 compared to baseline vs. 18.95% on placebo (p=0.158)
Oxthera AB, of Stockholm Oxabact  Bimodal enteric biotherapy containing lyophilized formulation of Oxalobacter formigenes Primary hyperoxaluria Completed enrollment in study designed to treat PH and prevent or delay kidney deterioration; top-line results expected in mid-2021

Notes

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

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