Company Product Description Indication Status
Phase I
Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Mast cell gastrointestinal disease 6-month treatment resulted in 64% mean reduction in Total Symptom Score (TSS)-8 compared to baseline; 5 of 7 participants had > 50% reduction in TSS-8
Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Eosinophil/mast cell-related diseases Placebo-controlled trial initiated to evaluate subcutaneously administered formulation in healthy volunteers
Mind Medicine Inc., of New York 18-MC (18-methoxycoronaridine) Alpha-3-beta-4 nicotinic receptor antagonist  Opioid use disorder Enrollment initiated in additional study in adult volunteers
Phase II
Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Eosinophilic esophagitis Initiated phase II/III trial expected to enroll ~300 participants with active disease, randomized 1-to-1-to-1 to 6 doses of study drug 1 mg/kg monthly, 1 mg/kg of study drug for first month followed by 5 doses of 3 mg/kg monthly or monthly placebo; co-primary endpoints are proportion of those achieving ≤6 eosinophils in single high-powered field and absolute change in dysphagia symptoms
Cassava Sciences Inc., of Austin, Texas PTI-125 Filamin A modulator Alzheimer's disease Initiated open-label extension study to monitor long-term safety and tolerability of study drug, dosed at 100 mg twice daily, for 12 months on ~100 individuals with mild to moderate disease, including those from prior studies; sites may initially slow pace of enrollment to minimize risk of exposure to coronavirus
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. TSC (trans sodium crocetinate) Vitamin A analogue Acute stroke Enrollment delay expected in ambulance-based PHAST-TSC trial due to coronavirus
Eloxx Pharmaceuticals Inc., of Waltham, Mass. ELX-02 CFTR gene modulator; ribosomal protein modulator Cystic fibrosis Enrollment in phase II program temporarily paused due to COVID-19 pandemic
Immutep Ltd., of Sydney Eftilagimod alfa  HLA class II antigen stimulator Metastatic breast cancer Phase IIb Aipac trial in HER2-negative/HR+ disease reported progression-free survival at 6 months (end of active therapy) of 63% for study drug plus paclitaxel vs. 54% for paclitaxel plus placebo, for unadjusted HR of 0.93; secondary endpoint of overall response rate was 48.3% for study drug vs. 38.4% for placebo; overall survival data expected in late 2020
Opko Health Inc., of Miami Rayaldee (calcifediol) Vitamin D prohormone Secondary hyperparathyroidism Interim results from ongoing trial in 20 adults with stage 5 chronic kidney disease requiring hemodialysis and with vitamin D insufficiency who completed at least 3 months of therapy showed no significant changes in serum calcium and phosphorus or increased incidence of adverse events; serum levels of active metabolite calcitriol were elevated; ≥30% decreases in intact parathyroid hormone were seen vs. baseline; full enrollment expected in second quarter of 2020, with top-line data expected in third quarter
Phase III
Allakos Inc., of Redwood City, Calif. Antolimab (AK-002) Sialic acid-binding Ig-like lectin 8 inhibitor Eosinophilic gastritis; eosinophilic duodenitis Initiated trial expected to enroll ~160 participants with active, biopsy-confirmed EG and/or EoD, randomized 1-to-1 to 1 mg/kg of study drug for first month followed by 5 doses of 3 mg/kg monthly or monthly placebo; co-primary endpoints are proportion achieving ≤ 4 eosinophils in 5 high-powered fields (hpfs) in stomach and/or ≤15 eosinophils in 3 hpfs in duodenum and absolute change in Total Symptom Score
Avadel Pharmaceuticals plc, of Dublin FT-218 (sodium oxybate) GABA B receptor agonist Narcolepsy Last visit completed in Rest-On study that enrolled 212 participants to assess effectiveness of study drug in Micropump controlled-release formulation to treat excessive daytime sleepiness and cataplexy under FDA special protocol assessment; top-line data expected in second quarter of 2020
H. Lundbeck A/S, of Copenhagen, Denmark Vyepti (eptinezumab-jjmr) CGRP receptor antagonist Chronic migraine Data published in Neurology showed pivotal Promise 2 study met primary endpoint of statistically significant reductions in mean monthly migraine days (MMDs) after first dose over months 1 to 3; preventive effects of recently approved drug were shown as early as day 1 post-infusion and sustained throughout study; one-third of those who took drug experienced 75% or > reduction in number of mean MMDs at month 1
Onconova Therapeutics Inc., of Newtown, Pa. Rigosertib RAS mimetic Myelodysplastic syndrome Completed planned enrollment of 360 higher-risk patients in Inspire trial
Phase IV
Opko Health Inc., of Miami Rayaldee (calcifediol) Vitamin D prohormone Secondary hyperparathyroidism Interim results from ongoing trial comparing study drug with 3 common treatment regimens in adults with stage 3 or 4 chronic kidney disease and vitamin D insufficiency showed that daily dose of 60 mcg of Rayaldee was only tested regimen that reliably raised serum total 25-hydroxyvitamin D to range of 50 ng/mL to 100 ng/mL; final study results expected in second half 2020

Notes

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