Company Product Description Indication Status
Phase I
Adamis Pharmaceuticals Corp., of San Diego Zimhi (naloxone) Opioid antagonist Healthy volunteers Data published in the Journal of Addiction Research and Adolescent Behavior showed intramuscular Zimhi produced higher levels of naloxone with more rapid absorption compared to intranasal Narcan (naloxone, Emergent Biosolutions Inc.)
Crispr Therapeutics AG, of Zug, Switzerland, and Vertex Pharmaceuticals Inc., of Cambridge, Mass. CTX-001  CRISPR/Cas9 gene-editing therapy Transfusion-dependent beta thalassemia and severe sickle cell disease TDT patient was transfusion independent 9 months after treatment with total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin and 99.8% F-cells (erythrocytes expressing fetal hemoglobin); SCD patient was free of vaso-occlusive crises at 4 months with total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin and 94.7% F-cells
Moleculin Biotech Inc., of Houston WP-1220   Inhibitor of p-STAT3 Cutaneous T-cell lymphoma Data published in Blood showed 3 patients had a median reduction of 70.8% in their CAILS scores on day 84; fourth ongoing patient had a 26.7% reduction after 56 days
Phase II
Antisense Therapeutics Ltd., of Melbourne, Australia ATL-1102  Inhibitor of CD49d expression  Duchenne muscular dystrophy A majority of the 7 patients had either increases or no change in their Performance of Upper Limb Test (PUL2.0) scores from baseline after 24 weeks of treatment
Faraday Pharmaceuticals Inc., of Seattle FDY-5301  Catalytically destroys hydrogen peroxide Reperfusion injury following ST elevation myocardial infarction Median infarct size (as a proportion of left ventricle) was 8.5% for patients treated with 2 mg/kg FDY-5301 compared to 14.9% for those on placebo; median left ventricular ejection fraction was 63.2% for 2 mg/kg FDY-5301 and 53.9% for placebo
Oramed Pharmaceuticals Inc., of New York ORMD-0801  Oral insulin capsule Type 2 diabetes Drug reduced HbA1c by 0.60% compared to a 0.06% reduction for placebo (p=0.036)
Scholar Rock Holding Corp., of Cambridge, Mass. SRK-015 Inhibitor of the activation of myostatin Spinal muscular atrophy Dose-dependent increases of up to 100-fold in serum latent myostatin after treatment of 2-mg/kg and 20-mg/kg doses of SRK-015; increases in serum latent myostatin levels in the first 4 weeks for patients were comparable to phase I study in healthy volunteers; preliminary pharmacokinetic profile exhibits dose proportionality and low variability; interim efficacy data expected in the first half of 2020
Viking Therapeutics Inc., of San Diego VK-2809 Liver-selective thyroid hormone receptor beta agonist Biopsy-confirmed nonalcoholic steatohepatitis Started study of about 340 patients comparing 4 dose regimens to placebo; primary endpoint is relative change in liver fat content from baseline to week 12; study will also evaluate histologic changes via hepatic biopsy after 52 weeks of dosing
Phase III
Allergan plc, of Dublin Ubrogepant Oral CGRP receptor antagonist Acute migraine Achieve II results published in The Journal of the American Medical Association showed treatment vs. placebo led to significantly greater rates of pain freedom at 2 hours with both 50-mg and 25-mg doses, and freedom from most bothersome migraine-associated symptom at 2 hours with 50-mg dose
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular risk reduction Reduce-IT subgroup analysis showed risk reductions in U.S. patients treated with 4 g/day vs. placebo across prespecified composite and individual primary and secondary endpoints, including 31% relative risk reduction and 6.5% absolute risk reduction in first occurrence of 5-point major adverse cardiovascular events 
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular risk reduction At prespecified 9-month interim analysis, Evaporate study showed slowing of noncalcified plaque (sum of LAP, fibrofatty and fibrous plaque) vs. placebo (35% vs. 43%, p=0.010), total plaque (noncalcified + calcified plaque) (p=0.0004), fibrous plaque (15% vs. 26%, p=0.011) and calcified plaque (-1% vs. 9%, p=0.001), after adjustment by baseline plaque, age, sex, diabetes status, baseline triglyceride levels and statin use but no significant change in primary endpoint of low attenuation plaque vs. placebo (74% vs. 94%, p=0.469); investigator-initiated study continuing to full 18-month review
Biondvax Pharmaceuticals Ltd., of Jerusalem M-001 Multimeric, multiepitope, single-protein, universal flu vaccine Influenza virus infection Full enrollment and randomization of 12,463 participants reached in pivotal trial, with results due by year-end 2020
Mezzion Pharma Co. Ltd., of Seoul, South Korea Udenafil  PDE5 inhibitor Congenital single ventricle heart disease In Fuel study that enrolled 400 adolescents with SVHD, those who took study drug over 6-month period showed improvement in exercise capacity measured by oxygen consumption at ventilatory anerobic threshold (VO2 at VAT); work rate at VAT and ventilatory equivalents of carbon dioxide at VAT also improved; peak VO2 improved but did not reach statistical significance
Morphosys AG, of Planegg, Germany Tafasitamab  B-lymphocyte antigen CD19 modulator Diffuse large B-cell lymphoma B-Mind study of tafasitamab with rituximab plus bendamustine passed preplanned, event-driven interim analysis for futility, and independent data monitoring committee recommended increasing the number of participants with relapsed/refractory disease from 330 to 450
Myovant Sciences Ltd., of Basel, Switzerland Relugolix GnRH receptor antagonist Advanced prostate cancer Hero study met primary efficacy endpoint, with 96.7% of participants achieving sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks; study achieved all 6 key secondary endpoints, including superiority to leuprolide acetate on rapid suppression of testosterone and prostate-specific antigen, all with "p" values < 0.0001
Santhera Pharmaceuticals AG, of Pratteln, Switzerland Idebenone Oxidoreductase inhibitor Duchenne muscular dystrophy Syros data showed long-term efficacy in slowing respiratory function loss in individuals with DMD under routine clinical care; published in Neuromuscular Disorders 


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