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Phase II clinical data reported in March 2020

Company Product Description Indication Phase II status


Aveo Oncology Inc., of Cambridge, Mass. Ficlatuzumab Hepatocyte growth factor inhibitory antibody  Relapsed/refractory acute myeloid leukemia CyFi-2 study discontinued due to COVID-19 pandemic
Blueprint Medicines Corp., of Cambridge, Mass. Ayvakit (avapritinib) KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist Indolent systemic mastocytosis Part 1 of Pioneer trial in 39 participants showed statistically significant mean decline of ~30% in total symptom score at 16 weeks, measured by Indolent SM Symptom Assessment Form, across all dose cohorts (25 mg, 50 mg and 100 mg) vs. ~3% in placebo cohort (p=0.001); reductions in scores deepened over time and, at cutoff date, 37 participants remained on study with median follow-up of 18 weeks; declines also seen in serum tryptase, bone marrow mast cells and KIT D816V allele burden and improvements in patient-reported quality of life; 25 mg once daily selected as part 2 dose
Can-Fite Biopharma Ltd., of Petah-Tikva, Israel Namodenoson Adenosine A3 receptor agonist; phosphatidylinositol 3 kinase subunit 3 inhibitor Hepatocellular cancer Enrolled 7 patients for the compassionate use program in HCC; no severe adverse events reported; 2 patients from former phase II study still undergoing treatment with namodenoson, each with an overall survival of >2.5 years
Delmar Pharmaceuticals Inc., of San Diego VAL-083 Adjuvant treatment (pre-temozolomide maintenance therapy)  MGMT-unmethylated glioblastoma multiforme Exceeded 50% enrollment in the adjuvant arm
Hutchison China Meditech Ltd. (Chi-Med), of Hong Kong
FGF1/FGF2/FGF3 receptor antagonist Mesothelioma Single-arm study initiated in individuals with advanced malignant disease who failed at least 1 line of systemic therapy; primary outcome measure is overall response rate; secondary outcome measures include disease control rate, time to and duration of response, and progression-free and overall survival
Ideaya Biosciences Inc., of South San Francisco IDE-196 Protein kinase C inhibitor Metastatic uveal melanoma and GNAQ/GNA11 hotspot mutation solid tumors Selected monotherapy dose of 400 mg twice daily (with 1-week 200-mg twice-daily run-in) and achieved first patient in for GNAQ/GNA11 non-MUM basket trial
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
Innovent Biologics Inc., of Suzhou, China Pemigatinib (IBI-375) FGFR1/2/3 inhibitor Advanced cholangiocarcinoma First patient dosed in registrational trial, enrolling patients with FGFR2 fusions or rearrangements who have progressed from at least 1 prior systemic therapy in China
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. VGX-3100 DNA medicine Anal dysplasia Interim results from open-label study showed VGX-3100 to be safe and effective; o 20 subjects who had results at the time of data review, 50% showed clearance of HPV-16/18-associated precancerous lesions and 75% demonstrated an overall decrease in the number of lesions 6 months after the start of treatment; no cases of anal cancer have been observed in the trial
ISA Pharmaceuticals BV, of Leiden, the Netherlands ISA-101b HPV16-specific vaccine Cervical cancer CervISA combination study with carboplatin/paclitaxel in 77 people with late-stage, HPV16-positive disease, published in Science Translational Medicine, showed longer (p=0.012) overall survival (OS) in those with good immune response (median OS 16.8 months) vs. weak response (median OS 11.2 months); 11 of 14 alive at end of study showed strong vaccine-induced response, including 9 stage IVa-IVb individuals with mean OS of 3 years
Nantkwest Inc., of Culver City, Calif. CD16-targeted natural killer (haNK) cells Allogeneic NK cell therapy Merkel cell carcinoma First of 33 participants with second- and third-line disease refractory to immune checkpoint inhibitors dosed in single-arm combination trial with super agonist N-803 (Immunitybio Inc.) and avelumab (Bavencio, EMD Serono Inc./Pfizer Inc.)
Oncolys Biopharma Inc., of Tokyo, and Chugai Pharmaceutical Co. Ltd., of Tokyo Telomelysin (OBP-301) Genetically modified type 5 adenovirus Locally advanced esophageal cancer Chugai enrolled first patient testing combination with radiotherapy in patients not eligible for surgical excision and definitive chemoradiotherapy
Oncopeptides AB, of Stockholm Melphalan flufenamide Peptide-drug conjugate Multiple myeloma Drug attained an ORR of 30% in patients with relapsed refractory MM, as assessed by the independent review committee, in the intent-to-treat analysis of the pivotal open-label Horizon study
Oncoquest Inc., of Edmonton, Alberta Oregovomab Anti-CA-125 antibody Ovarian cancer Report in Gynecologic Oncology at median of 42 months follow-up showed risk of progression and death were reduced by > 50% with study drug plus carboplatin and paclitaxel vs. carboplatin/paclitaxel alone; report in Cancer Immunology, Immunotherapy from same study showed chemoimmunotherapy combo increased presence of CA125-specific CD8+T lymphocytes measured in peripheral blood compared to chemotherapy, correlating with clinical outcomes
QED Therapeutics Inc., of San Francisco, a subsidiary of Bridgebio LLC Infigratinib (BGJ-398) Oral FGFR1-3 selective tyrosine kinase inhibitor Solid tumors Patients dosed in trial testing drug in patients whose disease is positive for FGFR1-3 gene fusions/translocations or other FGFR alterations; open-label study to assess overall response rate as primary endpoint
Redhill Biopharma Ltd., of Tel Aviv, Israel Yeliva (opaganib) Sphingosine kinase 2 inhibitor Prostate cancer Investigator-sponsored study initiated at Medical University of South Carolina evaluating study drug in up to 60 participants with metastatic castration-resistant disease progressing during treatment with androgen-signaling blockers
Sellas Life Sciences Group Inc., of New York Nelipepimut-S in combination with granulocyte-macrophage colony-stimulating factor Cancer vaccine Ductal carcinoma in situ of the breast Drug shown capable of inducing an antigen-specific antitumor immune response in DCIS patients even after a single vaccination; trial enrolled patients who were HLA-A2+ or A3+ positive, express HER2 at IHC 1+, 2+, or 3+ levels, and are pre- or post-menopausal
Theralase Technologies Inc., of Toronto TLD-1433 Light-activated photodynamic compound Non-muscle invasive bladder cancer Notified by all of its Canadian clinical study sites that enrollment of new patients and conducting the first treatment procedure has been temporarily placed on hold pending resolution of the COVID-19 global pandemic
Vaccitech Ltd., of Oxford, U.K. VTP-800 Recombinant virus-based vaccine Prostate cancer In phase IIa Advance combination study with anti-PD-1 in 23 men with metastatic castration-resistant disease, 5 (22%) had > 50% reduction in PSA level at any timepoint compared to baseline (median of 88 ng/ml); 4 of these maintained that response when tested 3 weeks later; 1 with response had PSA level of 0 at 24 weeks
VBI Vaccines Inc., of Cambridge, Mass. VBI-1901 GM-CSF receptor agonist Glioblastoma First of about 20 participants with first recurrent disease dosed in phase IIa study, in combination with AS-01B (Glaxosmithkline plc) adjuvant or GM-CSF; initial immunologic data from adjuvant arm expected in fourth quarter 2020
Company Product Description Indication Phase II status


Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 Free fatty acid formulation of omega-3 pentaenoic acid Elevated triglycerides Started Enhance-IT study testing drug against Vascepa (icosapent ethyl, Amarin Corp. plc) in reducing triglyceride levels and other lipid markers; trial also will gather data on bioavailability and blood levels of eicosapentaenoic acid and other omega-3 fatty acids
Phasebio Pharmaceuticals Inc., of Malvern, Pa. PB-1046 Sustained-release vasoactive intestinal peptide analogue Pulmonary arterial hypertension Pausing enrollment in study due to COVID-19
Tenax Therapeutics Inc., of Morrisville, N.C. Levosimendan Calcium sensitizer Pulmonary hypertension and heart failure with preserved ejection fraction Completed enrollment in Help trial; primary endpoint is change from baseline in pulmonary capillary wedge pressure with bicycle exercise (25 watts) at week 6 following 5weekly infusions; top-line data expected in second quarter of 2020
Company Product Description Indication Phase II status


Asana Biosciences LLC, of Lawrenceville, N.J. Gusacitinib Dual SYK/JAK kinase inhibitor Atopic dermatitis Achieved primary endpoint of a statistically significant reduction in the Eczema Area and Severity Index score at the 60-mg and 80-mg doses vs. placebo at week 12
Mediwound Ltd., of Yavne, Israel Escharex Topical enzyme proteolytic therapy Venous leg ulcers Enrollment suspended due to COVID-19 outbreak
Palvella Therapeutics Inc., of Wayne, Pa. PTX-022 (Qtorin 3.9% rapamycin anhydrous gel) mTOR inhibitor Pachyonychia congenita Completed enrollment in phase II/III Valo study in adults; top-line results expected in fourth quarter of 2020
Company Product Description Indication Phase II status


Matinas Biopharma Holdings Inc., of Bedminster, N.J. MAT-9001 (icosapent + docosapentaenoic acid + heneicosapentaenoic acid) Omega-3 fatty acid-based therapeutic Hypertriglyceridemia Enrollment paused in Enhance-It comparative study vs. Vascepa (icosapent ethyl, Amarin Corp. plc)
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
Synlogic Inc., of Cambridge, Mass. SYNB-1618 Synthetic biotic that consumes phenylalanine Phenylketonuria Enrollment expected to be delayed due to COVID-19
Zealand Pharma A/S, of Copenhagen Dasiglucagon Glucagon analogue Hypoglycemia after gastric bypass bariatric surgery Patients receiving 80 ?g and 200 ?g dasiglucagon spent an average of 27.5 minutes and 14 minutes in post-meal hypoglycemia (plasma glucose <3.9 mmol/L), compared to 62 minutes for placebo (p<0.05 vs. placebo for both)
Company Product Description Indication Phase II status


Akero Therapeutics Inc., of San Francisco AKR-001 FGF receptor agonist; FGF-21 ligand Nonalcoholic steatohepatitis All dose groups (28 mg, 50 mg, 70 mg) in phase IIa Balanced study met primary endpoint of absolute change from baseline in liver fat (12.3%, 13.4% and 14.1%, respectively), measured by MRI-PDFF at week 12
Albireo Pharma Inc., of Boston Elobixibat Ileal sodium bile acid cotransporter inhibitor Nonalcoholic fatty liver disease; nonalcoholic steatohepatitis Enrollment nearly complete; top-line data expected by mid-2020
Albireo Pharma Inc., of Boston Elobixibat Ileal bile acid transporter inhibitor Nonalcoholic steatohepatitis/fatty liver disease Achieved full patient enrollment; on track to report top-line data mid-2020
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
Bausch Health Companies Inc., of Laval, Quebec Rifaximin (soluble solid dispersion) RNA polymerase inhibitor Overt hepatic encephalopathy Top-line data showed study drug dosed at 40 mg twice daily with standard-of-care lactulose met primary endpoint of time to OHE resolution and was statistically significantly superior to placebo plus standard of care, with median resolution time of 21.1 hours vs. 62.7 hours, respectively
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Nonalcoholic steatohepatitis; nonalcoholic fatty liver disease Enrollment paused in pharmacokinetics/pharmacodynamics study in pediatric patients with NASH and in separate study in adults with NAFLD
Inventiva SA, of Daix, France Lanifibranor Oral small molecule that actives all 3 PPAR isoforms Nonalcoholic steatohepatitis Last visit of last patient out of 247 enrolled in phase IIb trial; publication of headline results expected in June 2020
Leading Biosciences Inc., of Carlsbad, Calif. LB-1148 (tranexamic acid) Serine protease inhibitor Bowel function Top-line data showed study drug provided 30% improvement in time to normal bowel function following cardiovascular surgery (p<0.001) vs. placebo, resulting in 1.1-day reduction in average ICU length of stay (LOS) and 1.2-day reduction in average hospital LOS
Orphomed Inc., of San Francisco ORP-101 (dibuprenorphine ethyl ether) Opioid receptor kappa antagonist/mu partial agonist Irritable bowel syndrome with diarrhea Enrollment paused due to COVID-19 pandemic
Seres Therapeutics Inc., of Cambridge, Mass. SER-287 Ecobiotic (encapsulated purified suspension of eubacterial spores) Ulcerative colitis Phase IIb study about 60% enrolled based on 201-patient target size but development adversely affected by clinical sites halting non-essential procedures, including endoscopies, due to COVID-19; trial may not achieve original enrollment target in second half of 2020
Company Product Description Indication Phase II status

Genitourinary/sexual function

Obseva SA, of Geneva OBE-022 PGF2 alpha antagonist Premature labor Data from phase IIa Prolong trial expected to report in second half of 2020, as planned
Company Product Description Indication Phase II status


Cellphire Inc., of Rockville, Md. Thrombosomes Platelet-derived hemostatic agent Thrombocytopenia FDA approved start of trial assessing multiple doses in bleeding patients with thrombocytopenia due to primary bone marrow disorders or chemotherapy, immunotherapy radiation therapy and/or refractory response to fresh platelet concentrate transfusion, with or without splenomegaly
Company Product Description Indication Phase II status


Hansa Biopharma AB, of Lund, Sweden Imlifidase Recombinant cysteine protease Kidney transplant of crossmatch-positive patients Of the 46 transplanted patients treated with the drug, 96% had a functioning graft after 6 months; 2-year death censored graft survival was 89% for 27 patients; overall graft survival was 80% for 30 patients
Orchard Therapeutics plc, of London OTL-103 WASP gene stimulator Wiskott-Aldrich syndrome Enrollment timelines in ongoing trial expected to shift by at least 3 months due to COVID-19 pandemic
Company Product Description Indication Phase II status


Algernon Pharmaceuticals Inc., of Vancouver, British Columbia Ifenprodil NDMA receptor glutamate antagonist COVID-19 CRO Novotech identified physicians in South Korea who agreed to conduct investigator-initiated trial of repurposed agent, approved to treat certain neurological conditions; development of study protocol underway
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 Submitted an IND to the FDA to test the drug in patients with mild to moderate respiratory illness after contracting the novel coronavirus; study will measure combined total symptom score for fever, myalgia, dyspnea and cough, as the primary endpoint
Cytodyn Inc., of Vancouver, Wash. Leronlimab CCR5 antagonist COVID-19 3 more patients treated, bringing total to 10
Moderna Inc., of Cambridge, Mass. mRNA-1647 mRNA vaccine Cytomegalovirus Enrollment complete for all 3 dose cohorts; first interim analysis expected in third quarter of 2020
Qurient Co. Ltd., of Seongnam, South Korea Telacebec Ubiquinol cytochrome C reductase 14 kDa inhibitor Tuberculosis Data from trial, now published in The New England Journal of Medicine, support continued development of telacebec, which could become the first universal regimen to overcome tuberculosis regardless of drug resistance status
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris  Kevzara (sarilumab) IL-6 receptor antagonist COVID-19 U.S. phase II/III trial initiated and will begin enrolling patients hospitalized with severe COVID-19 infection
Revive Therapeutics Ltd., of Toronto Bucillamine  Antioxidant COVID-19 Plans to run a clinical trial testing the drug in the U.S. and in Asia-Pacific countries
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Kevzara (sarilumab) Monoclonal antibody targeting interlukin-6 COVID-19 First patient treated in an ex-U.S. phase II/III trial
Company Product Description Indication Phase II status


Immunovant Inc., of New York IMVT-1401 Fully human monoclonal antibody targeting the neonatal Fc receptor Thyroid eye disease 65% mean reduction in total IgG was observed from baseline to end of treatment, with a pharmacodynamic response nearly identical to modeled predictions for dosing regimen tested in trial
Orthotrophix Inc., of Oakland, Calif. TPX-100 Matrix extracell phosphoglycoprotein modulator Osteoarthritis In participants with mild to severe disease, knees treated with study drug showed statistically significant improvement vs. placebo in functions based on 17 measures in combined KOOS score and WOMAC scales; benefits seen by 6 months (p=0.02) and maintained through 12 months (p=0.04); pathological 3D shape changes in femur in treated knees also were reduced vs. placebo by 6 months (p=0.01) and maintained through 12 months (p=0.02)
Company Product Description Indication Phase II status


Acceleron Pharma Inc., of Cambridge, Mass. ACE-083 GDF-8 antagonist Charcot Marie Tooth disease Did not achieve statistically significant improvements in functional endpoints vs. placebo; development discontinued
American Brivision Corp., of Fremont, Calif. ABV-1505 Inhibitor of the norepinephrine transporter Adult attention deficit hyperactivity disorder First patient enrolled
Auris Medical Holding Ltd., of Hamilton, Bermuda AM-125 Intranasal betahistine  Acute peripheral vertigo In addition to the ongoing intranasal dose-escalation portion, open-label testing of oral betahistine for reference purposes has commenced
Axsome Therapeutics Inc., of New York AXS-05 (dextromethorphan + bupropion) NMDA receptor antagonist; nicotinic acetylcholine receptor antagonist; opioid receptor sigma agonist 1 Alzheimer’s disease agitation Accelerating the completion of the Advance-1 phase II/III trial; top-line results now expected in early second quarter of 2020
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
Cortexyme Inc., of South San Francisco COR-388 Gingipain inhibitor Alzheimer’s disease Phase II/III Gain trial enrollment has reached 300 of target 570 patients; top-line results expected in Q4 2021
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
Engage Therapeutics Inc., of Summit, N.J. Staccato alprazolam Inhaled formulation of benzodiazepine Active epileptic seizure Phase IIb States study met primary endpoint, with proportion of responders (50 of 76) achieving cessation of seizure activity within 2 minutes of treatment administration and no recurrence within 2 hours; phase III study expected to start in 2020
Irlab Therapeutics AB, of Stockholm IRL-752 5-HT 7 receptor antagonist; alpha adrenoceptor antagonist Parkinson's disease Results of phase IIa study, published in Movement Disorders, show potential for study drug to improve balance and reduce fall risk by increasing levels of norepinephrine and dopamine in frontal cortex and activating genes involved in nerve cell connections
Neuraly Inc., of Germantown, Md. NYL-01 Glucagon-like peptide 1 receptor agonist Parkinson’s disease Dosed first of about 240 patients in the study to test the safety, tolerability and efficacy of the drug
Ovid Therapeutics Inc., of New York Soticlestat (OV-935/TAK-935) Cholesterol 24-hydroxylase inhibitor CDKL5 deficiency disorder and Dup15q syndrome Of the 5 patients with CDKL5 deficiency disorder, 3 patients had 45%, 61% and 63% fewer motor seizures during the maintenance phase; of the 6 patients with Dup15q syndrome, the 1 patient with pure motor seizures saw a 90% reduction in seizure frequency during the maintenance phase; of the other 5 Dup15q patients with mixed seizure types, 3 patients with myoclonic seizures showed a 60%, 66% and 100% reduction over the maintenance period and 2 patients had absence seizures and showed a 78% and 74% reduction during the maintenance phase
Theranexus SAS, of Lyon, France THN-102 Combination of modafinil and flecainide Parkinson’s disease Data showed trial in 75 patients with debilitating, excessive daytime sleepiness (EDS) met primary endpoint and significantly increased the proportion of patients no longer suffering from EDS; doses of 200 mg modafinil/2 mg flecainide showed significant superiority vs. placebo in reducing EDS measured using Epworth Sleepiness Scale, with improvements of 3.9 points in patients after treatment vs. 2.4 points for placebo (p=0.01)
Uniqure NV, of Amsterdam, the Netherlands AMT-130 HTT gene inhibitor Huntington's chorea First 2 patients are enrolled, but procedures have been temporarily postponed due to the expanding impact of the COVID-19 pandemic
Vivoryon Therapeutics AG, of Halle, Germany PQ-912 Small-molecule inhibitor of glutaminyl cyclase Early Alzheimer's disease Trial has added exploratory parameters selected with the rationale of advancing less invasive diagnostic technologies, including Winterlight Labs speech assessment, the use of EEG to test neuronal network activity and connectivity, as well as a set of blood-based biomarkers
Xenon Pharmaceuticals Inc., of Burnaby, British Columbia XEN-1101 Dual KCNQ voltage-gated potassium channel-2/channel-3 stimulator Focal epilepsy Top-line data now expected in first half of 2021 due to slower enrollment related to COVID-19 pandemic; blinded safety data to date suggest study drug well-tolerated, with discontinuation rate lower than modeled
Company Product Description Indication Phase II status


Graybug Vision Inc., of Redwood City, Calif. GB-102 Small-molecule multiple receptor tyrosine kinase inhibitor, sunitinib malate, in microparticle formulation Anti-VEGF-responsive wet age-related macular degeneration Enrollment closed early
Horizon Therapeutics plc, of Dublin Tepezza (teprotumumab-trbw) Monoclonal antibody targeting insulin-like growth factor 1 receptor Thyroid eye disease Pooled efficacy data from phase II and phase III studies showed drug effectively reduces proptosis (eye bulging) in patients with TED regardless of age, gender and smoking status; at week 24 of treatment, across all subgroups, significantly more patients receiving Tepezza (n=84) experienced an improvement of at least 2 mm in proptosis vs. placebo (n=87) (p<0.001 for all)
Opthea Ltd., of Melbourne, Australia OPT-302 Blocks activity of VEGF-C and VEGF-D Diabetic macular edema Completed patient dosing and week 12 visits in the study testing OPT-302 plus Eylea (aflibercept, Regeneron Pharmaceuticals Inc.)
Proqr Therapeutics NV, of Leiden, the Netherlands QR-1123 RHO gene modulator Retinitis pigmentosa Enrollment paused due to COVID-19 pandemic
Santen Pharmaceutical Co. Ltd., of Osaka, Japan, and Tracon Pharmaceuticals Inc., of San Diego DE-122 (carotuximab) Endoglin inhibitor Wet age-related macular degeneration Discontinued development in wet AMD after top-line data indicated DE-122 did not improve visual acuity when combined with Lucentis (ranibizumab) as compared to single-agent Lucentis treatment
Company Product Description Indication Phase II status


Cytodyn Inc., of Vancouver, Washington Leronlimab CCR5 antagonist  Graft-vs.-host disease First patient treated
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. VGX-3100 DNA medicine that works by stimulating a specific immune response to HPV-16 and HPV-18 Anal dysplasia Cleared high-risk HPV 16/18-associated precancerous lesions in 50% of patients, and reduced the number of lesions in 75% of patients
Sensorion SA, of Montpellier, France SENS-401 Designed to protect and preserve inner ear tissue from damage that can cause progressive or sequelar hearing impairment Sudden sensorineural hearing loss Patient recruitment rates indicate data will be available by midyear 2021, later than previously announced
Company Product Description Indication Phase II status


Bellus Health Inc., of Laval, Quebec BLU-5937 P2X3 antagonist Chronic cough Dose-escalation, placebo-controlled Relief trial fully enrolled 68 participants, and 75% completed dosing; top-line data due in mid-2020
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
Faron Pharmaceuticals Oy, of Turku, Finland Traumakine  CD73 agonist; interferon-beta ligand Acute respiratory distress syndrome FDA accepted the protocol design for the next study, which will exclude the concomitant use of glucocorticoids; pilot study will enroll 60 patients with data used to adjust size of pivotal study
Healios K.K., of Tokyo HLCM-051 Stem cells Acute respiratory distress syndrome Continues to make enrolment progress in Japan; in One-Bridge study, inclusion criteria allow patients who develop pneumonia-induced ARDS caused by COVID-19
Synairgen plc, of Southampton, U.K. SNG-001 (inhaled interferon beta) Interferon-beta ligand COVID-19 First participant dosed at initial trial site
Verona Pharma plc, of London Ensifentrine (pressurized metered-dose inhaler) Dual PDE3/PDE4 inhibitor Chronic obstructive pulmonary disease Part A single-dose portion of trial in moderate to severe disease showed statistically significant increase in lung function vs. placebo, measured by FEV1; improvements in peak FEV1 corrected for placebo showed general dose response (ranging from 47 mL to 391 mL, p<0.05 for doses 300 µg and >); improvements in average FEV1 over 4 hours corrected for placebo showed general dose response and over 12 hours showed dose response and durability of effect over dosing interval; enrollment in part B delayed due to COVID-19 pandemic

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