Company Product Description Indication Status
Phase I
Biomx Inc., of Ness Ziona, Israel BX-001 Phage therapy Acne Top-line results showed study met primary endpoint of safety and tolerability as well as a statistically significant (p=0.036) reduction of Cutibacterium acnes levels for the high dose vs. placebo
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Trispecific recombinant fusion protein conjugate composed of variable regions of heavy and light chains of anti-CD16 and anti-CD33 antibodies and modified form of IL-15 Acute myeloid leukemia Started retreatment of first patient to receive lowest dose in phase I/II trial; following initial treatment, patient achieved stable disease with respect to number of AML blasts observed in bone marrow before and after and showed no adverse events
Kiniksa Pharmaceuticals Inc., of Hamilton, Bermuda Mavrilimumab Fully human monoclonal antibody targeting GM-CSFRα COVID-19 Pilot study of 6 patients with severe COVID-19 pneumonia and hyperinflammation showed all had resolved fever, without progressing to mechanical ventilation; follow-on controlled study in Italy planned; company engaging with FDA regarding path for potential phase II/III development in COVID-19 pneumonia
Matinas Biopharma Holdings Inc., of Bedminster,  N.J. MAT-2203 (oral amphotericin B) Fungicidal Cryptococcal meningitis Uganda National Drug Authority informed all affected sponsors and clinical investigators that recruitment of new clinical trial participants in the county would be immediately suspended as a result of COVID-19; as a result, enrollment of new patients in phase I/II Enact trial has been temporarily paused
Mersana Therapeutics Inc., of Cambridge, Mass. XMT-1536 Sodium phosphate cotransporter 2b (NaPi2b) inhibitor Ovarian and non-small-cell lung cancer Ongoing dose-escalation study showed confirmed responses and durable stable disease (SD) in heavily pretreated patients; at 43 mg/m2 dose, 2/7 achieved partial responses (PRs) and 4/7 achieved SD for disease control rate (DCR) of 6/7 (86%); in subset of evaluable patients treated at >30 mg/m2 with higher NaPi2b expression, 5/15 achieved PR and 6/15 achieved SD for DCR of 11/15 (73%); in subset with lower NaPi2b expression, 0/9 achieved PR and 5/9 achieved SD for DCR of 5/9 (55%)
Proqr Therapeutics NV, of Leiden, the Netherlands QR-421a USH2A gene modulator Usher syndrome Interim analysis of phase I/II Stellar trial that enrolled 14 adults (8 to single dose of study drug and 6 to sham) showed sham treatment produced no consistent pattern of response above noise level; 1 of 4 in low-dose (50 µg) group classified as responder with onset of action observed by 3 month visit and benefit maintained for 6 months or >; 1 of 4 in mid-dose (100 µg) group classified as responder with onset of action observed by 3 months; 100-µg cohort will be expanded to additional participants homozygous for exon 13 mutations, with dose escalation to 200 µg planned in parallel, but enrollment paused due to COVID-19 pandemic
Proqr Therapeutics NV, of Leiden, the Netherlands QR-504a  TCF4 gene modulator  Corneal dystrophy Trial start delayed due to COVID-19 pandemic
Phase II
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
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
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
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)
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
Proqr Therapeutics NV, of Leiden, the Netherlands QR-1123  RHO gene modulator Retinitis pigmentosa Enrollment paused due to COVID-19 pandemic
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
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
Phase III
Biocardia Inc., of San Carlos, Calif. Cardiamp Autologous bone marrow-derived stem cell therapy Heart failure Following prespecified safety review, independent data safety monitoring board recommended pivotal trial continue as planned
Dicerna Pharmaceuticals Inc., of Lexington, Mass. Nedosiran LDHA gene inhibitor Primary hyperoxaluria Initial observations on 14 participants from Phyox1 trial enrolled in Phyox3 suggested study drug was well-tolerated, with no injection-site reactions or drug-related severe adverse events; on at least 2 visits, normalization or near-normalization of urinary oxalate levels were seen in 4 patients who received at least 3 monthly doses
Eyegate Pharmaceuticals Inc., of Waltham, Mass. Ocular bandage gel (dexamethasone, iontophoresis delivery) Glucocorticoid receptor agonist Dry eye disease Study drug showed 25% improvement in central corneal region staining vs. 15% for control (Refresh Preservative-Free lubricant, Allergan plc), 8.2% improvement over 2-week period vs. short-term worsening and no benefit for control in high order ocular aberrations, and trend in improved best corrected visual acuity vs. no improvement for control
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Fibrosis; primary biliary cholangitis COVID-19 pandemic not expected to affect receipt of FDA feedback from Resolve-It trial in fibrosis, where final visits of first 1,000 participants occurred and database related to cohort was locked on schedule at end of February; extension phase of Resolve-It continues but screening of new participants temporarily halted; initiation of phase III study in PBC placed on hold
Iterum Therapeutics plc, of Dublin Sulopenem Broad-spectrum penem beta-lactam antibiotic Complicated and uncomplicated urinary tract infection Top-line data from Sure 1 and 2 studies expected early in second quarter of 2020
Mimetogen Pharmaceuticals Inc., of Montreal Tavilermide  Nerve growth factor mimetic Dry eye disease Completed enrollment of the 600-patient MIM-728 study; data expected in the fourth quarter of 2020
Proqr Therapeutics NV, of Leiden, the Netherlands Sepofarsen CEP290 gene modulator  Leber congenital amaurosis 10 Enrollment in pivotal trial paused due to COVID-19 pandemic
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Activator of Nrf2  Connective tissue disease-associated pulmonary arterial hypertension Stopped Catalyst study due to risk of COVID-19; initial review of data by the data safety monitoring board suggest the study is unlikely to meet the primary endpoint of improvement in 6-minute walk distance compared to placebo at week 24
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Activator of Nrf2  Autosomal dominant polycystic kidney disease Pausing enrollment in study due to COVID-19


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