Company Product Description Indication Status
Phase I
Asclepix Therapeutics Inc., of Baltimore AXT-107 Dual VEGF-A/VEGF-C inhibitor; Tie2 stimulator Diabetic macular edema First of about 18 participants dosed in phase I/IIa Congo trial assessing single intravitreal injection of 0.1 mg (low dose), 0.25 mg (mid-dose) or 0.5 mg (high dose)
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 (dexmedetomidine, sublingual) Alpha 2A adrenoceptor agonist Agitation Phase Ib/II Tranquility trial in 54 participants with agitation related to dementia met primary and secondary endpoints at 60-mcg dose (n=20), including reductions in total scores in 3 primary agitation scales
Chemomab Ltd., of Tel Aviv, Israel CM-101 CCR3 chemokine modulator Nonalcoholic fatty liver disease In phase Ib Spark study in 16 NAFLD participants with normal liver function, exploratory analysis of pharmacodynamic parameters, including measurement of collagen turnover and fibrotic biomarkers such as Pro-C3, Pro-C4, C3M and TIMP1, resulted in reduction of markers vs. no change or slight elevation in placebo group
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo Cholesterol binding agent; lipid metabolism modulator Niemann-Pick disease type C Initial data showed all 8 participants enrolled in long-term extension study had disease stability or improvement as measured by 17-domain NPC Severity Score scale
Ideaya Biosciences Inc., of South San Francisco IDE-196 Protein kinase C inhibitor Metastatic uveal melanoma First participant dosed in combination study with cMET inhibitor Xalkori (crizotinib, Pfizer Inc.) 
Inhibrx Inc., of San Diego INBRX-106 Hexavalent OX40 agonist Solid tumors In part 1 (single agent dose escalation) of study in 20 participants, maximum administered dose was 3 mg/kg and maximum tolerated dose was not reached; clinical benefit seen in multiple tumor types, including those generally considered hot and cold, and in people relapsed or refractory to checkpoint inhibitors; biomarker sampling confirmed agonist activity at dose levels ranging from 0.0003 to 0.3 mg/kg
Replimune Group Inc., of Woburn, Mass. RP-3 (oncolytic virus) CD40 ligand receptor agonist; CDw137 agonist Advanced solid tumors First participant dosed in trial assessing study drug alone and in combination with anti-PD-1 therapy
Phase II
Arch Biopartners Inc., of Toronto Metablok LSALT peptide COVID-19 infection University of Calgary Cumming School of Medicine joined as trial site in study targeting prevention of acute lung or kidney injury and other complications in people hospitalized with moderate to severe infection
Atyr Pharma Inc., of San Diego ATYR-1923 Neuropilin 2 modulator COVID-19 infection Study in people hospitalized with moderate to severe infection (n=32) met primary safety endpoint; single dose of 3 mg/kg resulted in median time to recovery of 5.5 days; 83% who received 3-mg/kg dose recovered by day 6 vs. 56% for placebo
Cerecor Inc., of Rockville, Md. CERC-002 Anti-LIGHT monoclonal antibody COVID-19-related acute respiratory distress syndrome Participants hospitalized with COVID-19-related ARDS showed improvement in primary endpoint of proportion alive and free of respiratory failure over 28-day study period vs. placebo (n=62, p=0.059); prespecified subgroup analysis of 60-year-old enrollees showed treatment led to >3-fold increase in likelihood of avoiding respiratory failure and death vs. placebo (n=33, p=0.054)
Destiny Pharma plc, of Brighton, U.K. XF-73 (exeporfinium chloride) Antimicrobial permeability enhancer Staphylococcal infection, including MRSA Phase IIb trial was fully enrolled by year-end 2020, meeting program timetable; results expected in first quarter of 2021
Inflarx GmbH, of Jena, Germany Vilobelimab (IFX-1) Anti-C5a antibody Vasculitis European Ixchange study completed enrollment; data expected by year-end 2021
Metacrine Inc., of San Diego MET-409  Farnesoid X receptor agonist  Nonalcoholic steatohepatitis with type 2 diabetes mellitus  Started the 120-patient study comparing MET-409 and/or Jardiance (empagliflozin, Eli Lilly and Co. and Boehringer Ingelheim GmbH) to placebo in a 12-week study evaluating the safety, tolerability and reductions in liver fat content; top-line data expected in the first half of 2022
Sanbio Co Ltd., of Tokyo SB-623  Allogeneic modified and cultured adult bone marrow-derived mesenchymal stem cells Stable chronic neurological motor deficits secondary to traumatic brain injury Data from the Stemtra study published in Neurology showed SB-623 produced an average 8.3-point improvement from baseline in the Fugl-Meyer Motor Scale (FMMS), compared to a 2.3-point improvement in the control group, at 6 months (p=0.040); 39.1% of patients treated with SB623 achieved at least a 10-point improvement in FMMS score compared to 6.7% of patients in the control group (p=0.039)
Phase III
Abbvie Inc., of North Chicago Skyrizi (risankizumab) Monoclonal antibody targeting interleukin-23 Active psoriatic arthritis In the Keepsake-1 and -2 studies, 57% and 51% of patients taking Skyrizi achieved ACR20 at week 24, respectively, compared to 34% and 27% of patients taking placebo, respectively (p<0.001 for both); 52% and 55% of patients taking Skyrizi achieved PASI90, respectively, compared to 10% of patients in both studies who received placebo (p<0.001 for both)
Aeglea Biotherapeutics Inc., of Austin, Texas Pegzilarginase Human arginase Arginase 1 deficiency Disclosed in SEC filing that COVID-19 has created limited access environment, impacting the ability for some sites in Peace study to schedule and complete initial screening visits; company now expects to complete enrollment by end of March 2021 and anticipates top-line data in fourth quarter of 2021; to date, 22 patients (75%) have been enrolled
Genmab A/S, of Copenhagen, Denmark, and Seagen Inc., of Bothell, Wash. Tisotumab vedotin Antibody-drug conjugate targeting tissue factor Recurrent or metastatic cervical cancer after 1 or 2 prior lines of systemic therapy Started the InnovaTV 301 study testing tisotumab vedotin compared to physician’s choice single-agent chemotherapy; primary endpoint is overall survival
Ocuphire Pharma Inc., of Farmington Hills, Mich. Nyxol Formulation of phentolamine mesylate Night vision disturbances Started patient recruitment and screening for the 160-patient Lynx-1 study; primary endpoint is the percentage of patients with at least 3 lines of improvement in mesopic, low-contrast, best-corrected distance visual acuity after 7 days; secondary endpoints include pupil diameter reductions, other visual acuity measures (distance and near) and safety and tolerability


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