Company Product Description Indication Status
Phase I
9 Meters Biopharma Inc., of Raleigh, N.C. Exenatide-XTEN (subcutaneous) GLP-1 agonist  Short bowel syndrome Top-line data from phase Ib/IIa study due by year-end 2020
Aptevo Therapeutics Inc., of Seattle, Wash. APVO-436 Anti-CD123 x anti-CD3 targeted bispecific antibody Acute myeloid leukemia; myelodysplastic syndrome Second complete remission observed in participant in cohort 6 (n=9) in ongoing trial; enrollment initiated in cohort 8 
Arcturus Therapeutics Holdings Inc., of San Diego LUNAR-COV19  Anti-SARS-CoV-2 mRNA vaccine COVID-19 infection Based on interim results from fully enrolled phase I/II ARCT-021 study, single 7.5-µg dose + prime-boost regimens will advance into later-stage trials; seroconversion seen in most receiving doses in 5-µg to 7.5-µg range; geometric mean titer levels for neutralizing antibodies were within range seen in convalescent plasma; dose-dependent anti-spike IgG titers increased over time through about day 43
Celltrion Inc., of Incheon, Korea CT-P59 COVID-19 spike glycoprotein inhibitor; moesin modulator COVID-19 infection Those dosed with study drug saw about 44% reduced mean clinical recovery time vs. average placebo recovery time; no one who received CT-P59 required hospitalization or antiviral therapy due to COVID-19
Daewoong Pharmaceutical Co. Ltd., of Seoul Fexuprazan H+ K+ ATPase inhibitor Gastroesophageal reflux disease Bridging study results, published in Alimentary Pharmacology and Therapeutics, showed no racial differences in drug's pharmacokinetic and pharmacodynamic properties 
Immunitybio Inc., of Culver City, Calif. Human adenovirus 5 vector-delivered SARS-CoV-2 S + N protein vaccine COVID-19 spike glycoprotein modulator/nucleoprotein modulator COVID-19 infection Data published in Medrxiv suggested candidate stimulated memory CD4+ and CD8+ T cells
Intellia Therapeutics Inc., of Cambridge, Mass. NTLA-2001 CRISPR associated endonuclease Cas9 modulator; TTR gene modulator Transthyretin amyloidosis First of up to 38 participants dosed in 2-part study in adults with hereditary disease with polyneuropathy
Noxxon Pharma NV, of Berlin NOX-A12 Stromal cell-derived factor 1 ligand inhibitor Brain cancer Data safety monitoring board confirmed it is safe and appropriate to start recruitment for highest planned dose in phase I/I trial testing NOX-A12 plus radiotherapy
Pierre Fabre SA, of Toulouse, France W-0180 Advanced solid tumors VISTA checkpoint inhibitor Study initiated in Spain; additional site activation expected in France and Spain
Vallon Pharmaceuticals Inc., of Philadelphia Dexamfetamine (oral immediate-release abuse-deterrent, or ADAIR) Synaptic vesicular amine transporter stimulator; trace amine associated receptor 1 agonist Attention deficit hyperactivity disorder In 16-person intranasal human abuse crossover comparative study, ADAIR showed blunted pharmacokinetic profile (lower Cmax, delayed Tmax and lower AUC, especially during early hours after administration) vs. crushed and snorted dextroamphetamine sulfate
Phase II
American Brivision Holding Corp., of Fremont, Calif. ABV-1505 Noradrenaline transporter inhibitor; unspecified transporter protein modulator Adult attention deficit hyperactivity disorder For part 1 of study, low and high doses met primary endpoints by passing required 40% of population in percentages of improvement in Adult Attention-Deficit/Hyperactivity Disorder Rating Scale-Investigator Rated-IV scores from baseline through 8 weeks of treatment, with 83.3% (n=5) in intent-to-treat population and 80.0% (n=4) in per-protocol
Amylyx Pharmaceuticals Inc., of Cambridge, Mass. AMX-0035 (ursodoxicoltaurine + sodium phenylbutyrate) Histone deacetylase inhibitor Alzheimer's disease Last participant completed final study visit in Pegasus trial; top-line data targeted for first half of 2021
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Woodcliff Lake, N.J. BAN-2401 (lecanemab) Beta-amyloid antagonist Alzheimer’s disease Phase IIb data showed patients who received placebo in core study entered open-label extension study with high brain amyloid levels and, in extension study, those patients showed rapid decrease in amyloid levels after 3 months of treatment with 10 mg/kg every other week, with further decreases observed after 6 and 12 months; for patients who received BAN-2401 10 mg/kg either monthly or every other week in core study, amyloid levels in the brain were already low upon entering open-label extension and remained low
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide C-type natriuretic peptide ligand Achondroplasia First of about 20 infants enrolled in study for those at risk of requiring cervicomedullary decompression surgery to alleviate foramen magnum compression; as secondary endpoint, study will assess effect on growth of foramen magnum volume
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide C-type natriuretic peptide ligand Genetic forms of short stature First of about 35 children enrolled in investigator-sponsored study; primary outcomes include change from baseline in annualized growth velocity and in height Z-score 
Bristol Myers Squibb Co., of New York Deucravacitinib (BMS-986165)  TYK2 inhibitor Psoriatic arthritis Study met primary endpoint of at least 20% improvement in ACR 20 at week 16; participants dosed at 6 mg (n=70) or 12 mg (n=67) also showed greater responses vs. placebo (n=66) at week 16, at 52.9% and 62.7% vs. 31.8%, respectively (p=0.0134 and p=0.0004, respectively)
EIP Pharma Inc., of Boston Neflamapimod p38 MAP kinase alpha inhibitor Lewy body dementia AscenD-LB study in mild to moderate disease met primary endpoint, with clinically relevant effect size improvement in cognition in those dosed at 40 mg 3 times daily vs. those who received placebo or study drug 40 mg twice daily, as assessed by Neuropsychological Test Battery (NTB); positive effect on NTB seen at week 4 and maintained throughout 16-week study period
Eisai Co. Ltd., of Tokyo Lenvima (lenvatinib)  Multiple receptor tyrosine kinase inhibitor Renal cell carcinoma Comparison of 2 doses (18 mg vs. 14 mg once daily) of study drug + everolimus (5 mg once daily) in people with clear-cell disease showed lower starting dose did not meet threshold for noninferiority vs. FDA-approved starting dose of 18 mg (p=0.2676)
Entera Bio Ltd., of Boston EB-613 Human parathyroid hormone Osteoporosis Ongoing trial fully enrolled with 161 participants; interim 3-month biomarker data expected in first quarter of 2021 with final bone mineral density data expected in second quarter 2021
Inmune Bio Inc., of La Jolla, Calif. Quellor TNF alpha ligand inhibitor COVID-19 infection First participant dosed in trial assessing study drug's ability to prevent progression of immune-mediated pulmonary complications resulting from SARS-CoV-2 infection
Leap Therapeutics Inc., of Cambridge, Mass. DKN-01 Dickkopf-1 ligand inhibitor Epithelial endometrial cancer Study drug showed single-agent activity in recurrent disease, particularly in biomarker-selected subgroups relating to DKK1 biology; those with Wnt signaling alteration had higher overall response rate, greater objective disease control rate and longer median overall survival (OS) than those without Wnt signaling alteration; subgroup of people with Wnt activating mutations had longest progression-free survival and OS of subgroups evaluated
Synact Pharma AB, of Lund, Sweden AP-1189 Dual melanocortin MC1/MC3 receptor agonist Rheumatoid arthritis In part 1 of phase IIa study in people with high disease activity, number of participants changing from severe to moderate CDAI score was higher than placebo in both treatment groups (50 mg and 100 mg) in dose-dependent manner; data safety monitoring board recommended study continue to randomized part 2 portion assessing both doses vs. placebo + methotrexate; top-line data from full study expected by end of second quarter of 2021
Phase III
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Woodcliff Lake, N.J. BAN-2401 (lecanemab) Beta-amyloid antagonist Alzheimer’s disease Data from currently enrolled patients in Clarity AD trial showed baseline characteristics consistent with phase IIb 
Calliditas Therapeutics AB, of Stockholm Nefecon Oral formulation of budesonide Primary IgA nephropathy Top-line results showed part A of Nefigard study met primary objective of demonstrating statistically significant reduction in urine protein creatinine ratio after 9 months of treatment with 16 mg of drug vs. placebo, with significant continued improvement at 12 months; trial also met key secondary endpoint, showing a statistically significant difference in estimated glomerular filtration rate vs. placebo
Nevakar Inc., of Bridgewater, N.J. NVK-002 Preservative-free eye drop; target undisclosed Myopia First patient completed 3-year enrollment in Champ study testing drug’s ability to slow progression of myopia in children; trial remains on track for 3-year data readout in 2022
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 Vaccine COVID-19 Interim efficacy analysis by independent data monitoring committee showed COVID-19 cases split between vaccinated individuals and those who received the placebo, indicating efficacy rate above 90%, at 7 days after the second dose
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone methyl Oral, once-daily activator of Nrf2 Alport syndrome Cardinal study in patients with chronic kidney disease caused by Alport syndrome met primary and key secondary endpoints at end of year 2; at week 100, in intent-to-treat (ITT) population, patients had statistically significant improvement vs. placebo in mean change from baseline in eGFR of 7.7 mL/min/1.73 m2 (p=0.0005); in modified ITT population, patients had statistically significant improvement vs. placebo in mean change from baseline in eGFR at week 100 of 11.3 mL/min/1.73 m2 (p<0.0001); NDA submission to FDA expected in first quarter of 2021
Regenerx Biopharmaceuticals Inc., of Rockville, Md. RGN-259 eye drops  Thymosin beta 4 ligand Dry eye syndrome Final patient in 700-subject Arise-3 trial completed treatment and follow-up
Viiv Healthcare Ltd., of London Cabotegravir Integrase inhibitor HIV prevention Independent data safety monitoring board recommended early unblinding of HPTN 084 study testing drug for HIV prevention in women; following prespecified interim analysis, DSMB indicated drug met primary objective of demonstrating superiority vs. daily oral emtricitabine/tenofovir disoproxil fumarate 200 mg and 300 mg; study showed cabotegravir was 89% more effective for pre-exposure prophylaxis


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