Company Product Description Indication Status
Phase I
89bio Inc., of San Francisco BIO89-100 FGF21 analogue Nonalcoholic steatohepatitis Top-line results from phase Ib/IIa trial showed all dose groups demonstrated significant reductions in liver fat at week 13, with relative reductions up to 60% vs. baseline and up to 70% vs. placebo, as measured by magnetic resonance imaging – proton density fat factor (MRI-PDFF); significant proportion of subjects responded to therapy with up to 88% and 71% of subjects achieving a ≥30% or a ≥50% reduction in liver fat vs. baseline, respectively; treatment also resulted in significant improvements in liver transaminases, with 35 U/L decrease in ALT from baseline in subjects with elevated baseline levels, and reductions in ProC3, a marker of fibrosis
Actinium Pharmaceuticals Inc., of New York Actimab-A Antibody-radiation conjugate targeting CD33 Relapsed/refractory acute myeloid leukemia Update in Actimab-A CLAG-M combination trial showed all patients in third dosing cohort, which was scheduled as the final cohort of the dose-escalation trial, completed treatment with a 0.75 uCi/kg dose of Actimab-A followed by CLAG-M and have cleared initial safety evaluation; results are expected by year-end
Celltrion Group, of Incheon, Korea CT-P59 Monoclonal antibody  COVID-19 Interim results showed safety, tolerability and promising pharmacokinetics profile in healthy volunteers
Enanta Pharmaceuticals Inc., of Watertown, Mass. EDP-297 Targeted following FXR agonist Nonalcoholic steatohepatitis Dosed first subjects; data expected in second quarter of 2021
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Designed to activate progenitor cells and regenerate auditory hair cells in the inner ear to restore hearing function Chronic sensorineural hearing loss Results from long-term durability study showed some patients had significant improvement in key measures of hearing that were sustained for up to 21 months
Immunity Pharma Ltd., of Jerusalem IPL-344 AKT protein kinase stimulator Amyotrophic lateral sclerosis Interim results from phase I/IIa study showed deterioration rate of ALSFRS-R in 6 participants treated > 4 months was 49% slower than before treatment (p=0.08); average slow vital capacity loss was 1.3% per month vs. average of 3.1% per month in general ALS population with ALSFRS-R <40
LB Pharmaceuticals Inc., of New York LB-102 Derivative of amisulpride Schizophrenia Data showed drug well-tolerated up to 150 mg/day; dosing in 100-mg twice-daily cohort discontinued due to occurrence of extrapyramidal symptoms, a biomarker typically associated with >80% dopamine receptor occupancy; plasma concentrations were about 2.5 times greater than published values of amisulpride at same dose, suggesting LB-102 could be dosed substantially lower than amisulpride
Pharmabcine Inc., of Daejeon, South Korea Olinvacimab VEGF-2 receptor antagonist Glioblastoma multiforme In phase Ib combination trial with Keytruda (pembrolizumab, Merck & Co. Inc.) in recurrent disease, 4 participants (44%) had stable disease, including 1 over 12 cycles; median overall survival was 7.2 months vs. 4 months average for in general recurrent GBM population
Pharmabcine Inc., of Daejeon, South Korea Olinvacimab VEGF-2 receptor antagonist Triple-negative breast cancer In phase Ib combination trial with Keytruda (pembrolizumab, Merck & Co. Inc.) in metastatic disease, 4 (36%) of 11 participants had partial response and 1 had complete response
Phase II
AB Science SA, of Paris Masitinib Tyrosine kinase inhibitor Multiple sclerosis In phase IIb/III study in 301 participants with progressive disease, study drug (4.5 mg/kg/day) slowed disease progression, which was primary endpoint, and showed reduction in risk of reaching level of disability requiring wheelchair mobility
Alzprotect SAS, of Lille, France AZP-2006 (ezeprogind) Tau protein modulator Progressive supranuclear palsy First 5 participants enrolled in phase IIa trial; efficacy endpoints include assessment of treatment on >20 disease markers targeting inflammation and neurodegeneration; completion expected by year-end 2021, with initial data in 2022
Blade Therapeutics Inc., of South San Francisco BLD-2660 Calpain inhibitor COVID-19-related pneumonia Blade-Conquer trial reached 120-subject enrollment goal and IDMC recommended continuation after planned review of blinded safety data; top-line results expected in fourth quarter of 2020
Boehringer Ingelheim Pharmaceuticals Inc., unit of Boehringer Ingelheim GmbH, of Ingelheim, Germany BI-425809 Beta-amyloid synthesis inhibitor; glycine transporter-1 inhibitor Schizophrenia Trial met primary endpoint, showing improved cognition in stable adults
Cassava Sciences Inc., of Austin, Texas Sumifilam Filamin A modulator Alzheimer's disease People treated with 50 mg or 100 mg twice-daily for 28 days showed statistically significant (p<0.05) improvements in biomarkers of disease pathology, neurodegeneration and neuroinflammation vs. placebo, achieving 98% response rate; those on study drug also showed directional improvements in tests of episodic memory and spatial working memory vs. placebo
Cyxone AB, of Stockholm Rabeximod Small-molecule therapeutic COVID-19 Plans to start trial; treatment is intended to reduce risk of developing severe respiratory symptoms caused by virus-related overactivation of immune system
Daiichi Sankyo Co. Ltd., of Tokyo Patritumab deruxtecan (U3-1402) Anti-HER3 antibody; topoisomerase I inhibitor Colorectal cancer Study initiated in about 80 previously treated patients with HER3-expressing advanced/metastatic disease; primary endpoint is objective response rate per RECIST v1.1
Diamedica Therapeutics Inc., of Minneapolis DM-199 Kallikrein 1 modulator Diabetic kidney disease  First participants dosed in DKD cohort of Redux trial; primary efficacy endpoints include effect on blood pressure, albuminuria and kidney function
Diamyd Medical AB, of Stockholm Diamyd Diabetes vaccine Type 1 diabetes Top-line results encompassing 109 patients from phase IIb Diagnode-2 trial showed statistically significant effect in predefined HLA subgroup; trial showed preservation of beta cell function at 15 months post-diagnosis, as measured by meal stimulated C-peptide; primary endpoint, defined as meal stimulated C-peptide in the entire trial population, was not met; based on results, company will pursue the HLA restricted responder subgroup in upcoming pivotal phase III program
Phase III
Alexion Pharmaceuticals Inc., of Boston, and Caelum Biosciences Inc., of Bordentown, N.J. CAEL-101 Amyloid protein deposition inhibitor AL amyloidosis Enrollment initiated in parallel studies in Mayo stage IIIa and IIIb disease in Cardiac Amyloid Reaching for Extended Survival (Cares) program, expected to enroll about 370 individuals; primary endpoint is overall survival
Astrazeneca plc, of Cambridge, U.K. AZD-1222 COVID-19 Spike glycoprotein modulator COVID-19 infection Trial in U.K. in collaboration with University of Oxford resumed following voluntary pause after clearance by MHRA
Avita Therapeutics Inc., of Valencia, Calif. Recell Cell therapy + autologous cell harvesting device  Vitiligo Pivotal study initiated to assess repigmentation in people with stable disease
Eli Lilly and Co., of Indianapolis, and Incyte, Corp., of Wilmington, Del. Baricitinib  JAK1/JAK2 inhibitor COVID-19 Median time to recovery was approximately 1 day shorter for patients treated with baricitinib plus remdesivir compared to remdesivir alone
Humanigen Inc., of Burlingame, Calif. Lenzilumab GM-CSF ligand inhibitor COVID-19 infection Data safety monitoring board unanimously recommended trial continue without modification after prespecified interim analysis for safety, futility, sample size and power; enrollment target of 300 participants expected to be reached in September 2020
Inflarx NV, of Jena, Germany IFX-1  Monoclonal anti-human complement factor C5a antibody Severe COVID-19 induced pneumonia Enrolled first of 360 patients in the phase III portion of the phase II/III study; primary endpoint is 28-day all-cause mortality; secondary endpoints include assessment of organ support and disease improvement; interim analysis after 180 patients enrolled
Myovant Sciences, of Basel, Switzerland Relugolix combination therapy Relugolix plus estradiol and norethindrone acetate  Uterine fibroids In the Liberty studies, mean changes in lumbar spine bone mineral density were comparable for relugolix combination therapy and placebo (week 12: -0.63% vs. 0.34%; week 24: -0.23% vs. 0.18%, respectively)
Novartis AG, of Basel, Switzerland Beovu (brolucizumab)  Antibody fragment targeting VEGF-A Diabetic macular edema Kite study met its primary endpoint of noninferiority for Beovu vs. aflibercept in mean change in best-corrected visual acuity at year 1
Pfizer Inc., of New York, and  Biontech SE, of Mainz, Germany BNT-162b2 mRNA-based vaccine COVID-19 prophylaxis Submitted an amended protocol to the FDA to increase enrollment to approximately 44,000 participants
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-COV2  Antiviral antibody cocktail COVID-19 Plan to run the Recovery (Randomized Evaluation of COVID-19 Therapy) study in at least 2,000 patients; primary endpoint is all-cause mortality 28 days after randomization


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