Company Product Description Indication Status
Phase I
9 Meters Biopharma Inc., of Raleigh, N.C. NM-002 Long-acting glucagon-like peptide-1 receptor agonist  Short bowel syndrome Of the 9 patients, 8 had meaningful declines in total stool output following each dose; plans to meet with FDA in the first quarter of 2021 to discuss plans for further development
Amphivena Therapeutics Inc., of South San Francisco AMV-564 Depletes myeloid-derived suppressor cells and activates T cells Solid tumors Dosed first patient in expansion study
Arecor Ltd., of Cambridge, U.K. AT-278 Ultra-concentrated rapid-acting insulin Diabetes Dosed first patient 
Arecor Ltd., of Cambridge, U.K. AT-247 Ultra-rapid acting insulin Type 1 diabetes Data published in Diabetes Care showed AT-247 produced an earlier insulin appearance, exposure and offset, and a  corresponding early glucose-lowering effect compared with both Novorapid (insulin, Novo Nordisk A/S) and Fiasp (insulin, Novo Nordisk)
Celyad Oncology SA, of Mont-Saint-Guibert, Belgium CYAD-101 Allogeneic CAR T  Refractory metastatic colorectal cancer Treated first patient in the expansion cohort of the Alloshrink study; preliminary data expected in the first half of 2021
Cerecor Inc., of Rockville, Md. CERC-007 Monoclonal antibody targeting IL-18 Relapsed or refractory multiple myeloma Treated first of about 30 patients in phase Ib/IIa study designed to test the safety, tolerability and response rate of CERC-007 and to determine the recommended phase II dose
Grünenthal GmbH, of Aachen, Germany NOP agonist Nociceptin/orphanin peptide receptor agonist Healthy volunteers (eventually peripheral neuropathic pain) Enrolled first of 76 participants in the study designed to test the safety and tolerability profile and to confirm the pharmacokinetic characteristics of the compound following single and multiple ascending doses; data expected in 2021
Kineta Inc., of Seattle KCP-506 Αlpha9/alpha10 nicotinic acetylcholine receptor antagonist Healthy volunteers (eventually chronic neuropathic pain) First of up to 88 participants dosed in the study testing the safety, tolerability and pharmacokinetics of KCP-506; data expected in the second half of 2021
Protagonist Therapeutics Inc., of Newark, Calif., and Johnson & Johnson, of New Brunswick, N.J. PN-235 (JNJ-77242113) Interleukin-23 receptor antagonist peptide Healthy volunteers First participant dosed in the study testing single ascending doses, multiple ascending doses and a crossover solid dose comparison; primary endpoint is safety; secondary outcomes include pharmacokinetics measurements of peak concentration and area under the curve
Sio Gene Therapies Inc., of New York AXO-AAV-GM1 Gene therapy expressing GLB1 GM1 gangliosidosis In the low-dose cohort of phase I/II trial, all 5 patients had stability of Vineland Adaptive Behavior Scales 3rd Edition (VABS-3) Growth Scale Value scores, Upright and Floor Mobility Score and Clinical Global Impression from baseline to 6 months; VABS-3 Subdomain Growth Scale Value remained stable or improved in 4 out of 5 patients
Valneva SE, of Saint-Herblain, France VLA-2001 Inactivated, adjuvanted COVID-19 vaccine COVID-19 prophylaxis Started 150-participant study testing the immunogenicity of 2 shots at 3 dose levels; selection of optimal dose expected early in the second quarter of 2021
Phase II
AB Science SA, of Paris Masitinib  Tyrosine kinase inhibitor targeting mast cells and macrophages Mild and moderate Alzheimer’s disease In phase IIb/III study AB09004, patients treated with masitinib had a better change in Alzheimer's Disease Assessment Scale-Cognitive Subscale compared to the control arm (p=0.0003); masitinib generated a significant change from baseline in Alzheimer’s Disease Cooperative Study Activities of Daily Living (p= 0.0381)
Abpro Corporation, of Woburn, Mass. ABP-300 Monoclonal antibody targeting SARS-CoV-2 COVID-19 First phase II/III trial site opened; remainder expected to open in 2021; study compares 2 dose levels to placebo 
Biolinerx Ltd., of Tel Aviv, Israel Motixafortide (BL-8040) Targets CXCR4 Second-line stage IV pancreatic ductal adenocarcinoma In the phase IIa Combat/Keynote-202 study testing motixafortide plus Keytruda (pembrolizumab, Merck & Co. Inc.) and chemotherapy, median overall survival was 6.5 months; median progression-free survival was 4 months; confirmed overall response rate was 13.2%
Concentric Analgesics Inc., of San Francisco Vocacapsaicin (CA-008) Capsaicin prodrug Pain associated with total knee arthroplasty surgery Compared to placebo, treatment with 36 mg dose produced an 18% reduction in pain at rest measured as area under the curve from 12-96 hours (p=0.0004) and a 30% reduction in opioid consumption from 0-96h (p<0.0001)
Vtv Therapeutics Inc., of High Point, N.C. Azeliragon  Advanced glycosylation product receptor antagonist  Alzheimer’s disease with type 2 diabetes Patients taking azeliragon had a 1.8-point decline on the 14-item Alzheimer’s Disease Assessment Scale – Cognitive Subscale compared to a 0.35-point decline for placebo
Phase III
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Rimegepant CGRP receptor agonist in orally disintegrating tablet Migraine Data published in The Lancet show drug superior to placebo in reduction of monthly migraine days, with treatment resulting in -4.3-day reduction in mean migraine days per month; about half of treated patients showed 50% or greater reduction in number of moderate to severe migraine days per month
Eyenovia Inc., of New York Microline Pilocarpine formulation delivered via Optejet dispenser Presbyopia First set of patients enrolled in Vision-1 study; top-line data expected in first half of 2021
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Anti-PD-1 antibody Advanced endometrial cancer Combination with Lenvima (lenvatinib, Eisai Co. Ltd.) showed significant improvement in overall survival, progression-free survival and objective response rate vs. chemotherapy in patients following prior systemic therapy; positive results were observed in mismatch repair proficient (pMMR) subgroup and the intention-to-treat study population, which includes both patients with endometrial carcinoma that is pMMR as well as those whose disease is microsatellite instability-high/mismatch repair deficient 
Noven Pharmaceuticals Inc., of Miami, a wholly owned subsidiary of Hisamitsu Pharmaceutical Co. Inc. Secuado (asenapine) transdermal system Psychotropic agent delivered via transdermal patch Schizophrenia Data published in the Journal of Clinical Psychiatry showed significant improvement in Positive and Negative Syndrome Scale (PANSS) total score at week 6 vs. placebo in adult patients; the difference in the least squares mean change from baseline in PANSS total score was -4.8 (1.634) and -6.6 (1.630) for the 7.6-mg/24h and 3.8-mg/24h groups vs. placebo
Novo Nordisk A/S, of Bagsvaerd, Denmark Semaglutide Long-acting GLP-1 analogue Alzheimer’s disease Intends to start pivotal phase IIIa program in about 3,700 people with early Alzheimer’s disease in first half of 2021
Polypid Ltd., of Petah Tikva, Israel D-Plex100 Designed to provide prolonged and controlled antibacterial activity Prevention of post-abdominal surgery incisional infection Enrolled first patient in Shield II study; primary endpoint is the infection rate, as measured by the proportion of subjects with at least 1 abdominal target incisional infection event within 30 days post-abdominal surgery

Notes

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