Company Product Description Indication Status
Phase I
Crinetics Pharmaceuticals Inc., of San Diego CRN-04777 Somatostatin 5 receptor agonist Congenital hyperinsulinism Initiated trial, expected to enroll up to 117 healthy adults; in addition to safety and tolerability, study will measure agent's ability to suppress insulin secretion following stimulation with glucose or sulfonylurea
Gemoab Monoclonals GmbH, of Dresden, Germany UniCAR02-T-CD123 cell therapy  CDw123 modulator; CDw137 agonist Acute myeloid leukemia Interim data from ongoing study in relapsed/refractory disease showed expansion of UniCAR-T-CD123 in peripheral blood and bone marrow, comparable to conventional CD123-targeting CAR Ts, and neutrophil recovery after treatment with targeting module TM123 stopped; first 3 fully treated patients showed 1 partial remission and 2 complete remissions with incomplete hematologic recovery
Imcyse SA, of Liege, Belgium IMCY-0098 Imotope (modified peptide) Type 1 diabetes First of 24 participants dosed in step 1 of phase Ib/IIa adaptive design Impact study exploring immune signature and safety of 2 doses vs. placebo in people with recent onset T1D; step 2 will enroll 60 participants
LB Pharmaceuticals Inc., New York LB-102 (amisulpride analog) Dopamine/5-HT7 antagonist Schizophrenia First healthy volunteer dosed in phase Ib PET imaging study to evaluate dopamine receptor occupancy and assist in phase II dose selection
Revelation Biosciences Inc., of Menlo Park, Calif. REVTx-99 TLR4 agonist Respiratory viral infection Single dose-escalation portion completed in 40 participants; multiple dose cohort enrollment to finish in February 2021, with top-line data by end of first quarter of 2021
Sio Gene Therapies Inc., of New York AXO-AAV-GM2 HEXA/HEXB gene stimulator GM2 gangliosidosis (Sandhoff or Tay Sachs disease) Gene therapy delivered surgically to first participant with infantile form of disease in phase I/II trial
Vaxart Inc., of South San Francisco VXA-CoV2-1 Recombinant viral vector vaccine COVID 19 infection Oral vaccine triggered CD8+ cytotoxic T-cell response to viral spike protein, increase in plasmablast cell number, up-regulation of mucosal homing receptor and increase in proinflammatory Th1 cytokines, but neutralizing antibodies not detected in serum and IgG responses not detected in most participants
Phase II
Biophytis SA, of Paris Sarconeos (BIO-101, 20‐hydroxyecdysone) Proto-oncogene Mas agonist COVID-19-related acute respiratory failure Recruitment completed (n=50) in part 1 of Cova study, with interim analysis expected in first quarter of 2021; following data monitoring committee recommendation to continue, pivotal phase III part 2 of Cova trial expected to begin enrolling 310 participants, initially in U.S. and Brazil, with full data expected in second quarter of 2021
Covaxx, subsidiary of United Biomedical Inc., both of Hauppauge, N.Y. UB-612 Multitope peptide-based vaccine COVID-19 infection First participant enrolled in trial in Taiwan expected collectively to recruit 3,850 adolescents, adults and seniors
Daiichi Sankyo Co. Ltd., of Tokyo Patritumab deruxtecan HER3-directed DXd antibody-drug conjugate  Non-small-cell lung cancer First participant with EGFR-mutated metastatic or locally advanced disease previously treated with TKI and platinum-based chemotherapy dosed in Herthena-Lung01 trial; primary endpoint is objective response rate assessed by blinded independent central review; secondary endpoints include duration of response, progression-free survival and disease control rate
Enlivex Therapeutics Ltd., of Nes Ziona, Israel Allocetra Autologous cell-based therapy COVID-19 infection At 28-day follow-up of 16 participants with severe (n=9) or critical (n=7) infection, mortality was 0/16 (0%) and 14/16 were recovered and discharged from hospital; 2, both with critical illness at time of treatment, were hospitalized in ICU on respirator 
PDS Biotechnology Corp., of Florham Park, N.J. PDS-0101 Cationic lipid nanoparticle vaccine Human papillomavirus-associated cancer NCI combination study with TGF-beta/anti-PD-L1 bifunctional fusion protein bintrafusp alfa (M-7824, Glaxosmithkline plc/Merck KGaA) and DNA-targeted immunocytokine NHS-IL12 (M-9241, Merck Serono) in people with advanced disease achieved preliminary objective response and will progress to full enrollment of about 20 participants
Viacyte Inc., of San Diego PEC-EnCap (VC01-103) Pluripotent stem cell-derived beta cell progenitors Type 1 diabetes First in initial cohort of 10 participants implanted with encapsulated cell therapy, with potential to expand to 70 people; data expected in second half of 2021
Phase III
Astrazeneca plc, of Cambridge, U.K. AZD-1222 COVID-19 spike glycoprotein modulator COVID-19 infection Primary analysis of trials, published as preprint in The Lancet, showed efficacy of 76% (CI: 59% to 86%) after first dose with protection maintained to second dose; with inter-dose interval of 12 weeks or more, efficacy increased to 82% (CI: 63%, 92%); positive PCR readings were reduced by 67% (CI: 49%, 78%) after single dose and 50% (CI: 38% to 59%) after 2-dose regimen
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Tokyo Lecanemab (BAN-2401) Beta amyloid antagonist Alzheimer's disease Target enrollment number increased by about 200 in pivotal Clarity AD trial in people with early disease to ensure robust dataset and mitigate potential impact of missed doses due to COVID-19 pandemic; data readout still expected by September 2022
Vectivbio Holding AG, of Basel, Switzerland Apraglutide Glucagon-like peptide 2 receptor agonist Short bowel syndrome First participant dosed in pivotal Stars trial in people with intestinal failure

Notes

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