Company Product Description Indication Status
Phase I
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. BCX-9930 Oral factor D inhibitor Paroxysmal nocturnal hemoglobinuria New data from phase I/II trial in treatment-naïve patients receiving doses through 400 mg twice daily showed hemoglobin levels increased by mean of 3.8 g/dL from baseline, maintained without transfusions; 3 of 4 patients responded with hemoglobin levels >11 to date; in all 4 patients, size of PNH red blood cell clone approached that of PNH granulocyte clone, indicating near-complete control of complement-mediated hemolysis; all 4 have shown reductions in LDH
Galapagos NV, of Mechelen, Belgium GLPG-3970 Small-molecule anti-inflammatory drug Psoriasis Dosed first patient 
IGM Biosciences Inc., of Mountain View, Calif. IGM-8444 IgM antibody targeting death receptor 5 protein Solid cancers and non-Hodgkin lymphoma Dosed first patient in trial testing monotherapy and combination with chemotherapy in relapsed and/or refractory solid cancers and NHL; initial data expected in 2021
Isofol Medical AB, of Gothenburg, Sweden Arfolitixorin Folate receptor antagonist Metastatic colorectal cancer Update on extension study portion of phase I/IIa trial following 16 weeks of treatment and beyond showed best overall response rate of 55% in 31 patients
Moderna Inc., of Cambridge, Mass. mRNA-1273 mRNA vaccine COVID-19 Second interim analysis data, published in The New England Journal of Medicine, found both 25-µg and 100-µg dose levels were generally well-tolerated in both age cohorts (56-70 and 71+); immune responses were dose-dependent, with the 100-µg dose eliciting higher binding and neutralizing antibody titers, supporting selection of the 100-µg dose for further study in phase III
Prelude Therapeutics Inc., of Wilmington, Del. PRT-1419  MCL1 inhibitor Relapsed/refractory high-risk myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkin's lymphoma and multiple myeloma Treated first patient in the dose-escalation study, which also includes a dose-expansion phase; primary objective is to identify dose-limiting toxicities, a maximum tolerated dose and a recommended phase II dose; secondary endpoints include safety and tolerability, pharmacokinetics and antitumor activity of PRT-1419
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-COV2 Cocktail of antibodies targeting SARS-CoV-2 COVID-19 In seronegative patients, high dose produced a mean time-weighted-average change from baseline nasopharyngeal viral load through day 7 of 0.60 log10 copies/mL greater reduction compared to placebo (p=0.03) in phase I/II/III trial; low dose produced a 0.51 log10 copies/mL greater reduction compared to placebo (p=0.06) 
Regenxbio Inc., of Rockville, Md. RGX-121 Gene therapy expressing iduronate-2-sulfatase Mucopolysaccharidosis type II Plans to enroll up to 6 additional patients in cohort 2 
Ultimovacs ASA, of Oslo, Norway UV-1 Peptide-based vaccine inducing specific T-cell response against telomerase Metastatic malignant melanoma Top-line results from first cohort of 20 patients testing combination with Keytruda (pembrolizumab, Merck & Co. Inc.) as first-line treatment confirm achievement of primary endpoints of safety and tolerability and indicate initial signs of clinical response; at 1 year, overall survival rate was 85%; median progression-free survival was not reached, indicating more than half did not demonstrate disease progression
Phase II
Akero Therapeutics Inc., of San Francisco Efruxifermin  FGF21 mimic Nonalcoholic steatohepatitis with compensated cirrhosis (F4), Child-Pugh Class A Completed enrollment of 30 patients with F4 fibrosis score; data from the F4 expansion cohort expected in the first half of 2021
Curevac NV, of Tübingen, Germany CVnCoV MRNA-based vaccine COVID-19 prophylaxis Treated first of 690 participants in the CV-NCOV-OO2 study testing different dose levels of the vaccine; data expected in the fourth quarter of 2020
Harbour Biomed Therapeutics Ltd., of Suzhou, China Batoclimab (HBM-9161) Anti-FcRn monoclonal antibody Myasthenia gravis First patient enrolled in the study
Harbour Biomed Therapeutics Ltd., of Suzhou, China Batoclimab (HBM-9161) Anti-FcRn monoclonal antibody Immune thrombocytopenia First patient enrolled in the study
Iliad Biotechnologies LLC, of Weston, Fla. BPZE-1 Live attenuated pertussis vaccine Pertussis prophylaxis Vaccine induced both IgG and IgA systemic immunity; 3-month challenge at revaccination produced 10% colonization compared to 70% colonization for Boostrix
Intrabio Inc., of Oxford, U.K. IB-1001 Modified amino acid Niemann-Pick disease type C Trial met its primary endpoint, the Clinical Impression of Change in Severity, and secondary endpoints, including the Scale for the Assessment and Rating of Ataxia and the Investigators' Clinical Global Impression of Change assessment
Lidds Ab, of Upsala, Sweden Liproca Depot Extended release 2-hydroxyflutamide Prostate cancer In the open-label extension of the LPC-004 study, 50% of the 12 patients continued to have low PSA levels for at least 10 months; other 6 patients had second treatment which reduced PSA levels by an average of 28% at 2-months post treatment
Ovid Therapeutics Inc., of New York Soticlestat (OV-935/TAK-935) Cholesterol 24-hydroxylase inhibitor Developmental and epileptic encephalopathies In the Arcade study, patients with CDKL5 deficiency disorder had a median motor seizure frequency reduction of 24% during the 12-week maintenance, which increased to a 50% reduction after 9 months of continuous treatment in Endymion long-term extension study; in 8 Dup15q patients, there was an increase in median motor seizure frequency during the 12-week maintenance period; the 4 patients in the Endymion study who reached 9 months of continuous treatment had a 74% reduction in median motor seizure frequency
Phase III
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran RNAi targeting hydroxyacid oxidase 1 Primary hyperoxaluria type 1 In the Illuminate-B study, lumasiran demonstrated a clinically meaningful reduction in spot urinary oxalate:creatinine ratio; secondary endpoints, including additional measures of urinary and plasma oxalate, were also positive
Eisai Co. Ltd., of Tokyo Lorcaserin Serotonin 2C receptor agonist  Dravet syndrome Started the Momentum 1 study in 58 patients; primary endpoint is percent change in frequency of convulsive seizures per 28 days
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. TNX-102 SL (cyclobenzaprine HCl sublingual tablets) Non-opioid, centrally acting analgesic Fibromyalgia After an interim analysis of the primary endpoint for the first 50% of participants, the independent data monitoring committee recommended the addition of 210 participants to the original sample size of 470 participants; company decided to complete the study with the enrolled 503 participants; data expected in the fourth quarter of 2020


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