Company Product Description Indication Status
Phase I
AIM Immunotech Inc., of Ocala, Fla. Ampligen and interferon alpha-2b TLR-3 agonist; interferon type I receptor agonist; ribonuclease stimulator; 2,5-oligoadenylate synthetase stimulator Cancer and mild to moderate COVID-19 Institutional review board approved protocol amendment to ongoing phase I/IIa study testing 2-drug combination as potential early-onset treatment, calling for randomization of additional 20 patients
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. BCX-9930 Complement factor D inhibitor Paroxysmal nocturnal hemoglobinuria  Significantly increased hemoglobin and reduced transfusions in ongoing dose-ranging trial in treatment-naïve patients and in patients with an inadequate response to C5 inhibitors; company plans to directly advance into pivotal trials in PNH and proof-of-concept trials in renal complement-mediated diseases in second half of 2021
CTI Biopharma Corp., of Seattle Pacritinib Oral kinase inhibitor with specificity for JAK2 Prevention of acute graft-vs.-host disease Data published in Clinical Cancer Research showed drug, in combination with sirolimus and low-dose tacrolimus, exhibited preliminary therapeutic activity in preventing acute GVHD after allogeneic hematopoietic cell transplantation; phase II portion of phase I/II study is ongoing
Dare Bioscience Inc., of San Diego DARE-HRT1 Intravaginal ring delivering bio-identical 17β-estradiol/progesterone continuously over 28-day period Vasomotor symptoms and genitourinary syndrome associated with menopause as part of hormone therapy regimen Completed patient recruitment 
Eucure Biopharma, of Boston, a subsidiary of Biocytogen Corp. YH-001 Anti-CTLA4 antibody Solid tumors Demonstrated encouraging antitumor activity in combination with Tuoyi (toripalimab, Junshi Biosciences Inc.) in dose-escalation trial in Australia
Faron Pharmaceuticals Oy, of Turku, Finland Bexmarilimab Anti-Clever-1 humanized antibody Solid tumors Updated data from 67 patients in phase I/II Matins trial in patients who have extinguished all treatment options showed survival benefit following 4 treatment cycles among 10 responding participants (partial response or stable disease); overall risk of death in those patients reduced by 88% vs. nonresponding patients
Harbour Biomed Holdings Ltd., of Suzhou, China HBM-4003 Cytotoxic T-lymphocyte protein-4 inhibitor Advanced melanoma and other solid tumors Dosed first patient in study testing combination with PD-1 drug teriprizumab
Rapt Therapeutics Inc., of South San Francisco RPT-193 CCR4 inhibitor Atopic dermatitis Phase Ib trial fully enrolled 31 participants with moderate to severe disease; data, including efficacy endpoints of EASI and pruritis Numerical Rating Scale, expected in first half of 2021
Rocket Pharmaceuticals Inc., of Cranbury, N.J. RP-L301  PKLR gene stimulator Pyruvate kinase deficiency Updated preliminary data showed durable normalization of hemoglobin levels up to 6 months following therapy in first participant and similar 3-month trends in second 
Terns Pharmaceuticals Inc., of Foster City, Calif. TERN-501  Thyroid hormone receptor beta agonist Nonalcoholic steatohepatitis First of about 90 healthy participants dosed; top-line data due in second half of 2021
Phase II
Azurrx Biopharma Inc., of Delray Beach, Fla. MS-1819 Lipase modulator Exocrine pancreatic insufficiency Trial to treat severe disease in people with cystic fibrosis fully enrolled 18 participants 12 and older; top-line data, including primary efficacy endpoint of coefficient of fat absorption levels, expected in second quarter of 2021
Bioage Labs Inc., of Richmond, Calif. BGE-175 PGD2 antagonist COVID-19 infection Trial initiated in 132 people ≥ 60 years old hospitalized with infection and not yet in respiratory failure; primary endpoint is proportion who die or progress to respiratory failure within 28 days of initial dose; top-line results expected by year-end 2021
Biophytis SA, of Paris Sarconeos (20‐hydroxyecdysone, BIO-101) Proto-oncogene Mas agonist COVID-19 infection Data monitoring committee for phase II/III Cova study in people 45 and older hospitalized with infection and severe respiratory effects issued favorable opinion on safety following scheduled interim analysis of 50 participants from part 1 and progression of enrollment, now at 97 of 155 needed for second interim analysis
Curevac NV, of Tübingen, Germany CVnCoV (SARS-CoV-2 mRNA vaccine) COVID-19 spike glycoprotein modulator COVID-19 infection Ongoing pivotal phase IIb/III Herald trial expected to include efficacy determination for selected emerging variants, based on ongoing trial amendment discussions with EMA; protocol amendment submitted to include secondary efficacy objective in ongoing phase IIa dose-confirmation trial in older adults in Peru and Panama; readouts from both trials expected in second quarter of 2021, potentially followed by MAA filing in same quarter
Leap Therapeutics Inc., of Cambridge, Mass. DKN-01 Dickkopf-1 ligand inhibitor Endometrial cancer In 22 women with epithelial endometrial cancer (EEC) dosed with study drug as monotherapy for whom DKK1 expression data was available, those with DKK1-high tumors (n=7) had greater overall response rate (14% vs. 0%), disease control rate (57% vs. 7%) and median progression-free survival (PFS, 3 months vs. 1.8 months) vs. those with DKK1-low tumors (n=15); in 24 women with EEC who received DKN-01 + paclitaxel, DKK1-high women (n=11) had improved median PFS (5.4 months vs. 1.8 months) vs. DKK1-low patients (n=9)
Neurorx Inc., of Radnor, Pa. Zyesami (inhaled aviptadil) VIP receptor agonist COVID-19 infection First participant dosed in phase II/III Avicovid-2 study in people with moderate and severe infection, aimed to prevent progression to respiratory failure
Phase III
Astrazeneca plc, of Cambridge, U.K. AZD-1222 COVID-19 spike glycoprotein modulator COVID-19 infection U.S. vaccine trial showed statistically significant efficacy of 79% at preventing symptomatic infection and 100% at preventing severe disease and hospitalization, based on interim analysis of 32,449 participants who accrued 141 symptomatic cases; primary analysis will be submitted to FDA for EUA
Genentech, unit of Roche Holding AG, of Basel, Switzerland  Tecentriq (atezolizumab) PD-L1 inhibitor Non-small-cell lung cancer  At interim analysis of IMpower010 study, Tecentriq met primary endpoint of disease-free survival vs. best supportive care, with statistically significant improvement as adjuvant therapy following surgery and chemotherapy in stage II-IIIA disease; data on intent-to-treat population did not cross threshold and overall survival data remained immature at time of analysis

Notes

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