Company Product Description Indication Status
Phase I
Aim Immunotech Inc., of Ocala, Fla. Ampligen (rintatolimod) Double-stranded RNA COVID-19 infection Recruitment began at Roswell Park Comprehensive Cancer Center in phase I/IIa combination study with interferon alpha-2b in individuals with cancer who have mild or moderate infection
Exicure Inc., of Chicago Cavrotolimod TLR9 agonist Solid tumors Phase Ib combination trial with Keytruda (pembrolizumab, Merck & Co. Inc.) showed confirmed overall response rate (ORR) of 21% (4 of 19) in dose-escalation stage across all doses, with confirmed ORR 33% (2 of 6) at highest and selected phase II dose (32 mg); target tumor shrinkage observed in 37% of participants
Immune-Onc Therapeutics Inc., of Palo Alto, Calif. IO-202 Leukocyte Ig-like receptor-4 antagonist Acute myeloid leukemia First participant dosed in study enrolling individuals with AML with monocytic differentiation and with chronic myelomonocytic leukemia; trial designed to identify optimal dose for planned expansion cohort and to assess biomarkers
Menarini Ricerche, unit of Menarini Group, of Pomezia, Italy, and Ryvu Therapeutics SA (previously Selvita SA), of Krakow, Poland MEN-1703 (SEL-24) Dual PIM/FLT3 inhibitor Acute myeloid leukemia First participant dosed in cohort expansion of Diamond-01 monotherapy trial in people with relapsed or refractory disease or previously untreated and unsuitable for chemotherapy
Verastem Oncology Inc., of Boston VS-6766 + defactinib RAF/MEK inhibitor + FAK inhibitor Ovarian cancer In ongoing investigator-initiated phase I/II Frame study, among 17 patients with low-grade serous disease (LGSOC), overall response rate (ORR) was 41% (n=7), all partial responses (PRs); among those with KRAS-G12 mutant LGSOC, ORR was 56% (5 of 9) and 50% (3 of 6) in those who received recommended phase II dosing regimen
Phase II
Adrenomed AG, of Germany Adrecizumab (HAM-8101) Monoclonal antibody targeting adrenomedullin  Vascular integrity in patients with septic shock In the AdrenOSS-2 study, 28-day all-cause mortality showed a trend toward a survival benefit for patients given adrecizumab; at day 14, there was a 45% relative mortality reduction for adrecizumab (p=0.057); Sequential Organ Failure Assessment score was reduced after treatment with adrecizumab (p<0.05)
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (Ifenprodil) N-methyl-D-aspartate receptor antagonist COVID-19 Data and safety monitoring board unanimously approved continuing the study
Arrowhead Pharmaceuticals Inc., of Pasadena, CA ARO-AAT RNAi targeting AAT Alpha-1 antitrypsin deficiency In the AROAAT2002 study, serum and total intrahepatic Z-AAT decreased in all 4 patients by up to 93% and 95%, respectively; 3 of 4 patients had reductions from baseline in intrahepatic Z-AAT polymer, with a maximum reduction of 97%; all 4 had reductions in ALT and GGT, with maximum reductions of 66% and 58%, respectively; 3 of 4 had greater than 20% reductions in transient elastography Fibroscan values
Eli Lilly and Co., of Indianapolis LY-CoV555 SARS-CoV-2 neutralizing antibody Mild to moderate COVID-19 In the Blaze-1 study, change from baseline in viral load at day 11 was improved for the 2,800-mg dose compared to placebo; 1.7% of the 302 patients who received any dose level of LY-CoV555 had a COVID-19-related hospitalization or ER visit compared to 6% of the 150 patients taking placebo
Galera Therapeutics LLC, of Malvern, Pa. Avasopasem manganese (GC-4419) Superoxide dismutase mimetic  Hospitalized, critically ill COVID-19 First of up to 50 patients dosed in study testing 28-day mortality; trial will also measure requirement for intensive care and mechanical ventilation, as well as organ function
Genelux Corp., of San Diego Olvi-Vec-primed Oncolytic virus Platinum-resistant/refractory ovarian cancer Median overall survival was 15.7 months for 27 patients; 33.3% of patients survived longer than 18 months; in patients with platinum-refractory disease, median OS was 15.2 months
Heron Therapeutics Inc., of San Diego HTX-011 Non-opioid analgesic Pain associated with open inguinal hernia repair surgery with mesh Data published in Surgery showed 90.5% of patients who received HTX-011 remained opioid-free through 72 hours postoperatively; 82.5% of patients remained opioid-free through day 28
Knopp Biosciences LLC, of Pittsburgh Dexpramipexole  Reduces eosinophil levels Moderate to severe eosinophilic asthma Completed enrollment in the study; top-line data expected in early 2021
Springworks Therapeutics Inc., of Stamford, Conn. Nirogacestat Gamma secretase inhibitor Aggressive fibromatosis Study sponsored by Children’s Oncology Group plans to enroll about 30 children and adolescents with progressive, unresectable disease, with recruitment beginning in September 2020; primary endpoint is 2-year progression-free survival
Verona Pharma plc, of London Ensifentrine  Phosphodiesterase 3 and 4 inhibitor Chronic obstructive pulmonary disease Data published in the International Journal of Chronic Obstructive Pulmonary Disease showed all 4 doses superior to placebo from week 2 through week 4 on the breathlessness subscale of the E-RS: COPD (p<0.05); all ensifentrine doses were superior to placebo on the Transitional Dyspnea Index focal score at weeks 2 and 4 (p<0.05)
Phase III
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-151 (topical roflumilast cream) PDE4 inhibitor  Plaque psoriasis Completed enrollment in the Dermis-1 and -2 studies; top-line data expected in the first quarter of 2021
Arena Pharmaceuticals Inc., of San Diego Etrasimod Sphingosine 1-phosphate receptor modulator Severely active ulcerative colitis Enrolled first patient in the Elevate UC 12 study; primary endpoint is clinical remission at 12 weeks assessed by the 3-domain, modified Mayo Score; secondary endpoints include efficacy of etrasimod on clinical response, symptomatic response and remission, endoscopic changes, corticosteroid-free remission and total healing
Aveo Oncology Inc., of Boston Tivozanib Vascular endothelial growth factor receptor tyrosine kinase inhibitor Third- and fourth-line renal cell carcinoma Data from the Tivo-3 study published in European Urology showed a median progression-free survival of 5.6 months for tivozanib compared to 3.9 months for sorafenib (p=0.02); overall survival slightly favored tivozanib (p=0.82) 
Phase IV
Horizon Therapeutics plc, of Dublin Krystexxa (pegloticase) Uricase stimulator  Chronic gout refractory to conventional therapies In the Mirror study published in the Journal of Rheumatology, 79% of the 14 patients who received Krystexxa and methotrexate maintained therapeutic response (defined as sUA <6 mg/dL) during month 6


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