Company Product Description Indication Status
Phase I
Carisma Therapeutics Inc., of Philadelphia CT-0508 HER2-targeted CAR macrophage Recurrent or metastatic HER2-overexpressing solid tumors Dosed first patient in study focusing on patients whose cancers do not have any approved HER2-targeted therapies or who do not respond to treatment
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. Trans sodium crocetinate (TSC) Oxygen-enhancing therapeutic Peripheral tissue oxygenation Dosed first participants in study designed to use transcutaneous oxygen monitoring device to evaluate effects of TSC on peripheral tissue oxygenation
Noxopharm Ltd., of Sydney Veyonda  Topoisomerase inhibitor and radiopharmaceutical COVID-19 Approved to move into second and final stage of Noxcovid-1 trial; in part 1, 1,800-mg dose was deemed sufficiently well-tolerated in patients with moderate COVID-19 to become preferred dose; part 2 will recruit minimum of 10 and up to 15 patients with moderate to severe lung dysfunction
Sorrento Therapeutics Inc., of San Diego STI-6129 Antibody-drug conjugate Amyloid light chain amyloidosis Treated first patient in phase Ib trial; open-label, dose-escalation study will enroll patients with advanced relapsed and/or refractory systemic disease, with primary objective of identifying phase II dose
Translate Bio Inc., of Lexington, Mass. MRT-5005 mRNA therapy; CFTR modulator Cystic fibrosis Second interim analysis from phase I/II study showed percent predicted forced expiratory volume in 1 second (ppFEV1), a measure of lung function, was assessed as a safety measure at predefined timepoints throughout the trial, with no pattern of increases in ppFEV1; repeat dosing generally safe and well-tolerated
Vaccitech Ltd., of Oxford, U.K. VTP-200 Prime-boost HPV vaccine  Human papillomavirus infection Dosed first patient in phase I/II HPV001 study to enroll 105 women with high-risk HPV infection
Phase II
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-252 Topical small-molecule inhibitor of JAK1 Vitiligo Enrolled first patient in phase IIa trial
Hovione LLC, of Cork, Ireland Minocycline Matrix metalloprotease inhibitor Dry eye caused by Meibomian gland dysfunction Completed trial in 270 patients; observed superior clinical outcome in the 70% of patients with positive inflammatory biomarker at baseline, who achieved statistically significant (p=0.02) improvement of 25 points in Visual Analogue Scale discomfort after 2 weeks of treatment, dropping further to 35 points by end of treatment (improvement greater than 50%); sign endpoint inferior Cornea Fluorescein Staining, had statistically significant improvement against vehicle in both active arms at day 57 (p=0.009)
Imara Inc., of Boston IMR-687 Small-molecule inhibitor of PDE9 Sickle cell disease Independent data monitoring committee for Ardent phase IIb study recommended opening higher dose treatment arm
Imara Inc., of Boston IMR-687 Small-molecule inhibitor of PDE9 Beta-thalassemia Independent data monitoring committee for Forte phase IIb study recommended opening higher dose treatment arm
Italfarmaco Group, of Milan, Italy Givinostat HDAC inhibitor Polycythemia vera Interim analysis published in Blood Cancer Journal from a follow-up of 4 years in 51 patients showed long-term benefit; more than 80% responded to treatment, according to ELN Response Criteria, with response maintained for duration of follow-up period; reduction in mean JAK2 allele burden also was observed
Maat Pharma SA, of Lyon, France MaaT-013 Microbiome ecosystem therapy Steroid-refractory, gastrointestinal-predominant acute graft-vs.-host disease Study in a high-risk population with grade III-IV GI-aGVHD met primary endpoint, demonstrating combined 33.3% complete response rate or very good partial response rate at day 28; overall response rate was 38.1% at day 28 and best overall response rate was 57.1%; company plans to launch pivotal trial
Reithera Srl, of Rome GRAd-COV2 Vaccine COVID-19 Started Covitar phase II/III study to test safety, immunogenicity and efficacy vs. placebo for the prevention of COVID-19 in adults 18 and older; the first part will be a phase II to confirm safety and immunogenicity and select the regimen for phase III
Retrotope Inc., of Los Altos, Calif. RT-001 Isotopically stabilized, synthetic linoleic acid Amyotrophic lateral sclerosis Dosed first patients; study to enroll about 40 subjects, with data available by the end of 2021
Spruce Biosciences Inc., of San Francisco Tildacerfont CRF-1 receptor antagonist Congenital adrenal hyperplasia Results from open-label study evaluating drug in patients with CAH due to 21-hydroxylase deficiency showed good correlation between salivary and serum assessments of 17-OHP and A4, indicating that measurement of hormones in saliva may offer a promising, non-invasive approach to more frequently assessing response to therapy in patients with CAH
Spruce Biosciences Inc., of San Francisco Tildacerfont CRF-1 receptor antagonist Congenital adrenal hyperplasia Results from 12-week phase IIa study testing 400-mg dose showed tildacerfont-treated patients with poor disease control had mean maximum reductions of 84% in adrenocorticotropic hormone (ACTH), 80% in 17-OHP, and 79% in A4 compared to baseline at 8:00 a.m.; 60% of patients achieved normalization of ACTH levels, and 40% achieved normalization of A4 levels during month 3
Phase III
Eli Lilly and Co., of Indianapolis Verzenio (abemaciclib) CDK 4/6 dual inhibitor Breast cancer Patient-reported outcomes in trial combining use with standard adjuvant endocrine therapy in patients with HR+, HER2-negative high-risk early stage disease showed, in 1 analysis, data indicated that most patients (70%-75%) in both arms reported being bothered "a little bit" or "not at all" by treatment-related side effects
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. Edaravone Neuroprotectant Amyotrophic lateral sclerosis Post-hoc analysis showed risk reduction observed for the exploratory composite estimate of time to death, tracheostomy, permanent assisted ventilation (PAV) and hospitalization; during open-label period, there were 2 deaths in edaravone-edaravone (EE) group and 4 in edaravone-placebo (EP) group; survival analysis of cumulative occurrence of milestone events showed 53% relative risk reduction in EE group
Regenerx Biopharmaceuticals Inc., of Rockville, Md. RGN-259 Thymosin beta 4 ligand; eye drops Dry eye syndrome Arise-3 study did not meet primary outcome measure; showed improvement in 1 of prespecified secondary symptom endpoints, improvement of ocular grittiness
Swedish Orphan Biovitrum AB, of Stockholm, and Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan Targeted C3 therapy Paroxysmal nocturnal hemoglobinuria Results from Pegasus study published in The New England Journal of Medicine demonstrated superiority in improving hemoglobin levels vs. eculizumab in adults at 16 weeks who had persistent anemia following treatment with eculizumab; superiority in significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.001); 85% of pegcetacoplan-treated patients were transfusion-free over 16 weeks vs. 15% of eculizumab-treated patients


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