Company Product Description Indication Status
Phase I
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 Sublingual thin-film formulation of dexmedetomidine Opioid withdrawal symptoms Enrolled first of about 125 patients in the dose-escalation Release study testing the safety, pharmacokinetics, tolerability and efficacy of BXCL-501; top-line data expected in first quarter of 2021
Bridgebio Pharma Inc., of San Francisco BBP-418  Enhances the function of fukutin-related protein Healthy volunteers (eventually limb girdle muscular dystrophy type 2i) First subject enrolled in study measuring the safety, tolerability, pharmacokinetics and food effect of BBP-418
Lyndra Therapeutics Inc., of Watertown, Mass.  LYN-005 Ultra-long-acting oral risperidone Schizophrenia Completed enrollment in the study; plans to start a phase II study this summer
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-COV2  Cocktail of 2 virus-neutralizing antibodies COVID-19 Started clinical trials testing 4 populations: hospitalized COVID-19 patients, non-hospitalized symptomatic COVID-19 patients, uninfected people in groups that are at high-risk of exposure (such as health care workers or first responders) and uninfected people with close exposure to a COVID-19 patient (such as the patient's housemate); first 2 adaptive studies will be in patients, with phase I portion focused on virologic and safety endpoints, phase II portion focused on virologic and clinical endpoints and the data from those phases used to refine the endpoints and determine size for the phase III
Phase II
Crinetics Pharmaceuticals Inc., of San Diego Paltusotine  Nonpeptide biased agonist of somatostatin sst2 receptor Acromegaly  Enrolled 28 of the 47 patients in the Acrobat Edge study of patients who have not achieved normal IGF-1 levels despite stable octreotide LAR or lanreotide depot therapy alone or combined with a dopamine agonist
Magenta Therapeutics Inc., of Cambridge, Mass. MGTA-456 Hematopoietic stem cell therapy  Inherited metabolic disorders Discontinued enrollment in study due to enrollment challenges, challenges of allogeneic stem cell transplant in patients with non-malignant disease and feedback from the FDA on endpoints and clinical trial design for a registration study
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID-19 mRNA vaccine COVID-19 prophylaxis Completed enrollment of 300 people, ages 18-54, and 50 people, ages 55 and older; expects to start phase III study in July
Partner Therapeutics Inc., of Lexington, Mass. Leukine (sargramostim) Recombinant human granulocyte-macrophage growth factor T-cell replete HLA-mismatched haploidentical stem cell transplant recipients who are receiving post-transplant cyclophosphamide Enrolled first patient in study testing Leukine compared to a matched control cohort of patients treated with granulocyte-macrophage growth factor
Pluristem Therapeutics Inc., of Haifa, Israel PLX cells Allogeneic mesenchymal-like cells Severe COVID-19 with acute respiratory distress syndrome Started the 140-patient study testing the number of ventilator-free days during the main 28-day study period; secondary endpoints include all-cause mortality, duration of mechanical ventilation, ICU-free days and hospitalization-free days
Relief Therapeutics Holding AG, of Geneva, and Neurorx Inc., of Radnor, Pa. RLF-100 (aviptadil) Vasoactive intestinal polypeptide  Critical COVID-19 with respiratory failure  Added Houston Methodist Hospital as a research site
Phase III
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Givlaari (givosiran) RNAi targeting aminolevulinic acid synthase 1 Acute hepatic porphyria Results of the Envision study published in The New England Journal of Medicine showed the drug reduced the annualized rate of composite porphyria attacks, defined as those attacks requiring hospitalization, urgent healthcare visit or intravenous hemin administration at home, by 74% compared to placebo
Clovis Oncology Inc., of Boulder, Colo. Rubraca (rucaparib) PARP inhibitor Front-line maintenance treatment of newly diagnosed advanced ovarian cancer Completed enrollment in the Athena study testing Rubraca plus Opdivo (nivolumab, Bristol Myers Squibb Co.); top-line data expected in the second half of 2021
Roche Holding AG, of Basel, Switzerland, and Exelixis Inc., of Alameda, Calif. Tecentriq (atezolizumab, Roche) and Cabometyx (cabozantinib, Exelixis) Monoclonal antibody targeting PD-L1 and a kinase inhibitor Metastatic non-small-cell lung cancer after treatment with an immune checkpoint inhibitor and platinum-containing chemotherapy Started the 350-patient Contact-01 study testing Tecentriq plus Cabometyx; primary endpoint is overall survival; secondary endpoints include progression-free survival, objective response rate and duration of response


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