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We are pioneering a new therapeutic approach to treat debilitating ear, nose and throat (ENT) diseases that impact the lives of millions of people.
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The Company is building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms are based on deep understandings of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.
Janssen Alzheimer Immunotherapy div JNJ is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Qualicaps is a global supplier of two-piece capsules and related encapsulation equipment. Employees are our most valuable internal asset and we foster a creative, challenging environment in which personal and professional achievements are recognized.
Madrigal Pharmaceuticals is a clinical-stage biopharmaceutical company pursuing novel therapeutics that target a specific thyroid hormone receptor pathway in the liver, which is a key regulatory mechanism common to a spectrum of cardio-metabolic and fatty liver diseases with high unmet medical need. The Company has advanced its lead candidate, resmetirom, also known as MGL-3196, a first-in-class, orally administered, small-molecule, liver-directed, thyroid hormone receptor (THR) β-selective agonist, through Phase 2 clinical trials in NASH. Primary and key secondary endpoints were achieved including reduction of liver fat on a sensitive non-invasive imaging test, lowering of multiple atherogenic lipids including LDL-cholesterol and triglycerides, and resolution of NASH on liver biopsy. Based on evidence of broad activity and a favorable safety profile, Madrigal has initiated a Phase 3 clinical study in NASH patients with advanced liver fibrosis, MAESTRO-NASH, in which the primary endpoint after one year of dosing is resolution of NASH and key secondary endpoints include LDL-cholesterol lowering and reduction in liver fibrosis. Clinical benefit in reducing progression to more advanced liver disease, including cirrhosis, is a long-term goal of this trial. A second Phase 3 clinical study, MAESTRO-NAFLD-1, has also been initiated. This 52-week non-invasive study in a broader segment of NASH patients will provide additional safety information to support the NASH indication and will include key efficacy endpoints: LDL-cholesterol lowering, triglyceride lowering, reduction of liver fat as determined by MRI-PDFF, as well as reduction of elevated liver enzymes and reduction in fibrosis biomarkers. This is a non-biopsy study in people with NASH, as documented using non-invasive techniques or historical liver biopsy.