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Sirion Therapeutics, Inc. is a Tampa, FL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Therigy® is a patient-focused specialty therapy management company. As an industry pioneer, Therigy continues to lead the specialty pharmacy market as the top choice in technology and consulting solutions—with the best possible outcomes for patients in mind.
Nightstar is a private biopharmaceutical company focused on the development of therapies for retinal dystrophies. The Company’s lead programme is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people. Gene therapy has the potential to be an effective treatment for choroideremia and a range of other retinal dystrophies. The Company’s lead investors are Syncona LLP and NEA Inc. Syncona is an evergreen investment company, taking an active role in identifying, supporting and developing technologies with the potential to impact significantly the healthcare market of the future. NEA is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors and geographies.
Cedarburg Hauser Pharmaceuticals is a Grafton, WI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.