X-Ceptor Therapeutics
X-Ceptor focused on the identification of novel drug candidates that interact with orphan nuclear receptors involved in cholesterol metabolism and cardiovascular disease. Several of its programs were partnered and further developed by large pharmaceutical companies.
Exelixis acquired X-Ceptor in 2004.
Variagenics was a pioneer in the field of pharmacogenomics, exploring the ways in which genetic variations among patients influence their response to particular drugs. It developed and commercialized proprietary gene variance detection technology to streamline drug development. Although its technology was ultimately absorbed into other companies, Variagenics contributed to the development of what is now understood as personalized medicine, a way of diagnosing medical conditions, selecting specific therapeutic drugs, and determining dosages based on a highly-tailored understanding of the patient’s individual genetic profile.
Ultragenyx was founded by Dr. Emil Kakkis, a widely recognized leader in the rare disease field. He and his team are focused on building a broad-based rare disease company addressing proteins or small molecules with clear disease targets and treatment mechanisms ready for development. The company’s lead program, UX-001, is a small molecule substrate replacement therapy for a rare muscle wasting disease, Hereditary Inclusion Body Myopathy (HIBM). Its portfolio also includes triheptanoin, a promising treatment for long-chain fatty acid oxidation disorders (FAOD), and UX003, a potential enzyme replacement therapy for mucopolysaccharidosis type 7 (MPS 7).
Ultragenyx completed a successful initial public offering (IPO) in 2014.
Based on proprietary tyrosine polymer technology, TYRX’s FDA-cleared lead product, AIGIS-Rx™, is an antibiotic-eluting mesh envelope into which pacemakers and defibrillators can be inserted prior to surgical implantation. Since coming onto the market in 2008, the product has been shown to substantially reduce the risk of high morbidity post-operative infections. TYRX has several antibiotic-eluting products that are in review or cleared for market including a fully resorbable CRM envelope, a surgical soft tissue repair mesh, and a hernia repair mesh.
TYRX won the 2012 Frost and Sullivan New Product Innovation Award, the Excellence in Surgical Products Award and the Web Health Award for HeartDeviceInfection.com. FierceMedicalDevices named TYRX as one of its “Fierce 15” Medical Device and Diagnostic Companies of 2012. The company was acquired by Medtronic, Inc. in early 2014.
The company is addressing a large unmet medical need: treating acute kidney injury (AKI) and chronic kidney disease (CKD). Thrasos is advancing therapeutic programs, led by THR-184, for renal diseases, including AKI and CDK. Thrasos' drug candidates have been shown to be orally active and to protect, repair and restore the cell and tissue function in the kidney and other organs.
Led by a proven management team, TESARO has in-licensed and is developing several promising drug candidates, including rolapitant and niraparib. Rolapitant is a potent selective neurokinin-1 receptor antagonist that has commenced Phase 3 clinical testing for the prevention of chemotherapy induced nausea and vomiting. Niraparib is an orally active and potent PARP inhibitor that will commence Phase 3 pivotal trials in both the ovarian and breast cancer settings.
TESARO was co-founded by former executives of MGI Pharma (acquired by Eisai Co., Ltd. for $3.9 billion) and Abraxis BioScience (acquired by Celgene for $2.9 billion). TESARO became a public company in 2012, raising $81 million.
After failing with its initial programs, TargeGen progressed its innovative JAK2 inhibitor from the discovery phase into clinical trials in record time, capitalizing on intense interest in the JAK2 marker within the pharmaceutical industry. Sanofi is currently developing TargeGen’s lead compound for the treatment of myelofibrosis and polycythemia vera, debilitating blood disorders for which there is a very large unmet medical need.
Sanofi acquired TargeGen in 2010 for up to $560 million in upfront and milestone payments.
Syntonix was formed to discover and develop novel therapeutic proteins for the treatment of chronic diseases such as hemophilia, anemia, multiple sclerosis and autoimmune disorders.
Biogen IDEC, which acquired Syntonix in 2007, is developing Syntonix’s lead product, recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B. The Syntonix product is the first product candidate in a new class of long-lasting clotting factor therapies that are being developed with the goals of reducing the burden of treatment for this condition and enhancing protection from bleeding.
Syndax is a late stage oncology company whose lead product candidate is entinostat, an oral, selective histone deacetylase inhibitor (HDACi) that is being developed to treat therapy-resistant cancers through an epigenetic mechanism. Entinostat was recently granted Breakthrough Therapy designation by the FDA based on data from the company's completed Phase 2b clinical trial for ER+ metastatic breast cancer.
Spherics focused on applying its oral drug delivery platform to develop products with improved therapeutic profiles.
