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Epizyme is a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative personalized therapeutics for patients with genetically defined cancers. We systematically identify the genetic alterations that create cancer causing genes, called oncogenes, select patients in whom the identified genetic alteration is found, and then design small molecule therapeutic product candidates to inhibit the oncogene. The clinical development plan for each of our therapeutic product candidates is directed towards patients with a particular genetically defined cancer. Our approach is part of a broader trend towards personalized therapeutics based on first identifying the underlying cause of a disease affecting specific patient populations, applying rational drug design tools to create a therapeutic to inhibit a molecular target in the identified disease pathway, and using a companion diagnostic to select the right patients for treatment. We have built a proprietary product platform that we use to create small molecule inhibitors of a 96-member class of enzymes known as histone methyltransferases, or HMTs. Genetic alterations can result in changes to the activity of HMTs, making them oncogenic. When Epizyme was founded, we recognized that the HMT class of enzymes might contain many potential oncogenes and presented the opportunity to discover, develop and commercialize multiple personalized therapeutics. We have prioritized 20 of the 96 HMTs as attractive targets for personalized therapeutics based on their oncogenic potential. Our two most advanced therapeutic product programs target the HMTs DOT1L (for the treatment of acute leukemias with genetic alterations of MLL) and EZH2 (for a genetically defined subtype of non-Hodgkin lymphoma and solid tumors including INI1-deficient tumors). We believe that our ongoing Phase 1 adult trial for EPZ-5676, targeting DOT1L, is the first clinical trial of an HMT inhibitor. In May 2014, we initiated a Phase 1b clinical trial for EPZ-5676 in pediatric patients with MLL-r leukemia, which is considered to be the last largely untreatable pediatric acute leukemia. We are also conducting a Phase 1/2 clinical trial of EPZ-6438, which is being developed for the treatment of non-Hodgkin lymphoma and solid tumors including INI1-deficient tumors such as synovial sarcoma and malignant rhabdoid tumors, or MRT. We were founded in 2007 and are led by a management team with extensive experience in the pharmaceutical industry. We have entered into therapeutic collaborations with Celgene, Eisai and GSK that have provided us with approximately $184 million in non-equity funding. As of June 30, 2014, we had $232.1 million in cash, cash equivalents and accounts receivables.
Impact Biomedicines, Inc. (Impact) is pioneering the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers. Led by a highly skilled and devoted team, Impact is advancing a late-stage pipeline centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor, which is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). Impact was formed in 2016 after acquisition of Sanofi’s full rights for the global development and commercialization of fedratinib. The Impact team intends to pursue multiple clinical indications for fedratinib to maximize its potential as a best-in-class therapy for JAK2-dependent diseases.
Applied Biophysics Inc is a Troy, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Beyond Air, Inc. is a clinical-stage medical device and biopharmaceutical company developing a revolutionary NO Generator and Delivery System, LungFit®, that uses NO generated from ambient air to deliver precise amounts of NO to the lungs for the potential treatment of a variety of pulmonary diseases. The LungFit® can generate up to 400 ppm of NO, for delivery either continuously or for a fixed amount of time and has the ability to either titrate dose on demand or maintain a constant dose. The Company is currently applying its therapeutic expertise to develop treatments for pulmonary hypertension in various settings, in addition to treatments for respiratory tract infections that are not effectively addressed with current standards of care. Beyond Air is currently advancing its revolutionary LungFit® for clinical trials for the treatment of severe lung infections such as acute viral pneumonia (including COVID-19) and nontuberculous mycobacteria (NTM). Additionally, Beyond Air is using ultra-high concentrations of NO with a proprietary delivery system to target certain solid tumors in the pre-clinical setting.
Pinetree Therapeutics is an award-winning, pre-clinical stage biotechnology company based in Cambridge, Massachusetts. We are dedicated to finding solutions for unmet medical need, such as drug resistance and tumor recurrence, in targeted therapy by degradation of disease-causing proteins with AbReptorTM platform. Novel degrader drugs have been exciting scientific field, due to its distinct mechanism of action (removal of disease-causing protein). AbReptorTM was developed for targeted degradation on membrane bound and extracellular proteins causing diseases. Receptor tyrosine kinase (RTK) and non-RTK inhibitors have made huge progress in the past few years, but their application is limited due to specificity, drug resistance, and on-target toxicity. Our platform`s RTK degraders promise to redefine their distinctive mutation-agnostic properties, differing from existing RTK therapeutics field. With the pre-clinical animal study, our lead molecule demonstrated on-target degradation, mutation agnostic anti-tumor effect, and potent anti-tumor effect in RTK inhibitor resistant tumor. Surprisingly, no signs of toxicity were found in rodent and cynomolgus monkey. Furthermore, it would unluck full potential to solve limitation of RTK and non-RTK therapeutics. Immune checkpoint inhibitors (ICIs) field made an initial breakthrough with poor response rate, high toxicity profiles, and exhausted poor clinical outcome. Our platform has full potential to solve the current problem of immune checkpoint inhibitors (ICIs), PD-(L)1 inhibitors. Our PD-(L)1 degrader is the first ever in-class to demonstrate enhanced anti-tumor immunity in pre-clinical animal studies. Pinetree Therapeutics` lead molecule is in pre-clinical IND stage, and we are committed to expanding our platforms to other target proteins, such as RTKs, non-RTKs, and ICIs. We believe our platform has the potential to transform the field of medicine and revolutionize the treatment of cancer and other diseases.