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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.
At LAVA, we are focused on applying our expertise in gamma-delta T-cell engagers (TCEs) to transform cancer therapy. We refer to our compounds as gamma-delta bispecific T-cell engagers, or gamma-delta bsTCEs. Our platform generates therapeutics that specifically activate a unique and relatively abundant effector gamma-delta T-cell subset called Vγ9Vδ2 (Vgamma9-Vdelta2) T-cells that have the ability to trigger activity of immune cells of both the innate and adaptive immune system. Our gamma-delta bsTCEs have demonstrated superior efficacy and safety profiles compared to other bsTCE approaches in preclinical studies and show preferential activity against tumor cells thereby potentially limiting toxicity in healthy tissue. To date, we are the only company developing bispecific gamma-delta T-cell engaging antibodies for the treatment of cancer. We are building a pipeline of gamma-delta bsTCEs that have applications in both solid and hematologic malignancies. Our lead program, LAVA-051, targets the tumor specific antigen CD1d, which can be overexpressed in multiple myeloma, chronic lymphocytic leukemia (CLL), and acute myeloid leukemia (AML), among other tumor types. LAVA-051 will enter a Phase I/IIa study in 1Q21.
Applied Molecular Therapeutics (AMT), a public biopharmaceutical company based in South San Francisco, California, is committed to developing novel, oral biological therapeutics to treat severe autoimmune, metabolic and inflammatory diseases. AMT is leveraging its propriety technology platforms to create gastrointestinal (GI)-select therapeutics that harness naturally occurring transport and targeting mechanisms to cross the protective barrier of the intestinal epithelium (IE). Once across the IE, the therapeutics gain privileged access to the immune cell-rich environment of the GI tissue as well as the hepatic portal system and downstream systemic circulation. With this privileged access, AMT`s goal is to develop transformative new oral therapeutic treatment options that offer patients greater efficacy and tolerability than available with today`s therapeutics. AMT has a robust pipeline of oral biologic product opportunities, including AMT-101 which is currently in Phase 1b clinical development for the treatment of adults with ulcerative colitis (UC). AMT-101 is a novel, gut-selective, investigational oral biologic fusion protein of interleukin 10 (IL-10), an anti-inflammatory cytokine. Derived from AMT`s proprietary platforms, AMT-101 is engineered to cross the selective barrier of the IE and enhance localized IL-10 directly within the immune-cell rich environment of GI tissue where up to 75 percent of immune cells reside. This direct targeting of the immune system creates the potential for AMT-101 to address, at its point of origin, the immune dysregulation that gives rise to UC and other inflammatory bowel diseases, and restore immune homeostasis with minimal systemic exposure and potentially fewer adverse events compared to systemic administration of IL-10. AMT has amassed a broad global IP portfolio surrounding its multiple technology platforms and therapeutic programs.
RAPT Therapeutics is a clinical stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases. Utilizing its proprietary discovery and development engine, the company develops highly selective small molecules that are designed to modulate the fundamental immune responses underlying these diseases. RAPT has rapidly discovered and advanced two unique drug candidates each targeting CCR4, including our lead oncology drug candidate, FLX475, now in clinical development and our lead inflammation drug candidate, RPT193, expected to enter the clinic in the second half of 2019. The company is also pursuing other discovery targets including GCN2 and HPK1 for the treatment of cancer.
Peptimmune, Inc. is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.