CAMBRIDGE, Mass., Dec. 11, 2017 (GLOBE NEWSWIRE) -- Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage company developing novel epigenetic therapies, announced today new preclinical data from its novel G9a program for sickle cell disease (SCD). The data were reported during an oral presentation at the 59 th Annual Meeting & Exposition of the American Society of Hematology (ASH) in Atlanta, Ga. In addition, the company announced that its drug development candidate, EZM8266, a G9a inhibitor, will begin IND-enabling studies in 2018.

It is widely understood within the SCD research community that elevation of fetal hemoglobin, which is normally silenced after birth, has disease-modifying potential for patients with ß-globinopathies, such as sickle cell disease and ß-thalassemia. To this end, multiple academic groups have previously discovered that inhibition of the histone methyltransferase (HMT) G9a leads to increased levels of fetal hemoglobin in preclinical in vitro studies. Building upon these findings, scientists from Epizyme leveraged their expertise in HMT drug discovery to generate potent, selective inhibitors of G9a with drug-like properties. A tool compound derived from these efforts induced on-target elevation of fetal hemoglobin in cell culture assays. Additionally, this compound elicited significant increases in mouse embryonic hemoglobin, which is the rodent developmental equivalent of human fetal hemoglobin. Epizyme believes these findings to be the first in vivo study to demonstrate reactivation of developmental hemoglobin with a G9a inhibitor.

"Sickle cell disease is considered one of the first genetically defined diseases, yet despite all the advances of modern medicine, it remains an area of significant medical need," said Richard Chesworth, DPhil, senior vice president of research at Epizyme. "There is proof in nature that fetal hemoglobin can have true disease-modifying potential. Our internally discovered small molecule further illustrates this disease modifying potential and differentiates Epizyme in the field of sickle cell disease."

Based on its research efforts, Epizyme has named the next drug development candidate in the company's pipeline, EZM8266—a potent, selective and orally bioavailable G9a inhibitor. Throughout 2018, Epizyme will work to advance EZM8266 toward clinical initiation, completing the necessary pre-IND work, including GLP toxicology studies. Epizyme holds worldwide development and commercialization rights to EZM8266.

"We are excited about the potential of our novel, internally discovered program," said Robert Bazemore, president and chief executive officer of Epizyme. "This mechanism, combined with our data, give us great confidence in targeting G9a. We look forward to advancing EZM8266 toward clinical development and the continued execution of our vision through 2020."

The oral presentation provides details on Epizyme's tool compound that supports further study of G9a inhibition and the reactivation of fetal hemoglobin (abstract #537): Reawakening of Human Fetal Hemoglobin and an Epigenetic Path to the Clinic for Sickle Cell Disease and Beta-Thalassemia: Identification of an Orally-Available, Potent, and Selective Euchromatic Histone Lysine Methyltransferase 1 and 2 (EHMT1/2) Inhibitor.

About Sickle Cell DiseaseSickle cell disease  (SCD) is an inherited red blood cell disorder. People with SCD have abnormal hemoglobin, called hemoglobin S or  sickle hemoglobin, in their red blood cells. A monogenic disease, SCD is most common in people with African ancestry. Approximately 300,000 people have sickle cell disease globally, with an estimated 150,000 of those patients located in the U.S. and Europe. Potential complications include stroke, vaso-occlusive crises (VOC) associated with pain attacks, acute chest syndrome and anemia. Recurrent acute pain crises and chronic long-term organ damage are the hallmarks for SCD. However, symptoms of SCD can vary significantly in different patients, and better treatments for SCD patients are needed. Life expectancy of SCD patients still lags the life expectancy of the general population.

About Epizyme, Inc. Epizyme, Inc. is a clinical-stage biopharmaceutical company committed to rewriting treatment for cancer and other serious diseases through novel epigenetic medicines. Epizyme is broadly developing its lead product candidate, tazemetostat, a first-in-class EZH2 inhibitor, with studies underway in both solid tumors and hematological malignancies, as a monotherapy and combination therapy in relapsed and front-line disease. The company is also developing a novel G9a program and its next development candidate, EZM8266, is targeting sickle cell disease. By focusing on the genetic drivers of disease, Epizyme's science seeks to match targeted medicines with the patients who need them. For more information, visit

Cautionary Note on Forward-Looking Statements Any statements in this press release about future expectations, plans and prospects for Epizyme, Inc. and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation of future clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; whether results from clinical studies will warrant meetings with regulatory authorities, submissions for regulatory approval or review by governmental authorities under the accelerated approval process; whether Fast Track Designation and Orphan Drug Designations will provide the benefits for which tazemetostat is eligible; expectations for regulatory approvals to conduct trials or to market products; whether the company's cash resources will be sufficient to fund the company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the company's therapeutic candidates; and other factors discussed in the "Risk Factors" section of the company's most recent Form 10-Q filed with the SEC and in the company's other filings from time to time with the SEC. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof and should not be relied upon as representing the company's views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.

Contacts:Cheya Pope, Epizyme, Inc. 617-229-7561

Monique Allaire, THRUST IR 617-895-9511

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