Date: 2017-12-11
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 59th American Society of Hematology (ASH) Annual Meeting in Orlando
Company: Epizyme (USA - MA)
Product: EZM8266
Action
mechanism: histone methyltransferase inhibitor
Disease: sickle cell disease (SCD)
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details:
Latest
news:
- • On December 11, 2017, Epizyme announced new preclinical data from its novel G9a program for sickle cell disease (SCD). The data were reported during an oral presentation at the 59th 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 ?-globinopathies, such as sickle cell disease and ?-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.
- 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.
Is
general: Yes
