Table 2. Selected clinical stage compounds that target epigenetic regulators. The availability of new research tools and data on epigenetic regulators has driven the development of a new wave of compounds. Many of these compounds recently entered Phase I trials, and companies now point to Phase II data as the next milestone that will be key to determining which molecules and their respective targets will pan out for the field. Table excludes class-selective and pan-histone deacetylase (HDAC) inhibitors.
Source: BCIQ: BioCentury Online Intelligence; BioCentury Archives

Company

Compound

Description

Indication

Status

Milestone(s)

Acetylon Pharmaceuticals Inc.

ACY-1215

Oral selective HDAC6 inhibitor

Multiple myeloma (MM)

Phase Ib

Start Phase II (early 2014); additional Phase Ib data (December 2013)

Astex Pharmaceuticals Inc.A

SGI-110

Small molecule DNA methyltransferase inhibitor

Liver cancer; acute myelogenous leukemia (AML); myelodysplastic syndrome (MDS); ovarian cancer

Phase II;
Phase I/II; Phase I/II; Phase I/II

Phase II data (December 2013)

Constellation Pharmaceuticals Inc.

CPI-0610

BET bromodomain inhibitor

Lymphoma

Phase I

Undisclosed

Epizyme Inc. (NASDAQ:EPZM); Eisai Co. Ltd. (Tokyo:4523; Osaka:4523)

EPZ-6438

Selective inhibitor of enhancer of zeste homolog 2 (EZH2)

Non-Hodgkin's lymphoma (NHL)

Phase I/II

Phase I data (2014); start Phase II (2014)

Epizyme; Celgene Corp. (NASDAQ:CELG)

EPZ-5676

Histone methyltransferase DOT1L (DOT1L) inhibitor

Leukemia

Phase I

Phase I data (4Q13)

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK)

GSK525762

Bromodomain inhibitor

Epithelial cancer

Phase I

Phase I data (YE14 or early 2015)

Oncoethix S.A.; Mitsubishi Tanabe Pharma Corp. (Tokyo:4508; Osaka:4508)

OTX015

Synthetic small molecule inhibitor of BET bromodomain containing 2 (BRD2), BRD3 and BRD4

Hematologic malignancies

Phase I

Unavailable

Resverlogix Corp. (TSX:RVX)

RVX-208B

Inhibitor of the BET family of bromodomain-containing proteins, including BRD4

Atherosclerosis

Phase IIb

Unavailable

AAcquired by Otsuka Pharmaceutical Co. Ltd. BCompany had reported top-line Phase II data from the 324-patient trial showing that RVX-208 missed the primary endpoint of 0.6% reduction in atheroma volume from baseline at 26 weeks, but subsequent analysis identified a 92-patient subgroup with 1.4% reduction in atheroma volume from baseline at 26 weeks.