19 November 2018 - - German drugmaker Boehringer Ingelheim and US-based Epizyme, Inc. (NASDAQ: EPZM) have forged a new global collaboration focused on the research, development and commercialization of novel small molecule inhibitors directed toward two previously unaddressed epigenetic targets as potential therapies for people with cancer, the companies said.

Specifically, these targets are enzymes within the helicase and histone acetyltransferase families that when dysregulated have been linked to the development of cancers that currently lack therapeutic options.

Epigenetic modification affects how genes are biologically regulated. More than half of cancers can stem from functional errors in epigenetic modification.

In particular cases, epigenetic dysregulation is associated with alterations in specific components of gene regulation, which can be used to identify patients most likely to benefit from the therapy.

What makes both targets compelling is not only the clear role they play in cancer but that both targets have patient stratification biomarkers, which will enable a focus on the patients most likely to benefit from these potential treatments.

Epizyme is in the discovery of the roles of such enzymes and their development as therapy targets.

The collaboration has a strategic goal to focus on lung and other solid tumor cancers in patients with defined mutations, sub-populations that currently lack precision medicine treatments.

Under the terms of the agreement, Boehringer Ingelheim and Epizyme will jointly research and develop a helicase program, with both parties sharing US commercialization responsibilities and Boehringer Ingelheim assuming responsibility for commercialization outside the US Epizyme and Boehringer Ingelheim will share research responsibilities for the HAT program, with Boehringer Ingelheim assuming responsibility for worldwide development and commercialization.

Epizyme will receive an upfront payment of USD 15m and an additional USD 5m in research funding in 2019.

The company is eligible to receive more than USD 280m in additional payments for research, development, regulatory and commercial milestones.

For the helicase programme, Epizyme will fund a portion of the global development costs, retain a share of US profits and receive tiered royalties on ex-US sales. For the HAT program, Epizyme is eligible to receive tiered royalties on worldwide sales.