EPZ-719 Shows Promise in Mice Models of MM

During the European Hematology Association (EHA) Annual Congress (EHA2021), which took place virtually this year, hematological experts and stakeholders discussed new advances in the field. According to Fierce Biotech, one presentation centered around the efficacy of EPZ-719 for multiple myeloma (MM). The therapy, developed by biopharmaceutical company Epizyme, conferred positive benefits in mice models. Interested in learning more? Check out Abstract S176.


Previously, researchers suggested that chromosome 4 and 14 rearrangements and genetic mutations played a role in certain multiple myeloma cases. In fact, this chromosomal abnormality is associated with anywhere from 15-20% of MM diagnoses, as well as worse patient outcomes. Prior research highlighted MMSET overexpression caused by this mutation as a possible therapeutic target for multiple myeloma. However, researchers were set back when MMSET was deemed as an undruggable target.

Thus, researchers changed their focus to SETD2 which, like MMSET, plays a role in gene expression, RNA splicing, and DNA damage repair. To begin, researchers sourced mice models of MM. While some mice had the specific genetic mutation, others did not. Next, researchers evaluated varying EPZ-719 doses. Ultimately, the findings showed that:

  • In mice receiving the highest three doses, tumor regression (reduction in tumor size) was seen.
  • EPZ-719 showed promise in mutated MM, prompting a 95% tumor growth inhibition. Additionally, EPZ-719 also showed promise for mice models without the specific mutation, inhibiting tumor growth.
  • Altogether, EPZ-719 could be a promising treatment on its own or in conjunction with another therapy.

Over the next few months, Epizyme hopes to submit an Investigational New Drug (IND) application. Approval of this application would allow Epizyme to begin running human clinical trials.

Multiple Myeloma (MM)

Multiple myeloma (MM) is a cancer beginning in plasma cells, a type of white blood cell. Normally, plasma cells play a role in immune health, creating antibodies, fighting infections, and destroying “foreign invaders.” However, in MM, the cancerous plasma cells crowd healthy cells out of the bone marrow. Further, MM cells create abnormal M proteins. Altogether, this causes a variety of health-related complications. Typically, MM is more common in males than females. Additional risk factors include age, being Black, or having a family history of multiple myeloma. Symptoms include:

  • Unintended weight loss
  • Bone pain (often in the spine or chest)
  • Nausea
  • Kidney damage and/or kidney failure
  • Appetite loss
  • Frequent infections
  • Leg weakness or numbness
  • Anemia (low red blood cell count)
  • Bone fractures
  • Fatigue
  • Mental fogginess
  • Constipation
  • Excessive thirst
  • Hypercalcemia (excess calcium in the blood)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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