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Apexian Pharmaceuticals Preclinical Studies With APX3330 Shut Down Inflammatory Signaling in Leukemia

December 19, 2018

INDIANAPOLIS–(BUSINESS WIRE)–An obscure sliver of RNA with a grim-sounding name is unexpectedly taking center stage as a linchpin in transforming pre-leukemic cells into full-blown leukemia. Apexian Pharmaceuticals’ latest studies in collaboration with Indiana University’s Simon Cancer Center investigator Dr. Reuben Kapur show promise for halting that transformation.

“Our ongoing pre-clinical research continues to identify the role that APX3330 plays in impacting disease including hematological disorders”

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These results, published December 6, 2018, in Cell Stem Cell, explain how a long noncoding RNA called Morrbid becomes hyperactivated after an acute inflammatory insult. Morrbid’s overzealous signaling functionally alters the bone marrow’s myeloid cells (precursors to white blood cells) when those cells are deficient in a tumor-suppressor gene called TET2. The cells mature rapidly, increase in absolute numbers, become resistant to programmed cell death, and churn out high levels of inflammatory proteins in a sustained, self-perpetuating response.

Inflammation has long been suspected of contributing to cancer, but these results establish a causative link. Dr. Kapur’s and Apexian’s results also support others’ findings that Morrbid controls myeloid cell lifespan.

Using a mouse model that mimics human acute myeloid leukemia, Dr. Kapur and Apexian’s Chief Science Officer, Mark Kelley, PhD demonstrated that Apexian’s flagship compound APX3330 could reduce Morrbid’s activity. Blocking both the precancerous cells’ growth and the cells’ production of inflammatory proteins.

Such anti-inflammatory therapy could be of clinical value in people carrying TET2 mutations.

“Our ongoing pre-clinical research continues to identify the role that APX3330 plays in impacting disease including hematological disorders,” said Dr. Mark Kelley, Chief Scientific Officer. “Inflammation plays a critical role in the growth and survival of blood stem cells with pre-leukemia mutations such as TET2 and increases the risk of blood related cancers. APX3330 blocked this change from pre-leukemia to leukemia in the mouse models.”

About Apexian Pharmaceuticals

Apexian Pharmaceuticals is a patient-centered biotechnology company dedicated to developing novel anti-cancer compounds that target the multiple functions of the APE1 protein. Apexian’s research is grounded and driven by the belief that “better is always possible.” Please visit for more information.


Roger D. Miller, COO
Telephone: 844-463-3330 x100