Sumitomo Pharma Oncology, a clinical-stage company focused on novel cancer therapeutics, announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for TP-3654, the company's proprietary investigational oral inhibitor of PIM kinases, for the treatment of myelofibrosis.

"This designation is an important milestone in the development of TP-3654 and highlights the need for potential new treatment options for patients with myelofibrosis," said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. "This rare hematologic cancer can also progress and worsen. We are excited about collaborating with investigators to advance this clinical-stage asset with the goal of improving patient outcomes."

The FDA's Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Myelofibrosis is a rare type of bone marrow cancer which disrupts an individual's normal production of blood cells.1 Myelofibrosis has approximately 1.5 reported cases per 100,000 people each year in the United States.2

"TP-3654 is an investigational oral inhibitor of PIM kinases. PIM kinases have potential antitumor and anti-fibrotic effects through multiple pathways, including induction of apoptosis.3,4 Notably, PIM kinase expression correlates with increased cell survival and reduced apoptosis in tumors, supporting the potential of PIM kinases as novel therapeutic targets,"3 explained Jatin J. Shah, M.D, Chief Medical Officer of Sumitomo Pharma Oncology, Inc. "PIM-1 expression is significantly elevated in myelofibrosis hematopoietic cells and therefore a potential therapeutic target for myelofibrosis."4

TP-3654 is currently being evaluated in a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with intermediate or high-risk primary or secondary myelofibrosis. It is being conducted in the United States and Japan.