Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, today announced the U.S. Food and Drug Administration granted Orphan Drug Designation for TP-1287, an investigational oral CDK9 inhibitor, for the treatment of Ewing sarcoma.

"We are delighted to have received this designation for TP-1287 which underscores the need for additional treatment options for patients with Ewing sarcoma," said Patricia S. Andrews, Chief Executive Officer and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. "We recognize the unmet need for novel treatments in this disease state and are excited to contribute to the advancement of this research with the goal of helping to improve 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.1 Ewing sarcoma is a rare type of cancer that occurs in bones or in the soft tissue around the bones. The disease occurs when a cell develops changes in its DNA that cause the cell to multiply quickly, resulting in a tumor of abnormal cells that is capable of invading and destroying healthy body tissue. The abnormal cells can break away and metastasize throughout the body. Ewing sarcoma can occur at any age, but it is more likely to occur in children and teenagers.2

"TP-1287 exhibits potent inhibition of intracellular kinases including CDK9. Inhibition of CDK9 leads to downregulation of key antiapoptotic proteins such as MCL-1, which in turn has been shown to inhibit tumor growth in preclinical models of hematologic malignancies and several tumor types,"3-6 detailed Jatin J. Shah, M.D., Chief Medical Officer of Sumitomo Pharma Oncology, Inc.

TP-1287 was also granted Rare Pediatric Disease Designation from the FDA for the treatment of Ewing sarcoma. A rare pediatric disease is one that is serious or life-threatening in which the serious or life-threatening manifestations primarily affect patients from birth to 18 years old.7

TP-1287 is currently being evaluated in a Phase 1, first-in-human study of oral TP-1287 in patients with advanced metastatic or progressive solid tumors who are refractory to, or intolerant of, established therapy known to provide clinical benefit for their condition, which is being conducted in the United States. To learn more about the study and eligibility for enrollment, visit clinicaltrials.gov (NCT03604783).

This is the fourth Orphan Drug Designation Sumitomo Pharma Oncology, Inc. has received in the last year. DSP-5336, the company's proprietary investigational small molecule inhibitor against the binding of menin and mixed-lineage leukemia (MLL) protein, was granted Orphan Drug Designation for the treatment of acute myeloid leukemia (NCT04988555). TP-3654, the company's proprietary investigational oral inhibitor of PIM kinases, was granted Orphan Drug Designation for the treatment of myelofibrosis (NCT04176198). DSP-0390, an investigational emopamil-binding protein (EBP) inhibitor, was also granted Orphan Drug Designation for the treatment of brain cancer (NCT05023551). These designations showcase the strength and diversity of SMP Oncology's pipeline and commitment to oncology research and development.

 

Source:prnewswire.com

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