Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment

Ewing sarcoma

Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, said the U.S. Food and Drug Administration (FDA) has 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.”

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.

“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,” said Jatin J. Shah, chief medical officer at Sumitomo Pharma Oncology.

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.

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 U.S.

About Sumitomo Pharma Oncology’s TP-1287

TP-1287 is an investigational oral phosphate prodrug of the CDK9 inhibitor alvocidib. TP-1287 is hydrolyzed enzymatically to yield alvocidib. Alvocidib binds at the ATP-binding site of CDK9, stopping phosphorylation by CDK9. 

This binding prevents productive transcription and causes reduction of messenger RNA (mRNA) in genes such as c-MYC and MCL-1.

Downregulation of c-MYC and MCL-1 transcription leads to apoptosis in a variety of tumor cells. 

Spanish Ewing sarcoma research

Ewing sarcoma is caused by a single oncogene that results from the fusion of two genes. Although a variety of genes may be involved, EWSR1 and FLI1 and the resulting cancer-driving oncogene, EWS-FLI, are found to be responsible in the majority of patients. 

Prompted by the need for a genetically tractable model that could be used to study the disease, researchers led by Cayetano González, ICREA research professor at IRB Barcelona in Spain, and Jaume Mora, scientific director at the SJD Pediatric Cancer Center Barcelona (PCCB), recently engineered Drosophila (a genus of small flies) transgenic strains that express a mutant variant of the human oncogene called EWS-FLIFS. 

They found that expression of the human EWS-FLIFS protein in certain types of Drosophila cells triggers the same oncogenic pathways known to account for EWS-FLI oncogenic activity in human patients.

The scientists hope their studies may result in compounds that could serve as lead molecules for the development of therapeutic drugs against the disease.

Explore other topics: CancerFDARegulatory approval

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