BioTuesdays

Mateon gets FDA rare pediatric disease designation for melanoma drug candidate

Mateon Therapeutics

Mateon Therapeutics’ (NASDAQ:MATN) CA4P received FDA pediatric disease designation for the treatment of stage 2b-to-4 melanoma due to genetic mutations that disproportionately affect pediatric patients.

As a single agent, CA4P resulted in stable disease in two of five melanoma patients who failed standard therapies. In combination with carboplatin and paclitaxel, CA4P resulted in a partial response in one of six melanoma patients who failed first-line therapy. 

Mateon expects that the combination of CA4P with ipilimumab, or other immunotherapies, would result in improved tumor control, compared to those immunotherapies alone.

“This builds on our previous rare pediatric disease designation for our lead drug candidate, OT-101, for pediatric brain cancer patients,” Dr. Vuong Trieu, Mateo’s CEO, said in a statement.

“We are looking to [continue] to refocus the company efforts in the U.S. [on] rare pediatric indications, while expanding our footprint to China,” he added.