Maeton’s OT101 Gets Rare Pediatric Disease Designation for Childhood Brain Tumor

The U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to Mateon‘s OT101 as a therapy for diffuse intrinsic pontine glioma (DIPG), a difficult-to-treat brain tumor that affects children.

The designation is designed to accelerate the development of therapies that can improve the management of serious or life-threatening conditions affecting less than 200,000 people in the U.S. and that mainly manifest in children.

“We are excited about this Rare Pediatric Disease designation for our lead anti-brain tumor drug candidate for pediatric DIPG patients who are in urgent need for therapeutic innovations,” Vuong Trieu, CEO of Mateon, said in a press release.

“We will continue to build on our pipeline of therapeutics for rare and orphan diseases, including cancers,” he said.

Gliomas are a type of brain tumor characterized by a low immune response near the tumor site. OT101 is an immuno-oncology treatment designed to inhibit a protein called transforming growth factor-beta 2 (TGFB2), which inactivates immune cells. By blocking TGFB2 action, OT101 restores the natural immune response against tumor cells and helps the body fight cancer. The therapy also was granted orphan drug designation by the FDA.

A completed Phase 2 trial showed OT101 induced durable responses in adults with recurrent and refractory gliomas. Fatih Uckun, Maeton’s chief medical officer, will present results regarding the safety and anti-tumor efficacy of OT101 at the 24^th Annual Meeting of the Society for NeuroOncology, to be held Nov. 20-24 in Phoenix.

“The durable objective responses achieved in adult patients with recurrent/refractory high-grade gliomas after treatment with our lead anti-TGF beta2 compound OT-101 contribute to our optimism that new treatment strategies leveraging OT101 may favorably change the therapeutic landscape for difficult-to-treat brain tumors with a very poor prognosis,” Uckun said.

“Our recent bioinformatics [based on computer predictions] research has revealed that the TGF beta2 gene product, which is the molecular target for OT101, may serve as a target for immunotherapy in pediatric high-grade gliomas, especially DIPG.” This establishes OT101 “as a promising drug candidate in the treatment of pediatric DIPG, an orphan disease with a low survival rate and no established or effective standard of care.”

Mateon announced last month that it had reached an agreement to acquire PointR Data, a company that develops artificial intelligence (AI) and machine learning technologies for the pharmaceutical and healthcare industries. The acquisition would allow Mateon to develop AI-driven immunotherapies.

“We are working toward integrating artificial intelligence and drug development capabilities under one roof to create a pipeline of therapeutics for the niche market of rare pediatric diseases often ignored by large pharma,” said Saran Saund, CEO at PointR.