On September 23, 2019 Mateon Therapeutics Inc. (OTCQB:MATN) reported that the US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for OT101/Trabedersen for the treatment of diffuse intrinsic pontine glioma (DIPG) as a drug for a "rare pediatric disease (Press release, Mateon Therapeutics, SEP 23, 2019, View Source [SID1234539855])."
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"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," said Dr. Vuong Trieu, Chief Executive Officer of Mateon. "We will continue to build on our pipeline of therapeutics for rare and orphan diseases, including cancers."
Brainstem tumors comprise approximately 10-15 percent of all pediatric brain tumors and DIPG is the most common brainstem tumor and the second most common malignant brain tumor of childhood. DIPG, is an orphan disease with a low survival rate and no established or effective standard of care. Despite numerous clinical trials of chemotherapeutic agents, immuno-oncology drugs and specific targeted therapies, no significant progress has been made in the treatment of DIPG and the prognosis remains dismal, with a mean OS of 9–12 months from the time of diagnosis, a median survival time of approximately 10 months, and a two-year OS rate of less than 10 percent. Five-year survival is less than 3 percent, and many long-term survivors have evidence of moderate or severe cognitive impairment, likely as a consequence of radiation therapy. Chemotherapy does not have an established role in the management of patients with DIPG. Furthermore, there is no standard treatment for progressive DIPG after the failure of radiation therapy and no salvage regimen has been shown to extend survival. Therefore, there is an urgent need for therapeutic innovations for treatment of DIPG, as reflected by multiple treatment modalities being evaluated in early neuro-oncology clinical trials.
OT101, a first-in-class RNA therapeutic, is designed to abrogate the immunosuppressive actions of TGF-beta 2. In a completed Phase 2 clinical study, OT-101 exhibited clinically meaningful single-agent activity and induced durable complete and partial responses in recurrent and refractory adult high-grade glioma patients, including adults with GBM. Further development of OT-101 may offer renewed hope for salvage therapy of pediatric DIPG patients who have this rare and fatal disease.
The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application, and may be sold or transferred. In August 2019, AstraZeneca has reportedly paid approximately $95 million to buy a priority review voucher from Swedish Orphan Biovitrum (Sobi) (View Source). Likewise, Biohaven Pharmaceutical Holding Company Ltd. reportedly paid approximately $105 million for a priority review voucher in March 2019 (View Source).
Dr. Fatih Uckun, the Chief Medical Officer of Mateon is scheduled to give both a talk and a poster presentation on the clinical safety and anti-brain tumor efficacy of OT101 at the 24th Annual Meeting of the Society for NeuroOncology in Phoenix, Arizona in November 2019.
"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," Dr. Uckun explained. "Our recent bioinformatics 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. These in silico target validation data recently accepted for publication in a peer-reviewed medical journal extend the promising clinical data on the therapeutic activity of OT101 in adults and young adults and further demonstrate the potential of 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."
Last month, Mateon announced that it had entered into a definitive agreement for the potential acquisition of PointR Data, Inc. (PointR), a privately-held developer of high-performance cluster-computing technologies for artificial intelligence. The proposed transaction would create a publicly-traded AI driven immuno-oncology company.
"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, PointR’s Chief Executive Officer.
"AI-based cognitive technologies have the potential to streamline our clinical development strategy for the portfolio drug candidates by amplifying our knowledge and understanding of the target pediatric cancers, their biology as well as structural and pharmacologic characteristics of the lead compounds", said Dr. Fatih Uckun, MD, PhD, the Chief Medical Officer of Mateon. "Furthermore, the combined use of AI and the Blockchain technology supported by the PointR AI computing platform has a very high impact potential for a better cancer care and especially patient-tailored cancer treatments for pediatric and young adult cancer patient populations."