Oblato, Inc. issued the following announcement on Dec. 10.
Oblato, Inc. (the Company), a wholly owned U.S. subsidiary of the Korean biotech company GtreeBNT Co., Ltd., announced it had official discussions with the FDA on a detailed plan for a phase 1/2 clinical trial to start developing a new treatment for Diffuse Intrinsic Pontine Glioma (DIPG), a rare pediatric disease, using its proprietary drug, OKN-007.
Through this FDA meeting, the Company obtained consent from the FDA on important matters, such as patient population, starting dose, approach for dose escalation, and criteria for evaluating disease response. Importantly, there was in depth discussion on how to actively utilize existing historical patient data instead of requiring a comparative group in consideration of the nature of DIPG, a severe rare pediatric glioma and the need for all patients to receive anti-cancer treatment. The FDA also gave detailed advice on pharmacokinetic assessment and standard of care radiation therapy during the clinical study. The detailed response from the FDA indicates their support and expectations for Oblato which is developing a new drug for the treatment of DIPG.
In particular, the FDA has been supportive of biopharmaceutical companies developing treatments for rare pediatric diseases. Furthermore, new drugs for rare pediatric diseases have been approved by the FDA if the efficacy was fully demonstrated even with a small clinical trial. In 2019, Vyondys 53 (golodirsen), a treatment for DMD (Duchenne muscular dystrophy) developed by Sarepta Therapeutics, was approved after just a single phase 1/2 clinical trial. Another example is Brineura (ceriponase alpha) that was developed by Biomarin and approved in 2017 for the treatment of CLN2/Batten disease after a single phase 1/2 clinical study.
"DIPG's nonclinical studies using OKN-007 have been recognized as supportive to the proposed phase 1/2 trial plan for DIPG and were also fully accepted by the FDA during the meeting. If our treatment shows efficacy in the clinical study for DIPG, we will be able to apply for an NDA after the trial is completed," stated an official from the Company.
The FDA has already granted Oblato the Rare Pediatric Disease Designation for DIPG, and the FDA has officially indicated that Oblato would be eligible for a Rare Pediatric Disease Priority Review Voucher.
About Oblato, Inc.
Oblato, Inc., a wholly-owned subsidiary of the Korean biotech company GtreeBNT, is incorporated in Delaware and has its principal place of business in New Jersey. Since 2016, the company has been developing a new drug, OKN-007, to treat brain cancers, especially, glioblastoma multiforme (GBM) as a rare disease and diffuse intrinsic pontine glioma (DIPG) as a rare pediatric disease. Currently, clinical trials are ongoing to investigate the safety and efficacy of a combination therapy with both OKN-007 and temozolomide for patients with both newly diagnosed and recurrent GBM. Oblato will also plan to conduct a Phase 1/2 trial in 2021 for DIPG.
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