PRINCETON, N.J., Dec. 10, 2020 /PRNewswire/ — 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.
지트리비앤티(28,950 -2.20%)는 미국 식품의약국(FDA)과 ‘OKN-007’을 산재적 내재성 뇌교종(DIPG)의 희귀소아질환 치료제로 개발하기 위해 임상 1·2상에 대한 협의를 가졌다고 8일 밝혔다.
지트리비앤티는 이번 논의을 통해 FDA로부터 임상의 환자군 선정, 용량 결정, 종양 진행 평가 계획 등 중요 사항에 대해 동의를 얻었다는 설명이다. 소아 희귀질환이란 점을 고려해 비교군을 실제 임상 시에 모집하는 대신, 기존 환자 데이터를 활용하는 방법에 대해서도 논의가 이뤄졌다. FDA는 이번 협의에서 약동력학 평가와 표준 방사선 치료법 등의 세세한 부분까지 조언했다고 회사 측은 전했다. 이는 중증 소아 희귀질환인 DIPG 치료제에 대한 기대를 표명한 것이란 해석이다.
FDA는 희귀 또는 중증 질환의 치료제를 개발하는 기업에 다양한 혜택을 주고 있다. 소아 희귀질환의 경우는 효과가 충분히 입증될 경우 빠른 시간 내 판매를 허가한 사례도 있다. 2019년 사렙타 테라퓨틱스의 뒤센형 근이영양증 치료제인 ‘Vyondys53’은 임상 1·2상만으로 신약 허가를 받았다. 2017년 바이오마린의 바텐병 치료제인 브리뉴라는 임상 1·2상의 단계에서 허가받았다.