Toray Industries, Inc. (headquarters: Chuo-ku, Tokyo; President: Akihiro Nikkaku; hereinafter referred to as “Toray”) announced that BONAC Corporation (headquarters: Kurume-shi, Fukuoka Prefecture; President: Hirotake Hayashi; hereinafter referred to as “BONAC”) has received Orphan Drug Designation to TRK-250*2, a nucleic acid medicine originated by BONAC, for the treatment of Idiopathic Pulmonary Fibrosis (IPF) *3, from the U.S. Food and Drug Administration (FDA). Toray is currently conducting a Phase 1 clinical trial with IPF patients in the U.S.
The orphan drug designation enables us to receive benefits such as 7-year marketing exclusivity, tax credit for clinical trials costs and waiver of a partial marketing application user fees in the US.
IPF is disorder with a poor prognosis and an unpredictable clinical course, in which fibrosis of intestinal pneumonia progresses irreversibly. Thus, the development of a novel drug with new mechanisms is expected to broaden the treatment options in clinical practice.
TRK-250 is a nucleic acid medicine that inhibits the progression of pulmonary fibrosis by selectively suppressing the expression of transforming growth factor-beta 1 (TGF-β1) protein, a key growth factor involved in lung fibrosis, at the gene expression level. One of the features of the agent is that it is a single strand long-chain nucleic acid with a unique molecular structure employing BONAC’s proprietary nucleic acid platform, and it is expected to be a new nucleic acid medicine that has overcome the issue of stability. Moreover, it is in an aerosol form that can be administered directly to the lung, which is expected to carry the agent efficiently to the target organ.
With TRK-250’s orphan status in hand, Toray and Bonac look forward to accelerating the development of TRK-250, and aim to launch the agent in the late 2020s to bring it to IPF patients even sooner.
*1: Orphan Drug Designation:
Orphan Drug Designation (ODD) is the program for accelerate development of drugs including biologicals that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease. The condition of designation and incentives by the program is different from country to country and drugs would qualify for orphan status if they were intended to treat a disease affecting less than 200,000 American citizens in the US.
*2: TRK-250(Toray’s development code)
Toray signed a licensing agreement in December 2015 with BONAC, who is the originator of the agent, and Toray has acquired exclusive rights of development, sales, and manufacturing (excluding those of the active pharmaceutical ingredient) for the agent for IPF treatment in Japan. Toray and BONAC have been collaborating in problem-solving and project management of non-clinical studies and development of an inhaler-based administration method, and Toray has initiated Phase 1 clinical trial for IPF patients in the U.S
*3: Idiopathic pulmonary fibrosis (IPF):
A disease that is considered to be the most difficult to treat among idiopathic interstitial pneumonia types. It is a chronic intractable disease with an associated mean survival time of 3 to 5 years after diagnosis. Interstitial pneumonia is a disease in which inflammation and injuries due to various causes on the wall of alveoli located at the end of lungs induce fibrosis with the accumulation of collagen fibers, leading to difficulty in breathing. Interstitial pneumonia, whose cause remains unknown, is called idiopathic interstitial pneumonia. IPF occurs in 10 out of 100,000 individuals, with an estimated to be more than 10,000 patients in Japan.
