Elixirgen Therapeutics And Nippon Shinyaku Enter Into An Option Agreement For The Development And Commercialization Of EXG-7001 For Duchenne Muscular Dystrophy

Elixirgen Therapeutics, Inc., a clinical-stage biotechnology company developing treatments for rare diseases and aging-associated diseases, today announced that it has entered into an option agreement with Nippon Shinyaku Co., Ltd. (Headquarters, Kyoto, Japan; President, Toru Nakai). Under the terms of the agreement, Elixirgen will be responsible for the development of EXG-7001 for the treatment of Duchenne Muscular Dystrophy (DMD) and Nippon Shinyaku may obtain exclusive worldwide rights to commercialize EXG-7001. Nippon Shinyaku will provide the funding for the developmental costs, and Elixirgen will receive an upfront payment and is eligible to receive additional development and sales-based milestone payments if the option were to be exercised. Following the exercise of the option rights by Nippon Shinyaku and regulatory approval in the United States, NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku, will market and commercialize EXG-7001.

“Current approaches for treating DMD focus on delivering or restoring an incomplete dystrophin protein, and there still remains a significant unmet need for a therapy that can successfully deliver a full-length dystrophin protein,” said Aki Ko, Chief Executive Officer of Elixirgen Therapeutics. “By design, EXG-7001 has the potential to deliver the full-length, complete dystrophin protein that is missing in DMD patients, regardless of their genetic mutation. We look forward to combining Elixirgen’s clinical and regulatory excellence with Nippon Shinyaku’s expertise and mission to improve the lives of those living with rare diseases.”

EXG-7001 is a locally administered, full-length dystrophin mRNA therapeutic that is currently in preclinical development for the treatment of DMD.

DMD is a rare, progressive muscular dystrophy characterized by severe muscle wasting and weakness. It is caused by genetic mutations in the DMD gene resulting in the dysfunction or absence of the dystrophin protein which plays an important role in healthy muscle cell function.

About Nippon Shinyaku
Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” Nippon Shinyaku aims to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. For more information, visit https://www.nippon-shinyaku.co.jp/english/.

About Elixirgen Therapeutics, Inc.
Elixirgen Therapeutics, Inc. is a clinical-stage biotechnology company developing treatments for rare diseases and aging-associated diseases using its ZSCAN4 technology and mRNA platform technologies. For more information, visit ElixirgenTx.com.