Batten-1 is a new proprietary and exclusive pharmaceutical product whose active ingredient is miglustat. The mechanism of action of this active ingredient blocks the accumulation of glycosphingolipids and neuroinflammation to prevent brain cell death.
A first Phase 1/2 clinical study has been initiated in Batten disease patients aged of 17 and older. It aims to study the safety and pharmacokinetics of miglustat in our target indication. Recent discussions with the U.S. Food and Drug Administration (FDA) have confirmed that a single pivotal Phase 2/3 study can lead to registration of Batten-1 as a treatment for Batten disease.
This study will be launched in the first half of 2023 on approximately 60 patients in the United States and Europe. A successful completion of this study could lead to rapid registration of the product in the United States and Europe through accelerated procedures, opening up a potential market for Theranexus estimated at $500 million in peak annual sales.