Batten-1

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.

About juvenile Batten disease

Juvenile Batten disease or Spielmeyer-Vogt disease, also known as CLN3 disease, is a genetic lysosomal disease affecting mainly the nervous system. Batten disease is a rare genetic disease affecting the metabolism of the central nervous system and systematically fatal, characterized by the pathological accumulation of glycosphingolipids in the brain. It affects about 1,500 patients in Europe and the United States.

Within lysosomal diseases, juvenile Batten disease belongs to a larger group of diseases called neuronal ceroid lipofuscinoses (NLC). More than 400 mutations in 13 genes have been associated with NLC variants, all of which show impairment of lysosomal function and are similar in clinical presentation. However, they differ from each other clinically in the age of onset of the first symptoms as well as in the speed of disease progression, these clinical differences being explained by the fact that each mutation will affect a different lysosomal protein. Among NLCs, only NLC2 currently benefits from a treatment, Brineura® from the biotechnology company Biomarin, this drug having been approved in 2017.

BBDF Foundation

Charlotte and Craig Benson established the Beyond Batten Disease Foundation in August 2008 after their five-year-old daughter Christiane was diagnosed with Juvenile Batten Disease. Together with other families affected by Batten disease and supporters who share their hope and determination, they work tirelessly to create a better future for Christiane and all children with Batten disease.

BBDF has invested more than $35 million raised through direct philanthropic giving, partnerships and collaborations with other organizations and institutions, and has invested in more than 40 separate research projects.

It chose to partner with Theranexus because of its experience and expertise in neurodegenerative and neurological lysosomal diseases in the field of preclinical, regulatory and clinical development.

Theranexus is pursuing its development program in juvenile Batten disease in partnership with the BBDF Foundation, for which it holds an exclusive worldwide license for the development and commercial exploitation of its Batten-1 asset.