Austin, Texas, March 2, 2021

Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.

CLN3, one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), is a rare, fatal, inherited, neurodegenerative disease for which there is no treatment. Since it was founded in 2008, BBDF has spearheaded the development of BBDF-101. Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe (GRAS) by the US Federal Drug Administration (FDA). Although trehalose is commonly used as a mild sweetener, laboratory studies demonstrate trehalose’s ability to clear the pathologic accumulation of cellular waste found in lysosomal storage disorders and some forms of adult neurodegenerative disease. These data prompted BBDF to provide the financing to support the development of BBDF-101 as a potential treatment of CLN3 disease.

Preclinical studies in CLN3 patient cells and animal models demonstrate trehalose-mediated clearance of cellular waste and the inhibition of disease progression which was enhanced by the addition of miglustat. These data show that miglustat can ameliorate chronic inflammation, prevent the accumulation of harmful gangliosides, and inhibit a competing enzyme (trehalase) thereby increasing trehalose exposure. The potential efficacy of BBDF-101 led to a December 12, 2019 agreement with Theranexus (EPA: ALTHX), a biopharmaceutical company developing drug candidates to treat neurological disorders, granting Theranexus a worldwide exclusive license to develop and to commercialize BBDF-101 for CLN3 disease.

BBDF and Theranexus plan to initiate a Phase I-III study with BBDF-101 for patients with CLN3 disease in 2021 at Texas Children’s Hospital (TCH) in Houston. The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will serve as the assessment site. BBDF has also recruited an Advisory Committee of leading experts on juvenile Batten disease including Jonathan Mink, MD and Erika Augustine, MD at the University of Rochester; Angela Schulz, MD and Miriam Nickel, MD at the University Medical Center Hamburg-Eppendorf; Forbes Porter, MD, PhD, and An Dang Do, MD, PhD, NICHD.

“This agreement enabled BBDF to save approximately $42M, the cost of generic miglustat, had we needed to buy commercially available miglustat to conduct our development program”, said BBDF Founder and Chairman Craig Benson. “We are incredibly grateful to Janssen for its support for the development of BBDF-101 for Batten Disease. We believe this collaboration will make it possible for us to move towards a clinical trial in an expedited and cost-effective way; and time is of the essence for children with this fatal condition.”

About Beyond Batten Disease Foundation Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $35 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, coordinated strategy that incorporates independent scientific resources and collaborates with related organizations to drive research in CLN3 disease. More information can be found at

About Zavesca (miglustat) Zavesca (miglustat), an orally active inhibitor of glucosylceramide synthase, is used as monotherapy in adult patients with mild to moderate type I Gaucher disease (GD1) for whom enzyme replacement therapy is not a therapeutic option. Zavesca is commercially available for the treatment of GD1 in 47 markets, including the US and the EU (since 2003). In the EU, Zavesca is also indicated for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare neurodegenerative genetic disorder. Zavesca is commercially available for the treatment of NP-C in 46 markets, including the EU (since 2009) and Japan (since 2012, marketed as Brazaves®).