Beyond Batten Disease Foundation (BBDF) has partnered with Actelion Pharmaceuticals, a Janssen company, 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 programme, and right of reference to data within the Zavesca new drug application to support the development of BBDF-101.
CLN3, one of a group of disorders known as neuronal ceroid lipofuscinoses, is a rare, fatal, inherited, neurodegenerative disease for which there is no treatment. BBDF has spearheaded the development of BBDF-101 since the foundation was founded in 2008.
Trehalose is a naturally occurring disaccharide which is Generally Regarded as Safe by the FDA. Although it 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 findings 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. The data show that miglustat can ameliorate chronic inflammation, prevent the accumulation of harmful gangliosides, and inhibit a competing enzyme thereby increasing trehalose exposure.
The potential efficacy of BBDF-101 led to a December 2019 agreement with Theranexus, a biopharmaceutical company developing drug candidates to treat neurological disorders, granting the company a worldwide exclusive licence to develop and to commercialise 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 in Houston. The Eunice Kennedy Shriver National Institute of Child Health and Human Development will serve as the assessment site, and the foundation has also recruited an advisory committee of leading experts on juvenile Batten disease.
BBDF Founder and Chairman Craig Benson said: “This agreement enabled BBDF to save approximately $42 million, the cost of generic miglustat, had we needed to buy commercially available miglustat to conduct our development programme.
“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.”
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