Actelion to Help Develop BBDF-101 for Juvenile Batten Disease

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease.

Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of BBDF-101, called miglustat, for clinical trials and an extended access program for the investigational medication.

“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,” Craig Benson, founder and chairman of BBDF, said in a press release.

Juvenile Batten disease (also called CLN3 disease) is caused by a mutation in the gene CLN3, disrupting the body’s ability to get rid of certain waste products. This leads to an accumulation of waste products, most notably a fatty substance called lipofuscin, within cells, which has toxic effects.

BBDF-101 contains a proprietary combination of miglustat and trehalose.

Trehalose is a sugar molecule that is used in some artificial sweeteners and generally regarded as safe by the U.S. Food and Drug Administration (FDA). Preclinical data have suggested that trehalose can promote the disposal of cellular waste products, according to BBDF.

Preclinical data also indicate that the waste product-disposing effects of trehalose are enhanced by the addition of miglustat, leading to inhibited disease progression and lessened inflammation in preclinical models. In the U.S. and elsewhere, miglustat is currently marketed by Actelion under the brand name Zavesca as a treatment for type 1 Gaucher disease, which is also caused by a buildup of certain cellular products.

“We are incredibly grateful to Janssen for its support for the development of BBDF-101 for Batten Disease,” Benson said. “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.”

BBDF and Theranexus, which are jointly developing BBDF-101, are planning to start a Phase 2/3 clinical trial testing the investigational medication in children with CLN3 disease at Children’s Hospital in Houston, Texas, this year. BBDF also has recruited a committee of experts to serve as advisors for the clinical trial.

The FDA gave a favorable opinion of the continued preclinical development plan for BBDF-101 late last year. It has also granted the investigational medication orphan and rare pediatric disease designations.