Amicus enters gene therapy field with purchase of Celenex's portfolio focused on Batten disease

Amicus Therapeutics announced Thursday the acquisition of Celenex, gaining global rights to the latter's licenses for 10 gene therapy programmes focused on Batten disease and other neurologic lysosomal storage disorders. Under the deal, Amicus will pay Celenex $100 million upfront in cash to acquire the assets, along with up to $15 million in development milestones and $262 million linked to regulatory filings and approval milestones across multiple programmes, as well as $75 million in milestones on tiered sales.

The gene therapy programmes were developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. Amicus noted that the lead programmes in CLN6, CLN3 and CLN8 Batten disease are potential first-to-market curative therapies, with CEO John Crowley commenting "I cannot think of a better foundation for Amicus' entry into gene therapies."  

Amicus explained that all of the acquired clinical and preclinical programmes, which also include those for Niemann Pick C, Wolman disease and Tay Sachs, utilise intrathecal delivery via the same AAV vector approach successfully applied in clinical trials for other rare central nervous system (CNS) disorders. The company added that the approach and technology represent a clinically validated gene delivery system for CNS disorders.  

The news comes after Amicus received accelerated approval in August from the FDA for Galafold (migalastat) for certain patients with Fabry disease, later pricing the therapy at $315 000 per year. For related analysis on the drugmaker, see ViewPoints: Does Amicus have another rabbit up its sleeve?

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