RARE Daily

Amicus Discontinues CLN6 Batten Disease Gene Therapy Program

January 11, 2022

In a corporate update, Amicus Therapeutics said it is discontinuing its lead CLN6 Batten disease gene therapy program following review of long-term extension study data.

“It was recently determined that any initial stabilization of disease progression at the two-year time point was not maintained through the long-term extension study,” the company noted.

Instead, the company will advance its CLN3 Batten disease program, which features a higher dose, different promoter, and an intracisternal magna route of delivery “pending further phase 1/2 clinical data and preclinical data expected in 2022” before projecting a timeline for any pivotal study.

Amicus also plans to further analyze and share the phase 1/2 data with key stakeholders in the CLN6 Batten disease community and work with the community to support continued research efforts to find better treatments and cures.

Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs that are characterized by progressive mental and motor deterioration, seizures, and early death. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell’s ability to recycle certain molecules. The disease has several forms, each form caused by a mutation in a different gene, with similar features and symptoms but vary in severity and age of onset. Most forms of Batten disease/NCLs usually begin during childhood with symptoms appearing between the ages of four and ten years. Rapid vision loss resulting in total blindness is usually the first symptom observed followed by seizures that typically present between ages five and 18 years. Life expectancy generally ranges from the late teens to the 30s. Although a cure has not been identified for Batten disease, treatment for the symptoms is available.

Amicus was pinning its hopes on the CLN6 program when it arranged in late September 2021 to go public through at reverse merger with a Perceptive Advisors SPAC.

Amicus lead program is focused on Pompe disease, for which the company has a PDUFA action date of May 29, 2022. But the news on Batten disease plus missed sales projections for its marketed Fabry disease drug Galafold, rattled investors, sending shares down 17 percent by the end of the day.  

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