Amicus Therapeutics Announces Phase 1/2 Study of Gene Therapy for CLN3 Batten Disease
Second Gene Therapy to
Initial Safety is Encouraging with No Serious Adverse Events at One Month
This first in human study of an investigational gene therapy in CLN3 Batten disease, a life-threatening genetic neurologic disorder that typically begins in early childhood and results in premature death, is currently being conducted at Nationwide Children’s Hospital (
“We are pleased to announce that the first child has been dosed in the Phase 1/2 study for CLN3 Batten disease,” said
The Phase 1/2 study is enrolling children aged 3 to 10 years with a confirmed diagnosis of CLN3 Batten disease in two sequential intrathecal dose groups: a one-time low-dose of AAV9-CLN3 (Group 1) and a one-time high-dose of AAV9-CLN3 (Group 2) after evaluation of Group 1 participants. Both groups will participate in the current study for a period of three years. Amicus plans to present clinical data from this study, including interim data, at future scientific congresses and other relevant venues. The primary outcome measures are safety and efficacy as determined using the physical disability subscale of the Unified Batten Disease Rating Scale (UBDRS) in CLN3 Batten disease. More information is available at www.clinicaltrials.gov: NCT03770572.
CLN3 Batten disease, the most common form of NCL results from a mutation in the CLN3 gene which primarily affects the nervous system. Children with this condition develop vision impairment, intellectual disability, progressive loss of motor function, speech difficulties, and seizures which worsen over time.
About Batten Disease
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. 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. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.
Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding, and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment, and seizures. Patients typically experience progressive loss of motor function and eventually those affected become wheelchair-bound, are then bedridden, and die prematurely.
This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to preliminary safety information from a Phase 1/2 study to investigate intrathecal administration of AAV9-CLN3 for the treatment of CLN3 Batten disease. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary information based on a single patient and reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented from this study or the CLN6 study, that the Company will be not able to demonstrate the safety and efficacy of intrathecal administration of AAV9-CLN3, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, obtain any desireable third party licenses or successfully commercialize AAV9-CLN3. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended
Vice President, Investor Relations & Corporate Communications
Source: Amicus Therapeutics, Inc.