FDA Grants Orphan Drug Designation To RGX-181 Gene Therapy For The Treatment Of CLN2 Form Of Batten Disease

ROCKVILLE, Md., Nov. 14, 2018 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

"We believe RGX-181 has the potential to correct the underlying genetic condition, halt progression and address many of the serious and life-threatening symptoms of CLN2 disease," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "CLN2 disease is an extremely debilitating disease in children with no cure and limited treatment options. Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop RGX-181 as a potential one-time treatment for children with CLN2 disease."

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

RGX-181 is designed to use REGENXBIO's NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS), which may induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease. REGENXBIO plans to submit an Investigational New Drug (IND) application for RGX-181 to the FDA in 2019 to enable initiation of a first-in-human clinical trial.

About RGX-181

RGX-181 is being developed as a novel, one-time treatment for CLN2 disease utilizing the NAV AAV9 vector to deliver the gene encoding for TPP1, the enzyme deficient in children with CLN2 disease. Following a single administration given by intracisternal injection, RGX-181 treatment is designed to modify cells in the CNS, thereby providing a durable source of TPP1 and allowing for long-term correction of cells throughout the CNS. In an animal model for CLN2 disease, treatment with RGX-181 has been shown to restore TPP1 activity to levels greater than those in non-affected animals, and to improve neurobehavioral function and survival. The extent of CNS correction observed in animal studies suggests that RGX-181 has the potential to be an important and suitable therapeutic option for patients with CLN2 disease.

About CLN2 Disease

Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a form of Batten disease, is a rare, pediatric-onset, autosomal recessive, neurodegenerative lysosomal storage disorder caused by mutations in the TPP1 gene. Mutations in the TPP1 gene, and subsequent deficiency in TPP1 enzymatic activity, result in lysosomal accumulation of storage material and degeneration of tissues including the brain and retina. CLN2 disease is characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, loss of vision and blindness, and premature death by mid-childhood. Onset of symptoms is generally between two to four years of age with initial features of recurrent seizures (epilepsy), language delay, and difficulty coordinating movements (ataxia). There is currently no cure for CLN2 disease. Current treatment options include palliative care or enzyme replacement therapy, wherein recombinant TPP1 is administered into the lateral ventricles via a permanently implanted device on a biweekly basis.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

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SOURCE REGENXBIO Inc.

Posted: November 2018

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