Batten Disease Gene Therapy Granted Orphan Drug Designation

Abeona Therapeutics receives FDA orphan drug designation for ABO-202 gene therapy program in infantile Batten disease.

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced Monday that the FDA has granted orphan drug designation to its ABO-202 program (AAV-CLN1), an adeno-associated viral (AAV)-based gene therapy for the treatment of infantile Batten disease.

A fatal lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis (INCL), infantile Batten disease is an inherited fatal genetic disease that primarily affects the nervous system in newborns and progresses rapidly.

“This designation builds on our clinical portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the scientific and clinical translation of these products for the severely underserved CLN1 patient population,” stated Timothy J. Miller, Ph.D., president & CEO of Abeona Therapeutics Inc. “The ABO-202 preclinical data from Dr. Steven Gray’s lab support the clinical translation for patients with infantile Batten disease, and provide valuable insight for potentially improving efficacy using a combination of delivery routes for CNS and whole-body benefit to remove the underlying pathology associated with the disease.”

ABO-202, developed with Gray, assistant professor, pediatrics and neurology, UT Southwestern, and the support of The Saoirse Foundation, Taylor’s Tale, Garrett the Grand Batten Fighter, Hayden’s Batten Disease Foundation, and the Batten Disease Support and Research Association, is anticipated to enter clinical trials in 2018.

“ABO-202 is an AAV gene therapy that has shown promising preclinical efficacy in the INCL animal model of disease by extending survival and improving muscle function when administered early in the disease course,” Gray said.

The preclinical data were presented last week at the WORLDSymposium held in San Diego. Key findings included:

CLN1 mice recapitulate the major features of the human disease manifestations;
- The data demonstrate that a single intrathecal (IT) injection of self-complementary adeno-associated virus 9 (scAAV9) encoding the human CLN1 gene to CLN1 mice at 1 week and 1 month (pre-symptomatic) significantly increased their survival, improved behavior and reduced motor deficits.
- Higher IT doses further improved these observations, suggesting that methods increasing CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic INCL mice.
- A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy 50 percent and improved potential treatment options for older animals with advanced disease manifestations.

Miller said that the orphan drug designation helps advance the ABO-202 program and that the company plans to initiate human clinical trials later this year.

(Source: Abeona Therapeutics Inc.)