ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease

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by Alice Melao |

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Newborn diagnosis

ABO-202, Abeona Therapeutics‘ investigational candidate for the treatment of infantile Batten disease has received orphan drug status by the European Medicines Agency.

This new status adds to the previously granted orphan drug and rare pediatric disease designation by the U.S. Food and Drug Administration.

These designations are expected to provide regulatory support, financial benefits, and expedite the clinical development and review of ABO-202.

ABO-202 is an adeno-associated virus (AAV) based gene therapy designed to deliver a correct copy of the mutated gene that causes infantile Batten disease. This therapy uses a weakened viral structure to deliver a normal copy of the CLN1 gene to target cells, restoring its normal function.

“This designation is an important step in our CLN1 program as it encourages us to proceed towards clinical development,” Carsten Thiel, PhD, CEO of Abeona Therapeutics, said in a press release. “We remain committed to our vision of developing transformational gene therapies for patients suffering from devastating rare diseases.”

Results of preclinical studies in mouse models of Batten disease revealed that a single dose of ABO-202 at early stages of the disease could significantly increase survival, improve behavior, and reduce motor deficits.

Higher doses of the therapy in a combination approach of intravenous injections and direct delivery into the spinal canal was able to improve treatment response and increase survival by 50 percent.

These results were presented in February during the WORLDSymposium in San Diego, California, in a poster presentation titled “Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis.”

Abeona will provide an update of the preclinical results of ABO-202 during the upcoming 21st Annual Meeting of the American Society for Gene and Cell Therapy in Chicago, Illinois, May 16-19.

“Batten disease, particularly CLN1 disease, is a severely debilitating disorder where patients do not currently have adequate therapeutic options,” Thiel said. “[The] compelling pre-clinical data underscore the viability of ABO-202 as a potential treatment for CLN1 disease in humans.”

Abeona anticipates initiation of human clinical trials to evaluate the safety, tolerability, and effectiveness of ABO-202 during 2018.