RGX-181 is under clinical development by RegenxBio and currently in Phase II for Batten Disease. According to GlobalData, Phase II drugs for Batten Disease have a 50% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how RGX-181’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

RGX-181 overview

RGX-181 is under development for the treatment of late infantile neuronal ceroid lipofuscinosis (LINCL), a form of Batten disease in children. It is administered by intracisternal route. The therapeutic candidate is developed using NAV technology which uses recombinant Adeno-associated virus 9 (rAAV9) vectors for the delivery of new genetic materials to cells.

RegenxBio overview

RegenxBio, formerly ReGenX Biosciences LLC, is a clinical-stage biotechnology company involved in the discovery and development of gene therapy. It is focused on the treatment of various retinal, metabolic, and neurodegenerative diseases. The company develops, commercializes, and licenses recombinant adeno associated virus (AAV) gene therapy. RegenxBio‘s products include AAV plasmids, AAV vector reporter systems, and custom AAV vectors. The company’s gene therapy product candidates deliver genes to cells for addressing genetic defects and enable cells in the body to produce therapeutic proteins or antibodies that act on the disease. RegenxBio employs its proprietary NAV technology platform, a gene delivery technology, to advance its product candidates. RegenxBio is headquartered in Rockville, Maryland, the US.

For a complete picture of RGX-181’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.