ATGTX-502 is under clinical development by Amicus Therapeutics 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 ATGTX-502’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.

ATGTX-502 overview

ATGTX-502 is under investigation for the treatment of Batten disease (neuronal ceroid lipofuscinosis). It is administered through the intrathecal route and comprises of non-replicating recombinant self-complementary adeno-associated virus vector serotype 9 (AAV9) containing the human CLN3 gene. The therapeutic candidate acts by targeting CLN3 (ceroid-lipofuscinosis neuronal 3).

Amicus Therapeutics overview

Amicus Therapeutics is a biotechnology company, which focuses on the research, development and commercialization of small molecule drugs and orally administered drugs known as pharmacological chaperones for the treatment of rare and orphan diseases. The company’s lead product, migalastat HCl, a small molecule, is indicated for the treatment of Fabry disease. The other products in the pipeline include ATB200/AT2221 for the treatment of Pompe disease, Fabry disease, and other lysosomal storage disorders; migalastat for other conditions; and enzyme replacement therapies for other rare diseases. It has presence in the US, the UK, Ireland, France, the Netherlands, Germany, Spain, Italy and Japan. Amicus Therapeutics is headquartered in Philadelphia, Philadelphia, the US.

For a complete picture of ATGTX-502’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.