TSHA-102 is under clinical development by Astellas Pharma and currently in Phase II for Rett Syndrome. According to GlobalData, Phase II drugs for Rett Syndrome have a 50% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how TSHA-102’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

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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.

TSHA-102 overview

TSHA-102 (TGTX-104) is under development for the treatment of rett syndrome. It is a gene replacement therapy and is administered by intrathecal route. The therapeutic candidate comprises of recombinant adeno-associated virus (rAAV) vector encoding with MECP2 gene. It is developed based on adeno associated virus (AAV) discovery platform and miRNA-Responsive Auto-Regulatory Element (miRARE) platform.

Astellas Pharma overview

Astellas Pharma (Astellas) discovers, develops, manufactures, and commercializes a wide range of pharmaceuticals. The company’s product portfolio comprises marketed products for the treatment of a wide range of urological diseases, cancer, nephrology, infectious diseases, and metabolic diseases, immunodeficiency, and transplantation. The company strives to develop new drugs to address high unmet medical needs with focus on biology and modality. Its pipeline consists of drug candidates for the treatment of urothelial cancer; gastric cancer; gastroesophageal junction cancer; anemia associated with chronic kidney disease (CKD) in dialysis; and acute myeloid leukemia (AML), among others. It operates in the Americas, Australia, Europe, and Asia and Oceania through a network of subsidiaries and affiliates. Astellas is headquartered in Chuo-Ku, Tokyo, Japan.

For a complete picture of TSHA-102’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 16 July 2024

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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 clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.