Astellas Pharma Inc (OTC:ALPMY) (OTC:ALPMF) has elected not to exercise its option to obtain an exclusive license to TSHA-120 under the Option Agreement between Astellas and Taysha Gene Therapies Inc (NASDAQ:TSHA).

In October 2022, Taysha gave Astellas an option to license its gene therapy candidate TSHA-120 in the neurodegenerative disorder giant axonal neuropathy (GAN) as part of a two-asset agreement. 

Astellas paid $20 million upfront, invested $30 million in Taysha to secure the two options, and agreed to decide on the GAN program after seeing the minutes of the end-of-phase 2 meeting with the FDA.

In 2022, Taysha submitted and reviewed with the FDA in a Type B end-of-Phase 2 meeting a subset of available evidence from a Phase 1/2 clinical trial investigating TSHA-120 for GAN. 

FDA feedback included the need to address the heterogeneity of disease progression in GAN and the effort-dependent nature of MFM32 as a primary endpoint in an unblinded study. 

To further discuss a potential regulatory path forward for TSHA-120, Taysha submitted a new comprehensive analysis to the FDA in June 2023.

FDA Type C meeting feedback indicated that the FDA recommends a randomized, double-blind, placebo-controlled trial as the optimal path to demonstrate efficacy in TSHA-120. 

The FDA also provided a potential path for a single-arm trial with an external control group matched with to-be-treated patients by multiple prognostic factors and recommended longer-term follow-up to account for potential bias.

Taysha will pursue external strategic options for the TSHA-120 program to potentially enable further program development.

Price Action: TSHA shares are down 6.13% at $3.06 on the last check Wednesday.