Taysha drops lead gene therapy following FDA feedback
20 Sep 2023
Gene Therapy
Dive Brief:
Taysha Gene Therapies is giving up on an experimental treatment for a rare neurodegenerative disease due to research recommendations from the Food and Drug Administration that the company believes are too difficult.
In a meeting with the FDA last year, Taysha went over some of the data from a Phase 1/2 trial of its treatment, known as TSHA-120. The agency gave feedback to Taysha, which later submitted more data. The two parties then met again this year to discuss what evidence would need to be gathered to support an approval application. Feedback from that meeting, however, suggested the path forward posed too many hurdles, Taysha said Tuesday.
“We have made the decision to discontinue further development of the program due to challenges related to the feasibility of the study designs,” CEO Sean Nolan said in a statement, referring to the additional research requirements the FDA proposed. At the same time, Taysha said its partner Astellas Pharma has opted not to exercise an option for a license to the therapy, which was being studied in patients with giant axonal neuropathy.
Dive Insight:
Taysha is now retrenching and focusing on another experimental drug, TSHA-102, for a rare neurological disorder called Rett Syndrome. The company now expects to reduce operating expenses enough to extend its cash runway into the fourth quarter of 2025.
While TSHA-120 was Taysha’s most advanced program, investors are most focused on the Rett Syndrome therapy, Cantor Fitzgerald analyst Kristen Kluska wrote in a note to clients. The company’s shares, which had dropped below $1, began climbing in August after the release of early results for TSHA-102 and a private placement financing of $150 million.
Taysha’s stock fell 6% to just over $3 in early trading Wednesday.
Astellas also has an option to license the Rett Syndrome therapy. The Japan-based drugmaker acquired both options when it paid $50 million last year to take a 15% ownership stake in Taysha. “We remain focused on the needs of patients impacted by devastating diseases and look forward to continuing our relationship with Taysha,” Richard Wilson, senior vice president at Astellas, said in the statement Tuesday.
Taysha debuted in April 2020 after spinning out of the University of Texas-Southwestern’s medical school. The company had ambitious plans to advance 15 projects; but after two years, it cut 35% of its staff and narrowed its research focus.
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