Investing.com -- Shares of Taysha Gene Therapies (NASDAQ: TSHA ) soared 19% today after the company announced pivotal trial design details for TSHA-102 in Rett Syndrome, which were enabled by IRSF natural history data and positive clinical data from Part A of the REVEAL trial. The FDA has given written alignment for a single-arm, open-label pivotal trial with primary endpoints focused on developmental milestone gains in patients aged six years and older, who will serve as their own control. The trial is expected to begin in the third quarter of 2025.

The Dallas-based biotech firm reported that all patients aged 6-21 years in the REVEAL Part A trial achieved at least one developmental milestone following treatment with TSHA-102. This is particularly notable given the near-zero likelihood of such improvements without treatment, based on the natural history data. The high dose of TSHA-102 consistently outperformed the low dose, with no serious adverse events or dose-limiting toxicities reported.

Citizens JMP analyst Silvan Tuerkcan responded positively to these developments, raising the price target on Taysha to $6.00 from $5.00 and maintaining a Market Outperform rating. Tuerkcan highlighted the objectivity of the endpoint and the FDA alignment as key positives. "The current response rate is 100%, with 2.2 milestones being met per patient vs. the 1+ required to qualify as responder," Tuerkcan stated. "This leaves a significant margin for Part B."

In addition to the trial updates, Taysha also announced the commencement of an underwritten public offering of its common stock and pre-funded warrants to purchase common stock. The company is advancing AAV-based gene therapies for severe monogenic diseases of the CNS, with TSHA-102 being their lead candidate for Rett Syndrome, a severe neurological disorder.

As Taysha prepares to submit the pivotal Part B trial protocol and statistical analysis plan as an amendment to the IND application within the current quarter, investor optimism is reflected in the stock’s significant upswing. With the FDA’s advice to submit the trial protocol and SAP as an amendment, eliminating the need for a formal end-of-phase meeting, the company is positioned to expedite the trial initiation process.

The positive clinical data and FDA alignment mark a significant milestone for Taysha as it moves closer to potentially delivering TSHA-102 to patients with Rett Syndrome, a condition with a high unmet medical need. With pivotal trial site activation and trial initiation activities anticipated in the third quarter of 2025, Taysha is poised to advance its gene therapy pipeline.

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