IRSF Day 1: TSHA Maintains Edge On Response Rate & Potential Regulatory Timing

研报英文原文证据摘录

IRSF Day 1: TSHA Maintains Edge On Response Rate & Potential Regulatory Timing

C O M PA N Y N O T E

2 9 J u n e 2 0 2 6

Taysha Gene Therapies, Inc. (TSHA) Overweight

CONCLUSION PRICE: US$6.73

At IRSF, TSHA and NGNE provided updates on their early clinical studies for TSHA-102 TARGET: US$12.00

and NGN-401, respectively, now with 12 months of minimum follow-up. NGN-401's 80% Our $12 PT is based on a DCF through

(8/10) responder rate at 12 months exceeds the pivotal Embolden success threshold (33% 2036E (12% discount rate) and assumes

response rate) however it is not be a true comparison to TSHA data given differences in future revenue forecasts for TSHA-102 in Rett

how milestones/skills are recorded and age difference across the two datasets. Our pixel syndrome, with total non-risk-adjusted peak

analysis of TSHA's NHS presentation identified three milestones with 6%+ incremental sales of $1.8B in the US and EU.

spontaneous gain potential when comparing 6 y/o vs. 3 y/o patients, suggesting that Biren Amin

enrolling patients ages 3+ in Embolden may introduce the risk of false positive responses Sr. Research Analyst, Piper Sandler & Co.

that TSHA's 6-year-old floor sidesteps. However, NGNE's NHS analysis restricted to CGI- 732 284-6909, biren.amin@psc.com

S of 4-5 similar to the Embolden trial. Michael Mazzola, Ph.D.

Research Analyst, Piper Sandler & Co.

• TSHA-102 leads on pivotal response rate at 12 Months. This morning, competitor 212 284-9397, michael.mazzola@psc.com

NGNE (not covered) provided an update on its Ph1/2 study. At the new June 16, 2026 Felicia Berben, PharmD

Research Analyst, Piper Sandler & Co. data cut, data from two additional patients (N=10 vs. N=8 efficacy-evaluable prior) with 212 284-9412, felicia.berben@psc.com

longer follow-up (min.