Taysha Gene Therapies

TSHA

Market cap $1.3B

Taysha Gene Therapies, Inc. ( TSHA ) Q3 2025 Earnings Call November 4, 2025 8:30 AM EST Company Participants Hayleigh Collins - Director of Corporate Communications & Investor Relations Sean Nolan - CEO & Chairman Sukumar Nagendran - President, Head of Research & Development and Director Kamran Alam - CFO & Corporate Secretary Conference Call Participants Kristen Kluska - Cantor Fitzgerald & Co., Research Division Salveen Richter - Goldman Sachs Group, Inc., Research Division Tazeen Ahmad - BofA Securities, Research Division Gil Blum - Needham & Company, LLC, Research Division Maurice Raycroft - Jefferies LLC, Research Division Jack Allen - Robert W. Baird & Co. Incorporated, Research Division Christopher Raymond - Raymond James & Associates, Inc., Research Division Yanan Zhu - Wells Fargo Securities, LLC, Research Division Mehdi Goudarzi - Truist Securities, Inc., Research Division Presentation Operator Good day, everyone and welcome to The Taysha Gene Therapies Third Quarter 2025 Earnings Call.

TSHA-102 granted Breakthrough Therapy designation by FDA Finalized FDA alignment on REVEAL pivotal trial protocol and SAP, including 6-month interim analysis that may expedite BLA submission, which was enabled by the rigorous developmental milestone evaluation in Part A REVEAL Phase 1/2 trials showing an unprecedented response rate Dosing of first patient in REVEAL pivotal trial scheduled for Q4 2025, with enrollment of additional patients expected to continue at multiple sites this quarter Presented new supplemental analysis of Part A REVEAL data reinforcing the broad and consistent, multi-domain impact of TSHA-102 on activities of daily living at the CNS Annual Meeting TSHA-102 continues to be generally well tolerated with no treatment-related SAEs or DLTs in the 12 patients treated in the Part A REVEAL Phase 1/2 trials as of October 2025 data cutoff Regained full unencumbered rights to TSHA-102 Rett syndrome program, enabling Taysha to focus on driving long-term value with full strategic flexibility and optionality Conference call and webcast today at 8:30 AM Eastern Time DALLAS, Nov. 04, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the third quarter ended September 30, 2025, and provided a corporate update. “The progress we've made in the third quarter of 2025 sets the stage for a potentially transformative period ahead for Taysha.

DALLAS, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the third quarter ended September 30, 2025, and host a corporate update conference call and webcast on Tuesday, November 4, 2025, at 8:30 AM Eastern Time.

The 2022 Option Agreement between Astellas and Taysha has expired Regaining full rights to TSHA-102 Rett syndrome program enables Taysha to focus on driving its long-term value with full strategic flexibility and optionality TSHA-102 has demonstrated compelling Part A REVEAL Phase 1/2 safety and efficacy data, received FDA Breakthrough Therapy designation and is advancing with a clear path to potential registration Dosing of the first patient in the REVEAL pivotal trial is scheduled this quarter DALLAS, Oct. 16, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that the Company has regained full rights to its lead TSHA-102 program in clinical evaluation for Rett syndrome. The 2022 Option Agreement between Astellas and Taysha, which had granted Astellas the exclusive option to enter a negotiation period to obtain an exclusive license to TSHA-102 in Rett syndrome and certain rights with respect to change in control transactions involving Taysha, has expired.

Previously disclosed 100% response rate across all 10 patients in Part A for pivotal trial primary endpoint of gain/regain of ≥ one natural history defined developmental milestone assessed via video-evidenced review by independent central raters New supplemental analysis of validated, structured efficacy scales provides supportive evidence of additional functional gains, with 100% of patients demonstrating multiple skill gains/improvements outside the natural history defined developmental milestones Findings reinforce the broad and consistent functional gains seen across the core domains that impact activities of daily living, with 22 developmental milestones and 165 additional skills/improvements achieved across the 10 patients post-TSHA-102 DALLAS, Oct. 09, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced results from a new supplemental data analysis from Part A of the REVEAL Phase 1/2 adult/adolescent and pediatric trials evaluating TSHA-102 in females with Rett syndrome at the 54th Child Neurology Society (CNS) Annual Meeting. The analysis provides supportive evidence of additional functional gains in skills and improvements across core disease characteristics that are outside of the natural history defined developmental milestones, further highlighting the consistent, multi-domain impact of TSHA-102 on activities of daily living.

Taysha Gene Therapies, Inc.'s lead asset is TSHA-102. This is an intrathecal AAV9 gene therapy for Rett with a 100% Phase 1/2 responder rate and favorable safety. The FDA granted it a Breakthrough Therapy Designation. Its pivotal REVEAL trial finalized with a 6-month interim that could accelerate a BLA submission. The new trial uses objective, blinded, video-rated milestone gains. This will make the results easier to interpret and potentially support an accelerated BLA.

DALLAS, Oct. 03, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that, on October 1, 2025, the Compensation Committee of Taysha's Board of Directors granted seven new employees, in the aggregate, options to purchase 1,289,600 shares of the Company's common stock in connection with their employment. The stock options were granted under the Taysha Gene Therapies, Inc. 2023 Inducement Plan as an inducement material to the individuals entering employment with Taysha in accordance with Nasdaq Listing Rule 5635(c)(4).

As of Oct. 2, 2025, two stocks in the health care sector could be flashing a real warning to investors who value momentum as a key criteria in their trading decisions.

Breakthrough Therapy designation granted based on FDA's review of available clinical evidence of safety and efficacy from all 12 patients treated in Part A of the REVEAL Phase 1/2 trials Finalized FDA alignment on REVEAL pivotal trial protocol and SAP following resolution of remaining clinical and statistical queries to IND application amendment; on track to begin patient enrollment in Q4 2025 Key pivotal trial design elements remain unchanged, including 6-month interim analysis to potentially expedite BLA submission based on the rigorous developmental milestone evaluation in Part A showing an unprecedented response rate at 6 months post-TSHA-102 that deepened over time DALLAS, Oct. 02, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TSHA-102, an intrathecally delivered AAV9 gene therapy with disease modifying potential, for the treatment of Rett syndrome. Additionally, the Company announced that it has finalized alignment with the FDA on the REVEAL pivotal trial protocol and statistical analysis plan (SAP) that are intended to support the planned Biologics License Application (BLA) submission for TSHA-102, following the resolution of remaining clinical and statistical queries.

DALLAS, Sept. 05, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that, on September 2, 2025, the Compensation Committee of Taysha's Board of Directors granted five new employees, in the aggregate, options to purchase 225,000 shares of the Company's common stock in connection with their employment. The stock options were granted under the Taysha Gene Therapies, Inc. 2023 Inducement Plan as an inducement material to the individuals entering employment with Taysha in accordance with Nasdaq Listing Rule 5635(c)(4).