Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call May 15, 2025 08:30 AM ET Speaker0 Greetings, and welcome to the Tatia Gene Therapies First Quarter twenty twenty five Earnings Call. At this time, all participants are in listen only mode. A question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Hayley Collins, Director, Head of Corporate Communications. Please go ahead. Speaker1 Thank ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-39536 Taysha Gene Therapies, Inc. (Exact Name of Registrant as Specified in its Charter) | Delaware | 84- ...

Exhibit 99.1 Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update Obtained written alignment from the FDA on key elements of TSHA-102 pivotal Part B trial design and next steps on enabling study initiation The FDA advised the Company to submit pivotal Part B trial protocol and associated statistical analysis plan as an amendment to the IND application, eliminating the need for formal end-of-phase meeting and potentially expediting study initiation and registration ...

DALLAS, May 08, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), 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 first quarter ended March 31, 2025, and host a corporate update conference call and webcast on Thursday, May 15, 2025, at 8:30 AM Eastern Time. Conference Call DetailsThursday, May 15, ...

Taysha Gene Therapies, Inc. (TSHA) is expected to deliver a year-over-year increase in earnings on lower revenues when it reports results for the quarter ended March 2025. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price.The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they ...

Financial Data and Key Metrics Changes - Research and development expenses increased to $66 million for the full year ended December 31, 2024, compared to $56.8 million for the previous year, driven by good manufacturing practices and additional clinical trial activities [29] - General and administrative expenses decreased to $29 million for the full year ended December 31, 2024, from $30 million in 2023, primarily due to a decrease in issuance costs [30] - Net loss for the full year ended December 31, 2024, was $89.3 million or $0.36 per share, compared to a net loss of $111.6 million or $0.96 per share for the previous year [30] - As of December 31, 2024, the company had $139 million in cash and cash equivalents, expected to support planned operating expenses into the fourth quarter of 2026 [31] Business Line Data and Key Metrics Changes - The TSHA-102 program is advancing with a focus on generating critical clinical data across two REVEAL Phase 1/2 trials for Rett syndrome [10][11] - Dosing of ten patients in Part A of the REVEAL trials has been completed, with both high and low doses demonstrating an encouraging safety profile [12][13] Market Data and Key Metrics Changes - The company is focused on the Rett syndrome market, which has a high unmet need due to the progressive nature of the disease and the significant burden on caregivers [24] Company Strategy and Development Direction - The strategy involves utilizing Part A of the trials to generate a dataset that informs the pivotal Part B trial, focusing on objective measures that capture improvements in core features of Rett syndrome [13][20] - The company aims to bring TSHA-102 to patients as expeditiously and safely as possible, with ongoing discussions with the FDA to solidify the regulatory pathway [16][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the differentiated gene therapy candidate, anticipating a transformative year ahead with key milestones expected in the first half of 2025 [32][33] - The FDA discussions have been constructive, with a focus on functional outcomes that are clinically meaningful to patients and caregivers [46][49] Other Important Information - The company has completed dosing in both the high and low dose cohorts, with ongoing evaluations of functional gains across multiple domains [15][27] - The FDA has been open-minded regarding trial design and endpoints, emphasizing the importance of clinically meaningful outcomes [63][64] Q&A Session Summary Question: Can you speak to the number of people involved in FDA discussions? - Management indicated that discussions typically involve 15 to 20 FDA representatives, including senior officials, and have not been impacted by recent changes in administration [38][39] Question: Any changes in FDA's expectations regarding functional gains? - Management stated that FDA discussions have remained consistent, focusing on clinically meaningful objective improvements across key domains [44][49] Question: What differentiates TSHA-102 from competitors in the gene therapy space? - Management highlighted the use of self-complementary technology in TSHA-102, which allows for quicker protein production and potentially faster clinical effects compared to competitors [56][57] Question: What is the timeline for reaching alignment with the FDA on endpoints? - Management expressed optimism about ongoing discussions and aims to provide a regulatory update coinciding with clinical data updates in the first half of 2025 [84][85] Question: What is the safety profile of the high dose patients? - Management reported no treatment-related serious adverse events or dose-limiting toxicities observed in the ten patients dosed, indicating a favorable safety profile [75][103]

Core Insights - Taysha Gene Therapies, Inc. reported a quarterly loss of $0.07 per share, which was better than the Zacks Consensus Estimate of a loss of $0.08, representing an earnings surprise of 12.50% [1] - The company generated revenues of $2.02 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 6.42%, but down from $3.6 million in the same quarter last year [2] - The stock has underperformed the market, losing about 1.7% since the beginning of the year compared to the S&P 500's gain of 1.3% [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.08 on revenues of $2.63 million, and for the current fiscal year, it is -$0.37 on revenues of $9.73 million [7] - The estimate revisions trend for Taysha Gene Therapies is mixed, resulting in a Zacks Rank 3 (Hold), indicating expected performance in line with the market [6] Industry Context - The Medical - Generic Drugs industry, to which Taysha Gene Therapies belongs, is currently ranked in the bottom 24% of over 250 Zacks industries, suggesting potential challenges ahead [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which could impact Taysha's stock performance [5]

Clinical Development - TSHA-102 is in clinical development for Rett syndrome, a rare neurodevelopmental disorder, with no approved disease-modifying therapies currently available[17]. - The REVEAL Phase 1/2 trials have completed dosing of 10 patients, with no treatment-related serious adverse events or dose-limiting toxicities reported as of February 17, 2025[19]. - Positive longer-term clinical data from the REVEAL trials indicate sustained clinical improvements in both adolescent/adult and pediatric cohorts[29]. - Improvements in motor skills, communication, and autonomic function were observed in adult patients as early as four weeks post-treatment, with effects persisting over time[31]. - The maximum tolerated dose established in Part A of the REVEAL trials will be administered during the pivotal Part B phase[26]. - The company aims to report safety and efficacy data from the REVEAL trials in the first half of 2025[19]. - TSHA-102 demonstrated improvements in multiple efficacy measures as early as four weeks post-treatment, with sustained improvements observed at week 52 for patient one and week 25 for patient two[34]. - In the pediatric trial, TSHA-102 was well-tolerated with no treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported as of February 17, 2025[35]. - Patient one showed a CGI-S score improvement from 5 (markedly ill) at baseline to 2 (minimally improved) by week 12, while patient two improved from a score of 4 (moderately ill) to 1 (very much improved) by week 8[37]. - Patient one improved hand function, able to hold an object for 3 minutes compared to 12 seconds at baseline, while patient two showed improved gait and stability when walking[37]. Regulatory Designations - TSHA-102 has received multiple designations from regulatory authorities, including orphan drug designation and Fast Track Designation from the FDA[20]. - TSHA-118 has received orphan drug designation and fast track designation from the FDA for the treatment of CLN1 disease[46]. - TSHA-105 has also received orphan drug designation and rare pediatric disease designation from the FDA for SLC13A5 deficiency[50]. - Orphan drug designation is granted for drugs intended to treat rare diseases affecting fewer than 200,000 individuals in the U.S., providing seven years of exclusivity upon FDA approval[120]. - Fast track designation allows for expedited review of products intended to treat serious conditions and may include rolling review of BLA sections[112]. - Breakthrough therapy designation provides intensive FDA interaction and guidance, expediting development for products showing substantial improvement over existing therapies[113]. Financial Overview - As of December 31, 2024, the company had incurred net losses of $89.3 million and $111.6 million for the years ended December 31, 2024 and 2023, respectively, with an accumulated deficit of $602.3 million[192]. - The company has financed operations with $671.0 million in gross proceeds from equity financings, including pre-IPO private placements and public offerings, but has not generated any revenue from product sales[192]. - The company had cash and cash equivalents of $139.0 million as of December 31, 2024, which is expected to fund operating expenses into the fourth quarter of 2026[200]. - The company expects to incur significant expenses and operating losses over the next several years as it conducts clinical trials and seeks regulatory approval for product candidates[199]. - The company may require additional capital to achieve its business objectives, and its ability to raise capital may be adversely impacted by global economic conditions[202]. - The company currently has no committed external source of funds and may need to raise additional capital through equity offerings or debt financing, which could dilute stockholder ownership[209]. Intellectual Property and Licensing - The company has in-licensed five U.S. patents, 14 foreign patents, and 54 pending foreign patent applications as of February 24, 2024, to protect its proprietary technology[87]. - The company in-licenses patents related to TSHA-102, with expected expiration dates ranging from 2038 to 2041, depending on the specific patent[87][88]. - The company issued 2,179,000 shares of common stock to UT Southwestern as part of the license agreement[69]. - The company paid a one-time upfront license fee of $3.0 million to Abeona for the CLN1 Agreement in fiscal year 2020[73]. - The company is obligated to pay up to $26.0 million in regulatory-related milestones and up to $30.0 million in sales-related milestones per licensed product under the Abeona CLN1 Agreement[73]. Market and Economic Challenges - The company faces significant uncertainty regarding coverage and reimbursement for its pharmaceutical products, which depend on third-party payors' decisions[168]. - The company may encounter challenges in obtaining adequate coverage and reimbursement for products administered under physician supervision due to higher associated prices[170]. - The Inflation Reduction Act of 2022 introduces price negotiation for certain high-expenditure drugs under Medicare, impacting the company's pricing strategies[176]. - Legislative changes have resulted in aggregate reductions of Medicare payments to providers, which will remain in effect through 2032 unless further action is taken[175]. - Increased scrutiny over drug pricing and reimbursement methodologies may lead to further regulatory challenges for the company[176]. Clinical Trial Regulations - The FDA requires the submission of an IND application before initiating clinical trials, which must become effective for trials to begin[102]. - Human clinical trials are conducted in three phases: Phase 1 focuses on safety and dosage, Phase 2 evaluates efficacy and side effects, and Phase 3 confirms efficacy and safety in a larger population[105]. - The FDA may suspend clinical trials if patients are exposed to unacceptable health risks or if the trial is unlikely to meet its objectives[104]. - Regulatory agencies require extensive monitoring and auditing of clinical activities and data throughout all phases of clinical development[106]. Company Operations and Risks - The company has a limited operating history and no history of commercializing products, making it difficult for investors to assess future viability[197]. - The company relies on collaboration with UT Southwestern for preclinical research and development, and any failure in this collaboration could materially harm its business[191]. - The company anticipates that negative public opinion and increased regulatory scrutiny of gene therapy may adversely impact the development of its product candidates[193]. - The company faces risks related to the development of its product candidates, including the need for significant investment and successful completion of clinical trials[214]. - The company may need to relinquish rights to technologies or product candidates if it raises additional funds through collaborations or licensing arrangements[210].

Clinical Trials - TSHA-102 clinical program has completed dosing of 10 patients in Part A of the REVEAL trials, with 6 in the high dose cohort and 4 in the low dose cohort[5] - The pivotal trial design update for TSHA-102 is anticipated in the first half of 2025[7] - Clinical data from both high and low dose cohorts in the REVEAL trials is expected in the first half of 2025[8][9] Financial Performance - Net loss for the full year 2024 was $89.3 million, or $0.36 per share, compared to a net loss of $111.6 million, or $0.96 per share, in 2023[12] - Research and development expenses for the full year 2024 were $66.0 million, an increase of 16.2% from $56.8 million in 2023[10] - Total operating expenses for 2024 were $99.8 million, up from $87.9 million in 2023, reflecting increased clinical trial activities[20] Cash and Assets - Cash and cash equivalents as of December 31, 2024, were $139.0 million, expected to support operations into Q4 2026[12] - Total assets decreased to $160.4 million in 2024 from $172.7 million in 2023, primarily due to a reduction in cash and cash equivalents[22] Administrative Expenses - General and administrative expenses decreased to $29.0 million in 2024 from $30.0 million in 2023, a reduction of 3.3%[11] Warrant Liability - The company reported a change in fair value of warrant liability resulting in a gain of $16,000 in 2024, compared to a loss of $34.7 million in 2023[20]

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of February 2025 data cutoff Completed dosing of the 10 patients in Part A of both REVEAL trials; maturing dataset continues to support advancement toward pivotal Part B trial Productive ongoing discussions with the FDA to solidify regulatory pathway for TSHA-102; update on pivota ...