Core Insights - Taysha Gene Therapies, Inc. will report its financial results for the full year ended December 31, 2024, on February 26, 2025, at 8:30 AM Eastern Time [1] - The company focuses on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system [2] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company dedicated to developing gene therapies for severe monogenic diseases, particularly targeting Rett syndrome [2] - The lead clinical program, TSHA-102, is aimed at treating Rett syndrome, which currently lacks approved disease-modifying therapies [2] - The management team possesses proven experience in gene therapy development and commercialization, leveraging a clinically and commercially validated AAV9 capsid [2]

Group 1 - Taysha Gene Therapies is a clinical-stage biotech company focused on developing gene therapies, with its leading candidate being TSHA-102 for Rett syndrome [1] - TSHA-102 utilizes "self-complementary" adeno viruses (scAAV) to deliver its treatment [1]

Company Performance - Taysha Gene Therapies, Inc. reported a quarterly loss of $0.09 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.08, representing an earnings surprise of -12.50% [1] - The company posted revenues of $1.79 million for the quarter ended September 2024, missing the Zacks Consensus Estimate by 4.54%, and a significant decline from $4.75 million in the same quarter last year [2] - Over the last four quarters, Taysha Gene Therapies has surpassed consensus EPS estimates three times but has not beaten consensus revenue estimates [2] Stock Performance - Taysha Gene Therapies shares have increased approximately 35% since the beginning of the year, outperforming the S&P 500's gain of 25.5% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.08 on revenues of $1.88 million, and for the current fiscal year, it is -$0.35 on revenues of $8.27 million [7] Industry Outlook - The Medical - Generic Drugs industry, to which Taysha Gene Therapies belongs, is currently ranked in the top 22% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

Clinical Development - TSHA-102 is in clinical development for Rett syndrome, with the first two adult patients dosed in 2023 and cohort 1 (low dose) considered complete[162]. - The Independent Data Monitoring Committee approved early dose escalation to cohort 2 (high dose) in the adolescent and adult trial, with the first patient dosed in Q2 2024[162]. - TSHA-102 was generally well tolerated, with no serious adverse events reported as of the 20-week assessment in cohort 2[162]. - TSHA-102 has received orphan drug designation and Fast Track Designation from the FDA for the treatment of Rett syndrome[165]. - The FDA granted Regenerative Medicine Advanced Therapy designation for TSHA-102, aimed at expediting development and review processes[165]. - The first adult patient in cohort 1 demonstrated sustained improvements in motor function and communication, with the ability to sit unassisted and increased vocalizations by week 52 post-treatment[181]. - The second adult patient showed a 25% reduction in seizure frequency at week 25 post-treatment, with a significant improvement in overall seizure control[184]. - The first adult patient reported a 35-point improvement in the Rett Syndrome Behavior Questionnaire (RSBQ) total score at week 52 compared to baseline[186]. - The first adult patient also exhibited a 17-point improvement in the Revised Motor Behavior Assessment (R-MBA) total score at week 52, indicating enhanced motor and functional skills[187]. - The first pediatric patient in cohort 1 was dosed in December 2023, with ongoing trials expanding into the UK and Canada[174]. - The maximum tolerated dose (MTD) established in Part A of the trials will guide the dose expansion in Part B, focusing on efficacy endpoints and study duration[178]. - The company expects to report safety and efficacy data from cohort 2 of both the adolescent and adult trial and the pediatric trial in the first half of 2025[162][164]. - The first pediatric patient improved her ability to hold an object for up to three minutes at week 12, compared to 12 seconds at baseline[205]. - The first pediatric patient experienced a seven-point increase in RSBQ Total Score at week 12 compared to baseline[208]. - The first pediatric patient showed improvements in multiple clinical domains, including fine and gross motor skills, and communication abilities[205]. - The second pediatric patient exhibited improvements in fine and gross motor skills, including the ability to stand up from a chair and walk up stairs[207]. - The first pediatric patient demonstrated an improvement in age equivalence scores across multiple domains in the MSEL-A assessment at week 12[214]. - The second pediatric patient demonstrated a 13-point improvement in RSBQ Total Score at week eight post-TSHA-102 administration compared to week four[221]. - The second pediatric patient had an increase in days reported seizure-free post-treatment, with a reduction in seizure frequency observed two weeks post-TSHA-102[224]. Financial Performance - The company has raised an aggregate of $671.0 million from various funding sources, including an IPO and follow-on offerings, to support operations[166]. - Net losses for the nine months ended September 30, 2024, were $70.5 million, compared to $159.3 million for the same period in 2023[168]. - As of September 30, 2024, the accumulated deficit was $583.5 million, with expectations of continued significant operating losses[169]. - Revenue for the three months ended September 30, 2024, was $1.8 million, a decrease of 62.4% from $4.7 million for the same period in 2023[277]. - Revenue for the nine months ended September 30, 2024, was $6.3 million, down from $11.8 million in 2023, a decline of 46.2%[287]. - Research and development expenses increased to $14.9 million for the three months ended September 30, 2024, up from $11.8 million in 2023, representing a 26.5% increase[279]. - Research and development expenses for the nine months ended September 30, 2024, totaled $50.7 million, an increase of 15.5% from $44.1 million in 2023[288]. - General and administrative expenses decreased to $7.9 million for the three months ended September 30, 2024, from $8.6 million in 2023, a reduction of 8.2%[280]. - General and administrative expenses for the nine months ended September 30, 2024, were $22.3 million, a decrease of 4.3% from $23.3 million in 2023[289]. - A non-cash impairment charge of $4.8 million was recorded for the three months ended September 30, 2024, compared to $0.6 million in the same period of 2023[281]. - Net loss for the three months ended September 30, 2024, was $25.5 million, significantly improved from a net loss of $117.1 million in 2023[277]. - A non-cash impairment charge of $4.8 million was recorded for the nine months ended September 30, 2024, compared to $0.6 million in the same period of 2023[290]. - Interest income increased to $2.1 million for the three months ended September 30, 2024, compared to $1.1 million in 2023, a growth of 90.9%[284]. - Interest income increased to $5.2 million for the nine months ended September 30, 2024, up from $1.7 million in the same period of 2023, primarily due to dividends from a money market fund[293]. - Interest expense significantly decreased to $0.1 million for the nine months ended September 30, 2024, from $4.3 million in the same period of 2023, a reduction of approximately $4.2 million[294]. - The company experienced a net change in cash, cash equivalents, and restricted cash of $13.7 million for the nine months ended September 30, 2024, down from $76.4 million in 2023[310]. - For the nine months ended September 30, 2024, the net cash used in operating activities was $62.9 million, compared to $56.9 million for the same period in 2023[310][311][312]. - Cash provided by financing activities for the nine months ended September 30, 2024, was $77.0 million, a decrease from $140.6 million in the same period of 2023[310][314]. - Investing activities used $0.4 million in cash for the nine months ended September 30, 2024, compared to $7.3 million in the same period of 2023[310][313]. - The company has not recognized any revenue from product sales to date and does not expect to generate revenue from product sales in the foreseeable future[269]. Strategic Initiatives - The company has deprioritized certain clinical-stage programs and is seeking external strategic options for further development[170]. - The company entered into a research, collaboration, and license agreement with UT Southwestern, obtaining exclusive, worldwide, royalty-free licenses for certain patent rights and know-how[250]. - The company issued 2,179,000 shares of common stock to UT Southwestern and has no future milestone or royalty obligations under the UT Southwestern Agreement, except for patent maintenance costs[252]. - The company paid a one-time upfront license fee of $3.0 million to Abeona for the CLN1 Agreement and 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[256]. - The company recognized a $3.0 million regulatory milestone payment in December 2021 related to the Abeona CLN1 Agreement following the approval of its CTA filing for TSHA-118[257]. - The company paid a one-time upfront license fee of $3.0 million to Abeona for the Rett Agreement and is obligated to pay up to $26.5 million in regulatory-related milestones and up to $30.0 million in sales-related milestones per licensed product[261]. - The company recognized a $3.5 million milestone payment in August 2023 related to the Rett Agreement after dosing the first patient with TSHA-102 in a clinical trial[262]. - The company recognized revenue from the Astellas Transactions for the nine months ended September 30, 2024, as research and development activities related to the Rett program were performed[268]. Future Outlook - The company anticipates needing substantial additional funding for ongoing operations and research and development, particularly for Rett clinical trials[304]. - The company expects existing cash and cash equivalents to fund operations into the fourth quarter of 2026, but will require additional capital for research, manufacturing, and commercialization activities[306]. - The company may need to rely on additional financing to achieve its business objectives, which may not be available on acceptable terms[309]. - The company is classified as a smaller reporting company, with a market value of shares held by non-affiliates below $700 million[321]. - The company may continue to take advantage of reduced disclosure requirements as an emerging growth company until it no longer qualifies[320].

Financial Performance - Net loss for Q3 2024 was $25.5 million, or $0.1 per share, significantly reduced from a net loss of $117.1 million, or $0.93 per share, in Q3 2023[8]. - Revenue for the three months ended September 30, 2024, was $1,788,000, compared to $4,746,000 for the same period in 2023, representing a decrease of 62.4%[15]. - Net loss for the three months ended September 30, 2024, was $25,524,000, compared to a net loss of $117,087,000 for the same period in 2023[15]. - Total operating expenses for the nine months ended September 30, 2024, were $77,838,000, up from $68,040,000 in 2023, an increase of 14.5%[15]. - Research and development expenses for the three months ended September 30, 2024, were $14,946,000, compared to $11,791,000 for the same period in 2023, an increase of 26.5%[15]. - General and administrative expenses for the nine months ended September 30, 2024, were $22,324,000, down from $23,328,000 in 2023, a decrease of 4.3%[15]. Cash and Assets - As of September 30, 2024, Taysha had $157.7 million in cash and cash equivalents, expected to support operations into Q4 2026[9]. - Cash and cash equivalents increased to $157,688,000 as of September 30, 2024, from $143,940,000 as of December 31, 2023[16]. - Total assets rose to $180,217,000 as of September 30, 2024, compared to $172,731,000 as of December 31, 2023[16]. - Total liabilities decreased to $91,420,000 as of September 30, 2024, from $97,794,000 as of December 31, 2023, a reduction of 6.4%[16]. - Additional paid-in capital increased to $674,643,000 as of September 30, 2024, from $587,942,000 as of December 31, 2023[16]. - Accumulated deficit increased to $583,520,000 as of September 30, 2024, compared to $513,007,000 as of December 31, 2023[16]. Clinical Development - TSHA-102 high dose was generally well tolerated with no serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported in two adolescent/adult patients and one pediatric patient as of data cutoff[1]. - Clinical data from the low dose cohort of TSHA-102 indicated early clinical improvements and functional gains across multiple domains within four weeks post-treatment, with sustained improvements over time[4]. - The FDA approved the use of the pivotal TSHA-102 product in REVEAL trials based on successful analytical comparability, allowing for the final commercial manufacturing process[3]. - Anticipated safety and efficacy data from both high and low dose cohorts of the REVEAL trials are expected in the first half of 2025[6]. - The Independent Data Monitoring Committee (IDMC) approved continued enrollment in the high dose cohort across both REVEAL trials, with eight patients dosed to date[1]. - The company plans to focus on objective measures for functional gains in the REVEAL trials, excluding the Rett Syndrome Behavior Questionnaire as a primary or secondary endpoint[3]. - Taysha aims to expedite the development plan for TSHA-102 through ongoing discussions with the FDA regarding trial design and endpoints[2]. Research and Development Expenses - Research and development expenses for Q3 2024 were $14.9 million, an increase of $3.1 million from $11.8 million in Q3 2023, driven by GMP batch activities and increased compensation for R&D employees[6].

Core Insights - Taysha Gene Therapies, Inc. reported progress in the development of TSHA-102, a gene therapy for Rett syndrome, with positive clinical data and FDA engagement [1][2][3] Clinical Development - TSHA-102 was well tolerated in patients, with no serious adverse events (SAEs) or dose-limiting toxicities (DLTs) reported in both adolescent/adult and pediatric cohorts [1][4] - The FDA approved the pivotal product for use in REVEAL trials based on successful analytical comparability [2][3] - Clinical data from both low and high dose cohorts of the REVEAL trials are expected to be reported in the first half of 2025 [1][5] Regulatory Engagement - Advanced discussions with the FDA regarding trial design, endpoints, and the use of a natural history dataset for Part B of the REVEAL trials have been established [2][3] - The company has aligned with the FDA on a meeting cadence to expedite the development plan for TSHA-102 [2][3] Financial Performance - Research and development expenses for Q3 2024 were $14.9 million, an increase from $11.8 million in Q3 2023, primarily due to GMP batch activities [5][6] - General and administrative expenses decreased to $7.9 million in Q3 2024 from $8.6 million in Q3 2023 [6] - The net loss for Q3 2024 was $25.5 million, significantly reduced from a net loss of $117.1 million in Q3 2023 [7] Cash Position - As of September 30, 2024, Taysha had $157.7 million in cash and cash equivalents, expected to support operations into Q4 2026 [8] Company Overview - Taysha Gene Therapies focuses on AAV-based gene therapies for severe monogenic diseases, with TSHA-102 as its lead program targeting Rett syndrome [10][12]

Core Insights - Taysha Gene Therapies, Inc. is advancing AAV-based gene therapies for severe monogenic diseases of the central nervous system (CNS) and has presented biodistribution data from studies on AAV9 gene therapy vectors in non-human primates (NHPs) [1][2][5] - The analysis indicates that intrathecal (IT) administration is an effective, safe, and minimally invasive method for delivering gene therapies targeting CNS diseases, including Rett syndrome [1][2][3] Company Overview - Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing AAV-based gene therapies for severe CNS diseases, with its lead program TSHA-102 targeting Rett syndrome [5] - TSHA-102 is designed as a one-time treatment to deliver a functional form of the MECP2 gene to address the genetic root cause of Rett syndrome [3][4] - The company has received multiple designations from regulatory agencies, including Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA [3] Research Findings - The analysis of five NHP studies showed that both IT and intra-cisterna magna (ICM) administration resulted in comparable biodistribution of AAV9 vectors across brain and spinal cord regions [1][2] - The findings support the clinical potential of IT administration for outpatient use in both children and adults, reinforcing Taysha's clinical development strategy [2] Rett Syndrome Context - Rett syndrome is a rare neurodevelopmental disorder primarily affecting females, characterized by loss of communication and motor functions, with no approved disease-modifying therapies currently available [4] - It is estimated that Rett syndrome affects between 15,000 and 20,000 patients in the U.S., EU, and U.K. due to mutations in the MECP2 gene [4]

Core Viewpoint - Taysha Gene Therapies, Inc. (TSHA) has received an upgrade to a Zacks Rank 1 (Strong Buy), indicating a positive outlook based on rising earnings estimates, which significantly influence stock prices [1][4]. Earnings Estimates and Revisions - The Zacks Consensus Estimate for Taysha Gene Therapies indicates an expected loss of -$0.35 per share for the fiscal year ending December 2024, reflecting a year-over-year change of 63.5% [9]. - Over the past three months, the Zacks Consensus Estimate for the company has increased by 16.8%, showcasing a trend of rising earnings estimates [9]. Zacks Rating System - The Zacks rating system is based on changes in earnings estimates, which are crucial for predicting near-term stock price movements [2][3]. - The system classifies stocks into five groups, with Zacks Rank 1 stocks historically generating an average annual return of +25% since 1988 [8]. - Taysha Gene Therapies' upgrade to Zacks Rank 1 places it in the top 5% of Zacks-covered stocks, indicating strong potential for market-beating returns in the near term [11]. Market Implications - Rising earnings estimates and the corresponding rating upgrade suggest an improvement in Taysha Gene Therapies' underlying business, likely leading to increased buying pressure and a higher stock price [6][4]. - The influence of institutional investors, who adjust their valuations based on earnings estimates, contributes to stock price movements, reinforcing the importance of earnings revisions [5].

Financial Data and Key Metrics Changes - Research and development expenses decreased to $15.1 million for Q2 2024 from $19.8 million in Q2 2023, primarily due to a milestone fee payable in the prior year [18] - General and administrative expenses increased to $7.3 million in Q2 2024 from $6 million in Q2 2023, attributed to higher noncash stock-based compensation and consulting fees [19] - Net loss for Q2 2024 was $20.9 million or $0.09 per share, compared to a net loss of $24.6 million or $0.38 per share in Q2 2023 [19] - As of June 30, 2024, the company had $172.7 million in cash and cash equivalents, with expectations to support operations into Q4 2026 [19] Business Line Data and Key Metrics Changes - The TSHA-102 program for Rett Syndrome showed promising safety and efficacy data from the low dose cohort in ongoing clinical trials [7][11] - The company initiated the high dose cohort and expanded its pediatric trial into Canada, indicating progress in clinical evaluations [7][10] Market Data and Key Metrics Changes - Rett Syndrome affects an estimated 15,000 to 20,000 patients in the U.S., EU, and UK, with no approved disease-modifying therapies currently available [8] Company Strategy and Development Direction - The company aims to develop a transformative therapeutic option for all patients suffering from Rett Syndrome, focusing on clinical trial execution and data collection [8][21] - The strategy includes advancing to high dose cohorts based on encouraging preliminary data from low dose cohorts, which is expected to inform regulatory discussions [12][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety profile and clinical effects of TSHA-102, emphasizing the potential to address significant unmet medical needs in Rett Syndrome [20][21] - The company plans to report safety and efficacy data from both low and high dose cohorts in the first half of 2025, indicating a commitment to transparency and data-driven decision-making [14][21] Other Important Information - The company completed a public follow-on offering, resulting in net proceeds of $76.8 million, which is expected to extend the cash runway into Q4 2026 [13] Q&A Session Summary Question: How will the high-dose data be measured for dose response? - Management indicated that preclinical data showed improvements at high doses and that consistent effects across clinical domains are expected [22] Question: What is the status of regulatory discussions? - The company has an upcoming Type B meeting with regulatory authorities to align on trial design and data sharing [26] Question: Will the timeline for data impact the timeline for FDA meetings? - Management stated that they aim to complete dosing and natural history analysis before engaging with the FDA, with flexibility in the timeline [42] Question: What are the baseline characteristics of high-dose patients? - Management noted that baseline characteristics are consistent with Rett Syndrome, with common clinical features expected across patients [48] Question: Are there any updates on safety events related to the immunosuppressive regimen? - Management confirmed that there have been no new significant adverse events reported and that discussions regarding the dosing stagger are ongoing [52]

Taysha Gene Therapies, Inc. (TSHA) came out with a quarterly loss of $0.09 per share versus the Zacks Consensus Estimate of a loss of $0.10. This compares to loss of $0.38 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 10%. A quarter ago, it was expected that this company would post a loss of $0.11 per share when it actually produced a loss of $0.10, delivering a surprise of 9.09%. Over the last four quarters, the company ha ...