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 ...

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 June 30, 2024 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 84-31995 ...

Financial Performance - Taysha Gene Therapies reported a net loss of $20.9 million, or $0.09 per share, for Q2 2024, compared to a net loss of $24.6 million, or $0.38 per share, in Q2 2023[5]. - Research and development expenses decreased to $15.1 million in Q2 2024 from $19.8 million in Q2 2023, primarily due to a milestone fee in the prior year[4]. - General and administrative expenses increased to $7.3 million in Q2 2024 from $6.0 million in Q2 2023, driven by higher stock-based compensation and consulting fees[5]. - The accumulated deficit widened from $(513,007,000) to $(557,996,000), indicating ongoing investment in growth despite losses[14]. Cash and Assets - Cash and cash equivalents as of June 30, 2024, were $172.7 million, providing a runway into Q4 2026[5]. - Total current assets as of December 31, 2023, amount to $149,868,000, with cash and cash equivalents at $143,940,000[12]. - Total assets increased from $172,731,000 to $200,393,000, indicating a strong growth trajectory[14]. - The company holds $2,000,000 in assets held for sale, indicating potential divestitures or restructuring[12]. - The company has $449,000 in restricted cash, which may be earmarked for specific future obligations[12]. Liabilities and Equity - Total liabilities decreased from $97,794,000 to $91,525,000, reflecting a reduction in current liabilities from $36,756,000 to $34,176,000[13]. - Stockholders' equity increased significantly from $74,937,000 to $108,868,000, driven by an increase in additional paid-in capital from $587,942,000 to $664,457,000[14]. - Deferred revenue decreased from $18,106,000 to $13,583,000, suggesting a potential shift in revenue recognition[13]. - Operating lease liabilities remain stable, with a slight decrease in the current portion from $18,953,000 to $18,134,000[13]. Clinical Trials and Developments - TSHA-102 was generally well tolerated with no serious adverse events reported in both pediatric and adolescent/adult trials[2]. - Dosing of the second patient in the high dose cohort of the adolescent/adult trial is scheduled for Q3 2024[4]. - Safety and efficacy data from cohort two (n=3) and updates from cohort one (n=2) are expected in the first half of 2025[4]. - The pediatric trial has received clearance to expand into Canada, enhancing the study's geographical reach[3]. - Taysha aims to report clinical data from both high and low dose cohorts in the first half of 2025, focusing on further informing regulatory discussions[2]. Capital Raising - The company completed a public follow-on offering with total net proceeds of $76.8 million[3]. - The increase in common stock issued from 186,960,193 to 201,381,450 shares reflects ongoing capital raising efforts[14].

Presented cohort one (low dose) data from both trials of TSHA-102 at 2024 IRSF Rett Syndrome Scientific Meeting that demonstrated durable improvements across consistent clinical domains and an encouraging safety profile in adult (up to 52 weeks) and pediatric (up to 22 weeks) patients TSHA-102 was generally well tolerated with no SAEs or DLTs as of initial six-week assessment in first patient in cohort two (high dose) of adolescent/adult trial; IDMC approved dosing of second (adolescent/adult trial) and fir ...