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 Finding ...

My name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens, OH, USA, a graduate degree in Business Management from Universidad Andina Simon Bolivar, Quito, Ecuador, and the Ph.D. degree in computer applications from the University of Alicante, Spain.Disclosure: I collaborate professionally with Edgar Torres H, who is also an author on Seeking ...

U.S. stocks were mixed, with the Dow Jones index falling more than 50 points on Thursday.Shares of Taysha Gene Therapies Inc (NASDAQ:TSHA) rose sharply during Thursday's session after the FDA granted the company Breakthrough Therapy designation to TSHA-102 for the treatment of Rett syndrome.Needham analyst Gil Blum maintained Taysha Gene Therapies with a Buy and raised the price target from $8 to $10.Taysha Gene Therapies shares jumped 50.4% to $4.78 on Thursday.Here are some other big stocks recording gain ...

Taysha Gene Therapies (NASDAQ:TSHA) jumped ~39% on Thursday after the U.S. FDA granted its Breakthrough Therapy designation for the company's lead candidate TSHA-102 as a treatment for Rett syndrome, a rare neurodevelopmental disorder. Additionally, the gene therapy developer stated that the ...

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 ...

Core Insights - 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 $0.07, marking an earnings surprise of -28.57% [1] - The company generated revenues of $1.99 million for the quarter ended June 2025, exceeding the Zacks Consensus Estimate by 15.73% and showing significant growth from $1.11 million a year ago [2] - Taysha Gene Therapies shares have increased approximately 59.5% year-to-date, outperforming the S&P 500's gain of 8.4% [3] Financial Performance - Over the last four quarters, Taysha Gene Therapies has surpassed consensus EPS estimates only once [2] - The current consensus EPS estimate for the upcoming quarter is -$0.07 on revenues of $1.73 million, and for the current fiscal year, it is -$0.31 on revenues of $7.51 million [7] Industry Context - The Medical - Generic Drugs industry, to which Taysha Gene Therapies belongs, is currently ranked in the top 41% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

PART I. FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements%20(Unaudited)) The company significantly increased cash to **$312.8 million** post-public offering, reporting a Q2 2025 net loss of **$26.9 million** due to higher R&D [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets rose to **$333.3 million** by June 30, 2025, driven by a public offering that boosted cash and stockholders' equity Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $312,761 | $139,036 | | Total current assets | $316,264 | $142,130 | | **Total assets** | **$333,331** | **$160,364** | | **Liabilities & Equity** | | | | Total current liabilities | $25,349 | $26,227 | | Term loan, net | $41,051 | $43,942 | | **Total liabilities** | **$84,604** | **$88,839** | | **Total stockholders' equity** | **$248,727** | **$71,525** | | **Total liabilities and stockholders' equity** | **$333,331** | **$160,364** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Q2 2025 revenue increased to **$2.0 million**, but operating expenses rose, leading to a widened net loss of **$26.9 million** for the quarter Condensed Consolidated Statements of Operations Highlights (in thousands) | Account | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Six Months 2025 (in thousands) | Six Months 2024 (in thousands) | | :--- | :--- | :--- | :--- | :--- | | Revenue | $1,986 | $1,112 | $4,288 | $4,523 | | Research and development | $20,141 | $15,073 | $35,706 | $35,730 | | General and administrative | $8,598 | $7,338 | $16,756 | $14,422 | | Loss from operations | ($26,753) | ($21,299) | ($48,174) | ($45,629) | | **Net loss** | **($26,882)** | **($20,928)** | **($48,411)** | **($44,989)** | | Net loss per common share | ($0.09) | ($0.09) | ($0.17) | ($0.19) | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash provided by financing activities, primarily from a public offering, led to a **$173.7 million** increase in cash for the six months ended June 30, 2025 Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Category | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | $(42,202) | $(41,302) | | Net cash used in investing activities | $(309) | $(341) | | Net cash provided by financing activities | $216,236 | $70,446 | | **Net increase in cash, cash equivalents and restricted cash** | **$173,725** | **$28,803** | [Notes to Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the company's biotech focus, liquidity from a **$215.6 million** public offering, Astellas option status, debt refinancing, and ongoing legal proceedings - The company is a clinical-stage biotechnology firm focused on AAV-based gene therapies for severe monogenic diseases of the central nervous system[30](index=30&type=chunk) - In May 2025, the company completed an underwritten public offering, raising approximately **$215.6 million in net proceeds**, which is expected to fund operations for at least twelve months from the issuance date of these financial statements[35](index=35&type=chunk)[36](index=36&type=chunk) - Under an agreement with Astellas, Taysha delivered the Rett Data Package for TSHA-102 in mid-2025, triggering a 90-day period for Astellas to decide whether to exercise its option for an exclusive license[63](index=63&type=chunk) - On August 7, 2025, the company entered into a new term loan agreement with Trinity Capital, drawing **$50.0 million** and terminating its previous loan facility. The new agreement provides for up to an additional **$50.0 million** in financing contingent on achieving certain regulatory milestones for TSHA-102[150](index=150&type=chunk) - The company is a nominal defendant in consolidated stockholder derivative lawsuits related to its August 2023 private placement and has received subpoenas from the SEC as part of an investigation into the same financing[141](index=141&type=chunk)[142](index=142&type=chunk)[143](index=143&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=45&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses TSHA-102's clinical progress, a **$48.4 million** net loss for H1 2025, and liquidity of **$312.8 million**, projecting funding into 2028 [Overview and Clinical Update](index=45&type=section&id=Overview%20and%20Clinical%20Update) The company's lead program, TSHA-102 for Rett syndrome, completed Part A dosing with good tolerability and is preparing for pivotal Part B enrollment in Q4 2025 - The lead clinical program, TSHA-102, is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies[161](index=161&type=chunk) - Dosing of all **12 patients** in Part A of the REVEAL trials is complete. As of August 4, 2025, TSHA-102 was generally well-tolerated with no treatment-related serious adverse events or dose-limiting toxicities[164](index=164&type=chunk) - The company has commenced site activation for the REVEAL pivotal Part B trial, with patient enrollment expected to begin in the **fourth quarter of 2025**[165](index=165&type=chunk) - TSHA-102 has received several key regulatory designations, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) from the FDA[167](index=167&type=chunk) [Results of Operations](index=66&type=section&id=Results%20of%20Operations) Q2 2025 revenue increased to **$2.0 million**, while R&D expenses rose by **$5.0 million** and G&A expenses by **$1.3 million**, primarily due to TSHA-102 activities Comparison of Operating Expenses (in millions) | Expense Category | Q2 2025 (in millions) | Q2 2024 (in millions) | Change (in millions) | | :--- | :--- | :--- | :--- | | Research and development | $20.1 | $15.1 | +$5.0 | | General and administrative | $8.6 | $7.3 | +$1.3 | - The increase in Q2 2025 R&D expenses was primarily driven by BLA-enabling process performance qualification manufacturing initiatives and clinical trial activities[238](index=238&type=chunk) [Liquidity and Capital Resources](index=68&type=section&id=Liquidity%20and%20Capital%20Resources) Cash and equivalents reached **$312.8 million** by June 30, 2025, supported by a **$215.6 million** public offering and new **$50.0 million** debt, funding operations into 2028 - The company had cash and cash equivalents of **$312.8 million** as of June 30, 2025[250](index=250&type=chunk) - In May 2025, the company raised total net proceeds of **$215.6 million** from an underwritten public offering[261](index=261&type=chunk) - The company believes its existing cash and cash equivalents will be sufficient to fund operating expenses and capital requirements into **2028**[264](index=264&type=chunk) - In August 2025, the company entered into a new term loan agreement, drawing an initial **$50.0 million** and repaying its existing term loan[252](index=252&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=77&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, the company is exempt from providing quantitative and qualitative disclosures about market risk - As a smaller reporting company, Taysha is not required to provide quantitative and qualitative disclosures about market risk[281](index=281&type=chunk) [Controls and Procedures](index=77&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control over financial reporting - Management concluded that as of June 30, 2025, the company's disclosure controls and procedures were effective[282](index=282&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[283](index=283&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=78&type=section&id=Item%201.%20Legal%20Proceedings) The company faces stockholder derivative lawsuits and an SEC investigation related to its August 2023 Private Placement, with litigation currently stayed - The company is a nominal defendant in consolidated stockholder derivative actions concerning its August 2023 Private Placement. The litigation is currently stayed until **September 30, 2025**[286](index=286&type=chunk) - The company and certain officers received subpoenas from the SEC in late 2024 related to the August 2023 PIPE and other public offerings. The SEC investigation is not a determination of any wrongdoing[287](index=287&type=chunk) [Risk Factors](index=78&type=section&id=Item%201A.%20Risk%20Factors) The company's success hinges on TSHA-102, facing risks from evolving clinical data, lack of pivotal trial experience, Astellas option uncertainties, and legal proceedings - The company is substantially dependent on the success of its lead product candidate, TSHA-102, which is its only product in clinical development[292](index=292&type=chunk) - Interim or preliminary results from clinical trials, such as those for TSHA-102, may change as more patient data becomes available, which could harm business prospects[290](index=290&type=chunk) - The company has never conducted pivotal clinical trials and may be unable to successfully execute the necessary trials for TSHA-102 to support a BLA submission[298](index=298&type=chunk) - Uncertainties exist regarding the option agreement with Astellas for TSHA-102. If Astellas exercises the option, it could be considered a sale of substantially all company assets, and potential disputes over terms could arise[293](index=293&type=chunk)[294](index=294&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=88&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the reporting period - The company reported no unregistered sales of equity securities for the period[313](index=313&type=chunk) [Other Information](index=88&type=section&id=Item%205.%20Other%20Information) The company disclosed a Rule 10b5-1 trading plan and a new **$50.0 million** term loan agreement with Trinity Capital, refinancing prior debt - On June 11, 2025, Sukumar Nagendran, President and Head of R&D, adopted a prearranged Rule 10b5-1 stock trading plan for the potential sale of up to **800,000 shares**[318](index=318&type=chunk) - On August 7, 2025, the company entered into a new loan agreement with Trinity Capital, drawing **$50.0 million** in an initial tranche and terminating its existing term loan[319](index=319&type=chunk)[327](index=327&type=chunk) [Exhibits](index=91&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the quarterly report, including corporate governance documents, pre-funded warrant forms, and officer certifications

Financial Data and Key Metrics Changes - Research and development expenses increased to $20.1 million for Q2 2025 from $15.1 million in Q2 2024, driven by BLA enabling processes and clinical trial activities [53] - General and administrative expenses rose to $8.6 million for Q2 2025 compared to $7.3 million in Q2 2024, primarily due to higher legal and professional fees [53] - Net loss for Q2 2025 was $26.9 million or $0.09 per share, compared to a net loss of $20.9 million or $0.09 per share for Q2 2024 [54] - As of June 30, 2025, the company had $312.8 million in cash and cash equivalents, reflecting gross proceeds of $230 million from a follow-on financing [54][55] Business Line Data and Key Metrics Changes - The REVEAL pivotal trial for TATIA-102 has commenced site activation, with patient enrollment expected to begin in Q4 2025 [11][12] - In the Part A data of the REVEAL trials, all 10 patients treated with TATIA-102 gained or regained one or more developmental milestones, indicating a 100% response rate [15][32] Market Data and Key Metrics Changes - Rett syndrome affects an estimated 15,000 to 20,000 patients across the U.S., Europe, and the UK, highlighting the market potential for TATIA-102 [12][13] Company Strategy and Development Direction - The company aims to redefine the treatment landscape for Rett syndrome with TATIA-102, a gene therapy targeting the genetic root cause of the disease [13][14] - The pivotal trial design focuses on the developmental plateau population, where the likelihood of achieving developmental milestones is approximately zero percent after age six [15][27] - The company has established a two-study approach to generate safety and efficacy data across the broad Rett syndrome population [19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made towards the potential registration of TATIA-102, with a clear path forward following alignment with the FDA [11][57] - The company anticipates reporting new supplemental clinical data from Part A of the REVEAL trials in Q4 2025, supporting the therapeutic impacts of TATIA-102 [57] Other Important Information - The company completed a public follow-on offering that extended its cash runway into 2028 [16] - The pivotal trial will evaluate the percentage of patients in the developmental plateau population who gain or regain developmental milestones, with each patient serving as their own control [20][22] Q&A Session Summary Question: Is the 100% response rate for the pivotal trial primary endpoint in Part A the bar for Part B? - Management noted that while the results in Part A are compelling, maintaining a 100% standard is difficult. The statistical plan for Part B will use a null hypothesis of 6.7% for milestone gains, which is significantly lower than the observed results in Part A [59][60] Question: Are there specific time points for milestone occurrences? - Management indicated that improvements are seen over time, with gains typically occurring at most follow-up visits. Some critical gains may not be captured in the milestone assessment but still represent significant improvements in daily functioning [63][66] Question: How does the gene therapy differentiate from competitors? - The company emphasized its unique approach in defining clinical efficacy through meaningful functional gains, supported by a comprehensive natural history dataset. This sets it apart from competitors and aligns with regulatory expectations [69][72] Question: Will there be pushback on using interim readouts for BLA filing? - Management confirmed that the interim analysis approach was discussed with the FDA and is based on robust data from Part A, which supports the primary endpoint at both six and twelve months [79][82] Question: What to expect from the new supplemental REVEAL Part A data in Q4? - Management anticipates sharing additional data points, including video documented milestones and their impact on daily living, at both a medical conference and a company update [84][85]

[Report Highlights and Corporate Update](index=1&type=section&id=Report%20Highlights%20and%20Corporate%20Update) The company advanced its TSHA-102 program for Rett syndrome and extended its cash runway into 2028 with a successful financing - Commenced site activation for the REVEAL pivotal trial for Rett syndrome, with patient enrollment anticipated to begin in **Q4 2025**, following a No Objection Letter from Health Canada and feedback from the FDA[1](index=1&type=chunk)[3](index=3&type=chunk) - TSHA-102 continues to be **generally well-tolerated**, with no treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) in the 12 patients treated in Part A of the Phase 1/2 trials[1](index=1&type=chunk)[7](index=7&type=chunk) - Data from the REVEAL Phase 1/2 trials showed a **100% response rate** for the pivotal trial's primary endpoint, which is the gain or regain of at least one developmental milestone[1](index=1&type=chunk) - Completed a public follow-on offering with gross proceeds of **$230 million**, extending the company's cash runway into **2028**[1](index=1&type=chunk)[7](index=7&type=chunk)[8](index=8&type=chunk) [TSHA-102 Program Highlights](index=2&type=section&id=TSHA-102%20Program%20Highlights) The company finalized the REVEAL pivotal trial design for TSHA-102 following alignment with the FDA on key study parameters - **REVEAL Pivotal Trial Design:** - **Study Type:** Single-arm, open-label, with each patient as their own control - **Dose:** Single high dose of TSHA-102 (1x10¹⁵ total vector genomes) via lumbar intrathecal injection - **Patient Population:** 15 females aged 6 to <22 years in the developmental plateau stage of Rett syndrome - **Primary Endpoint:** Response rate, defined as the percentage of patients who gain or regain at least one developmental milestone from a predefined list of 28 - **Analysis:** A 12-month primary analysis and a 6-month interim analysis are planned[4](index=4&type=chunk) - The FDA has provided written alignment on an **extrapolation approach** for a safety-focused study in females aged 2 to <6 years, which is intended to enable access for a broader patient population[4](index=4&type=chunk) - Data presented at the IRSF Scientific Meeting highlighted **positive clinical results**, caregiver research supporting the trial's primary endpoint, and preclinical data on biodistribution[7](index=7&type=chunk) [Financial Performance](index=3&type=section&id=Financial%20Performance) The company reported a higher net loss driven by increased R&D expenses but maintained a strong cash position Q2 2025 vs Q2 2024 Performance | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Research & Development Expenses | $20.1M | $15.1M | +$5.0M | | General & Administrative Expenses | $8.6M | $7.3M | +$1.3M | | Net Loss | $26.9M | $20.9M | +$6.0M | | Net Loss Per Share | $0.09 | $0.09 | $0.00 | - The increase in R&D expenses was primarily driven by BLA-enabling manufacturing initiatives, REVEAL clinical trial activities, and higher compensation from increased headcount[5](index=5&type=chunk) - As of June 30, 2025, the company had **$312.8 million in cash and cash equivalents**, which is expected to support operations into **2028**[8](index=8&type=chunk) [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Operating expenses of $28.7 million resulted in a net loss of $26.9 million for the three months ended June 30, 2025 | (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Revenue | $1,986 | $1,112 | | Total operating expenses | $28,739 | $22,411 | | Loss from operations | $(26,753) | $(21,299) | | **Net loss** | **$(26,882)** | **$(20,928)** | | Net loss per common share | $(0.09) | $(0.09) | [Condensed Consolidated Balance Sheet Data](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheet%20Data) Total assets grew to $333.3 million, driven by a significant increase in cash and cash equivalents to $312.8 million | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Cash and cash equivalents** | **$312,761** | **$139,036** | | Total current assets | $316,264 | $142,130 | | **Total assets** | **$333,331** | **$160,364** | | Total current liabilities | $25,349 | $26,227 | | **Total liabilities** | **$84,604** | **$88,839** | | **Total stockholders' equity** | **$248,727** | **$71,525** | [Outlook and Corporate Information](index=3&type=section&id=Outlook%20and%20Corporate%20Information) The company outlines key upcoming milestones for its lead candidate TSHA-102, an AAV9-based gene therapy for Rett syndrome - **Anticipated Milestones for Q4 2025:** - Begin patient enrollment for the REVEAL pivotal trial - Report new supplemental clinical data from Part A of the REVEAL Phase 1/2 trials[7](index=7&type=chunk) - TSHA-102 is an investigational gene therapy for Rett syndrome that uses a novel **miRARE technology** to regulate MECP2 gene expression and has received **Regenerative Medicine Advanced Therapy (RMAT)**, **Fast Track**, and **Orphan Drug** designations from the FDA[10](index=10&type=chunk) - Rett syndrome is a rare genetic disorder affecting an estimated **15,000 to 20,000 patients** in the U.S., EU, and U.K., with no currently approved therapies that treat the genetic root cause[11](index=11&type=chunk)

Core Insights - Taysha Gene Therapies has commenced site activation for the REVEAL pivotal trial, with patient enrollment expected to begin in Q4 2025 following regulatory feedback from Health Canada and the FDA [1][3][4] - The TSHA-102 treatment has shown a 100% response rate in the primary endpoint of gaining or regaining developmental milestones in the Phase 1/2 trials, indicating strong therapeutic potential [1][5][10] - The company has successfully raised $230 million through a public follow-on offering, extending its cash runway into 2028 [1][8] Company Updates - Taysha Gene Therapies is focused on advancing AAV-based gene therapies for severe monogenic diseases, particularly targeting Rett syndrome with its lead product TSHA-102 [2][12] - The REVEAL pivotal trial is designed as a single-arm, open-label study, with a primary endpoint assessing the percentage of patients who gain or regain developmental milestones [4][5] - The company has received various designations from the FDA, including Regenerative Medicine Advanced Therapy and Fast Track, which facilitate the development of TSHA-102 [10][12] Financial Performance - For Q2 2025, Taysha reported a net loss of $26.9 million, compared to a net loss of $20.9 million in Q2 2024, with research and development expenses increasing to $20.1 million [7][15] - Cash and cash equivalents as of June 30, 2025, were $312.8 million, bolstered by the recent follow-on offering [8][16] - General and administrative expenses rose to $8.6 million in Q2 2025, primarily due to higher legal and professional fees [6][15]