Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q4 2022 Earnings Conference Call March 28, 2023 4:30 PM ET Company Participants Hayleigh Collins - Director and Head of Corporate Communications Sean Nolan - Chief Executive Officer Sukumar Nagendran - President and Head of Research and Development Kamran Alam - Chief Financial Officer Conference Call Participants Whitney Ijem - Canaccord Genuity Jack Allen - Robert W. Baird & Co. Silvan Tuerkcan - JMP Securities Yun Zhong - BTIG David Hoang - SMBC Nikko Securities ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ WASHINGTON, DC 20549 FORM 10-K Commission File Number: 001-39536 Taysha Gene Therapies, Inc. (Exact Name of Registrant as Specified in its Charter) | Delaware | 84-31995 ...

Financial Data and Key Metrics Changes - Research and development expenses decreased to $16.4 million for Q3 2022 from $39.5 million in Q3 2021, a reduction of $23.1 million attributed to lower costs in various areas [37] - General and administrative expenses were $8.7 million for Q3 2022, down from $11.2 million in Q3 2021, reflecting a decrease of approximately $2.5 million [38] - Net loss for Q3 2022 was $26.3 million or $0.64 per share, compared to a net loss of $51.2 million or $1.35 per share in Q3 2021 [39] - As of September 30, 2022, cash and cash equivalents stood at $34.3 million, excluding a recent $50 million investment from Astellas and $25.6 million from a follow-on offering [40] Business Line Data and Key Metrics Changes - The GAN program (TSHA-120) demonstrated clinically meaningful slowing of disease progression across all therapeutic dose cohorts, with effects observed up to five years post-dosing [21] - TSHA-102 for Rett syndrome is the first gene therapy in clinical development, with preclinical data showing significant improvements in survival and behavior in Rett knockout mice [28][30] Market Data and Key Metrics Changes - The partnership with Astellas includes a $50 million investment for 15% of Taysha's outstanding shares, highlighting the market opportunity for TSHA-120 and TSHA-102 [13] - The company anticipates updates on regulatory pathways and clinical data in early 2023, which could impact market perception and stock performance [19][42] Company Strategy and Development Direction - The strategic investment from Astellas is aimed at supporting the development of TSHA-120 for GAN and TSHA-102 for Rett syndrome, indicating a focus on gene therapy for serious genetic diseases [11][12] - The company plans to leverage Astellas' expertise in gene therapy to enhance its capabilities and expedite the development of its product candidates [16][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming FDA meeting to discuss the pathway to a BLA filing for TSHA-120, with expectations for regulatory updates in January 2023 [42] - The company is optimistic about the potential for its gene therapies to address significant unmet medical needs in neurodegenerative diseases [87] Other Important Information - The company has outlined possible scenarios for regulatory approval in Europe and the U.S., with a focus on achieving conditional and accelerated approvals based on existing data [25][26] - TSHA-102 has received orphan drug and rare pediatric disease designations from the FDA, enhancing its development prospects [34] Q&A Session Summary Question: Does Astellas have experience in manufacturing AAV9 ACE genetic medicine? - Management noted that while Astellas' experience is primarily with AAV8, the manufacturing processes are similar, allowing for effective collaboration [48][49] Question: What are the specified outcomes from the FDA meeting for Astellas to opt in? - Management indicated that Astellas is aware of the range of possible outcomes and is focused on the robustness of the data set rather than specific outcomes [51][53] Question: How will the registrational trial design differ from the ongoing Phase 1/2 in Rett syndrome? - Management stated that the registrational study will focus on the pediatric population, with similar assessment buckets but tailored endpoints for children [56][61] Question: What is the expected translatability of outcomes from adult patients to pediatrics? - Management explained that dosing for CNS delivered drugs is different, and after age four, the same dose is expected for both adults and children [67][69] Question: What is the bar for success in the initial clinical data for Rett syndrome? - Management emphasized that safety is paramount, and preliminary efficacy will be assessed across various endpoints, with expectations for better outcomes in children [72][78] Question: What triggers the start of the pediatric study? - Management indicated that patient experience and follow-up in the adult study will be the main triggers for initiating the pediatric study [84][87]

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q2 2022 Results Conference Call August 11, 2022 8:00 AM ET Company Participants Dr. Kimberly Lee - Chief Corporate Affairs Officer RA Session - President, Founder, and Chief Executive Officer Dr. Suyash Prasad - Chief Medical Officer and Head, R&D Kamran Alam - Chief Financial Officer Dr. Frederick Porter - Chief Technical Officer Conference Call Participants Joon Lee - Jo Securities Gil Blum - Needham & Company Mike Ulz - Morgan Stanley Eun Yang - Jefferies Jack Al ...

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, 2022 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 ...

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q1 2022 Earnings Conference Call May 16, 2022 8:00 AM ET Company Participants Kimberly Lee - Chief Corporate Affairs Officer RA Session II - President, Founder, and Chief Executive Officer Suyash Prasad - Chief Medical Officer and Head, R&D Kamran Alam - Chief Financial Officer Conference Call Participants Gil Blum - Needham & Company Elizabeth Webster - Goldman Sachs Kevin DeGeeter - Oppenheimer & Company Jack Allen - Baird Yun Zhong - BTIG Laura Chico - Wedbush Se ...

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, 2022 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 84-3199 ...

Taysha Gene Therapies (NASDAQ:TSHA) Q4 2021 Earnings Conference Call March 31, 2022 8:00 AM ET Company Participants RA Session II – President, Chief Executive Officer & Founder Kimberly Lee – Corporate Affairs Officer Suyash Prasad – Chief Medical Officer & Head, Research & Development Kamran Alam – Chief Financial Officer Conference Call Participants Elizabeth Webster – Goldman Sachs Joon Lee – Truist Securities Mike Ulz – Morgan Stanley Jack Allen – Baird Kevin DeGeeter – Oppenheimer Gil Blum – Needham an ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 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 84-3199512 ( ...

PART I. FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for Taysha Gene Therapies, Inc. as of September 30, 2021, reflecting a significant increase in operating losses and cash used in operations driven by escalated research and development activities [Condensed Consolidated Balance Sheets](index=3&type=section&id=Balance%20Sheets) As of September 30, 2021, the company's total assets were $241.0 million, a decrease from $258.9 million at year-end 2020, mainly due to a reduction in cash and cash equivalents, while total liabilities increased substantially to $100.6 million from $7.6 million, primarily driven by a new term loan and a build-to-suit lease liability, consequently decreasing total stockholders' equity to $140.4 million from $251.3 million Condensed Consolidated Balance Sheet Highlights (in thousands) | | September 30, 2021 | December 31, 2020 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $188,785 | $251,253 | | Total current assets | $197,170 | $257,879 | | Total assets | $241,042 | $258,881 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $43,214 | $7,129 | | Build-to-suit lease liability | $26,607 | $— | | Term loan | $27,812 | $— | | Total liabilities | $100,648 | $7,579 | | Total stockholders' equity | $140,394 | $251,302 | | Total liabilities and stockholders' equity | $241,042 | $258,881 | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Statements%20of%20Operations) For the three months ended September 30, 2021, the company reported a net loss of $51.2 million, compared to a $15.0 million loss in the same period of 2020, with the nine-month net loss significantly increasing to $124.1 million from $41.7 million year-over-year, primarily due to a substantial rise in research and development expenses to $94.0 million from $19.6 million Operating Results (in thousands, except per share data) | | For the Three Months Ended Sep 30, | For the Nine Months Ended Sep 30, | | :--- | :--- | :--- | | | **2021** | **2020** | **2021** | **2020** | | Research and development | $39,528 | $11,057 | $94,025 | $19,633 | | General and administrative | $11,153 | $3,984 | $29,518 | $5,002 | | **Loss from operations** | **$(50,681)** | **$(15,041)** | **$(123,543)** | **$(24,635)** | | **Net loss** | **$(51,187)** | **$(15,042)** | **$(124,137)** | **$(41,693)** | | Net loss per common share | $(1.35) | $(1.28) | $(3.31) | $(3.73) | [Condensed Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit)](index=5&type=section&id=Statements%20of%20Convertible%20Preferred%20Stock%20and%20Stockholders'%20Equity%20(Deficit)) These statements detail the changes in stockholders' equity, showing a decrease from $251.3 million to $140.4 million for the nine months ended September 30, 2021, primarily driven by a net loss of $124.1 million, partially offset by $13.2 million in stock-based compensation Changes in Stockholders' Equity for the Nine Months Ended Sep 30, 2021 (in thousands) | Description | Amount | | :--- | :--- | | Balance as of December 31, 2020 | $251,302 | | Stock-based compensation | $13,229 | | Net loss | $(124,137) | | **Balance as of September 30, 2021** | **$140,394** | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Statements%20of%20Cash%20Flows) For the nine months ended September 30, 2021, net cash used in operating activities significantly increased to $76.8 million from $10.9 million in the prior-year period, with net cash used in investing activities at $13.0 million, while financing activities provided $30.0 million from a new term loan, resulting in an overall decrease of $59.8 million in cash, cash equivalents, and restricted cash Summary of Cash Flows for the Nine Months Ended Sep 30 (in thousands) | | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(76,784) | $(10,881) | | Net cash used in investing activities | $(13,034) | $(3,031) | | Net cash provided by financing activities | $29,978 | $292,546 | | **Net (decrease) increase in cash** | **$(59,840)** | **$278,634** | [Notes to Financial Statements](index=8&type=section&id=Notes%20to%20Financial%20Statements) The notes provide detailed explanations of the company's financial status and operations, including its liquidity position, significant transactions like a new term loan and acquisition of TSHA-120 rights, and a subsequent 'at the market' offering agreement - The company had cash and cash equivalents of **$188.8 million** as of September 30, 2021, which management believes is sufficient to fund planned operations for at least the next twelve months[35](index=35&type=chunk) - In August 2021, the company entered into a Loan and Security Agreement with Silicon Valley Bank for a term loan facility of up to **$100.0 million**, and drew **$30.0 million** on the closing date[49](index=49&type=chunk) - In March 2021, the company acquired exclusive worldwide rights to **TSHA-120** for the treatment of Giant Axonal Neuropathy (GAN) from Hannah's Hope Fund for an upfront payment of **$5.5 million**[71](index=71&type=chunk)[72](index=72&type=chunk) - In October 2021, the company entered into a Sales Agreement for an 'at the market' offering to sell up to **$150.0 million** of its common stock[112](index=112&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=22&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition, results of operations, and business outlook, highlighting the advancement of a portfolio of 27 gene therapy candidates for CNS diseases, significant increases in operating losses to $124.1 million due to higher R&D and G&A expenses, and the belief that existing cash and term loan access will fund operations into the second half of 2023 [Overview and Recent Developments](index=22&type=section&id=Overview%20and%20Recent%20Developments) Taysha, a patient-centric gene therapy company focused on CNS diseases, is advancing a portfolio of 27 product candidates, recently acquiring exclusive worldwide rights to **TSHA-120** for GAN and securing a term loan agreement for up to **$100 million**, drawing an initial **$30 million** to bolster its financial position, while expecting continued significant operating losses as it advances its pipeline - The company is advancing a deep portfolio of **27 gene therapy product candidates** for monogenic CNS diseases, with options to acquire four additional programs[117](index=117&type=chunk)[123](index=123&type=chunk) - In April 2021, the company acquired exclusive worldwide rights to **TSHA-120**, a clinical-stage AAV9 gene therapy for giant axonal neuropathy (GAN)[117](index=117&type=chunk) - In August 2021, the company entered into a term loan agreement for up to **$100 million** and drew an initial **$30 million**[120](index=120&type=chunk) [Our Pipeline](index=24&type=section&id=Our%20Pipeline) The company's pipeline targets a broad range of neurological indications, with detailed updates on key programs including **TSHA-120** for GAN showing dose-dependent slowing of disease progression, **TSHA-102** for Rett Syndrome demonstrating regulated gene expression and improved safety in preclinical models, and other programs like **TSHA-101** and **TSHA-118** advancing with expected clinical data - **TSHA-120** (GAN): Phase 1/2 trial data showed a **statistically significant improvement** versus historical controls, with the **1.8x10¹⁴ vg dose halting disease progression**, and a Bayesian analysis confirmed these findings[144](index=144&type=chunk)[148](index=148&type=chunk)[149](index=149&type=chunk) - **TSHA-102** (Rett Syndrome): Preclinical data published in 'Brain' showed the **miRARE platform extended knockout survival by 56%** and **attenuated behavioral side effects** compared to unregulated constructs, with an **IND/CTA submission is planned for November 2021**[175](index=175&type=chunk)[176](index=176&type=chunk)[177](index=177&type=chunk) - **TSHA-101** (GM2 Gangliosidosis): A **Phase 1/2 trial is ongoing** in Canada, with **preliminary clinical safety and HEX A enzyme activity data expected in December 2021**[117](index=117&type=chunk)[166](index=166&type=chunk) - **TSHA-118** (CLN1 Disease): The company plans to **initiate a Phase 1/2 clinical trial by year-end 2021** and expects **preliminary clinical data in the first half of 2022**[117](index=117&type=chunk)[173](index=173&type=chunk) [Results of Operations](index=45&type=section&id=Results%20of%20Operations) This section provides a comparative analysis of the company's financial performance, showing that for the nine-month period, R&D expenses increased by **$74.4 million** to $94.0 million due to higher manufacturing costs, employee compensation, and third-party research expenses, while G&A expenses increased by **$24.5 million** to $29.5 million, primarily from increased compensation and professional fees Comparison of Operating Expenses (in thousands) | | For the Nine Months Ended Sep 30, | | :--- | :--- | | | **2021** | **2020** | | Research and development | $94,025 | $19,633 | | General and administrative | $29,518 | $5,002 | | **Total operating expenses** | **$123,543** | **$24,635** | - The **$74.4 million increase** in nine-month R&D expenses was primarily due to a **$29.7 million increase** in manufacturing and raw material costs, a **$24.4 million increase** in employee compensation, and a **$20.3 million increase** in third-party R&D consulting fees[219](index=219&type=chunk) - The **$24.5 million increase** in nine-month G&A expenses was mainly attributable to a **$13.2 million increase** in compensation (including **$6.2 million in stock-based compensation**) and an **$11.3 million increase** in professional fees[220](index=220&type=chunk) [Liquidity and Capital Resources](index=47&type=section&id=Liquidity%20and%20Capital%20Resources) The company has historically funded operations through equity and debt, holding **$191.4 million** in cash, cash equivalents, and restricted cash as of September 30, 2021, and believes existing cash combined with full access to its term loan facility will fund operations **into the second half of 2023**, though **substantial** additional funding will be required for future development and commercialization - As of September 30, 2021, the company had cash, cash equivalents, and restricted cash of **$191.4 million**[225](index=225&type=chunk) - The company believes its existing cash and cash equivalents, along with full access to the term loan facility, will fund operations **into the second half of 2023**[228](index=228&type=chunk) - Future funding requirements are **substantial** and depend on the progress of clinical trials, manufacturing scale-up, and potential commercialization activities[227](index=227&type=chunk)[228](index=228&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=50&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a **smaller reporting company**, Taysha Gene Therapies is **not required to provide the information** requested under this item - The company is a **smaller reporting company** as defined by Rule 12b-2 of the Exchange Act and is **not required to provide the information** under this Item[242](index=242&type=chunk) [Item 4. Controls and Procedures](index=50&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, with the participation of the CEO and CFO, evaluated the company's disclosure controls and procedures and concluded that as of September 30, 2021, these controls were **effective**, with **no material changes** in internal control over financial reporting during the quarter - The Chief Executive Officer and Chief Financial Officer concluded that as of September 30, 2021, the company's disclosure controls and procedures were **effective**[243](index=243&type=chunk) - There were **no changes in internal control over financial reporting** during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[244](index=244&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=52&type=section&id=Item%201.%20Legal%20Proceedings) The company states that it is **not currently subject to any material legal proceedings** - The company is **not currently a party to any legal proceedings that are likely to have a material adverse effect on its business**[248](index=248&type=chunk) [Item 1A. Risk Factors](index=52&type=section&id=Item%201A.%20Risk%20Factors) This section updates previously disclosed risk factors, highlighting a new risk concerning indebtedness from the August 2021 Term Loan Agreement, which contains **restrictive covenants** that could **limit operational flexibility**, and a breach could result in an **event of default** and **acceleration of debt repayment** - The Term Loan Agreement entered into on August 12, 2021, contains various **covenants that limit the company's ability** to, among other things, incur debt, merge, dispose of assets, make investments, and pay dividends[250](index=250&type=chunk)[253](index=253&type=chunk) - A breach of these covenants could result in an **event of default**, allowing lenders to declare all outstanding amounts immediately due and payable[251](index=251&type=chunk) - The ability to draw down an **additional $60.0 million** under the term loan is **subject to meeting specified conditions** or obtaining lender approval, which may not be achieved[252](index=252&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=53&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports **no unregistered sales** of equity securities during the period and confirms there has been **no material change in the planned use of proceeds** from its Initial Public Offering - There were **no recent sales of unregistered equity securities**[255](index=255&type=chunk) - There has been **no material change in the planned use of proceeds** from the company's initial public offering[257](index=257&type=chunk) [Item 3. Defaults Upon Senior Securities](index=53&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is **not applicable** to the company - **Not applicable**[259](index=259&type=chunk) [Item 4. Mine Safety Disclosures](index=53&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is **not applicable** to the company - **Not applicable**[260](index=260&type=chunk) [Item 5. Other Information](index=53&type=section&id=Item%205.%20Other%20Information) The company reports **no information** for this item - **None**[261](index=261&type=chunk) [Item 6. Exhibits](index=54&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the quarterly report, which include the company's Amended and Restated Certificate of Incorporation and Bylaws, the Loan and Security Agreement dated August 12, 2021, and certifications by the Principal Executive Officer and Principal Financial Officer - The exhibits list includes **key corporate documents and the Loan and Security Agreement** from August 12, 2021[264](index=264&type=chunk)