Taysha Gene Therapies Reports Second Quarter 2024 Financial Results and Provides Corporate Update

Rhea-AI Summary

Taysha Gene Therapies (NASDAQ: TSHA) reported Q2 2024 financial results and provided a corporate update. Key highlights include:

• Presented encouraging data from TSHA-102 trials for Rett syndrome in adult and pediatric patients
• Dosed first patient in high-dose cohort of adolescent/adult trial with no serious adverse events
• Raised $76.8 million in June 2024 offering, extending cash runway into Q4 2026
• Q2 2024 net loss of $20.9 million ($0.09 per share)
• Cash and equivalents of $172.7 million as of June 30, 2024

The company plans to report clinical data from high-dose cohorts and update on low-dose cohorts for both REVEAL trials in 1H 2025.

Positive

• Presented encouraging TSHA-102 trial data showing durable improvements in adult and pediatric Rett syndrome patients
• Raised $76.8 million, extending cash runway into Q4 2026
• Received IDMC approval to proceed with high-dose cohort dosing in both trials
• Reduced net loss to $20.9 million in Q2 2024 from $24.6 million in Q2 2023
• Strong cash position of $172.7 million as of June 30, 2024

Negative

• Research and development expenses of $15.1 million in Q2 2024
• General and administrative expenses increased to $7.3 million in Q2 2024 from $6.0 million in Q2 2023
• Net loss of $20.9 million in Q2 2024, despite reduction from previous year

Insights

Financial Analyst

Taysha Gene Therapies' Q2 2024 results show mixed financial signals. The company's R&D expenses decreased by $4.7 million year-over-year, primarily due to a one-time milestone payment in 2023. However, G&A expenses increased by $1.3 million, driven by higher stock-based compensation and professional fees. The net loss narrowed to $20.9 million ($0.09 per share) from $24.6 million ($0.38 per share) in Q2 2023.

Notably, Taysha strengthened its balance sheet with a public offering raising $76.8 million in net proceeds. This extends their cash runway into Q4 2026, providing a solid financial foundation for ongoing clinical trials. With $172.7 million in cash and cash equivalents as of June 30, 2024, Taysha appears well-positioned to fund its operations and key milestones in the near term.

Medical Research Analyst

Taysha's TSHA-102 gene therapy for Rett syndrome is showing promising early results. The low-dose cohort data from both adult and pediatric trials demonstrated durable improvements across multiple clinical domains, including motor skills, communication and autonomic function. Importantly, the treatment appears well-tolerated with no serious adverse events reported.

The advancement to high-dose cohorts in both trials, with IDMC approval, suggests confidence in the therapy's safety profile. The scheduled dosing of additional patients in Q3 2024 and expected data readout in 1H 2025 are critical milestones. These upcoming results, particularly from the high-dose cohorts, will be pivotal in determining TSHA-102's potential efficacy and safety at higher doses, which could be important for its therapeutic impact and regulatory pathway.

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 first (pediatric trial) patient in cohort two, which is scheduled for Q3 2024  

Cohort two (n=3) and cohort one (n=2) data from both trials (adolescent/adult and pediatric) expected in 1H 2025

$76.8 million net proceeds from June 2024 public follow-on offering extends anticipated cash runway into Q4 2026

   Conference call and live webcast today at 8:30 AM Eastern Time

DALLAS, Aug. 12, 2024 (GLOBE NEWSWIRE) --  Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the second quarter ended June 30, 2024, and provided a corporate update.

“In the second quarter, we made strong progress across our TSHA-102 clinical program, including reporting encouraging preliminary data in our pediatric trial and longer-term data in our adolescent and adult trial following the low dose of TSHA-102. We dosed the first patient in the high dose cohort of our adolescent and adult trial, and TSHA-102 was generally well tolerated as of the initial six-week assessment. Subsequently, we received IDMC approval to proceed with dosing the second adolescent/adult and first pediatric patient in the high dose cohort of our REVEAL trials. This progress enables us to build on the promising low dose data that demonstrated an encouraging safety profile and improvements across consistent clinical domains impacting daily activities in adult and pediatric patients suffering from Rett syndrome,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “We remain focused on clinical trial execution and data collection that will further inform discussions with regulatory authorities on the development plan for the next phase of our studies.”

Mr. Nolan continued, “Dosing of the second patient in our adolescent and adult trial and the first patient in our pediatric trial in the high dose cohort is scheduled for the third quarter of 2024. We are moving toward reporting cohort-based updates with more mature data sets to provide more fulsome updates on our clinical data. In line with this decision, we plan to report clinical data from the high dose cohorts and an update on clinical data from the low dose cohorts in both REVEAL trials in the first half of 2025. With our balance sheet strengthened and runway extended, we believe we are in an excellent position to execute on key value-creating milestones.”

Recent Corporate and Program Highlights

• Completed public follow-on offering with total net proceeds of $76.8 million with anticipated cash runway into the fourth quarter of 2026
  

REVEAL Phase 1/2 Adolescent and Adult Trial (Canada and United States (U.S.)): a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome due to MECP2 loss-of-function mutation.

• Presented longer-term data from cohort one (low dose, n=2) of 5.7x10¹⁴ total vector genomes (vg) at the 2024 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting:
  • Generally well tolerated with no serious adverse events (SAEs) related to TSHA-102 or dose-limiting toxicities (DLTs) as of the 52- and 36-week assessment for patient one and two, respectively
  • Sustained and new improvements across multiple efficacy measures and clinical domains relative to baseline, including fine and gross motor skills, communication/socialization, autonomic function and seizure events, through 52- and 25-weeks post-treatment for patient one and two, respectively
• Dosed the first patient in cohort two (high dose, n=3) of 1x10¹⁵ total vg, and TSHA-102 was generally well tolerated with no SAEs or DLTs as of the initial six-week assessment
• Enrolled the second patient in cohort two and scheduled dosing for the current quarter, following Independent Data Monitoring Committee (IDMC) review of initial six-week clinical data from the first patient dosed in cohort two
  

REVEAL Phase 1/2 Pediatric Trial (U.S., United Kingdom (U.K.) and Canada): an open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females aged 5 to 8 years old with Rett syndrome due to MECP2 loss-of-function mutation.

• Health Canada cleared the pediatric clinical trial application (CTA), enabling expansion of the ongoing U.S. and U.K. REVEAL pediatric trial into Canada
• Presented preliminary data from cohort one (low dose, n=2) of 5.7x10¹⁴ total vg at the 2024 IRSF Rett Syndrome Scientific Meeting:
  • Generally well-tolerated with no SAEs related to TSHA-102 or DLTs as of the 22- and 11-week assessment for patient one and two, respectively
  • Initial improvements across multiple efficacy measures and clinical domains relative to baseline, including fine and gross motor skills, communication/socialization, autonomic function and seizure events, as of 12- and eight-weeks post-treatment for patient one and two, respectively
• Enrolled the first pediatric patient in cohort two (high dose, n=3) of 1x10¹⁵ total vg and scheduled dosing for the current quarter, following IDMC approval to proceed with the Company’s request for early advancement to cohort two after review of the initial six-week safety data from the first patient treated with the high dose of TSHA-102 in the adolescent and adult trial
  

Anticipated Milestones

• REVEAL Adolescent and Adult Trial
  • Dosing of the second patient in cohort two (high dose) scheduled for the third quarter of 2024
  • Safety and efficacy data from cohort two (n=3) and an update on safety and efficacy data from cohort one (n=2) expected in the first half of 2025
    
    
• REVEAL Pediatric Trial
  
  
  • Dosing of the first patient in cohort two (high dose) scheduled for the third quarter of 2024
  • Safety and efficacy data from cohort two (n=3) and an update on safety and efficacy data from cohort one (n=2) expected in the first half of 2025
    

Second Quarter 2024 Financial Highlights

Research and Development Expenses: Research and development expenses were $15.1 million for the three months ended June 30, 2024, compared to $19.8 million for the three months ending June 30, 2023. The $4.7 million decrease was primarily due to a milestone fee payable to Abeona Therapeutics Inc. during the three months ended June 30, 2023, following the dosing of the first patient in the REVEAL Phase 1/2 adolescent and adult trial.

General and Administrative Expenses: General and administrative expenses were $7.3 million for the three months ended June 30, 2024, compared to $6.0 million for the three months ended June 30, 2023. The increase of $1.3 million was primarily due to $0.9 million of higher stock-based compensation expenses and $0.4 million of higher consulting, professional fees and other expenses.

Net loss: Net loss for the three months ended June 30, 2024, was $20.9 million, or $0.09 per share, compared to a net loss of $24.6 million, or $0.38 per share, for the three months ended June 30, 2023.

Cash and cash equivalents: As of June 30, 2024, Taysha had $172.7 million in cash and cash equivalents. Taysha expects that its current cash resources will support planned operating expenses and capital requirements into the fourth quarter of 2026.

Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 8:30 a.m. ET to review its financial and operating results and provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13747741. The live webcast and replay may be accessed by visiting Taysha’s website.

About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.

About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.

About Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, and our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials and reporting data from our clinical trials, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and our current cash resources supporting our planned operating expenses and capital requirements into the fourth quarter of 2026. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (“SEC”) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2023, which is available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

Taysha Gene Therapies, Inc. Condensed Consolidated Statements of Operations (in thousands, except share and per share data)
		For the Three Months Ended June 30,								For the Six Months Ended June 30,
			2024				2023				2024				2023
Revenue		$	1,112			$	2,395			$	4,523			$	7,101
Operating expenses:
Research and development			15,073				19,791				35,730				32,305
General and administrative			7,338				5,988				14,422				14,739
Total operating expenses			22,411				25,779				50,152				47,044
Loss from operations			(21,299	)			(23,384	)			(45,629	)			(39,943	)
Other income (expense):
Change in fair value of warrant liability			195				-				(142	)			-
Change in fair value of term loan			(1,279	)			-				(2,332	)			-
Interest income			1,440				223				3,133				542
Interest expense			(27	)			(1,440	)			(56	)			(2,814	)
Other (expense) income			42				3				37				(5	)
Total other income (expense), net			371				(1,214	)			640				(2,277	)
Net loss		$	(20,928	)		$	(24,598	)		$	(44,989	)		$	(42,220	)
Net loss per common share, basic and diluted		$	(0.09	)		$	(0.38	)		$	(0.19	)		$	(0.66	)
Weighted average common shares outstanding, basic and diluted			232,821,553				64,244,531				232,035,448				63,755,435

Taysha Gene Therapies, Inc. Condensed Consolidated Balance Sheet Data   (in thousands, except share and per share data)
		June 30, 2024				December 31, 2023
ASSETS
Current assets:
Cash and cash equivalents		$	172,743			$	143,940
Restricted cash			449				449
Prepaid expenses and other current assets			3,278				3,479
Assets held for sale			2,000				2,000
Total current assets			178,470				149,868
Restricted cash			2,151				2,151
Property, plant and equipment, net			10,513				10,826
Operating lease right-of-use assets			8,971				9,582
Other non-current assets			288				304
Total assets		$	200,393			$	172,731
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable		$	8,718			$	6,366
Accrued expenses and other current liabilities			11,875				12,284
Deferred revenue			13,583				18,106
Total current liabilities			34,176				36,756
Term loan, net			37,835				40,508
Operating lease liability, net of current portion			18,134				18,953
Other non-current liabilities			1,380				1,577
Total liabilities			91,525				97,794
Stockholders' equity
Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized, and no shares issued and outstanding as of June 30, 2024, and December 31, 2023			—				—
Common stock, $0.00001 par value per share; 400,000,000 shares authorized and 201,381,450 and 186,960,193 issued and outstanding as of June 30, 2024, and December 31, 2023, respectively			2				2
Additional paid-in capital			664,457				587,942
Accumulated other comprehensive income			2,405				—
Accumulated deficit			(557,996	)			(513,007	)
Total stockholders’ equity			108,868				74,937
Total liabilities and stockholders' equity		$	200,393			$	172,731

Company Contact:
Hayleigh Collins
Director, Head of Corporate Communications and Investor Relations
Taysha Gene Therapies, Inc.
hcollins@tayshagtx.com

Media Contact:
Carolyn Hawley
Inizio Evoke
Carolyn.hawley@inizioevoke.com

FAQ

What were Taysha Gene Therapies' (TSHA) Q2 2024 financial results?

Taysha reported a net loss of $20.9 million ($0.09 per share) for Q2 2024, compared to a net loss of $24.6 million ($0.38 per share) in Q2 2023. The company had $172.7 million in cash and cash equivalents as of June 30, 2024.

What progress has Taysha (TSHA) made in its TSHA-102 clinical trials for Rett syndrome?

Taysha presented encouraging data from low-dose cohorts in both adult and pediatric trials, showing durable improvements across clinical domains. The company has also dosed the first patient in the high-dose cohort of the adolescent/adult trial and received IDMC approval to proceed with high-dose cohort dosing in both trials.

When does Taysha (TSHA) expect to report clinical data from its REVEAL trials?

Taysha plans to report clinical data from the high-dose cohorts and an update on clinical data from the low-dose cohorts in both REVEAL trials in the first half of 2025.

How has Taysha (TSHA) strengthened its financial position?

Taysha completed a public follow-on offering in June 2024, raising net proceeds of $76.8 million. This has extended the company's anticipated cash runway into the fourth quarter of 2026.