Taysha Gene Therapies Provides Update on Deprioritized Pipeline Programs
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Insights
The strategic realignment of Taysha Gene Therapies' pipeline, including the transfer of intellectual property for several deprioritized programs, is a significant move that could have implications for the company's financial health. By terminating their previous loan and entering into a new agreement that extends their cash runway into 2026, Taysha is signaling a strengthened financial position. This extended runway could provide the company with more time to focus on their lead programs without the immediate pressure of fundraising.
From a financial perspective, the transfer of rights for programs like TSHA-120 to NINDS and TSHA-101 back to Queen's University may alleviate some of the company's R&D expenses while potentially providing future milestone payments or royalties if these programs are successful under new stewardship. For investors, this could mean a more streamlined company with a focused approach on its most promising assets, possibly leading to a more efficient use of capital and a clearer path to profitability.
In the biotechnology sector, the prioritization of product pipelines is a crucial aspect of strategic management, particularly for clinical-stage companies like Taysha. The focus on AAV-based gene therapies for CNS diseases is a high-stakes area given the complexity of these conditions and the significant unmet medical needs. Taysha's decision to deprioritize certain programs reflects an industry-wide trend where companies often have to make tough choices to concentrate resources on the most promising therapies.
For industry stakeholders, the transfer of investigational drugs and IP to institutions such as NINDS and Queen's University is noteworthy. It suggests a collaborative approach to drug development that could lead to shared risks and benefits. This could potentially accelerate the development of treatments for rare diseases like GAN and GM2 gangliosidosis, as academic and research institutions often have specialized expertise and access to patient populations for clinical trials.
The update on Taysha's deprioritized pipeline programs is of particular interest to the medical research community. The transfer of the IND application for TSHA-120 to NINDS could be a pivotal step for the continuation of research in giant axonal neuropathy (GAN), a rare and often fatal neurological disorder. This move could ensure that the investigational therapy continues to be evaluated in clinical trials, which is essential for potential future treatment options.
Similarly, the transfer of rights for TSHA-101 back to Queen's University could have positive implications for research into GM2 gangliosidosis. As universities often have the capability to pursue research objectives that are not immediately commercially viable, this could lead to advancements in understanding and treating this rare genetic disorder. The provision of TSHA-118 to RUSH University Medical Center for an individual-patient IND request also demonstrates Taysha's commitment to supporting patient access to investigational treatments, which is a critical aspect of rare disease research.
DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.
Taysha has been working to find ways to advance its deprioritized programs. On November 13, 2023, Taysha terminated its existing loan and security agreement and entered into a new loan and security agreement that provides consent to allow the Company to transfer intellectual property (IP) for several deprioritized programs to third parties in a more efficient manner. The Company’s new loan and security agreement also extended its cash runway into 2026.
Recent progress on previously deprioritized pipeline programs includes:
- TSHA-120: The Company initiated the transfer of the United States Food and Drug Administration Investigational New Drug (IND) application and investigational clinical trial material for TSHA-120 in giant axonal neuropathy (GAN) to clinical trial collaborator National Institute of Neurological Disorders and Stroke (NINDS), creating an opportunity for continued clinical evaluation of TSHA-120 in GAN. Additionally, the Company entered discussions with the originating advocacy organization regarding TSHA-120 in an effort to transfer rights back to the advocacy organization to move the program forward.
- TSHA-101: The Company transferred rights back to Queen’s University (Queen’s) for TSHA-101 in GM2 gangliosidosis, resulting in Queen’s regaining exclusive IP to the program.
- TSHA-104 and TSHA-112: The Company transferred rights back to the originating institution for select programs, including TSHA-104 in SURF1-associated Leigh syndrome and TSHA-112 in APBD.
- TSHA-118: The Company provided investigational clinical trial material for TSHA-118 in CLN1 to support an individual-patient investigator-initiated IND request from RUSH University Medical Center for the treatment of a patient with CLN1 disease.
“Today’s announcement demonstrates meaningful progress to advance important development work for several deprioritized programs. Creating options for these programs has been a focus since the Company completed a management change in December 2022, and the new loan and security agreement afforded the flexibility to implement certain opportunities,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “As we continue to focus on advancing our lead TSHA-102 program for the treatment of Rett syndrome, we are pleased that we can ensure these programs are provided to the right advocates, clinicians and scientific experts who can potentially move these programs forward for the benefit of patients.”
The Company continues to explore potential partnerships and opportunities for its other deprioritized programs to help support their further potential development.
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 statements concerning Taysha’s cash runway and potential partnerships and opportunities for Taysha’s deprioritized programs. 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, 2022, and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, both of which are 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.
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
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