Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy and a 2023 Corporate Outlook
January 31 2023 - 4:01PM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric,
clinical -stage gene therapy company focused on developing and
commercializing AAV-based gene therapies for the treatment of
monogenic rare diseases of the central nervous system (CNS), today
provided an update on the TSHA-120 program in giant axonal
neuropathy (GAN) and a corporate outlook for 2023.
“We expect to deliver on several key milestones in 2023,
including the generation of first-in-human adult clinical data in
Rett syndrome, CTA submission to MHRA to enable initiation of our
pediatric Rett syndrome program and submission of an IND for Rett
syndrome in the U.S. to further expand our clinical study
footprint. For our GAN program, we received the formal FDA meeting
minutes and recently submitted follow up questions to clarify some
of their recommendations including the feasibility of a proposed
study design and the totality of evidence required for BLA
submission. Their feedback will help inform next steps for the
program in this ultra-rare indication with no approved treatments,”
said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha.
“I believe that the operational, structural and personnel actions
recently implemented position us well to execute across our
near-term milestones and deliver on our commitments to key
stakeholders, especially
patients.”
Clinical Program Updates
TSHA-120 in GAN:
- Receipt of formal written meeting
minutes from FDA in January 2023 following completion of Type B
end-of-Phase 2 meeting
- Overall approach to manufacturing of
pivotal/to-be marketed product deemed appropriate pending review of
a planned submission of Chemistry, Manufacturing, and Controls
(CMC) data package for TSHA-120
- FDA acknowledged MFM32 as an
acceptable endpoint with a recommendation to dose additional
patients in a double-blind, placebo-controlled design to support
BLA submission
- Awaiting response from FDA on follow
up questions the Company submitted on recommended design and
totality of evidence required for BLA submission
TSHA-102 in Rett syndrome:
- Dosing of the first adult patient
with Rett syndrome anticipated in H1 2023
- Initial available clinical data for
TSHA-102 in the adult Rett syndrome study expected in H1 2023 with
planned quarterly updates on available clinical data primarily on
safety from the adult study thereafter
- Company anticipates submission of a
CTA to UK MHRA for TSHA-102 in pediatric patients with Rett
syndrome in mid-2023
- Company plans to submit an IND
application for Rett syndrome to FDA in H2 2023
Corporate Updates
- Operational, structural and
personnel changes implemented following thorough business review to
enhance execution
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today
at 4:30 pm ET to provide regulatory feedback from FDA on the
GAN program and 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 13736009. The
live webcast and replay may be accessed by visiting Taysha’s
website
at https://ir.tayshagtx.com/news-events/events-presentations.
An archived version of the webcast will be available on the website
for 30 days.
About Taysha Gene
Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to
eradicate monogenic CNS disease. With a singular focus on
developing curative medicines, we aim to rapidly translate our
treatments from bench to bedside. We have combined our team’s
proven experience in gene therapy drug development and
commercialization with the world-class UT Southwestern Gene Therapy
Program to build an extensive, AAV gene therapy pipeline focused on
both rare and large-market indications. Together, we leverage our
fully integrated platform—an engine for potential new cures—with a
goal of dramatically improving patients’ lives. More information is
available at 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 the
potential of our product candidates, such as TSHA-120 and TSHA-102
and including our preclinical 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, the potential for these product
candidates to receive regulatory approval from the FDA or
equivalent foreign regulatory agencies, and whether, if approved,
these product candidates will be successfully distributed and
marketed, the potential market opportunity for these product
candidates, our corporate growth plans and the impacts of our
corporate operational, structural and personnel changes.
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, 2021 and
our Quarterly Report on Form 10-Q for the quarter ended September
30, 2022, 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. Such risks may
be amplified by the impacts of the COVID-19 pandemic. 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:Kimberly Lee, D.O. Chief
Corporate Affairs OfficerTaysha Gene
Therapiesklee@tayshagtx.com
Media Contact:Carolyn HawleyEvoke
Canalecarolyn.hawley@evokegroup.com
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