Taysha Gene Therapies, Inc. (Nasdaq: TSHA), 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 reported financial results for
the first quarter ended March 31, 2023, and provided a corporate
update.
“We continue to make significant progress with
our two lead clinical programs and remain on track to deliver on
multiple key milestones, including the generation of first-in-human
clinical data for TSHA-102 in adult patients with Rett syndrome,
the submission of a CTA to the MHRA to initiate expansion of
TSHA-102 in pediatric patients, the submission of an IND
application to the FDA for TSHA-102, and obtaining further clarity
from the FDA on the regulatory path forward for TSHA-120 in GAN,”
said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha.
“Screening is completed, and dosing is now scheduled for our first
potential patient in the adult Rett syndrome study. For GAN, our
comprehensive analyses of the totality of data for TSHA-120
continues to be encouraging and includes compelling findings with
potential to further support a regulatory path forward.”
Sukumar Nagendran, M.D., President, and Head of
R&D added, “We plan to seek a formal meeting with the FDA to
discuss the totality of findings from the functional, biological,
and electrophysiological assessments of TSHA-120 in GAN,
anticipated in the third quarter of this year. In the near term, we
look forward to hosting an R&D Day in June where we will
overview the GAN disease state and share the comprehensive
analyses, as well as provide an update on our Rett program. For
TSHA-102, new preclinical data supporting the efficacy and safety
of TSHA-102 and the miRARE technology in Rett syndrome will be
presented as part of a poster presentation at the upcoming ASGCT
conference. We believe that the clinical and preclinical data
generated to date across our Rett syndrome and GAN programs
reinforce our gene therapy approach, and the therapeutic potential
to address severe unmet needs in monogenic central nervous system
disease.”
Recent Corporate Highlights
TSHA-102 in Rett
syndrome: a self-complementary intrathecally
delivered AAV9 gene transfer therapy in clinical evaluation for
Rett syndrome, a rare genetic neurodevelopmental disorder caused by
mutations in the X-linked MECP2 gene. TSHA-102 utilizes a novel
miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed
to regulate cellular MECP2 expression. TSHA-102 has received Orphan
Drug and Rare Pediatric Disease designations from the FDA and has
been granted Orphan Drug designation from the European
Commission.
- Phase 1/2 REVEAL
trial in adult patients with Rett syndrome
- Completed screening and scheduled
dosing for first potential adult patient with dosing anticipated in
Q2 2023
- Initial available Phase 1/2 clinical
data, primarily on safety, expected in Q2 2023, with planned
quarterly updates on available clinical data thereafter
- Continued dosing of adult patients
with Rett syndrome in the REVEAL trial in H2 2023
- CTA submission
to UK MHRA for TSHA-102 in pediatric patients with Rett syndrome
anticipated in mid-2023
- IND application
submission to U.S. FDA for Rett syndrome expected in H2 2023
- New preclinical
data for TSHA-102 in Rett syndrome to be presented as a poster
presentation at the upcoming ASGCT 26th Annual Meeting on Friday,
May 19 at 12-2 PM PT; these data and available clinical data from
Phase 1/2 REVEAL trial will be presented in upcoming R&D Day in
June
TSHA-120 for giant axonal neuropathy
(GAN): a self-complimentary intrathecally delivered AAV9
gene therapy in clinical evaluation for GAN, an ultra-rare
inherited genetic neurodegenerative disorder with no approved
treatments. TSHA-120 has received Orphan Drug and Rare Pediatric
Disease designations from the FDA and has been granted Orphan Drug
designation from the European Commission.
- Completed CMC
module 3 amendment submission detailing commercial process product
manufacturing and drug comparability analysis; awaiting FDA
feedback
- R&D Day in
June 2023 to overview new findings from totality of data
evaluation, including comprehensive analyses of functional,
biological, and electrophysiological assessments of TSHA-120 in
GAN
- Submission of a
formal meeting request to the FDA in Q2 2023 to discuss alternative
study designs, additional objective measures and regulatory path
forward; formal meeting anticipated in Q3 2023
First Quarter 2023 Financial Highlights
Research and Development
Expenses: Research and development expenses were $12.5
million for the three months ended March 31, 2023, compared to
$38.2 million for the three months ending March 31, 2022. The $25.7
million decrease was due to reduced research and development
compensation as a result of lower headcount of $10.7 million. The
decrease was also due to reduced manufacturing and other raw
material purchases of $7.1 million. We also incurred $6.4 million
reduced expense in non-clinical studies related to translational
and toxicology studies and $1.5 million lower expense in other
research and development activities.
General and Administrative
Expenses: General and administrative expenses were
$8.8 million for the three months ended March 31, 2023, compared to
$11.5 million for the three months ended March 31, 2022. The
decrease of $2.7 million was due to reduced general and
administrative compensation as a result of lower headcount and
reduced consulting and professional fees.
Net loss: Net loss for the
three months ended March 31, 2023 was $17.6 million
or $0.28 per share, as compared to a net loss
of $50.3 million, or $1.32 per share, for the three
months ended March 31, 2022. The net loss for the three months
ended March 31, 2023 was partially offset by revenue
of $4.7 million recognized related to the Astellas
Transactions.
Cash and cash
equivalents: As of March 31, 2023, Taysha had $63.4
million in cash and cash equivalents. Taysha continues to expect
that its current cash resources will support planned operating
expenses and capital requirements into the first quarter of
2024.
Conference Call and Webcast Information
Taysha management will hold a conference call
and webcast today at 4:30 pm ET to review its financial
and operating results and to provide a corporate update. The
dial-in number for the conference call is 855-327-6837
(U.S./Canada) or 631-891-4304 (international). The conference ID
for all callers is 10021767. 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. Together, we leverage our fully integrated platform 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, 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, the forecast of our cash runway and the implementation and
potential impacts of our strategic pipeline prioritization
initiatives. 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, 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. 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.
Taysha Gene Therapies,
Inc.Condensed Consolidated Statements of
Operations(in thousands, except share and per share
data)(Unaudited)
|
For the three months ended March 31, 2023 |
For the three months ended March 31, 2022 |
|
|
|
Revenue: |
|
|
Service Revenue |
$ |
4,706 |
|
$ |
- |
|
Operating
expenses: |
|
|
Research and development |
|
12,514 |
|
|
38,182 |
|
General and administrative |
|
8,751 |
|
|
11,469 |
|
Total operating expenses |
|
21,265 |
|
|
49,651 |
|
Loss from
operations |
|
(16,559 |
) |
|
(49,651 |
) |
Other income
(expense): |
|
|
|
|
|
Interest Income |
|
319 |
|
|
14 |
|
Interest expense |
|
(1,374 |
) |
|
(672 |
) |
Other expense |
|
(8 |
) |
|
(8 |
) |
Total other expense |
|
(1,063 |
) |
|
(666 |
) |
Net loss |
$ |
(17,622 |
) |
$ |
(50,317 |
) |
|
|
|
Net loss per common share,
basic and diluted |
$ |
(0.28 |
) |
$ |
(1.32 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
63,260,905 |
|
|
38,174,717 |
|
|
Taysha Gene Therapies,
Inc.Condensed Consolidated Balance Sheet
Data(in thousands, except share and per share
data)(Unaudited)
|
March 31, 2023 |
|
December 31, 2022 |
|
|
|
|
ASSETS |
|
|
Current assets: |
|
|
Cash and cash equivalents |
$ |
63,425 |
|
$ |
87,880 |
|
Prepaid expenses and other current liabilities |
|
8,933 |
|
|
8,537 |
|
Total current assets |
|
72,358 |
|
|
96,417 |
|
|
|
|
Restricted cash |
|
2,637 |
|
|
2,637 |
|
Property, plant and equipment,
net |
|
14,642 |
|
|
14,963 |
|
Operating lease right-of-use
assets |
|
10,647 |
|
|
10,943 |
|
Other noncurrent assets |
|
1,316 |
|
|
1,316 |
|
Total
assets |
$ |
101,600 |
|
$ |
126,276 |
|
|
|
|
LIABILITIES,
CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS' (DEFICIT)
EQUITY |
|
|
Current liabilities: |
|
|
Accounts payable |
$ |
9,002 |
|
$ |
10,946 |
|
Accrued expenses and other current liabilities |
|
16,602 |
|
|
18,287 |
|
Deferred revenue |
|
28,851 |
|
|
33,557 |
|
Total current liabilities |
|
54,455 |
|
|
62,790 |
|
Term loan, net |
|
38,161 |
|
|
37,967 |
|
Operating lease liability, net
of current portion |
|
19,928 |
|
|
20,440 |
|
Other noncurrent
liabilities |
|
4,004 |
|
|
4,130 |
|
Total liabilities |
|
116,548 |
|
|
125,327 |
|
|
|
|
|
|
|
Preferred stock, $0.00001 par
value per share; 10,000,000 shares authorized and no shares issued
and outstanding as of March 31, 2023 and December 31, 2022 |
|
- |
|
|
- |
|
Stockholders'
(deficit) equity |
|
|
Common stock, $0.00001 par
value per share; 200,000,000 shares authorized and 63,473,349 and
63,207,507 issued and outstanding as of March 31, 2023 and December
31, 2022, respectively |
|
1 |
|
|
1 |
|
Additional paid-in
capital |
|
404,114 |
|
|
402,389 |
|
Accumulated deficit |
|
(419,063 |
) |
|
(401,441 |
) |
Total stockholders’ (deficit) equity |
|
(14,948 |
) |
|
949 |
|
Total liabilities,
convertible preferred stock, and stockholders' (deficit)
equity |
$ |
101,600 |
|
$ |
126,276 |
|
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communications carolyn.hawley@canalecomm.com
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