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 fourth quarter and full-year ended December 31, 2022, and
provided a corporate update.
“The actions taken early this year to improve execution and
expedite progress on our two lead clinical programs in Rett
syndrome and GAN are having a positive effect,” said Sean P. Nolan,
Chairman and Chief Executive Officer of Taysha. “We recently
initiated screening of the first potential adult subject for the
REVEAL Rett syndrome trial and remain on track to dose the first
patient and deliver initial available first-in-human adult data,
primarily on safety, for TSHA-102 in the first half of the year.
Additionally, we recently submitted a protocol amendment to allow
patients as young as 15 years old to be included in the study,
which we believe will further expedite enrollment. We remain on
track to submit a CTA to the MHRA in mid-2023 to conduct a
pediatric Rett syndrome trial, and plan to submit an IND to the FDA
for Rett syndrome in the second half of 2023. For TSHA-120 in GAN,
based on the constructive feedback recently received from the FDA
in response to our follow up questions to the formal Type B
end-of-Phase 2 meeting minutes, coupled with the positive
preliminary assessment of the ongoing comprehensive data analyses,
we plan to submit a formal meeting request to the Agency in the
second quarter of 2023 to discuss the potential regulatory pathway
forward for this ultra-rare disease with no approved
treatment.”
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 inherited
genetic neurodevelopmental disorder. 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.
- Screening initiated for first
potential adult patient with Rett syndrome for the Phase 1/2 REVEAL
trial
- Submitted protocol amendment
expanding enrollment eligibility to include subjects ≥ 15
years
- Dosing of the first adult patient
with Rett syndrome anticipated in H1 2023
- Initial available Phase 1/2 clinical
data, primarily on safety, for TSHA-102 in adult patients with Rett
syndrome expected in H1 2023, with planned quarterly updates on
available clinical data thereafter
- 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
- Continued dosing of adult patients
with Rett syndrome in the REVEAL trial in H2 2023
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 to FDA detailing commercial process product
manufacturing and drug comparability analysis
- Receipt of FDA’s response to
Taysha’s follow up questions to the formal Type B end-of-Phase 2
meeting minutes
- FDA clarified MFM32, the primary
efficacy scale discussed at the FDA Type B end-of-Phase 2 meeting,
as a relevant primary endpoint only in the setting of a randomized
double blind placebo controlled trial and acknowledged Taysha's
challenge in executing and enrolling such a study design due to the
ultra-rare nature of GAN
- FDA is open to regulatory
flexibility in a controlled trial setting and willing to consider
alternative study designs utilizing objective measurements to
demonstrate a relatively large treatment effect that is
self-evident and clinically meaningful
- Ongoing comprehensive analyses of
functional, biological and electrophysiological assessments as part
of totality of data evaluation to inform future interactions with
the FDA
- Submission of a formal meeting
request to the FDA planned in Q2 2023
Fourth Quarter and Full-Year 2022 Financial
Highlights
Research and Development Expenses: Research and
development expenses were $13.9 million for the three months ended
December 31, 2022, compared to $37.9 million for the three months
ended December 31, 2021. Research and development expenses were
$91.2 million for the full year ended December 31, 2022, compared
to $131.9 million for the full year ended December 31, 2021. The
$40.7 million decrease was primarily attributable to a decrease of
$20.3 million in research and development manufacturing and other
raw material purchases and a $9.0 million decrease in license fees.
The decrease in research and development expenses for the year
ended December 31, 2022 was also attributable to a $12.0 million
decrease in third-party research and development fees, mainly
related to non-clinical studies and toxicology studies and a $4.7
million decrease in compensation expense as a result of lower
headcount. Overall, lower research and development expenses for the
year ended December 31, 2022 were partially offset by higher
clinical trial expenses of $2.4 million and higher severance
expense of $2.9 million in 2022.
General and Administrative
Expenses: General and administrative expenses were
$7.3 million for the three months ended December 31, 2022, compared
to $11.8 million for the three months ended December 31, 2021.
General and administrative expenses were $37.4 million for the year
ended December 31, 2022, compared to $41.3 million for the year
ended December 31, 2021. The decrease of approximately $3.9 million
was primarily attributable to $5.0 million of lower consulting
professional fees and lower compensation expenses driven by lower
headcount in 2022. Lower general and administrative expenses were
partially offset by $1.1 million of severance expense.
Net loss: Net loss for the three months
ended December 31, 2022 was $55.7 million, or $0.99 per
share, as compared to a net loss of $50.4 million,
or $1.32 per share, for the three months
ended December 31, 2021. In November 2022, we recorded a $36.4
million non-cash, non-recurring impairment charge related to the
North Carolina manufacturing facility. The net loss for the three
months ended December 31, 2022 was partially offset by revenue of
$2.5 million recognized related to the Astellas Transactions. Net
loss for the full year ended December 31, 2022 was $166.0 million
or $3.78 per share, as compared to a net loss of $174.5 million, or
$4.64 per share, for the full year ended December 31, 2021.
Cash and cash equivalents: As of December
31, 2022, Taysha had $87.9 million in cash and cash equivalents.
The Company 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 877-407-0792 (U.S./Canada) or 201-689-8263
(international). The conference ID for all callers is 13736479. 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—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 TSHA-102 and TSHA-120 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.
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 |
For the twelve months ended |
|
December 31 2022 |
December 31 2021 |
December 31 2022 |
December 31 2021 |
|
|
|
|
|
Revenue: |
|
|
|
|
Service Revenue |
$ |
2,502 |
|
|
- |
|
$ |
2,502 |
|
$ |
- |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
13,861 |
|
|
37,918 |
|
|
91,169 |
|
|
131,943 |
|
General and administrative |
|
7,341 |
|
|
11,806 |
|
|
37,360 |
|
|
41,324 |
|
Impairment of long-lived assets |
|
36,420 |
|
|
- |
|
|
36,420 |
|
|
- |
|
Total operating expenses |
|
57,622 |
|
|
49,724 |
|
|
164,949 |
|
|
173,267 |
|
Loss from
operations |
|
(55,120 |
) |
|
(49,724 |
) |
|
(162,447 |
) |
|
(173,267 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
|
|
Interest Income |
|
199 |
|
|
29 |
|
|
249 |
|
|
172 |
|
Interest expense |
|
(796 |
) |
|
(691 |
) |
|
(3,798 |
) |
|
(1,428 |
) |
Other |
|
(6 |
) |
|
- |
|
|
(18 |
) |
|
- |
|
Total other income (expense) |
|
(603 |
) |
|
(662 |
) |
|
(3,567 |
) |
|
(1,256 |
) |
Net loss |
$ |
(55,723 |
) |
$ |
(50,386 |
) |
$ |
(166,014 |
) |
$ |
(174,523 |
) |
|
|
|
|
|
Net loss per common share,
basic and diluted |
$ |
(0.99 |
) |
$ |
(1.32 |
) |
$ |
(3.78 |
) |
$ |
(4.64 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
56,386,130 |
|
|
38,110,597 |
|
|
43,952,015 |
|
|
37,650,566 |
|
|
|
|
|
|
Taysha Gene Therapies,
Inc.Condensed Consolidated Balance Sheet
Data(in thousands, except share and per share
data)(Unaudited)
|
December 31, 2022 |
December 31, 2021 |
|
|
|
ASSETS |
|
|
Current assets: |
|
|
Cash and cash equivalents |
$ |
87,880 |
|
$ |
149,103 |
|
Prepaid expenses and other current assets |
|
8,537 |
|
|
10,499 |
|
Total current assets |
|
96,417 |
|
|
159,602 |
|
|
|
|
Restricted cash |
|
2,637 |
|
|
2,637 |
|
Property, plant and equipment,
net |
|
14,963 |
|
|
50,610 |
|
Operating lease right-of-use
assets |
|
10,943 |
|
|
- |
|
Other noncurrent assets |
|
1,316 |
|
|
1,107 |
|
Total
assets |
$ |
126,276 |
|
$ |
213,956 |
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY |
|
|
Current liabilities: |
|
|
Accounts payable |
$ |
10,946 |
|
$ |
21,763 |
|
Accrued expenses and other current liabilities |
|
18,287 |
|
|
29,983 |
|
Deferred revenue |
|
33,557 |
|
|
- |
|
Total current liabilities |
|
62,790 |
|
|
51,746 |
|
Build-to-suit lease
liability |
|
- |
|
|
25,900 |
|
Term loan, net |
|
37,967 |
|
|
37,192 |
|
Operating lease liability, net
of current portion |
|
20,440 |
|
|
- |
|
Other noncurrent
liabilities |
|
4,130 |
|
|
3,735 |
|
Total liabilities |
|
125,327 |
|
|
118,573 |
|
|
|
|
|
|
|
Stockholders’
equity |
|
|
Common stock, $0.00001 par
value per share; 200,000,000 shares authorized and 63,207,507
issued and outstanding as of December 31, 2022 and 38,473,945
outstanding as of December 31, 2021 |
|
1 |
|
|
- |
|
Additional paid-in
capital |
|
402,389 |
|
|
331,032 |
|
Accumulated deficit |
|
(401,441 |
) |
|
(235,649 |
) |
Total stockholders’ equity |
|
949 |
|
|
95,383 |
|
Total liabilities and
stockholders' equity |
$ |
126,276 |
|
$ |
213,956 |
|
Company Contact:Hayleigh Collins Director, Head
of Corporate CommunicationsTaysha Gene
Therapieshcollins@tayshagtx.com
Media Contact:Carolyn HawleyEvoke
Canalecarolyn.hawley@evokegroup.com
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