Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric,
pivotal-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) in both rare
and large patient populations, today reported financial results for
the second quarter ended June 30, 2022, and provided a corporate
update.
“We are excited to announce important progress in giant axonal
neuropathy (GAN) including stabilization and improvement of sensory
nerve function, a definitive clinical endpoint, in patients with
GAN following treatment with TSHA-120,” said RA Session II,
President, Founder and CEO of Taysha. “In addition, we now have
positive comparability data demonstrating that our commercial grade
and clinical trial material are comparable across all key quality
attributes. Importantly, we believe positive feedback received from
the MHRA, in conjunction with robust comparability data for
TSHA-120 and comprehensive clinical data generated to date, further
support our ongoing regulatory engagement. We expect additional
regulatory feedback, including from the FDA, by year-end. In Rett
syndrome, we are highly encouraged by late-breaking neonatal data
in preclinical mouse models demonstrating near normalization of
survival and normalization of behavior. We look forward to
reporting preliminary Phase 1/2 clinical data in adult females with
Rett syndrome by year-end 2022.”
Recent Corporate Highlights
TSHA-120 for giant axonal neuropathy (GAN): an
intrathecally dosed AAV9 gene therapy in clinical development for
the rare inherited genetic disorder GAN. TSHA-120 has received
orphan drug and rare pediatric disease designations from the FDA
and orphan drug designation from the European Commission.
- TSHA-120-treated patients with GAN demonstrated durable
neurophysiological improvements in the sensory nerve action
potential (SNAP), a definitive clinical endpoint, compared to rapid
and irreversible decline in sensory function early in life in
untreated patients based on natural history
- Natural history data suggest that
sensory function is unrecoverable once SNAP reaches zero
- All patients who were 9 years and
older in the natural history study demonstrated zero SNAP
response
- Treatment with TSHA-120 resulted in
stabilization and improvement of sensory nerve function with
durability of improvement in SNAP
- Pathology from nerve biopsies in all
evaluable samples confirmed the presence of regenerative nerve
fibers in 100% of TSHA-120-treated patients (n=11)
- TSHA-120 commercial grade and clinical
trial material considered comparable across all key quality
attributes as assessed by an extensive panel of release assays and
next-generation sequencing
- Positive regulatory
feedback from MHRA supports regulatory strategy
- MHRA found
functional clinical data, eye pathology and visual acuity, and
nerve biopsy data compelling. Agency agreed with commercial
manufacturing and release testing strategy, including potency
assays and recommended dosing a few patients with commercial grade
material, which will be released in September 2022. MHRA was
supportive of Taysha’s proposal to perform validation work on MFM32
as a key clinical endpoint
TSHA-102 in Rett syndrome: a
self-complementary intrathecally delivered AAV9 gene replacement
therapy in clinical development for Rett syndrome. TSHA-102
utilizes the novel miRNA-Responsive Auto-Regulatory Element
(miRARE) platform to regulate transgene expression genotypically on
a cell-by-cell basis. TSHA-102 is the first-and-only gene therapy
in clinical development for Rett syndrome. 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.
- Late-breaking positive preclinical
data for TSHA-102 in Rett syndrome demonstrated near normalization
of survival and normalization of behavior in neonatal knockout Rett
mice, a model significantly more severe than the human
phenotype
Anticipated 2022 Milestones
- Further regulatory update, including from FDA, for TSHA-120 in
GAN by year-end 2022
- First-in-human preliminary Phase 1/2 data for TSHA-102 in Rett
syndrome by year-end 2022
Second Quarter 2022 Financial Highlights
Research and Development (R&D) Expenses:
Research and development expenses were $23.1 million for the three
months ended June 30, 2022, compared to $30.6 million for the three
months ended June 30, 2021. The $7.5 million decrease was primarily
attributable to a decrease of $3.8 million in third-party R&D,
primarily related to GLP toxicology studies, a decrease of $3.2
million in R&D manufacturing costs, and lower employee
compensation expenses of $0.5 million.
General and Administrative (G&A)
Expenses: General and administrative expenses were
$9.9 million for the three months ended June 30, 2022, compared to
$10.1 million for the three months ended June 30, 2021. The
decrease of approximately $0.2 million was primarily attributable
to a decrease of $1.1 million in professional fees related to
market research, recruiting, accounting, and patient advocacy
activities. This was partially offset by $0.9 million of
incremental employee compensation expenses.
Net loss: Net loss for the three months
ended June 30, 2022, was $33.9 million, or $0.84 per
share, as compared to a net loss of $40.9 million,
or $1.09 per share, for the three months ended June
30, 2021.
Cash and cash equivalents: As of June 30,
2022, the Company had cash and cash equivalents of $66.2 million.
Taysha continues to expect that its current cash and cash
equivalents, in addition to full access to its existing term loan
facility, is sufficient to fund operating expenses into the fourth
quarter of 2023.
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast with
slides today at 8:00 am ET / 7:00 am CT 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 13730848. 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, 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, 2021, and our Quarterly Report on
Form 10-Q for the quarter ended June 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.
Taysha Gene Therapies,
Inc.Condensed Consolidated Statements of
Operations(in thousands, except share and per share
data)(Unaudited)
|
|
For the Three MonthsEnded June
30, |
|
For the Six MonthsEnded June
30, |
|
|
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
23,118 |
|
$ |
30,643 |
|
$ |
60,917 |
|
$ |
54,497 |
|
General and
administrative |
|
|
9,867 |
|
|
10,129 |
|
|
21,336 |
|
|
18,365 |
|
Total operating expenses |
|
|
32,985 |
|
|
|
40,772 |
|
|
|
82,253 |
|
|
|
72,862 |
|
|
Loss from
operations |
|
|
(32,985 |
) |
|
|
(40,772 |
) |
|
|
(82,253 |
) |
|
|
(72,862 |
) |
|
Other income
(expense): |
|
|
|
|
|
|
|
|
|
Interest income |
|
|
27 |
|
|
|
40 |
|
|
|
41 |
|
|
|
106 |
|
|
Interest expense |
|
|
(912 |
) |
|
|
(194 |
) |
|
|
(1,761 |
) |
|
|
(194 |
) |
|
Other expense |
|
|
(3 |
) |
|
|
- |
|
|
|
(11 |
) |
|
|
- |
|
|
Total other expense, net |
|
|
(888 |
) |
|
|
(154 |
) |
|
|
(1,731 |
) |
|
|
(88 |
) |
|
Net loss |
|
$ |
(33,873 |
) |
|
$ |
(40,926 |
) |
|
$ |
(83,984 |
) |
|
$ |
(72,950 |
) |
|
|
|
|
|
|
|
|
|
|
|
Net loss per common share,
basic and diluted |
|
$ |
(0.84 |
) |
|
$ |
(1.09 |
) |
|
$ |
(2.14 |
) |
|
$ |
(1.96 |
) |
|
Weighted average common shares
outstanding, basic and diluted |
|
|
40,142,403 |
|
|
|
37,479,164 |
|
|
|
39,163,996 |
|
|
|
37,237,115 |
|
|
Taysha Gene Therapies,
Inc.Condensed Consolidated Balance Sheet
Data(in thousands, except share and per share
data)(Unaudited)
|
|
|
|
|
|
|
June 30,2022 |
|
December 31,2021 |
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
66,239 |
|
|
$ |
149,103 |
|
Prepaid expenses and other
current assets |
|
|
10,596 |
|
|
|
10,499 |
|
Total current assets |
|
|
76,835 |
|
|
|
159,602 |
|
Restricted cash |
|
|
2,637 |
|
|
|
2,637 |
|
Deferred lease asset |
|
|
643 |
|
|
|
667 |
|
Property, plant and equipment,
net |
|
|
61,011 |
|
|
|
50,610 |
|
Other non-current assets |
|
|
1,206 |
|
|
|
440 |
|
Total
assets |
|
$ |
142,332 |
|
|
$ |
213,956 |
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY |
|
|
|
|
Current liabilities |
|
|
|
|
Accounts payable |
|
$ |
23,967 |
|
|
$ |
21,763 |
|
Accrued expenses and other
current liabilities |
|
|
18,986 |
|
|
|
29,983 |
|
Total current liabilities |
|
|
42,953 |
|
|
|
51,746 |
|
Build-to-suit lease
liability |
|
|
25,609 |
|
|
|
25,900 |
|
Term Loan, net |
|
|
37,580 |
|
|
|
37,192 |
|
Other non-current
liabilities |
|
|
3,480 |
|
|
|
3,735 |
|
Total liabilities |
|
|
109,622 |
|
|
|
118,573 |
|
|
|
|
|
|
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, 2022 and December 31, 2021 |
|
|
- |
|
|
|
- |
|
Common stock, $0.00001 par
value per share; 200,000,000 shares authorized and 41,020,086 and
38,473,945 issued and outstanding as of June 30, 2022 and December
31, 2021, respectively |
|
|
1 |
|
|
|
- |
|
Additional paid-in
capital |
|
|
352,342 |
|
|
|
331,032 |
|
Accumulated deficit |
|
|
(319,633 |
) |
|
|
(235,649 |
) |
Total stockholders’
equity |
|
|
32,710 |
|
|
|
95,383 |
|
Total liabilities and
stockholders' equity |
|
$ |
142,332 |
|
|
$ |
213,956 |
|
Company Contact:Kimberly Lee, D.O. Chief
Corporate Affairs OfficerTaysha Gene
Therapiesklee@tayshagtx.com
Media Contact:Carolyn HawleyEvoke
Canalecarolyn.hawley@evokegroup.com
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jul 2023 to Jul 2024