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 first quarter ended March 31, 2022 and provided a corporate
update.“In 2022, we are focused on advancing our key programs in
Rett syndrome and GAN. We initiated clinical development of
TSHA-102 in Rett syndrome and expect preliminary clinical data from
the REVEAL study by year-end,” said RA Session II, President,
Founder and CEO of Taysha. “Recently, the European Commission
granted orphan drug designation for TSHA-120 for GAN, further
highlighting the unmet need for treatment options for these
patients and the important potential of TSHA-120. We have completed
a commercially representative GMP batch for TSHA-120 with release
testing currently underway. Our existing capital resources, along
with full access to the term loan facility, should fund operating
expenses and capital requirements into the fourth quarter of 2023.”
Recent Corporate Highlights
TSHA-120 for giant axonal neuropathy (GAN): an
intrathecally dosed AAV9 gene therapy currently being evaluated in
a clinical trial for the treatment of GAN, a rare inherited genetic
disorder that affects both the central and peripheral nervous
systems and is caused by loss-of-function mutations in the gene
coding for gigaxonin. TSHA-120 is designed to deliver a
functional copy of the GAN gene to the CNS and PNS. TSHA-120 has
already received orphan drug and rare pediatric disease
designations from the U.S. Food and Drug Administration (FDA) and
orphan drug designation from the European Commission.
- Received orphan drug designation from the European Commission
for GAN in April 2022
- Reported positive clinical efficacy and safety data for high
dose cohort and long-term durability data across all therapeutic
dose cohorts for TSHA-120 in GAN
- Efficacy data for high dose cohort demonstrated clinically
meaningful and statistically significant improvement in MFM32 by
Year 1 compared to natural history (n=3)
- Long-term durability data across all therapeutic dose cohorts
demonstrated a 10-point improvement in mean change in MFM32 by Year
3 compared to estimated natural history decline of 24 points
(n=5)
- Biopsy data in five of six patient samples analyzed to date
confirmed active regeneration of nerve fibers following treatment
with TSHA-120 (n=6)
- 53 patient-years of clinical data support the safety and
tolerability profile of TSHA-120
- Commercially representative GMP batch completed and release
testing underway
- Regulatory feedback for TSHA-120 in
GAN expected mid-2022
TSHA-102 in Rett syndrome: a
self-complementary intrathecally delivered AAV9 gene replacement
therapy under development for the treatment of Rett syndrome.
TSHA-102 utilizes the novel 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.
- CTA approved by Health Canada in March 2022
- Published preclinical data for TSHA-102 in Rett syndrome
presented at the International Rett Syndrome Foundation (IRSF) Rett
Syndrome Scientific Meeting and the ASCEND Rett Syndrome National
Summit in April 2022
- Initiation of clinical development with the REVEAL study, an
open-label, dose escalation, randomized, multicenter Phase 1/2
clinical trial in adult female patients with Rett syndrome
- Sainte-Justine Mother and Child University Hospital Center in
Montreal, Quebec, Canada selected as initial clinical site under
the direction of Dr. Elsa Rossignol, principal investigator
- Key assessments to include Rett-specific and global
assessments, quality of life, biomarkers and neurophysiology and
imaging
- Preliminary clinical data for
TSHA-102 in Rett syndrome expected by year-end 2022
AAV9 Gene Replacement for CLN7 Batten
disease: an investigational AAV9 intrathecally dosed
gene replacement therapy designed to deliver a full-length copy of
the CLN7 gene to potentially treat CLN7 disease, a rapidly
progressing rare lysosomal storage disease with no approved
treatments. The clinical development of the CLN7 program is being
funded by UT Southwestern (UTSW), Children’s Health and Children’s
Medical Center Foundation.
- Reported positive preliminary
clinical safety data for first-generation construct in CLN7 Batten
disease from UTSW-sponsored clinical trial
- Data from three patients dosed presented at the 18th Annual
WORLDSymposium
- Fourth patient with CLN7 disease
dosed at 1.0 x 1015 total vg
Anticipated Milestones
- Regulatory update for TSHA-120 in GAN by mid-2022
- Preliminary clinical data from the REVEAL study for TSHA-102 in
Rett syndrome by year-end 2022
- Initiation of clinical development for TSHA-105 in SLC13A5
deficiency
- Continued clinical development of the first-generation
construct for CLN7 disease in 2022
- Continued clinical development for
TSHA-118 in CLN1 disease
First Quarter 2022 Financial Highlights
Research and Development (R&D) Expenses:
Research and development expenses were $37.8 million for the three
months ended March 31, 2022, compared to $23.9 million for the
three months ended March 31, 2021. The increase of approximately
$13.9 million was primarily attributable to an increase of $9.3
million in employee compensation, which included $2.2 million of
severance and one-time termination costs in connection with the
strategic reprioritization of programs completed in March 2022 and
$1.0 million of non-cash stock-based compensation. Additionally, in
the three months ended March 31, 2022, we incurred an increase of
$2.9 million of expenses in research and development manufacturing
and other raw material purchases. We also incurred an increase of
$1.7 million of third-party research and development consulting
fees, primarily related to GLP toxicology studies and clinical
study activities.
General and Administrative (G&A)
Expenses: General and administrative expenses were
$11.5 million for the three months ended March 31, 2022, compared
to $8.2 million for the three months ended March 31, 2021. The
increase of approximately $3.3 million was primarily attributable
to $2.9 million of incremental compensation expense, which included
$0.4 million of severance and one-time termination costs and $0.7
million of non-cash stock-based compensation. We also incurred an
increase of $0.4 million in professional fees related to insurance,
investor relations/communications, accounting, and market
research.
Net loss: Net loss for the three months
ended March 31, 2022 was $50.1 million, or $1.31 per
share, as compared to a net loss of $32.0 million,
or $0.87 per share, for the three months ended March
31, 2021.
Cash and cash equivalents: As of March 31,
2022, we had $96.6 million in cash and cash equivalents. This
excludes approximately $12 million in gross proceeds generated from
the sale of common stock, par value $0.00001 per share, under our
existing at-the-market facility in April 2022.
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast 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 13729044. 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 March 31, 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 March
31, |
|
|
2022 |
|
|
|
2021 |
|
Operating
expenses: |
|
|
|
Research and development |
$ |
37,799 |
|
|
$ |
23,854 |
|
General and
administrative |
|
11,469 |
|
|
|
8,236 |
|
Total operating expenses |
|
49,268 |
|
|
|
32,090 |
|
Loss from
operations |
|
(49,268 |
) |
|
|
(32,090 |
) |
Other income
(expense): |
|
|
|
Interest income |
|
14 |
|
|
|
66 |
|
Interest expense |
|
(849 |
) |
|
|
- |
|
Other expense |
|
(8 |
) |
|
|
- |
|
Total other expense, net |
|
(843 |
) |
|
|
66 |
|
Net loss |
$ |
(50,111 |
) |
|
$ |
(32,024 |
) |
|
|
|
|
Net loss per common share,
basic and diluted |
$ |
(1.31 |
) |
|
$ |
(0.87 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
38,174,717 |
|
|
|
36,992,377 |
|
Taysha Gene Therapies,
Inc.Condensed Consolidated Balance Sheet
Data(in thousands, except share and per share
data)(Unaudited)
|
March 31, 2022 |
|
December 31, 2021 |
ASSETS |
|
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
96,630 |
|
|
$ |
149,103 |
|
Prepaid expenses and other
current assets |
|
10,261 |
|
|
|
10,499 |
|
Total current assets |
|
106,891 |
|
|
|
159,602 |
|
Restricted cash |
|
2,637 |
|
|
|
2,637 |
|
Deferred lease asset |
|
655 |
|
|
|
667 |
|
Property, plant and equipment,
net |
|
55,120 |
|
|
|
50,610 |
|
Other non-current assets |
|
673 |
|
|
|
440 |
|
Total
assets |
$ |
165,976 |
|
|
$ |
213,956 |
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY |
|
|
|
Current liabilities |
|
|
|
Accounts payable |
$ |
21,997 |
|
|
$ |
21,763 |
|
Accrued expenses and other
current liabilities |
|
26,620 |
|
|
|
29,983 |
|
Total current liabilities |
|
48,617 |
|
|
|
51,746 |
|
Build-to-suit lease
liability |
|
25,752 |
|
|
|
25,900 |
|
Term Loan, net |
|
37,386 |
|
|
|
37,192 |
|
Other non-current
liabilities |
|
3,496 |
|
|
|
3,735 |
|
Total liabilities |
|
115,251 |
|
|
|
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 March 31, 2022 and December 31, 2021 |
|
- |
|
|
|
- |
|
Common stock, $0.00001 par
value per share; 200,000,000 shares authorized and 38,473,945
issued and outstanding as of March 31, 2022 and December 31,
2021 |
|
- |
|
|
|
- |
|
Additional paid-in
capital |
|
336,485 |
|
|
|
331,032 |
|
Accumulated deficit |
|
(285,760 |
) |
|
|
(235,649 |
) |
Total stockholders’
equity |
|
50,725 |
|
|
|
95,383 |
|
Total liabilities and
stockholders' equity |
$ |
165,976 |
|
|
$ |
213,956 |
|
Company Contact:Kimberly Lee, D.O. Chief
Corporate Affairs OfficerTaysha Gene
Therapiesklee@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
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