Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the
Company”), 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 third quarter ended
September 30, 2023, and provided corporate and clinical updates.
“Prior to initiating the REVEAL trial, the
expectation of seeing a clinical benefit in adults with stage four
Rett syndrome was low due to the advanced and relentless
progression of the disease. We are highly encouraged by the
positive 12-week data from the first adult patient and initial
four-week data from the second adult patient in the low dose
TSHA-102 cohort,” said Sean P. Nolan, Chairman and Chief Executive
Officer of Taysha. “Importantly, response was seen across multiple
clinical domains in both stage four patients with different genetic
mutation severity and phenotypic expression, including autonomic
function, socialization, and gross and fine motor skills. These
early improvements in both patients, coupled with the sustained
response through week 12 in the first patient, support the
transformative potential of TSHA-102 across multiple genotypes of
Rett syndrome.”
Dr. Elsa Rossignol, M.D., FRCP, FAAP, Associate
Professor in Neuroscience and Pediatrics at the Université de
Montréal, and Principal Investigator of the REVEAL trial at the CHU
Sainte-Justine added, “The two adult patients dosed with TSHA-102
have different mutations in their MECP2 gene that manifest in
different phenotypes and clinical severity. Following treatment,
both patients experienced improvement in key clinical domains
impacting activities of daily living, including breathing
dysrhythmia, autonomic function, socialization, and gross and fine
motor skills. Both patients display significantly reduced breathing
dysrhythmia, with less breath holding spells and infrequent
hyperventilation, improved limb perfusion and vastly improved
interest in social communication and activities. In addition, the
first patient experienced sustained and new improvements, with
restored movement in her legs and the gained ability to sit
unassisted for up to 15 minutes for the first time in over a
decade. Further, her hand function improved with the gained ability
to grasp objects with her non-dominant hand and transfer them to
her dominant hand for the first time since infancy. Following
treatment, the second patient’s posture, gait and stability
improved, resulting in straighter posture and smoother movements
when walking. Her hand stereotypies also improved for the first
time since regression at age three: she now displays less forceful
hand wringing and her hands are often open and relaxed, providing
new opportunities for fine motor skill learning. In addition, her
seizures are much less frequent. I’m encouraged by the early
positive signals and consistent improvement seen in both patients
following treatment.”
Recent Corporate Highlights
- Presented two
posters at the European Society of Gene & Cell Therapy (ESGCT)
30th Annual Congress on new preclinical in vitro data supporting
the miRARE technology, and initial clinical data from the first
adult patient dosed in the REVEAL Phase 1/2 trial
- United States
(U.S.) Food and Drug Administration (FDA) granted Fast Track
Designation to TSHA-102 for Rett syndrome
- Entered into a
loan and security agreement with Trinity Capital and terminated
existing loan and security agreement with Silicon Valley Bank,
extending cash runway into 2026; no financial covenants or warrants
associated with the loan and security agreement with Trinity
Capital
Recent Clinical 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
miRARE technology designed to mediate levels of MECP2 in the CNS on
a cell-by-cell basis without risk of overexpression.
TSHA-102 is being evaluated in the REVEAL Phase
1/2 adult trial, a first-in-human, open-label, randomized,
dose-escalation and dose-expansion study in Canada evaluating the
safety and preliminary efficacy of TSHA-102 in adult females with
Rett syndrome due to MECP2 loss-of-function mutation.
Results from the first patient
(large MECP2 deletion; associated
with severe phenotype) and second patient (missense
MECP2 mutation; associated with milder
phenotype) with late motor deterioration stage four Rett syndrome
dosed with TSHA-102 in the low dose cohort:
- Generally
well-tolerated with no treatment-emergent serious adverse events
(SAEs) as of 20-week assessment post-treatment for patient one and
six-week assessment for patient two
- Based on clinical observations by
the Principal Investigator (PI), both patients demonstrated
improvement in multiple clinical domains, with sustained and new
improvements in patient one 12-weeks post-treatment and initial
improvements in patient two four-weeks post-treatment, including:
- Autonomic function: improved
breathing patterns and sleep quality/duration (patient one) reduced
seizures and improved breathing patterns (patient two)
- Socialization: improved social
interest and vocalization (patient one) improved social interest
(patient two)
- Gross motor skills: gained ability
to sit unassisted and move legs (patient one) improved posture,
gait and stability (patient two)
- Fine motor
skills: improved hand function (patient one) improved hand
stereotypies (patient two)
- Seizure Diary
demonstrated comparable seizure events relative to baseline
through 20-weeks post-treatment in patient one and reduced seizure
events relative to baseline through day 33 post-treatment for
patient two, based on caregiver-reported medical history
- Clinical improvements demonstrated in both patients across key
efficacy measures include:
- Patient one:
sustained improvement through 12-weeks in Clinical Global
Impression–Improvement (CGI-I), Clinical Global Impression–Severity
(CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), with new
improvements in Revised Motor Behavior Assessment (R-MBA), Parental
Global Impressions–Improvement (PGI-I) and Rett Syndrome Hand
Function Scale (RSHFS)
- Patient two: improvement four-weeks
post-treatment in CGI-I, PGI-I, RSBQ and R-MBA
-
Figure accompanying this
announcement is available
at: https://www.globenewswire.com/NewsRoom/AttachmentNg/9b39103b-685c-4849-9072-97f32658320c.
Additional information
on available clinical data is available in the Company’s quarterly
report on Form 10-Q for the quarter ended September 30, 2023, to be
filed with the SEC.
- Independent Data Monitoring
Committee (IDMC) provided clearance to dose third adult patient
based on available data
Upcoming Milestones
- Dosing of third
adult patient and completion of dosing in cohort one (low dose) in
the adult trial in Canada anticipated in the fourth quarter of
2023/first quarter of 2024
- Further updates
on available clinical data from the low dose cohort expected in the
first quarter of 2024
- Dosing of first
pediatric Rett syndrome patient in the U.S. anticipated in the
first quarter of 2024
- U.K. Medicines
and Healthcare products Regulatory Agency (MHRA) response to
Clinical Trial Application (CTA) for TSHA-102 in pediatric patients
with Rett syndrome expected by year-end 2023
Third Quarter 2023 Financial Highlights
Research and Development
Expenses: Research and development expenses were $11.8
million for the three months ended September 30, 2023, compared to
$16.8 million for the three months ended September 30, 2022. The
net change was due to a $9.3 million decrease due to lower
compensation expense as a result of reduced headcount, lower
licensing milestone fees, fewer manufacturing batches and fewer raw
material purchases. This was partially offset by a $4.3 million
increase in activity surrounding ongoing clinical trial efforts in
the Rett syndrome REVEAL adult and pediatric studies.
General and Administrative (G&A)
Expenses: General and administrative expenses were $8.6
million for the three months ended September 30, 2023, compared to
$8.7 million for the three months ended September 30, 2022. The
decrease of $0.1 million was due to reduced compensation expense
due to lower headcount of $2.0 million and reduced consulting and
professional fees of $0.7 million, partially offset by $2.6 million
issuance costs allocated to the liability-classified pre-funded
warrants issued in connection with the private placement financing
completed in August 2023.
Net loss: Net loss for the
three months ended September 30, 2023, was $117.1 million, or $0.93
per share, as compared to a net loss of $26.5 million, or $0.65 per
share, for the three months ended September 30, 2022, due to a
non-cash expense of $100.5 million recorded in Q3 2023 from a
change in the fair value of warrant liability from pre-funded
warrants in connection with the private placement financing
completed in August 2023.
Cash and cash equivalents: As
of September 30, 2023, the Company had cash and cash equivalents of
$164.3 million. The Company expects that its existing cash and cash
equivalents will fund operating expenses and capital requirements
into 2026.
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 corporate and clinical
updates. 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 13741244. 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 the reproducibility
and durability of any favorable results initially seen in our first
and second patients dosed in the REVEAL trial 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 timing of our clinical trials, including
reporting data therefrom, the forecast of our cash runway and the
Company’s expectations regarding funding, operating and working
capital expenditures. 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, and our Quarterly Report on
Form 10-Q for the quarter ended September 30, 2023, 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. 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 Balance Sheet Data |
(in thousands, except share and per share data) |
(Unaudited) |
|
|
|
September 30,2023 |
|
December 31,2022 |
ASSETS |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
164,278 |
|
|
$ |
87,880 |
|
Prepaid expenses and other current assets |
|
|
5,529 |
|
|
|
8,537 |
|
Assets held for sale |
|
|
2,000 |
|
|
|
— |
|
Total current assets |
|
|
171,807 |
|
|
|
96,417 |
|
Restricted cash |
|
|
2,637 |
|
|
|
2,637 |
|
Property, plant and equipment,
net |
|
|
11,169 |
|
|
|
14,963 |
|
Operating lease right-of-use
assets |
|
|
9,852 |
|
|
|
10,943 |
|
Other non-current assets |
|
|
304 |
|
|
|
1,316 |
|
Total
assets |
|
$ |
195,769 |
|
|
$ |
126,276 |
|
LIABILITIES AND
STOCKHOLDERS' (DEFICIT) EQUITY |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
7,520 |
|
|
$ |
10,946 |
|
Accrued expenses and other current liabilities |
|
|
13,638 |
|
|
|
18,287 |
|
Deferred revenue |
|
|
18,759 |
|
|
|
33,557 |
|
Warrant liability |
|
|
140,534 |
|
|
|
— |
|
Total current liabilities |
|
|
180,451 |
|
|
|
62,790 |
|
Deferred revenue, net of current
portion |
|
|
2,951 |
|
|
|
— |
|
Term loan, net |
|
|
38,548 |
|
|
|
37,967 |
|
Operating lease liability, net of
current portion |
|
|
19,101 |
|
|
|
20,440 |
|
Other non-current
liabilities |
|
|
3,832 |
|
|
|
4,130 |
|
Total liabilities |
|
|
244,883 |
|
|
|
125,327 |
|
|
|
|
|
|
|
|
|
|
|
Stockholders' (deficit)
equity |
|
|
|
|
Preferred stock, $0.00001 par
value per share; 10,000,000 shares authorized and no shares issued
and outstanding as of September 30, 2023 and December 31,
2022 |
|
|
— |
|
|
|
— |
|
Common stock, $0.00001 par value
per share; 200,000,000 shares authorized and 186,960,193 and
63,207,507 issued and outstanding as of September 30, 2023 and
December 31, 2022, respectively |
|
|
2 |
|
|
|
1 |
|
Additional paid-in capital |
|
|
511,632 |
|
|
|
402,389 |
|
Accumulated deficit |
|
|
(560,748 |
) |
|
|
(401,441 |
) |
Total stockholders’ (deficit) equity |
|
|
(49,114 |
) |
|
|
949 |
|
Total liabilities and
stockholders' (deficit) equity |
|
$ |
195,769 |
|
|
$ |
126,276 |
|
|
|
|
|
|
|
|
|
|
Taysha Gene Therapies, Inc. |
Condensed Consolidated Statement of
Operations |
(in thousands, except share and per share data) |
(Unaudited) |
|
|
|
For the Three MonthsEnded
September 30, |
|
For the Nine MonthsEnded
September 30, |
|
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Revenue |
|
$ |
4,746 |
|
|
$ |
— |
|
|
$ |
11,847 |
|
|
$ |
— |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
11,791 |
|
|
|
16,774 |
|
|
|
44,096 |
|
|
|
78,462 |
|
General and administrative |
|
|
8,589 |
|
|
|
8,683 |
|
|
|
23,328 |
|
|
|
30,019 |
|
Impairment of long-lived assets |
|
|
616 |
|
|
|
— |
|
|
|
616 |
|
|
|
— |
|
Total operating expenses |
|
|
20,996 |
|
|
|
25,457 |
|
|
|
68,040 |
|
|
|
108,481 |
|
Loss from
operations |
|
|
(16,250 |
) |
|
|
(25,457 |
) |
|
|
(56,193 |
) |
|
|
(108,481 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
|
Change in fair value of warrant liability |
|
|
(100,456 |
) |
|
|
— |
|
|
|
(100,456 |
) |
|
|
— |
|
Interest income |
|
|
1,109 |
|
|
|
9 |
|
|
|
1,651 |
|
|
|
50 |
|
Interest expense |
|
|
(1,471 |
) |
|
|
(1,078 |
) |
|
|
(4,285 |
) |
|
|
(2,493 |
) |
Other expense |
|
|
(19 |
) |
|
|
(1 |
) |
|
|
(24 |
) |
|
|
(12 |
) |
Total other expense, net |
|
|
(100,837 |
) |
|
|
(1,070 |
) |
|
|
(103,114 |
) |
|
|
(2,455 |
) |
Net loss |
|
$ |
(117,087 |
) |
|
$ |
(26,527 |
) |
|
$ |
(159,307 |
) |
|
$ |
(110,936 |
) |
Net loss per common share, basic
and diluted |
|
$ |
(0.93 |
) |
|
$ |
(0.65 |
) |
|
$ |
(1.88 |
) |
|
$ |
(2.79 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
|
125,700,799 |
|
|
|
40,937,808 |
|
|
|
84,630,796 |
|
|
|
39,761,764 |
|
|
Company Contact:Hayleigh Collins Director, Head
of Corporate Communications and Investor RelationsTaysha Gene
Therapies, Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communications carolyn.hawley@canalecomm.com
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