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 second quarter ended June 30, 2023, and provided a corporate
update.
“We are pleased with the progress we have made this quarter in
the clinical evaluation of our two lead investigational programs.
For TSHA-102 in Rett syndrome, we believe the initial safety
profile and significant clinical improvements seen in the first
adult patient with severe disease four weeks post-treatment
reinforces the transformative potential of our gene therapy to
address the root cause of Rett syndrome. Importantly, these early
data indicate that the miRNA-Responsive Auto-Regulatory Element
(miRARE) technology is mediating MECP2 expression in the CNS on a
cell-by-cell basis, supporting the regulatory control of miRARE. We
are highly encouraged by the initial data for TSHA-102 and are
focused on continuing to explore its therapeutic potential, with
the dosing of the second patient expected in the third quarter. We
also received FDA clearance to initiate clinical development of
TSHA-102 in pediatric patients in the U.S. and have submitted a CTA
to the MHRA for TSHA-102 in pediatric patients with Rett syndrome,
which will expand our clinical evaluation to children with earlier
stages of disease progression,” said Sean P. Nolan, Chairman and
Chief Executive Officer of Taysha. “For TSHA-120 in GAN, our new
comprehensive data analysis utilizing the Disease Progression Model
(DPM) was submitted to the FDA, and we plan to review the potential
regulatory pathway for TSHA-120 with the Agency expected in the
third quarter.”
Mr. Nolan continued, “Our successful completion of a $150
million PIPE from top-tier investors significantly bolsters our
balance sheet and we believe highlights the enthusiasm for our
TSHA-102 program and the early clinical readout of the first
patient treated in the REVEAL trial. By extending our cash runway
into the third quarter of 2025, we can focus on execution as we
endeavor to deliver on key value-creating milestones.”
Dr. Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor
Neuroscience and Pediatrics at CHU Sainte-Justine, affiliated to
the Université de Montréal, and Principal Investigator of the
REVEAL trial added, “The efficacy response observed following
treatment with TSHA-102 in the first adult with an advanced stage
of Rett syndrome is promising. Prior to treatment, the patient was
in a constant state of hypertonia, had limited body movement,
required constant back support, and had lost fine and gross motor
function early in childhood. Following treatment, we have observed
improvements in breathing patterns, vocalization and motor skills.
The patient was able to sit unassisted for the first time in over a
decade, and she demonstrated the ability to unclasp her hands and
hold an object steadily for the first time since infancy. I believe
that the patient achieving these milestones so early in treatment,
coupled with the improvements in breathing patterns and quality of
sleep that we have observed, are highly encouraging and support the
potential of TSHA-102. I am honored to work with the Rett syndrome
community and help patients and families suffering from this
devastating disease.”
Rett syndrome is a rare neurodevelopmental disorder caused by
mutations in the X-linked MECP2 gene. The disorder is characterized
by intellectual disabilities, loss of communication, seizures,
slowing and/or regression of development, motor and respiratory
impairment, and shortened life expectancy. Rett syndrome caused by
a pathogenic/likely pathogenic MECP2 mutation is estimated to
affect between 15,000 and 20,000 patients in the U.S., EU and
UK.
Recent Corporate Highlights
$150 million private placement financing strengthens
balance sheet and, together with existing cash and cash
equivalents, extends cash runway into the third quarter of
2025
- Private placement led by new
investor, RA Capital Management, with participation from a large
institutional investor, PBM Capital, RTW Investments, LP, Venrock
Healthcare Capital Partners, TCGX, Acuta Capital Partners, Kynam
Capital Management, LP, Octagon Capital, Invus, GordonMD® Global
Investments LP, and B Group Capital
- Cash runway expected to fund
operational plans into the third quarter of 2025
- Net proceeds to primarily fund
clinical development of TSHA-102 in Rett syndrome and provide
support for program activities for TSHA-120 in GAN, working
capital, and other general corporate purposes
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 platform designed to
mediate levels of MECP2 in the CNS on a cell-by-cell basis without
risk of overexpression. 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.
TSHA-102 is being evaluated in the REVEAL Phase 1/2 trial, a
first-in-human, open-label, randomized, dose-escalation and
dose-expansion study evaluating the safety and preliminary efficacy
of TSHA-102 in adult females with Rett syndrome due to MECP2
loss-of-function mutation. Primary efficacy endpoints are patient
assessments by clinicians using the Clinical Global Impressions
Scale – Improvement (CGI-I), Rett Syndrome Hand Function Scale, and
Revised Motor Behavior Assessment (R-MBA). Secondary endpoints
include patient assessments by clinicians and caregivers using the
Clinical Global Impressions Scale – Severity (CGI-S), the Rett
Syndrome Behavior Questionnaire (RSBQ) and other clinical
assessment scales.
Results from the first adult patient dosed in cohort one
(low dose) with TSHA-102 in the REVEAL Phase 1/2
trial:
- Well-tolerated safety profile with
no treatment-emergent SAEs as of six-week assessment
post-treatment
- The following were demonstrated in
key efficacy measures four weeks post-treatment:
- Clinical Global Impressions –
Improvement (CGI-I) scale adapted to Rett syndrome, a
clinician-reported assessment of overall improvement using a
seven-point scale (one=“very much improved” and seven=“very much
worse”), demonstrated a score of two indicating “much
improved”
- Clinical Global Impressions –
Severity (CGI-S) scale, a clinician-reported assessment of overall
severity of a patient’s illness using a seven-point scale,
demonstrated a one-point improvement from the baseline score of six
(“severely ill”) to a score of five (“markedly ill”)
-
Rett Syndrome Behavior Questionnaire (RSBQ), a
45-item questionnaire to assess Rett syndrome characteristics,
demonstrated a total score improvement of 23 points from the
baseline score of 52 to a score of 29
- Seizure diary demonstrated no
quantifiable seizure events through week five
post-treatment
- No marked changes observed four
weeks post-treatment in the Revised Motor Behavior Assessment
(R-MBA), a 24-question clinician-reported scale measuring disease
behaviors of Rett syndrome
- Initial efficacy data and clinical
observations supported by video evidence from PI six-weeks
post-treatment indicate clinical improvements in multiple domains,
including:
- Autonomic function with improvements
in breathing patterns and sleep quality/duration, including the
normalization of night-time behavior
- Vocalization with increased social
interest
- Gross motor skills with the gained
ability to sit unassisted for three minutes
- Fine motor skills and hand function
with the gained ability to hold an object, unclasp her hands and
use her fingers to touch a screen
- Further updates on available
clinical data expected quarterly
- Dosing of second patient cleared by
the Independent Data Monitoring Committee (IDMC) and expected in Q3
2023, with continued dosing of adult patients in second half of
2023
- U.S. FDA cleared the IND application
for TSHA-102 in pediatric patients with Rett syndrome
- CTA submitted to U.K. MHRA for
TSHA-102 in pediatric patients with Rett syndrome
TSHA-120 for giant axonal neuropathy (GAN): a
self-complementary 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.
- At R&D Day in June 2023, Taysha
provided an overview of new comprehensive data analysis and
development of disease progression model (DPM), which the Company
believes has the potential to address FDA feedback regarding the
heterogeneity of GAN and effort-dependent nature of MFM32 as the
primary endpoint in an unblinded study
- New comprehensive data analysis
utilizing the DPM submitted as meeting request to the FDA; feedback
for a potential regulatory pathway for TSHA-120 expected in Q3
2023
- FDA feedback on CMC module 3
amendment concluded that the analytical data is sufficient to
support the comparability of pivotal lot and release for use in
clinical studies
Second Quarter 2023 Financial Highlights
Research and Development Expenses: Research and
development expenses were $19.8 million for the three months ended
June 30, 2023, compared to $23.5 million for the three months
ending June 30, 2022. The $3.7 million decrease was due to lower
compensation expense as a result of reduced headcount and fewer
manufacturing batches and raw material purchases.
General and Administrative
Expenses: General and administrative expenses were
$6.0 million for the three months ended June 30, 2023, compared to
$9.9 million for the three months ended June 30, 2022. The decrease
of $3.9 million was due to reduced general and administrative
compensation as a result of lower headcount, consulting and
professional fees.
Net loss: Net loss for the three months
ended June 30, 2023 was $24.6 million or $0.38 per share,
as compared to a net loss of $34.1 million,
or $0.85 per share, for the three months ended June
30, 2022.
Cash and cash equivalents: As of June 30,
2023, Taysha had $45.1 million in cash and cash equivalents. Taysha
expects to receive gross proceeds of $150 million from the Private
Placement, which is expected to close August 16, 2023, before
deducting placement agent commissions and offering expenses. The
net proceeds from the private placement, combined with the current
cash and cash equivalents, are expected to fund its operational
plans and capital requirements into the third quarter of 2025.
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today
at 8:30 a.m. 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 13740092. The
live webcast can be accessed here:
https://viavid.webcasts.com/starthere.jsp?ei=1624983&tp_key=25b742b70a. An
archived version of the webcast will be available for 30 days and
can be accessed by visiting Taysha’s website
at https://ir.tayshagtx.com/news-events/events-presentations.
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
patient 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 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, statements associated with the timing, size and completion
of the Private Placement, 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 March 31, 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. 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
Balance Sheet Data(in thousands, except share and
per share data)(Unaudited)
|
June 30, 2023 |
December 31, 2022 |
|
|
|
ASSETS |
|
|
Current assets: |
|
|
Cash and cash equivalents |
$ |
45,083 |
|
$ |
87,880 |
|
Prepaid expenses and other
current liabilities |
|
9,032 |
|
|
8,537 |
|
Total current assets |
|
54,115 |
|
|
96,417 |
|
|
|
|
Restricted cash |
|
2,637 |
|
|
2,637 |
|
Property, plant and equipment,
net |
|
14,139 |
|
|
14,963 |
|
Operating lease right-of-use
assets |
|
10,348 |
|
|
10,943 |
|
Other non-current assets |
|
304 |
|
|
1,316 |
|
Total
assets |
$ |
81,543 |
|
$ |
126,276 |
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' (DEFICIT) EQUITY |
|
|
Current liabilities: |
|
|
Accounts payable |
$ |
10,766 |
|
$ |
10,946 |
|
Accrued expenses and other
current liabilities |
|
19,631 |
|
|
18,287 |
|
Deferred revenue |
|
26,909 |
|
|
33,557 |
|
Total current liabilities |
|
50,641 |
|
|
62,790 |
|
Deferred revenue, net of
current portion |
|
6,212 |
|
|
Term loan, net |
|
38,354 |
|
|
37,967 |
|
Operating lease liability, net
of current portion |
|
19,528 |
|
|
20,440 |
|
Other non-current
liabilities |
|
3,922 |
|
|
4,130 |
|
Total liabilities |
|
118,657 |
|
|
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 June 30, 2023 and December 31, 2022 |
|
- |
|
|
- |
|
|
|
|
Common stock, $0.00001 par
value per share; 200,000,000 shares authorized and 64,432,637 and
63,207,507 issued and outstanding as of June 30, 2023, and December
31, 2022, respectively |
|
1 |
|
|
1 |
|
Additional paid-in
capital |
|
406,546 |
|
|
402,389 |
|
Accumulated deficit |
|
(443,661 |
) |
|
(401,441 |
) |
Total stockholders’ (deficit)
equity |
|
(37,114 |
) |
|
949 |
|
Total liabilities and
stockholders' (deficit) equity |
$ |
81,543 |
|
$ |
126,276 |
|
Taysha Gene Therapies,
Inc.Condensed Consolidated Statements of
Operations(in thousands, except share and per share
data)(Unaudited)
|
For the three months ended June 30, 2023 |
For the three months ended June 30, 2022 |
For the six months ended June 30, 2023 |
For the six months ended June 30, 2022 |
|
|
|
|
|
Revenue: |
|
|
|
|
Service Revenue |
$ |
2,395 |
|
$ |
- |
|
$ |
7,101 |
|
$ |
- |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
19,791 |
|
|
23,506 |
|
|
32,305 |
|
|
61,688 |
|
General and administrative |
|
5,988 |
|
|
9,867 |
|
|
14,739 |
|
|
21,336 |
|
Total operating expenses |
|
25,779 |
|
|
33,373 |
|
|
47,044 |
|
|
83,024 |
|
Loss from
operations |
|
(23,384 |
) |
|
(33,373 |
) |
|
(39,943 |
) |
|
(83,024 |
) |
Other income
(expense): |
|
|
|
|
Interest Income |
|
223 |
|
|
27 |
|
|
542 |
|
|
41 |
|
Interest expense |
|
(1,440 |
) |
|
(743 |
) |
|
(2,814 |
) |
|
(1,415 |
) |
Other expense |
|
3 |
|
|
(3 |
) |
|
(5 |
) |
|
(11 |
) |
Total other income (expense) |
|
(1,214 |
) |
|
(719 |
) |
|
(2,277 |
) |
|
(1,385 |
) |
Net loss |
$ |
(24,598 |
) |
$ |
(34,092 |
) |
$ |
(42,220 |
) |
$ |
(84,409 |
) |
Net loss per common share,
basic and diluted |
$ |
(0.38 |
) |
$ |
(0.85 |
) |
$ |
(0.66 |
) |
$ |
(2.16 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
64,244,531 |
|
|
40,142,403 |
|
|
63,755,435 |
|
|
39,163,996 |
|
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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