- Initiated global natural history study for
pediatric patients with MYBPC3 mutation to support clinical
development of TN-201 gene therapy
- Presented preclinical data supporting both
TN-201 and PKP2 gene therapy programs at the European Society of
Gene and Cell Therapy conference demonstrating significant and
durable disease reversal and survival in severe murine models of
disease
- Initiated cGMP manufacturing to support the
IND filing for TYA-11631 small molecule for HFpEF
Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology
company with a mission to discover, develop and deliver curative
therapies that address the underlying causes of heart disease,
today reported business and program updates and third quarter 2021
financial results.
“The third quarter of 2021 was a significant marker in Tenaya’s
history with the successful completion of our IPO, and it also
represented a period of growth for the company as we continued to
make progress on our key pipeline programs and operations,” said
Faraz Ali, CEO of Tenaya. “We are pleased to announce the
initiation of our global natural history study for pediatric
patients with MYBPC3 mutations, as this supports our TN-201 gene
therapy program as well as our evolution towards becoming a
clinical-stage company. The initiation of cGMP manufacturing for
TYA-11631 is an important operational milestone and another step in
the direction of our vision for modality-agnostic drug discovery.
The first-ever presentation of data supporting our PKP2 gene
therapy program at an important scientific conference provides new
hope for individuals and families fighting the leading genetic
cause of Arrhythmogenic Right Ventricular Cardiomyopathy, a severe
disease with high unmet need.”
Business and Program Updates
- TN-201 – MYBPC3 Gene Therapy Program for Genetic HCM
(gHCM):
- Pre-clinical data on the treatment in a severe MYBPC3 mutant
mouse model of gHCM with TN-201 demonstrating extended survival to
18 months after a single dose and continued improvement in
functional measures was presented at the annual meeting of the
European Society of Gene and Cell Therapy (ESGCT) in October
2021.
- Tenaya has initiated a global natural history study to improve
our understanding of disease progression and unmet need in
individuals carrying mutations in the MYBPC3 gene, with an initial
focus on pediatric patients under the age of 18. For additional
information about the natural history study, please visit
www.clinicaltrials.gov using Identifier NCT05112237. These efforts
may support the evaluation of Tenaya’s TN-201 gene therapy product
candidate in this patient population during clinical
development.
- Tenaya has initiated IND-enabling activities and intends to
submit an investigational new drug (IND) application or clinical
trial application (CTA) to the U.S. Food and Drug Administration
(FDA) or European Medicine Agency (EMA), respectively, in
2022.
- TYA-11631 – HDAC6 Inhibitor (HDAC6i) Small Molecule for
Heart Failure with Preserved Ejection Fraction (HFpEF)
- Tenaya has initiated cGMP manufacturing activities to support
the production of TYA-11631 at larger scales. Tenaya had previously
announced initiation of IND-enabling activities and intends to
submit an IND to the FDA in 2022.
- PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right
Ventricular Cardiomyopathy (ARVC)
- Pre-clinical data on the treatment of a severe mouse model of
ARVC due to PKP2 gene mutations with Tenaya’s gene therapy product
construct demonstrated prevention of symptoms and continued,
significant improvement in functional measures and survival benefit
at 15 weeks after a single dose as presented in a poster at ESGCT.
Mutations due to the PKP2 gene are the leading genetic cause of
ARVC, a severe disease estimated to effect more than 70,000
patients in the US alone and with no approved disease-specific
therapies.
- Strong balance sheet with net proceeds of $188.5 million
from the sale of 13.8 million shares of its common stock in the
Company’s initial public offering (IPO), bringing cash, cash
equivalents and investments in marketable securities to $280.5
million as of September 30, 2021.
Third Quarter 2021 Financial Highlights
- Cash Position: As of September 30, 2021, Tenaya had
cash, cash equivalents and investments in marketable securities of
$280.5 million which included net proceeds from the Company’s IPO
in August 2021. Tenaya expects its current cash, cash equivalents
and investments in marketable securities will be sufficient to fund
its current operating plan at least into the second half of
2023.
- Research & Development (R&D) Expenses: R&D
expenses were $12.9 million for the third quarter of 2021. Non-cash
stock-based compensation included in R&D expense was $0.3
million for the third quarter of 2021.
- General & Administrative (G&A) Expenses: G&A
expenses were $5.4 million for the third quarter of 2021. Non-cash
stock-based compensation included in G&A expense was $0.6
million for the third quarter of 2021.
- Net Loss: Net loss was $18.3 million, or $0.68 on a per
share basis, for the third quarter of 2021.
About Tenaya Therapeutics
Tenaya Therapeutics is a biotechnology company committed to a
bold mission: to discover, develop and deliver curative therapies
that address the underlying drivers of heart disease. Founded by
leading cardiovascular scientists from Gladstone Institutes and the
University of Texas Southwestern Medical Center, Tenaya is
developing therapies for rare genetic disorders as well as for more
prevalent heart conditions through three distinct but interrelated
product platforms: Gene Therapy, Cellular Regeneration and
Precision Medicine. For more information, visit
www.tenayatherapeutics.com.
Forward Looking Statements
This press release contains forward-looking statements as that
term is defined in Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. Statements in
this press release that are not purely historical are
forward-looking statements. Such forward-looking statements
include, among other things, statements regarding the potential of
and expectations regarding Tenaya’s product candidates and
programs, including TN-201 and TYA-11631; statements regarding the
expected timing of IND and CTA submissions to the FDA or EMA,
respectively, for Tenaya’s product candidates; statements regarding
the sufficiency of Tenaya’s cash, cash equivalents and investments
in marketable securities to fund its operating plan; and statements
by Tenaya’s chief executive officer. Words such as “expects,”
“intends,” “potential,” “utility,” and “will,” and similar
expressions are intended to identify forward-looking statements.
The forward-looking statements contained herein are based upon
Tenaya’s current expectations and involve assumptions that may
never materialize or may prove to be incorrect. Actual results
could differ materially from those projected in any forward-looking
statements due to numerous risks and uncertainties, including but
not limited to: risks associated with the process of discovering,
developing and commercializing drugs that are safe and effective
for use as human therapeutics and operating as an early clinical
stage company including the potential for Tenaya’s product
candidates to cause serious adverse events; Tenaya’s ability to
develop, initiate or complete preclinical studies and clinical
trials for, obtain approvals for and commercialize any of its
product candidates for heart failure patients or other patient
populations; the timing, progress and results of preclinical
studies and clinical trials for TN-201 and TYA-11631 and Tenaya’s
other product candidates and programs; Tenaya’s ability to raise
any additional funding it will need to continue to pursue its
business and product development plans; negative impacts of the
COVID-19 pandemic on Tenaya’s manufacturing and operations,
including preclinical studies and clinical trials; the timing,
scope and likelihood of regulatory filings and approvals; the
potential for any clinical trial results to differ from
preclinical, interim, preliminary, topline or expected results;
Tenaya’s ability to develop a proprietary drug discovery platform
to build a pipeline of product candidates; Tenaya’s manufacturing,
commercialization and marketing capabilities and strategy; the loss
of key scientific or management personnel; competition in the
industry in which Tenaya operates; Tenaya’s reliance on third
parties; Tenaya’s ability to obtain and maintain intellectual
property protection for its product candidates; general economic
and market conditions; and other risks. Information regarding the
foregoing and additional risks may be found in the section entitled
“Risk Factors” in documents that Tenaya files from time to time
with the Securities and Exchange Commission. These forward-looking
statements are made as of the date of this press release, and
Tenaya assumes no obligation to update the forward-looking
statements, or to update the reasons why actual results could
differ from those projected in the forward-looking statements,
except as required by law.
TENAYA THERAPEUTICS, INC.
Condensed Statements of Operations and
Comprehensive Loss (In thousands, except share and per share
data) (Unaudited)
Three Months Ended
September 30,
Nine Months Ended
September 30,
2021
2020
2021
2020
Operating expenses:
Research and development
$
12,944
$
8,480
$
33,440
$
22,735
General and administrative
5,356
1,972
13,202
5,855
Total operating expenses
18,300
10,452
46,642
28,590
Loss from operations
(18,300
)
(10,452
)
(46,642
)
(28,590
)
Other income (expense), net:
Interest income
23
7
41
82
Change in fair value of convertible
preferred stock tranche liability
—
151
—
75
Other income (expense), net
15
(1
)
31
356
Total other income (expense), net
38
157
72
513
Net loss before income tax expense
(18,262
)
(10,295
)
(46,570
)
(28,077
)
Income tax expense
—
—
—
—
Net loss
(18,262
)
(10,295
)
(46,570
)
(28,077
)
Other comprehensive loss:
Unrealized loss on marketable
securities
(23
)
—
(23
)
—
Comprehensive loss
$
(18,285
)
$
(10,295
)
$
(46,593
)
$
(28,077
)
Net loss per share, basic and diluted
$
(0.68
)
$
(10.29
)
$
(4.75
)
$
(29.65
)
Weighted-average shares used in computing
net loss per share, basic and diluted
26,895,716
1,000,052
9,808,162
947,009
TENAYA THERAPEUTICS, INC.
Condensed Balance Sheets (In
thousands)
September 30,
December 31,
2021
2020
ASSETS
(Unaudited)
Current assets:
Cash and cash equivalents
$
117,077
$
128,535
Investments in marketable securities
163,388
—
Prepaid expenses and other current
assets
4,357
1,429
Total current assets
284,822
129,964
Property and equipment, net
30,891
17,185
Operating lease right-of-use assets
12,006
—
Restricted cash, non-current
547
547
Other non-current assets
3,528
465
Total assets
$
331,794
$
148,161
LIABILITIES, CONVERTIBLE PREFERRED
STOCK AND STOCKHOLDERS’ EQUITY (DEFICIT)
Current liabilities:
Accounts payable
$
6,171
$
1,017
Accrued expenses and other current
liabilities
5,607
3,161
Deferred rent and other lease liabilities,
current
—
863
Operating lease liabilities, current
1,914
—
Total current liabilities
13,692
5,041
Deferred rent and other lease liabilities,
non-current
—
3,662
Operating lease liabilities,
non-current
14,307
—
Other non-current liabilities
96
19
Total liabilities
28,095
8,722
Commitments and contingencies
Convertible preferred stock
—
220,754
Stockholders’ equity (deficit):
Common stock
4
—
Additional paid-in capital
433,112
1,584
Notes receivable from stockholders
(12
)
(87
)
Accumulated other comprehensive loss
(23
)
—
Accumulated deficit
(129,382
)
(82,812
)
Total stockholders’ equity (deficit)
303,699
(81,315
)
Total liabilities, convertible preferred
stock and stockholders’ equity (deficit)
$
331,794
$
148,161
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211110006154/en/
Investors Leone Patterson Tenaya Therapeutics
IR@tenayathera.com
Media Kit Rodophele Ten Bridge Communications
krodophele@tenbridgecommunications.com
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