Design Therapeutics Reports GeneTAC™ Portfolio Progress and Second Quarter 2021 Results
August 09 2021 - 4:05PM
Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company
developing treatments for degenerative genetic disorders, today
reported recent progress with its portfolio of novel small molecule
gene targeted chimeras (GeneTACsTM), as well as business highlights
and second quarter 2021 financial results.
“So far in 2021, we’ve made substantial progress
as a company, highlighted by the compelling new data from ongoing
IND-enabling studies with our lead GeneTAC program for Friedreich
ataxia. Importantly, we’ve observed well-tolerated GeneTAC doses in
rodents and non-human primates that produced ample biodistribution
into key tissues affected by the disease, including the brain,
increasing our confidence in the potential of this program as a
disease-modifying treatment for patients,” said João Siffert, M.D.,
president and chief executive officer of Design Therapeutics. “With
plans to begin clinical development in the first half of 2022, we
are pleased to welcome Dr. Elizabeth Gordon to the team, who brings
decades of valuable experience in overseeing US and ex-US
regulatory affairs and successful regulatory submissions.”
“Design is well positioned to advance our
research and development activities targeting a number of
nucleotide repeat expansion diseases, enabled by a talented team
that includes multiple new additions in our R&D organization
and a strong balance sheet to fuel our pipeline of novel GeneTAC
programs,” added Pratik Shah, Ph.D., co-founder and executive chair
of Design Therapeutics. “We are now preparing for the important
transition to a clinical-stage organization, bringing us another
step closer to delivering a new class of genomic medicines for a
range of serious disorders currently without approved
treatments.”
Pipeline Progress
- New Data from IND-enabling Studies with GeneTAC Product
Candidate for Friedreich Ataxia (FA) Support Initiation of Clinical
Trial in First Half of 2022: Data from IND-enabling
studies in rodents and non-human primates showed that multidose
systemic administration of the company’s FA GeneTAC was well
tolerated and achieved higher concentrations in the CNS (cerebrum,
cerebellum, brainstem and spinal cord), heart, and skeletal muscle
than needed to restore frataxin (FXN) gene expression. In addition,
the company observed that exposure to low nanomolar (nM)
concentrations of its FA GeneTAC in neurons and cardiomyocytes
derived from FA patient stem cells in in vitro experiments led to
robust and durable increases in FXN mRNA, as well as an increase in
endogenous protein reaching levels comparable to unaffected
individuals.
- On-track for FA GeneTAC Initiation of Clinical Trial in
First Half of 2022: Design has received scientific advice
from the European Medicines Agency consistent with the favorable
feedback previously received from the U.S. Food and Drug
Administration (FDA), supporting the development plan for its FA
GeneTAC. In addition, the company has successfully manufactured
both drug substance and product at a scale sufficient for clinical
use. Design anticipates initiating a Phase 1 clinical trial in
patients with Friedreich ataxia in the first half of 2022, with
initial topline clinical data expected in the second half of
2022.
- Continued Progress across
GeneTAC Portfolio with Myotonic Dystrophy with Preclinical Data to
be Reported in Second Half of 2021: Design has continued
to advance its second GeneTAC program focused on treating myotonic
dystrophy type-1 (DM1), a genetic disorder that causes progressive
muscle weakness and for which there are currently no approved
treatment options. The company plans to report preclinical data
supporting its potential to develop a disease-modifying treatment
for patients with DM1 at a medical meeting in the second half of
2021. In addition, leveraging its GeneTAC platform, Design is
progressing several earlier-stage programs targeting diseases
caused by inherited nucleotide repeat expansions, which represent
significant pipeline opportunities to address serious unmet medical
needs.
Business Highlights
- Leadership Team
Strengthened with Appointment of Elizabeth Gordon, Ph.D. as Senior
Vice President of Regulatory Affairs: Dr. Gordon is a
recognized expert in regulatory affairs with over 30 years of
experience in the pharmaceutical industry and at the FDA. Dr.
Gordon most recently served as senior vice president of regulatory
affairs at Amplyx Pharmaceuticals, and before that as vice
president, regulatory affairs at Shire Pharmaceutics and vice
president of regulatory affairs at Lumena Pharmaceuticals. Earlier
in her career, Dr. Gordon served in the Center for Biologics
Evaluation and Research and the Center for Drug Evaluation and
Research at the FDA, where she was instrumental in developing
policy for the regulation of biological products. Dr. Gordon earned
her Ph.D. in microbiology from the University of Rhode Island.
- Board of Directors Expanded
with Key Appointments: In June, Design further enhanced
its board with the appointments of industry veterans, Heather
Behanna, Ph.D., principal of SR One, and Deepa Prasad, managing
director of WestRiver Group, as directors of the company.
Second Quarter 2021 Financial
Results
- Net loss for the second quarter
ended June 30, 2021 was $7.6
million.
- Research and development expenses
for the second quarter of 2021 were $5.0 million.
- General and administrative expenses
for the second quarter of 2021 were $2.7 million.
- As of June 30, 2021, the company had cash, cash
equivalents and investment securities of $402.8 million.
About Design TherapeuticsDesign
Therapeutics is a biotechnology company developing a new class of
therapies based on a platform of gene targeted chimera (GeneTAC™)
small molecules. The company’s GeneTAC molecules are designed to
either turn on or turn off a specific disease-causing gene to
address the underlying cause of disease. Design’s lead program is
focused on the treatment of Friedreich ataxia, followed by a
program in myotonic dystrophy type-1 and discovery efforts for
multiple other serious degenerative disorders caused by nucleotide
repeat expansions. For more information, please visit
designtx.com.
Forward Looking
StatementsStatements in this press release that are not
purely historical in nature are "forward-looking statements" within
the meaning of the Private Securities Litigation Reform Act of
1995. These statements include, but are not limited to, statements
related to: the progress and expected timing of Design’s
development programs and any clinical trials; the effectiveness of
Design’s GeneTAC program in the treatment of Friedreich ataxia and
myotonic dystrophy type-1; the potential advantages of these
GeneTAC programs; Design’s continued growth; and the strength of
Design’s balance sheet and the adequacy of cash on hand. Because
such statements are subject to risks and uncertainties, actual
results may differ materially from those expressed or implied by
such forward-looking statements. Words such as "believes,"
"anticipates," "plans," "expects," "intends," "will," "goal,"
"potential" and similar expressions are intended to identify
forward-looking statements. These forward-looking statements are
based upon Design’s current expectations and involve assumptions
that may never materialize or may prove to be incorrect. Actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include, without limitation,
risks associated with the process of discovering, developing and
commercializing therapies that are safe and effective for use as
human therapeutics and operating as a development stage company;
Design’s ability to develop, initiate or complete preclinical
studies and clinical trials for, obtain approvals for and
commercialize any of its product candidates; the risk that
promising early research or clinical trials do not demonstrate
safety and/or efficacy in later preclinical studies or clinical
trials; preliminary or expected results; changes in Design’s plans
to develop and commercialize its product candidates; the risk that
Design may not obtain approval to market its product candidates;
uncertainties associated with performing clinical trials,
regulatory filings and applications; risks associated with reliance
on third parties to successfully conduct clinical trials; changes
in Design’s plans to develop and commercialize its product
candidates; Design’s ability to raise any additional funding it
will need to continue to pursue its business and product
development plans; regulatory developments in the United States and
foreign countries; Design’s reliance on key third parties,
including contract manufacturers and contract research
organizations; Design’s ability to obtain and maintain intellectual
property protection for its product candidates; our ability to
recruit and retain key scientific or management personnel;
competition in the industry in which Design operates; and market
conditions. For a more detailed discussion of these and other
factors, please refer to Design’s filings with the Securities and
Exchange Commission. You are cautioned not to place undue reliance
on these forward-looking statements, which speak only as of the
date hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement and Design undertakes no
obligation to revise or update this press release to reflect events
or circumstances after the date hereof, except as required by
law.
Contact: Chelcie
Lister THRUST
Strategic
Communications (910)
777-3049 chelcie@thrustsc.com
DESIGN THERAPEUTICS,
INC.CONDENSED STATEMENTS OF
OPERATIONS
(in thousands, except share and per share
data)(unaudited)
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Grant revenue |
$ |
— |
|
|
$ |
31 |
|
|
$ |
— |
|
|
$ |
173 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
5,027 |
|
|
|
1,061 |
|
|
|
8,902 |
|
|
|
1,438 |
|
General and administrative |
|
2,660 |
|
|
|
433 |
|
|
|
4,465 |
|
|
|
821 |
|
Total operating expenses |
|
7,687 |
|
|
|
1,494 |
|
|
|
13,367 |
|
|
|
2,259 |
|
Loss from operations |
|
(7,687 |
) |
|
|
(1,463 |
) |
|
|
(13,367 |
) |
|
|
(2,086 |
) |
Other income (expense), net |
|
51 |
|
|
|
21 |
|
|
|
217 |
|
|
|
(19 |
) |
Net loss |
$ |
(7,636 |
) |
|
$ |
(1,442 |
) |
|
$ |
(13,150 |
) |
|
$ |
(2,105 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
$ |
(0.14 |
) |
|
$ |
(0.06 |
) |
|
$ |
(0.36 |
) |
|
$ |
(0.08 |
) |
Weighted-average shares of common
stock outstanding, basic and diluted |
|
55,081,397 |
|
|
|
25,597,154 |
|
|
|
36,459,244 |
|
|
|
25,558,779 |
|
|
|
DESIGN THERAPEUTICS,
INC.CONDENSED BALANCE SHEETS
(in
thousands)(unaudited)
|
June 30, |
|
|
December 31, |
|
|
2021 |
|
|
2020 |
|
|
(unaudited) |
|
|
|
|
|
Assets |
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
Cash, cash equivalents and investment securities |
$ |
402,836 |
|
|
$ |
36,091 |
|
Prepaid expense and other current assets |
|
2,039 |
|
|
|
142 |
|
Total current assets |
|
404,875 |
|
|
|
36,233 |
|
Property and equipment, net |
|
271 |
|
|
|
71 |
|
Deferred offering costs |
|
— |
|
|
|
212 |
|
Total assets |
$ |
405,146 |
|
|
$ |
36,516 |
|
Liabilities, Convertible
Preferred Stock and Stockholders’ Equity (Deficit) |
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Accounts payable |
$ |
1,585 |
|
|
$ |
1,399 |
|
Accrued expenses |
|
1,820 |
|
|
|
931 |
|
Total current liabilities |
|
3,405 |
|
|
|
2,330 |
|
Other long-term liabilities |
|
140 |
|
|
|
145 |
|
Total liabilities |
|
3,545 |
|
|
|
2,475 |
|
Convertible preferred stock |
|
— |
|
|
|
45,356 |
|
Total stockholders’ equity
(deficit) |
|
401,601 |
|
|
|
(11,315 |
) |
Total liabilities, convertible preferred stock and stockholders’
equity (deficit) |
$ |
405,146 |
|
|
$ |
36,516 |
|
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