Design Therapeutics Reports Pipeline Progress and Third Quarter 2021 Results
November 09 2021 - 4:05PM
Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company
developing treatments for degenerative genetic disorders, today
reported recent pipeline progress and third quarter 2021 financial
results.
“At Design, our mission is to bring a new class
of small-molecule genomic medicines to patients with inherited
degenerative diseases. We’ve taken major strides toward achieving
our goals, with advancements across our pipeline and GeneTACTM
platform, and expansion of our leadership team to support our
expected near-term transition to a clinical-stage organization,”
said João Siffert, M.D., president and chief executive officer of
Design Therapeutics. “We expect 2022 will be a milestone-rich and
transformational year for the company, including initiation of the
clinical program in Friedreich ataxia, progress with our myotonic
dystrophy type-1 GeneTACTM program, and expansion of our pipeline
with several earlier stage programs that represent exciting
opportunities to treat additional diseases caused by nucleotide
repeat expansions.”
Recent Pipeline Highlights
-
Friedreich Ataxia (FA) GeneTACTM
Program On-track for Clinical Initiation in First Half of
2022: In ongoing IND-enabling studies, Design’s FA
GeneTACTM clinical candidate has been shown to be well tolerated in
repeat dose GLP toxicity studies in rats and non-human primates at
doses that exceed what we estimate to be biologically active in the
clinic. Design remains on track to initiate a Phase 1 clinical
trial in patients with FA in the first half of 2022, with initial
topline clinical data expected in 2022.
- Positive
Preclinical Data Reported Highlighting Disease-Modifying Potential
of DM1 GeneTACTM Molecules for
Myotonic Dystrophy Type-1 (DM1): Design reported new
preclinical data from its novel DM1 GeneTACTM program at the 2021
Virtual Myotonic Dystrophy Foundation Annual Conference in
September 2021, including demonstration of near-complete resolution
of disease-causing foci and correction of splicing defects in DM1
patient cells. Preclinical in vivo studies demonstrated
distribution of DM1 GeneTACTM molecules to key target tissues
including skeletal muscle and heart, achieving tissue
concentrations that reduced nuclear foci and corrected mRNA
splicing in our in vitro experiments. These data support the
continued advancement of the DM1 program and underscore the broader
potential of GeneTACTM molecules to treat multiple additional
nucleotide repeat expansion diseases. The company remains on track
to initiate its clinical program in DM1 in 2023.
Upcoming Investor Conference Presentations
-
33rd Annual Virtual Piper
Healthcare Conference: João Siffert, M.D., president and
chief executive officer and Sean Jeffries, Ph.D., chief operating
officer, will participate in a fireside chat during the 33rd Annual
Virtual Piper Healthcare Conference, being held November 30 –
December 2, 2021. The pre-recorded fireside chat will be available
to registered participants beginning on November 22, 2021.
The webcast will be available in the investor section of the
company's website at www.designtx.com and will be archived for 30
days following the presentation.Third Quarter 2021
Financial Results
- Net loss for the third quarter ended
September 30, 2021, was $11.3 million.
- Research and development expenses
for the third quarter of 2021 were $8.5 million.
- General and administrative expenses
for the third quarter of 2021 were $2.8 million.
- As of September 30, 2021,
the company had cash, cash equivalents and investment securities of
$393.7 million.
About Design TherapeuticsDesign
Therapeutics is a biotechnology company developing a new class of
therapies based on its platform of GeneTAC™ gene targeted chimera
small molecules. The company’s GeneTACTM 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; expected timing for
initial topline data; the effectiveness of Design’s GeneTACTM
program in the treatment of Friedreich ataxia and myotonic
dystrophy type-1; the potential advantages of these GeneTACTM
programs; Design’s continued growth, including the expected
expansion of our pipeline and the timing thereof; 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,"
“designed to,” "anticipates," “on track,” "plans," "expects,"
“estimate,” "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 and preclinical studies; 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; Design’s 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 (“SEC”),
including under the “Risk Factors” heading of Design’s Quarterly
Report on Form 10-Q for the quarter ended September 30, 2021, as
filed with the SEC on November 9, 2021. 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.STATEMENTS OF OPERATIONS(in
thousands, except share and per share
data)(unaudited)
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
Grant revenue |
$ |
- |
|
|
$ |
20 |
|
|
$ |
- |
|
|
$ |
193 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
8,539 |
|
|
1,914 |
|
|
17,441 |
|
|
3,352 |
|
General and administrative |
2,798 |
|
|
489 |
|
|
7,263 |
|
|
1,310 |
|
Total operating expenses |
11,337 |
|
|
2,403 |
|
|
24,704 |
|
|
4,662 |
|
Loss from operations |
(11,337 |
) |
|
(2,383 |
) |
|
(24,704 |
) |
|
(4,469 |
) |
Other income, net |
19 |
|
|
63 |
|
|
236 |
|
|
44 |
|
Net loss |
$ |
(11,318 |
) |
|
$ |
(2,320 |
) |
|
$ |
(24,468 |
) |
|
$ |
(4,425 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and diluted |
$ |
(0.21 |
) |
|
$ |
(0.09 |
) |
|
$ |
(0.57 |
) |
|
$ |
(0.17 |
) |
Weighted-average shares of common stock outstanding, basic and
diluted |
55,155,030 |
|
|
25,701,737 |
|
|
42,759,656 |
|
|
25,606,779 |
|
|
|
|
|
|
|
|
|
|
|
|
|
DESIGN THERAPEUTICS, INC. |
|
BALANCE SHEETS |
|
(in thousands) |
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
2021 |
|
2020 |
|
|
(unaudited) |
|
|
|
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash, cash equivalents and investment securities |
$ |
393,718 |
|
$ |
36,091 |
|
Prepaid expense and other current assets |
1,572 |
|
142 |
|
Total current assets |
395,290 |
|
36,233 |
|
Property and equipment, net |
1,320 |
|
71 |
|
Right-of-use asset |
3,743 |
|
— |
|
Deferred offering costs |
— |
|
212 |
|
Total assets |
$ |
400,353 |
|
$ |
36,516 |
|
Liabilities, Convertible Preferred Stock and Stockholders’
Equity (Deficit) |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
$ |
2,216 |
|
$ |
1,399 |
|
Accrued expenses and other current liabilities |
3,289 |
|
931 |
|
Total current liabilities |
5,505 |
|
2,330 |
|
Operating lease liability, net |
3,273 |
|
— |
|
Other long-term liabilities |
11 |
|
145 |
|
Total liabilities |
8,789 |
|
2,475 |
|
Convertible preferred stock |
— |
|
45,356 |
|
Total stockholders’ equity (deficit) |
391,564 |
|
(11,315 |
) |
Total liabilities, convertible preferred stock and stockholders’
equity (deficit) |
$ |
400,353 |
|
$ |
36,516 |
|
|
|
|
|
|
|
|
|
|
|
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