Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle
disease company focused on advancing innovative life-transforming
therapeutics for people living with genetically driven diseases,
today reported financial results for the fourth quarter and full
year 2023 and recent business highlights.
“We’ve had an exciting start to 2024 highlighted by Dyne’s first
clinical data demonstrating proof-of-concept in our DM1 and DMD
programs that validated the promise of the FORCE™ platform in
developing targeted therapeutics for people living with rare muscle
diseases. We were thrilled to see dose-dependent results in our
ACHIEVE trial in DM1 as well as meaningful improvement in myotonia
at the lowest dose, and in our DELIVER trial to exceed the level of
dystrophin production reported for the standard of care for DMD
exon 51 with a fraction of the dose. The strength of these data
formed the foundation of the $345 million offering we completed in
January 2024, which has extended our projected cash runway through
2025,” said Joshua Brumm, president and chief executive
officer of Dyne. “In 2024, we are focused on leveraging the
favorable safety profiles for both DYNE-101 and DYNE-251 and the
adaptive nature of our ACHIEVE and DELIVER trials to optimize dose
and dose regimen. We plan to provide an update from both trials in
the second half of 2024, including reporting data from multiple,
higher dose cohorts. We also continue to pursue expedited
regulatory pathways as part of our commitment to move with a sense
of urgency to deliver potentially transformative therapies for the
DM1 and Duchenne communities.”
Highlights from Clinical Programs
Phase 1/2 ACHIEVE Trial of DYNE-101 in DM1
- In January 2024, Dyne reported
initial clinical data from the Phase 1/2 ACHIEVE trial of DYNE-101
in adult patients with myotonic dystrophy type 1 (DM1).
- The initial efficacy assessment was
based on data from 32 patients enrolled in the randomized,
placebo-controlled multiple ascending dose (MAD) portion of the
trial, including 6-month data from the 1.8 mg/kg (approximate ASO
dose) cohort and 3-month data from the 3.4 mg/kg Q4W cohort.
DYNE-101 demonstrated a dose-dependent splicing correction as well
as an increase in muscle delivery and DMPK knockdown. Patients
treated with 1.8 mg/kg Q4W of DYNE-101 also experienced functional
improvement in myotonia as measured by video hand opening time
(vHOT) and overall improvement in the Myotonic Dystrophy Health
Index (MDHI) patient reported outcome, including the fatigue
subscale, suggesting potential benefit in the central nervous
system.
- Safety and tolerability data were
based on 45 patients enrolled through the 5.4 mg/kg Q8W cohort of
the MAD portion. DYNE-101 demonstrated a favorable safety profile
as of the data cutoff date1.
- The ACHIEVE trial is fully enrolled
through the 5.4 mg/kg Q8W cohort and is currently enrolling
participants in the 6.8 mg/kg Q8W cohort.
Phase 1/2 DELIVER Trial of DYNE-251 in DMD
- In January 2024, Dyne reported
initial clinical data from the Phase 1/2 DELIVER trial of DYNE-251
in Duchenne muscular dystrophy (DMD).
- The initial efficacy assessment was
based on 6-month data from 6 male patients with DMD amenable to
exon 51 skipping enrolled in the 5 mg/kg (approximate PMO dose)
cohort of the randomized, placebo-controlled MAD portion of the
trial. Once every 4-week administration of DYNE-251 reached levels
of dystrophin expression, exon skipping and percent dystrophin
positive fibers that exceeded levels reported in a clinical trial
for the current weekly standard of care for DMD exon 51,
eteplirsen, at 6 months2 with a 24-fold lower total PMO dose.
- Safety and tolerability data were
based on 37 patients enrolled through the 20 mg/kg cohort of the
MAD portion. DYNE-251 demonstrated a favorable safety profile as of
the data cutoff date1.
- The DELIVER trial is fully enrolled
through the 20 mg/kg Q4W cohort and is currently enrolling
participants in the 40 mg/kg Q8W cohort.
Other Business Highlights
- In January 2024, Dyne completed an
underwritten public offering of 19,722,500 shares of its common
stock at a public offering price of $17.50 per share, which
included 2,572,500 shares issued upon the exercise in full by the
underwriters of their option to purchase additional shares of
common stock in the offering. The gross proceeds to Dyne from the
offering were approximately $345.1 million, before deducting
underwriting discounts and commissions and offering expenses
payable by Dyne.
Upcoming Events & Key 2024 Milestones
- Initial data from ACHIEVE and
DELIVER reported in January 2024 will be featured in oral
presentations at the Muscular Dystrophy Association (MDA) Clinical
and Scientific Conference on March 6, 2024.
- Dyne anticipates reporting data from
multiple, higher dose cohorts from both the ACHIEVE and the DELIVER
trials in the second half of 2024 with the goal of initiating
registrational cohorts by the end of 2024.
Fourth Quarter and Full Year 2023 Financial
Results
Cash position: Cash, cash equivalents and
marketable securities were $123.1 million as of December 31, 2023.
In January 2024, the Company completed an underwritten public
offering of 19,722,500 shares of its common stock for net proceeds
of approximately $323.9 million. The Company expects that its cash,
cash equivalents and marketable securities as of December 31, 2023,
together with the net proceeds from the January 2024 underwritten
public offering, will be sufficient to fund its operations through
2025.
Research and development (R&D) expenses:
R&D expenses were $58.8 million and $33.2 million for the
quarters ended December 31, 2023 and 2022, respectively. R&D
expenses were $210.8 million and $142.8 million for the years ended
December 31, 2023 and 2022, respectively.
General and administrative (G&A) expenses:
G&A expenses were $8.8 million and $7.0 million for the
quarters ended December 31, 2023 and 2022, respectively. G&A
expenses were $31.4 million and $28.2 million for the years ended
December 31, 2023 and 2022, respectively.
Net loss: Net loss for the quarter ended
December 31, 2023 was $66.6 million, or $1.09 per basic and diluted
share. This compares with a net loss of $38.8 million, or $0.74 per
basic and diluted share, for the quarter ended December 31, 2022.
Net loss for the year ended December 31, 2023 was $235.9 million,
or $3.95 per basic and diluted share. This compares with a net loss
of $168.1 million, or $3.23 per basic and diluted share, for the
year ended December 31, 2022.
About Dyne Therapeutics
Dyne Therapeutics is a clinical-stage muscle disease company
focused on advancing innovative life-transforming therapeutics for
people living with genetically driven diseases. With its
proprietary FORCE™ platform, Dyne is developing modern
oligonucleotide therapeutics that are designed to overcome
limitations in delivery to muscle tissue. Dyne has a broad pipeline
for serious muscle diseases, including clinical programs for
myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy
(DMD) and a preclinical program for facioscapulohumeral muscular
dystrophy (FSHD). For more information, please
visit https://www.dyne-tx.com, and follow us on X,
LinkedIn and Facebook.
Forward-Looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. All statements, other
than statements of historical facts, contained in this press
release, including statements regarding Dyne’s strategy, future
operations, prospects and plans, objectives of management, the
potential of the FORCE platform, the trial design of the DYNE-101
and DYNE-251 clinical trials, the anticipated timelines for
reporting data from the DYNE-101 and DYNE-251 clinical trials and
initiating registrational cohorts, plans to optimize dose and dose
regimen for DYNE-101 and DYNE-251 and the sufficiency of Dyne’s
cash resources for the period anticipated, constitute
forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. The words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,”
“may,” “might,” “objective,” “ongoing,” “plan,” “predict,”
“project,” “potential,” “should,” or “would,” or the negative of
these terms, or other comparable terminology are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Dyne
may not actually achieve the plans, intentions or expectations
disclosed in these forward-looking statements, and you should not
place undue reliance on these forward-looking statements. Actual
results or events could differ materially from the plans,
intentions and expectations disclosed in these forward-looking
statements as a result of various important factors, including:
uncertainties inherent in the identification and development of
product candidates, including the initiation and completion of
preclinical studies and clinical trials; uncertainties as to the
availability and timing of results from preclinical studies and
clinical trials; the timing of and Dyne’s ability to initiate and
enroll patients in clinical trials; whether results from
preclinical studies and initial data from early clinical trials
will be predictive of the final results of the clinical trials or
future trials; whether Dyne’s cash resources will be sufficient to
fund the Company’s foreseeable and unforeseeable operating expenses
and capital expenditure requirements; as well as the risks and
uncertainties identified in Dyne’s filings with the Securities and
Exchange Commission (SEC), including the Company’s most recent Form
10-K and in subsequent filings Dyne may make with the SEC. In
addition, the forward-looking statements included in this press
release represent Dyne’s views as of the date of this press
release. Dyne anticipates that subsequent events and developments
will cause its views to change. However, while Dyne may elect to
update these forward-looking statements at some point in the
future, it specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing Dyne’s views as of any date subsequent to the date of
this press release.
1. Safety data as of December 6, 20232. No head-to-head trials
have been conducted comparing DYNE-251 to eteplirsen. Eteplirsen
data may not be directly comparable due to differences in trial
protocols, dosing regimens and patient populations. Accordingly,
these cross-trial comparisons may not be reliable. Eteplirsen data
from J Neuromuscul Dis. 2021; 8(6): 989–1001
Dyne Therapeutics, Inc. |
|
Condensed Consolidated Statement of
Operations |
|
(in thousands, except share and per share
data) |
|
|
Three Months Ended |
|
|
Year Ended |
|
|
December 31, |
|
|
December 31, |
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
$ |
58,843 |
|
|
$ |
33,191 |
|
|
$ |
210,762 |
|
|
$ |
142,760 |
|
General and administrative |
|
8,846 |
|
|
|
6,955 |
|
|
|
31,400 |
|
|
|
28,202 |
|
Total operating expenses |
|
67,689 |
|
|
|
40,146 |
|
|
|
242,162 |
|
|
|
170,962 |
|
Loss from operations |
|
(67,689 |
) |
|
|
(40,146 |
) |
|
|
(242,162 |
) |
|
|
(170,962 |
) |
Other (expense) income, net |
|
1,050 |
|
|
|
1,319 |
|
|
|
6,225 |
|
|
|
2,863 |
|
Net loss |
$ |
(66,639 |
) |
|
$ |
(38,827 |
) |
|
$ |
(235,937 |
) |
|
$ |
(168,099 |
) |
Net loss per share, basic and
diluted |
$ |
(1.09 |
) |
|
$ |
(0.74 |
) |
|
$ |
(3.95 |
) |
|
$ |
(3.23 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
61,393,409 |
|
|
|
52,817,413 |
|
|
|
59,683,851 |
|
|
|
51,976,343 |
|
|
Dyne Therapeutics, Inc. |
|
Condensed Consolidated Balance Sheet Data |
|
(in thousands) |
|
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
Assets |
|
|
|
|
|
|
Cash, cash equivalents and marketable securities |
|
$ |
123,100 |
|
|
$ |
256,012 |
|
Other assets |
|
|
41,982 |
|
|
|
50,313 |
|
Total Assets |
|
$ |
165,082 |
|
|
$ |
306,325 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
Liabilities |
|
|
73,790 |
|
|
|
53,961 |
|
Stockholders' equity |
|
|
91,292 |
|
|
|
252,364 |
|
Total liabilities and stockholders' equity |
|
$ |
165,082 |
|
|
$ |
306,325 |
|
|
Contacts:
InvestorsAmy Reillyareilly@dyne-tx.com
857-341-1203
MediaStacy Nartkersnartker@dyne-tx.com
781-317-1938
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