Dyne Therapeutics Reports Third Quarter 2023 Financial Results and Provides Update on Significant Progress for ACHIEVE and DELIVER Trials and Upcoming Clinical Milestones
30 Outubro 2023 - 8:30AM
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 third quarter of 2023 and
provided an update on progress and upcoming milestones for its
Phase 1/2 ACHIEVE and DELIVER clinical trials.
“We’ve made tremendous progress in our ACHIEVE and DELIVER
trials with more than 72 patients enrolled and over 300 doses
administered thus far. To date, the safety profile in the clinic
has been favorable for DYNE-101 and DYNE-251 and has supported dose
escalation to a combined nine cohorts across both trials. The
enthusiasm amongst the DM1 and DMD communities reflects the
significant unmet need and the potential for our investigational
therapies to deliver transformative outcomes for patients,” said
Joshua Brumm, president and chief executive officer of Dyne. “We
are moving with great excitement towards reporting initial data
from ACHIEVE and DELIVER, including the important biomarker of
splicing and functional outcome of myotonia in DM1 and dystrophin
in DMD. We look forward to sharing both readouts around the time of
the J.P. Morgan Healthcare Conference in early January 2024.”
ACHIEVE Trial of DYNE-101 in DM1
Clinical Update
- ACHIEVE is a Phase 1/2 global clinical trial evaluating
DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1) who
are 18 to 49 years of age. ACHIEVE, which is designed to be a
registrational trial, consists of a 24-week multiple ascending dose
(MAD), randomized, placebo-controlled period, a 24-week open-label
extension (OLE) and a 96-week long-term extension. The primary
endpoints are safety and tolerability, with secondary endpoints of
pharmacokinetics and pharmacodynamics, including change from
baseline in splicing, as well as measures of muscle strength and
function.
- In the MAD portion of ACHIEVE:
- Enrollment is complete in the 1.8 mg/kg (approximate ASO dose)
cohort (n=16) evaluating once every four-week dosing and the 3.4
mg/kg cohort evaluating once every four-week dosing (n=16) and
every eight-week dosing (n=8). A total of 40 patients have been
enrolled in these two cohorts with over 150 doses administered to
date.
- All patients who completed the 24-week MAD portion of ACHIEVE
have entered the 24-week OLE.
- The safety profile of DYNE-101 supported dose escalation to
cohort 3 (up to 6.8 mg/kg), which is currently enrolling
participants. Per the protocol, further dose escalation to cohort 4
(up to 10.2 mg/kg) will be based on review of safety data from
cohort 3, when available, and the trial overall.
- To date, no participants have demonstrated treatment-emergent
anemia and there have been no discontinuations.
Planned Reporting of Initial Data
- In early January 2024 at a company event around the time of the
42nd Annual J.P. Morgan Healthcare Conference, Dyne anticipates
reporting from the ACHIEVE trial:
- Safety and tolerability data from multiple cohorts.
- Splicing data and analysis of the video hand opening time
(vHOT) myotonia functional assessment from at least the 1.8 mg/kg
cohort at 6 months.
DELIVER Trial of DYNE-251 in DMD
Clinical Update
- DELIVER is a Phase 1/2 global clinical trial evaluating
DYNE-251 in ambulant and non-ambulant males with Duchenne muscular
dystrophy (DMD) who are ages 4 to 16 and have mutations amenable to
exon 51 skipping. DELIVER, which is designed to be a registrational
trial, consists of a 24-week MAD, randomized, placebo-controlled
period, a 24-week OLE and a 96-week long-term extension. The
primary endpoints are safety, tolerability and change from baseline
in dystrophin levels as measured by Western blot. Secondary
endpoints include measures of muscle function, exon skipping and
pharmacokinetics.
- In the MAD portion of DELIVER:
- Enrollment is complete through the 10 mg/kg (approximate PMO
dose) cohort evaluating once every four-week dosing. A total of 32
patients have been enrolled across the first five cohorts (0.7
mg/kg (n=6), 1.4 mg/kg (n=6), 2.8 mg/kg (n=6), 5 mg/kg (n=6) and 10
mg/kg (n=8)) with over 175 doses administered to date.
- All patients who completed the 24-week MAD portion of DELIVER
have entered the 24-week OLE.
- The safety profile of DYNE-251 supported dose escalation to
cohort 6 (up to 20 mg/kg), which is currently enrolling
participants. Per the protocol, further dose escalation to cohort 7
(up to 40 mg/kg) will be based on review of safety data from cohort
6, when available, and the trial overall.
- To date, no participants have demonstrated treatment-emergent
anemia and there have been no discontinuations.
Planned Reporting of Initial Data
- In early January 2024 at a company event around the time of the
42nd Annual J.P. Morgan Healthcare Conference, Dyne anticipates
reporting from the DELIVER trial:
- Safety and tolerability data from multiple cohorts.
- Dystrophin expression data from the 5 mg/kg cohort at 6
months.
Third Quarter 2023 Financial Results
Cash position: Cash, cash equivalents and
marketable securities were $157.8 million as of September 30, 2023,
which is anticipated to fund operations through 2024.
Research and development (R&D) expenses:
R&D expenses were $55.3 million for the quarter ended September
30, 2023, compared to $34.7 million for the quarter ended September
30, 2022.
General and administrative (G&A) expenses:
G&A expenses were $7.0 million for the quarter ended September
30, 2023, compared to $7.6 million for the quarter ended September
30, 2022.
Net loss: Net loss for the quarter ended
September 30, 2023 was $60.2 million, or $0.99 per basic and
diluted share. This compares with a net loss of $41.4 million, or
$0.80 per basic and diluted share, for the quarter ended September
30, 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 anticipated timelines for
reporting data from the DYNE-251 and DYNE-101 clinical trials, the
trial design of the DYNE-251 and DYNE-101 clinical trials, and the
sufficiency of Dyne’s existing 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 clinical trials will be predictive of the
results of later preclinical studies and clinical 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-Q 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.
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Dyne Therapeutics, Inc. |
|
Condensed Consolidated Statement of Operations
(Unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
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Three Months EndedSeptember 30, |
|
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Nine Months EndedSeptember 30, |
|
|
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2023 |
|
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2022 |
|
|
2023 |
|
|
2022 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
55,251 |
|
|
$ |
34,670 |
|
|
$ |
151,918 |
|
|
$ |
109,570 |
|
General and administrative |
|
|
7,022 |
|
|
|
7,609 |
|
|
|
22,556 |
|
|
|
21,247 |
|
Total operating expenses |
|
|
62,273 |
|
|
|
42,279 |
|
|
|
174,474 |
|
|
|
130,817 |
|
Loss from operations |
|
|
(62,273 |
) |
|
|
(42,279 |
) |
|
|
(174,474 |
) |
|
|
(130,817 |
) |
Other (expense) income, net |
|
|
2,063 |
|
|
|
894 |
|
|
|
5,175 |
|
|
|
1,545 |
|
Net loss |
|
$ |
(60,210 |
) |
|
$ |
(41,385 |
) |
|
$ |
(169,299 |
) |
|
$ |
(129,272 |
) |
Net loss per share—basic and
diluted |
|
$ |
(0.99 |
) |
|
$ |
(0.80 |
) |
|
$ |
(2.86 |
) |
|
$ |
(2.50 |
) |
Weighted-average common shares
outstanding used in net loss per share—basic and
diluted |
|
|
61,109,917 |
|
|
|
51,795,446 |
|
|
|
59,107,795 |
|
|
|
51,692,899 |
|
|
|
|
|
Dyne Therapeutics, Inc. |
|
Condensed Consolidated Balance Sheet Data
(Unaudited) |
|
(in thousands) |
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
2023 |
|
2022 |
|
Assets |
|
|
|
|
|
Cash, cash equivalents and marketable securities |
|
$ |
157,823 |
|
$ |
256,012 |
|
Other assets |
|
|
47,421 |
|
|
50,313 |
|
Total assets |
|
$ |
205,244 |
|
$ |
306,325 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
Liabilities |
|
|
53,206 |
|
|
53,961 |
|
Stockholders’ equity |
|
|
152,038 |
|
|
252,364 |
|
Total liabilities and stockholders’ equity |
|
$ |
205,244 |
|
$ |
306,325 |
|
Contacts:
Investors Amy Reilly areilly@dyne-tx.com
857-341-1203
Media Stacy Nartker snartker@dyne-tx.com
781-317-1938
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