Initiation of global Phase 3
HARBOR™ trial for del-desiran (AOC 1001) in
DM1 on track for this quarter
Avidity to report FSHD data from
FORTITUDE™ trial this quarter and DMD data from
EXPLORE44™ trial in 2H24
Presented positive long-term del-desiran data
from MARINA-OLE™ showing reversal of disease progression in
people living with myotonic dystrophy type 1 across multiple
endpoints; same key endpoints, with vHOT as primary, agreed for
phase 3 HARBOR trial
Cash position of $915
million at the end of Q124 following successful private
placement
SAN
DIEGO, May 9, 2024 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), today reported
financial results for the first quarter ended March 31, 2024, and highlighted recent corporate
progress.
"As we move forward with the planned initiation of our global
Phase 3 HARBOR™ study for del-desiran this quarter, we continue to
work diligently to improve people's lives by advancing our AOC
platform," said Sarah Boyce,
president and chief executive officer at Avidity. "In March, we
announced new long-term del-desiran data from our MARINA-OLE study
showing reversal of disease progression in people living with
DM1 across multiple endpoints including video hand opening time
(vHOT), muscle strength and activities of daily living when
compared to END-DM1 natural history data. Importantly, we also
secured agreement with global regulators on the inclusion of these
endpoints in the HARBOR study, including vHOT as the primary
endpoint. We look forward to sharing preliminary data in
approximately half of the study participants in our Phase 1/2
FORTITUDE™ trial in people living with FSHD this quarter and 5
mg/kg cohort data from our Phase 1/2 EXPLORE44™ trial of
people living with DMD44 in the second half of this year."
"Following an oversubscribed equity raise of $400 million, our cash position of $915 million at the end of the first quarter
provides us with funding into late 2026," said Mike MacLean, chief financial officer and chief
business officer at Avidity. "With our positive long-term data for
del-desiran and two additional data readouts planned from our FSHD
and DMD44 clinical programs this year, we continue to make
significant progress in advancing our clinical programs."
Recent Highlights
- Avidity announced that it received Breakthrough Therapy
designation from the FDA for del-desiran for the treatment of
DM1
- The company announced it accelerated the global Phase 3
HARBORTM study initiation to Q2 2024 following agreement
with multiple regulators on study design. The primary endpoint is
video hand opening time (vHOT) and key secondary endpoints include
muscle strength and activities of daily living
- Avidity introduced delpacibart etedesiran as the approved
international nonproprietary name of AOC 1001, abbreviated as
del-desiran
- Presented positive del-desiran long-term 4 mg/kg data from
MARINA-OLE™ study in March 2024. Data
showed:
- Reversal of disease progression in people living with myotonic
dystrophy type 1 (DM1) across multiple endpoints including vHOT,
muscle strength and activities of daily living when compared to
END-DM1 natural history data
- Consistent and durable improvements in the following:
- Myotonia (video hand opening time, or vHOT)
- Multiple measures of strength:
- Hand grip
- Quantitative Muscle Testing (QMT) total score which includes
hand grip; elbow extension and elbow flexion; knee extension and
knee flexion, and ankle dorsiflexion
- DM1-Activ, a patient reported outcome (PRO) that measures
activities of daily living (e.g., taking a shower, visiting family
or friends, and walking up stairs)
- With over 265 infusions totaling 61.1 patient-years of
exposure, del-desiran continues to demonstrate favorable safety and
tolerability
- The FDA granted AOC 1044 with Rare Pediatric Disease
Designation in February 2024 for
DMD44
Upcoming Milestones
- Upcoming anticipated milestones include:
- In the second quarter of 2024, initiation of global Phase 3
HARBOR™ trial of del-desiran for adults living with DM1
- In the second quarter of 2024, share preliminary data in
approximately half of participants in the Phase 1/2 FORTITUDE™
trial of AOC 1020 in people living with FSHD
- In the second half of 2024, share 5 mg/kg cohort data from the
Phase 1/2 EXPLORE44™ trial of AOC 1044 in people living with
DMD44
First Quarter 2024 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash,
cash equivalents and marketable securities totaled $915.9 million as of March
31, 2024, which reflects a gross $400
million raise from a private placement.
- Collaboration Revenue: Collaboration revenue relates to
Avidity's research collaboration and license partnerships.
Collaboration revenues of $3.5
million in the first quarter of 2024 related to partnerships
with Bristol Myers Squibb and Eli Lilly and Company as compared to
$2.2 million related to a partnership
with Eli Lilly and Company in the first quarter of 2023. There was
no revenue related to the partnership with Bristol Myers Squibb in
2023.
- Research and Development (R&D) Expenses: R&D
expenses include external and internal costs associated with
research and development activities. These expenses were
$66.8 million for the first quarter
of 2024 compared with $47.8 million
for the first quarter of 2023. The increases were primarily driven
by the advancement of del-desiran, AOC 1020 and AOC 1044, as well
as internal and external costs related to the expansion of the
company's overall research capabilities.
- General and Administrative (G&A) Expenses:
G&A expenses primarily consist of employee-related expenses,
professional fees, insurance costs and patent filing and
maintenance fees. These expenses were $13.9
million for the first quarter of 2024 compared with
$12.1 million for the first quarter
of 2023. The increases were primarily due to higher personnel costs
to support the company's expanded operations.
About Avidity
Avidity Biosciences, Inc.'s mission is
to profoundly improve people's lives by delivering a new class of
RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™).
Avidity is focused on revolutionizing the field of RNA with its
proprietary AOCs, which are designed to combine the specificity of
monoclonal antibodies with the precision of oligonucleotide
therapies to address targets and diseases previously unreachable
with existing RNA therapies. Utilizing its proprietary AOC
platform, Avidity demonstrated the first-ever successful targeted
delivery of RNA into muscle and is leading the field with clinical
development programs for three rare muscle diseases: myotonic
dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and
facioscapulohumeral muscular dystrophy (FSHD). Avidity is
broadening the reach of AOCs with its advancing and expanding
pipeline including programs in cardiology and immunology through
internal discovery efforts and key partnerships. Avidity is
headquartered in San Diego, CA.
For more information about our AOC platform, clinical development
pipeline and people, please visit
www.aviditybiosciences.com and engage with us on
LinkedIn and X.
Forward-Looking Statements
Avidity cautions readers
that statements contained in this press release regarding matters
that are not historical facts are forward-looking statements. These
statements are based on the company's current beliefs and
expectations. Such forward-looking statements include, but are not
limited to, statements regarding: plans to initiate the global
Phase 3 HARBOR™ trial of del-desiran for people living with DM1;
the anticipated release of data from the FORTITUDE™ and
EXPLORE44™ trials, including the timing thereof and cohort dosage
data; the characterization of data associated with del-desiran from
the MARINA-OLE™ study; plans for the progression of research and
development initiatives, including in cardiology and immunology;
Avidity's financial position, cash balance and ability to fund its
operations; Avidity's growth and related needs; and the potential
of AOCs to target a range of different cells and tissues beyond the
liver, and to treat cardiac and immunological diseases.
The inclusion of forward-looking statements should not be
regarded as a representation by Avidity that any of these plans
will be achieved. Actual results may differ from those set forth in
this press release due to the risks and uncertainties inherent in
Avidity's business and beyond its control, including, without
limitation: Avidity may not be able to resolve the partial clinical
hold related to the serious adverse event which occurred in the
Phase 1/2 MARINA® trial; additional data related to
Avidity's current clinical programs that continues to become
available may be inconsistent with the data produced as of the
respective data cutoff dates, further analysis of existing data and
analysis of new data may lead to conclusions different from those
established as of the date hereof, and such data may not meet
Avidity's expectations; unexpected adverse side effects to, or
inadequate efficacy of, Avidity's product candidates that may delay
or limit their development, regulatory approval and/or
commercialization; later developments with the FDA and other global
regulators that could be inconsistent with the feedback received to
date regarding the proposed design and protocol for the Phase 3
HARBOR trial; Avidity's approach to the discovery and development
of product candidates based on its AOC platform is unproven;
potential delays in the commencement, enrollment, data readouts and
completion of preclinical studies or clinical trials; the success
of its preclinical studies and clinical trials for the company's
product candidates; Avidity's dependence on third parties in
connection with preclinical and clinical testing and product
manufacturing; Avidity may not realize the expected benefits of its
collaborations; regulatory developments in the United States and foreign countries;
Avidity could exhaust its available capital resources sooner than
it currently expects; and other risks described in Avidity's Annual
Report on Form 10-K for the fiscal year ended December 31, 2023 and subsequent filings with the
SEC. Avidity cautions readers not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof,
and the company undertakes no obligation to update such statements
to reflect events that occur or circumstances that arise after the
date hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement, which is made under the safe
harbor provisions of the Private Securities Litigation Reform Act
of 1995.
Investor Contact:
Geoffrey
Grande, CFA
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot
Rai
(619) 837-5016
media@aviditybio.com
Avidity Biosciences,
Inc.
|
Selected Condensed
Financial Information
|
(in thousands,
except per share data)
|
(unaudited)
|
|
|
|
|
|
|
Statements of
Operations
|
|
|
Three Months Ended
March 31,
|
|
|
2024
|
|
2023
|
Collaboration
revenue
|
|
|
$
3,543
|
|
$
2,233
|
Operating
expenses:
|
|
|
|
|
|
Research and development
|
|
|
66,832
|
|
47,765
|
General and administrative
|
|
|
13,898
|
|
12,064
|
Total operating
expenses
|
|
|
80,730
|
|
59,829
|
Loss from
operations
|
|
|
(77,187)
|
|
(57,596)
|
Other income,
net
|
|
|
8,332
|
|
5,202
|
Net loss
|
|
|
$
(68,855)
|
|
$
(52,394)
|
Net loss per share,
basic and diluted
|
|
|
$
(0.79)
|
|
$
(0.74)
|
Weighted-average shares
outstanding,
basic and diluted
|
|
|
87,212
|
|
70,433
|
|
|
|
|
|
|
Balance
Sheets
|
|
March
31,
|
|
December
31,
|
|
|
2024
|
|
2023
|
Assets
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
Cash, cash equivalents and marketable securities
|
|
$
915,873
|
|
$
595,351
|
Prepaid and other assets
|
|
|
18,601
|
|
15,956
|
Total current
assets
|
|
|
934,474
|
|
611,307
|
Property and equipment,
net
|
|
|
8,655
|
|
8,381
|
Restricted
cash
|
|
|
295
|
|
295
|
Right-of-use
assets
|
|
|
7,625
|
|
8,271
|
Other assets
|
|
|
425
|
|
301
|
Total assets
|
|
|
$
951,474
|
|
$
628,555
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
Accounts payable and other liabilities
|
|
|
$
48,852
|
|
$
52,315
|
Deferred revenue, current portion
|
|
|
26,103
|
|
28,365
|
Total current
liabilities
|
|
|
74,955
|
|
80,680
|
Lease liabilities, net
of current portion
|
|
|
5,421
|
|
6,213
|
Deferred revenue, net
of current portion
|
|
|
40,199
|
|
40,898
|
Total
liabilities
|
|
|
120,575
|
|
127,791
|
Stockholders'
equity
|
|
|
830,899
|
|
500,764
|
Total liabilities and
stockholders' equity
|
|
|
$
951,474
|
|
$
628,555
|
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SOURCE Avidity Biosciences, Inc.