Advancing three clinical development programs
– DM1, DMD, FSHD - with data anticipated from each program over the
next 12 months
SAN
DIEGO, Aug. 8, 2023 /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 and recent highlights for the second quarter
ended June 30, 2023. Avidity ended
the second quarter of 2023 with cash, cash equivalents and
marketable securities totaling $577
million.
![(PRNewsfoto/Avidity Biosciences, Inc.) (PRNewsfoto/Avidity Biosciences, Inc.)](https://mma.prnewswire.com/media/974628/Avidity_Biosciences_Logo.jpg)
"We are executing well across all three of our clinical
development programs in three distinct rare muscle diseases – DM1,
DMD and FSHD. We plan to report data from each of these clinical
programs over the next 12 months," said Sarah Boyce, president and chief executive
officer at Avidity. "With the positive topline data from MARINA™
and the easing of the partial clinical hold, we are working to
finalize the Phase 3 study design and global regulatory path for
AOC 1001 in DM1, while advancing our programs for DMD and FSHD.
There are no approved treatment options for these muscle diseases.
We are working to bring these much-needed therapies to people as
quickly as possible."
"At the close of Q2, we maintain a strong cash balance of
$577 million. We are funded into the
second half of 2025, allowing us to execute on advancing our
clinical development programs in rare muscle diseases, expanding
our AOC platform and broadening our pipeline in immunology,
cardiology and other select indications outside of muscle," said
Mike MacLean, chief financial
officer and chief business officer at Avidity. "As leaders in the
RNA field, we are committed to investing in our platform, pipeline
and people as we revolutionize a new class of targeted RNA
therapeutics for people living with rare diseases."
Recent Highlights
- The company is now enrolling participants living with Duchenne
muscular dystrophy with mutations amenable to exon 44 skipping
(DMD44) into the AOC 1044 EXPLORE44™ trial
- The Phase 1/2 MARINA trial concluded with 38 participants
enrolled; 37 participants completed MARINA and have rolled over
into the MARINA-OLE™
- In May, the U.S. Food and Drug Administration (FDA) eased the
partial clinical hold on AOC 1001. Avidity is now dose escalating
approximately 12 participants from 2 mg/kg to 4 mg/kg of AOC 1001
in the MARINA-OLE; in parallel, the company is finalizing a Phase 3
study design and a global regulatory path for AOC 1001
- In April, FDA granted Fast Track Designation for AOC 1044
- In April, AOC 1001 topline data demonstrating functional
improvement, disease modification and favorable safety and
tolerability profile in people living with myotonic dystrophy type
1 (DM1) were presented at the AAN Annual meeting. AOC 1001 provided
directional improvements in multiple functional endpoint
assessments including myotonia, measures of strength and mobility,
as well as meaningful DMPK reduction and splicing changes.
Upcoming Milestones
- The company continues to advance three distinct rare disease
clinical programs with AOC 1001 for DM1, AOC 1020 for the treatment
of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for
the treatment of DMD44. Upcoming milestones include:
-
- Data from healthy volunteers in the EXPLORE44 trial planned for
the fourth quarter of 2023
- A first look at data from the MARINA-OLE trial planned for
first half of 2024
- Data from a preliminary assessment in approximately half of
participants in the FORTITUDE™ trial planned for the first half of
2024
Second Quarter 2023 Financial Results
Cash, Cash Equivalents and Marketable
Securities: Cash, cash equivalents and marketable
securities totaled $576.5 million as
of June 30, 2023, compared to
$610.7 million as of December 31, 2022.
- Collaboration Revenue: Collaboration revenue, including
reimbursable expenses, primarily relates to Avidity's partnership
with Eli Lilly and Company and totaled $2.3
million for the second quarter of 2023 compared with
$2.2 million for the second quarter
of 2022, and $4.5 million for the
first six months of 2023 compared with $4.0
million for the first six months of 2022.
- Research and Development (R&D) Expenses: R&D
expenses include external and internal costs associated with
research and development activities. These expenses were
$42.6 million for the second quarter
of 2023 compared with $39.8 million
for the second quarter of 2022, and $90.4
million for the first six months of 2023 compared with
$67.5 million for the first six
months of 2022. The increases were primarily driven by the
advancement of AOC 1001, 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 $12.3
million for the second quarter of 2023 compared with
$8.7 million for the second quarter
of 2022, and $24.3 million for the
first six months of 2023 compared with $17.3
million for the first six months of 2022. The increases were
primarily due to higher personnel costs and professional fees 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 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 Twitter.
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: the anticipated
timing of release of data from the MARINA-OLE™, EXPLORE44™ and
FORTITUDE™ trials; the design of and prospects for a Phase 3 trial
and regulatory pathway for AOC 1001; plans the progression of
clinical programs for AOC 1001, AOC 1044 and AOC 1020 and the
timing thereof; the sufficiency of Avidity's cash balance to meet
the company's operational needs and the expected cash runway; the
potential of Avidity's product candidates to treat rare diseases
and Avidity's efforts to bring them to people suffering from
applicable diseases; the potential of AOCs to target a range of
different cells and tissues beyond the liver, and to treat cardiac
and immunological diseases; the continued advancement of programs
with collaboration partners, including Eli Lilly and Company; and
Avidity's plans to expand its AOC platform and to invest in its
pipeline programs.
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, 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,
which may result in delays in the clinical development of AOC 1001;
additional participant data related to AOC 1001 that continues to
become available may be inconsistent with the data produced as of
the most recent date cutoff, and further analysis of existing data
and analysis of new data may lead to conclusions different from
those established as of such date cutoff; 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, or may result in additional clinical
holds which may not be timely lifted, recalls or product liability
claims; Avidity is early in its development efforts; Avidity's
approach to the discovery and development of product candidates
based on its AOC platform is unproven, and the company does not
know whether it will be able to develop any products of commercial
value; 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, including acceptance of INDs and
similar foreign regulatory filings and the proposed design of
future clinical trials; Fast Track Designation by the FDA may not
lead to a faster development or regulatory review or approval
process; Avidity could exhaust its available capital resources
sooner than it currently expects and fail to raise additional
needed funds; and other risks described in Avidity's Annual Report
on Form 10-K for the fiscal year ended December 31, 2022, filed with the Securities and
Exchange Commission (SEC) on February 28,
2023, and in 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:
Kathleen
Gallagher
(858) 401-7900 x550
investors@aviditybio.com
Media Contact:
Navjot
Rai
(858) 401-7900 x550
media@aviditybio.com
Avidity Biosciences, Inc.
|
Selected Condensed Financial
Information
|
(in thousands, except per share
data)
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
Statements of Operations
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Collaboration
revenue
|
|
$
2,316
|
|
$
2,178
|
|
$
4,549
|
|
$
3,973
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and development
|
|
42,672
|
|
39,789
|
|
90,437
|
|
67,477
|
General and administrative
|
|
12,278
|
|
8,688
|
|
24,342
|
|
17,255
|
Total operating
expenses
|
|
54,950
|
|
48,477
|
|
114,779
|
|
84,732
|
Loss from
operations
|
|
(52,634)
|
|
(46,299)
|
|
(110,230)
|
|
(80,759)
|
Other income,
net
|
|
5,609
|
|
609
|
|
10,811
|
|
834
|
Net loss
|
|
$
(47,025)
|
|
$
(45,690)
|
|
$
(99,419)
|
|
$
(79,925)
|
Net loss per share,
basic and diluted
|
|
$
(0.66)
|
|
$
(0.92)
|
|
$
(1.40)
|
|
$
(1.63)
|
Weighted-average shares
outstanding,
basic and diluted
|
|
71,390
|
|
49,927
|
|
70,914
|
|
49,091
|
|
|
|
|
|
|
|
|
|
Balance Sheets
|
|
|
|
|
June
30,
2023
|
|
December 31,
2022
|
Assets
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash, cash equivalents and marketable securities
|
|
|
|
$
576,503
|
|
$
610,727
|
Prepaid and other assets
|
|
|
|
|
|
18,031
|
|
12,215
|
Total current
assets
|
|
|
|
|
|
594,534
|
|
622,942
|
Property and equipment,
net
|
|
|
|
|
|
7,776
|
|
6,254
|
Restricted
cash
|
|
|
|
|
|
295
|
|
251
|
Right-of-use
asset
|
|
|
|
|
|
7,755
|
|
8,755
|
Other assets
|
|
|
|
|
|
424
|
|
598
|
Total assets
|
|
|
|
|
|
$
610,784
|
|
$
638,800
|
Liabilities and Stockholders'
Equity
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
Accounts payable and other liabilities
|
|
|
|
|
|
$
41,434
|
|
$
46,867
|
Deferred revenue, current portion
|
|
|
|
|
|
3,810
|
|
5,041
|
Total current
liabilities
|
|
|
|
|
|
45,244
|
|
51,908
|
Lease liabilities, net
of current portion
|
|
|
|
|
|
6,362
|
|
7,582
|
Deferred revenue, net
of current portion
|
|
|
|
|
|
—
|
|
1,235
|
Total
liabilities
|
|
|
|
|
|
51,606
|
|
60,725
|
Stockholders'
equity
|
|
|
|
|
|
559,178
|
|
578,075
|
Total liabilities and
stockholders' equity
|
|
|
|
|
|
$
610,784
|
|
$
638,800
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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SOURCE Avidity Biosciences, Inc.