Avidity reported new positive AOC 1001 data at
World Muscle Society (WMS) Congress in October demonstrating
consistent improvement in multiple additional functional endpoints
and favorable long-term safety and tolerability in people living
with myotonic dystrophy type 1 (DM1)
AOC 1044 receives Orphan Designation for the
treatment of DMD in US and EU
Avidity remains on track to report data
readouts from three clinical development programs – DM1, DMD and
FSHD – over the next nine months
SAN
DIEGO, Nov. 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 third
quarter ended September 30, 2023.
Avidity ended the third quarter of 2023 with cash, cash equivalents
and marketable securities totaling $542.6
million.
"In early October we announced new positive AOC 1001 data that
demonstrated improvement in multiple additional functional
endpoints, as well as favorable long-term safety and tolerability,
in people living with DM1," said Sarah
Boyce, president and chief executive officer. "In addition,
we announced the completion of the 12-participant dose escalation
from 2 mg/kg to 4 mg/kg as part of the partial clinical hold
easement and look forward to finalizing the Phase 3 study design
and global regulatory path for AOC 1001. We continue to make
significant strides across all three of our clinical development
programs, remaining on track to report data this year for AOC 1044
in DMD and in the first half of 2024 for both AOC 1001 in DM1 and
AOC 1020 in FSHD".
"With funding into the second half of 2025 and continued
execution across our portfolio, we remain very confident in the
progress we are making in advancing our clinical development
programs for DM1, DMD and FSHD," said Mike
MacLean, chief financial officer and chief business officer.
"The recently presented positive AOC 1001 data at World Muscle
Society (WMS) Congress reinforces our confidence in our AOC
platform, which spans multiple therapeutic areas, and our ability
to deliver on our vision to profoundly improve people's lives by
revolutionizing the delivery of RNA therapeutics".
Recent Highlights
- In October 2023, Avidity
announced new positive AOC 1001 data demonstrating improvement in
multiple additional functional endpoints and favorable long-term
safety and tolerability in people with DM1 at WMS. The new data
demonstrated improvement in additional functional measures
including hand grip, muscle strength (Manual Muscle Testing
composite score and both upper and lower Quantitative Muscle
Testing composite scores) and patient reported outcomes, augmenting
previously reported positive data showing improvements in myotonia,
muscle strength and mobility.
- Data from the dose escalation of 12 participants from 2 mg/kg
to 4 mg/kg of AOC 1001 as part of the easement of the partial
clinical hold showed no neurological events and no MRI changes
following dosing. Avidity continues to work as quickly as possible
to resolve the partial clinical hold. In parallel, the company is
finalizing a Phase 3 study design and a global regulatory path for
AOC 1001.
- Avidity has initiated dosing participants living with Duchenne
muscular dystrophy with mutations amenable to exon 44 skipping
(DMD44).
- In August and October 2023, the
FDA and EMA respectively, granted Orphan Designation for AOC 1044
for DMD44.
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 Q4 of this year
- A first look at efficacy 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
Third Quarter 2023 Financial Results
Cash, Cash Equivalents and Marketable
Securities: Cash, cash equivalents and marketable
securities totaled $542.6 million as
of September 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 $2.8
million for the third quarter of 2023 compared with
$2.5 million for the third quarter of
2022, and $7.4 million for the first
nine months of 2023 compared with $6.5
million for the first nine 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
$47.7 million for the third quarter
of 2023 compared with $37.3 million
for the third quarter of 2022, and $138.2
million for the first nine months of 2023 compared with
$104.8 million for the first nine
months of 2022. The increases were primarily driven by the
advancement of AOC 1001, AOC 1020 and AOC 1044, as well as external
and internal 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 $13.7
million for the third quarter of 2023 compared with
$10.1 million for the third quarter
of 2022, and $38.1 million for the
first nine months of 2023 compared with $27.3 million for the first nine 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 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: Avidity's efforts to resolve the
partial clinical hold related to AOC 1001; the characterization of
safety, tolerability and functional data associated with AOC 1001;
expectations related to the MARINA-OLE™ study and AOC
1001; the anticipated timing of release of data from the
MARINA-OLE, EXPLORE44™ and FORTITUDE™ trials;
expectations regarding a Phase 3 study and global regulatory path
for AOC 1001; Avidity's balance sheet and the ability of Avidity's
cash balance to meet the company's operational needs; plans for the
progression of clinical programs for AOC 1001, AOC 1044 and AOC
1020 and the timing thereof; the continued advancement of programs
with collaboration partners, including Eli Lilly and Company; the
potential of Avidity's product candidates to treat rare diseases
and Avidity's efforts to bring them to people suffering from
applicable diseases; 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, 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 data cutoff, and further analysis of
existing data and analysis of new data may lead to conclusions
different from those established as of such data 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; 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:
Geoffrey
Grande, CFA
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot
Rai
(619) 837-5016
media@aviditybio.com
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|
|
|
|
|
|
|
Avidity Biosciences,
Inc.
|
Selected Condensed
Financial Information
|
(in thousands,
except per share data)
|
(unaudited)
|
|
|
|
|
|
|
|
|
|
|
Statements of
Operations
|
|
|
Three Months Ended
September 30,
|
|
Nine Months Ended
September 30,
|
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Collaboration
revenue
|
|
|
$
2,818
|
|
$
2,482
|
|
$
7,367
|
|
$
6,455
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Research and development
|
|
|
47,714
|
|
37,317
|
|
138,151
|
|
104,794
|
General and administrative
|
|
|
13,729
|
|
10,094
|
|
38,071
|
|
27,349
|
Total operating
expenses
|
|
|
61,443
|
|
47,411
|
|
176,222
|
|
132,143
|
Loss from
operations
|
|
|
(58,625)
|
|
(44,929)
|
|
(168,855)
|
|
(125,688)
|
Other income,
net
|
|
|
6,267
|
|
1,330
|
|
17,078
|
|
2,164
|
Net loss
|
|
|
$
(52,358)
|
|
$
(43,599)
|
|
$
(151,777)
|
|
$
(123,524)
|
Net loss per share,
basic and diluted
|
|
|
$
(0.71)
|
|
$
(0.82)
|
|
$
(2.11)
|
|
$
(2.45)
|
Weighted-average shares
outstanding,
basic and diluted
|
|
|
74,097
|
|
53,069
|
|
71,987
|
|
50,432
|
|
|
|
|
|
|
|
|
|
|
Balance
Sheets
|
|
|
|
|
|
September
30,
|
|
December
31,
|
|
|
|
|
|
|
2023
|
|
2022
|
Assets
|
|
|
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
|
Cash, cash equivalents and marketable securities
|
|
|
|
|
$
542,583
|
|
$
610,727
|
Prepaid and other assets
|
|
|
|
|
|
|
12,904
|
|
12,215
|
Total current
assets
|
|
|
|
|
|
|
555,487
|
|
622,942
|
Property and equipment,
net
|
|
|
|
|
|
|
8,130
|
|
6,254
|
Restricted
cash
|
|
|
|
|
|
|
295
|
|
251
|
Right-of-use
asset
|
|
|
|
|
|
|
8,907
|
|
8,755
|
Other assets
|
|
|
|
|
|
|
344
|
|
598
|
Total assets
|
|
|
|
|
|
|
$
573,163
|
|
$
638,800
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
|
Accounts payable and other liabilities
|
|
|
|
|
|
|
$
46,748
|
|
$
46,867
|
Deferred revenue, current portion
|
|
|
|
|
|
|
1,968
|
|
5,041
|
Total current
liabilities
|
|
|
|
|
|
|
48,716
|
|
51,908
|
Lease liabilities, net
of current portion
|
|
|
|
|
|
|
6,979
|
|
7,582
|
Deferred revenue, net
of current portion
|
|
|
|
|
|
|
—
|
|
1,235
|
Total
liabilities
|
|
|
|
|
|
|
55,695
|
|
60,725
|
Stockholders'
equity
|
|
|
|
|
|
|
517,468
|
|
578,075
|
Total liabilities and
stockholders' equity
|
|
|
|
|
|
|
$
573,163
|
|
$
638,800
|
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SOURCE Avidity Biosciences, Inc.