Avidity is on-track to initiate global Phase 3
HARBOR™ trial of AOC 1001 for DM1 in mid-2024
Company to share first-look at long-term
efficacy and safety data from MARINA-OLE™ trial in people
living with DM1 at MDA Clinical & Scientific Conference and via
webcast on March 4, 2024
Avidity to report data from clinical
programs for people living with FSHD and DMD44 later this
year
SAN
DIEGO, Feb. 28, 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 fourth quarter and year ended
December 31, 2023, and highlighted
recent corporate progress.
"With proven delivery of RNA to muscle and robust efficacy and
long-term safety data from our ongoing clinical development
programs, we are revolutionizing the delivery of RNA with our AOC
technology," said Sarah Boyce,
president and chief executive officer at Avidity. "2024 is poised
to be a transformative year for Avidity as we plan to initiate the
global Phase 3 HARBOR™ trial for people living with DM1 and report
data from all three of our clinical development programs to treat
rare muscle diseases – DM1, FSHD and DMD44. We also plan to advance
our cardiology and additional skeletal muscle programs toward
clinical development. We know how important these potential new
treatments are for the patient community and are working diligently
to bring these much-needed therapies to people living with rare
diseases."
"We are pleased to be in a strong financial position, with
$595 million at the close of 2023 and
a cash runway through 2025," said Mike
MacLean, chief financial officer and chief business officer
at Avidity. "We are well positioned to rapidly advance our clinical
development programs, strategically progress our pipeline and build
the infrastructure needed as part of Avidity's next phase of
growth."
Clinical Development Programs - Achievements &
Updates
AOC 1001
- 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 living with myotonic dystrophy
type 1 (DM1). These data augmented previously presented positive
topline data from the Phase 1/2 MARINA® trial in
April 2023 showing improvements in
myotonia, muscle strength and mobility.
- Avidity will share a first-look at long-term efficacy and
safety data from the MARINA-OLE™ trial of AOC 1001 in people living
with DM1 via poster presentation during the 2024 MDA Clinical &
Scientific Conference March 3-6 in
Orlando, Florida. The company will
also host a live webcast event on March
4 at 8:00 a.m. ET as part of
its ongoing investor and analyst event series.
- Avidity is on-track to initiate the global Phase 3 HARBOR™
trial of AOC 1001 for adults living with DM1 in mid-2024.
AOC 1020
- In February 2023, the FDA and the
European Medicines Agency (EMA) granted Orphan Designation for AOC
1020 for facioscapulohumeral muscular dystrophy (FSHD), and in
January 2023, the FDA granted AOC
1020 Fast Track Designation for FSHD.
- In the second quarter of 2024, Avidity is planning to share
preliminary data in approximately half of the study participants in
the Phase 1/2 FORTITUDE™ trial of AOC 1020 in FSHD.
AOC 1044
- In December 2023, Avidity
announced positive AOC 1044 data in healthy volunteers showing
unprecedented delivery to muscle and up to 1.5% exon skipping from
the Phase 1/2 EXPLORE44™ clinical trial for the treatment of
Duchenne muscular dystrophy 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. The FDA granted AOC 1044 Fast Track Designation in
April 2023 and Rare Pediatric Disease
Designation in February 2024 for
DMD44.
- In the second half of 2024, Avidity is planning to share 5
mg/kg cohort data from the Phase 1/2 EXPLORE44™ trial of people
living with DMD44.
Collaboration Announcements
- In November 2023, Avidity
announced the expansion of a global licensing and research
collaboration with Bristol Myers Squibb. The expanded collaboration
will focus on the discovery, development and commercialization of
up to five cardiovascular targets with $100
million upfront and potential cumulative milestone payments
to Avidity of up to $2.2
billion.
Organizational Highlights
- Avidity announced in January 2024
the appointment of Eric B. Mosbrooker as Chief Strategy Officer.
Mr. Mosbrooker previously served as a member of Avidity's Board of
Directors.
Fourth Quarter and Year-End 2023 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash,
cash equivalents and marketable securities totaled $595.4 million as of December 31, 2023, which reflects $160.5 million raised in 2023, inclusive of
approximately $100 million through
our collaboration with Bristol Myers Squibb and $60.5 million through our "at the market
offering" program.
- Collaboration Revenue: Collaboration revenue, including
reimbursable expenses, primarily relates to Avidity's partnership
with Eli Lilly and Company and totaled $2.2
million for the fourth quarter of 2023 compared with
$2.8 million for the fourth quarter
of 2022, and $9.6 million for the
full year 2023 compared with $9.2
million for the full year 2022.
- Research and Development (R&D) Expenses: R&D
expenses include external and internal costs associated with
research and development activities. These expenses were
$52.8 million for the fourth quarter
of 2023 compared with $45.6 million
for the fourth quarter of 2022, and $191.0
million for the full year 2023 compared with $150.4 million for the full year 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 $16.1
million for the fourth quarter of 2023 compared with
$10.4 million for the fourth quarter
of 2022, and $54.2 million for the
full year 2023 compared with $37.7
million for the full year 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 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 a global Phase
3 trial for people living with DM1; the anticipated release of data
from the MARINA-OLE™, FORTITUDE™ and EXPLORE44™ trials and the
timing thereof; the characterization of safety, tolerability and
functional data associated with Avidity's clinical development
programs; plans for the progression of research and development
initiatives, including in cardiology and skeletal muscle; Avidity's
collaboration with Bristol Myers Squibb; Avidity's financial
position, cash balance and ability to fund its operations;
Avidity's growth and related needs; plans for the progression of
Avidity's pipeline and clinical programs for AOC 1001, AOC 1044 and
AOC 1020, and the timing thereof; 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,
and Avidity's position in the RNA field.
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, which may result in delays in
the clinical development of AOC 1001; 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; 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
December 31,
|
|
Twelve Months Ended
December 31,
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Collaboration
revenue
|
$
2,193
|
|
$
2,769
|
|
$
9,560
|
|
$
9,224
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
52,817
|
|
45,610
|
|
190,968
|
|
150,404
|
General and
administrative
|
16,119
|
|
10,384
|
|
54,190
|
|
37,733
|
Total operating
expenses
|
68,936
|
|
55,994
|
|
245,158
|
|
188,137
|
Loss from
operations
|
(66,743)
|
|
(53,225)
|
|
(235,598)
|
|
(178,913)
|
Other income,
net
|
6,300
|
|
2,754
|
|
23,378
|
|
4,918
|
Net loss
|
$
(60,443)
|
|
$
(50,471)
|
|
$
(212,220)
|
|
$
(173,995)
|
Net loss per share,
basic and diluted
|
$
(0.79)
|
|
$
(0.88)
|
|
$
(2.91)
|
|
$
(3.34)
|
Weighted-average shares
outstanding, basic and diluted
|
76,052
|
|
57,296
|
|
73,012
|
|
52,162
|
Balance
Sheets
|
December 31,
2023
|
|
December 31,
2022
|
Assets
|
|
|
|
Current
assets:
|
|
|
|
Cash, cash equivalents
and marketable securities
|
$
595,351
|
|
$
610,727
|
Prepaid and other
assets
|
15,956
|
|
12,215
|
Total current
assets
|
611,307
|
|
622,942
|
Property and equipment,
net
|
8,381
|
|
6,254
|
Restricted
cash
|
295
|
|
251
|
Right-of-use
assets
|
8,271
|
|
8,755
|
Other assets
|
301
|
|
598
|
Total assets
|
$
628,555
|
|
$
638,800
|
Liabilities and
Stockholders' Equity
|
|
|
|
Current
liabilities:
|
|
|
|
Accounts payable and
other liabilities
|
$
52,315
|
|
$
46,867
|
Deferred revenue,
current portion
|
28,365
|
|
5,041
|
Total current
liabilities
|
80,680
|
|
51,908
|
Lease liabilities, net
of current portion
|
6,213
|
|
7,582
|
Deferred revenue, net
of current portion
|
40,898
|
|
1,235
|
Total
liabilities
|
127,791
|
|
60,725
|
Stockholders'
equity
|
500,764
|
|
578,075
|
Total liabilities and
stockholders' equity
|
$
628,555
|
|
$
638,800
|
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SOURCE Avidity Biosciences, Inc.