LA JOLLA, Calif., Nov. 9, 2021 /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 third quarter and nine months ended September 30, 2021 and highlighted recent
corporate progress.
"This past quarter we initiated our Phase 1/2
MARINATM trial for AOC 1001, which transitions us into a
clinical stage company. This trial is important for both AOC 1001
and our platform as it is expected to generate the first proof of
concept data on the AOC platform's potential to deliver RNA therapy
outside of the liver – a first for the field of RNA conjugates and
for Avidity," said Sarah Boyce, president and chief executive
officer. "This is also an important milestone for the myotonic
dystrophy community who currently have no therapeutic options. We
look forward to sharing preliminary data on AOC 1001 in the second
half of next year as we work to achieve our ambitious agenda of
having AOCs for DM1, DMD and FSHD in the clinic by the end of
2022."
"Following completion of our successful first follow-on
financing in August, we are well funded into 2024 with a cash
balance of $413 million at quarter
end. Given these significant financial resources, we are well
positioned to complete the ongoing MARINA trial for AOC 1001, as
well as initiate proof of concept clinical trials in 2022 for AOC
1044, the first of our DMD programs, and for our AOC FSHD program
while we continue our investment in expanding our AOC platform,"
said Mike MacLean, chief financial
officer.
Recent Highlights:
- Initiated first clinical trial for an AOC – transitioning
Avidity into a clinical stage company
-
- Initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with
myotonic dystrophy type 1 (DM1)
-
- Received clearance from the U.S. Food and Drug Administration
(FDA) to proceed with the Phase 1/2 MARINA trial under Avidity's
initial new drug application (IND) in July
2021
- Enrolled the first patient in the MARINA trial in October 2021, marking the first time a person has
been dosed with an AOC
- AOC 1001 was granted Orphan Designation by the FDA and the
European Medicines Agency (EMA)
- AOC 1001 was granted Fast Track Designation by the FDA in
October 2021
- Entered a collaboration with a key physician network for a
natural history study in FSHD
-
- Avidity is supporting the natural history study called the
Motor Outcomes to Validate Evaluations Plus (MOVE+) Study run by
the facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial
Research Network (FSHD CTRN) to enhance the understanding of how to
utilize whole-body MRI and other tools to identify specific
biomarkers for FSHD that can accelerate and support future clinical
trial design.
- Completed a follow-on financing, resulting in net proceeds of
$155.1 million
Upcoming Events
Volume 3 of Avidity's Virtual Investor and Analyst Event Series
will be focused on FSHD and will be held on Thursday, December
9, 2021 at 8am PT/11am ET.
To register for the live video webcast, please visit the "Events
and Presentations" page in the "Investors" section of
Avidity's website. A replay of the webcast will be archived on
Avidity's website following the event.
Art Levin, Ph.D., Avidity's chief
scientific officer, and W. Michael
Flanagan, Ph.D., Avidity's chief technical officer, will
discuss the scientific rationale and potential benefits of
leveraging AOCs for FSHD.
The virtual event will also feature Jeffrey Statland, M.D., professor, Department of
Neurology, University of Kansas Medical
Center, to discuss the clinical impact of FSHD and the
ongoing natural history studies. Dr. Statland is one of the
principal investigators in the ongoing MOVE and MOVE+ natural
history studies for people living with FSHD.
Third Quarter 2021 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash,
cash equivalents and marketable securities totaled $413.0 million as of September 30, 2021, compared to $328.1 million as of December 31, 2020.
- 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 third quarter of 2021 compared with
$1.7 million for the third quarter of
2020, and $7.5 million for the first
nine months of 2021 compared with $4.6
million for the first nine months of 2020.
- Research and Development (R&D) Expenses: R&D
expenses include external and internal costs associated with
research and development activities. These expenses were
$24.8 million for the third quarter
of 2021 compared with $9.5 million
for the third quarter of 2020, and $68.2
million for the first nine months of 2021 compared with
$24.0 million for the first nine
months of 2020. The increases were primarily driven by the
advancement of AOC 1001, AOC 1044 and the AOC FSHD program, as well
as 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 $6.6
million for the third quarter of 2021 compared with
$3.8 million for the third quarter of
2020, and $18.8 million for the first
nine months of 2021 compared with $8.6
million for the first nine months of 2020. The increases
were primarily due to higher personnel costs. The year-to-date
increase was also due to higher professional fees and insurance
costs.
About Avidity Biosciences
Avidity Biosciences, Inc.'s mission is to profoundly improve
people's lives by delivering a new class of RNA therapeutics -
Antibody Oligonucleotide Conjugates (AOCsTM). Avidity's
proprietary AOCs are designed to combine the specificity of
monoclonal antibodies with the precision of oligonucleotide
therapies to target the root cause of diseases previously
untreatable with RNA therapeutics. Avidity's lead product
candidate, AOC 1001, is designed to treat patients with myotonic
dystrophy type 1 (DM1). AOC 1001 has commenced clinical testing
with the ongoing Phase 1/2 MARINATM trial in adults with
DM1. Its advancing and expanding pipeline also includes programs in
facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular
Dystrophy (DMD), muscle atrophy and Pompe disease. The company is
planning for AOC 1044, the lead of three programs for the treatment
of DMD, and its AOC FSHD program to enter the clinic in 2022.
Avidity is also broadening the reach of AOCs beyond muscle tissues
through both internal discovery efforts and key partnerships as the
company continues to deliver on the RNA revolution. Avidity is
headquartered in La Jolla, CA. For
more information about our science, 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 potential to develop a meaningful pipeline of novel AOC
therapeutics; the potential of AOC 1001 in patients with DM1 and
the expected timing of preliminary data from the MARINA trial; the
progression of clinical programs for AOC 1044 and the company's AOC
FSHD program and timing of planned clinical trials; the expected
benefits associated with Orphan Designation and Fast Track
Designation; the sufficiency of the company's current financial
position to fund its development programs, investments in its
pipeline and platform, and operations into 2024; the potential to
apply Avidity's AOC approach to new targets beyond muscle tissues;
and the broad potential of AOCs to treat rare and serious 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 the
business, including, without limitation: 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 and completion of clinical trials;
disruption to its operations from the COVID-19 pandemic; risks that
the benefits associated with Orphan Designation may not be
realized, including that Orphan Designation exclusivity may not
effectively protect a product from competition and that such
exclusivity may not be maintained; the success of its preclinical
studies and clinical trials for the company's product candidates;
the results of preclinical studies and early clinical trials are
not necessarily predictive of future results; Avidity's dependence
on third parties in connection with preclinical testing and product
manufacturing; unexpected adverse side effects or inadequate
efficacy of its product candidates that may limit their
development, regulatory approval and/or commercialization, or may
result in recalls or product liability claims; Avidity's dependence
on third parties for existing collaborations and Avidity may not
realize any benefits from such 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; Avidity could use its available capital resources
sooner than it currently expects; and other risks described in
prior press releases and in filings with the Securities and
Exchange Commission (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 exist 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.
Company Contact:
Kathleen Gallagher
(858) 401-7900
kath.gallagher@aviditybio.com
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,
|
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Collaboration
revenue
|
|
|
$
2,163
|
|
$
1,746
|
|
$
7,474
|
|
$
4,645
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
24,831
|
|
9,455
|
|
68,214
|
|
23,983
|
General and
administrative
|
|
|
6,612
|
|
3,757
|
|
18,764
|
|
8,646
|
Total operating
expenses
|
|
|
31,443
|
|
13,212
|
|
86,978
|
|
32,629
|
Loss from
operations
|
|
|
(29,280)
|
|
(11,466)
|
|
(79,504)
|
|
(27,984)
|
Other income
(expense), net
|
|
|
6
|
|
27
|
|
33
|
|
(96)
|
Net loss
|
|
|
$
(29,274)
|
|
$
(11,439)
|
|
$
(79,471)
|
|
$
(28,080)
|
Net loss per share,
basic and diluted
|
|
|
$
(0.68)
|
|
$
(0.31)
|
|
$
(2.01)
|
|
$
(1.72)
|
Weighted-average
shares outstanding,
basic and diluted
|
|
|
43,265
|
|
37,420
|
|
39,477
|
|
16,361
|
|
|
|
|
|
|
|
|
|
|
Balance
Sheets
|
|
|
|
|
|
September
30,
|
|
December
31,
|
|
|
|
|
|
|
2021
|
|
2020
|
Assets
|
|
|
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and marketable securities
|
|
|
|
$
413,029
|
|
$
328,141
|
Prepaid and
other assets
|
|
|
|
|
|
|
8,929
|
|
3,537
|
Total current
assets
|
|
|
|
|
|
|
421,958
|
|
331,678
|
Property and
equipment, net
|
|
|
|
|
|
|
3,220
|
|
1,468
|
Restricted
cash
|
|
|
|
|
|
|
251
|
|
251
|
Other
assets
|
|
|
|
|
|
|
488
|
|
501
|
Total
assets
|
|
|
|
|
|
|
$
425,917
|
|
$
333,898
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
|
Accounts payable
and other accrued liabilities
|
|
|
|
$
18,157
|
|
$
10,897
|
Deferred
revenue, current portion
|
|
|
|
|
|
|
4,261
|
|
3,690
|
Total current
liabilities
|
|
|
|
|
|
|
22,418
|
|
14,587
|
Lease liabilities,
net of current portion
|
|
|
|
|
|
|
877
|
|
938
|
Deferred revenue, net
of current portion
|
|
|
|
|
|
|
7,745
|
|
12,150
|
Total
liabilities
|
|
|
|
|
|
|
31,040
|
|
27,675
|
Stockholders'
equity
|
|
|
|
|
|
|
394,877
|
|
306,223
|
Total liabilities and
stockholders' equity
|
|
|
|
|
|
|
$
425,917
|
|
$
333,898
|
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SOURCE Avidity Biosciences, Inc.