LA JOLLA, Calif., March 15, 2021 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
pioneering a new class of oligonucleotide-based therapies called
Antibody Oligonucleotide Conjugates (AOCs™), today reported
financial results for the fourth quarter and year ended
December 31, 2020 and highlighted
recent corporate progress.
"In 2020, we made significant advances across our AOC pipeline
and platform. Our work supports Avidity's evolution to a
clinical-stage company as we plan to advance AOC 1001 into the
clinic in the second half of this year and progress our FSHD and
DMD programs," said Sarah Boyce,
President and Chief Executive Officer. "Our discovery efforts
continue to focus on expanding our AOC platform into additional
muscle diseases and other tissues as we begin to realize our
vision of profoundly improving people's lives by revolutionizing
the delivery of RNA treatments."
"Last year we built a solid foundation for growth, anchored by
our successful IPO and strong financial position with $328 million in cash at year-end," said
Mike MacLean, Chief Financial
Officer. "We are investing in our platform and have assembled an
experienced team in RNA therapeutics and rare diseases to deliver
on our discovery, clinical and commercial objectives."
AOC Platform and Pipeline Highlights
- Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline
for Untreated Rare Muscle Diseases. Avidity advanced its
first-in-class, lead rare disease program, AOC 1001, toward the
clinic and entered into a collaboration with Myotonic Dystrophy
Clinical Research Network supporting END-DM1, a natural history
study to advance the understanding of disease progression in
patients with myotonic dystrophy type 1 (DM1). The company
plans to initiate a Phase 1/2 clinical study of AOC 1001 in adults
with DM1 in the second half of 2021.
Avidity also advanced additional programs in its muscle franchise
including a program for facioscapulohumeral muscular dystrophy
(FSHD) and three programs for Duchenne muscular dystrophy (DMD).
The AOC FSHD program and the lead AOC DMD program targeting Exon 44
are the most advanced. In 2022, following additional preparatory
preclinical studies and regulatory clearance, Avidity plans to
commence clinical trials for both of these programs.
- Demonstrated Preclinical Proof-of-Concept in Skeletal Muscle
and Other Tissues; Advancing Beyond Muscle with Discovery Efforts
and Partnering. In preclinical models, Avidity's AOCs
demonstrated robust mRNA reductions in skeletal muscle, cardiac
muscle, activated B- and T-cells and tumor
infiltrating lymphocytes, macrophages and the liver.
Avidity is advancing beyond muscle with its own discovery efforts
and through partnering, as evidenced by its strategic
collaborations with Eli Lilly in immunology and MyoKardia, a
wholly-owned subsidiary of Bristol Meyers Squibb, in cardiac
tissue.
Organizational Highlights
- Appointed Experienced and Diverse Team Members to Management
and Board of Directors. Avidity has assembled a full management
team with deep expertise in the discovery, development and
commercialization of RNA therapeutics and rare diseases. Avidity
also welcomed Jean Kim and
Tamar Thompson to its Board of
Directors.
Fourth Quarter and Year-End 2020 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash,
cash equivalents and marketable securities totaled $328.1 million as of December 31, 2020, which includes net proceeds of
$274.1 million from the company's IPO
in June 2020, compared to
$94.6 million as of December 31, 2019.
- Collaboration Revenue: Collaboration revenue solely
related to our partnership with Lilly, including reimbursable
expenses, was $2.1 million for the
fourth quarter of 2020 compared with $1.4
million for the fourth quarter of 2019, and $6.8 million for the full year 2020 compared with
$2.3 million for the full year
2019.
- Research and Development (R&D) Expenses: R&D
expenses, including external and internal costs associated with
research activities, primarily relate to the progression of the
company's research on AOC 1001 and other muscle programs. These
expenses were $13.6 million for the
fourth quarter of 2020 compared with $5.6
million for the fourth quarter of 2019, and $37.6 million for the full year 2020 compared
with $14.5 million for the full year
2019. The increases were primarily driven by the progression of AOC
1001 toward the clinic, as well as other programs.
- 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 $4.8
million for the fourth quarter of 2020 compared with
$1.8 million for the fourth quarter
of 2019, and $13.5 million for the
full year 2020 compared with $5.1
million for the full year 2019. The increases were primarily
due to higher personnel costs (including noncash stock-based
compensation), professional fees and insurance costs related to
being a public company, as well as higher patent filing fees.
About Avidity Biosciences
Avidity Biosciences, Inc. is driven to change lives with a new
class of therapies called Antibody Oligonucleotide Conjugates
(AOCs) that are designed to overcome current limitations of
oligonucleotide therapies in order to treat a wide range of serious
diseases. Avidity's proprietary AOC platform combines the tissue
selectivity of monoclonal antibodies and the precision of
oligonucleotide therapies to access previously undruggable tissue
and cell types and more effectively target underlying genetic
drivers of diseases. Avidity's lead product candidate, AOC 1001, is
designed to treat myotonic dystrophy type 1, and its other muscle
programs are focused on the treatment of Duchenne muscular
dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease
and muscle atrophy. In addition to its muscle franchise, Avidity
has research efforts focused on immune, cardiac and other cell
types.
Avidity is headquartered in La Jolla, CA. For more
information about Avidity's science, pipeline and people,
please visit www.aviditybiosciences.com and engage with
Avidity on LinkedIn.
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 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; Avidity's
evolution to a clinical-stage company; Avidity's plans to initiate
a Phase 1/2 clinical trial of AOC 1001 in patients with DM1 and the
expected timing thereof; Avidity's plans to submit a regulatory
filing and commence a clinical trial of its AOC FSHD program and
the expected timing thereof; Avidity's plans to submit a regulatory
filing and commence a clinical trial of its AOC DMD program and the
expected timing thereof; Avidity's plans to expand its AOC platform
into additional muscle diseases and other tissues; and the broad
potential of AOCs to treat 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: the company is early in
development efforts and all of its development programs are in the
preclinical or discovery stage; the company's approach to the
discovery and development of product candidates based on the 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 the company's operations from the
COVID-19 pandemic; the success of its preclinical studies and
clinical trials for its product candidates; the results of
preclinical studies and early clinical trials are not necessarily
predictive of future results; the company may not be able to show
utility of AOCs in cardiac or other tissue and may not realize any
benefits from its collaborations; its 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; 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; risks related to
integration of new management personnel; and other risks described
in the company's prior press releases and in its 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.
Contacts:
Company:
Mike MacLean
(858) 401-7900
mikemaclean@aviditybio.com
Media and Investors:
Amy Conrad
Juniper Point
(858) 366-3243
amy@juniper-point.com
Avidity
Biosciences, Inc.
|
Selected Condensed
Financial Information
|
(in thousands,
except per share data)
|
(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
Statements of
Operations
|
|
|
Three Months
Ended
|
|
Twelve Months
Ended
|
|
|
|
December
31,
|
|
December
31,
|
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
Collaboration
revenue
|
|
|
$
2,142
|
|
$
1,445
|
|
$
6,787
|
|
$
2,319
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
13,619
|
|
5,645
|
|
37,602
|
|
14,539
|
General and
administrative
|
|
|
4,816
|
|
1,847
|
|
13,462
|
|
5,112
|
Total operating
expenses
|
|
|
18,435
|
|
7,492
|
|
51,064
|
|
19,651
|
Loss from
operations
|
|
|
(16,293)
|
|
(6,047)
|
|
(44,277)
|
|
(17,332)
|
Other income
(expense), net
|
|
|
18
|
|
(4,354)
|
|
(78)
|
|
(7,402)
|
Net loss
|
|
|
$
(16,275)
|
|
$
(10,401)
|
|
$
(44,355)
|
|
$
(24,734)
|
Net loss per share,
basic and diluted
|
|
|
$
(0.43)
|
|
$
(3.75)
|
|
$
(2.05)
|
|
$
(9.12)
|
Weighted-average
shares outstanding,
basic and diluted
|
|
|
37,455
|
|
2,776
|
|
21,663
|
|
2,713
|
|
|
|
|
|
|
|
|
|
|
Balance
Sheets
|
|
|
|
|
|
December
31,
|
|
|
|
|
|
|
2020
|
|
2019
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and marketable securities
|
|
|
|
|
|
$
328,141
|
|
$
94,578
|
Prepaid and
other assets
|
|
|
|
|
|
3,537
|
|
1,098
|
Total current
assets
|
|
|
|
|
|
331,678
|
|
95,676
|
Property and
equipment, net
|
|
|
|
|
|
1,468
|
|
631
|
Restricted
cash
|
|
|
|
|
|
|
251
|
|
—
|
Other
assets
|
|
|
|
|
|
501
|
|
600
|
Total
assets
|
|
|
|
|
|
$
333,898
|
|
$
96,907
|
Liabilities,
Convertible Preferred Stock and Stockholders' Equity
(Deficit)
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
|
Accounts payable
and other accrued liabilities
|
|
|
|
|
|
$
10,897
|
|
$
3,622
|
Deferred
revenue, current portion
|
|
|
|
|
|
3,690
|
|
3,840
|
Long-term debt,
current portion
|
|
|
|
|
|
|
—
|
|
2,774
|
Total current
liabilities
|
|
|
|
|
|
|
14,587
|
|
10,236
|
Lease liabilities,
net of current portion
|
|
|
|
|
|
|
938
|
|
393
|
Deferred revenue, net
of current portion
|
|
|
|
|
|
|
12,150
|
|
15,100
|
Long-term debt, net
of current portion
|
|
|
|
|
|
|
—
|
|
1,770
|
Other long-term
liabilities
|
|
|
|
|
|
|
—
|
|
45
|
Total
liabilities
|
|
|
|
|
|
|
27,675
|
|
27,544
|
Convertible preferred
stock
|
|
|
|
|
|
|
—
|
|
134,720
|
Stockholders' equity
(deficit)
|
|
|
|
|
|
306,223
|
|
(65,357)
|
Total liabilities,
convertible preferred stock and
stockholders' equity (deficit)
|
|
|
|
|
|
|
$
333,898
|
|
$
96,907
|
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SOURCE Avidity Biosciences, Inc.