LA JOLLA, Calif., May 12, 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 first quarter ended March 31, 2021 and highlighted recent corporate
progress.
"AOC 1001, the first program from our AOC platform, is advancing
toward the clinic and is on track to begin a Phase 1/2 study in
adults with DM1 in the second half of this year. Our skeletal
muscle pipeline is also progressing as planned while we continue to
expand our discovery efforts to other indications," said Sarah
Boyce, president and chief executive officer. "We are proud to
be at the forefront of RNA innovation by delivering on the promise
of our AOC platform and pipeline."
Mike MacLean, chief financial
officer, added, "We are off to a great start this year with a
strong financial position of over $300
million at the end of the first quarter. This allows us to
progress AOC 1001 while continuing to invest in our other pipeline
programs and our AOC platform, which we believe is a sustainable
engine with the potential to have a broad impact across both rare
and other serious diseases."
AOC Platform and Pipeline Highlights
- Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline
for Untreated Rare Muscle Diseases.
In April 2021, Avidity
presented preclinical data for AOC 1001 for myotonic dystrophy type
1 (DM1) in an oral presentation as part of the Emerging Science
Session at the American Academy of Neurology (AAN) 2021 Virtual
Annual Meeting. Avidity scientists have demonstrated activity and
potency of siRNAs against the dystrophy protein kinase (DMPK) gene
in muscle cells derived from patients with DM1. These data
demonstrated activity in the nucleus and cytoplasm. The
presentation also showed that single doses of AOC 1001 produced 75%
reductions in DMPK mRNA expression in preclinical studies that were
maintained for months post-dosing. Following regulatory clearance,
Avidity remains on track with plans to begin 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. Avidity remains on track with plans to
commence clinical trials for both programs in 2022, following
additional preparatory preclinical studies and regulatory
clearance.
Upcoming Events
- Avidity's Virtual Investor & Analyst Event Series –
Volume 1. Recently, Avidity launched its Virtual
Investor and Analyst Event Series. The first virtual event
entitled "Engineering Antibody Oligonucleotide Conjugates" will be
held on Wednesday, May
19th, 2021 at 8am
PT/11am ET.
The virtual event will feature a panel discussion with leading RNA
therapeutics key opinion leaders Phillip D.
Zamore, Ph.D., Chair, RNA Therapeutics Institute at UMASS
and Steven F. Dowdy, Ph.D.,
Professor of Cellular and Molecular Medicine at UCSD. To
register for the event or for more information, please email
investors@aviditybio.com.
First Quarter 2021 Financial Results
- Cash, Cash Equivalents and Marketable
Securities: Cash, cash equivalents and marketable
securities totaled $307.9 million as
of March 31, 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 $2.7 million for the first quarter of 2021
compared with $1.4 million for the
first quarter of 2020.
- 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 $20.7 million for the first quarter of 2021
compared with $5.5 million for the
first quarter of 2020. The increase was primarily driven by the
progression of AOC 1001 toward the clinic, as well as research for
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 $5.9
million for the first quarter of 2021 compared with
$2.0 million for the first quarter of
2020. The increase was primarily due to higher personnel costs
(including noncash stock-based compensation), professional fees and
insurance costs related to being a public company.
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 cells, cardiac tissue 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 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 initiation of a clinical trial of AOC 1001 in
patients with myotonic dystrophy type 1 and other planned clinical
trials, and the timing thereof; the potential to identify new
targets beyond skeletal muscle that can be targeted with Avidity's
AOC approach; 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 and all of its development
programs are in the preclinical or discovery stage; 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; 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; 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 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.
Contacts:
Company:
Kathleen Gallagher
(858) 401-7900
kath.gallagher@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
March 31,
|
|
|
|
|
2021
|
|
|
2020
|
|
Collaboration
revenue
|
|
|
$
|
2,704
|
|
|
$
|
1,358
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
|
20,677
|
|
|
|
5,544
|
|
General and
administrative
|
|
|
|
5,884
|
|
|
|
1,964
|
|
Total operating
expenses
|
|
|
|
26,561
|
|
|
|
7,508
|
|
Loss from
operations
|
|
|
|
(23,857)
|
|
|
|
(6,150)
|
|
Other income
(expense), net
|
|
|
|
13
|
|
|
|
65
|
|
Net loss
|
|
|
$
|
(23,844)
|
|
|
$
|
(6,085)
|
|
Net loss per share,
basic and diluted
|
|
|
$
|
(0.64)
|
|
|
$
|
(2.14)
|
|
Weighted-average
shares outstanding, basic and diluted
|
|
|
|
37,521
|
|
|
|
2,845
|
|
|
|
|
|
|
|
|
|
|
|
Balance
Sheets
|
|
March
31,
|
|
|
December
31,
|
|
|
|
2021
|
|
|
2020
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and marketable securities
|
|
$
|
307,914
|
|
|
$
|
328,141
|
|
Prepaid and
other assets
|
|
|
4,036
|
|
|
|
3,537
|
|
Total current
assets
|
|
|
311,950
|
|
|
|
331,678
|
|
Property and
equipment, net
|
|
|
2,187
|
|
|
|
1,468
|
|
Restricted
cash
|
|
|
|
251
|
|
|
|
251
|
|
Other
assets
|
|
|
364
|
|
|
|
501
|
|
Total
assets
|
|
$
|
314,752
|
|
|
$
|
333,898
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
|
Accounts payable
and other accrued liabilities
|
|
$
|
13,157
|
|
|
$
|
10,897
|
|
Deferred
revenue, current portion
|
|
|
4,357
|
|
|
|
3,690
|
|
Total current
liabilities
|
|
|
|
17,514
|
|
|
|
14,587
|
|
Lease liabilities,
net of current portion
|
|
|
|
951
|
|
|
|
938
|
|
Deferred revenue, net
of current portion
|
|
|
|
10,171
|
|
|
|
12,150
|
|
Total
liabilities
|
|
|
|
28,636
|
|
|
|
27,675
|
|
Stockholders'
equity
|
|
|
286,116
|
|
|
|
306,223
|
|
Total liabilities and
stockholders' equity
|
|
|
$
|
314,752
|
|
|
$
|
333,898
|
|
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SOURCE Avidity Biosciences, Inc.