Accelerated approval path confirmed for
del-zota; Biologics License Application (BLA) submission planned
for year end 2025
On track to complete enrollment in del-desiran
HARBORTM Phase 3 and potential registrational
del-brax biomarker cohort as well as initiate global pivotal trial
for del-brax by mid-2025
Eric Mosbrooker expands role to Chief
Commercial Officer; Charles Calderaro III, Chief
Technical Officer, and Kat Lange, Chief Business Officer, join as
company prepares for global commercialization and continued
pipeline expansion
Avidity to present at 43rd Annual J.P. Morgan
Healthcare Conference on Tuesday, January
14
SAN
DIEGO, Jan. 8, 2025 /PRNewswire/ -- Avidity
Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company
committed to delivering a new class of RNA therapeutics called
Antibody Oligonucleotide Conjugates (AOCs™), today announced its
progress and planning for a transformational year in 2025. These
major anticipated milestones include executing on three potentially
registrational programs, filing a Biologics License Application
(BLA) for delpacibart zotadirsen (del-zota) for people living with
Duchenne muscular dystrophy mutations amenable to exon 44 skipping
(DMD44), preparing for multiple product launches in DMD44, myotonic
dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy
(FSHD) and building a global, fully integrated company to support a
growing AOC pipeline in neuromuscular diseases and precision
cardiology.
"We look forward to an exciting year ahead as we prepare to
become a commercial organization and transition to the next stage
of delivering AOCs to people living with rare diseases who have no
or limited treatment options. In 2025, we plan to file our first
BLA, accelerate commercial preparations for three potential product
launches in rapid succession and broaden our pipeline," said
Sarah Boyce, President and Chief
Executive Officer at Avidity. "We are very pleased that the
accelerated approval path is open for del-zota in DMD44 and that
global regulators are aligned on a registrational path for
del-desiran in DM1. We are now setting our sights on finalizing the
global registrational path for del-brax in FSHD, including a
potential accelerated approval strategy in the U.S. We continue to
focus on rapid execution and are on track to fully enroll
del-desiran's DM1 Phase 3 HARBOR™ trial as well as the biomarker
cohort of the del-brax FORTITUDE™ trial on time. As we expand the
reach of the AOC platform to precision cardiology and
next-generation technology innovations, we continue to make many
new and revolutionary advances for patients, our company and the
biotech industry."
Expanding the Leadership Team to Support Anticipated
Growth
To execute on the full range of strategic initiatives and growth
anticipated in 2025 and beyond, Avidity continues to enhance and
deepen the company's leadership team:
- Eric B. Mosbrooker expands his role to Chief Commercial Officer
(CCO) to lead Avidity's multiple global product launches;
- Charles (Chuck) Calderaro III,
joins as Chief Technical Officer (CTO) to lead technical
development and operations; and,
- Kat Lange joins as Senior Vice President and Chief Business
Officer (CBO) to lead investor relations and business
development.
"With Eric transitioning to CCO as well as Chuck and Kat
joining, we are expanding our team and the organizational structure
to propel Avidity forward to becoming a global, commercial company
launching therapies in three separate rare diseases to profoundly
improve the lives of people living with DMD44, DM1 and FSHD. As we
prepare for the first of these potential launches, our early
initiatives include building global medical affairs and commercial
teams devoted to serving the needs of these rare disease
communities that are waiting for treatments," continued Ms.
Boyce.
2025 Upcoming Clinical and Regulatory Highlights
- Delpacibart zotadirsen (del-zota) for the treatment of
DMD44:
- Planned BLA submission year end 2025
- The U.S. Food and Drug Administration (FDA) confirmed the
accelerated approval path is available for del-zota and that the
clinical data package from the EXPLORE44TM program could
support a BLA filing
- Presentation of topline data from the EXPLORE44 trial (Q1)
- Presentation of topline data from the ongoing
EXPLORE44-OLETM trial (Q4)
- Delpacibart etedesiran (del-desiran) for the treatment
of DM1:
- Presentation of additional data analyses from the Phase 1/2
MARINA® trial (Q1)
- Completion of enrollment of the ongoing Phase 3
HARBORTM trial (mid-2025)
- Update from the ongoing MARINA-OLETM trial including
long-term 4mg/kg and safety data (Q4)
- Planned marketing application submissions in 2026, including in
the U.S. and European Union
- Delpacibart braxlosiran (del-brax) for the treatment of
FSHD:
- Regulatory alignment on a global Phase 3 trial design (Q2)
- Alignment on a potential accelerated approval path for the
ongoing FORTITUDE™ biomarker cohort (Q2)
- Completion of enrollment of the FORTITUDE biomarker cohort
(Q2)
- Presentation of topline data from the FORTITUDE trial (Q2)
- Initiation of a global, potentially registrational trial in
FSHD (Q2)
J.P. Morgan Healthcare Conference – Webcast
Information
Sarah Boyce, President and Chief
Executive Officer, will be presenting at the 43rd
Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 14, 2025 at 7:30 a.m. PT / 10:30 a.m.
ET. A live webcast of the event, up-to-date event
details and an archived replay will be available on the "Events and
Presentations" page in the "Investors" section of Avidity's website
at
https://aviditybiosciences.investorroom.com/events-and-presentations.
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 neuromuscular diseases:
myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD)
and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also
advancing two wholly-owned precision cardiology development
candidates addressing rare genetic cardiomyopathies. In addition,
Avidity is broadening the reach of AOCs with its advancing and
expanding pipeline including programs in cardiology and immunology
through 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 plans for a BLA submission for del-zota
and the timing thereof; the status of three of Avidity's programs
as potentially registrational; the status of Avidity's ongoing
clinical trials and cohorts therein, including but not limited to
initiation, enrollment, design and goals; the ability for del-zota
and del-brax to achieve accelerated approval; the presentation of
additional data, analyses and other updates from Avidity's ongoing
clinical programs and the timing thereof; planned marketing
applications for del-desiran in the U.S. and European Union and the
timing thereof; Avidity's plans to become a global commercial
organization and the status of its commercialization efforts; the
expansion of the AOC platform; the goals of Avidity's personnel
changes and the early initiatives prior to a first potential
product launch; Avidity's plans to present at the J.P. Morgan
Healthcare Conference; and plans and objectives of management for
future operations. 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: the data and results produced in Avidity's
ongoing clinical trials as of the most recent respective cutoff
dates may not be indicative of final results, may not support BLA
submissions or accelerated approvals, may not be satisfactory to
the FDA and other regulators, and new analyses of existing data and
results may produce different conclusions than established as of
the date hereof; the FDA and other regulators may change their
positions on the statuses of Avidity's clinical trials and product
candidates; even if approved, Avidity may not be able to execute
any successful product launches; Avidity's efforts to build a
global commercial organization may be unsuccessful; 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 Avidity's ongoing
clinical trials; Avidity's dependence on third parties in
connection with clinical testing and product manufacturing;
legislative, judicial and 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:
Kat Lange
(619) 837-5014
investors@aviditybio.com
Media Contact:
(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.
