Avidity plans to initiate global Phase 3
HARBOR™ trial of AOC 1001 for DM1
mid-2024
Company to report data from all three ongoing
clinical programs in 2024
-- AOC 1001 data for DM1
in Q1, AOC 1020 data for FSHD in Q2, and AOC 1044 data for DMD44 in
2H --
Avidity to advance wholly-owned and partnered
cardiology programs toward clinical development
Avidity appoints Eric B. Mosbrooker as Chief
Strategy Officer
SAN
DIEGO, Jan. 5, 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 announced its
2024 corporate priorities and catalysts for the next stage of
growth. In mid-2024, Avidity plans to initiate the global Phase 3
HARBOR™ trial of AOC 1001 for adults living with myotonic
dystrophy type 1 (DM1). The robust data package of AOC 1001 from
the Phase 1/2 MARINA® trial and open-label extension
study, MARINA-OLE™, has demonstrated consistent improvements
across multiple functional endpoints including myotonia, muscle
strength and mobility, and long-term favorable safety and
tolerability results in people living with DM1. Avidity also plans
to report data in 2024 from all three of its ongoing clinical
programs targeting three distinct rare muscle diseases:
DM1, Duchenne muscular dystrophy with mutations amenable
to exon 44 skipping (DMD44) and facioscapulohumeral muscular
dystrophy (FSHD) while advancing its cardiology programs toward
clinical development. In addition, Avidity announced the
appointment of Eric B. Mosbrooker as Chief Strategy Officer.
2024 Upcoming Milestones & Key Highlights
- Initiation of global Phase 3 HARBOR™ trial of AOC 1001 for
DM1 in mid-2024
- Data readouts from all three clinical programs for three
distinct muscle diseases
- Q1 2024: First look at AOC 1001 efficacy data from
MARINA-OLE™ trial in people living with DM1
- Q2 2024: AOC 1020 data from a preliminary assessment in
approximately half of patients in Phase 1/2 FORTITUDE™ trial in
FSHD
- 2H 2024: First look at AOC 1044 data in people living with
DMD44 from Phase 1/2 EXPLORE44™ trial
- Dose-escalation of remaining study participants from 2 mg/kg to
4 mg/kg of AOC 1001 in the MARINA-OLE trial
- Advance wholly-owned and partnered cardiology programs toward
clinical development following recent expansion of cardiology
collaboration with Bristol Myers Squibb
- Strong cash balance with funding through 2025
"In 2024, we will continue to build on the remarkable functional
data and long-term safety data from our DM1 clinical program and
unprecedented data from our DMD44 clinical program while executing
across multiple significant research and development initiatives.
We plan to initiate the global pivotal study for people living with
DM1, report data from all three of our clinical programs in rare
muscle diseases, and advance our cardiology programs toward
clinical development," said Sarah
Boyce, president and chief executive officer. "In addition,
as we evolve from a research and development company to an
integrated global organization, we are excited to have Eric
Mosbrooker join our leadership team. His proven track record in
global commercial operations and deep understanding of our
company's strategic imperatives as part of his tenure on our board
will be valuable to the company in our next phase of growth."
With more than 20 years of experience in global commercial
operations, Mr. Mosbrooker brings a wealth of expertise and
leadership in areas including global pricing, marketing,
distribution, and product launches in the life sciences industry.
He has expertise in gene therapy, rare metabolic diseases,
additional orphan conditions and oncology. Mr. Mosbrooker, who
served two and a half years as a member of Avidity's board of
directors, has stepped down from his board position to join the
management team.
"I am honored to join Avidity's management team and enthusiastic
about the immense potential of our AOC platform to improve the
lives of people affected by serious diseases," said Mr. Mosbrooker,
chief strategy officer at Avidity. "With a catalyst-rich 2024
ahead, I am excited to bring a global commercial view and strategic
insights as we fulfill our mission to profoundly improve people's
lives by revolutionizing a new class of targeted RNA
therapeutics."
Prior to joining Avidity, Mr. Mosbrooker served as the Chief
Operations Officer for Cognoa, where he spearheaded commercial
initiatives, program management, product development, and business
operations. He also previously held the roles of Chief Commercial
Officer at Audentes Therapeutics, where he oversaw the gene therapy
business unit, and Senior Vice President of the Global Orphan
Business Unit at Horizon Pharmaceuticals. Mr. Mosbrooker holds a
Bachelor of Science in Industrial Engineering from the University of Wisconsin – Madison.
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 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: Avidity's planned milestones,
their prospects for success and the timing thereof; Avidity's
corporate priorities; 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; plans for the progression of research and development
initiatives, including in cardiology; Avidity's collaboration with
Bristol Myers Squibb; Avidity's cash balance and ability to fund
its operations; the appointment of a chief strategy officer and its
importance to Avidity; Avidity's progress and evolution as a
company; 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, or may result in clinical holds which may not be
timely lifted (if at all), recalls or product liability claims;
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, 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 fail to raise additional needed funds; and
other risks described in Avidity's Annual Report on Form 10-K for
the fiscal year ended December 31,
2022, filed with the Securities and Exchange Commission
(SEC) on February 28, 2023, and in
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
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SOURCE Avidity Biosciences, Inc.