Participants currently enrolled in MARINA and
MARINA-OLE™ trials may continue to be treated with AOC
1001
Avidity received Investigational New Drug
(IND) clearance for FSHD and DMD studies from FDA; programs now
advancing into the clinic
Company to host investor webcast today at
8:30 a.m. ET / 5:30 a.m. PT
SAN
DIEGO, Sept. 27, 2022 /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 that
the U.S. Food and Drug Administration (FDA) has placed a partial
clinical hold on new participant enrollment in the Phase 1/2
MARINA™ clinical trial of AOC 1001 in adults with myotonic
dystrophy type 1 (DM1). Close to 40 participants are currently
enrolled in the MARINA and MARINA open label extension
(MARINA-OLE™) trials.
All participants, whether they are on AOC 1001 or placebo, may
continue in their current dosing cohort although no additional
participants may be enrolled until the partial clinical hold is
resolved. All participants in MARINA may roll over into the
MARINA-OLE where they will receive AOC 1001 as planned. DM1 is an
underrecognized, progressive and often fatal neuromuscular disease
with no approved treatment options.
The partial clinical hold is in response to a serious adverse
event reported in a single participant in the 4mg/kg cohort of the
MARINA study. Avidity is working closely with the FDA and the trial
investigator to assess the cause of this event. The company is
taking all necessary steps to resolve the partial clinical hold on
new participant enrollment as quickly as possible.
"The safety of participants enrolled in our clinical studies is
our first priority. We are doing a thorough analysis and will work
diligently with the FDA and the trial investigator to follow the
progress of this participant and to resume new participant
enrollment as soon as we can," said Sarah
Boyce, president and chief executive officer at Avidity. "We
share the sense of urgency with the DM1 community for effective
therapies and we remain confident that AOC 1001 has the potential
to address important unmet needs of people living with DM1. We want
to thank each participant in the study, their families and the
investigators for their continued contributions."
"We continue to look forward to the preliminary assessment of
the MARINA study in Q4 and, with our two recent IND clearances, we
are now advancing AOC 1020 for FSHD and AOC 1044 for DMD into the
clinic this year as planned," added Ms. Boyce.
Avidity remains on track to conduct a preliminary assessment of
safety, tolerability and key biomarkers in approximately half of
the study participants in the MARINA trial in the fourth quarter of
2022.
Avidity received IND clearance from the FDA to proceed with the
clinical trial of AOC 1020 for the treatment of facioscapulohumeral
muscular dystrophy (FSHD) and AOC 1044 for the treatment of
Duchenne muscular dystrophy (DMD) with mutations amenable to exon
44 skipping. These programs are now advancing into the clinic.
Today's Webcast Information
Avidity's management team
will host a webcast and conference call at 8:30 a.m. ET / 5:30 a.m.
PT today, September 27, 2022.
The live call can be accessed by dialing 866-652-5200 (US) and
412-317-6060 (International) and requesting Avidity Biosciences. A
live webcast will also be available on 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.
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'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 now has three programs in clinical
development. The company's lead product candidate, AOC 1001, is
designed to treat people with myotonic dystrophy type 1 (DM1). AOC
1001 is currently in Phase 1/2 development with the ongoing
MARINA™ trial and MARINA-OLE™ in adults with DM1. The next
programs in the company's advancing and expanding pipeline are AOC
1044, the lead of three programs for the treatment of DMD, and AOC
1020, designed to treat people living with FSHD. The FDA has
cleared Avidity to proceed into the clinic with both AOC 1020 and
AOC 1044. 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 San Diego,
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: expectations for Avidity's
interactions with the FDA, the ongoing investigation into the
underlying cause of the serious adverse event for the affected
patient, and the anticipated impact of, and Avidity's ability to
resolve, the partial clinical hold and resume enrollment in and
complete the MARINA study, and to conduct and present data from the
preliminary assessment of the MARINA study and the timing thereof;
the progression of clinical programs for AOC 1001, AOC 1044 and AOC
1020 and timing thereof; and the potential of AOC 1001 to treat
DM1. 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 may not be
able to resolve the partial clinical hold and the analysis related
to the underlying cause of the serious adverse event may result in
delays in the MARINA study or an inability to compete the study;
unexpected adverse side effects or inadequate efficacy of its
product candidates that may delay or limit their development,
regulatory approval and/or commercialization, or may result in
clinical holds, 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 and completion of
preclinical studies or clinical trials; 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 and
clinical testing and product manufacturing; 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; disruption to its operations from the COVID-19 pandemic or
the war in Ukraine; 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.
Investor Contact:
Kathleen
Gallagher
(858) 401-7900 x550
investors@aviditybio.com
Media Contact:
Navjot
Rai
(858) 401-7900 x550
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.