FSHD Society to host Avidity webinar on
positive initial data from the Phase 1/2 FORTITUDE™ trial for
people living with FSHD today at 1:00 p.m.
ET
SAN
DIEGO, June 20, 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 it is
partnering with the FSHD Society and joining the global community
of patients, caregivers, and healthcare providers to raise
awareness of facioscapulohumeral muscular dystrophy in support of
World FSHD Day. The FSHD Society is hosting a webinar with Avidity
today at 1:00 p.m. ET to share recent
unprecedented del-brax data from its Phase 1/2 FORTITUDE™
trial in people living with FSHD, a rare, hereditary disorder
marked by life-long, relentless loss of muscle function,
significant pain, fatigue, and progressive disability.
"This World FSHD Day, we are excited to be joining the FSHD
Society to share recent unprecedented data from our FORTITUDE study
with the patient and caregiver community," said Sarah Boyce, president and chief executive
officer at Avidity. "We know the FSHD community is eagerly waiting
for an approved therapy and we are committed to bringing forward a
treatment as quickly as possible to the many families worldwide who
are affected by FSHD. We are extremely thankful for the important
contributions of patients, caregivers and families that continue to
better inform our ongoing research and development as we advance
del-brax for people living with FSHD."
"As the world's largest research-focused patient organization,
we are dedicated to speeding the delivery of treatments and a cure
for FSHD. With the remarkable data from Avidity's FORTITUDE study
presented last week, we feel invigorated that there may be a
potential treatment to change the course of this relentlessly
progressive disease for people and families affected by FSHD," said
Mark Stone, president and chief
executive officer of FSHD Society. "We remain deeply committed to
our collaborations with industry leaders such as Avidity who are
accelerating innovative research. World FSHD Day is an important
opportunity to recognize the partnership, commitment and work that
is necessary to bring more support and more hope to families
impacted by FSHD every day."
In the Phase 1/2 study, del-brax, the first
investigational therapy designed to treat the underlying cause of
FSHD, demonstrated unprecedented and consistent reductions of
greater than 50% in DUX4 regulated genes, trends of functional
improvement, and favorable safety and tolerability in people living
with FSHD. Currently, there are no approved therapies for the
treatment of FSHD.
In an online webinar today hosted by the FSHD Society, the
Avidity team, joined by Dr. Jeffrey
Statland, University of Kansas Medical
Center and FORTITUDE trial investigator, will present the
FORTITUDE trial initial findings, focusing on what these results
mean for the FSHD community.
Date: Thursday,
June 20, 2024
Time: 10:00 a.m. Pacific Time
/ 1:00 p.m. Eastern Time
How to join: You can register to join the webinar on the
FSHD Society's website
(https://www.fshdsociety.org/event/fshd-university-fortitude-trial-interim-results/).
A recording of this webinar will be available on FSHD Society's
FSHD University at a later date.
Every June 20th, people around the
world join in activities to raise awareness of FSHD through World
FSHD Day and to recognize patients and families worldwide who are
affected by FSHD. This year, Avidity participated in the 31st
Annual FSHD Society International Research Congress (FSHD IRC),
where Jeffrey M. Statland, M.D.,
Professor of Neurology, University of Kansas
Medical Center, and FORTITUDE trial investigator, presented
the new initial data from the FORTITUDE study. Avidity also
participated in the FSHD Connect Conference, an educational
conference specifically for people living with FSHD and their
families. Both conferences are organized by the FSHD Society, the
world's largest research-focused patient organization for FSHD.
About the Phase 1/2 FORTITUDE™ trial
The FORTITUDE™ trial is a randomized, placebo-controlled,
double-blind, Phase 1/2 clinical trial designed to evaluate single
and multiple doses of delpacibart braxlosiran or
del-brax (AOC 1020) in approximately 39 adult participants
with facioscapulohumeral muscular dystrophy (FSHD). FORTITUDE will
evaluate the safety, tolerability, pharmacokinetics, and
pharmacodynamics of del-brax administered intravenously,
with the primary objective being the safety and tolerability of
del-brax in FSHD patients. Activity of del-brax will
be assessed using key biomarkers, including magnetic resonance
imaging (MRI) measures of muscle volume and composition. Though the
Phase 1/2 trial is not statistically powered to assess functional
benefit, it will explore the clinical activity of del-brax
including measures of mobility and muscle strength as well as
patient reported outcomes and quality of life measures.
Participants will have the option to enroll in an open-label
extension study at the end of the treatment period in the FORTITUDE
study. For more information about the FORTITUDE trial, visit
the FORTITUDE study website or
visit http://www.clinicaltrials.gov and search for
NCT05747924.
About Del-brax (AOC 1020)
Del-brax (AOC 1020) is designed to treat the underlying
cause of FSHD, which is caused by the abnormal expression of a gene
called double homeobox 4 or DUX4. The abnormal expression of DUX4
protein leads to changes in gene expression in muscle cells that
are associated with the life-long, progressive loss of muscle
function in patients with FSHD. Del-brax aims to reduce the
expression of DUX4 mRNA and DUX4 protein in muscles in people with
FSHD. Del-brax consists of a proprietary monoclonal antibody
that binds to the transferrin receptor 1 (TfR1) conjugated with a
siRNA targeting DUX4 mRNA. In preclinical studies, a single
intravenous dose with the murine version of del-brax
prevented development of muscle weakness demonstrated by three
functional assays - treadmill running, in vivo force and compound
muscle action potential. Del-brax is currently in Phase 1/2
development as part of the FORTITUDE™ trial in adults
with FSHD. The U.S. Food and Drug Administration (FDA) and the
European Medicines Agency (EMA) have granted Orphan designation for
del-brax and the FDA has granted del-brax Fast Track
designation.
About Facioscapulohumeral Muscular Dystrophy
(FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is a
rare, progressive, and variable hereditary muscle-weakening
condition marked by significant pain, fatigue, and disability. It
is characterized by progressive and often asymmetric skeletal
muscle loss that initially causes weakness in muscles in the face,
shoulders, arms and trunk and progresses to weakness in muscles in
the lower body. FSHD is an autosomal dominant disease caused by the
aberrant expression of the DUX4 (double homeobox 4) gene in the
skeletal muscle, which activates genes that are toxic to muscle
cells and leads to a series of downstream events that result in
skeletal muscle wasting and compromised muscle function. Skeletal
muscle weakness results in physical limitations throughout the
whole body, including an inability to lift arms for more than a few
seconds, loss of ability to show facial expressions and serious
speech impediments. These symptoms cause many people affected by
FSHD to become dependent on the use of a wheelchair for mobility.
Currently, there are no approved treatments for people living with
FSHD.
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:
the characterization of safety, tolerability and functional data
associated with del-brax from the Phase 1/2 FORTITUDE™
trial; the impact of such data on the advancement of
del-brax; timeline for advancing del-brax; the
status and potential of del-brax as the first
investigational therapy designed to treat the underlying cause of
FSHD; the potential of Avidity's product candidates to treat rare
diseases and Avidity's efforts to bring them to people suffering
from applicable diseases; and the potential of AOCs™ to target a
range of different cells and tissues beyond the liver, and to treat
cardiac and immunological diseases. This press release also
contains estimates and other statistical data made by independent
parties and by us. This data involves a number of assumptions and
limitations, and the reader is cautioned not to give undue weight
to such estimates.
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 those beyond its control, including, without
limitation: preliminary results of a clinical trial are not
necessarily indicative of final results and additional participant
data related to del-brax that continues to become available
may be inconsistent with the data produced as of the date hereof,
and further analysis of existing data and analysis of new data may
lead to conclusions different from those established as of the data
cutoff; 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 additional clinical holds which may not be timely lifted,
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; 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; and
other risks described in Avidity's Annual Report on Form 10-K for
the fiscal year ended December 31,
2023, filed with the Securities and Exchange Commission
(SEC) on February 28, 2024, 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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