SAN DIEGO, Oct. 24, 2016 /PRNewswire/ -- aTyr Pharma,
Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the
discovery and development of Physiocrine-based therapeutics to
address severe, rare diseases, today announced that its product
candidate Resolaris™ was granted Fast Track designation by the US
Food and Drug Administration (FDA) for the treatment of
facioscapulohumeral muscular dystrophy (FSHD), making it the first
known therapeutic candidate for the treatment of FSHD to receive
the designation. Resolaris, a designated Orphan Drug in FSHD,
is currently being studied in a Phase 1b/2 clinical program.
"This Fast Track designation, which is granted to drug
candidates addressing serious conditions and that demonstrate the
potential to address unmet medical needs, represents another step
forward for our rare muscle disease franchise," said John Mendlein, PhD, CEO of aTyr Pharma. "This
designation highlights the significant need to develop and
ultimately approve meaningful new therapeutics to treat patients
with rare myopathies, such as FSHD."
Fast Track is a process designed by the FDA to facilitate the
development and expedite the review of new drugs, biologics or
treatments. Product candidates may be eligible for priority review
at the time of a Biologic License Application (BLA) filing and may
also be eligible to submit completed sections of the BLA on a
rolling basis. Importantly, the therapy's sponsors are eligible for
more frequent written communication and meetings with the FDA, the
benefit of which may be to develop a pivotal study design which
more closely meets the FDA's criteria, thereby creating a more
collaborative and efficient pathway to approval.
Resolaris is currently being studied in a Phase 1b/2 clinical
program for three types of rare myopathies with an immune
component. The Company expects to report data from the following
three clinical trials in December
2016:
- Phase 1b/2 study in patients with early-onset FSHD (003
Trial)
- Phase 1b/2 study in patients with FSHD and Limb Girdle Muscular
Dystrophy 2B (LGMD2B) (004 Trial)
- Phase 1b/2 Extension study in patients with FSHD (005
Trial)
The primary objectives of these trials are to establish a safety
and tolerability database and to explore and establish activity
signals, such as various endpoints and biomarkers, which will best
inform aTyr's clinical development path forward, including
endpoints for a BLA.
About Resolaris™
aTyr Pharma is developing Resolaris as a potential
first-in-class intravenous protein therapeutic for the treatment of
rare myopathies with an immune component. Resolaris is derived from
a naturally occurring protein released in vitro by human
skeletal muscle cells. aTyr believes Resolaris has the potential to
provide therapeutic benefit to patients with rare myopathies with
an immune component characterized by excessive immune cell
involvement.
About FSHD
Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic
myopathy affecting an estimated 19,000 people in the United States for which there are no
approved treatments. The primary clinical phenotype of FSHD is
debilitating skeletal muscle deterioration and weakness. The
symptoms of FSHD often appear early in the face, shoulder blades,
upper arms, lower legs and trunk, and can affect certain muscles
while adjacent muscles remain healthy. In addition to muscle
weakness, FSHD patients often experience debilitating fatigue and
chronic pain. The disease is typically diagnosed by the presence of
a characteristic pattern of muscle weakness and other clinical
symptoms, as well as through genetic testing. To learn more about
FSHD, please visit
www.fshsociety.org.
About Early-Onset FSHD
While FSHD can manifest at any age, the onset of symptoms in
many patients occurs before the age of 18. We refer to this
patient population as early onset FSHD. aTyr has selected
those patients with onset of symptoms before the age of ten for its
current clinical trial. Within the early onset population are
individuals with symptom onset at less than five years of age, with
progression in disease prior to age ten. These individuals have
generally the most severe muscle symptoms and extra-muscular
manifestations such as auditory deficits and retinal complications
that may result in vision loss. This sub-group of early onset
patients are often referred to as having "infantile onset" FSHD.
Estimates of prevalence vary; however, aTyr believes the "infantile
onset" FSHD population is approximately 1,000 in the U.S.
About LGMD2B
Limb girdle muscular dystrophy (LGMD) refers to a group of rare
genetic myopathies, of which there are more than 20 different
subtypes, none with approved therapies. LGMD affects an estimated
16,000 patients in the U.S., approximately 3,000 of who have
LGMD2B. LGMD2B is a recessive genetic disease caused by mutations
in the dysferlin gene. Patients experience debilitating muscle
weakness and atrophy as well as immune cell invasion in the
skeletal muscle. Patients are primarily assessed for clinical
symptoms to assess skeletal muscle health. To learn more about
LGMD2B please visit www.jain-foundation.org.
About Physiocrines
Physiocrines comprise naturally occurring proteins that aTyr
believes promote homeostasis, a fundamental process of restoring
stressed or diseased tissue to a healthier state. Physiocrines are
extracellular signaling regions of tRNA synthetases, an ancient
family of enzymes that catalyze a key step in protein synthesis.
aTyr is currently focused on Physiocrines that act as endogenous
modulators of the immune system. Physiocrines offer the opportunity
for modulating biological pathways through newly discovered,
naturally occurring mechanisms, many of which may provide
advantages over other types of immune-modulatory therapeutics,
including the potential for improved patient outcomes and reduced
side effect profiles.
About aTyr Pharma
aTyr Pharma is engaged in the discovery and clinical development
of innovative medicines for patients suffering from severe rare
diseases using its knowledge of Physiocrine biology, a newly
discovered set of physiological modulators. The Company's lead
candidate, Resolaris™, is a potential first-in-class intravenous
protein therapeutic for the treatment of rare myopathies with an
immune component. Resolaris is currently in a Phase 1b/2 clinical
trial in adult patients with facioscapulohumeral muscular dystrophy
(FSHD); a Phase 1b/2 trial in adult patients with limb-girdle
muscular dystrophy 2B (LGMD2B or dysferlinopathies) or FSHD; and a
Phase 1b/2 trial in patients with an early onset form of FSHD. aTyr
has built an intellectual property estate, to protect its pipeline,
comprising over 80 issued or allowed patents and over 230 pending
patent applications that are owned or exclusively licensed by aTyr,
including over 300 potential Physiocrine-based protein
compositions. aTyr's key programs are currently focused on severe,
rare diseases characterized by immune dysregulation for which there
are currently limited or no treatment options. For more
information, please visit http://www.atyrpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Litigation Reform Act.
Forward-looking statements are usually identified by the use of
words such as "anticipates," "believes," "estimates," "expects,"
"intends," "may," "plans," "projects," "seeks," "should," "will,"
and variations of such words or similar expressions. We intend
these forward-looking statements to be covered by such safe harbor
provisions for forward-looking statements and are making this
statement for purposes of complying with those safe harbor
provisions. These forward-looking statements, including statements
regarding the potential of Resolaris, the ability of the Company to
undertake certain development activities (such as clinical trial
enrollment and the conduct of clinical trials) and accomplish
certain development goals, and the timing of initiation of
additional clinical trials and of reporting results from our
clinical trials reflect our current views about our plans,
intentions, expectations, strategies and prospects, which are based
on the information currently available to us and on assumptions we
have made. Although we believe that our plans, intentions,
expectations, strategies and prospects as reflected in or suggested
by those forward-looking statements are reasonable, we can give no
assurance that the plans, intentions, expectations or strategies
will be attained or achieved. Furthermore, actual results may
differ materially from those described in the forward-looking
statements and will be affected by a variety of risks and factors
that are beyond our control including, without limitation, risks
associated with the discovery, development and regulation of our
Physiocrine-based product candidates, as well as those set forth in
our most recent Annual Report on Form 10-K for the year ended
December 31, 2015 and in our
subsequent SEC filings. Except as required by law, we assume no
obligation to update publicly any forward-looking statements,
whether as a result of new information, future events or
otherwise.
Contact:
Mark
Johnson
Sr. Director, Investor Relations
mjohnson@atyrpharma.com
858-223-1163
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