SAN DIEGO, Oct. 6, 2016 /PRNewswire/ -- aTyr Pharma,
Inc. (Nasdaq: LIFE), today announced that additional clinical data
from aTyr's Phase 1b/2 Trial (002) in adult patients with FSHD were
presented at the 21st International Congress of the
World Muscle Society in Granada, Spain. The poster presentation is titled "A
Randomized, Double-blinded, Placebo-controlled, Multiple Ascending
Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics,
Immunogenicity, and Biological Activity of ATYR1940 (Resolaris™) in
Adult Patients with Facioscapulohumeral Muscular Dystrophy."
"In today's presentation we have provided additional data
analyses which reaffirm encouraging trends in two meaningful
clinical assessments from our adult FSHD (002) trial," commented
John Mendlein, PhD, CEO of aTyr
Pharma. "In December, we look forward to sharing further top-line
updates from our Phase 1b/2 clinical program with Resolaris in
three different rare myopathies from our early onset FSHD (003)
trial, our LGMD2B/FSHD (004) trial, and our first FSHD extension
(005) trial."
Today's presentation includes additional detailed data from the
adult FSHD (002) trial covering safety, tolerability, PK, and
clinical assessments. Encouraging activity was seen in the
patient-reported outcomes, known as the Individualized
Neuromuscular Quality of Life Assessment (INQoL), and the
physician-reported functional assessment Manual Muscle Testing
(MMT).
Patients treated with Resolaris were generally improved compared
with placebo as assessed by INQoL, with patients in cohort 3 (3.0
mg/kg for 12 weeks) showing the greatest improvement compared with
cohort 1 and cohort 2 (0.3 mg/kg and 1.0 mg/kg for 4 weeks
respectively). Patients in cohort 3 reported a ~9.9% improvement in
INQoL compared with a ~15.6% worsening in the placebo group at week
14. Five out of the six patients on Resolaris in cohort 3
showed overall improvement in their INQoL score at week 14, versus
zero out of two patients on placebo.
A trend for improvement in MMT results with Resolaris treatment
was also observed compared with placebo, especially in the upper
limbs. In general, there was an association between changes in
INQoL and MMT, whereby all patients who experienced an improvement
in muscle function also showed improvement in INQoL scores.
Patients in cohort 3 reported ~0.7% improvement in MMT compared
with ~1.4% worsening in the placebo group at week 14. Three out of
the six patients on Resolaris in cohort 3 showed overall
improvement in their MMT score at week 14, versus zero out of two
patients on placebo.
Conclusions of the Adult FSHD (002)
Trial:
- Over the dose and duration studied, Resolaris was found to be
generally safe in adult patients with FSHD, and was generally well
tolerated with the exception of one patient who experienced
infusion related reactions as previously reported and as discussed
in more detail in our poster presentation.
- PK properties were dose-proportional and generally consistent
throughout the study, with no measurable impact from anti-drug
antibodies (ADAs).
- Clinical activity was supported by signals of improvement in
INQoL questionnaire responses and MMT measures.
- Other exploratory measures, including lower extremity targeted
MRI, did not demonstrate activity. Variability in image acquisition
may have diminished the opportunity to show activity in this
measurement.
- Patients in cohorts 2 and 3 were allowed to enroll in the
ongoing long-term extension study (005) investigating the safety
and efficacy of Resolaris, for which aTyr expects to provide an
update in December 2016.
"We are excited to have observed signals of activity across two
clinical assessments with both INQoL and MMT, which is a first
for FSHD patients," said Sanjay
Shukla, MD, MS, Chief Medical Officer of aTyr Pharma.
"Directional improvement in two separate assessments that should
otherwise decline over time may be evidence of how clinically
meaningful Resolaris could be for FSHD patients, and we look
forward to building upon the clinical activity and safety data with
our additional data later this year."
For additional information please refer to aTyr Pharma's
investor relations website (www.atyrpharma.investorroom.com) to
access today's full poster presentation.
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. Early onset FSHD
occurs in individuals who experience symptoms of progressive muscle
involvement as juveniles, and some of these patients suffer from a
particularly severe form of the disease. To learn more about FSHD,
please visit
www.fshsociety.org.
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 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. To
protect this pipeline, aTyr has built an intellectual property
estate 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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