SAN DIEGO, Aug. 10, 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 operating results
for the second quarter ended June 30,
2016.
"We continue to execute on our vision to bring innovative
therapeutics to patients severely afflicted by rare diseases.
During this last quarter, we made considerable progress toward that
goal on three fronts: first, we advanced Resolaris in three
distinct opportunities to help patients with rare myopathies with
an immune component (RMIC)," said John
Mendlein, PhD, Chief Executive Officer of aTyr Pharma.
"Second, we further elucidated the mechanism of the Resokine
pathway which includes affecting T-cell activation; and finally, we
have advanced our second program, iMod.Fc, to cGMP manufacturing
for GLP safety studies and are on track to have that candidate
enter the clinic in 2017. We also want to thank the 35
facioscapulohumeral muscular dystrophy (FSHD) and 10 limb-girdle
muscular dystrophy 2B (LGMD2B) patients that have participated or
are participating in our clinical trials, as well as the physicians
and investigators that make these studies possible across 14 global
sites."
Recent Highlights and Upcoming Milestones
Following the encouraging results from our previously announced
Phase 1b/2 trial of Resolaris in patients diagnosed with adult
FSHD, our 002 trial, we continued to enroll patients across three
ongoing clinical studies for three different types of rare
myopathies with an immune component. The primary objectives of
these studies are to establish a safety and tolerability database
and to explore and establish activity signals, such as various
functional endpoints and biomarkers, which will best inform our
clinical development path forward, including endpoints for a
Biologics License Application (BLA).
Highlights from our ongoing Resolaris program include:
- Early Onset FSHD (003) Trial – Our Phase 1b/2 trial is
enrolling patients diagnosed with early-onset FSHD, which is often
the most severe form of FSHD. Patients entering the trial
experienced onset of disease by the age 10, and the trial includes
a Stage 1, with patients ages 16 to 25, and a Stage 2, with
patients ages 12 to 15. We expect to announce data from the first
four patients enrolled in Stage 1 in the fourth quarter of
2016.
- LGMD2B/FSHD (004) Trial – Our Phase 1b/2 trial in adult
patients diagnosed with either FSHD or LGMD2B completed enrollment
ahead of schedule in May with 18 total patients (8 with FSHD and 10
with LGMD2B), exceeding our stated target enrollment of 16
patients. We expect to announce results in the fourth quarter of
2016.
- First Extension (005) Trial – Our long-term extension
study of Resolaris for our adult FSHD Phase 1b/2 trial (002)
continues to dose patients that were eligible to roll over. We
expect to announce an update from these patients in the fourth
quarter of 2016.
- Second Extension (006) Trial – During the quarter, we
initiated a long-term extension study of adult FSHD, early-onset
FSHD, and LGMD2B patients from our ongoing 003 and 004 trials.
Our scientists, clinical operations professionals, and
clinicians continued to advance our science, protein manufacturing,
and programs with recent highlights, including:
- Mechanism of Action – The Resokine pathway includes
interactions with activated T-cells. Our scientists continue to
elucidate the role of the Resokine pathway in affecting the level
of activation of T-cells by CD3 & CD28. Resolaris is a 56kD
protein, with a naturally occurring sequence of amino acids, that
harnesses the Resokine pathway and interacts with T-cells to 'place
a brake' on T-cell activation.
- iMod.Fc Program – iMod.Fc, our second product
development candidate, comprises an Fc region of an antibody
genetically engineered to a protein, derived from the Resokine
pathway. Fc fusion proteins, such as iMod.Fc, include an additional
antibody domain to enhance pharmacokinetic, or PK, and distribution
characteristics. We plan to evaluate its therapeutic potential in
patients with rare pulmonary diseases with an immune and/or
fibrotic component. We continue to prepare for clinical trial
initiation in 2017 by initiating large scale drug substance cGMP
manufacturing.
- Natural History Study Collaboration – We recently
entered into a collaboration with The Cooperative International
Neuromuscular Research Group on a longitudinal natural history
study, comprised of 53 patients, to advance knowledge of early
onset FSHD. We provide financial support to this study, which was
initially implemented by the FSH Society, the FSHD Global Research
Foundation and Muscular Dystrophy Canada.
- Expanding Leadership – In May, we appointed Grove
Matsuoka as Senior Vice President, Product Programs and Planning.
Mr. Matsuoka's most recent role was Senior VP, Commercialization at
CoDa Therapeutics, Inc. Prior to that, he worked in various
positions at Amgen, Inc., most recently as Director, Medical
Affairs, Strategic Planning and Operations, where he established
and managed the strategic and operational function for the newly
formed Medical Affairs organization. He was also Clinical Research
Project Team Leader for an Fc Fusion Program while at Amgen.
- Robust Patent Estate – We have recently been issued or
allowed an additional 17 patents that are now part of an
intellectual property estate comprising over 80 issued or allowed
patents and over 230 pending patent applications that we own or
exclusively license, including over 300 potential Physiocrine-based
protein compositions.
Second Quarter 2016 Financial Results
Research and development expenses were $11.3 million and $7.5
million for the quarters ended June
30, 2016 and 2015, respectively. The increase of
$3.8 million was due primarily to a
$1.9 million increase in clinical and
non-clinical development costs for Resolaris, a $0.8 million increase related to compensation
expenses resulting from increased headcount in research and
development functions, including $0.1
million in non-cash stock-based compensation, a $0.7 million increase related to cGMP
manufacturing of Resolaris to support future clinical trials, and a
$0.4 million increase in other
pre-clinical development costs.
Sequentially, there was a $2.4
million decrease related to the cGMP manufacturing of
Resolaris in the second quarter of 2016 versus the first quarter of
2016, as much of the investment required to supply future clinical
trials was spent in the first quarter of 2016, partially offset by
a $1.2 million increase in clinical
and non-clinical development costs for Resolaris.
General and administrative expenses were $4.1 million and $3.4
million for the quarters ended June
30, 2016 and 2015, respectively. The increase of
$0.7 million was due primarily to a
$0.5 million increase in non-cash
stock-based compensation and a $0.2
million increase in costs associated with being a public
company.
First Half 2016 Financial Results
Research and development expenses were $23.3 million and $14.1
million for the six months ended June
30, 2016 and 2015, respectively. The increase of
$9.2 million was due primarily to a
$4.7 million increase related to cGMP
manufacturing of Resolaris to support future clinical trials, a
$3.5 million increase in clinical and
non-clinical development costs for Resolaris, a $1.6 million increase related to compensation
expenses resulting from increased headcount in research and
development functions, including $0.3
million of non-cash stock-based compensation, and
$0.7 million in other pre-clinical
development costs. The increase was offset by a decrease
related to a one-time $1.4 million
non-cash expense for the assignment of certain intellectual
property rights in the prior year period.
General and administrative expenses were $8.2 million and $5.7 million for the six months ended
June 30, 2016 and 2015, respectively.
The increase of $2.5 million was
due primarily to a $1.8 million
increase in personnel costs resulting from increased headcount
inclusive of $1.0 million of non-cash
stock-based compensation and a $0.6
million increase in costs associated with being a public
company.
Financial Guidance
As of June 30, 2016, we had $96.6 million in
cash, cash equivalents and investments and 23.7 million shares of
common stock outstanding.
We currently expect that our cash, cash equivalents and
investments will be sufficient to fund our anticipated operations
into 2018.
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 provide advantages
over engineered immune-modulatory therapeutics, including the
potential for improved patient outcomes and reduced side effect
profiles.
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. 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 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 whom 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 LGMD2 please visit www.jain-foundation.org.
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™ or iMod.Fc, 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, the timing of initiation of
additional clinical trials and of reporting results from our
clinical trials and projected cash expenditures 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.
ATYR PHARMA
INC.
|
Condensed
Consolidated Statements of Operations
|
(unaudited, in
thousands, except share and per share data)
|
|
|
Three Months
Ended June
30,
|
Six Months
Ended
June 30,
|
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
$
|
11,307
|
|
$
|
7,502
|
|
$
|
23,307
|
|
$
|
14,095
|
General and
administrative
|
|
4,126
|
|
|
3,396
|
|
|
8,241
|
|
|
5,725
|
Total operating
expenses
|
|
15,433
|
|
|
10,898
|
|
|
31,548
|
|
|
19,820
|
Loss from
operations
|
|
(15,433)
|
|
|
(10,898)
|
|
|
(31,548)
|
|
|
(19,820)
|
Other income
(expenses), net
|
|
50
|
|
|
(182)
|
|
|
78
|
|
|
(331)
|
Net loss
|
|
(15,383)
|
|
|
(11,080)
|
|
|
(31,470)
|
|
|
(20,151)
|
Accretion to
redemption value of redeemable convertible preferred
stock
|
|
—
|
|
|
(15)
|
|
|
—
|
|
|
(15)
|
Net loss attributable
to common stockholders
|
|
(15,383)
|
|
|
(11,095)
|
|
|
(31,470)
|
|
|
(20,166)
|
Net loss per share
attributable to common stockholders, basic and diluted
|
$
|
(0.65)
|
|
$
|
(0.74)
|
|
$
|
(1.33)
|
|
$
|
(2.53)
|
Weighted average
shares outstanding, basic and diluted
|
|
23,672,527
|
|
|
14,901,473
|
|
|
23,655,366
|
|
|
7,955,973
|
ATYR PHARMA
INC.
|
Condensed
Consolidated Balance Sheets
|
(in
thousands)
|
|
|
|
June 30,
|
|
December 31,
|
|
|
2016
|
|
2015
|
|
|
(unaudited)
|
|
|
|
Cash, cash
equivalents and available-for-sale investments
|
|
$
|
96,581
|
|
$
|
125,349
|
Other
assets
|
|
|
2,139
|
|
|
2,533
|
Property and
equipment, net
|
|
|
1,766
|
|
|
1,793
|
Total
assets
|
|
$
|
100,486
|
|
$
|
129,675
|
|
|
|
|
|
|
|
Accounts payable,
accrued expenses and other liabilities
|
|
$
|
10,428
|
|
$
|
9,483
|
Total commercial bank
debt
|
|
|
3,489
|
|
|
5,142
|
Stockholders'
equity
|
|
|
86,569
|
|
|
115,050
|
Total liabilities and
stockholders' equity
|
|
$
|
100,486
|
|
$
|
129,675
|
Contact:
Mark Johnson
Sr. Director,
Investor Relations
mjohnson@atyrpharma.com
858-223-1163
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SOURCE aTyr Pharma, Inc.