aTyr Pharma Announces Third Quarter 2023 Results and Provides Corporate Update
November 09 2023 - 4:00PM
aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a
clinical stage biotechnology company engaged in the discovery and
development of first-in-class medicines from its proprietary tRNA
synthetase platform, today announced third quarter 2023 results and
provided a corporate update.
“During the third quarter we made meaningful
progress with our clinical development program for our lead
therapeutic candidate, efzofitimod, in interstitial lung disease
(ILD),” said Sanjay S. Shukla, M.D., M.S., President and Chief
Executive Officer of aTyr. “We had a positive data and safety
monitoring board (DSMB) review for our global pivotal Phase 3
EFZO-FIT™ study in patients with pulmonary sarcoidosis, a major
form of ILD with high unmet medical need. This study continues to
enroll in the U.S., Europe and Japan and based on current
projections, we expect to complete enrollment in this study early
in the second quarter of 2024. Additionally, we dosed the first
patient in our Phase 2 EFZO-CONNECT™ study in patients with
systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD),
which is currently enrolling in the U.S.”
Third Quarter 2023 and Subsequent Period
Highlights
- Continued
enrollment in the global pivotal Phase 3 EFZO-FIT™ study to
evaluate the efficacy and safety of efzofitimod in patients with
pulmonary sarcoidosis. This is a randomized, double-blind,
placebo-controlled, 52-week study consisting of three parallel
cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of
efzofitimod or placebo dosed intravenously monthly for a total of
12 doses. The study intends to enroll up to 264 subjects with
pulmonary sarcoidosis. The study is open for enrollment at nearly
all of the centers intended in the U.S., Europe and Japan and is
expected to expand to include centers in Brazil. Based on current
enrollment projections, the Company expects to complete enrollment
in the study early in the second quarter of 2024.
-
Completed a positive DSMB review for the Phase 3
EFZO-FIT™ study. Data from the pre-planned,
interim analysis of safety and tolerability of efzofitimod in
patients with pulmonary sarcoidosis, which included the evaluation
of patients who completed treatment, was evaluated. There were no
drug-related serious adverse events, consistent with prior studies.
The DSMB assessed that the study could continue unmodified and that
the drug does not pose any undue risk to the patient that warrants
additional safety measures.
- Dosed the
first patient in the Phase 2 EFZO-CONNECT™ study to evaluate the
efficacy, safety and tolerability of efzofitimod in patients with
SSc-ILD. This proof-of-concept study is a randomized,
double-blind, placebo-controlled, 28-week study consisting of three
parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of
efzofitimod or placebo dosed intravenously monthly for a total of 6
doses. The study intends to enroll 25 patients at multiple centers
in the U.S. The primary objective of the study is to evaluate the
efficacy of multiple doses of intravenous efzofitimod on pulmonary,
cutaneous and systemic manifestations in patients with
SSc-ILD.
-
Peer-reviewed article for efzofitimod published in the
journal Frontiers in
Pharmacology. The publication, titled,
“Exposure-response analyses of efzofitimod in patients with
pulmonary sarcoidosis,” highlights a positive exposure response
demonstrated by efzofitimod across multiple clinically relevant
endpoints in the Phase 1b/2a study in patients with pulmonary
sarcoidosis.
- Presented
two posters for efzofitimod at the European Respiratory Society
(ERS) International Congress 2023. The posters presented
new data from a pooled, post hoc analysis from the Phase 1b/2a
study of efzofitimod in patients with pulmonary sarcoidosis that
further demonstrates efficacy and findings that identify the
expression of neuropilin-2 (NRP2), efzofitimod’s binding partner,
in the skin of patients with SSc-ILD.
- Poster for
efzofitimod accepted for presentation at the upcoming American
College of Rheumatology (ACR) Convergence 2023. The
conference is scheduled to take place November 10 – 15, 2023, in
San Diego, CA. The poster presents new data demonstrating the
effects of efzofitimod in preclinical models of rheumatoid
arthritis (RA) and RA-associated lung fibrosis.
- Poster 1322 –
Efzofitimod, a First-in-Class NRP2-targeting Immunomodulator,
Ameliorates Rheumatoid Arthritis and Associated Lung Fibrosis in
Preclinical Models on Monday, November 13, 2023, from 9:00 a.m. to
11:00 a.m. PST.
Third Quarter 2023 Financial Highlights
and Cash Position
- Cash &
Investment Position: Cash, restricted cash, cash
equivalents and investments as of September 30, 2023, were $105.6
million. Based on the Company’s current operational plans and
existing cash, the Company updates its prior guidance and believes
its cash runway will be sufficient to fund the Company’s operations
through the filing of a Biologics License Application (BLA) for
efzofitimod in pulmonary sarcoidosis.
- R&D
Expenses: Research and development expenses were $10.3
million for the third quarter of 2023, which consisted primarily of
clinical trial costs for the Phase 3 EFZO-FIT™ and Phase 2
EFZO-CONNECT™ studies, manufacturing costs for the efzofitimod
program and research and development costs for the efzofitimod and
discovery programs.
- G&A
Expenses: General and administrative expenses were $2.6
million for the third quarter of 2023.
-
Collaboration and License Revenue: Collaboration
and license revenue related to the Kyorin Agreement was $0.4
million for the third quarter of 2023, which consisted of drug
product material sold to Kyorin for the Japan portion of the
EFZO-FIT™ study.
About Efzofitimod
Efzofitimod is a first-in-class biologic
immunomodulator in clinical development for the treatment of
interstitial lung disease (ILD), a group of immune-mediated
disorders that can cause inflammation and fibrosis, or scarring, of
the lungs. Efzofitimod is a tRNA synthetase derived therapy that
selectively modulates activated myeloid cells through neuropilin-2
to resolve inflammation without immune suppression and potentially
prevent the progression of fibrosis. aTyr is currently
investigating efzofitimod in the global Phase 3 EFZO-FIT™ study in
patients with pulmonary sarcoidosis, a major form of ILD, and in
the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis
(SSc, or scleroderma)-related ILD. These forms of ILD have limited
therapeutic options and there is a need for safer and more
effective, disease-modifying treatments that improve outcomes.
About aTyr
aTyr is a clinical stage biotechnology company
leveraging evolutionary intelligence to translate tRNA synthetase
biology into new therapies for fibrosis and inflammation. tRNA
synthetases are ancient, essential proteins that have evolved novel
domains that regulate diverse pathways extracellularly in humans.
aTyr’s discovery platform is focused on unlocking hidden
therapeutic intervention points by uncovering signaling pathways
driven by its proprietary library of domains derived from all 20
tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod,
a first-in-class biologic immunomodulator in clinical development
for the treatment of interstitial lung disease, a group of
immune-mediated disorders that can cause inflammation and
progressive fibrosis, or scarring, of the lungs. For more
information, please visit www.atyrpharma.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Forward-looking statements are usually
identified by the use of words such as “believes,” “expects,”
“intends,” “may,” “plans,” “potential,” “will,” “project,” 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 include statements
regarding our belief that we will have sufficient cash runway to
fund the Company’s operations through the filing of a BLA for
efzofitimod for pulmonary sarcoidosis; the expected size of, and
number and nationality of patients to be enrolled in, the EFZO-FIT™
and EFZO-CONNECT™ studies; the potential therapeutic benefits and
applications of efzofitimod; and timelines and plans with respect
to certain development activities and development goals, including
our expectation that our Phase 3 EFZO-FIT™ study of efzofitimod in
patients with pulmonary sarcoidosis will complete enrollment early
in the second quarter of 2024. These forward-looking statements
also 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 these
forward-looking statements, are reasonable, we can give no
assurance that the plans, intentions, expectations or strategies
will be attained or achieved. All forward-looking statements are
based on estimates and assumptions by our management that, although
we believe to be reasonable, are inherently uncertain. Furthermore,
actual results may differ materially from those described in these
forward-looking statements and will be affected by a variety of
risks and factors that are beyond our control including, without
limitation, our assumptions and expectations underlying our belief
that we will have sufficient cash runway to fund the Company’s
operations through the filing of a BLA for efzofitimod for
pulmonary sarcoidosis may not be accurate, the fact that NRP2 and
tRNA synthetase biology is not fully understood, uncertainty
regarding the ultimate long-term impact of evolving macroeconomic
and geopolitical conditions, the risk of delays in our clinical
trials, risks associated with the discovery, development and
regulation of our product candidates, including the risk that
results from clinical trials or other studies may not support
further development, the risk that we may cease or delay
preclinical or clinical development activities for any of our
existing or future product candidates for a variety of reasons, the
fact that our collaboration agreements are subject to early
termination, and the risk that we may not be able to raise the
additional funding required for our business and product
development plans, as well as those risks set forth in our most
recent Annual Report on Form 10-K, Quarterly Reports on form 10-Q
and in our other 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. |
|
Consolidated Statements of Operations |
|
(in thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
Nine Months Ended |
|
|
|
September 30, |
|
|
September 30, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
(unaudited) |
|
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
|
License and collaboration agreement revenues |
|
$ |
353 |
|
|
$ |
— |
|
|
$ |
353 |
|
|
$ |
— |
|
Total revenues |
|
|
353 |
|
|
|
— |
|
|
|
353 |
|
|
|
— |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
10,319 |
|
|
$ |
9,867 |
|
|
$ |
29,538 |
|
|
$ |
27,898 |
|
General and administrative |
|
|
2,649 |
|
|
|
3,625 |
|
|
|
9,775 |
|
|
|
10,556 |
|
Total operating expenses |
|
|
12,968 |
|
|
|
13,492 |
|
|
|
39,313 |
|
|
|
38,454 |
|
Loss from operations |
|
|
(12,615 |
) |
|
|
(13,492 |
) |
|
|
(38,960 |
) |
|
|
(38,454 |
) |
Total other income (expense), net |
|
|
1,273 |
|
|
|
247 |
|
|
|
3,324 |
|
|
|
634 |
|
Consolidated net loss |
|
|
(11,342 |
) |
|
|
(13,245 |
) |
|
|
(35,636 |
) |
|
|
(37,820 |
) |
Net loss attributable to
noncontrolling interest in Pangu BioPharma Limited |
|
|
2 |
|
|
|
1 |
|
|
|
7 |
|
|
|
3 |
|
Net loss attributable to aTyr
Pharma, Inc. |
|
$ |
(11,340 |
) |
|
$ |
(13,244 |
) |
|
$ |
(35,629 |
) |
|
$ |
(37,817 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.20 |
) |
|
$ |
(0.46 |
) |
|
$ |
(0.69 |
) |
|
$ |
(1.34 |
) |
Shares used in computing net
loss per share, basic and diluted |
|
|
57,885,393 |
|
|
|
28,663,047 |
|
|
|
51,700,864 |
|
|
|
28,184,698 |
|
ATYR PHARMA INC. |
|
Condensed Consolidated Balance Sheets |
|
(in thousands) |
|
|
|
|
|
|
|
|
|
|
September 30, |
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
|
|
(unaudited) |
|
|
|
|
Cash, cash equivalents, restricted cash and available-for-sale
investments |
|
$ |
105,582 |
|
|
$ |
69,311 |
|
Other receivables |
|
|
2,087 |
|
|
|
11,775 |
|
Property and equipment,
net |
|
|
5,644 |
|
|
|
3,059 |
|
Operating lease, right-of-use
assets |
|
|
6,812 |
|
|
|
7,250 |
|
Financing lease, right-of-use
assets |
|
|
1,791 |
|
|
|
1,248 |
|
Prepaid expenses and other
assets |
|
|
3,153 |
|
|
|
3,143 |
|
Total assets |
|
$ |
125,069 |
|
|
$ |
95,786 |
|
|
|
|
|
|
|
|
Accounts payable, accrued
expenses and other liabilities |
|
$ |
11,648 |
|
|
$ |
12,968 |
|
Current portion of operating
lease liability |
|
|
774 |
|
|
|
630 |
|
Current portion of financing
lease liability |
|
|
459 |
|
|
|
264 |
|
Long-term operating lease
liability, net of current portion |
|
|
12,548 |
|
|
|
9,633 |
|
Long-term financing lease
liability, net of current portion |
|
|
1,437 |
|
|
|
1,007 |
|
Total stockholders’
equity |
|
|
98,203 |
|
|
|
71,284 |
|
Total liabilities and stockholders’ equity |
|
$ |
125,069 |
|
|
$ |
95,786 |
|
Contact:Ashlee DunstonDirector, Investor
Relations and Public Affairsadunston@atyrpharma.com
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