On November 10, 2022 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported third quarter 2022 results and provided a corporate update (Press release, aTyr Pharma, NOV 10, 2022, View Source [SID1234623874]).
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"The third quarter saw the initiation of EFZO-FIT, a global pivotal Phase 3 study of our lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, the most prevalent form of interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Given current market conditions, we intend to focus our resources on the EFZO-FIT study, which is our largest value driver, to ensure a timely and successful completion of this study."
"As part of this prioritization, we have made the strategic decision not to use internal resources to initiate a Phase 1 study of ATYR2810 this year. The data we have generated for ATYR2810 firmly support its therapeutic potential in rare aggressive tumors, thus we intend to pursue alternative non-dilutive funding avenues, including academic collaborations, to advance this program."
Third Quarter 2022 and Subsequent Period Highlights
Dosed the first patient in EFZO-FIT, a global pivotal Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a 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 once a month for a total of 12 doses. The study is currently enrolling and intends to enroll up to 264 subjects with pulmonary sarcoidosis at multiple centers in the U.S., Europe and Japan.
Announced the publication of results of the Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis in the peer-reviewed medical journal CHEST. The study demonstrated that efzofitimod was safe and well-tolerated at all doses and exhibited a consistent dose response on key efficacy endpoints and improvements compared to placebo, including measures of steroid reduction, lung function, sarcoidosis symptom measures and inflammatory biomarkers.
Received U.S. Food and Drug Administration (FDA) Fast Track designation for efzofitimod for the treatment of systemic sclerosis (SSc, or scleroderma)-associated ILD. Fast Track designation helps facilitate development and expedite the review of drugs to treat serious or life-threatening diseases with unmet medical need. Fast Track designation provides certain benefits, including enhanced interactions with the FDA throughout the development program, as well as eligibility for accelerated approval, priority review and rolling review.
Presented a poster at the European Respiratory Society International Congress 2022 on findings for an antibody for immunohistochemical detection of neuropilin-2 (NRP2) protein, efzofitimod’s binding partner, in patient tissue samples. The antibody may provide a useful tool for patient selection or stratification for sarcoidosis, other ILD, oncology or other indications where NRP2 is implicated.
Completed IND-enabling activities and received a notice of allowance for a patent for ATYR2810, a fully humanized monoclonal antibody targeting NRP2, in preclinical development for cancer.
Announced a research collaboration with Dualsystems Biotech AG, a company specializing in custom proteomics, aimed at accelerating drug discovery and generating new therapeutics based on aTyr’s extensive intellectual property portfolio. Under the collaboration, which is exclusive with respect to tRNA related molecules, Dualsystems will utilize their proprietary receptor screening technology and research expertise to attempt to identify and validate 10 new target receptors for tRNA synthetases by 2025. aTyr previously worked with Dualsystems to identify fibroblast growth factor 4 (FGFR4) as the target receptor for a fragment of alanyl-tRNA synthetase (AARS).
Third Quarter 2022 Financial Highlights and Cash Position
Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of September 30, 2022, were $79.6 million.
R&D Expenses: Research and development expenses were $9.9 million for the third quarter of 2022, which consisted of product development and manufacturing costs for the efzofitimod and ATYR2810 programs, as well as startup costs for the Phase 3 EFZO-FIT study.
G&A Expenses: General and administrative expenses were $3.6 million for the third quarter of 2022.
Shares Outstanding: Common shares outstanding were 29,009,382 as of September 30, 2022.
Conference Call and Webcast Details
aTyr will host a conference call and webcast today at 5:00 p.m. EST / 2:00 p.m. PST to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: View Source An audio replay will be available for at least 90 days following the event.
About Efzofitimod
aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates innate and adaptive immune response in inflammatory disease states. aTyr’s lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr is currently conducting EFZO-FIT, a Phase 3 study of efzofitimod in pulmonary sarcoidosis event.