On March 9, 2023 aTyr Pharma, Inc. (Nasdaq: LIFE) ("aTyr" or the "Company"), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported fourth quarter and full year 2022 results and provided a corporate update (Press release, aTyr Pharma, MAR 9, 2023, View Source [SID1234628467]).
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"2022 was an important year for aTyr as we advanced our lead therapeutic candidate, efzofitimod, to a global pivotal Phase 3 study 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. "This study, which is known as EFZO-FIT, is expected to be the largest interventional study in sarcoidosis to date and is currently enrolling patients at multiple centers in the U.S., Europe and Japan."
"We are also expanding the clinical development program for our novel immunomodulator efzofitimod with plans to initiate a Phase 2 proof-of-concept study in patients with systemic sclerosis (SSc, or scleroderma)-associated ILD (SSc-ILD) this year, which increases the potential market opportunity in these forms of ILD with high unmet medical need. As a result of program prioritization and proceeds from a recent public offering, we project that we will have sufficient cash to fund both of our efzofitimod clinical trials and the company’s operations into 2026. We are very proud of our progress and look forward to the year ahead as we continue our pursuit to develop a new class of medicines."
Fourth Quarter 2022 and Subsequent Period Highlights
Continued enrollment 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 intends to enroll up to 264 subjects with pulmonary sarcoidosis and is currently enrolling at multiple centers in the U.S., Europe and Japan.
Kyorin Pharmaceutical Co., Ltd. (Kyorin), aTyr’s partner for the development and commercialization of efzofitimod for ILD in Japan, dosed the first patient in Japan in EFZO-FIT. This achievement triggered a $10.0 million milestone payment to aTyr in the fourth quarter. Under the collaboration and license agreement with Kyorin, aTyr has received $20.0 million in upfront and milestone payments to date and is eligible to receive up to an additional $155.0 million in the aggregate upon the achievement of certain development, regulatory and sales milestones, as well as tiered royalties on any net sales in Japan. Kyorin has the exclusive rights to develop and commercialize efzofitimod in Japan for all forms of ILD.
Announced U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for a Phase 2 randomized, double-blind, placebo-controlled, proof-of-concept study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This is expected to be a 28-week study with 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. It is expected that the study will enroll 25 patients with progressive disease who are currently receiving background mycophenolate therapy at multiple centers in the U.S. The primary objective of the study will be to evaluate the efficacy of multiple doses of intravenous efzofitimod on pulmonary, cutaneous and systemic manifestations in patients with SSc-ILD. The Company plans to initiate this study in 2023.
Received European Union (EU) orphan drug designation for efzofitimod for the treatment of sarcoidosis from the European Commission based on the opinion of the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for EMA orphan status, a product must, among other things, be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating for which either no satisfactory method of diagnosis, prevention or treatment exists, or if such method exists, the medicine is of significant benefit to those affected by such condition. EMA orphan drug designation provides certain benefits, including the potential for 10 years of market exclusivity following market authorization, potential reduction in regulatory fees and a centralized EU market authorization process.
Announced that the Company will present data on the mechanism of action and an exposure-efficacy analysis for efzofitimod at the upcoming American Thoracic Society (ATS) 2023 International Conference, which is scheduled to take place May 19 – 24 in Washington, DC.
Received a Notice of Allowance from the European Patent Office for a patent covering the use of efzofitimod in combination with the anti-fibrotic pirfenidone for the treatment of lung inflammation or fibrosis.
Announced latent transforming growth factor beta binding protein 1 (LTBP1) as the target of a domain of aspartyl-tRNA synthetase (DARS). LTBP1 is a key regulator of transforming growth factor beta (TGF-β), a central player in the pathogenesis of fibrotic diseases. The Company expects to present additional findings around the interaction between LTBP1 and this domain of DARS at an upcoming scientific conference.
Raised gross proceeds of approximately $52.0 million through the issuance of 23,125,000 shares of common stock in February 2023 from a public offering, which included the partial exercise of the underwriters’ option to purchase additional shares.
Year Ended 2022 Financial Highlights and Cash Position
Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of December 31, 2022, were $69.3 million. Subsequent to the end of the year, the Company received a $10.0 million milestone payment in connection with the Kyorin Agreement and generated gross proceeds of approximately $52.0 million from the public offering of common stock. Based on the Company’s current operational plans and existing cash, aTyr believes the Company’s cash runway will extend into 2026.
R&D Expenses: Research and development expenses were $42.8 million for the year ended 2022, which consisted of clinical trial costs for the Phase 3 EFZO-FIT study, manufacturing costs for the efzofitimod and ATYR2810 programs, and research and development for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $14.0 million for the year ended 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 immune responses 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 patients.