Financial Performance Overview of aTyr Pharma

aTyr Pharma is a clinical-stage biopharmaceutical company developing novel therapeutics based on tRNA synthetase biology. The company’s lead product candidate is efzofitimod, which is being evaluated for the treatment of pulmonary sarcoidosis and systemic sclerosis-associated interstitial lung disease (SSc-ILD).

Regulatory Milestones

• aTyr has received orphan drug and Fast Track designations from regulatory authorities in the U.S. and Europe for efzofitimod in the treatment of pulmonary sarcoidosis and SSc.
• The company’s partner Kyorin Pharmaceutical has also received orphan drug designation for efzofitimod in Japan for the treatment of sarcoidosis.

Clinical Development Progress

• In 2021, aTyr reported positive results from a Phase 1b/2a clinical trial of efzofitimod in pulmonary sarcoidosis, demonstrating safety, tolerability and improvements in key efficacy endpoints.
• Based on these results, aTyr initiated a global Phase 3 pivotal trial (EFZO-FIT) in 2022 to evaluate the efficacy and safety of efzofitimod in pulmonary sarcoidosis. The study is expected to enroll up to 264 patients.
• In 2024, aTyr also initiated a Phase 2 proof-of-concept study (EFZO-CONNECT) to evaluate efzofitimod in patients with SSc-ILD.

Collaboration and Licensing

• In 2020, aTyr entered into a collaboration and licensing agreement with Kyorin Pharmaceutical for the development and commercialization of efzofitimod in Japan.
• Under the agreement, Kyorin has exclusive rights to develop and commercialize efzofitimod in Japan and is responsible for funding all related activities. aTyr is eligible to receive up to $155 million in milestone payments and tiered royalties on net sales in Japan.
• In 2023, Kyorin dosed the first patient in Japan for the EFZO-FIT study, triggering a $10 million milestone payment to aTyr.

Discovery Platform and Pipeline

• Using efzofitimod as a model, aTyr has developed a process to advance novel tRNA synthetase domains from concept to therapeutic candidates.
• The company has identified target receptors for domains of two additional tRNA synthetases and is working to further elucidate their therapeutic potential.
• aTyr’s pipeline includes two additional product candidates:
  • ATYR0101, a fusion protein derived from aspartyl-tRNA synthetase that targets fibrosis
  • ATYR0750, a fusion protein derived from alanyl-tRNA synthetase that targets inflammation and fibrosis

Financial Position and Liquidity

• As of December 31, 2024, aTyr had $75.1 million in cash, cash equivalents, restricted cash and available-for-sale investments.
• The company believes its current cash resources will be sufficient to fund operations for at least one year.
• aTyr has financed its operations primarily through the sale of equity securities, venture debt, and revenue from licensing and collaboration agreements.
• In 2023, the company raised $48.1 million through a public offering and an additional $40.3 million through an at-the-market (ATM) offering program in 2024.
• aTyr expects its cash requirements to increase as it advances efzofitimod and other pipeline candidates through clinical development and prepares for potential commercialization.

Financial Performance Trends

• aTyr has incurred net losses since inception, with an accumulated deficit of $532 million as of December 31, 2024.
• Research and development expenses increased by $12.1 million in 2024 compared to 2023, primarily due to the advancement of the EFZO-FIT study and increased manufacturing efforts for efzofitimod.
• General and administrative expenses increased by $0.8 million in 2024, driven by higher personnel-related costs and professional fees.
• Collaboration revenue from Kyorin decreased from $0.4 million in 2023 to $0.2 million in 2024, as the revenue was primarily from the sale of drug product material.
• Other income, net, decreased from $4.5 million in 2023 to $3.9 million in 2024, primarily due to lower interest income on the company’s cash balances.

Outlook and Future Funding Needs

• aTyr expects its research and development expenses to continue increasing as it advances efzofitimod and other pipeline candidates through clinical development and prepares for potential commercialization.
• The company will need to raise additional capital through equity offerings, collaborations, or other means to fund its operations beyond the next 12 months.
• aTyr’s ability to secure additional funding will depend on various factors, including the progress of its clinical programs, the potential for future collaborations, and market conditions.
• Failure to raise sufficient capital could delay or force the company to scale back its development programs or other operations.

In summary, aTyr Pharma is a clinical-stage biopharmaceutical company making progress in the development of its lead product candidate, efzofitimod, for the treatment of pulmonary sarcoidosis and SSc-ILD. The company has achieved important regulatory milestones and is advancing its pipeline of tRNA synthetase-based therapeutics. While aTyr has a strong cash position, it will need to secure additional funding to support its ongoing and future development activities as it moves closer to potential commercialization.