aTyr’s ATYR1923 for Pulmonary Sarcoidosis Now Named Efzofitimod

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by Vanda Pinto PhD |

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aTyr Pharma has announced that its investigational immunomodulatory therapy for pulmonary sarcoidosis will now be called efzofitimod instead of ATYR1923.

The change comes after the United States Adopted Names (USAN) Council and the World Health Organization’s (WHO) International Nonproprietary Name (INN) expert committee selected efzofitimod as the generic name for the therapy candidate.

These expert panels are responsible for selecting simple, informative, and unique nonproprietary — generic — drug names.

“The assignment of the nonproprietary name efzofitimod for ATYR1923 is an important step as we continue to advance this potentially disease-modifying immunomodulator to its next stage of development,” Sanjay S. Shukla, MD, president and CEO of aTyr, said in a press release.

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The company is planning to launch a clinical trial of its therapy candidate in pulmonary sarcoidosis later this year, with a goal of obtaining enough data to support the filing of a marketing application with regulatory authorities.

“We look forward to initiating a registrational trial of efzofitimod in our lead indication, pulmonary sarcoidosis, this year,” Shukla said.

Sarcoidosis is an inflammatory disease in which small clumps of immune cells, called granulomas, can form in several parts of the body. The condition is designated pulmonary sarcoidosis when granulomas appear in the lungs.

Efzofitimod was developed to bind neuropilin-2 (NRP-2), a protein located at the surface of immune cells that contributes to inflammation and the formation of granulomas in pulmonary sarcoidosis.

Preclinical studies showed that efzofitimod could reduce inflammation-dependent scarring and lower the levels of pro-inflammatory molecules in the lungs, which prompted researchers to conduct a proof-of-concept Phase 1b/2a clinical trial (NCT03824392) in adults with pulmonary sarcoidosis.

The trial, completed in July 2021, assessed the safety, tolerability, and preliminary effectiveness of efzofitimod in 37 patients diagnosed with pulmonary sarcoidosis at least six months before entering the study.

The participants were randomly assigned to receive one of three monthly doses of efzofitimod — 1, 3, or 5 mg/kg — or a placebo, given directly into the bloodstream (intravenously) for six months.

Top-line data revealed that all three doses were generally safe and well-tolerated, with no reports of treatment-associated serious adverse events and no signs of unwanted immune responses.

Regarding efficacy, the highest dose showed the greatest benefits, with patients experiencing clinically meaningful improvements in lung function and general health, along with reduced shortness of breath, cough, and fatigue, compared with those on a placebo.

In addition, the individuals on the highest dose reduced their corticosteroid use by 58% from the study’s start — a 22% greater reduction compared with patients on a placebo.

“aTyr is working to develop a new class of medicines based on extracellular tRNA synthetase biology,” Shukla said. “By restoring immune balance through selective modulation of NRP2, efzofitimod is the first tRNA synthetase-derived and NRP2-targeting therapy to demonstrate clinical activity in patients.”

As ATYR1923, efzofitimod was granted orphan drug designation earlier this year by the U.S. Food and Drug Administration (FDA) for the treatment of sarcoidosis. Orphan drug status provides aTyr with regulatory support and financial benefits to accelerate the therapy’s clinical development, as well as seven-year period of marketing exclusivity if it is approved.