1st Patient Dosed in Efzofitimod Trial for Pulmonary Sarcoidosis

The main goal of the clinical trial is to reduce the use of corticosteroids

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by Steve Bryson, PhD |

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A Phase 3 clinical trial testing the ability of efzofitimod to safely improve lung function and reduce corticosteroid usage in adults with pulmonary sarcoidosis has dosed its first participant.

The aTyr Pharma-sponsored study, dubbed EFZO-FIT, will take place at multiple centers in North America, Europe, and Japan. Contact and location information is available for those interested in learning more.

“We are delighted to begin patient dosing in EFZO-FIT,” Sanjay Shukla, MD, aTyr’s president and CEO, said in a press release. “With multiple centers in the U.S. open for enrollment, this very important study for patients with pulmonary sarcoidosis is underway.”

“This is an important step forward to delivering a transformative, disease-modifying therapy that we believe can reduce the burden of steroids and provide clinically meaningful outcomes for patients with this complex disease,” Shukla said.

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Sarcoidosis is indicated by the abnormal formation of small clumps of inflammatory cells, called granulomas, in organs and tissues. In about 90% of cases, granulomas form in the lungs, a condition called pulmonary sarcoidosis, which can lead to inflammation and scarring of lung tissue.

Standard prescribed medicine for sarcoidosis include corticosteroids, such as prednisone, which can help control and reduce inflammation. Long-term use, however, can cause harmful side effects.

Efzofitimod (formerly known as ATYR1923) is designed to dampen the immune responses associated with lung inflammation by modulating the activity of NRP2, a protein found at elevated levels in immune cells, including in granulomas, during inflammation. The therapy has the potential to ease lung inflammation, lower corticosteroid doses, and reduce side effects.

In preclinical studies, it slowed the influx of inflammatory immune cells into the lungs of mouse models, potentially easing inflammation.

A Phase 1 clinical trial (ACTRN12617001446358) with healthy adults demonstrated efzofitimod was generally well tolerated at all tested doses. More recently, it safely improved lung function, reduced symptoms, and lowered the need for oral corticosteroids compared to a placebo in a Phase 1/2 study (NCT03824392).

The one-year Phase 3 EFZO-FIT study intends to enroll 264 adults, ages 18–75, with a confirmed diagnosis of pulmonary sarcoidosis for at least six months. Eligible participants must have received stable doses of oral corticosteroids, between 7.5 and 25 mg/day, for at least three months before the study. The trial also includes a forced corticosteroid dose reduction.

Participants will be randomly assigned one of two efzofitimod doses (3 and 5 mg/kg) or a placebo, administered by intravenous (into-the-vein) infusion every four weeks for 48 weeks (about a year).

The study’s main goal is to reduce corticosteroid dosage, with secondary goals including changes in lung function and lung health, as assessed by King’s Sarcoidosis Questionnaire (KSQ)-Lung score.

“We are excited to collaborate with aTyr as they advance this study of a promising new treatment that could potentially improve the lives of sarcoidosis patients worldwide,” Mary McGowan, CEO of the Foundation for Sarcoidosis Research, said.

In the U.S., efzofitimod has been granted orphan drug status, which supports the therapy’s development, and fast-track designation to accelerate its review process for approval.

Recently, aTyr, in collaboration with the University Hospital Bonn, Germany developed a highly sensitive test to detect NRP2. This test may help reveal NRP2’s role in pulmonary sarcoidosis and potentially act as a biomarker to monitor and classify patients.