Final visit completed in Phase 3 EFZO-FIT clinical trial of efzofitimod
Top-line results are expected in the third quarter of this year
The final participant has completed the last visit as part of EFZO-FIT, a Phase 3 clinical trial testing Atyr Pharma’s experimental treatment efzofitimod, in adults with pulmonary sarcoidosis. Top-line results are expected in the coming months.
“Completing this first-ever global Phase 3 and largest interventional study conducted for patients with sarcoidosis to date is a major accomplishment for aTyr and the broader sarcoidosis community,” Sanjay S. Shukla, MD, president and CEO of Atyr, said in a company press release.
Sarcoidosis is an inflammatory disease where small clusters of immune cells, called granulomas, form in different parts of the body. Pulmonary sarcoidosis, where the disease affects the lungs, leads to symptoms like shortness of breath, coughing, and chest pain.
Oral corticosteroids are the first-line treatment for sarcoidosis, but can cause serious side effects when used in high doses or for long periods. Other treatments can reduce the need for oral corticosteroids, but they also weaken the immune system, increasing the risk of infections.
“Current treatment options for sarcoidosis are limited and rely heavily on oral corticosteroids, which often have significant side effects,” Shukla said. EFZO-FIT “provides an opportunity to evaluate a treatment such as efzofitimod that may have the potential to reduce steroid burden and improve quality of life in patients with this chronic disease.”
How would efzofitimod work in sarcoidosis?
Efzofitimod targets activated myeloid cells, immune cells that help in the inflammatory response, through a protein on their surface called neuropilin-2 (NRP2). By binding to NRP2, which is abundant in granulomas, efzofitimod should reduce inflammation and prevent scarring from getting worse without weakening the immune system.
In a Phase 1b/2a clinical study (NCT03824392), monthly infusions of efzofitimod directly into the bloodstream — at 1, 3, or 5 mg/kg — for 20 weeks (about 4.5 months) helped reduce patients’ use of daily oral corticosteroids while easing symptoms. The two higher doses also reduced relapses, when symptoms worsen suddenly or new ones appear.
The Phase 3 EFZO-FIT study (NCT05415137) is assessing the safety and efficacy of efzofitimod against a placebo in 268 adults with pulmonary sarcoidosis in the U.S., Europe, Japan, and Brazil. Efzofitimod was infused at 3 or 5 mg/kg, every four weeks, for a total of 12 doses.
The main goal of EFZO-FIT is to watch for changes in the mean daily dose of oral corticosteroids from the study’s start to week 48. Secondary goals include changes in the King’s Sarcoidosis Questionnaire score, a measure of how sarcoidosis affects lung-related, health-related quality of life, and forced vital capacity, a measure of lung function. The proportion of participants able to discontinue steroids will also be assessed.
“We thank the patients, investigators, patient advocacy organizations and our partner, Kyorin Pharmaceutical … for helping to make this study possible, and we look forward to sharing top-line results,” Shukla said. Kyorin is aTyr’s partner for developing and commercializing efzofitimod for pulmonary sarcoidosis and certain other lung diseases in Japan.
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