Pulmonary sarcoidosis treatment efzofitimod Phase 3 trial to go on
Independent review board recommends trial continue without modifications
A global Phase 3 clinical trial evaluating efzofitimod, Atyr Pharma’s investigational therapy, in people with pulmonary sarcoidosis will continue as planned following a third pre-planned interim safety review.
An independent data safety and monitoring board (DSMB) examined available safety and tolerability data from all 268 participants enrolled in the trial, dubbed EFZO-FIT (NCT05415137), and recommended its continuation without modifications. DSMBs consist of independent specialists who routinely conduct interim analyses on clinical trials to ensure patient safety and data integrity and provide guidance on modifications, if necessary.
“We are pleased to report yet another positive safety review for efzofitimod, which includes all 268 patients that have been enrolled in our global pivotal Phase 3 EFZO-FIT study,” Sanjay S. Shukla, MD, president and CEO of Atyr, said in a company press release.
Sarcoidosis is an inflammatory disease marked by the formation of granulomas, or clumps of inflammatory cells, in tissues and organs. In pulmonary sarcoidosis, these granulomas form in the lungs, leading to inflammation and tissue damage that drives scarring, making breathing difficult.
Corticosteroids, which have the anti-inflammatory and immunosuppressive properties, are a standard treatment for pulmonary sarcoidosis, but their long-term use is associated with serious side effects.
Pulmonary sarcoidosis treatment
Administered directly into the bloodstream, efzofitimod (formerly ATYR1923) is a first-in-class immunomodulating therapy. It’s designed to modulate the activity of NRP2, a protein on the surface of certain immune cells implicated in granuloma formation and inflammation.
Efzofitimod’s goal is to reduce inflammation without entirely suppressing immune responses, thereby improving lung function and lessening symptoms related to pulmonary sarcoidosis.
In a previous Phase 1b/2a trial (NCT03824392), three doses of the therapy (1, 3, and 5 mg/kg) were tested against a placebo, when administered once a month for six or more months. Participants were also on daily oral corticosteroids, which were tapered in the study’s first two months.
Data showed that efzofitimod treatment eased symptoms, improved lung function, and lowered the need for corticosteroids. A follow-up analysis indicated that the two highest doses of efzofitimod (3 and 5 mg/kg) significantly reduced relapse rates or the need to increase corticosteroids.
The ongoing Phase 3 EFZO-FIT study is evaluating the one-year safety and efficacy of these efzofitimod doses (3 and 5 mg/kg) versus a placebo in 268 adults with pulmonary sarcoidosis, ages 18-75. Patients will continue corticosteroid treatment with a forced taper during the trial.
The study, conducted at 97 sites in the U.S., the U.K., Europe, Brazil, and Japan, began dosing patients in 2022 and completed enrollment in mid-2023.
Its primary objective is to determine whether the therapy can reduce corticosteroid use. Its secondary goals are changes in lung function and sarcoidosis symptoms. The trial, which passed a similar pre-planned safety review in late 2023, is expected to wrap up in 2025.
“Safety is paramount when looking to provide a disease modifying treatment for a chronic condition such as pulmonary sarcoidosis, where reducing or replacing a toxic standard of care such as oral corticosteroids could be highly meaningful and improve quality of life for patients,” Shukla said.
Efzofitimod has received fast track designation in the U.S. for pulmonary sarcoidosis and orphan drug status in the U.S., the European Union, and Japan for sarcoidosis. These designations are intended to speed a therapy’s development.
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