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.
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Administered directly into the bloodstream, efzofitimod (formerly ATYR1923) is a firstin-
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.