Transitioning to new treatments is focus of March 25 virtual town hall

FSR and aTyr Pharma will host panel of sarcoidosis experts and advocates

Steve Bryson, PhD avatar

by Steve Bryson, PhD |

Share this article:

Share article via email
An announcement illustration of person with a megaphone.

In advance of Sarcoidosis Awareness Month in April, the Foundation for Sarcoidosis Research (FSR) and aTyr Pharma will hold a virtual town hall about sarcoidosis and considerations for transitioning from standard corticosteroids to new therapies.

The meeting, which is open to anyone, will be held online at 12 p.m. EDT on March 25. Those interested in attending can register for free on the event page and are invited to complete an online survey that will help shape the discussion. For those unable to attend, a replay will be available on FSR’s YouTube channel.

“We look forward to this discussion to help educate patients on evaluating sarcoidosis treatments and considerations for transitioning to a new therapy,” Sanjay S. Shukla, MD, aTyr’s president and CEO, said in a company press release.

Recommended Reading
Two people sitting at a table and taking part in a discussion.

Earlier moves to specialist, better therapies welcome, patients say

Town hall includes panel of experts in treatment and advocacy

The town hall will include a panel of experts with experience in sarcoidosis treatment and advocacy, including Mary McGowan, FSR’s CEO, and Jim Kuhn, a sarcoidosis patient and FSR’s patient advocate. Shukla, as well as Shambhu Aryal, MD, the medical director of the Inova Lung Transplant Program and Inova Sarcoidosis Center at Inova Fairfax Hospital, will round out the panel.

The moderator will be John Carlin, host of the FSR Sarc Fighter podcast and co-chair of FSR’s patient advisory committee.

“We are pleased to collaborate with aTyr and other members of our esteemed panel for this important event to discuss new and emerging treatment options for sarcoidosis and what to expect when transitioning from steroid treatment to another therapy,” McGowan said.

In sarcoidosis, an overactive immune system triggers the formation of small clumps of inflammatory cells, called granulomas, in tissues and organs. When they form in the lungs, which occurs most often, the disease is referred to as pulmonary sarcoidosis and can lead to scar formation (fibrosis), permanently affecting lung function.

We look forward to this discussion to help educate patients on evaluating sarcoidosis treatments and considerations for transitioning to a new therapy.

Current sarcoidosis treatments include corticosteroids, commonly known as steroids, and other immunosuppressive therapies. However, “while steroids are a treatment option for many people with sarcoidosis, they have limited efficacy and serious side effects when used long term,” Shukla said.

Efzofitimod, aTyr’s non-steroidal investigational therapy, is designed to dampen the immune activity associated with lung inflammation. Infused directly into the bloodstream, it binds to and modulates the activity of NRP2, a protein found at elevated levels in immune cells, including in granulomas.

As such, efzofitimod has the potential to suppress inflammation, prevent lung fibrosis, and lessen the use of corticosteroids and their associated side effects.

Conducted in partnership with the FSR, aTyr sponsored a previous Phase 1b/2a clinical trial (NCT03824392) that tested efzofitimod in 37 adults with pulmonary sarcoidosis.

Top-line data showed the therapy was superior to a placebo at reducing steroid use, improving lung function, easing symptoms, and lowering inflammatory biomarkers. It was also found to be generally safe and well-tolerated, with no reports of treatment-related serious adverse events or unwanted immune activity.

Recommended Reading
An illustration of the word

FSR trains 1st patient volunteers to boost sarcoidosis awareness

Phase 3 EFZO-FIT trial may still be enrolling patients

An aTyr Pharma-sponsored Phase 3 trial, called EFZO-FIT (NCT05415137), is evaluating efzofitimod in up to 264 adults with pulmonary sarcoidosis. With dosing initiated last year, the study may still be enrolling at sites in the U.S., Puerto Rico, Japan, the Netherlands, and Spain.

Participants are randomly assigned to receive monthly infusions of either efzofitimod (3 or 5 mg/kg) or a placebo for about a year. Reducing corticosteroid use is the trial’s primary goal, and secondary measures include changes in lung function and other sarcoidosis-related symptoms.

EFZO-FIT is expected to wrap up in January 2025.

“New non-steroidal treatments that are safe and can help patients maintain adequate symptom control while improving quality of life are on the horizon,” Shukla said. “Efzofitimod is currently in clinical development for pulmonary sarcoidosis and may in the future provide a better alternative to steroid treatment.”

The experimental therapy received orphan drug and fast track designations in the U.S. and orphan drug status in Europe for sarcoidosis. These designations are meant to speed efzofitimod’s clinical development and regulatory review.