Sarcoidosis Advocate Appointed Patient Advisor at aTyr
aTyr Pharma, which is developing a candidate therapy for pulmonary sarcoidosis called ATYR1923, has appointed Andrea Wilson — a sarcoidosis patient and advocate who co-founded the Foundation for Sarcoidosis Research (FSR) — as a patient advisor.
“We are pleased during Sarcoidosis Awareness Month to welcome Andrea, a leading advocate for sarcoidosis, as a patient advisor to aTyr,” said Sanjay S. Shukla, MD, aTyr’s president and CEO, said in a press release.
“Andrea has dedicated the past 20 years to promoting awareness and generating support to accelerate research to find a cure for this debilitating disease,” Shukla said. “Her firsthand knowledge as a patient combined with her longstanding advocacy experience and relationship to the sarcoidosis community will help support patient strategies for our ATYR1923 clinical program in pulmonary sarcoidosis,” he added.
“I know through personal experience the need for new treatment options for patients with sarcoidosis, including alternatives to corticosteroids,” said Wilson, who served previously as president and board member of FSR, the leading international non-profit organization dedicated to finding a cure for sarcoidosis and improving patient care.
“I look forward to supporting aTyr as they continue development of ATYR1923 as a potential new therapeutic for this chronic, debilitating disease,” Wilson said.
Sarcoidosis is an inflammatory disease that can lead to the formation of small abnormal lumps or nodules — called granulomas — in virtually any organ in the body, but most often in the lungs (classified as pulmonary sarcoidosis).
Estimated to affect 150,000 to 200,000 Americans, pulmonary sarcoidosis is part of a larger group of more than 200 chronic lung disorders — known collectively as interstitial lung diseases (ILDs).
ILDs cause inflammation and scarring (fibrosis) in the tissue in and around the lung air sacs, impairing the lungs’ ability to transfer oxygen to the bloodstream.
Current treatments for pulmonary sarcoidosis include corticosteroids and other immunosuppressive therapies, which have limited effectiveness and are linked to serious side effects when used long-term.
As such, there is a high unmet need for more tolerable and effective treatments for this rare condition.
ATYR1923 is a fusion protein that works by selectively binding to and changing the activity of neuropilin-2 (NRP2), a protein that plays a key role in regulating inflammatory responses, including in the lungs.
It is expected to suppress immune responses in uncontrolled inflammatory disease states, such as those found in ILDs, to resolve inflammation and prevent subsequent fibrosis.
Preclinical studies have shown that ATYR1923 reduces the levels of pro-inflammatory and pro-scarring molecules, and significantly lessens inflammation-dependent fibrosis. This led to improvement in lung function in ILD animal models.
A proof-of-concept, fully-enrolled Phase 1b/2a clinical trial (NCT03824392) is evaluating the safety, tolerability, immunogenicity, pharmacokinetics, and preliminary effectiveness of ATYR1923 in 37 adults with pulmonary sarcoidosis.
ATYR1923’s immunogenicity concerns its ability to trigger an unwanted immune response, while pharmacokinetics refers to its movement into, through, and out of the body.
Participants, recruited at 15 sites across the U.S., are assigned randomly to receive into-the-vein (intravenous) infusions of either one of three doses of ATYR1923 (1, 3, or 5 mg/kilogram) or a placebo, once a month for six months.
A pre-planned interim analysis from the first 15 participants suggested the therapy is generally safe and well-tolerated, with no reports of treatment-related serious adverse events. These findings are in line with previous findings from a Phase 1 study (ACTRN12617001446358) conducted in healthy volunteers in Australia.
While full results from the trial, which was conducted in collaboration with FSR, first were expected by September 2020, aTyr announced these data were likely to be delayed due to the COVID-19 pandemic. Data is expected later this year.
The company also tested ATYR1923 in COVID-19 patients with severe respiratory complications in a Phase 2 clinical trial (NCT04412668), which is now complete.
aTyr entered a collaboration and license agreement with Kyorin Pharmaceutical last year to develop and commercialize ATYR1923 in Japan.