Most people may never have heard of sarcoidosis, an inflammatory disease that affects about 1.2 million people worldwide.
In the U.S. alone, there are are approximately 200,000 people with sarcoidosis, and nearly 3 times more common in African Americans – and more severe– than the caucasian population. Not to mention, it is twice as common in women than in men.
Characterized by the formation of granulomas — clumps of inflammatory cells — in one or more organs in the body, sarcoidosis affects people of all ages but is most common among people who are 20 to 39 years of age.
While sarcoidosis can occur in any organ, more than 90% of patients with sarcoidosis will have the lungs affected, which is called pulmonary sarcoidosis. Left undiagnosed or untreated, the condition of patients with pulmonary sarcoidosis could degenerate into a chronic, progressive disease. Chronic, unresolved lung inflammation may result in scarring (fibrosis) that permanently damages the lung tissue and can lead to lung failure and death.
Between 1% and 5% of patients with sarcoidosis die from complications, according to reports, so there is a growing need for effective therapies.
This unmet therapy need is fueling the growth of the sector. The only approved treatments for sarcoidosis include the steroid prednisone, which includes inexpensive generics, and Achtar Gel by Mallinckrodt– an expensive treatment rarely used.
The standard of care for patients with pulmonary sarcoidosis involves treatment with corticosteroids and other immunosuppressive therapies. But currently available treatments have limited evidence of effectiveness and can cause serious long-term side effects.
The Need For New Treatment
There is always a need for new treatment options for sarcoidosis patients. Biotherapeutics company, aTyr Pharma Inc. LIFE reports its pipeline drug efzofitimod could be an effective therapy option for pulmonary sarcoidosis.
aTyr says its mission is to translate findings from its tRNA synthetase platform into new therapeutics for fibrosis, inflammation and cancer.
The company focuses on the extracellular functionality and signaling pathways of transfer RNA (tRNA) synthetases. Built on more than a decade of foundational science, aTyr says it has built a global intellectual property estate directed to all 20 human tRNA synthetases and certain associated signaling pathways, with over 300 protein compositions patented.
Efzofitimod is a potential first-in-class, disease-modifying therapy for patients with fibrotic lung disease.
Efzofitimod works by selectively modulating Neuropilin-2 (NRP2) to downregulate the innate and adaptive immune responses in uncontrolled inflammatory disease states in order to resolve inflammation and prevent subsequent fibrosis.
aTyr is initially investigating efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD) which is made up of a group of immune-mediated disorders that can cause progressive lung fibrosis. Sarcoidosis is the most prevalent form of ILD and fibrosis occurs in 1 in 5 cases.
Pivotal Phase 3 EFZO-FIT™ Study
The EFZO-FIT™ study is supported by safety and efficacy data from a Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis. Efzofitimod has been granted U.S. Food and Drug Administration (FDA) Orphan Drug and Fast Track designations for sarcoidosis.
According to aTyr, the EFZO-FIT™ study is a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis.
The 52-week study will consist of three parallel cohorts randomized equally to either 3 mg/kg or 5 mg/kg of efzofitimod or placebo dosed intravenously once per month for 12 doses.
The study intends to enroll 264 subjects with pulmonary sarcoidosis at multiple centers in North America, Europe and Japan. The trial design will incorporate a forced steroid taper.
The study's primary endpoint is steroid reduction, while the secondary endpoints include measures of lung function and sarcoidosis symptoms.
With possibly no real marketed therapies for pulmonary sarcoidosis yet, aTyr could be in a position to be a significant player. There could be an opportunity for efzofitimod in sarcoidosis and other ILD, and aTyr believes it could even be looking at a $2-3 billion market opportunity for efzofitimod in sarcoidosis and other inflammatory forms of ILD including connective tissue disease-associated interstitial lung disease, such as scleroderma-ILD, and chronic hypersensitivity pneumonitis.
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