Clinical trials can be helpful tools in determining the safety, efficacy, tolerability, recommended dose, and pharmacokinetics of specific drugs (among other things). In doing so, these trials help tell researchers (and the FDA) what can and should become available for patients and what should not. According to Sarcoidosis News, researchers recently evaluated ATYR1923 within a Phase 1b/2a clinical trial. The therapy, designed to treat adult patients with pulmonary sarcoidosis, was shown to be both safe and effective. Additionally, the treatment helped reduce sarcoidosis-associated symptoms, as well as improve lung function. 

ATYR1923

Developed by aTyr Pharma, ATYR1923 is described as:

A potential first-in-class, disease modifying therapy for patients with severe inflammatory lung diseases with high unmet medical need. ATYR1923 works by selectively modulating Neuropilin-2 (NRP2) to downregulate the innate and adaptive immune responses…to resolve inflammation and prevent subsequent fibrosis.

The treatment is administered intravenously. In preclinical studies, ATYR1923 significantly reduces pro-inflammatory and pro-scarring molecule rates, which in turn reduced fibrosis. So within this particular clinical trial, researchers sought to evaluate the therapy’s safety, efficacy, pharmacokinetic profile, and tolerability. Altogether, 37 patients enrolled. During treatment, patients received, 1mg/kg, 3mg/kg, or 5mg/kg of either ATYR1923 or a placebo over a 24-week period. Findings include:

• While 5mg ATYR1923 was the most effective of the three doses, researchers determined that 1mg, 3mg, and 5mg were all safe and relatively well-tolerated.
• 33% of patients who received 5mg ATYR1923 were able to stop using corticosteroids. Additionally, patients within the 5mg cohort were also able to reduce overall corticosteroid use by 58%.
• This high dose group also saw an improvement in forced vital capacity (FVC), which is a key marker of how well the lungs are functioning. 
• Patients who received ATYR1923 over the placebo also experienced lowered inflammatory and sarcoidosis biomarkers, cough, fatigue, and shortness of breath. Ultimately, this highlights how ATYR1923 has the potential to greatly improve quality of life (QOL) for patients with pulmonary sarcoidosis.

In the future, aTyr Pharma will work with the FDA to determine a path towards drug approval and more clinical testing.

Sarcoidosis

Normally, our immune system protects us from “foreign invaders” or potentially harmful substances within our bodies. When the immune system recognizes these foreign invaders, it releases chemicals to destroy them, causing some inflammation. In patients with sarcoidosis, this inflammation remains. More so, the excess immune cells form clumps called granulomas, which can disrupt organ function. Sarcoidosis often originates in the lungs (pulmonary sarcoidosis), lymph nodes, or skin, though it can affect other organs. Symptoms include:

• Depression and anxiety
• Swollen lymph nodes
• Nodules under the skin OR lesions on the skin
• Unintended weight loss
• Fatigue
• Coughing and/or wheezing
• Light sensitivity
• Irregular heartbeat
• Joint pain and swelling
• Shortness of breath and/or difficulty breathing
• Chest pain

Learn more about sarcoidosis.