Prior to the open of U.S. markets today, biotherapeutics company aTyr Pharma Inc. (LIFE:NASDAQ), which is focused on developing medicines that utilize biological pathways to address chronic lung diseases, announced "positive results from its Phase 1b/2a double-blind, placebo-controlled clinical trial of its lead therapeutic candidate, ATYR1923, in 37 patients with pulmonary sarcoidosis, a major form of interstitial lung disease (ILD)."
The firm noted that in the study its ATYR1923 was shown to be well-tolerated at all dosage levels and that no adverse reactions were recorded. aTyr advised further that in the study, ATYR1923 demonstrated a consistent dose response for key efficacy outcomes versus placebo. Patients in the study who were given ATYR1923 showed improvements in steroid reduction, lung function, general sarcoidosis symptoms, and inflammation.
The company's President and CEO, Sanjay S. Shukla, M.D., M.S., commented, "We are delighted by the results of this study, which provide the first clinical proof-of-concept for ATYR1923, as well as validation for our tRNA synthetase biology platform and Neuropilin-2 as a target. The consistency in dose response and clinically meaningful benefit observed, along with ATYR1923's favorable safety and tolerability profile, give us great confidence that ATYR1923 could be a transformative, disease modifying therapy for pulmonary sarcoidosis patients."
"Based on the results of this study, we plan to meet with the U.S. Food and Drug Administration to present these data and our plans for subsequent clinical development and path to registration for ATYR1923 for pulmonary sarcoidosis, and we expect to initiate a registrational trial next year," Dr. Shukla added.
Robert Baughman, M.D., Professor of Medicine and Pulmonologist at the University of Cincinnati Medical Center stated, "I am very impressed by this study, which is one of the best that I have seen conducted in sarcoidosis, a patient population that is highly underserved by current treatment options…The dose response and consistent response seen across multiple efficacy measures, without added toxicity, in this patient population with advanced disease is notable. Importantly, ATYR1923 demonstrated an improvement in several indicators of quality of life, a high priority for patients, by a much larger margin than I would expect in a trial of this size and duration."
The Cleveland Clinic's Chair of the Dept. of Pulmonary Medicine and Director of Diffuse Parenchymal Lung Disease Daniel Culver, D.O., remarked, "The dose response and consistent results across almost every endpoint are a remarkable finding, and as good as could be expected in this small study. The ability to taper patients off steroids while controlling disease symptoms in the ATYR1923 treatment groups is particularly compelling and supports advancement of ATYR1923 into the next phase of development."
The company pointed out that ATYR1923 met its key primary endpoints in the study including a 58% reduction in steroid reduction for the group treated with the highest dosage of 5.0 mg/kg. The firm noted that 33% of the patients in this treatment group were able to taper completely off the use of steroids. In addition, aTyr said that participants in this group demonstrated "clinically meaningful improvements in forced vital capacity (FVC) of 3.3% and all sarcoidosis symptom measures, including shortness of breath, cough, and fatigue."
The company indicated that the Phase 1b/2a ATYR1923 clinical study enrolled 37 individuals diagnosed with pulmonary sarcoidosis. Patients in the randomized, multiple-ascending dose trial were split into three cohorts and were intravenously administered monthly doses of 1.0 mg/kg, 3.0 mg/kg and 5.0 mg/kg of ATYR1923 or placebo for six months.
The firm stated that the trial's main purpose was "to evaluate safety, tolerability, immunogenicity, and pharmacokinetic profile of multiple doses of ATYR1923 compared to placebo." A secondary goal was to study ATYR1923's potential steroid-sparing effects and overall effects on lung function.
The company explained that "pulmonary sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body." The disease effects around 200,000 people in the U.S. The condition ranges from mild symptoms all the way up to chronic and extremely debilitating levels that can cause permanent loss of lung function and death.
The firm listed that it believes that ATYR1923 shows strong promise as a therapeutic treatment for patients with severe inflammatory lung diseases. ATYR1923 was described in the report as "a fusion protein comprised of the immuno-modulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody and is a selective modulator of neuropilin-2 that downregulates innate and adaptive immune response in inflammatory disease states."
HQ'd in San Diego, California, aTyr Pharma is a biotherapeutics company that focuses on developing novel and innovative therapeutics based on novel immunological pathways. The firm's research and development efforts are centered around the extracellular functionality and signaling pathways of tRNA synthetases.
aTyr Pharma started the day with a market cap of around $92.7 million with approximately 16.9 million shares outstanding and a short interest of about 2.5%. LIFE shares opened nearly 15% higher today at $6.30 (+$0.82, +14.96%) over Frida's $5.48 closing price. The stock has traded today between $5.84 and $8.49 per share and is currently trading at $8.13 (+$2.65, +48.36%).
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