
Core Insights - The interim analysis of the Phase 2 EFZO-CONNECT™ study indicates that three out of four patients treated with efzofitimod showed clinically significant improvement in the modified Rodnan Skin Score (mRSS) at 12 weeks [1][3][7] - Efzofitimod is generally safe and well tolerated across all doses, with no serious adverse events reported [1][7] Company Overview - aTyr Pharma, Inc. is a clinical stage biotechnology company focused on developing first-in-class medicines from its proprietary tRNA synthetase platform [2][9] - The lead therapeutic candidate, efzofitimod, is being investigated for the treatment of interstitial lung disease (ILD) related to systemic sclerosis (SSc) [8][9] Study Details - The EFZO-CONNECT™ study is a Phase 2 randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with limited or diffuse SSc-ILD [4] - The study involves 28 weeks of treatment with three parallel cohorts randomized to receive either 270 mg or 450 mg of efzofitimod or placebo, with a total of 25 patients expected to be enrolled [4] Key Findings - The interim analysis revealed stable or improved mRSS for all patients, with a notable improvement of 4 points or greater for three out of four efzofitimod-treated patients with diffuse SSc-ILD [7] - Preliminary signals of improvement were observed in inflammatory biomarkers such as interferon gamma (IFN-γ) and monocyte chemoattractant protein-1 (MCP-1) [7] Disease Context - Systemic sclerosis (SSc) is a chronic autoimmune disease that can lead to interstitial lung disease (SSc-ILD), affecting approximately 100,000 people in the U.S., with up to 80% potentially developing ILD [6] - Current treatment options for SSc-ILD are limited and primarily focus on slowing lung function decline, often with significant toxicity [6]