- Topline data remains on track for second quarter of 2024 - MIAMI, Jan. 02, 2024 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today announced that all patients have been enrolled in the Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy (“SBMA”), also known as Kennedy's Disease.

-2024年第二季度的Topline数据仍在正常运行中-迈阿密，2024年1月2日（环球通讯社）-Avenue Therapeutics，Inc.（纳斯达克股票代码：ATXI）（“Avenue”或“公司”），一家专注于开发和商业化治疗神经系统疾病的专业制药公司，今天宣布，所有患者均已参加AJ201治疗脊髓和延髓性肌萎缩症（SBMA）（也称为肯尼迪病）的1b/2a期临床试验。

AJ201 is currently the lead drug candidate in the clinic for SBMA, and topline data are anticipated in the second quarter of 2024. “We are pleased with the speed of enrollment in our Phase 1b/2a study evaluating AJ201 for the treatment of SBMA, reflecting the large unmet need in this debilitating neurodegenerative disease,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue.

AJ201目前是SBMA诊所的主要候选药物，预计将在2024年第二季度提供topline数据。Avenue首席执行官亚历山德拉·麦克莱恩（AlexandraMacLean）医学博士说：“我们对评估AJ201治疗SBMA的1b/2a期研究的入学速度感到满意，这反映了这种衰弱的神经退行性疾病的巨大需求尚未得到满足。”。

“We are intently focused on advancing this much needed drug for patients and their caregivers, and we look forward to reporting topline data in the second quarter of 2024.” The 12-week, multicenter, randomized, double-blind Phase 1b/2a clinical trial of AJ201 enrolled 25 patients, randomly assigned to AJ201 (600 mg/day) or placebo.

“我们专注于为患者及其护理人员推进这种急需的药物，我们期待着在2024年第二季度报告topline数据。”AJ201的12周，多中心，随机，双盲1b/2a期临床试验招募了25名患者，随机分配到AJ201（600毫克/天）或安慰剂。

The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA. Although the disease is slow in progression, limiting the potential to demonstrate clinical efficacy over 12 weeks, Avenue has included as secondary endpoints various biomarkers addressing proof of target engagement, which could reflect the likelihood of clinical success.

该研究的主要终点是评估AJ201在临床和遗传定义的SBMA受试者中的安全性和耐受性。尽管该疾病进展缓慢，限制了在12周内证明临床疗效的潜力，但Avenue已将各种生物标志物作为次要终点，以解决靶标参与的证据，这可能反映了临床成功的可能性。

These biomarkers include the pharmacodynamic data measuring change from baseline in mutant androgen receptor protein levels in skeletal muscle and changes i.

这些生物标志物包括测量骨骼肌突变型雄激素受体蛋白水平与基线变化的药效学数据和i的变化。