― Phase 2b ReDUX4 clinical trial indicates improvements in functional, structural, and patient-reported outcomes; findings informed the design and choice of efficacy endpoints in the ongoing Phase 3 clinical trial― ― Topline data for the Phase 3 REACH clinical trial of losmapimod in FSHD expected during 4Q’24― CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced the publication of results from its Phase 2b clinical trial of losmapimod for the treatment of facioscapulopumeral muscular dystrophy (FSHD).

―2b期ReDUX4临床试验表明功能，结构和患者报告结果有所改善；研究结果为正在进行的第三阶段临床试验的疗效终点的设计和选择提供了依据，该临床阶段的生物制药公司Fulcrum Therapeutics，Inc.®（Nasdaq:FULC）于2024年5月8日在马萨诸塞州剑桥市进行了losmapimod在FSHD中的第三阶段REACH临床试验的Topline数据，该公司专注于改善遗传定义的罕见疾病患者的生活，今天宣布公布了losmapimod治疗面capulopumeral肌营养不良症（FSHD）的2b期临床试验结果。

The data are published in the peer-reviewed journal The Lancet Neurology. FSHD is a serious, rare, and progressively disabling disease characterized by fat infiltration of skeletal muscle leading to muscular atrophy involving primarily the face, shoulder girdle, upper arms, abdomen, and lower limbs.

这些数据发表在同行评议的《柳叶刀神经病学》杂志上。FSHD是一种严重，罕见且逐渐致残的疾病，其特征是骨骼肌脂肪浸润，导致肌肉萎缩，主要涉及面部，肩带，上臂，腹部和下肢。

It is caused by the aberrant expression of the DUX4 protein. It is characterized by accumulating muscle weakness and functional impairment. There are no approved disease-modifying treatments for FSHD. “The publication of these results, which informed the design and choice of efficacy endpoints in our Phase 3 clinical trial, also provide important validation for the therapeutic potential of losmapimod,' said Patrick Horn M.D., Ph.D., Fulcrum’s chief medical officer.

它是由DUX4蛋白的异常表达引起的。它的特征是累积的肌肉无力和功能障碍。FSHD尚无批准的疾病缓解治疗方法。“这些结果的公布为我们的3期临床试验中疗效终点的设计和选择提供了依据，也为losmapimod的治疗潜力提供了重要的验证，”Fulcrum首席医疗官PatrickHorn医学博士说。

'Looking ahead, we remain on track to report topline data for REACH in the fourth quarter of 2024, which will bring us one step closer to addressing the high unmet needs of the FSHD patient community. With an unwavering commitment to our patients, we continue to take important steps toward a potential NDA filing and commercial launch .

“展望未来，我们仍将在2024年第四季度报告REACH的topline数据，这将使我们更接近解决FSHD患者群体的高度未满足需求。凭借对患者的坚定承诺，我们继续朝着潜在的NDA申请和商业启动迈出重要的一步。