Viridian Therapeutics, Inc. VRDN reported upbeat results for the second quarter on Wednesday.The company posted quarterly losses of $(1.00) per share which beat the analyst consensus estimate of $(1.02) per share. The company reported quarterly sales of $75.000 thousand which beat the analyst consensus estimate of $43.643 thousand.“Veligrotug’s recent Breakthrough Therapy Designation as well as the continued and consistent performance of veligrotug across all of the endpoints and timepoints in our pivotal c ...

Corporate Presentation August 2025 Cautionary note regarding forward-looking statements This presentation contains forward-looking statements. These statements may be identified by the use of words such as, but not limited to, "anticipate," "believe," "become," "continue," "could," "design," "estimate," "expect," "intend," "may," "might," "on track," "plan," "potential," "predict," "project," "should," "target," "will," or "would" or other similar terms or expressions that concern our expectations, plans an ...

现金流与财务状况 - 截至2025年3月31日，公司现金为6.37亿美元，预计资金可支持运营至2027年下半年[12] 临床试验与产品研发 - Veligrotug在THRIVE和THRIVE-2试验中显示出强大的临床效果，所有主要和次要终点均达到统计学显著性[31] - 预计Veligrotug的BLA申请将在2025年下半年提交，欧盟MAA申请将在2026年上半年提交[12] - VRDN-003的REVEAL-1和REVEAL-2试验正在招募患者，预计2026年上半年将公布顶线数据[12] - 预计VRDN-006的健康志愿者数据将在2025年第三季度公布，IND申请计划于2025年年底提交[12] - 预计VRDN-008的IND申请将在2025年年底提交[12] - Veligrotug在15周的主要终点中，70%的患者实现了眼球突出反应，而安慰剂组仅为5%[57] - Veligrotug在15周时的眼球突出平均变化为-2.89毫米，安慰剂组为-0.48毫米，p值均小于0.0001[55] - Veligrotug在15周时，54%的患者实现了双眼复视完全消失，而安慰剂组为12%[55] - Veligrotug在15周的临床活动评分（CAS）为0或1的比例为64%，安慰剂组为18%[55] - Veligrotug在15周的整体反应率为67%，安慰剂组为5%[55] - Veligrotug在52周时，70%的眼球突出反应者维持了反应[68] - Veligrotug的半衰期为40-50天，IGF-1水平增加约4倍[104] - Veligrotug的94%患者完成了治疗课程，显示出良好的耐受性[87] - VRDN-003的药代动力学模型显示，Q4W和Q8W给药方案可实现3-10 mg/kg的veligrotug预期暴露水平[107] - 在二期TED临床试验中，3 mg/kg和10 mg/kg的veligrotug静脉注射显示出强劲的临床活性[108] - REVEAL试验预计在2026年上半年交付顶线结果，以支持2026年底的BLA提交，参与者数量为117[115] 市场潜力与竞争 - 当前TED市场年销售额约为20亿美元，市场潜力巨大[26] - Veligrotug和VRDN-003有潜力成为TED治疗的最佳选择[12] - FcRn抑制剂市场预计到2030年将接近100亿美元，仅MG和CIDP的市场规模[133] 不良事件与安全性 - Veligrotug在15周的治疗相关不良事件发生率为71%，而安慰剂组为24%[64] - Veligrotug在15周的听力损害不良事件的安慰剂调整发生率为5.5%[66] - Veligrotug组中85%的参与者出现任何治疗相关不良事件（TEAE），而安慰剂组为68%[87] 未来展望 - 预计Veligrotug将在2026年成为TED的IV治疗首选[12] - 公司正在推进FcRn抑制剂组合，计划在2026年和2027年进行BLA和MAA提交[17]