Why Is Dyne Therapeutics Stock Soaring On Monday?
Why Is Dyne Therapeutics Stock Soaring On Monday?
DYNE-101 demonstrated an improvement in myotonia as measured by video hand opening time (vHOT) in all reported cohorts. Monday, Dyne Therapeutics Inc (NASDAQ:DYN) released clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
周一，达因疗法公司（纳斯达克股票代码：DYN）公布了其正在进行的针对1型肌强直性营养不良症（DM1）患者的 DYNE-101 1/2期ACHIEVE试验，以及正在进行的针对愿意跳过外显子51的杜兴氏肌肉萎缩症（DMD）患者的 DYNE-251 1/2期DELIVER试验的临床数据。
Earlier this year, Dyne Therapeutics released initial clinical data from both trials.
今年早些时候，达因疗法发布了这两项试验的初步临床数据。
In the ACHIEVE trial, patients in the 5.4 mg/kg Q8W cohort had a 27% mean splicing correction from baseline across a broad, 22-gene panel at 3 months, with all participants demonstrating splicing correction.
在ACHIEVE试验中，5.4 mg/kg Q8W队列的患者在3个月时在广泛的22个基因组中平均剪接校正率为基线的27％，所有参与者均表现出剪接校正。
DYNE-101 demonstrated an improvement in myotonia as measured by video hand opening time (vHOT) in all reported cohorts.
根据所有报告的队列的视频手部开启时间（VHOT）来衡量，DYNE-101 显示出肌张力有所改善。
The 1.8 mg/kg Q4W group had a mean 3.1-second benefit in myotonia at 3 months, increasing to 4.4 seconds at 12 months.
1.8 mg/kg Q4W 组在 3 个月时肌张力受益平均为 3.1 秒，在 12 个月时增加到 4.4 秒。
In addition, the 5.4 mg/kg Q8W cohort had a mean 4.5-second improvement in myotonia at 3 months.
此外，5.4 mg/kg Q8W队列在3个月时肌张力平均改善了4.5秒。
DYNE-101 demonstrated an improvement in muscle strength and early and sustained potential benefit in the 10-meter Walk/Run Test and 5-times to Stand Test.
在 10 米步行/跑步测试和 5 次站立测试中，DYNE-101 显示肌肉力量有所改善，并有早期和持续的潜在益处。
Dyne reported efficacy data from 8 male patients with DMD amenable to exon 51 skipping enrolled in the 10 mg/kg (approximate PMO dose) cohort of the randomized, placebo-controlled MAD portion of the DYNE-251 DELIVER trial.
达因报告了8名跳过外显子51的男性患者的疗效数据，这些患者加入了 DYNE-251 DELIVER 试验随机、安慰剂对照的 MAD 部分 10 mg/kg（大约 PMO 剂量）队列中。
10 mg/kg of DYNE-251 Q4W demonstrated dose-dependent exon skipping and dystrophin expression.
10 mg/kg 的 DYNE-251 Q4W 表现出剂量依赖性外显子跳跃和肌营养不良蛋白表达。
DYNE-251 reached levels of dystrophin expression that exceeded levels reported in a clinical trial for the current weekly standard of care for DMD exon 51, eteplirsen, at 6 months1 with a 12-fold lower PMO dose.
DYNE-251 在 6 个月时达到的肌萎缩蛋白表达水平超过了当前 DMD 外显子 51 每周护理标准 eteplirsen 的临床试验中报告的水平，PMO 剂量降低了 12 倍。
DYNE-251 also demonstrated encouraging trends in multiple functional endpoints.
DYNE-251 在多功能终端方面也显示出令人鼓舞的趋势。
Patients treated with 10 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin level of 3.22% of normal and a 2.97% change (unadjusted for muscle content) from baseline at 6 months.
接受10 mg/kg DYNE-251 Q4W治疗的患者的平均绝对肌萎缩素水平为正常水平的3.22％，与基线相比在6个月时变化2.97％（未调整肌肉含量）。
Eteplirsen reached a mean absolute unadjusted dystrophin level of 0.30% of normal and a 0.06% change from baseline at 6 months.
Eteplirsen在6个月时达到了未经调整的平均绝对肌营养素水平为正常值的0.30％，与基线相比变化了0.06％。
When adjusting for muscle content, the DYNE-251 treated group reached 7.64% mean absolute dystrophin, greater than levels reported by peptide conjugate PMOs in clinical development.
调整肌肉含量时，DYNE-251 治疗组的平均绝对肌营养不良蛋白达到了 7.64%，高于肽偶联物 pMO 在临床开发中报告的水平。
Trends in improvement were observed in multiple functional endpoints in the 10 mg/kg DYNE-251 Q4W group at 6 months.
在 10 mg/kg DYNE-251 Q4W 组中，在 6 个月时观察到多个功能终点的改善趋势。
Price Action: DYN shares are up 27.80% at $35.36 at the last check Monday.
价格走势：周一最后一次检查时，DYN股价上涨27.80％，至35.36美元。
译文内容由第三方软件翻译。