Solid Bio's DMD Gene Therapy Shows Promising Early Data, No New Safety Concerns, But Fails To Cheer Investors
Solid Bio's DMD Gene Therapy Shows Promising Early Data, No New Safety Concerns, But Fails To Cheer Investors
Solid Bio公司的DMD基因疗法显示有希望的早期数据，没有新的安全担忧，但未能鼓舞投资者
Solid Biosciences Inc (NASDAQ: SLDB) reported interim functional, biomarker data, and patient-reported outcome measures from its ongoing IGNITE Phase 1/2 trial of its lead gene therapy candidate, SGT-001 in Duchenne muscular dystrophy (DMD).
固体生物科学公司(纳斯达克：SLDB) 已报告的临时功能、生物标志物数据，以及患者报告的结果度量从其正在进行的点火阶段1/2试验其领先的候选基因治疗药物SGT-001在杜氏肌营养不良症(DMD)中得到了广泛应用。
- While IGNITE-DMD was designed to assess the microdystrophin protein expression and distribution, the company instead chose to highlight the functional and patient-reported outcomes at the 12-month mark.
- Analysts and investors are less certain about the takeaway, driving the stock price lower.
- Data will be presented at the Muscular Dystrophy Association’s virtual clinical and scientific conference.
- Sarepta Therapeutics Inc’s Duchenne gene therapy, which failed in a Phase 2 study readout, recently sported 1.7 point improvement in NSAA total score (all-important functional endpoint) compared to 0.9 on placebo.
- Solid Bio spurred even smaller improvement on the NSAA score at a mean of 1.0 point in the low dose group and 0.3 points in the high dose group.
- The untreated control cohort exhibited a mean decline of 4.0 points.
- On the 6-minute walk test, patients in the low-dose cohort walked 37 meters more on average, while the high-dose cohort had a mean improvement of 49.7 meters. Patients in the untreated control cohort exhibited a decline of 8.5 meters.
- As for FVC, within a year untreated control cohort exhibited a mean decline of 10.7% on an absolute basis, while patients in the low dose and high dose cohorts exhibited a mean improvement of 3.9% and 16.7%, respectively, over the same period.
- The biomarker data were more mixed. Though the high-dose group saw a sustained decline in creatine kinase (a marker of damage) of around 50%, the low dose cohort saw CK increase 166%, while the control group had an average CK increase of 17%.
- Starting with the 7th patient in IGNITE-DMD, Solid is testing a second-generation construct that would cut the viral load by half and be administered within a modified regimen.
- The Phase 1/2 DMD study was held down by clinical holds for almost three years. In March 2018, the FDA instituted a clinical hold after one patient was hospitalized due to a drop in platelet and red blood cell levels, followed by an immune reaction.
- The agency cleared the study to resume in June 2018, only to halt it again in November 2019 after a patient developed complications similar to those that led to the first clinical hold.
- Finally, in October last year, the FDA lifted the hold as the company agreed to lower the maximum weight of patients in the study, a change intended to boost safety.
- Price Action: SLDB shares are trading 10.3% lower at $8.53 in premarket on the last check Tuesday.
- 虽然IGNITE-DMD旨在评估微营养不良蛋白的表达和分布，但该公司选择在12个月时强调功能和患者报告的结果。
- 分析师和投资者对外卖不太确定，导致股价走低。
- 数据将在肌肉营养不良协会的虚拟临床和科学会议上公布。
- Sarepta治疗公司的杜兴基因疗法，它第二阶段研究读数失败最近，与安慰剂组的0.9分相比，NSAA总分(所有重要的功能终点)提高了1.7分。
- Solid Bio对NSAA评分的改善更小，低剂量组平均为1.0分，高剂量组为0.3分。
- 未经治疗的对照组平均下降4.0分。
- 在6分钟步行测试中，低剂量组的患者平均多步行37米，而高剂量组的患者平均改善49.7米。未经治疗的对照组患者下降了8.5米。
- 至于用力肺活量(FVC)，在一年内，未经治疗的对照组的绝对值平均下降了10.7%，而同期低剂量组和高剂量组的患者分别平均改善了3.9%和16.7%。
- 生物标记物的数据更是喜忧参半。尽管高剂量组的肌酸激酶(一种损伤标志)持续下降了约50%，但低剂量组的CK上升了166%，而对照组的CK平均上升了17%，而高剂量组的CK持续下降了约50%，而低剂量组的CK上升了166%，而对照组的CK平均上升了17%。
- 从IGNITE-DMD的第7名患者开始，Solid公司正在测试一种第二代结构，这种结构将把病毒载量减少一半，并在修改后的方案中使用。
- 1/2期DMD研究被临床搁置了近三年。2018年3月，FDA在一名患者因血小板和红细胞水平下降而住院后，实施了临床搁置，随后出现了免疫反应。
- 该机构批准了这项研究于2018年6月恢复，但在一名患者出现与导致首次临床搁置的并发症类似的并发症后，该研究于2019年11月再次停止。
- 最后，去年10月，美国食品药品监督管理局解除了这一禁令。由于该公司同意降低研究中患者的最大体重，这一变化旨在提高安全性。
- 价格行动：在周二的最后一次检查中，SLDB的股价在盘前交易中下跌10.3%，至8.53美元。
译文内容由第三方软件翻译。
以上内容仅用作资讯或教育之目的，不构成与富途相关的任何投资建议。富途竭力但不能保证上述全部内容的真实性、准确性和原创性。