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基因療法顯示有希望的早期數據，沒有新的安全擔憂，但未能鼓舞投資者
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).
固體生物科學公司(納斯達克：SLDB) 已報告的臨時功能、生物標誌物數據，以及患者報告的結果度量從其正在進行的點火階段1/2試驗其領先的候選基因治療藥物SGT-001在杜氏肌營養不良症(DMD)中得到了廣泛應用。
- 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.
- 雖然IGNITE-DMD旨在評估微營養不良蛋白的表達和分佈，但該公司選擇在12個月時強調功能和患者報告的結果。
- 分析師和投資者對外賣不太確定，導致股價走低。
- 數據將在肌肉營養不良協會的虛擬臨牀和科學會議上公佈。
- Sarepta治療公司的杜興基因療法，它第二階段研究讀數失敗最近，與安慰劑組的0.9分相比，NSAA總分(所有重要的功能終點)提高了1.7分。
- Solid Bio對NSAA評分的改善更小，低劑量組平均為1.0分，高劑量組為0.3分。
- 未經治療的對照組平均下降4.0分。
- 在6分鐘步行測試中，低劑量組的患者平均多步行37米，而高劑量組的患者平均改善49.7米。未經治療的對照組患者下降了8.5米。
- 至於用力肺活量(FVC)，在一年內，未經治療的對照組的絕對值平均下降了10.7%，而同期低劑量組和高劑量組的患者分別平均改善了3.9%和16.7%。
- 生物標記物的數據更是喜憂參半。儘管高劑量組的肌酸激酶(一種損傷標誌)持續下降了約50%，但低劑量組的CK上升了166%，而對照組的CK平均上升了17%，而高劑量組的CK持續下降了約50%，而低劑量組的CK上升了166%，而對照組的CK平均上升了17%。
- 從IGNITE-DMD的第7名患者開始，Solid公司正在測試一種第二代結構，這種結構將把病毒載量減少一半，並在修改後的方案中使用。
- 1/2期DMD研究被臨牀擱置了近三年。2018年3月，FDA在一名患者因血小板和紅細胞水平下降而住院後，實施了臨牀擱置，隨後出現了免疫反應。
- 該機構批准了這項研究於2018年6月恢復，但在一名患者出現與導致首次臨牀擱置的併發症類似的併發症後，該研究於2019年11月再次停止。
- 最後，去年10月，美國食品藥品監督管理局解除了這一禁令。由於該公司同意降低研究中患者的最大體重，這一變化旨在提高安全性。
- 價格行動：在週二的最後一次檢查中，SLDB的股價在盤前交易中下跌10.3%，至8.53美元。
譯文內容由第三人軟體翻譯。
以上內容僅用作資訊或教育之目的，不構成與富途相關的任何投資建議。富途竭力但無法保證上述全部內容的真實性、準確性和原創性。