Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst
Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst
Danon disease is caused by mutations in the LAMP2 gene, which contains instructions for the production of an enzyme called lysosomal-associated membrane protein-2 (LAMP-2). The data showed that RP-A501 was generally well tolerated.On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.
在週一，rocket pharmaceuticals公司（納斯達克：RCKT）公佈了在患有Danon病的男性患者中RP-A501的長期安全性和有效性研究結果。
Danon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability.
Danon病是一種罕見的X連鎖顯性遺傳疾病，其臨床特點爲心肌病（心臟肌肉僵硬）、骨骼肌病（無力）和智力障礙。
Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides
另請閱讀：FDA拒絕了rocket pharmaceuticals的罕見免疫疾病基因療法，要求提供更多數據，股票下跌。
Danon disease is caused by mutations in the LAMP2 gene, which contains instructions for the production of an enzyme called lysosomal-associated membrane protein-2 (LAMP-2). The data showed that RP-A501 was generally well tolerated.
Danon病是由LAMP2基因的突變引起的，該基因包含生產一種叫做溶酶體相關膜蛋白-2（LAMP-2）的酶的指令。數據表明，RP-A501通常耐受良好。
All evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of left ventricular (LV) mass index by ≥10% at 12 months (sustained up to 54 months) after treatment.
所有可評估的Danon病患者在治療後12個月表現出LAMP2蛋白表達（持續至60個月），並在12個月時減少左心室（LV）質量指數≥10%（持續至54個月）。
These data were presented at the American Heart Association (AHA) Scientific Sessions 2024.
這些數據在美國心臟協會（AHA）2024科學會議上展示。
Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months.
在隨訪24個月的兒童患者和隨訪60個月的成人/青少年患者中觀察到持續的臨床顯著改善證據。
All evaluable patients in the Phase 1 trial demonstrated:
在第一階段試驗中，所有可評估的患者表現出：
- Cardiac LAMP2 protein expression at 12 months and thereafter.
- Reduction or stabilization of Left ventricular mass index (LV mass index)– the median reduction from baseline to the most recent visit of 24%.
- Preservation of normal LV ejection fraction (LVEF).
- Reduction or stabilization of cardiac biomarkers (median cTnI and NTproBNP reductions of 84% and 57%, respectively).
- The study also showed the absence of clinical signs of heart failure in patients treated with RP-A501.
- 心臟LAMP2蛋白在12個月及之後的表達。
- 左心室質量指數（LV質量指數）的減少或穩定——從基線到最近一次訪問的中位數減少爲24%。
- 保持正常的左心室射血分數（LVEF）。
- 心臟生物標誌物的減少或穩定（中位數cTnI和NTproBNP的減少分別爲84%和57%）。
- 研究還表明接受RP-A501治療的患者沒有心力衰竭的臨床跡象。
William Blair views the long-term follow-up favorably and says it supports RP-A501's durable efficacy.
William Blair對長期隨訪持積極態度，並表示這支持RP-A501的持久療效。
Overall, the updated data from the Phase I study of RP-A501 de-risks the pivotal trial, as significant improvements in both co-primary endpoints were seen across all six treated patients.
總體而言，RP-A501一期研究的更新數據降低了關鍵試驗的風險，因爲在六名接受治療的患者中均觀察到了兩個主要終點的顯著改善。
The analyst looks forward to additional updates on the pivotal study and the company's prelaunch preparations in 2025.
分析師期待關於關鍵研究和公司在2025年預發佈準備的更多更新。
"Although we continue to see RP-A501 as the main stock driver in the near term given its large total addressable market, we believe the approval of RP-L201 in leukocyte adhesion deficiency type I (LAD-I) and the completion of the rolling BLA for RP-L102 in Fanconi anemia (FA) could be incremental catalysts for Rocket in 2025," William Blair writes.
「儘管我們認爲RP-A501在近期仍將是主要的股票驅動力，因爲其巨大潛在市場，但我們相信，RP-L201在白細胞黏附缺陷類型I (LAD-I) 的批准以及RP-L102在範可尼貧血 (FA) 的滾動BLA的完成可能會成爲rocket pharmaceuticals在2025年的增量催化劑，」William Blair寫道。
Price Action: RCKT stock is down 3.44% at $13.19 at the last check on Tuesday.
價格動態：截至週二最後一次檢查，RCKt股票下跌3.44%，報13.19美元。
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譯文內容由第三人軟體翻譯。
