Arcturus' Inhaled MRNA Therapeutic In Cystic Fibrosis Study Highlights Potential Amid Competitive Landscape: Analyst
Arcturus' Inhaled MRNA Therapeutic In Cystic Fibrosis Study Highlights Potential Amid Competitive Landscape: Analyst
Friday, Arcturus Therapeutics Holdings Inc (NASDAQ:ARCT) presented Phase 1 results in healthy volunteers and Phase 1b interim data in people with cystic fibrosis for ARCT-032, an inhaled investigational mRNA therapeutic, at the European Cystic Fibrosis Conference.
納斯達克上市公司Arcturus Therapeutics Holdings Inc (NASDAQ:ARCT) 在歐洲囊性纖維化大會上發佈了Phase 1的健康志願者和囊性纖維化患者Phase 1b的中期結果，針對ARCT-032的吸入式探究性mRNA治療方法。
ARCT-032 administration was generally safe and well tolerated, with no serious or severe adverse events in healthy volunteers and the first four dosed participants with cystic fibrosis.
在健康志願者和四名囊性纖維化的首批接受劑量者中，ARCT-032的施用總體安全無憂，未發生嚴重或嚴重不良反應。
The Phase 1b trial showed improvements in FEV1 (Forced Expiratory Volume in 1 second) in the four adults with cystic fibrosis after two inhaled administrations.
Phase 1b試驗顯示，接受兩次吸入後四名成人囊性纖維化患者的FEV1 (第一秒鐘強制呼氣容積)有所改善。
The absolute change in percent predicted FEV1 averaged +4.0% on Day 8 (5 days after 2nd dose). The relative change in FEV1 averaged +5.8% on Day 8.
第8天（第二次用藥5天后），預測FEV1百分比絕對變化平均爲+4.0％，相對變化平均爲+5.8％。
The observed increases in FEV1 are encouraging and consistent with the previously reported data in the CF ferret model that demonstrated markedly improved mucociliary clearance (MCC) after a single dose of ARCT-032.
FEV1的提高是令人鼓舞的，並與之前在CF雪貂模型中數據的報道相一致，此模型證明使用ARCT-032的單次劑量後，粘液纖毛清除顯着改善。
Of the four participants in Phase 1b to date, one had 2 Class I mutations, and the other three had F508del mutations and were being treated with Vertex Pharmaceuticals Inc's (NASDAQ:VRTX) Trikafta.
在第一階段截至目前的四位參與者中，一位有2種1類突變體，另外三位有F508del突變體且正在接受Vertex Pharmaceuticals公司 (NASDAQ:VRTX) 的Trikafta治療。
No bronchospasm or febrile reactions were observed. Dose-related, mild-to-moderate febrile reactions occurred in some healthy volunteers.
未觀察到支氣管痙攣或發熱反應，健康志願者中有輕度至中度的發熱反應與劑量相關。
William Blair notes that despite important limitations such as the small patient sample size, differences in baseline characteristics, and significant within-patient variability, the overall data is encouraging in two key areas:
William Blair指出，儘管存在重要的限制因素，如患者樣本量較小，基線特徵差異以及嚴重的患者內變異性等，但總體數據在兩個關鍵領域是令人鼓舞的：
- Firstly, in light of Vertex's recent studies on its next-generation Vanza triple treatment, which did not show superior FEV1 improvement compared to Trikafta, William Blair is intrigued by the potential additional benefits observed in three patients with amendable mutations.
- Second, the 4% absolute benefit and over 5.8% relative improvement seen in Class I null mutation patients—who currently lack treatment options—are comparable to the early Phase I results of ivacaftor during its CFTR modulator development.
- 首先，鑑於Vertex最近關於其下一代Vanza三聯治療的研究，並未顯示FEV1的改善優於Trikafta，因此William Blair對在三名可糾正突變體患者中觀察到的潛在附加益處感到好奇。
- 其次，在null1類突變患者中看到的4％絕對益處和超過5.8％相對改善 -- 目前缺乏治療選擇 -- 與CFTR調節劑發展早期的ivacaftor的 Phase I 的結果相當。
The analyst says the results are also promising compared to a ferret model of cystic fibrosis, which accurately mimics key aspects of the disease, such as mucus buildup.
該分析師稱，與囊性纖維化雪貂模型相比，這些結果也很有前途，該模型能準確地模擬疾病的重要方面，如粘液積聚。
William Blair reiterates the Outperform rating on Arcturus, saying it is a leader in self-amplifying mRNA vaccine technology and advancing an mRNA therapeutics platform with a differentiated lipid nanoparticle delivery system.
William Blair重申其對Arcturus的Outperform評級，稱其是自我放大mRNA疫苗技術的領導者，並正在推進具有差異性的脂質納米粒輸送系統的mRNA治療平台。
Price Action: ARCT shares are down 27.9% at $30.72 at last check Friday.
價格行動：截至上週五，ARCT股價下跌27.9％至每股30.72美元。
Photo by Minerva Studio via Shutterstock
照片由 Shutterstock 的Minerva Studio提供。
譯文內容由第三人軟體翻譯。