Goldman Sachs Bullish On Avidity Biosciences, Cites Scalable RNA Therapeutics Platform For Rare Diseases
Goldman Sachs Bullish On Avidity Biosciences, Cites Scalable RNA Therapeutics Platform For Rare Diseases
The company's pipeline includes Del-brax in the Phase 1/2 Fortitude trial for facioscapulohumeral muscular dystrophy (FSHD) and del-zota in a Phase 1/2 Explore44 trial in Duchenne muscular dystrophy (DMD) for patients amenable to exon 44 skipping.Goldman Sachs has initiated coverage on Avidity Biosciences Inc (NASDAQ:RNA) based on multi-blockbuster, first-to-market commercial opportunities for its investigational candidates.
高盛已開始對avidity biosciences進行研究覆蓋，基於多個巨型爆款，首家進入市場的商業機會，針對其調查候選人。
Avidity Biosciences is a clinical-stage biotechnology company that develops RNA therapeutics (antibody oligonucleotide conjugates) for muscular dystrophies.
avidity biosciences是一家臨床階段的生物技術公司，開發用於肌肉營養不良的RNA治療（抗體寡核苷酸結合物）。
The company's lead asset, del-desiran (AOC 1001), is currently in the Phase 3 HARBOR trial in patients with myotonic dystrophy type 1 (DM1).
公司的主要資產del-desiran（AOC 1001）目前正在進行第3期HARBOR試驗，針對患有肌症型營養不良1型（DM1）的患者。
The company's pipeline includes Del-brax in the Phase 1/2 Fortitude trial for facioscapulohumeral muscular dystrophy (FSHD) and del-zota in a Phase 1/2 Explore44 trial in Duchenne muscular dystrophy (DMD) for patients amenable to exon 44 skipping.
公司的產品管線包括Del-brax在進行第1/2期Fortitude試驗，針對肌肉鬆弛性肌肉營養不良（FSHD），以及Del-zota在進行第1/2期Explore44試驗，針對對外顯子44跳躍敏感的杜甫肌肉營養不良（DMD）患者。
Goldman Sachs has initiated with a Buy rating and a price target of $59.
高盛已發出買入評級，並設定了59美元的價格目標。
The analyst sees a blockbuster commercial opportunity for del-brax in FSHD and a positive view of the clinical outlook and registrational path, with a 60% probability of success.
分析師認爲Del-brax在FSHD中有巨型商業機會，並對臨床前景和登記路徑持積極看法，成功概率爲60%。
Del-brax is well positioned to be first-to-market, and the analyst highlights significant revenue opportunity, with an estimated peak sales of $2.7 billion.
Del-brax有望成爲首家進入市場的產品，並且分析師強調巨大的營業收入機會，預計達到27億美元的銷售高峰。
Goldman Sachs analyst also says Del-desiran's Phase 1/2 data positions the company to potentially be a first-to-market drug in myotonic dystrophy. The analyst projects unadjusted peak sales of $4 billion and is optimistic about the trial's outcome, with a probability of success of 75%.
高盛分析師還表示，Del-desiran的第1/2期數據使該公司有望成爲肌症型營養不良的首家進入市場藥物。分析師預測未調整的銷售高峰達40億美元，並對試驗結果持樂觀態度，成功概率爲75%。
In August, Avidity Biosciences released Del-zota (AOC 1044) 5 mg/kg data that demonstrated a statistically significant increase of 25% of normal dystrophin production and 37% in exon 44 skipping in people living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the Phase 1/2 EXPLORE44 trial.
在8月份，avidity biosciences發佈了Del-zota（AOC 1044）5mg/kg的數據，表明相較於在進行適合於exon 44 skipping（DMD44）的杜歐肌營養不良突變患者中，正常二氫矽丁生產增加了25%，第44外顯子缺失增加了37%，在1/2期EXPLORE44試驗中有統計學上顯著的提升。
In addition, AOC 1044 5 mg/kg reduced creatine kinase levels to near normal with greater than 80% reduction compared to baseline.
此外，AOC 1044 5 mg/kg將肌酸激酶水平降低至接近正常水平，與基準相比減少超過80%。
Price Action: RNA stock is down 0.07% at $43.47 at last check Tuesday.
價格走勢：RNA股票最近一次檢查時下跌0.07%，報43.47美元。星期二。
Photo via Shutterstock
圖片來自shutterstock。
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譯文內容由第三人軟體翻譯。