Fulcrum Therapeutics Upgraded: BofA Debates Pivotal Study Success For Losmapimod In Rare Genetic Muscle Disease
Fulcrum Therapeutics Upgraded: BofA Debates Pivotal Study Success For Losmapimod In Rare Genetic Muscle Disease
BofA Securities has upgraded Fulcrum Therapeutics (NASDAQ:FULC) ahead of the company's phase 3 losmapimod readout, expected by the end of October for Facioscapulohumeral muscular dystrophy (FSHD).
美銀證券在Fulcrum Therapeutics（納斯達克股票代碼：FULC）的第三階段發佈之前，已經對Fulcrum Therapeutics（納斯達克股票代碼：FULC）進行了升級，該數據預計將於10月底發佈面部肩肱肌萎縮症（FSHD）。
FSHD is a rare genetic muscle disease affecting a child's facial muscles, shoulders, upper arms, and lower legs. These muscles weaken and shrink (atrophy).
FSHD 是一種罕見的遺傳性肌肉疾病，會影響孩子的面部肌肉、肩膀、上臂和小腿。這些肌肉變弱和收縮（萎縮）。
The analyst notes that although a previous pivotal study failed using the DUX4 expression endpoint, the company conducted a post-hoc analysis and designed a new study with the endpoint "reachable workspace" (RWS), which has sparked debate in the investment community.
該分析師指出，儘管先前使用DUX4表達式端點的一項關鍵研究失敗了，但該公司進行了事後分析，並設計了一項以 「可訪問工作空間」（RWS）終點爲終點的新研究，這引發了投資界的爭論。
Two key opinion leaders (KOLs) expressed optimism about the upcoming phase 3 results, citing the functional benefits seen in phase 2 using RWS, which measures upper extremity function. Despite being a newer endpoint, the KOLs believe RWS accurately tracks disease progression.
兩位關鍵意見領袖（KOL）對即將到來的第三階段結果表示樂觀，他們列舉了在第二階段使用測量上肢功能的RWS所帶來的功能益處。儘管是一個較新的終點，但KOL認爲RWS可以準確地追蹤疾病進展。
They view the stabilization of RWS in phase 3 as clinically meaningful and believe replicating the phase 2 results—showing a 10% placebo-adjusted change at 48 weeks—would be a success.
他們認爲RWS在3期的穩定具有臨床意義，並認爲複製2期結果（顯示48周時安慰劑調整後的變化爲10％）將是成功的。
However, the analyst remains uncertain about the study's likelihood of success due to the unpredictability of the control arm's response.
但是，由於控制部門的反應不可預測，分析師仍不確定該研究的成功可能性。
BofA upgraded the stock from Underperform to Neutral and increased the price target from $5 to $10, as the analyst increased the likelihood of success (LOS) from 35% to 50%
由於分析師將成功可能性（LOS）從35％提高到50％，美國銀行將該股從表現不佳上調至中性，並將目標股價從5美元上調至10美元
"We await the ph 3 readout before further de-risking the program and note we continue to view the pociredir story in sickle cell disease (SCD) as a stock overhang," the analyst adds.
該分析師補充說：「在進一步降低該計劃的風險之前，我們正在等待ph3的公佈結果，並注意到我們繼續將鐮狀細胞病（SCD）中的pociredir故事視爲股票積壓。」
BofA notes that losmapimod is expected to be the first treatment for FSHD, but it may face future competition from Avidity Biosciences Inc (NASDAQ:RNA) and Roche Holdings AG (OTC:RHHBY), which are further behind in development.
美國銀行指出，losmapimod有望成爲FSHD的首種治療方法，但未來可能會面臨來自Avidity Biosciences Inc（納斯達克股票代碼：RNA）和羅氏控股公司（場外交易代碼：RHHBY）的競爭，後者的開發進一步落後。
Price Action: FULC stock is up 1.30% at $8.651 at last check Monday.
價格走勢：週一最後一次檢查時，FULC股價上漲1.30％，至8.651美元。
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譯文內容由第三人軟體翻譯。