PTC Therapeutics Ends Utreloxastat Development After Rare Neurodegenerative Disease Trial Misses Goal, Analyst Maintains Outperform With Focus On Sepiapterin, Huntington's Programs
PTC Therapeutics Ends Utreloxastat Development After Rare Neurodegenerative Disease Trial Misses Goal, Analyst Maintains Outperform With Focus On Sepiapterin, Huntington's Programs
While modest numerical benefit was recorded on the primary endpoint, and a favorable clinical effect was correlated with lowering plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints.On Tuesday, PTC Therapeutics Inc (NASDAQ:PTCT) revealed data from the global Phase 2 placebo-controlled CardinALS study of Utreloxastat in amyotrophic lateral sclerosis patients.
週二，ptc therapeutics公司（納斯達克: ptct）公佈了全球第2期安慰劑對照CardinALS研究中Utreloxastat在肌萎縮性側索硬化症患者中的數據。
The study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.
該研究未達到減緩疾病進展的主要終點，即複合ALSFRS-R和死亡分析。
Also Read: FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
此外閱讀：FDA覈准PTC Therapeutics基因治療作爲首個可直接在大腦內給藥的有條件批准。
While modest numerical benefit was recorded on the primary endpoint, and a favorable clinical effect was correlated with lowering plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints.
雖然主要終點記錄了適度的數值好處，並且有利的臨床效應與降低血漿神經絲輕鏈（NfL）相關聯，但沒有達到統計學意義（p=0.52）。此外，次要療效終點也未達到統計學顯著性。
Amyotrophic lateral sclerosis (ALS), sometimes referred to as motor neuron disease or Lou Gehrig's disease, is a rare neurodegenerative disease that affects motor neurons in the brain and spinal cord.
肌萎縮性側索硬化（ALS），有時被稱爲運動神經元疾病或路易·格里希症，是一種影響大腦和脊髓中的運動神經元的罕見神經退行性疾病。
Utreloxastat was demonstrated to be safe and well-tolerated in the CardinALS trial. However, further development is not planned due to the lack of efficacy and biomarker signal.
在CardinALS試驗中，Utreloxastat被證明是安全且耐受良好的。然而，由於缺乏療效和生物標誌信號，不計劃進行進一步開發。
William Blair notes that while the CardinALS trial results were disappointing, the program was not a major factor in its investment thesis. As a result, ending the program does not affect the firm's Outperform rating on the stock.
William Blair指出，儘管CardinALS試驗結果令人失望，但該項目並未成爲其投資論點的主要因素。因此，終止該計劃不會影響公司對該股票的超額評級。
The analyst highlights the potential approval and launch of sepiapterin as a key growth driver over the next year, particularly in treating Phenylketonuria (PKU). William Blair believes the market is undervaluing this opportunity, with a PDUFA decision expected in July.
分析師強調sepiapterin的潛在覈準和上市將成爲未來一年內的關鍵增長驅動力，特別是在治療苯丙酮尿症（PKU）方面。William Blair認爲市場低估了這個機會，PDUFA決定預計將於7月出台。
The analyst also points to growing investor interest in the company's Huntington's disease program. Given the strong commercial prospects of this small-molecule therapy, positive discussions with regulators about an accelerated approval pathway for PTC-518 could significantly boost the stock. A Type C meeting with the FDA is planned for the fourth quarter to discuss the regulatory pathway.
該分析師還指出，投資者對該公司亨廷頓病項目日益增長的興趣。鑑於這種小分子療法的強大商業前景，與監管機構就 ptc therapeutics 的PTC-518獲得快速批准途徑的積極討論可能會極大地提振股價。已計劃於第四季度與FDA進行C型會議，討論監管途徑。
Additionally, the company aims to file a U.S. marketing application for vatiquinone in Friedreich's ataxia (FA) by late 2024. William Blair expects approval based on the drug's clinical data and the FDA's recent neurology approvals, especially as no treatments exist for pediatric FA patients.
此外，該公司計劃於2024年底前提交vatiquinone在弗裏德雷希共濟失調症（FA）的美國營銷申請。William Blair預計基於藥物臨床數據和FDA最近的神經學批准，尤其是對於兒童FA患者目前尚無治療方法的情況下，會獲得批准。
On Wednesday, PTC Therapeutics agreed to sell its Rare Pediatric Disease Priority Review Voucher for $150 million.
週三，ptc therapeutics同意以15000萬美元賣出其罕見兒童疾病優先審查券。
PTC was granted the voucher along with the FDA approval of Kebilidi (eladocagene exuparvovec-tneq) for children and adults with AADC deficiency.
在FDA批准Kebilidi（eladocagene exuparvovec-tneq）用於兒童和成人的AADC缺乏症後，PTC獲得了該審查券。
Price Action: PTCT stock is down 1.98% at $45.10 during the premarket session at last check Wednesday.
股價走勢：PTCt股票週三最後一次檢查預市交易時下跌1.98%，報45.10美元。
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Illustration of Phrama lab worker created with MidJourney.
藥廠實驗室工作者插圖，由MidJourney創建。
譯文內容由第三人軟體翻譯。
