Scholar Rock's Muscle Wasting Drug Excels In Pivotal Trial, Plans FDA Submission Next Year
Scholar Rock's Muscle Wasting Drug Excels In Pivotal Trial, Plans FDA Submission Next Year
In the main efficacy population (ages 2-12), the mean difference in change from baseline in HScholar Rock Holding (NASDAQ:SRRK) stock is trading higher on Monday after the company released topline results from the Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy.
納斯達克的scholar rock控股（NASDAQ:SRRK）股票週一交易上漲，原因是公司發佈了脊髓性肌肉萎縮症SAPPHIRE試驗阿皮特格羅馬布第3期臨床試驗的頭部結果。
The study achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function in SMA patients on chronic dosing of standard-of-care therapies.
該研究實現了其主要終點，顯示了阿皮特格羅馬布相對安慰劑在脊髓性肌肉萎縮症患者慢性標準護理療法中運動功能的統計顯著和臨床意義上的改善。
Standard therapies include Biogen Inc's (NASDAQ:BIIB) Spinraza (nusinersen) and Roche Holdings AG's (OTC:RHHBY) Evrysdi (risdiplam).
標準治療包括Biogen Inc（納斯達克：BIIB）的Spinraza（努西尼生）和Roche Holdings AG的場外交易Evrysdi（利司地普蘭）。
In the main efficacy population (ages 2-12), the mean difference in change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) was 1.8 points (p=0.0192) for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (n=106) compared to placebo (n=50).
在主要有效人群（2-12歲）中，所有接受10mg/kg和20mg/kg阿皮特格羅馬布（n=106）的患者與安慰劑（n=50）相比，Hammersmith功能性運動量表擴展（HFMSE）的基線變化均值差異爲1.8分（p=0.0192）。
Patients receiving 20 mg/kg of apitegromab (n=53) showed a 1.4-point mean difference compared to placebo (p=0.1149).
接受20mg/kg阿皮特格羅馬布的患者（n=53）與安慰劑相比，平均差異爲1.4分（p=0.1149）。
The prespecified analysis of the 10 mg/kg dose showed that patients receiving 10 mg/kg of apitegromab (n=53) improved by 2.2 points (nominal p=0.0121) compared to placebo.
預設分析顯示，接受10 mg/kg阿皮特格羅馬布的患者（n=53）在比較安慰劑時改善了2.2分（名義p=0.0121）。
Motor function outcomes were meaningful and consistent across the main efficacy population, and the exploratory population ages 13-21 favored apitegromab (n=22) compared to placebo (n=10).
運動功能結果在主要有效人群和探索性人群（13-21歲）中都具有意義且一致，這些人群有利於阿皮特格羅馬布（n=22）而非安慰劑（n=10）。
Thirty percent of patients receiving apitegromab had a >3-point improvement in HFMSE compared to 12.5% of patients on placebo.
接受阿皮特格羅馬布的患者中，30％的患者HFMSE得分>3分且優於12.5％的接受安慰劑的患者。
Patients receiving apitegromab demonstrated early motor function improvement compared to placebo from the first measured time point at 8 weeks, benefit expanded at 52 weeks as measured by HFMSE.
接受apitegromab治療的患者從第一個記錄的8周時點開始表現出早期的運動功能改善，52周時HFMSE測量顯示其獲益不斷擴大。
Treatment with apitegromab was well-tolerated across all age groups.
治療apitegromab在所有年齡組中均具有良好的耐受性。
The company plans to submit an FDA and European marketing applications in the first quarter of 2025.
公司計劃在2025年第一季度提交FDA和歐洲營銷申請。
Price Action: SRRK stock is up 320.2% to $31.43 at last check Monday.
價格走勢：SRRk股價上漲320.2%，至31.43美元，截至週一最新數據。
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Photo: Shutterstock
Photo: shutterstock
譯文內容由第三人軟體翻譯。