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.
Scholar Rock Holding（納斯達克：SRRK）股票週一上漲，此前該公司發佈了在脊髓肌肉萎縮症的SAPPHIRE三期試驗中，apitegromab的頂線結果。
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，表明apitegromab在標準治療藥物長期使用的脊髓肌肉萎縮症患者中，在運動功能方面與安慰劑相比存在顯著且臨床意義重大的改善。
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（奴西嗪）和Roche Holdings AG（場外交易：RHHBY）的Evrysdi（利斯地平）。
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apitegromab的患者（10 mg/kg和20 mg/kg，n=106）的Hammersmith Functional Motor Scale Expanded（HFMSE）基線變化的平均差異爲1.8分（p=0.0192），與安慰劑（n=50）相比。
Patients receiving 20 mg/kg of apitegromab (n=53) showed a 1.4-point mean difference compared to placebo (p=0.1149).
接受20 mg/kg apitegromab的患者（n=53）顯示與安慰劑相比的1.4分平均差異（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.
10 mg/kg劑量的預設分析顯示，接受10 mg/kg apitegromab的患者（n=53）較安慰劑改善了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人群（13-21歲）中都具有重要意義且一致，13-21歲的探索人群有22名傾向於apitegromab（n=22）而不是安慰劑（n=10）。
Thirty percent of patients receiving apitegromab had a >3-point improvement in HFMSE compared to 12.5% of patients on placebo.
30%接受apitegromab的患者HFMSE有超過3分的改善，而安慰劑患者中爲12.5%。
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治療的患者首次在8周第一個測量時間點表現出早期的運動功能改善，到52周時通過HFMSE測量，益處擴大。
Treatment with apitegromab was well-tolerated across all age groups.
各年齡組對apitegromab治療的耐受性良好。
The company plans to submit an FDA and European marketing applications in the first quarter of 2025.
該公司計劃在2025年第一季度提交FDA和歐洲的營銷申請。
Price Action: SRRK stock is up 320.2% to $31.43 at last check Monday.
股價走勢：SRRk股票上漲320.2%，最近一次檢查週一報收於31.43美元。
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Photo: Shutterstock
Photo: shutterstock
