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（纳斯达克：SRRK）股票周一上涨，此前该公司发布了在脊髓肌肉萎缩症的SAPPHIRE三期试验中，apitegromab的顶线结果。
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.
该研究实现了其主要终点，表明apitegromab在标准治疗药物长期使用的脊髓肌肉萎缩症患者中，在运动功能方面与安慰剂相比存在显著且临床意义重大的改善。
Standard therapies include Biogen Inc's (NASDAQ:BIIB) Spinraza (nusinersen) and Roche Holdings AG's (OTC:RHHBY) Evrysdi (risdiplam).
标准治疗包括Biogen Inc（纳斯达克：BIIB）的Spinraza（奴西嗪）和Roche Holdings AG（场外交易：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岁）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）基线变化的平均差异为1.8分（p=0.0192），与安慰剂（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）显示与安慰剂相比的1.4分平均差异（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剂量的预设分析显示，接受10 mg/kg apitegromab的患者（n=53）较安慰剂改善了2.2分（名义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).
运动功能结果在主要有效人群和探索人群（13-21岁）中都具有重要意义且一致，13-21岁的探索人群有22名倾向于apitegromab（n=22）而不是安慰剂（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有超过3分的改善，而安慰剂患者中为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治疗的患者首次在8周第一个测量时间点表现出早期的运动功能改善，到52周时通过HFMSE测量，益处扩大。
Treatment with apitegromab was well-tolerated across all age groups.
各年龄组对apitegromab治疗的耐受性良好。
The company plans to submit an FDA and European marketing applications in the first quarter of 2025.
该公司计划在2025年第一季度提交FDA和欧洲的营销申请。
Price Action: SRRK stock is up 320.2% to $31.43 at last check Monday.
股价走势：SRRk股票上涨320.2%，最近一次检查周一报收于31.43美元。
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Photo: Shutterstock
Photo: shutterstock