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（納斯達克股票代碼：SRRK）週一股價走高，此前該公司公佈了apitegromab治療脊髓性肌萎縮症的3期SAPPHIRE試驗的主要結果。
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.
該研究達到了其主要終點，表明在長期服用標準護理療法的SMA患者中，與安慰劑相比，apitegromab在運動功能方面有統計學意義和臨床意義的改善。
Standard therapies include Biogen Inc's (NASDAQ:BIIB) Spinraza (nusinersen) and Roche Holdings AG's (OTC:RHHBY) Evrysdi (risdiplam).
標準療法包括百健公司（納斯達克股票代碼：BIIB）的Spinraza（nusinersen）和羅氏控股股份公司（場外交易代碼：RHHBY）的Evrysdi（risdiplam）。
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).
在主要療效人群（年齡2-12歲）中，所有接受阿匹特格羅單抗10 mg/kg和20 mg/kg（n=106）的患者與安慰劑（n=106）的患者相比，與基線的平均變化差異爲1.8點（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).
與安慰劑相比，接受20 mg/kg的apitegromab（n = 53）的患者顯示出1.4個百分點的平均差異（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.
對10毫克/千克劑量的預設分析表明，與安慰劑相比，接受10 mg/kg阿匹特格羅單抗（n=53）的患者改善了2.2個百分點（標稱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).
在主要療效人群中，運動功能結果是有意義且一致的，與安慰劑（n=10）相比，13-21歲的探索性人群更傾向於apitegromab（n=22）。
Thirty percent of patients receiving apitegromab had a >3-point improvement in HFMSE compared to 12.5% of patients on placebo.
在接受阿匹特格羅單抗治療的患者中，有30％的HFMSE改善了3個百分點，而服用安慰劑的患者的這一比例爲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.
與安慰劑相比，接受阿匹特格羅單抗治療的患者在8周的首次測量時間點開始表現出早期的運動功能改善，根據HFMSE的測量，受益範圍擴大到52周。
Treatment with apitegromab was well-tolerated across all age groups.
所有年齡組的apitegromab治療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.
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.
價格走勢：在週一的最後一次檢查中，sRK股價上漲320.2％，至31.43美元。
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Photo: Shutterstock
照片：Shutterstock