Novartis AG (NYSE:NVS) announced topline results from the six-month, double-blind period of the Phase 3 APPEAR-C3G study of iptacopan for C3 glomerulopathy (C3G), a group of related conditions that cause the kidneys to malfunction.
Approximately 50% of C3G patients progress to kidney failure within ten years of diagnosis. Each year, approximately 1-2 people per million worldwide are newly diagnosed with C3G.
Last week, the FDA approved iptacopan, under the brand name Fabhalta, as the first oral monotherapy for a rare blood disorder, paroxysmal nocturnal hemoglobinuria.
The study met its primary endpoint, with iptacopan (200 mg twice daily) demonstrating superiority compared to placebo in providing clinically meaningful and statistically significant proteinuria (protein in urine) reduction on top of background therapy at six months.
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The safety profile of iptacopan was consistent with previously reported data.
Potential regulatory submissions are expected in 2024. The APPEAR-C3G study continues for a six-month, open-label period, in which all patients receive iptacopan, including those previously receiving a placebo.
In addition, enrollment is ongoing in a separate cohort of adolescent patients with C3G.
Novartis also announced results from the extension period of the Phase 3 APPLY-PNH trial of oral monotherapy Fabhalta (iptacopan) in PNH patients who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy.
Continuous Fabhalta treatment (200 mg twice daily) for 48 weeks enabled sustained hemoglobin-level increases to near-normal (12 g/dL or more), blood transfusion avoidance, and reduced patient-reported fatigue in the majority of patients; comparable benefits emerged in those patients switching from anti-C5 therapy to Fabhalta in the extension.
Price Action: NVS shares are up 0.53% at $96.81 during the premarket session on the last check Monday.
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諾華公司（紐約證券交易所代碼：NVS）公佈了iptacopan對C3腎小球病（C3G）進行爲期六個月的雙盲期 APPEAR-C3G 研究的總體結果，C3G是一組導致腎臟功能失調的相關疾病。
大約50％的C3G患者在診斷後的十年內發展爲腎衰竭。每年，全球每百萬人中約有1-2人被新診斷出患有C3G。
上週，美國食品藥品管理局批准名爲Fabhalta的iptacopan作爲首款治療罕見血液疾病（陣發性睡眠性血紅蛋白尿症）的口服單一療法。
該研究達到了其主要終點，與安慰劑相比，iptacopan（200 mg，每日兩次）在六個月的背景治療的基礎上，在減少具有臨床意義且具有統計學意義的蛋白尿（尿液中的蛋白質）方面具有優越性。
另請閱讀: 諾華概述仿製藥業務分拆後的純戰略進展。
iptacopan的安全性特徵與先前報告的數據一致。
潛在的監管文件預計將在2024年提交。APPEAR-C3G 研究持續了六個月的開放標籤期，在此期間，所有患者都接受了 iptacopan，包括之前接受安慰劑的患者。
此外，另一組C3G青少年患者的招生工作正在進行中。
諾華還公佈了口服單一療法Fabhalta（iptacopan）的3期APPLY-PNH試驗的延期結果，該試驗適用於儘管之前進行了抗C5治療，但仍有殘留貧血（血紅蛋白<10 g/dL）的PNH患者。
持續治療 Fabhalta（每天兩次 200 mg）持續 48 周，可使血紅蛋白水平持續升高至接近正常水平（12 g/dL 或以上），避免輸血，並減少大多數患者報告的疲勞；在延期從抗C5療法轉向Fabhalta的患者中，也出現了類似的益處。
價格走勢：在週一的最後一次盤前交易中，NVS股價上漲0.53％，至96.81美元。
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