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Adverum Biotechnologies Announces Positive 52-Week LUNA and 4-Year OPTIC Results, and Provides Key Pivotal Program Design Elements

Adverum Biotechnologies Announces Positive 52-Week LUNA and 4-Year OPTIC Results, and Provides Key Pivotal Program Design Elements

adverum biotechnologies公佈了積極的52周LUNA和4年OPTIC結果,並提供了關鍵的關鍵計劃設計元素
GlobeNewswire ·  11/18 20:00

- 52-week LUNA data combined with follow-up from OPTIC at 4 years continue to support long-term potential best-in-class product profile of Ixo-vec

- 52周LUNA數據結合OPTIC的4年跟進繼續支持Ixo-vec長期潛力的最佳產品特性

- 6E10 dose in LUNA maintains visual and anatomic endpoints and demonstrates potential best-in-class injection-free rates and reduction in injection burden

- LUNA中6E10劑量維持視覺和解剖終點,並顯示出注射無負擔的最佳潛力和減少注射負擔

- No LUNA patients who received local steroid prophylaxis had inflammation at week 52 or at any subsequent visit, and 100% of OPTIC 2E11 patients were free of inflammation at year 1 and through year 4

- 所有接受局部類固醇預防的LUNA患者在第52周或後續就診中均未出現炎症,100%的OPTIC 2E11患者在1年和4年內均無炎症

- 6E10 with steroid eye drops or topical steroids to progress into two registrational studies; initial ARTEMIS Phase 3 non-inferiority study will evaluate a broad patient population; on track and expected to initiate in 1H 2025

- 6E10與類固醇眼藥水或局部類固醇進展到兩個註冊研究;初步ARTEMIS III期非劣效性研究將評估廣泛的患者群體;按計劃,預計在2025年上半年啓動

- Investor & analyst webcast, including a key opinion leader panel, to be held Monday, November 18th at 7:30 a.m. EST

- 投資者和分析師網絡廣播,包括一個關鍵意見領袖小組,將於11月18日星期一上午7:30(美國東部時間)舉行

REDWOOD CITY, Calif., Nov. 18, 2024 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy to preserve sight for life in highly prevalent ocular diseases, today announced topline 52-week results from the LUNA Phase 2 trial, new 4-year OPTIC long-term follow-up data and key pivotal program design elements.

加利福尼亞州紅木城,2024年11月18日(環球新聞通訊)—— Adverum Biotechnologies, Inc.(納斯達克股票代碼:ADVM),一家在臨床階段的公司,開創了利用基因療法治療高度流行的眼科疾病以維持視力的先河,今天宣佈來自LUNA第二階段試驗的52周整體結果、新的4年OPTIC長期跟蹤數據和關鍵的關鍵性程序設計要素。

"We are thrilled to report 52-week LUNA data and 4-year OPTIC data that continue to support Ixo-vec as a transformative and potential best-in-class therapy, which may provide patients who have wet AMD with potentially life-long benefit and a predictable safety profile. Both OPTIC 2E11 results and LUNA efficacy data at 52 weeks show maintenance of visual and anatomic endpoints with over 80% reduction in injection burden and greater than 50% injection freedom. These consistent results are bolstered by our OPTIC long-term data where we have demonstrated stable therapeutic aflibercept levels through 5 years. The data across both studies support a reliable long-term benefit and a predictable safety profile," stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. "Ultimately, Ixo-vec is a potential paradigm-shifting solution for patients with wet AMD, where real-world evidence suggests that up to 57% of patients stop anti-VEGF treatment within 5 years, and the vast majority of patients end up losing vision. Designed as a single, one-time intravitreal injection, Ixo-vec has the potential to extend therapeutic benefit from weeks to years. Today's 4-year OPTIC data suggest that Ixo-vec may preserve vision for the life of wet AMD patients."

「我們很高興地報告52周的LUNA數據和4年的OPTIC數據,這些數據繼續支持Ixo-vec作爲一種變革性和潛在的最佳療法,可能爲患有溼性AMD的患者提供潛在的終生好處和可預測的安全性資料。OPTIC 2E11結果和LUNA在52周的療效數據顯示,在注射負擔減少超過80%和注射自由度超過50%的情況下,視覺和解剖端點得以維持。這些一致的結果得到了我們的OPTIC長期數據的支持,我們在那裏證明了5年內治療性阿夫利攝坦水平的穩定。兩項研究的數據支持可靠的長期好處和可預測的安全性資料,」Adverum Biotechnologies的總裁兼首席執行官Laurent Fischer萬.D.表示。「最終,Ixo-vec是溼性AMD患者的潛在範式轉變解決方案,現實世界證據表明,高達57%的患者在5年內停止抗VEGF治療,而且絕大多數患者最終失去視力。作爲一次性玻璃體內注射設計的Ixo-vec有潛力將治療效果從幾周延長到幾年。今天的4年OPTIC數據顯示,Ixo-vec可能爲溼性AMD患者保持視力。」

"We have designed our Ixo-vec Phase 3 pivotal program to establish gene therapy as a standard of care for all wet AMD patients. Our ARTEMIS trial design considers feedback from key stakeholders, including global regulatory authorities, key opinion leaders, and patients, thereby optimizing for Ixo-vec's potential clinical, regulatory and commercial success," stated Rabia Gurses Ozden, MD, Chief Medical Officer at Adverum. "Today's LUNA 52-week data support our decision to advance the 6E10 dose and topical-eyedrops-only prophylaxis into Phase 3. One of the unique, and in my view, profound aspects of this LUNA update was the near unanimous patient preference for Ixo-vec, as assessed via a pre-specified patient survey. The vast majority preferred Ixo-vec over their prior intravitreal injections. No patients on topical eyedrops alone stated that the steroid eyedrops were difficult to manage. And 100% of patients who received Ixo-vec 6E10 and eyedrops alone preferred Ixo-vec over prior anti-VEGF treatments."

「我們已設計Ixo-vec第三階段關鍵性程序,以確立基因療法作爲所有溼性AMD患者的標準護理。我們的ARTEMIS試驗設計考慮了關鍵利益相關者的反饋,包括全球監管機構、關鍵意見領袖和患者,從而優化Ixo-vec的潛在臨床、監管和商業成功,」Adverum的首席醫療官Rabia Gurses Ozden醫生表示。「今天的LUNA 52週數據支持我們將6E10劑量和僅限局部眼藥水預防措施推進到第三階段的決定。就我而言,這次LUNA更新的獨特而深遠的方面之一是幾乎所有患者都選擇了Ixo-vec,這通過預先指定的患者調查進行了評估。絕大多數患者更喜歡Ixo-vec而不是他們之前的玻璃體內注射。在僅使用局部眼藥水的患者中,沒有一位患者表示激素眼藥水難以使用。而且100%的患者認爲接受Ixo-vec 6E10和眼藥水單獨使用的Ixo-vec比以前的抗VEGF治療更好。」

"The LUNA 52-week clinical data further establish that the 6E10 dose of Ixo-vec has the potential to meaningfully reduce treatment burden for patients with wet AMD, even among patients with highly active disease who are receiving frequent dosing," said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas, Professor of Clinical Ophthalmology, Blanton Eye Institute, Houston Methodist Hospital and a principal investigator for LUNA. "Encouragingly, the 6E10 dose with extended prophylaxis also resulted in less inflammation. Taking these LUNA results together with 4-year data from the 2E11 dose in OPTIC, the totality of data indicates a predictable immune response with inflammation that, if it occurs, is manageable with local steroids, doesn't impact vision, and ultimately resolves. These data from LUNA and OPTIC studies suggest a favorable benefit-risk profile for patients, which I believe many patients would consider if Ixo-vec were available in routine clinical practice. I look forward to working with the Adverum team as Ixo-vec advances toward pivotal studies next year."

「LUNA 52周的臨床數據進一步證明,Ixo-vec的6E10劑量有潛力實質性降低溼性AMD患者的治療負擔,即使在接受頻繁給藥的高度活躍病患者中也是如此。」德克薩斯州視網膜顧問研究所研究主任,醫學博士、哲學博士,休斯敦衛理公協會眼科教授,Blanton眼科研究所的查爾斯·維科夫博士說道,「令人鼓舞的是,採用延長預防措施的6E10劑量也減少了炎症。將這些LUNA結果與OPTIC中2E11劑量的4年數據結合起來,數據顯示出可預測的免疫反應,如果發生,可通過局部類固醇進行管理,不影響視力,最終會得到解決。這些來自LUNA和OPTIC研究的數據表明,對於患者而言,這表明了一個有利的效益-風險特徵,我相信許多患者會考慮在常規臨床實踐中使用Ixo-vec。我期待着與Adverum團隊合作,支持Ixo-vec在明年進軍關鍵研究。」

LUNA Phase 2 Trial and OPTIC First-in-Human Trial - Background and Baseline Prior Anti-VEGF Injections

LUNA第二階段試驗和OPTIC首次人體試驗 - 背景和基線之前的抗-VEGF注射

LUNA is an ongoing double-masked, randomized Phase 2 trial. 60 patients with wet AMD were randomized equally across two dose cohorts, 6E10 or 2E11 vg/eye. The trial is evaluating multiple prophylactic regimens, including topical steroid eyedrops (difluprednate) with or without Ozurdex and with or without oral steroids. LUNA is designed to inform the selection of both the Ixo-vec dose and prophylactic regimen for Phase 3 registrational trials.

LUNA是一項正在進行的雙盲隨機第二階段試驗。60名溼性AMD患者在兩個劑量組之間平均隨機分配,6E10或2E11 vg/眼。該試驗正在評估多種預防方案,包括局部類固醇眼藥水(difluprednate)與或不與Ozurdex及與或不與口服類固醇的組合。LUNA旨在爲第三階段註冊試驗選擇Ixo-vec劑量和預防方案提供參考。

OPTIC is an ongoing, open-label, dose-ranging first-in-human trial. 30 patients with wet AMD requiring frequent IVT injections were enrolled equally across two doses, 2E11 or 6E11. Patients received either six weeks of prophylactic topical steroid eye drops or 13 days of prophylactic oral steroids. The OPTIC trial was a two-year study, with an optional 3-year extension.

OPTIC是一項正在進行的開放標籤劑量選擇首次人體試驗。30名需要頻繁進行靜脈注射的溼性AMD患者被平均招募到兩個劑量組中,2E11或6E11。患者接受了六週的局部類固醇眼藥水的預防或13天的口服類固醇的預防。OPTIC試驗爲期兩年,具有可選的3年延長。

The LUNA and OPTIC data cutoff dates were August 29, 2024, and August 21, 2024, respectively. At the data cutoff date for LUNA, 57 patients had completed the 52-week study visit, with 3 discontinuations due to adverse events unrelated to study drug. 23 OPTIC patients elected to participate in the OPTIC extension. At the data cutoff date for OPTIC, 21 patients had completed the 4-year study visit, with 2 discontinuations unrelated to study drug.

LUNA和OPTIC的數據截止日期分別爲2024年8月29日和2024年8月21日。在LUNA的數據截止日期,57名患者完成了爲期52周的研究訪視,其中由於與研究藥物無關的不良事件而中止的有3例。23名OPTIC患者選擇參與OPTIC擴展。在OPTIC的數據截止日期,21名患者完成了爲期4年的研究訪視,其中有2例與研究藥物無關的中止。

Both LUNA and OPTIC were designed to assess a broad wet AMD population, including hard-to-treat patients with severe disease who required frequent anti-VEGF injections before enrolling in the trial. At baseline, mean annualized prior anti-VEGF injections in the year prior to enrolling in LUNA and OPTIC were 10.1 (2.6 SD) and 9.9 (1.9 SD), respectively.

LUNA和OPTIC的設計旨在評估廣泛的溼性AMD人群,包括在註冊試驗前需要頻繁進行抗VEGF注射的重症患者。在基線時,LUNA和OPTIC註冊前一年抗VEGF的平均年度注射次數分別爲10.1(標準差2.6)和9.9(標準差1.9)。

LUNA 52-week Analysis Topline Data Summary

LUNA 52周分析頂線數據摘要

  • Both doses of Ixo-vec maintained visual and anatomic endpoints through 52 weeks.
    • Best Corrected Visual Acuity (BCVA) - least squares mean BCVA change from baseline at week 52 (95% CI)1:
      • 6E10: -2.1 (-4.8, 0.7)
      • 2E11: -1.8 (-4.6, 0.9)
  • 兩種劑量的Ixo-vec在52周內維持了視覺和解剖終點。
    • 最佳矯正視力(BCVA) - 從基線到第52周的最小平方平均BCVA變化(95% CI)1:
      • 6E10: -2.1 (-4.8, 0.7)
      • 2E11: -1.8 (-4.6, 0.9)

1. Excludes 1 participant at each dose with letter loss due to cataract

1. 排除每個劑量中因白內障導致的字母丟失的1名參與者

    • Central Subfield Thickness (CST) - least squares mean CST (μm) change from baseline at week 52 (95% CI):
      • 6E10: -10.2 (-29.0, 8.5)
      • 2E11: -21.9 (-40.4, -3.3)
    • 中心亞區厚度(CST) - 從基線到第52周的最小平方平均CST(μm)變化(95% CI):
      • 6E10: -10.2 (-29.0, 8.5)
      • 2E11: -21.9 (-40.4, -3.3)
  • Both doses of Ixo-vec achieved an industry leading treatment burden reduction and proportion patients who were injection free through 52 weeks.
    • Treatment Burden Reduction - % reduction in mean annualized anti-VEGF injections:
      • 6E10: 88% treatment burden reduction
      • 2E11: 92% treatment burden reduction
    • Proportion of Patients Injection Free:
      • 6E10: 54% injection free, with 75% of patients with ≤1 injection
      • 2E11: 69% injection free, with 79% of patients with ≤1 injection
  • Both doses of Ixo-vec were well tolerated, with local steroids effectively managing inflammation when present.
    • No 6E10 patients had inflammation at week 52 or at any subsequent visit2.
    • No Ixo-vec-related serious adverse events. All Ixo-vec-related AEs were either mild or moderate: no episcleritis, vasculitis, retinitis, choroiditis, vascular occlusion, or hypotony.
    • The most common Ixo-vec-related AEs were dose-dependent anterior inflammation responsive to local corticosteroids and anterior pigmentary changes with no impact on vision.
    • No new onset inflammation after week 30.
  • 兩種劑量的Ixo-vec在控制治療負擔減少和無注射患者比例方面達到了行業領先水平,持續時間爲52周。
    • 治療負擔減少 - 年化抗VEGF注射平均減少的百分比:
      • 6E10: 88%治療負擔減少
      • 2E11: 92%治療負擔減少
    • 無注射患者比例:
      • 6E10: 54%無注射,75%的患者僅接受≤1次注射
      • 2E11: 69%無注射,79%的患者僅接受≤1次注射
  • 兩劑Ixo-vec均耐受良好,當存在時局部類固醇有效控制炎症。
    • 在第52周或任何後續訪問中,沒有6E10患者出現炎症。
    • 沒有與Ixo-vec相關的嚴重不良事件。所有與Ixo-vec有關的不良事件要麼是輕微的,要麼是中度的:沒有前鞏膜炎、血管炎、視網膜炎、脈絡膜炎、血管閉塞或眼壓低。
    • 與Ixo-vec相關的最常見不良事件是劑量依賴性的前部炎症,對局部皮質類固醇有反應,以及前部色素變化,對視力沒有影響。
    • 第30周之後沒有新發生的炎症。

2. Inflammation defined as grade ≥ 1 AC/VC cells

2.炎症定義爲AC/VC細胞等級≥1

  • 6E10 dose with topical eyedrops as prophylactic regimen selected for pivotal program, providing a predictable long-term favorable safety profile.
    • No patients at 6E10 with topical eyedrops had inflammation at week 52 or at any subsequent visit.
    • Only one subject had inflammation, which resolved by year 1.
  • LUNA results underscored by sub-group analyses that support potential best-in-class product profile and position Ixo-vec for potential clinical, regulatory and commercial success.
    • Demonstrated consistent benefit in both patients with ≤300 μm baseline CST ("dry") and patients with > 300 μm baseline CST ("wet").
    • Demonstrated maintenance of visual and anatomic outcomes in injection-free patients.
    • Demonstrated even more robust clinical activity in patients with less treatment burden (experienced patients with <6 injections in year prior to LUNA).
  • Results from our LUNA patient preference survey demonstrate strong preference for Ixo-vec over prior anti-VEGF therapies and acceptability of steroid regimen.
    • 93% (n=56) of LUNA patients at 52 weeks prefer Ixo-vec, including accompanying steroid regimen, over prior treatments. Patient preference for Ixo-vec over prior treatments increased over time, from 88% (n=57) at 26 weeks.
    • 95% (n=56) of LUNA patients would elect to receive Ixo-vec in the other eye if both eyes had wet AMD.
    • 96% (n=56) of LUNA patients would recommend Ixo-vec to their family or friends with wet AMD.
    • 100% (n=10) of patients on the 6E10 pivotal dose and topical eyedrop steroid regimen prefer Ixo-vec over prior treatments for wet AMD.
    • 100% (n=10) of patients on the 6E10 pivotal dose and topical eyedrop steroid regimen would elect to receive Ixo-vec in other eye if both eyes had wet AMD.
    • 100% (n=10) of patients on the 6E10 pivotal dose and topical eyedrop steroid regimen would recommend Ixo-vec to their family or friends with wet AMD.
    • No patients receiving topical eyedrop alone prophylaxis (n=20) stated it was difficult to manage.
  • 6E10劑量與局部眼藥水作爲預防方案選定用於關鍵項目,提供可預測的長期有利安全性概況。
    • 在6E10的患者中,使用局部眼藥水的患者在第52周或任何後續訪問中均未出現炎症。
    • 只有一名受試者出現了炎症,且在第一年時已自行好轉。
  • LUNA的結果通過子組分析得到了強調,支持潛在的最佳產品形象,併爲Ixo-vec在臨床、監管和商業成功的潛力奠定了基礎。
    • 在基線CSt≤300 μm(「幹」)和基線CSt>300 μm(「溼」)的患者中均展示了一致的益處。
    • 在沒有注射的患者中維持了視覺和解剖結果。
    • 在治療負擔更輕的患者中(在LUNA之前一年內接受
  • 我們的LUNA患者偏好調查結果顯示,患者對Ixo-vec的偏好明顯高於以前的抗VEGF療法,並且對類固醇方案的接受度較高。
    • 在52周時,93%(n=56)的LUNA患者更喜歡Ixo-vec(包括伴隨的類固醇方案),而不是之前的治療。患者對Ixo-vec的偏好隨着時間的推移而增加,從26周時的88%(n=57)上升。
    • 95%(n=56)的LUNA患者表示,如果兩隻眼睛都有溼性年齡相關性黃斑變性,他們會選擇在另一隻眼中接受Ixo-vec。
    • 96%(n=56)的LUNA患者會向有溼性年齡相關性黃斑變性的家人或朋友推薦Ixo-vec。
    • 100%(n=10)接受6E10關鍵劑量和局部眼藥水類固醇方案的患者更傾向於選擇Ixo-vec,而不是以前的溼性年齡相關性黃斑變性治療。
    • 100%(n=10)接受6E10關鍵劑量和局部眼藥水類固醇方案的患者表示,如果兩隻眼睛都有溼性年齡相關性黃斑變性,他們會選擇在另一隻眼中接受Ixo-vec。
    • 100%(n=10)接受6E10關鍵劑量和局部眼藥水類固醇方案的患者會向有溼性年齡相關性黃斑變性的家人或朋友推薦Ixo-vec。
    • 接受局部眼藥水單獨預防(n=20)的患者表示管理起來並不困難。

OPTIC (2E11) 4-year Analysis Topline Data Summary

OPTIC(2E11)4年分析總體數據摘要

  • Patients in OPTIC received 9.9 mean annualized injections prior to receiving Ixo-vec. Despite significant treatment need at baseline, these patients continue to experience long-term benefit from Ixo-vec through at least 4 years of follow up, including maintenance of vision, durability of anatomical improvements and sustained reduction in anti-VEGF treatment burden. Aflibercept levels have been demonstrated up to 5-years post-treatment.
    • Patients had an 86% reduction in annualized anti-VEGF injections through year 4, with a robust reduction in treatment burden demonstrated in each year following Ixo-vec administration.
      • Through Year 1: 84% reduction in anti-VEGF injections
      • Through Year 2: 81% reduction in anti-VEGF injections
      • Through Year 3: 84% reduction in anti-VEGF injections
      • Through Year 4: 86% reduction in anti-VEGF injections
    • 4-year OPTIC data underscore Ixo-vec's reliable long-term benefit.
      • Nearly 50% of patients were injection free through 4 years following Ixo-vec treatment.
      • 78% of OPTIC participants who were injection free through year 1 remained injection free through year 4.
      • 88% of OPTIC participants who were injection free through year 2 remained injection free through year 4.
      • Durable aqueous aflibercept protein levels up to 5 years after a single Ixo-vec IVT injection.
  • Ixo-vec at 2E11 was generally well tolerated and demonstrated a favorable safety profile.
    • Inflammation was dose dependent, did not impact vision and, when present, was responsive to local corticosteroids.
    • Long-term data establish a 10-fold safety margin from highest dose tested in nAMD.
  • 在OPTIC研究中,患者在接受Ixo-vec治療之前平均每年接受9.9次注射。儘管基線時存在顯著的治療需求,但這些患者在至少4年的隨訪中仍持續感受到Ixo-vec的長期益處,包括視力維持、解剖改善的持久性和抗VEGF治療負擔的持續減少。研究證明,阿弗利珠單抗的水平在治療後可持續到5年。
    • 患者在接受Ixo-vec治療的第4年中,抗VEGF注射的年均減少率爲86%,在Ixo-vec給藥後的每一年中都表現出穩健的治療負擔減少。
      • 第一年:抗VEGF注射減少84%
      • 第二年:抗VEGF注射減少81%
      • 第三年:抗VEGF注射減少84%
      • 第四年:抗VEGF注射減少86%
    • 4年的OPTIC數據強調了Ixo-vec的可靠長期益處。
      • 近50%的患者在接受Ixo-vec治療後的4年中無需注射。
      • 在第一年內獲得注射自由的OPTIC參與者中,有78%在第四年內仍然保持注射自由。
      • 在第二年內獲得注射自由的OPTIC參與者中,有88%在第四年內仍然保持注射自由。
      • 在單次Ixo-vec IVt注射後,持續的水溶性阿柏西普蛋白水平可維持至5年。
  • Ixo-vec在2E11劑量下通常耐受良好,並顯示出有利的安全性特徵。
    • 炎症與劑量相關,不影響視力,並且當出現時,對局部皮質類固醇有反應。
    • 長期數據確立了nAMD中測試的最高劑量的10倍安全裕度。

Key Design Elements of the Ixo-vec Phase 3 Pivotal Program

Ixo-vec三期關鍵程序的關鍵設計要素

  • The company plans to conduct two, double-masked, randomized Phase 3 clinical trials.
  • The initial 284-patient, US-based ARTEMIS Phase 3 study is expected to enroll a broad patient population, including both treatment-naïve and treatment-experienced wet AMD patients.
  • The primary endpoint, measured at an average of weeks 52 and 56, is non-inferiority (NI) in mean BCVA change from baseline between Ixo-vec (6E10 vg/eye) and aflibercept (2mg Q8W). The non-inferiority margin for this study is -4.5 letters.
  • All patients will receive three monthly loading doses of aflibercept prior to Ixo-vec.
  • The study will utilize a sham in the control arm to support masking. Patients in both arms will be eligible for supplemental injections of aflibercept and will receive topical steroid eye drops.
  • This trial design is based on our end-of-Phase 2 feedback from the U.S. Food and Drug Administration (FDA).
  • ARTEMIS remains on track and is expected to initiate in 1H 2025.
  • 該公司計劃進行兩項雙盲隨機的第三階段臨床試驗。
  • 初始的284名患者、美國基礎的ARTEMIS第三階段研究預計將招募廣泛的患者群體,包括治療 näive 和有治療經驗的溼性年齡相關性黃斑變性患者。
  • 主要終點是在52周和56周時的平均值,測量Ixo-vec(6E10 vg/眼)和阿弗利布單抗(2mg Q8W)基線BCVA變化的非劣效性(NI)。本研究的非劣效性邊際爲-4.5字母。
  • 所有患者在接受Ixo-vec之前將接收三個月的阿弗利布單抗負荷劑量。
  • 該研究將在對照組中使用假藥以支持盲法。兩個治療組的患者均可接受額外的阿弗利布單抗注射,並會接受局部類固醇眼藥水。
  • 該試驗設計基於美國食品藥品監督管理局(FDA)對我們第二階段結束時的反饋。
  • ARTEMIS仍按計劃推進,預計將在2025年上半年啓動。

Updated Cash Runway Guidance

更新的現金運營指導

As of September 30, 2024, the company had $153.2 million in cash, cash equivalents and short-term investments. The company expects to be able to fund operations into the second half of 2025, which does not include completion of the ARTEMIS Phase 3 trial.

截至2024年9月30日,該公司擁有15320萬美元的現金、現金等價物和短期投資。該公司預計能夠爲運營提供資金,直到2025年下半年,這不包括ARTEMIS第三階段試驗的完成。

Webcast Details

網絡研討會細節

The live webcast will be accessible under Events and Presentations in the Investors section of the company's website. Listeners can access the webcast through this link: A replay will be available on the company's website shortly after the conclusion of the webcast.

直播網絡研討會將在公司網站投資者部分的事件和演示中提供。聽衆可以通過此鏈接訪問網絡研討會:網絡研討會結束後不久,回放將在公司網站上提供。

About Wet Age-Related Macular Degeneration

關於溼性年齡相關性黃斑變性

Wet AMD, also known as neovascular AMD or nAMD, is a VEGF driven advanced form of AMD affecting approximately 10% of patients living with AMD associated with the build-up of fluid in the macula and the retina. Wet AMD is a leading cause of blindness in people over 65 years of age, with approximately 20 million individuals worldwide living with this condition. New cases of wet AMD are expected to grow significantly worldwide as populations age. AMD is expected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases. Additionally, wet AMD is a bilateral disease, and incidence of nAMD in the second eye is up to 42% in the first two to three years. The current standard of care requires frequent life-long repeated bolus injections of anti-VEGF in the eye. IVT gene therapy has the promise to preserve vision and reduce most or all injections for the life of the patient by delivering stable therapeutic levels of anti-VEGF to control macular fluid.

Wet AMD, also known as neovascular AMD or nAMD, is a VEGF driven advanced form of AMD affecting approximately 10% of patients living with AMD associated with the build-up of fluid in the macula and the retina. Wet AMD is a leading cause of blindness in people over 65 years of age, with approximately 2000萬 individuals worldwide living with this condition. New cases of wet AMD are expected to grow significantly worldwide as populations age. AMD is expected to impact 28800萬 people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases. Additionally, wet AMD is a bilateral disease, and incidence of nAMD in the second eye is up to 42% in the first two to three years. The current standard of care requires frequent life-long repeated bolus injections of anti-VEGF in the eye. IVt gene therapy has the promise to preserve vision and reduce most or all injections for the life of the patient by delivering stable therapeutic levels of anti-VEGF to control macular fluid.

About Ixo-vec in Wet AMD

關於Ixo-vec在溼性年齡相關性黃斑變性中的應用

Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician's office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF, optimize patient compliance and improve vision outcomes for patients with wet AMD. In recognition of the need for new treatment options for wet AMD, FDA granted Fast Track designation for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the EMA and the Innovation Passport from the United Kingdom's Medicines and Healthcare Products Regulatory Agency for the treatment of wet AMD.

Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVm-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVt injection in the physician's office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF, optimize patient compliance and improve vision outcomes for patients with wet AMD. In recognition of the need for new treatment options for wet AMD, FDA granted Fast Track designation for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the EMA and the Innovation Passport from the United Kingdom's Medicines and Healthcare Products Regulatory Agency for the treatment of wet AMD.

About Adverum Biotechnologies

關於Adverum生物技術公司

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians' offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit .

adverum biotechnologies(納斯達克:ADVM)是一家臨床階段公司,旨在將基因治療確立爲高度普遍的眼科疾病的新標準治療,期望開發出恢復視力和預防失明的功能性療法。利用其專有的玻璃體內(IVT)平台的能力,adverum正在開發耐用的單次給藥療法,旨在在醫生辦公室中提供,以消除治療這些疾病所需的頻繁眼內注射。adverum正在評估其新型基因治療候選藥物ixoberogene soroparvovec(Ixo-vec,以前稱爲ADVm-022),作爲一種一次性IVT注射,針對患有新生血管或溼性年齡相關性黃斑變性的患者。此外,通過克服與現有治療方案相關的挑戰,adverum期望能夠改變治療標準,保護視力,並在全球範圍內產生深遠的社會影響。欲了解更多信息,請訪問。

Forward-looking Statements

前瞻性聲明

Statements contained in this press release regarding events or results that may occur in the future are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding: the long-term potential best-in-class product profile of Ixo-vec; potential best-in-class injection-free rates and reduction in injection burden of Ixo-vec; the trial design of the Ixo-vec Phase 3 pivotal program and anticipated initiation timing; the potential of Ixo-vec to be transformative and a best-in-class therapy; the potential life-long therapeutic benefit and predictable safety profile of Ixo-vec; the potential of Ixo-vec to shift the treatment paradigm for patients with wet AMD; the ability to establish gene therapy as a standard of care for wet AMD patients; the likelihood of clinical, regulatory and commercial success of Ixo-vec; the Company's cash sufficiency and runway; and other statements that are not historical fact. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum's novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; the potential for future complications or side effects in connection with use of Ixo-vec; and risks associated with market condition. Additional risks and uncertainties facing Adverum are set forth under the caption "Risk Factors" and elsewhere in Adverum's Securities and Exchange Commission (SEC) filings and reports, including Adverum's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on November 4, 2024 and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

本新聞稿中關於可能在未來發生的事件或結果的聲明是根據1995年《私人證券訴訟改革法》的定義,屬於「前瞻性聲明」。此類聲明包括但不限於關於:Ixo-vec的長期潛力最佳產品特徵;Ixo-vec的無注射率和減少注射負擔的潛力;Ixo-vec三期關鍵方案的試驗設計及預期啓動時間;Ixo-vec具有變革性和最佳療法的潛力;Ixo-vec可能帶來的終身治療益處和可預測的安全特徵;Ixo-vec可能改變溼性AMD患者的治療範式;確立基因治療作爲溼性AMD患者標準醫療的能力;Ixo-vec的臨床、監管和商業成功的可能性;公司的現金充足性和可持續性;以及其他不是歷史事實的聲明。實際結果可能因各種風險和不確定性而與這種前瞻性聲明中的預期結果存在重大差異,包括涉及的固有風險,其中包括但不限於:adverum的新技術,使得預測臨床試驗開始和完成的時間變得困難;監管不確定性;登記不確定性;早期臨床試驗結果不總是預測未來臨床試驗和結果的有效性;使用Ixo-vec可能出現的未來併發症或副作用的潛力;以及與市場條件相關的風險。adverum面臨的其他風險和不確定性在題爲「風險因素」的標題下及adverum的證券交易委員會(SEC)文件和報告的其他部分中列出,包括adverum截至2024年9月30日的季度報告(Form 10-Q),該報告於2024年11月4日向SEC提交,以及隨後向SEC提交的文件。本新聞稿中包含的所有前瞻性聲明僅在作出聲明的日期有效。adverum沒有義務更新此類聲明,以反映作出聲明後發生的事件或存在的情況,除非法律要求。

Inquiries:

查詢:

Adverum Investor Relations

Adverum投資者關係

Email: ir@adverum.com

電子郵件:ir@adverum.com


譯文內容由第三人軟體翻譯。


以上內容僅用作資訊或教育之目的,不構成與富途相關的任何投資建議。富途竭力但無法保證上述全部內容的真實性、準確性和原創性。
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