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Annexon, Inc. Reports Positive Real-World Evidence Study Results Supporting ANX005 for Guillain-Barré Syndrome Treatment

Annexon, Inc. Reports Positive Real-World Evidence Study Results Supporting ANX005 for Guillain-Barré Syndrome Treatment

Annexon, Inc. 報告支持 ANX005 治療吉蘭-巴雷綜合症的積極真實世界證據研究結果
Quiver Quantitative ·  2024/12/16 07:41

Real-world evidence study shows ANX005 improves muscle strength and functional outcomes in Guillain-Barré Syndrome compared to current treatments.

真實世界證據研究顯示,ANX005相比於當前治療方法改善了格林-巴利綜合症患者的肌肉力量和功能結果。

Quiver AI Summary

Quiver AI 概要

Annexon, Inc. announced positive topline results from a real-world evidence study supporting its drug ANX005 as a potential treatment for Guillain-Barré Syndrome (GBS), a serious condition with no FDA-approved therapies. The study compared a matched cohort of 79 GBS patients treated with ANX005 to another cohort treated with standard therapies, finding that those receiving ANX005 showed significantly greater and quicker improvements in muscle strength and functional outcomes. Notably, fewer patients on ANX005 required mechanical ventilation, and they spent less time in the ICU. The results, which align with findings from Annexon's earlier Phase 3 trial, are encouraging for the potential regulatory submission of ANX005 as a treatment for GBS, with plans for a U.S. Biologics License Application in the first half of 2025.

Annexon, Inc. 宣佈了來自真實世界證據研究的積極頂線結果,支持其藥物ANX005作爲格林-巴利綜合症(GBS)潛在治療方案,該病是一種沒有FDA批准治療的嚴重疾病。該研究比較了79名接受ANX005治療的GBS患者與另一組接受標準治療的患者,發現接受ANX005的患者在肌肉力量和功能結果方面表現出顯著更大且更快的改善。值得注意的是,接受ANX005治療的患者中,需要機械通氣的患者更少,且他們在重症監護室的時間也更短。這些結果與Annexon早期進行的3期臨牀試驗的發現一致,令人鼓舞,支持將ANX005作爲治療GBS的潛在監管提交計劃,計劃在2025年上半年提交美國生物製品許可申請。

Potential Positives

潛在的積極因素

  • Positive topline results from a real-world evidence study support ANX005 as a potential treatment for Guillain-Barré Syndrome (GBS), a condition with no FDA-approved therapies.
  • ANX005 demonstrated early and greater improvements in muscle strength and functional outcomes compared to standard treatments like IVIg or PE.
  • Fewer patients treated with ANX005 required mechanical ventilation, indicating a potential reduction in the overall healthcare burden associated with GBS.
  • The study results strengthen the company's position as it prepares for a planned U.S. Biologics License Application submission in the first half of 2025.
  • 來自真實世界證據研究的積極頂線結果支持ANX005作爲格林-巴利綜合症(GBS)的潛在治療方案,這種疾病沒有FDA批准的治療。
  • 與標準治療方法如IVIg或市盈率相比,ANX005表現出更早且更顯著的肌肉力量和功能結果改善。
  • 接受ANX005治療的患者中,需要機械通氣的患者更少,這表明可能減輕與GBS相關的整體醫療負擔。
  • 研究結果強化了公司在2025年上半年計劃提交美國生物製品許可申請的準備。

Potential Negatives

潛在負面因素

  • Potential regulatory concerns may arise if the FDA does not find the data from the real-world evidence study sufficient for filing a Biologics License Application (BLA).
  • The possibility exists that regulatory authorities could require additional information or studies prior to the approval of ANX005, which could delay its market entry.
  • The company has a history of net operating losses, raising concerns about its financial stability and ability to fund ongoing clinical programs.
  • 如果FDA認爲來自真實世界證據研究的數據不足以提交生物製品許可申請(BLA),可能會出現潛在的監管擔憂。
  • 監管機構可能會要求在批准ANX005之前提供額外的信息或研究,這可能會延遲其市場進入。
  • 該公司有淨運營虧損的歷史, raising concerns about its financial stability and ability to fund ongoing clinical programs.

FAQ

常見問題

What are the key findings of the ANX005 real-world evidence study?

ANX005真實世界證據研究的主要發現是什麼?

The study indicates ANX005 provides earlier and greater improvements in muscle strength and disability compared to IVIg or PE for GBS patients.

研究表明,與IVIg或市盈率相比,ANX005在提高肌肉力量和殘疾方面效果更早且更顯著,適用於GBS患者。


How does ANX005 compare to standard treatments for GBS?

ANX005與GBS的標準治療方法相比如何?

ANX005 demonstrated faster recovery, reduced need for mechanical ventilation, and shorter ICU stays compared to standard treatments like IVIg or plasma exchange.

ANX005在恢復速度上表現更快,減少了機械通氣的需求,並且相較於標準治療如IVIg或血漿置換,ICU住院時間更短。


When will Annexon submit the U.S. Biologics License Application for ANX005?

Annexon將何時提交ANX005的美國生物製品許可申請?

Annexon plans to submit the U.S. Biologics License Application for ANX005 in the first half of 2025.

Annexon計劃在2025年上半年提交ANX005的美國生物製品許可申請。


What is Guillain-Barré Syndrome (GBS)?

什麼是格林巴利綜合症(GBS)?

GBS is a severe, acute neuromuscular disease causing rapid-onset paralysis and nerve damage, with no FDA-approved treatments available.

GBS是一種嚴重的急性神經肌肉疾病,會導致快速發作的癱瘓和神經損傷,目前沒有FDA批准的治療方法。


What is the design of the ANX005 clinical study?

ANX005臨牀研究的設計是什麼?

The study used a matched cohort design, comparing 79 real-world GBS patients to those treated with ANX005 in a Phase 3 trial.

該研究使用了匹配隊列設計,比較了79名真實世界的GBS患者與在三期試驗中接受ANX005治療的患者。

Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.

免責聲明:這是由GlobeNewswire分發的新聞稿的人工智能生成摘要。用於總結這份稿件的模型可能會出錯。請在這裏查看完整發佈。


$ANNX Insider Trading Activity

$ANNX內部交易活動

$ANNX insiders have traded $ANNX stock on the open market 12 times in the past 6 months. Of those trades, 4 have been purchases and 8 have been sales.

$ANNX的內部人士在過去6個月內在公開市場交易過$ANNX股票12次。在這些交易中,4次爲購買,8次爲出售。

Here's a breakdown of recent trading of $ANNX stock by insiders over the last 6 months:

以下是過去6個月內部人士對$ANNX股票的近期交易明細:

  • WILLIAM H. CARSON has traded it 4 times. They made 4 purchases, buying 12,800 shares and 0 sales.
  • TED YEDNOCK (EVP & CHIEF INNOVATION OFFICER) has traded it 6 times. They made 0 purchases and 6 sales, selling 27,514 shares.
  • MICHAEL OVERDORF (EVP & CHIEF BUSINESS OFFICER) sold 784 shares.
  • JENNIFER LEW (EVP & CHIEF FINANCIAL OFFICER) sold 1,104 shares.
  • WILLIAm H. CARSON 交易了4次。他們進行了4次購買,購買了12,800股,並沒有出售。
  • TED YEDNOCk (執行副總裁兼首席創新官) 交易了6次。他們沒有購買,進行了6次出售,出售了27,514股。
  • MICHAEL OVERDORF (執行副總裁兼首席業務官) 出售了784股。
  • JENNIFER LEW (執行副總裁兼財務長) 出售了1,104股。

To track insider transactions, check out Quiver Quantitative's insider trading dashboard.

要跟蹤內部交易,請查看Quiver Quantitative的內部交易儀表。

$ANNX Hedge Fund Activity

$ANNX 對沖基金活動

We have seen 79 institutional investors add shares of $ANNX stock to their portfolio, and 76 decrease their positions in their most recent quarter.

我們看到79家機構投資者在最近的季度中增加了$ANNX股票的持有量,76家減少了他們的持倉。

Here are some of the largest recent moves:

以下是最近的一些重大變動:

  • STATE STREET CORP added 2,068,294 shares (+116.6%) to their portfolio in Q3 2024
  • PICTET ASSET MANAGEMENT HOLDING SA removed 1,644,591 shares (-100.0%) from their portfolio in Q2 2024
  • SIO CAPITAL MANAGEMENT, LLC added 1,433,155 shares (+inf%) to their portfolio in Q3 2024
  • MARSHALL WACE, LLP removed 1,401,291 shares (-82.7%) from their portfolio in Q3 2024
  • GOLDMAN SACHS GROUP INC added 1,308,187 shares (+219.4%) to their portfolio in Q3 2024
  • BLACKROCK, INC. added 1,060,427 shares (+16.1%) to their portfolio in Q3 2024
  • GMT CAPITAL CORP removed 1,031,579 shares (-35.1%) from their portfolio in Q3 2024
  • 州街CORP在2024年第三季度向其投資組合添加了2,068,294股(+116.6%)
  • 巴黎銀行資產管理控股SA在2024年第二季度從其投資組合中移除了1,644,591股(-100.0%)
  • SIO資本管理公司在2024年第三季度向其投資組合添加了1,433,155股(+inf%)
  • 馬歇爾威斯有限公司在2024年第三季度從其投資組合中移除了1,401,291股(-82.7%)
  • 高盛集團有限公司在2024年第三季度向其投資組合添加了1,308,187股(+219.4%)
  • 貝萊德公司在2024年第三季度增加了1,060,427股(+16.1%)到他們的投資組合中
  • GMT資本公司在2024年第三季度從他們的投資組合中移除1,031,579股(-35.1%)

To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.

要跟蹤對沖基金的股票投資組合,請查看Quiver Quantitative的機構持有情況儀表。

Full Release

完整發佈




Real-World Evidence Study Strengthens the Body of Evidence Supporting ANX005 for Treatment of GBS



真實世界證據研究加強了支持ANX005治療GBS的證據基礎




ANX005 Phase 3 Population Was Matched 1:1 on Prespecified Criteria with Patients in International GBS Outcomes Study (IGOS)



ANX005第三階段的受試者在預先規定的標準下與國際GBS結果研究(IGOS)中的患者進行了1:1匹配




Matched Cohort Study Showed Early and Greater Benefits of ANX005 over IVIg or PE in Muscle Strength and Functional Outcomes Across Multiple Measurements



匹配隊列研究顯示,ANX005在肌肉力量和功能結果的多個測量指標上的早期和更大益處,相較於IVIg或市盈率




Conference Call and Webcast Today at 8:30 a.m. ET



今天上午8:30(東部時間)召開電話會議並進行網絡廣播



BRISBANE, Calif., Dec. 16, 2024 (GLOBE NEWSWIRE) --

Annexon, Inc.

(Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today announced positive topline results from a real-world evidence (RWE) study supporting ANX005 as a potential treatment for Guillain-Barré Syndrome (GBS). GBS is a rapid-onset and acute neuromuscular disease with no U.S. Food and Drug Administration (FDA)-approved treatments. ANX005, the most advanced targeted immunotherapy in development for GBS, is designed to rapidly block C1q and complement activity with a single dose to halt disease progression during the critical progressive phase of the disease.


加利福尼亞州布里斯班,2024年12月16日(環球新聞)--

Annexon公司

(納斯達克:ANNX)是一家生物製藥公司,正在推動一種針對嚴重的經典補體介導的神經炎症疾病的新型療法的晚期臨牀平台,這些疾病影響身體、大腦和眼睛。今天宣佈了支持ANX005作爲格林-巴利綜合症(GBS)潛在治療方法的真實世界證據(RWE)研究的積極初步結果。GBS是一種快速發作和急性的神經肌肉疾病,目前沒有美國食品和藥物管理局(FDA)批准的治療方法。ANX005是針對GBS的最先進的靶向免疫療法,旨在通過單次劑量快速阻斷C1q和補體活動,以在疾病的關鍵進展階段阻止疾病進展。



Working in collaboration, the IGOS investigators and Annexon established a cohort of 79 real-world patients from the IGOS global patient registry that was matched based on key prespecified prognostic factors to the cohort of 79 patients treated with ANX005 30 mg/kg from Annexon's completed Phase 3 study conducted outside the United States. Patients in the ANX005 Phase 3 population had moderate to severe disease, and the matching level demonstrates that the Phase 3 population is represented within the global GBS patient spectrum captured in IGOS.


Working in collaboration, the IGOS investigators and Annexon established a cohort of 79 real-world patients from the IGOS global patient registry that was matched based on key prespecified prognostic factors to the cohort of 79 patients treated with ANX005 30 mg/kg from Annexon's completed Phase 3 study conducted outside the United States. Patients in the ANX005 Phase 3 population had moderate to severe disease, and the matching level demonstrates that the Phase 3 population is represented within the global GBS patient spectrum captured in IGOS.



Patients treated with ANX005 showed faster and greater improvement in muscle strength and disability compared to patients in the matched IGOS cohort treated with IVIg or PE. The comparison also showed that fewer patients treated with ANX005 required mechanical ventilation. Further, ANX005-treated patients were observed to spend less time on ventilation and less time in the intensive care unit (ICU). These findings indicate that ANX005 may decrease the overall burden of GBS care.


Patients treated with ANX005 showed faster and greater improvement in muscle strength and disability compared to patients in the matched IGOS cohort treated with IVIg or PE. The comparison also showed that fewer patients treated with ANX005 required mechanical ventilation. Further, ANX005-treated patients were observed to spend less time on ventilation and less time in the intensive care unit (ICU). These findings indicate that ANX005 may decrease the overall burden of GBS care.



"We're highly encouraged by the growing body of evidence demonstrating consistent, robust effects of ANX005 treatment across the Phase 3 trial and in this real-world study of patients with GBS," said Douglas Love, president and chief executive officer of Annexon. "We look forward to discussing these data and our overall regulatory package with regulators as we prepare for our planned U.S. Biologics License Application submission in the first half of 2025."


"We're highly encouraged by the growing body of evidence demonstrating consistent, robust effects of ANX005 treatment across the Phase 3 trial and in this real-world study of patients with GBS," said Douglas Love, president and chief executive officer of Annexon. "We look forward to discussing these data and our overall regulatory package with regulators as we prepare for our planned U.S. Biologics License Application submission in the first half of 2025."



Hugh Willison, MBBS, PhD, professor emeritus of neurology, University of Glasgow and a member of the IGOS Steering Committee added: "In this analysis, patients treated with a single dose of ANX005 showed improved and more rapid benefit on muscle strength and disability over matched patients treated with multiple days of IVIg or PE. Recognizing the common role of complement biology in GBS pathogenesis, it's reasonable to expect these results could translate well to a broad population of patients with GBS."


休·威利森,MBBS,博士,格拉斯哥大學神經學名譽教授,IGOS指導委員會成員補充道:「在這項分析中,接受單劑量ANX005治療的患者在肌肉力量和殘疾方面的改善比接受多天IVIg或PE治療的匹配患者更快、更明顯。鑑於補體生物學在GBS發病機制中的共同作用,合理預期這些結果可能很好地轉化爲廣泛的GBS患者群體。」




Key findings comparing ANX005 30 mg/kg to IVIg or PE:



比較ANX005 30 mg/kg與IVIg或PE的關鍵發現:



  • By week 1, patients treated with ANX005 showed more than a 10-point improvement in muscle strength over patients treated with IVIg or PE, a clinically meaningful benefit as measured by Medical Research Council (MRC) sumscore and an indicator for future recovery potential

    1

    (

    p

    < 0.0001)

  • 在第一週,接受ANX005治療的患者在肌肉力量上比接受IVIg或PE的患者提高了超過10分,這是通過醫療研究委員會(MRC)總分評估的臨牀意義明顯的好處,也是未來恢復潛力的指標。

    1

    (

    p



  • Patients treated with ANX005 were approximately twice as likely to be in a better state of health than patients on IVIg or PE on the GBS-Disability Scale (GBS-DS) at multiple timepoints throughout the study, including at week 8, the primary endpoint for the Phase 3 trial (

    p

    = 0.0459)

  • 在整個研究過程中,接受ANX005治療的患者在GBS-殘疾量表(GBS-DS)上的健康狀態比接受IVIg或PE的患者更好,可能性大約是後者的兩倍,包括在第8周,這是第3階段試驗的主要終點(

    p

    = 0.0459)


  • Approximately half the number of patients treated with ANX005 (n=15 of 79) required mechanical ventilation compared with patients treated with IVIg or PE (n=32 of 79) (

    p

    = 0.022)

  • 接受ANX005治療的患者中,大約有一半(n=15 of 79)需要機械通氣,相比於接受IVIg或PE治療的患者(n=32 of 79)(

    p

    = 0.022)


  • ANX005-treated patients were observed to spend fewer days on mechanical ventilation and fewer days in the ICU (median of 12 fewer days for each measure,

    p

    = n.s.*)

  • 接受ANX005治療的患者在機械通氣和ICU的住院天數上均少於其他患者(每項指標的中位數少12天,

    p

    = n.s.*)


"GBS is a traumatizing disease that can affect anyone, anywhere, at any time, resulting in nerve damage, severe weakness and acute paralysis," said Lisa Butler, executive director, GBS/CIDP Foundation International. "These new data from the ANX005 real-world study support the value of ANX005 as a potential novel targeted therapy for GBS. After decades with limited treatment options, none of which are FDA-approved, the GBS community deserves a future where patients can be hopeful for a quicker recovery and better outcomes."


「GBS是一種創傷性疾病,任何人、在任何地方、任何時間都可能受到影響,導致神經損傷、嚴重虛弱和急性癱瘓,」GBS/CIDP國際基金會的執行董事Lisa Butler說道。「來自ANX005真實世界研究的新數據支持ANX005作爲GBS潛在新靶向治療的價值。在數十年裏,治療選擇有限且沒有一種獲得FDA批准之後,GBS社區理應期待一個患者能夠更加快速恢復和取得更好結果的未來。」




Conference Call and Webcast Information

Annexon management will hold a conference call and webcast today at 8:30 a.m. ET to discuss topline results from its real-world evidence (RWE) study in GBS. The dial-in number for the conference call is 1-877-407-0784 (U.S./Canada) or 1-201-689-8560 (international). The conference ID for all callers is 13750635. The live webcast and replay may be accessed by visiting Annexon's website at



電話會議和網絡直播信息

Annexon管理層將在今天上午8:30(東部時間)舉行電話會議和網絡直播,討論其在GBS中進行的真實世界證據(RWE)研究的初步結果。電話會議的撥入號碼是1-877-407-0784(美國/加拿大)或1-201-689-8560(國際)。所有撥入者的會議ID是13750635。可以通過訪問Annexon的網站訪問直播和重播。



Call me: Click here. Participants can use guest dial-in numbers above and be answered by an operator or they can click the Call me link for instant telephone access to the event (dial-out). The Call me link will be made active 15 minutes prior to scheduled start time.


給我打電話:點擊這裏。參與者可以使用上面的來賓撥入號碼並由運營商接聽,或者他們可以點擊「給我打電話」鏈接以即刻撥號參加活動(撥出)。 「給我打電話」鏈接將在預定開始時間前15分鐘激活。




About the Real-World Evidence Study Comparing ANX005 Treatment to IGOS Matched Cohort

Working in collaboration, the IGOS investigators and Annexon conducted the RWE study. The RWE study applied a well-accepted statistical method of propensity score matching to establish 1:1 cohorts of patients matched on key prespecified prognostic factors of disease severity (muscle strength and GBS disability score measured at the time of hospitalization, prior to treatment). The same analytical and statistical approaches used to measure efficacy in the Phase 3 trial were applied to assess treatment effect in the matched populations (n=79 in each cohort). IGOS is a global, prospective, observational, multicenter cohort study that enrolled 2,000 patients who were followed for one to three years. Consistent with global standards of care (SoC), patients in the IGOS registry were treated with IVIg or PE. Published literature has demonstrated that more severe patients experience less benefit from IVIg or PE, further highlighting the unmet need in this patient population.

2, 3, 4, 5



關於將ANX005治療與IGOS匹配隊列進行比較的真實世界證據研究

IGOS研究者與Annexon合作開展了真實世界證據(RWE)研究。RWE研究採用了一種被廣泛接受的傾向評分匹配統計方法,建立了在關鍵的預先設定的疾病嚴重程度預後因素(住院時測量的肌肉力量和GBS殘疾評分)上匹配的1:1患者隊列。用於評估第3期試驗中療效的相同分析和統計方法被應用於匹配人群(每個隊列79人)的治療效果評估。IGOS是一項全球前瞻性觀察性多中心隊列研究,招募了2,000名患者,並進行了爲期一到三年的跟蹤。根據全球護理標準(SoC),IGOS登記的患者接受了IVIg或市盈率(PE)治療。已發佈的文獻表明,較重的患者從IVIg或市盈率(PE)中獲益較少,進一步強調了該患者群體未滿足的需求。

2, 3, 4, 5




About ANX005

Annexon's lead investigational therapy, ANX005, is a first-of-its kind selective, targeted and rapid-acting agent designed to reduce inflammation and nerve damage by stopping C1q activity in the peripheral and central nervous systems. In GBS, ANX005 is designed to seek out C1q and prevent its binding to targets on peripheral nerves. ANX005 is administered intravenously and has been observed to act almost immediately in blocking C1q function. The aim of an effective treatment in GBS is to rapidly stop the autoimmune damage on nerve cells, allowing patients to regain muscle strength sooner and to regain independence and return to pre-illness activities. ANX005 has received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as orphan drug designation from the European Medicines Agency for the treatment of GBS.



關於ANX005

Annexon的主要研究藥物ANX005是一種首創的選擇性、靶向且快速作用的藥物,旨在通過阻止C1q在外周和中樞神經系統的活性來減少炎症和神經損傷。在GBS中,ANX005旨在尋找C1q並防止其與外周神經的靶點結合。ANX005通過靜脈給藥,已被觀察到在阻止C1q功能上幾乎立即生效。GBS中有效治療的目標是迅速停止對神經細胞的自身免疫損傷,使患者能夠更快恢復肌肉力量,恢復獨立並返回到生病前的活動。ANX005已獲得美國食品藥品監督管理局的快速通道和孤兒藥資格,以及歐洲藥品管理局對GBS治療的孤兒藥資格。




About the ANX005 Phase 3 Trial



關於ANX005三期臨牀試驗



Positive data from a previously reported Phase 3 study demonstrated statistically significant effects of 30mg/kg ANX005 treatment over placebo on multiple measures of GBS, including on the primary endpoint GBS-DS. Patients treated with ANX005 demonstrated a higher likelihood of being in a better state of health at week 1 on the GBS-DS, a benefit that was observed across the 26-week study period. Early, robust and durable treatment effects were observed, which resulted in expedited recovery and reduced days on mechanical ventilation, allowing patients to walk approximately one month earlier. ANX005 was generally well-tolerated with a safety profile similar to placebo.


來自之前報道的三期研究的積極數據表明,30mg/kg ANX005治療對多個GBS指標與安慰劑相比具有統計學顯著效果,包括主要終點GBS-DS。接受ANX005治療的患者在第一週GBS-DS中表現出更高的健康狀態,受益於整個26周的研究期間。觀察到早期、強勁和持久的治療效應,這導致恢復加快,並減少機械通氣天數,使患者能夠提前大約一個月行走。ANX005通常耐受良好,其安全性特徵與安慰劑相似。




About Guillain-Barré Syndrome (GBS)

GBS is a severe disease resulting from an acute autoantibody and classical complement-mediated attack on peripheral nerves that generally occurs post-infection in otherwise healthy persons. It is an acute, rapidly progressive neurological disease with a narrow timeframe for therapeutic intervention. GBS results in the hospitalization of more than 22,000 people annually in the U.S. and Europe. The peripheral nerve damage progresses rapidly, causing acute neuromuscular paralysis that can lead to significant morbidity, disability and mortality. Currently, there are no approved treatments for GBS in the United States. The long-term disease burden associated with GBS has led to a multi-billion-dollar annual economic cost to the U.S. healthcare system alone.



關於吉蘭-巴雷綜合徵(GBS)

GBS是一種嚴重疾病,因急性自身抗體和經典補體介導的攻擊外周神經,通常發生在感染後的健康人群中。它是一種急性、快速進展的神經系統疾病,治療干預的時間非常有限。GBS每年導致美國和歐洲超過22,000人住院。外周神經損傷迅速進展,導致急性神經肌肉癱瘓,可能導致顯著的發病率、殘疾和死亡。目前,美國尚無批准的GBS治療。GBS相關的長期疾病負擔使得美國醫療系統每年產生數十億美元的經濟成本。




About Annexon

Annexon Biosciences (Nasdaq: ANNX) is harnessing therapeutics against classical complement-driven neuroinflammation to advance potentially first-in-kind treatments for millions of people living with serious neuroinflammatory diseases of the body, brain and eye. Our novel scientific approach focuses on C1q, the initiating molecule of the classical complement cascade, which can inappropriately lead to severe tissue damage and loss. By targeting C1q, our immunotherapies are designed to stop neuroinflammatory diseases where they start. Our pipeline spans three diverse therapeutic areas – autoimmune, neurodegenerative and ophthalmic diseases – and includes targeted investigational drug candidates designed to address the unmet needs of over 8 million people worldwide. Annexon's mission is to deliver game-changing therapies to patients so that they can live their best lives. To learn more visit

annexonbio.com

.



關於Annexon

Annexon Biosciences (Nasdaq: ANNX) is harnessing therapeutics against classical complement-driven neuroinflammation to advance potentially first-in-kind treatments for millions of people living with serious neuroinflammatory diseases of the body, brain and eye. Our novel scientific approach focuses on C1q, the initiating molecule of the classical complement cascade, which CAN inappropriately lead to severe tissue damage and loss. By targeting C1q, our immunotherapies are designed to stop neuroinflammatory diseases where they start. Our pipeline spans three diverse therapeutic areas – autoimmune, neurodegenerative and ophthalmic diseases – and includes targeted investigational drug CANdidates designed to address the unmet needs of over 800萬 people worldwide. Annexon's mission is to deliver game-changing therapies to patients so that they CAN live their best lives. To learn more visit

annexonbio.com

.



*n.s. = not significant


*n.s. = 不顯著




References



參考文獻




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Walgaard, et al., 2011. Early recognition of poor prognosis in Guillain-Barré syndrome.

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Hughes RAC, Swan AV, van Doorn PA. 用於格林-巴利綜合徵的靜脈免疫球蛋白。

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Van der Meché FGA, Schmitz PIM. A randomized trial comparing intravenous immune globulin and plasma exchange in Guillain–Barré syndrome.

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Van der Meché FGA,Schmitz PIm。隨機試驗比較靜脈注射免疫球蛋白和血漿置換在格林-巴利綜合症中的應用。

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Walgaard C, Lingsma HF, Ruts L, et al. Second intravenous immunoglobulin dose in patients with Guillain-Barré syndrome with poor prognosis (SID-GBS): a double-blind, randomized, placebo-controlled trial.

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Walgaard C,Lingsma HF,Ruts L等。對預後不良的格林-巴利綜合症患者進行第二次靜脈免疫球蛋白劑量的研究(SID-GBS):一項雙盲、隨機、安慰劑對照試驗。

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2021;20(4):275-283.




Forward Looking Statements



前瞻性聲明



This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as "aim," "anticipate," "assume," "believe," "contemplate," "continue," "could," "design," "due," "estimate," "expect," "goal," "intend," "may," "objective," "plan," "positioned," "potential," "predict," "seek," "should," "suggest," "target," "on track," "will," "would" and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: ability of ANX005 to stop C1q activity; ability to bring ANX005 to patients worldwide as soon as possible; the clinical and regulatory status of ANX005; the overall treatment potential of ANX005; the ability to translate the results of the RWE study to a broad population of GBS patients; the timing and outcomes of ongoing and future interactions with regulatory bodies, including the FDA; the adequacy and sufficiency of the RWE data to support marketing application; the anticipated timeline of our planned Biologics License Application (BLA) submission in the first half of 2025; the potential therapeutic benefit of ANX005 or any other product candidates on GBS, or other autoimmune, neurodegenerative and ophthalmic diseases; potential benefit of ANX005, if approved, compared to IVIg/plasma exchange or other existing therapies; and market size for GBS and other therapeutic areas of interest for Annexon. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the potential that FDA or EU regulators may not find the data from the RWE study sufficient for filing of a BLA; the potential that FDA and comparable foreign regulatory authorities may require additional information or studies prior to the approval of ANX005; the potential that regulatory authorities may not find the results of the ANX005 trial conducted outside the United States translate to a broad population of GBS patients; the potential for any final clinical trial results to differ from preliminary or topline results; the company's history of net operating losses; the company's ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company's product candidates; the effects of public health crises on the company's clinical programs and business operations; the company's ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company's product candidates; the company's reliance on third-party suppliers and manufacturers; the outcomes of any future collaboration agreements; and the company's ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled "Risk Factors" contained in the company's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q and the company's other filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.


本新聞稿包含根據1933年《證券法》第27A條和1934年《證券交易法》第21E條的定義的前瞻性聲明。在某些情況下,您可以通過術語如「目標」、「預計」、「假設」、「相信」、「考慮」、「繼續」、「可能」、「設計」、「應有」、「估計」、「期待」、「目標」、「打算」、「也許」、「目標」、「計劃」、「定位」、「潛在」、「預測」、「尋求」、「應該」、「建議」、「目標」、「按計劃」、「將」、「會」等其他類似表達識別前瞻性聲明,這些表達是對未來事件和未來趨勢的預測或指示,或這些術語的否定或其他可比術語。除了新聞稿中包含的歷史事實以外,所有聲明均爲前瞻性聲明。這些前瞻性聲明包括但不限於關於:ANX005停止C1q活性的能力;儘快將ANX005帶給全球患者的能力;ANX005的臨牀和監管狀態;ANX005的整體治療潛力;將RWE研究結果轉化爲廣泛GBS患者群體的能力;與監管機構(包括FDA)ongoing和未來互動的時機和結果;RWE數據的充分性和足夠性以支持上市申請;在2025年上半年提交我們的生物製品許可申請(BLA)的預期時間表;ANX005或其他產品候選藥物對GBS或其他自身免疫、神經退行性和眼科疾病的潛在治療益處;如果獲得批准,相比於IVIg/血漿置換或其他現有療法ANX005的潛在益處;以及GBS及其他Annexon interest的治療領域的市場規模。前瞻性聲明不保證未來的表現,且受可能導致實際結果和事件與預期大相徑庭的風險和不確定性的影響,包括但不限於與以下方面相關的風險和不確定性:FDA或EU監管機構可能不會認爲RWE研究的數據足以提交BLA;FDA和其他國家監管機構可能需要在批准ANX005之前額外的信息或研究;監管機構可能不會認爲在美國以外進行的ANX005試驗結果能有效轉化爲廣泛的GBS患者群體;最終臨牀試驗結果可能與初步或頂線結果不同;公司淨經營虧損的歷史;公司獲取必要資本以資助其臨牀項目的能力;公司產品候選藥物的臨牀開發早期階段;公共衛生危機對公司臨牀項目和業務運營的影響;公司獲得產品候選藥物的監管批准併成功商業化的能力;公司產品候選藥物的任何不良副作用或其他特性;公司對第三方供應商和製造商的依賴;任何未來合作協議的結果;以及公司能否充分維持其產品候選藥物的知識產權。關於這些和其他風險的詳細描述請參見公司在SEC提交的《10-K年度報告》和《10-Q季度報告》中「風險因素」部分。公司在本新聞稿中所作的任何前瞻性聲明均依據1995年《私人證券訴訟改革法》及其修正案發佈,僅在本新聞稿發佈日期有效。除非法律要求,公司不承擔公開更新任何前瞻性聲明的義務,無論是由於新信息、未來事件或其他原因。




Investor Contact:

Joyce Allaire
LifeSci Advisors, LLC

jallaire@lifesciadvisors.com



投資者聯繫人:

喬伊斯·阿拉爾
生命科學顧問公司

jallaire@lifesciadvisors.com




Media Contact:

Sheryl Seapy
Real Chemistry
949-903-4750

sseapy@realchemistry.com



媒體聯繫人:

Sheryl Seapy
真實化學
949-903-4750

sseapy@realchemistry.com



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