share_log

Updated Results of Phase 3 ALLELE Study Presented at 66th American Society of Hematology Annual Meeting Confirm Efficacy, Safety and Durability of Novel Allogeneic Cell Therapy Tabelecleucel in Relapsed or Refractory Epstein-Barr Virus Positive...

Updated Results of Phase 3 ALLELE Study Presented at 66th American Society of Hematology Annual Meeting Confirm Efficacy, Safety and Durability of Novel Allogeneic Cell Therapy Tabelecleucel in Relapsed or Refractory Epstein-Barr Virus Positive...

更新的第三階段ALLELE研究結果在第66屆美國血液學會年會上發佈,確認了新型異體電芯治療Tabelecleucel在復發或難治性埃潑斯坦-巴爾病毒陽性患者中的療效、安全性和持久性...
PR Newswire ·  12/07 12:33

Updated Results of Phase 3 ALLELE Study Presented at 66th American Society of Hematology Annual Meeting Confirm Efficacy, Safety and Durability of Novel Allogeneic Cell Therapy Tabelecleucel in Relapsed or Refractory Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease (EBV+ PTLD)

第66屆美國血液學會年會上發佈的第3期ALLELE研究更新結果確認了新型異基因細胞療法Tabelecleucel在複發性或難治性EBV陽性移植後淋巴增殖性疾病(EBV+ PTLD)中的療效、安全性和持久性。

Tabelecleucel treated relapsed or refractory EBV+ PTLD patients achieved a 50.7% Objective Response Rate (ORR), 23.0-month Median Duration of Response (DOR), and Median Overall Survival (OS) of 18.4 months

接受Tabelecleucel治療的複發性或難治性EBV+ PTLD患者實現了50.7%的客觀緩解率(ORR),23.0個月的緩解持續時間(DOR),以及18.4個月的中位總生存期(OS)。

Safety findings consistent with prior studies with no reports of cytokine release syndrome, tumor flare reactions, immune effector cell-associated neurotoxicity syndrome, and organ rejection,

安全性結果與先前研究一致,未報告細胞因子釋放綜合徵、腫瘤放大反應、免疫效應細胞相關神經毒性綜合徵和器官排斥等情況。

Biologics license application (BLA) under priority review by U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2025

生物製品許可申請(BLA)正在接受美國食品藥品監督管理局(FDA)的優先審查,預計於2025年1月15日前完成處方藥用戶費法案(PDUFA)目標行動日期。

If approved, tabelecleucel would be the first approved allogeneic, off-the-shelf, T-cell therapy in U.S. and only FDA-approved treatment for R/R EBV+ PTLD an ultra-rare, acute, and potentially deadly hematologic malignancy that occurs after life-saving transplantation

如果獲批,tabelecleucel將成爲美國首個獲批的異基因現貨萬億細胞療法並且是R/R EBV+ PTLD的唯一FDA批准治療方案。 這是一種在生命拯救移植後發生的超罕見、急性且潛在致命的血液惡性腫瘤。

PARSIPPANY, N.J., Dec. 7, 2024 /PRNewswire/ -- Pierre Fabre Pharmaceuticals Inc., today announced updated results presented at the 66th American Society of Hematology (ASH) Annual Meeting of the pivotal Phase 3 ALLELE study of tabelecleucel in adults and children two years of age and older with relapsed or refractory (R/R) EBV+ PTLD following solid organ transplant (SOT) or hematopoietic cell transplant (HCT). The oral presentation titled "Updated Clinical Results: A Multicenter, Open-Label, Phase 3 Study of Tabelecleucel for Solid Organ or Allogeneic Hematopoietic Cell Transplant Recipients with Epstein–Barr Virus-Driven Post Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab Plus Chemotherapy," confirmed the efficacy, safety and durability of responses observed in prior studies.

2024年12月7日,新澤西州帕西帕尼/ -- Pierre Fabre Pharmaceuticals Inc. 今日宣佈,針對接受實體器官移植(SOT)或造血幹細胞移植(HCT)後復發或難治的成年人和兩歲及以上兒童的EBV+ PTLD(R/R)患者進行的第3期重要ALLETELE研究的更新結果在第66屆美國血液學會(ASH)年會上發佈。口頭報告題爲「更新的臨床結果:Tabelecleucel用於Rituximab或Rituximab Plus 化療方案失敗後實體器官或異基因造血幹細胞移植術後EB病毒驅動的移植後淋巴增殖性疾病的多中心、開放標籤、第3期研究」,確認了先前研究中觀察到的療效、安全性和持久性反應。

"These clinically meaningful results highlight the potential of tabelecleucel in improving survival for R/R EBV+ PTLD patients, who after undergoing a potentially life-saving solid organ or hematopoietic cell transplant suddenly face yet another life-threatening illness. Currently, these patients have no FDA-approved treatment options and experience poor overall survival of only weeks to a few months following the failure of first-line treatment," said presenter and clinical investigator, Armin Ghobadi, MD, Professor of Medicine and Clinical Director of the Center for Gene and Cellular Immunotherapy at Washington University in St. Louis. "The updated data confirm the potential of tabelecleucel as a practice changing advance in addressing the significant unmet need in EBV+ PTLD."

「這些具有臨床意義的結果突顯了Tabelecleucel在改善R/R EBV+ PTLD患者生存機會方面的潛力。這些患者在潛在拯救生命的實體器官或造血幹細胞移植後突然面臨着另一種威脅生命的疾病。目前,這些患者沒有FDA批准的治療選擇,並在一線治療失敗後的幾周至幾個月內生存率很低,」報告人兼臨床調查員阿明·戈巴迪博士說,他是聖路易斯華盛頓大學基因與細胞免疫療法中心的臨床主任。 「更新的數據證實了Tabelecleucel在解決EBV+ PTLD領域重大未滿足需求中的潛力。」

The presentation of ALLELE data encompassed a larger cohort of 75 patients (49 SOT, 26 HCT) compared to the 43 patients included in the previously published data in The Lancet Oncology. The updated findings showed patients receiving tabelecleucel achieved a 50.7% objective response rate (ORR) with SOT ORR at 51.0% and HCT ORR at 50.0%. The median duration of response was 23 months, and median overall survival was 18.4 months.

allele數據的展示涵蓋了75名患者(49名SOt,26名HCT),而此前在《柳葉刀腫瘤學》發表的數據中包括了43名患者。更新的研究結果顯示,接受tabel臨售的患者達到了50.7%的客觀反應率(ORR),SOt ORR爲51.0%,HCt ORR爲50.0%。反應持續時間的中位數爲23個月,中位總生存時間爲18.4個月。

In the study, patients received a median of two cycles of therapy. Each cycle of tabelecleucel included three infusions given on days 1, 8 and 15 with an imaging assessment of efficacy around day 28. Infusions were given on an outpatient basis 67% of the time.

在這項研究中,患者平均接受了兩個療程。每個tabel臨售週期包括在第1、8和15天進行三次輸液,約在第28天進行有效性成像評估。大約67%的時間輸液是在門診基礎上進行的。

Safety findings presented were consistent with previously published data. Serious treatment emergent AEs (TEAEs) and fatal TEAEs were reported in 65.4% and 19.2% of HCT and 61.2% and 18.4% of SOT patients, respectively. No fatal TEAEs were reported by investigators to be treatment related and there were no reports of cytokine release syndrome, tumor flare or infusion reactions, immune effector cell-associated neurotoxicity syndrome, or transmission of infectious diseases. No events of graft vs host disease or organ rejection were reported as tabelecleucel related.

所呈現的安全研究結果與先前發表的數據一致。 HCt患者和SOt患者分別報告了65.4%和19.2%的嚴重治療相關的不良事件(TEAEs),以及61.2%和18.4%的死亡性TEAEs。調查人員報告稱,沒有死亡性TEAEs與治療有關,並且沒有報道細胞因子釋放綜合徵、腫瘤閃爍或輸液反應、免疫效應細胞相關神經毒性綜合徵、傳染性疾病的傳播。沒有報告tabel臨售相關的移植物抗宿主病或器官排斥事件。

"The consistency of these data enhances our confidence in the potential tabelecleucel may bring to people living with R/R EBV+ PTLD. These patients and their families face a difficult journey to undergo a potentially life-saving transplant only to be diagnosed with this very rare form of cancer," said Adriana Herrera, Chief Executive Officer of Pierre Fabre Pharmaceuticals Inc., the new Pierre Fabre Laboratories pharmaceutical subsidiary in the United States. "We look forward to the FDA target action date in January 2025 and if approved, the subsequent transfer of the BLA license from Atara Biotherapeutics, so we can bring this novel allogeneic cell therapy to people living with R/R EBV+ PTLD who urgently need new treatment options."

「這些數據的一致性增強了我們對tabel臨售可能爲患有復發/難治EBV+ PTLD的患者帶來的潛在價值的信心。這些患者及其家人經歷了進行潛在挽救生命的移植手術的艱難旅程,卻被診斷患有這種非常罕見的癌症,」Pierre Fabre製藥公司的首席執行官Adriana Herrera表示。Inc.,這家位於美國的Pierre Fabre Laboratories製藥新子公司。「我們期待2025年1月的FDA目標行動日期,如果獲得批准,將從Atara Biotherapeutics轉移BLA許可證,以便我們將這種新型同基因異體細胞療法帶給那些迫切需要新治療選擇的患有復發/難治EBV+ PTLD的患者。」

In August 2024, the FDA accepted the BLA submitted by Atara Biotherapeutics and granted priority review for tabelecleucel indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with EBV+ PTLD who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. The BLA is supported by pivotal and supportive data covering more than 430 patients treated with tabelecleucel across multiple life-threatening diseases.

2024年8月,FDA接受了atara biotherapeutics提交的BLA,併爲表達EBV+ PTLD的tabelecleucel作爲成人和兩歲及兩歲以上的兒童單藥治療而進行了優先審查,這些患者接受過至少一種先前治療。對於固體器官移植患者,先前治療包括化療,除非化療不合適。BLA得到關鍵和支持性數據支持,這些數據覆蓋了超過430名患者,這些患者接受了tabelecleucel治療,涵蓋了多種危及生命的疾病。

Tabelecleucel is an allogeneic, off-the-shelf, EBV-specific T-cell immunotherapy designed to selectively target and eliminate EBV-infected cells. Unlike autologous CAR-T therapies, allogeneic T-cells are derived from third-party donors and are not genetically modified. Immune cells are collected from the blood of healthy donors and exposed to Epstein-Barr virus antigens to help enrich for T cells that recognize EBV. These EBV T cells are expanded, characterized, kept alive and stored for future use to treat patients.

Tabelecleucel是一種異基因、零庫存、EBV特異性t細胞免疫療法,旨在有選擇地靶向和消除EBV感染的細胞。與自體CAR-t療法不同,異基因t細胞來源於第三方供體,並且不進行基因修改。免疫細胞從健康獻血者的血液中收集,並暴露於EB病毒抗原,以幫助富集識別EBV的t細胞。這些EBV t細胞被擴增、表徵、保持活性並存儲以供將來治療患者。

Tabelecleucel was granted marketing authorization under the brand name EBVALLO in December 2022 by the European Commission (EC) as a monotherapy for the treatment of adult and pediatric patients two years of age and older with r/r EBV+ PTLD who have received at least one prior therapy.

Tabelecleucel在2022年12月獲得了歐洲委員會(EC)批准,以EBVALLO品牌名授權上市,用作成人和兩歲及兩歲以上的兒童r/r EBV+ PTLD的單藥治療,這些患者接受過至少一種先前治療。

In December 2023, Atara announced an expanded global partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tabelecleucel, building on an initial partnership covering Europe, Middle East, Africa, and other select emerging markets.

2023年12月,Atara宣佈與Pierre Fabre Laboratories擴大全球合作伙伴關係,涵蓋tabelecleucel在美國及其他全球商業市場的發展,此舉基於最初在歐洲、中東、非洲和其他新興市場的合作伙伴關係。

About EBV+PTLD
EBV+ PTLD is an ultra-rare, acute, and potentially deadly hematologic malignancy that occurs after transplantation when patient T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone solid organ transplant (SOT) or allogeneic HCT. Poor median survival of 3 weeks and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patients for whom standard of care failed, underscoring the significant need for new therapeutic options.

關於EBV+ PTLD
EBV+ PTLD是一種超稀有的急性、潛在致命的造血系統惡性腫瘤,在患者進行移植後,由於免疫抑制導致患者的t細胞免疫反應受損而發生。在標準治療失敗的EBV+ PTLD患者中,HCt和SOt的中位生存期分別爲3周和4.1個月,突出了對新療法的重大需求。

About Pierre Fabre Pharmaceuticals and Pierre Fabre Laboratories
The mission of Pierre Fabre Pharmaceuticals (PFP) is to deliver breakthrough therapies in oncology and rare diseases to patient populations with high unmet needs and limited treatment options. Our belief is that every time we care for a single person, we make the whole world better.

關於Pierre Fabre Pharmaceuticals和Pierre Fabre Laboratories
Pierre Fabre Pharmaceuticals(PFP)的使命是爲患有高度未滿足需求和有限治療選擇的患者群體提供腫瘤學和罕見疾病的突破性療法。我們相信,每次爲一個人提供關懷,我們就讓整個世界變得更美好。

PFP is the US pharmaceutical subsidiary of Pierre Fabre Laboratories, a foundation-owned company with seven decades of impact. Pierre Fabre Laboratories is a global healthcare company, established in 43 countries, with 10,000 employees, and with products distributed in 120 territories across the globe.

PFP是Pierre Fabre Laboratories在美國的製藥子公司,Pierre Fabre Laboratories是一家有七十年曆史的基金所有的公司。Pierre Fabre Laboratories是一家全球醫療保健公司,在43個國家設立機構,在全球120個領土銷售產品,擁有1萬名員工。

The Pierre Fabre Laboratories foundation ownership enhances the ability of the company to create long-term value for patients. Partnerships and acquisitions drive its innovative precision treatment pipeline and are enabled by the unique corporate structure.

Pierre Fabre Laboratories基金會所有權增強了公司爲患者創造長期價值的能力。合作伙伴關係和收購推動了其創新的精準治療技術管線,並受到獨特企業結構的支持。

Building on the legacy of Pierre Fabre Laboratories, innovation is the life blood of PFP and patient experience drives everything the company does. PFP aspires to design and develop therapeutic solutions inspired by patients and healthcare professionals; draw on science and nature as perpetual sources of inspiration; develop long-term partnerships with researchers and innovators worldwide; and place pharmaceutical ethics and climate transition at the heart of our action.

在Pierre Fabre Laboratories的傳統基礎上,創新是PFP的生命線,患者體驗驅動着公司的一切。PFP的願景是設計和開發由患者和醫療保健專業人員啓發的治療方案;以科學和自然作爲永恒的啓發源;與全球研究人員和創新者建立長期合作伙伴關係;並將製藥倫理和氣候轉變置於我們行動的核心。

Pierre Fabre Pharmaceuticals has therapies in development for Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD), NRAS-mutant melanoma, non-small cell lung cancer with mutation or amplification of MET, and X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). Pierre Fabre Pharmaceuticals is headquartered in Parsippany, NJ.

Pierre Fabre Pharmaceuticals正在開發愛潑斯坦-巴爾病毒陽性移植後淋巴增生性疾病(EBV+ PTLD)、NRAS突變的黑色素瘤、具有MEt基因突變或擴增的非小細胞肺癌,以及X連鎖低汗性外胚層發育不全(XLHED)的治療療法。Pierre Fabre Pharmaceuticals總部位於新澤西州帕西帕尼。

SOURCE Pierre Fabre Pharmaceuticals

資訊 Pierre Fabre Pharmaceuticals

WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM?

想要您公司的新聞在PRNEWSWIRE.COM上特色呈現嗎?

440k+
440k+

Newsrooms &
新聞發佈室&

Influencers
影響力人士
9k+
9k+

Digital Media
數字媒體

Outlets
Outlets
270k+
270k+

Journalists
記者

Opted In
Opted In
GET STARTED
開始使用
声明:本內容僅用作提供資訊及教育之目的,不構成對任何特定投資或投資策略的推薦或認可。 更多信息
    搶先評論