European Medicines Agency (EMA) Grants Orphan Drug Designation for Moleculin's Treatment of Acute Myeloid Leukemia (AML)
European Medicines Agency (EMA) Grants Orphan Drug Designation for Moleculin's Treatment of Acute Myeloid Leukemia (AML)
– Next-generation non-cardiotoxic anthracycline Annamycin with notable signs of clinical efficacy in AML in combination with Cytarabine achieves preliminary CRc rate of 60% in 2nd line AML subjects in a European clinical trial
— 下一代無心臟毒性蒽環素安那黴素在急性髓細胞白血病中具有明顯的臨床療效跡象，與阿糖胞苷聯合使用，初步的cRC率爲60％和 歐洲臨床試驗中的急性髓細胞白血病受試者
– There are approximately 160,000 people with AML worldwide
— 全球大約有 160,000 名反洗錢患者
– Annamycin continues to show no signs of cardiotoxicity (N=82 across multiple studies); Lower toxicity profile than traditional intensive therapy
— 安那黴素仍然沒有心臟毒性跡象（多項研究中N=82）；毒性特徵低於傳統的強化療法
– Annamycin is advancing towards pivotal AML study in 2024 and may qualify for an accelerated approval pathway
— 安那黴素正朝着2024年的關鍵反洗錢研究邁進，可能有資格進入加速批准途徑
HOUSTON, April 18, 2024 — Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to Annamycin for the treatment of AML.
休斯頓，2024 年 4 月 18 日 — Moleculin Biotech有限公司，（納斯達克股票代碼：MBRX）（Moleculin或公司）是一家臨床階段的製藥公司，擁有廣泛的針對難以治療的腫瘤和病毒的候選藥物組合。該公司今天宣佈，歐洲藥品管理局（EMA）已授予用於治療急性髓細胞白血病的安那黴素孤兒藥稱號。
Annamycin is the Company's next-generation anthracycline that has been designed to be non-cardiotoxic (unlike currently prescribed anthracyclines) and has shown to be non-cardiotoxic in the 82 subjects treated in multiple studies in the U.S. and in Europe. Furthermore, Annamycin has recently shown in Moleculin's European clinical study for the treatment of AML using Annamycin in combination with Cytarabine (MB-106) a preliminary 60% composite complete response (CRc) rate in 2nd line subjects (N=10) and an overall interim CRc of 39% in all subjects (N=18), regardless of the prior number of therapies, in the European trial. Durability of the CRc's is developing. One subject has surpassed the one-year mark with a durable complete response. Recruitment in MB-106 has ended for 2nd line subjects while recruitment for 1st line and 3rd line subjects continue. Annamycin is currently in development for the treatment for AML and STS lung mets, and the Company believes the drug may have the potential to treat additional indications.
安那黴素是公司的下一代蒽環素，被設計爲無心臟毒性（與目前處方的蒽環類藥物不同），在美國和歐洲的多項研究中治療的82名受試者中，安那黴素已被證明對無心臟毒性。此外，安那黴素最近在Moleculin的歐洲臨床研究中顯示，使用安那黴素與阿糖胞苷（MB-106）聯合治療急性髓細胞白血病的初步複合完全反應（cRC）率爲60％和 在歐洲試驗中，直線受試者（N=10），所有受試者（N=18）的總體中期cRC爲39％（N=18）。cRC 的耐久性正在發展。一個受試者已經超過一年大關，得到了持久的全面回應。MB-106 的 2 人招募已經結束和 招生 1 名時直線科目st 直線和 3第三方 直線主題仍在繼續。安那黴素目前正在開發用於治療急性髓細胞白血病和性病肺炎的治療，該公司認爲該藥物可能有可能治療其他適應症。
"We are pleased to receive Orphan Drug Designation from the EMA for Annamycin, which further supports our ongoing efforts to advance this compelling next-generation anthracycline for the treatment of hard-to-treat cancers," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Combined with the Orphan Drug Designation we already have in the US and with the new composition of matter patent just awarded by the US Patent and Trademark Office with coverage through 2040, we believe the commercial exclusivity of Annamycin is now very well protected. We continue to be encouraged by the growing body of promising interim clinical data demonstrated by Annamycin. We remain focused on advancing our clinical and regulatory strategies toward our next phase of development, including the planned commencement of a pivotal registration study as a 2nd line therapy in AML before year end."
Moleculin董事長兼首席執行官沃爾特·克萊普評論說：“我們很高興獲得EMA頒發的安那黴素孤兒藥稱號，這進一步支持了我們爲推進這種引人注目的下一代蒽環類藥物用於治療難以治療的癌症所做的持續努力。”“結合我們在美國已經獲得的孤兒藥稱號，再加上美國專利和商標局剛剛授予的涵蓋期至2040年的新的物質專利組合，我們認爲安那黴素的商業排他性現在得到了很好的保護。安那黴素顯示出越來越多的令人鼓舞的中期臨床數據，這繼續令我們感到鼓舞。我們將繼續專注於推進下一階段開發的臨床和監管戰略，包括計劃啓動一項名爲 2 的關鍵註冊研究和 年底前對急性髓細胞白血病進行線路治療。”
The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of potential market exclusivity in the European Union if approved.
EMA向旨在治療、診斷或預防罕見、危及生命或慢性衰弱的疾病或病症的藥物和生物製劑授予孤兒藥資格，這些疾病或病症影響歐盟每10,000人中不到5人。孤兒認定爲公司提供了某些好處，包括降低監管費用、臨床方案援助、研究補助金，以及獲得批准後在歐盟長達10年的潛在市場獨家經營權。
Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory AML, in addition to Orphan Drug Designation for the treatment of STS lung mets.
安那黴素目前已獲得美國食品藥品監督管理局頒發的用於治療復發或難治性急性髓細胞白血病的快速通道狀態和孤兒藥認定，此外還有用於治療STS肺炎的孤兒藥認定。
About Moleculin Biotech, Inc.
關於 Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs, for hard-to-treat tumors and viruses. The Company's lead program, Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. All interim and preliminary data discussed above is subject to change.
Moleculin Biotech, Inc. 是一家臨床階段的製藥公司，針對難以治療的腫瘤和病毒的產品線不斷擴大，包括二期臨床項目。該公司的主要項目安那黴素是下一代蒽環素，旨在避免多藥耐藥機制，幾乎沒有心臟毒性。安那黴素目前正在開發中，用於治療復發或難治性急性髓系白血病（AML）和軟組織肉瘤（STS）肺轉移。上面討論的所有中期和初步數據可能會發生變化。
Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers, and WP1220, an analog to WP1066, for the topical treatment of cutaneous T-cell lymphoma. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as cancer indications including brain tumors, pancreatic and other cancers.
此外，該公司正在開發免疫/轉錄調節劑 WP1066，一種免疫/轉錄調節劑，能夠抑制p-stat3和其他致癌轉錄因子，同時刺激針對腦腫瘤、胰腺和其他癌症的自然免疫反應，以及用於局部治療皮膚 T 細胞淋巴瘤的 WP1066 類似物 WP1220。Moleculin 還參與開發抗代謝藥物產品組合，包括用於潛在病毒治療的 WP1122，以及包括腦腫瘤、胰腺癌和其他癌症在內的癌症適應症。
For more information about the Company, please visit www.moleculin.com and connect on Twitter, LinkedIn and Facebook.
如需了解有關公司的更多信息，請訪問 www.moleculin.com 然後連接 推特， 領英 和 Facebook。
Forward-Looking Statements
前瞻性陳述
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the timing of the commencement of a pivotal registration study of Annamycin as a 2nd line therapy in AML before year end. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
本新聞稿中的一些陳述是1933年《證券法》第27A條、1934年《證券交易法》第21E條和1995年《私人證券訴訟改革法》所指的前瞻性陳述，涉及風險和不確定性。本新聞稿中的前瞻性陳述包括但不限於開始對安那黴素作爲 2 的關鍵註冊研究的時機和 年底前對急性髓細胞白血病進行線路治療。儘管Moleculin認爲，截至發佈之日，此類前瞻性陳述中反映的預期是合理的，但事實證明，預期可能與此類前瞻性陳述所表達或暗示的結果存在重大差異。Moleculin試圖通過包括 “相信”、“估計”、“預期”、“計劃”、“項目”、“打算”、“潛力”、“可能”、“可能”、“可能”、“將”、“應該”、“大約” 或其他表示未來事件或結果不確定性的詞語來識別這些前瞻性陳述。這些陳述只是預測，涉及已知和未知的風險、不確定性和其他因素，包括在項目1A下討論的因素。我們最近向美國證券交易委員會（SEC）提交的10-K表格中的 “風險因素”，並在我們的10-Q表文件和向美國證券交易委員會提交的其他公開文件中不時更新。本新聞稿中包含的任何前瞻性陳述僅代表截至發佈日期。我們沒有義務更新本新聞稿中包含的任何前瞻性陳述，以反映其發佈日期之後發生的事件或情況，也沒有義務反映意外事件的發生。
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
MBRX@jtcir.com
投資者聯繫人：
JTC Team, LLC
珍妮·托馬斯
(833) 475-8247
MBRX@jtcir.com
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SOURCE Moleculin Biotech, Inc.
來源 Moleculin Biotech, Inc.
譯文內容由第三人軟體翻譯。