Marvel Biosciences Announces Promising New Data From Rett Syndrome Study and Plans to Discuss Orphan and Rare Disease Status With the FDA
Marvel Biosciences Announces Promising New Data From Rett Syndrome Study and Plans to Discuss Orphan and Rare Disease Status With the FDA
Calgary, Alberta--(Newsfile Corp. - November 26, 2024) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), are pleased to share additional data from its ongoing preclinical Rett syndrome study conducted in collaboration with the iBraiN Institute. Marvel also announces plans to engage with the FDA to discuss these promising pilot results.
卡爾加里,阿爾伯塔--(資訊 corp. - 2024年11月26日) - Marvel Biosciences Corp.(tsxv:MRVL)(otcqb:MBCOF)及其全資子公司Marvel Biotechnology Inc.(統稱爲「公司」或「Marvel」)很高興分享其與iBraiN研究所合作進行的正在進行的臨床前雷特綜合症研究的額外數據。Marvel還宣佈計劃與FDA接洽,討論這些有前景的試點結果。
The study evaluated MB204, Marvel's lead compound, in comparison to Trofinetide, the only FDA and Health Canada approved treatment for Rett syndrome. Mice were treated for two weeks with either compound, and social behaviour endpoints were assessed when the treatment was ceased and weekly thereafter. New data from our collaborations reveal the MB204 exhibited sustained post-treatment benefits, surpassing Trofinetide.
該研究評估了MB204,Marvel的主要compound,與FDA和健康加拿大批准的唯一雷特綜合症治療藥物Trofinetide進行比較。小鼠接受了爲期兩週的任何compound治療,並在停止治療時及隨後每週評估社會行爲終點。來自我們合作的新數據顯示,MB204表現出持續的治療後益處,超過了Trofinetide。
Key Data Highlights:
關鍵數據亮點:
- MB204 demonstrated strong carry-over effects on multiple social behaviour endpoints, persisting for 21 days post-treatment. This new data suggests a durable post-treatment and potential neuromodulator effect.
- In contrast, Trofinetide showed minimal carry-over effects, lasting only 14 days.
- Full study results are expected in Q1 of 2025.
- MB204在多個社會行爲終點上表現出強大的延續效應,持續了治療後21天。這個新數據表明了持久的治療後效果和潛在的神經調節作用。
- 相比之下,Trofinetide表現出微弱的延續效應,僅持續了14天。
- 完整的研究結果預計將在2025年第一季度公佈。
Building on these results in two models of autism, Marvel intends to begin the process with the FDA of obtaining Orphan and/or rare disease designation for MB204 in Rett Syndrome. Such designation could provide market exclusivity, tax incentives, and priority review waivers.
在這兩個自閉症模型的基礎上,Marvel打算開始與FDA的過程,爲MB204在雷特綜合症中獲得孤兒藥和/或罕見疾病的認定。這樣的認定可以提供市場獨佔權、稅收優惠和優先審查豁免。
"MB204 continues to exceed our expectations in pre-clinical studies," said Dr. Mark Williams, Chief Science Officer of Marvel Biosciences. "MB204's long-last effects are particularly exciting and encouraging."
「MB204在臨床前研究中持續超出我們的預期,」Marvel Biosciences的首席科學官馬克·威廉姆斯博士說。「MB204的長效作用尤其令人興奮和鼓舞。」
"Alongside this program, we are advancing preclinical studies in Fragile X syndrome and recently secured a major grant to support preclinical research in Alzheimer's disease," added Rod Matheson, CEO of Marvel Biosciences. "Achieving orphan or rare disease designation for MB204 could also add significant value to our lead asset."
「在這個項目的同時,我們正在推動對脆弱X綜合徵的臨床前研究,並最近獲得了一項重大補助金,以支持對阿爾茨海默病的臨床前研究,」Marvel Biosciences的CEO Rod Matheson補充道。「獲得MB204的孤兒藥或罕見病的資格也可能爲我們的首要資產增加顯著價值。」
About Marvel Biosciences Corp.
關於奇妙生物科技公司:
Marvel Biosciences Corp., and its wholly owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company that utilizes a "drug redevelopment" approach to drug development. Historically, when a new class of drug is developed, it is optimized for a particular target, but typically only approved for a specific disease. Often, a new disease is identified which involves the same target, however, pending the remaining patent life, the originally approved drug may not have sufficient time left for it to be commercially viable to be developed for the new disease indication. Marvel develops new synthetic chemical derivatives of the original approved drug for the new disease indication. Patent protection is sought, as the new potential asset is developed by the Company. The Company believes the business model results in significantly less risk, cost and time to develop its assets compared to traditional biotechnology companies.
Marvel Biosciences公司及其全資子公司Marvel生物技術公司是加拿大卡爾加里一家臨床前階段的藥品開發生物技術公司,利用"藥品重新開發"方法進行藥物開發。歷史上,當一種新的藥物類別被開發出來時,它被優化爲特定的作用靶標,但通常只被批准用於特定的疾病。通常,會發現涉及相同作用靶標的新疾病,但原始批准的藥物可能由於專利剩餘存續期不足,不足以對新的疾病適用。Marvel爲新的疾病適應研究和開發原批准藥物的新合成化學衍生物。由公司開發的新潛在資產,我們將會尋求專利保護。該公司認爲這種商業模式與傳統生物技術公司相比,能夠更少風險、成本和時間地開發其資產。
Marvel Biotechnology Inc. has currently developed several new chemical entities, using synthetic chemical derivatives of known, off-patent drugs, that inhibit the A2a adenosine receptor with application to neurological diseases (depression & anxiety, Alzheimer's, ADHD), and the non-neurological conditions of cancer and non-alcoholic steatohepatitis. Marvel is also exploring additional undisclosed targets to expand its asset pipeline.
Marvel Biotechnology Inc.目前已經開發出幾種新化學實體,使用已知的過期藥物的合成化學衍生物來抑制A2a腺苷受體,適用於神經系統疾病(抑鬱症和焦慮症、阿爾茨海默病、ADHD)、癌症和非酒精性脂肪肝等非神經系統疾病。Marvel還在探索其他未公開的目標,以擴大其資產流水線。
Contact Information
聯繫信息
Marvel Biosciences Corp.
J. Roderick (Rod) Matheson, Chief Executive Officer or
Dr. Mark Williams, President and Chief Science Officer
Tel: 403 770 2469
Marvel Biosciences Corp.
J. Roderick(Rod) Matheson,首席執行官。
馬克·威廉姆斯博士,總裁和首席科學官
電話:403 770 2469
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this press release.
無論是TSX Venture交易所還是其監管服務提供商(如TSXV政策中所定義的那樣),都不對本新聞稿的充分性或準確性負責。
All information contained in this news release with respect to the Company and its subsidiary, (collectively, the "Parties") were supplied by Marvel, respectively, for inclusion herein and each parties' directors and officers have relied on each other for any information concerning such Party.
本新聞發佈中所有有關公司及其子公司(統稱「各方」)的信息均由Marvel分別提供,供本新聞發佈納入,並且各自的董事和高管人員都僅就有關方面相互依賴獲得了信息。
This news release may contain forward-looking statements and other statements that are not historical facts. Forward-looking statements are often identified by terms such as "will", "may", "should", "anticipate", "expects" and similar expressions. All statements other than statements of historical fact, included in this release, including, without limitation, statements regarding the future plans and objectives of the Company are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those anticipated in such statements. Important factors that could cause actual results to differ materially from the expectations of the Company and include other risks detailed from time to time in the filings made by the Company under securities regulations.
本新聞發佈可能包含前瞻性聲明和其他陳述,而不是歷史事實。前瞻性聲明通常用諸如「將」、「可能」、「應該」、「預期」和類似表達方式來識別。本發佈中包含的所有陳述,除歷史事實陳述外,包括但不限於有關公司未來計劃和目標的陳述,都是有風險和不確定性的前瞻性聲明。不保證這類聲明會證明是準確的,實際結果和未來事件可能會與這類聲明所預期的有所不同。可能導致實際結果與公司預期不同的重要因素包括本新聞發佈所述各方的風險以及其他風險和不確定性,其許多情況超出公司的控制範圍。因此,公司不能保證上述事件將按照此處或全部披露的條款發生,也不能保證時間的履行。本新聞發佈中包含的前瞻性聲明明確受到此警告聲明的約束。本新聞發佈中包含的前瞻性聲明是截至本新聞發佈日期所述的,公司將根據加拿大證券法的規定明確更新或修訂包含在其中的任何前瞻性聲明。
The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. As a result, the Company cannot guarantee that the above events on the terms will occur and within the time disclosed herein or at all. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company will update or revise publicly any of the included forward-looking statements as expressly required by Canadian securities law.
讀者應注意,用於準備任何前瞻性信息的假設可能被證明是不正確的。由於衆所周知的和未知的風險、不確定性和其他因素,許多情況會導致預測結果與實際結果不同。因此,公司無法保證上述事件將按照此處或全部披露的條款發生,也無法保證時間的履行。讀者應謹慎,不要過度依賴任何前瞻性信息。儘管管理層在準備時認爲這些信息是合理的,但實際結果可能證明是不正確的。本新聞發佈中含有的前瞻性聲明受到此警示聲明的明確限制,本新聞發佈中含有的前瞻性聲明是截至本新聞發佈日期所述的,公司將根據加拿大證券法的規定明確更新或修訂包含在其中的任何前瞻性聲明。