Thiogenesis Announces Important Core Patent Allowed in Europe
Thiogenesis Announces Important Core Patent Allowed in Europe
San Diego, California--(Newsfile Corp. - November 4, 2024) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing novel thiol compounds, including precursors to cysteamine that have potent antioxidant and anti-inflammatory properties for mitochondrial diseases and related metabolic conditions; is pleased to announce that one of its core patents titled, "Methods For The Treatment of Cysteamine Sensitive Disorders," has been allowed by the European Patent Office (European Patent No. 18858763.8). The patent is scheduled to expire in September 2037. The new patent allowance in Europe is in addition to patents granted in this same patent family in United States in November 2021 and in Japan in 2023.
加利福尼亞州聖地亞哥--(新聞資訊公司 - 2024年11月4日)- Thiogenesis Therapeutics, Corp.(TSXV: TTI)("Thiogenesis"或"公司")是一家臨床階段的生物技術公司,開發包括半胱氨酸前體在內的新型硫醇化合物,這些化合物具有強效的抗氧化和抗炎性,用於線粒體疾病和相關代謝狀況;很高興地宣佈,其名爲"半胱氨酸敏感疾患治療方法"的核心專利之一已獲得歐洲專利局批准(歐洲專利號18858763.8)。該專利將於2037年9月到期。歐洲的新專利授權是在2021年11月在美國和2023年在日本同一專利家族中授予的專利的補充。
About TTI-0102 and Cysteamine
有關TTI-0102和半胱氨酸
Thiogenesis' lead compound, TTI-0102, is a new chemical entity (NCE) that is an asymmetric disulfide consisting of two different thiols, moreover it is a prodrug that is metabolized into the thiol cysteamine. Thiols, like TTI-0102 and cysteamine, have a functional SH group (containing sulfur and hydrogen) and are versatile bio-active molecules that are known to be involved in a number of biochemical reactions and metabolic processes, making them promising candidates for a number of therapeutic applications.
Thiogenesis的主導化合物TTI-0102是一種新的化學實體(NCE),是由兩種不同的硫醇組成的不對稱二硫化物,此外,它是一種代謝成胺基硫醇半胱氨酸的前體。像TTI-0102和半胱氨酸這樣的硫醇具有功能性SH基團(含硫和氫) ,是多功能的生物活性分子,已知參與多種生化反應和代謝過程,使其成爲多種治療應用的有希望的候選藥物。
In particular, certain thiols like cysteamine are precursors to the critical antioxidant glutathione. Glutathione is considered one of the most important antioxidants because it: is uniquely transported into the mitochondria, effectively neutralizes free radicals in the mitochondria and enhances the overall antioxidant defense system. As a result, TTI-0102 has the potential to significantly reduce oxidative stress in the mitochondria - the 'powerplant' of the cell that produces both ATP energy to power cells and volatile free radicals as a by-product. Oxidative stress occurs when there is an imbalance of too many free radicals versus antioxidants in the mitochondria, which can damage cells and exacerbate disease. Oxidative stress is a hallmark of many chronic diseases such as inherited mitochondrial diseases and related metabolic disorders such as fatty liver disease, diabetes and obesity.
特別是,像半胱氨酸這樣的某些硫醇是抗氧化劑谷胱甘肽的前體。谷胱甘肽被認爲是最重要的抗氧化劑之一,因爲它:獨特地被運輸到線粒體,有效中和線粒體中的自由基,並增強整體抗氧化防禦系統。因此,TTI-0102有潛力顯著減少線粒體中的氧化應激-細胞的「動力工廠」,產生用於爲細胞供能的ATP能量以及揮發性自由基作爲副產品。氧化應激是由於線粒體中自由基與抗氧化劑不平衡而導致的,這可能損害細胞並惡化疾病。氧化應激是許多慢性疾病的標誌,例如遺傳性線粒體疾病和相關代謝紊亂,如脂肪肝病、糖尿病和肥胖症。
TTI-0102 was developed to address the challenges facing thiol-based drugs, including their short half live, adverse side effects and dosing limitations. As a prodrug, it only becomes active after oral administration, leading to a well-tolerated and sustained release of cysteamine that can last over 24 hours. Its active moiety has been previously approved for the lysosomal disease nephropathic cystinosis. As a result, TTI-0102 is eligible for the accelerated 505 (b)(2) regulatory pathway in the U.S. and a similar hybrid pathway in the European Union.
TTI-0102是針對硫醇類藥物面臨的挑戰而開發的,包括它們短暫半衰期、不良副作用和給藥限制。作爲一種前藥,在口服後才會變得活性,導致半胱氨酸的耐受性好且持續釋放,可以持續超過24小時。其活性基團此前已獲批用於溶酶體疾病——囊性脂酰病。因此,TTI-0102符合美國的加速505(b)(2)監管途徑以及歐盟類似的混合途徑。
"We are extremely pleased to have this important European patent allowed in addition to our previously granted US and Japanese patents. They provide the Company with significant long term IP protection of our novel thiol compounds, just as we are anticipating the initiation of Phase 2 clinical trials in MELAS, Leigh syndrome and pediatric MASH," said Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "TTI-0102 has the potential to be the best-in-class method of generating intracellular glutathione and has the promise of yielding meaningful clinical benefits for patients with diseases that have a strong oxidative stress component."
"我們非常高興獲得這一重要的歐洲專利許可,此外我們之前獲得的美國和日本專利。它們爲公司提供了對我們創新硫醇化合物的重要長期知識產權保護,正值我們預計即將啓動MELAS、Leigh綜合症和兒童MASH第2期臨床試驗之際,"Thiogenesis首席執行官Patrice Rioux醫學博士、博士稱。"TTI-0102有望成爲產生胞內谷胱甘肽的最佳方法,並有望爲患有強氧化應激成分疾病的患者帶來有意義的臨床益處。"
About MELAS
關於MELAS
Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS") is a rare, inherited mitochondrial disorder caused by a mutation in mitochondrial DNA. Initial symptoms include seizures, vomiting, muscle weakness, and fatigue; longer term the disease may cause a loss of motor skills and intellectual disability. Oxidative stress plays an important role in mitochondria and is a potential pathological mechanism of mitochondrial disease, making it a viable target for the treatment of MELAS and other mitochondrial diseases. The prevalence of MELAS is not well understood; however, it has been estimated to occur in approximately 15,000 patients in the US and up to 20,000 in the EU.
線粒體腦肌病伴乳酸性酸中毒和類似卒中發作("MELAS")是一種罕見的遺傳線粒體疾病,由線粒體DNA突變引起。早期症狀包括抽搐、嘔吐、肌無力和疲勞;疾病的長期症狀可能導致運動能力喪失和智力障礙。氧化應激在線粒體中起着重要作用,是線粒體疾病的潛在病理機制,使其成爲MELAS和其他線粒體疾病治療的可行靶點。MELAS的患病率尚不清楚;然而,據估計在美國約有約15,000名患者,歐盟高達20,000名。
About Leigh Syndrome
關於Leigh綜合症
Leigh syndrome is a rare inherited genetic disease that results from the disruption of normal mitochondrial function, it is usually diagnosed in infancy and affects an estimated 1/40,000 births. Initial symptoms for Leigh syndrome include impaired or weak sucking/breastfeeding capability, loss of motor and communication skills, respiratory issues, poor muscle development, loss of appetite and seizures. There is currently no cure for Leigh syndrome, oxidative stress is a distinguishing feature of the disease.
Leigh綜合症是一種罕見的遺傳性疾病,由於正常線粒體功能受到干擾而導致,通常在嬰兒期診斷,約每4萬次分娩中有1例患病。Leigh綜合症的早期症狀包括吸吮/哺乳能力受損或弱、失去運動和交流技能、呼吸問題、肌肉發育不良、食慾減退和抽搐。目前尚無Leigh綜合症的治癒方法,氧化應激是該疾病的一個顯著特徵。
About Pediatric MASH
關於兒科MASH
Pediatric Metabolic Dysfunction-Associated Steatohepatitis (MASH) is a disease of the liver in children and is a more severe form of pediatric Nonalcoholic Fatty Liver Disease ("NAFLD"). It is often linked to obesity and characterized by inflammation, accumulation of fat and scarring (fibrosis) in the liver. There are estimated to be well-over 1,000,000 children in the U.S. with pediatric MASH, for which there are no approved drugs or treatments.
兒科代謝性功能障礙相關脂肪肝炎(MASH)是兒童肝臟疾病的一種更嚴重形式,通常與肥胖有關,特徵是肝臟中的炎症、脂肪積累和瘢痕(纖維化)。據估計,美國有超過100萬兒童患有兒科MASH,目前尚無批准的藥物或治療方法。
About Thiogenesis
關於Thiogenesis
Thiogenesis Therapeutics, Corp. (TSXV: TTI) is a clinical-stage biopharmaceutical company operating through its wholly owned subsidiary based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Prodrugs are drugs that contain previously approved active ingredients and are modified so that they only become active when metabolized. For regulatory purposes prodrugs can use existing third-party safety data in regulatory submissions in the streamlined 505 (b)(2) regulatory pathway in the U.S., and its equivalent hybrid system in Europe, to proceed into human efficacy trials with regulatory approval. Prodrugs may enhance the profile of the active ingredient to increase its bioavailability and reduce side effects. The Company's initial target indications include MELAS, Leigh syndrome, pediatric MASH and Rett syndrome.
Thiogenesis Therapeutics, Corp.(TSXV: TTI)是一家臨床階段的生物製藥公司,通過其位於加利福尼亞州聖迭戈的全資子公司運作。該公司在TSX創業公司上市。Thiogenesis正在開發含硫代韋髓醇的前藥,作爲之前批准的硫化物化合物的前體,有潛力治療嚴重的兒科疾病,臨床需求尚未滿足。前藥是包含先前批准的活性成分的藥物,並經過修改,只有在代謝後才會變得活性。出於監管目的,前藥可以在美國的簡化505(b)(2)監管途徑中使用現有的第三方安全數據進行監管申報,在歐洲採用其等效的混合制度,獲得監管批准後進入人類有效性試驗。前藥可能改善活性成分的特性,增加其生物利用度並減少副作用。公司初步目標適應症包括MELAS、Leigh綜合徵、兒科MASH和Rett綜合徵。
For further information, please contact:
如需更多信息,請聯繫:
Brook Riggins, Director, and CFO
董事兼CFO Brook Riggins
Email: info@thiogenesis.com
Tel.: (888) 223-9165
電子郵件:info@thiogenesis.com
電話:(888) 223-9165
Forward Looking Statements
前瞻性聲明
This news release contains certain forward-looking statements and forward-looking information (collectively referred to herein as "forward- looking statements") within the meaning of Canadian securities laws including, without limitation, statements with respect to the future investments by the Company. All statements other than statements of historical fact are forward-looking statements. Undue reliance should not be placed on forward-looking statements, which are inherently uncertain, are based on estimates and assumptions, and are subject to known and unknown risks and uncertainties (both general and specific) that contribute to the possibility that the future events or circumstances contemplated by the forward-looking statements will not occur. Although the Company believes that the expectations reflected in the forward-looking statements contained in this press release, and the assumptions on which such forward-looking statements are made, are reasonable, there can be no assurance that such expectations will prove to be correct. Readers are cautioned not to place undue reliance on forward-looking statements included in this document, as there can be no assurance that the plans, intentions, or expectations upon which the forward-looking statements are based will occur. By their nature, forward-looking statements involve numerous assumptions, known and unknown risks and uncertainties that contribute to the possibility that the predictions, forecasts, projections and other forward-looking statements will not occur, which may cause the Company's actual performance and results in future periods to differ materially from any estimates or projections of future performance or results expressed or implied by such forward-looking statements. The forward-looking statements contained in this news release are made as of the date hereof and the Company does not undertake any obligation to update publicly or to revise any of the included forward-looking statements, except as required by applicable law. The forward-looking statements contained herein are expressly qualified by this cautionary statement.
本新聞稿包含某些前瞻性聲明和前瞻性信息(統稱爲"前瞻性聲明"),根據加拿大證券法,其中包括但不限於公司未來的投資。所有除歷史事實陳述之外的陳述均爲前瞻性聲明。不應過度依賴前瞻性陳述,這些陳述是本質上不確定的,基於估計和假設,並受到已知和未知的風險和不確定性(無論是一般還是具體),這些風險和不確定性有助於可能導致前瞻性陳述所考慮的未來事件或情況未發生。儘管公司認爲本新聞稿中所包含的前瞻性陳述反映出理性,即製作這種前瞻性陳述的假設是合理的,但不能保證這些期望將證明正確。讀者被告知不要將其納入本文件中所包含的前瞻性陳述,因爲無法保證所基於的計劃、意圖或期望將發生。根據其本質,前瞻性陳述涉及許多假設、既知和未知的風險和不確定性,這些因素有助於可能導致前瞻性陳述、預測、投影和其他前瞻性陳述未發生,這可能導致公司未來時期的表現和結果與任何估計或預測的未來表現或結果(無論是暗示或明示地表達)不一致。本新聞稿中包含的前瞻性陳述是截至本項聲明之日做出的,公司不承擔任何更新或修訂所包含的前瞻性陳述和向期望進行或基於其中一個目的而作出的任何變化的義務,除非適用法律要求。本新聞稿中包含的前瞻性陳述應受到本警示聲明的顯式限制。
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this news release.
TSX Venture交易所及其監管服務提供方(如TSX Venture Exchange的政策所定義的)對此新聞發佈的充分性或準確性不負任何責任。
譯文內容由第三人軟體翻譯。