Tiziana Life Sciences Files for Orphan Drug Designation for Intranasal Foralumab
Tiziana Life Sciences Files for Orphan Drug Designation for Intranasal Foralumab
NEW YORK, May 13, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) ("Tiziana" or the "Company"), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced it has submitted an FDA request to obtain Orphan Drug Designation for intranasal foralumab for the treatment of non-active secondary progressive Multiple Sclerosis (na-SPMS). This request would make foralumab the first therapy for na-SPMS to receive Orphan Drug Designation. Our request is supported by clinical and non-clinical evidence of Foralumab's effectiveness in na-SPMS. The prevalence estimates, in part, are supported from the Brigham & Women's Hospital, Boston, Massachusetts, longitudinal study, the CLIMB data of which allowed the estimate of na-SPMS in the population.
紐約，2024年5月13日（GLOBE NEWSWIRE）——通過新的藥物遞送途徑開發突破性免疫調節療法的生物技術公司Tiziana Life Sciences, Ltd.（納斯達克股票代碼：TLSA）（“Tiziana” 或 “公司”）今天宣佈，它已提交美國食品藥品管理局申請，要求獲得用於治療非活性繼發性進行性多發性硬化的鼻內福魯單抗的孤兒藥認定（NA-SPMS）。這一要求將使foralumab成爲第一種獲得孤兒藥稱號的Na-spms療法。我們的要求得到了Foralumab在NA-SPMS中的有效性的臨床和非臨床證據的支持。患病率估計值在一定程度上得到了馬薩諸塞州波士頓布里格姆婦女醫院的縱向研究的支持，該研究的CLIMB數據允許估計人群中的NA-SPM。
Foralumab, a fully human anti-CD3 monoclonal antibody, is a biological drug candidate that has been shown to stimulate T regulatory cells when dosed intranasally.[1] At present, 10 patients with na-SPMS have been dosed in an open-label intermediate-size Expanded Access (EA) Program with an additional 20 patients recently allowed to enter the program by the FDA. All patients in this expanded access program have either improved or stabilized on treatment with foralumab. 70% of patients have seen an improvement in fatigue after six months of treatment. Fatigue is a debilitating symptom for many MS patients and is measured by the Modified Fatigue Impact Scale (MFIS). None of the patients have declined in key clinical measures. In addition, intranasal foralumab is being studied in a Phase 2a, randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial (NCT06292923) with a data readout planned for 2025.
Foralumab是一種完全人源性的抗CD3單克隆抗體，是一種生物候選藥物，已被證明在鼻內給藥時能刺激T調節細胞。[1] 目前，一項開放標籤的中等規模擴大准入（EA）計劃已爲10名NA-SPM患者服藥，FDA最近還允許另外20名患者加入該計劃。該擴大准入計劃中的所有患者在使用foralumab的治療後均已改善或穩定下來。70％的患者在接受六個月的治療後疲勞有所改善。疲勞是許多多發性硬化症患者的一種使人衰弱的症狀，由改進的疲勞衝擊量表（MFIS）來衡量。所有患者的關鍵臨床指標均未下降。此外，一項2a期隨機、雙盲、安慰劑對照、多中心、劑量範圍試驗（NCT06292923）正在研究鼻內foralumab，並計劃於2025年讀出數據。
"Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the U.S.," commented Gabriele Cerrone, Chairman, acting CEO, and founder of Tiziana Life Sciences. "Orphan Drug Designation allows for up to seven years of marketing exclusivity if the product is ultimately approved for its designated indication, as well as providing the opportunity for other financial incentives to assist with development. It therefore carries significant value to our company and shareholders," he concluded.
Tiziana Life Sciences董事長、代理首席執行官兼創始人Gabriele Cerrone評論說：“美國食品藥品管理局向旨在治療罕見疾病或病症的藥物或生物製劑授予孤兒藥稱號，其定義是影響美國少於20萬人的疾病或病症。”“如果該產品最終獲得指定適應症的批准，孤兒藥指定允許長達七年的上市獨家經營權，並提供其他經濟激勵措施以協助開發的機會。因此，它爲我們的公司和股東帶來了巨大的價值。” 他總結道。
Once submitted, applications are reviewed by the FDA's Orphan Drug Designation program, which determines whether all criteria for Orphan Drug Designation approval have been met. Applications are reviewed by the Orphan Drug Designation program within 90 days of receipt.
申請提交後，將由美國食品藥品管理局的孤兒藥指定計劃進行審查，該計劃確定孤兒藥指定批准的所有標準是否得到滿足。孤兒藥指定計劃將在收到申請後的90天內對申請進行審查。
About Foralumab
關於 Foralumab
Activated T cells play an important role in the inflammatory process. Foralumab, the only fully human anti-CD3 monoclonal antibody (mAb), binds to the T cell receptor and dampens inflammation by modulating T cell function, thereby suppressing effector features in multiple immune cell subsets. This effect has been demonstrated in patients with COVID and with multiple sclerosis, as well as in healthy normal subjects. The non-active SPMS intranasal foralumab Phase 2 trial began screening patients in November of 2023. Immunomodulation by nasal anti-CD3 mAb represents a novel avenue for treatment of neuroinflammatory and neurodegenerative human diseases.[1],[2]
活化的 T 細胞在炎症過程中起着重要作用。Foralumab是唯一一種完全人源的抗CD3單克隆抗體（mAb），它與T細胞受體結合並通過調節T細胞功能來抑制炎症，從而抑制多個免疫細胞亞群中的效應器特徵。這種效果已在COVID和多發性硬化症患者以及健康的正常受試者中得到證實。非活性SPMS鼻內foralumab 2期試驗於2023年11月開始篩查患者。鼻腔抗CD3 mAb的免疫調節是治療神經炎症和神經退行性人類疾病的新途徑。[1]，[2]
About Tiziana Life Sciences
關於 Tiziana 生命科學
Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana's innovative nasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana's lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb, has demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana's technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.
Tiziana Life Sciences是一家臨床階段的生物製藥公司，使用變革性藥物遞送技術開發突破性療法，以實現免疫療法的替代途徑。與靜脈注射（IV）給藥相比，Tiziana的創新鼻腔治療有可能提高療效、安全性和耐受性。Tiziana的主要候選藥物鼻內foralumab是唯一一種全人源抗CD3單克隆抗體，迄今爲止在研究中已在患者中表現出良好的安全性和臨床反應。Tiziana的替代免疫療法途徑技術已獲得專利，有多項應用在等待中，預計將允許廣泛的在研應用。
For further inquiries:
如需進一步查詢：
Tiziana Life Sciences Ltd
Paul Spencer, Business Development and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com
Tiziana 生命科學有限公司
保羅·斯賓塞，業務發展和投資者關係
+44 (0) 207 495 2379
電子郵件：info@tizianalifesciences.com
Investors:
Irina Koffler
LifeSci Advisors, LLC
646.970.4681
ikoffler@lifesciadvisors.com
投資者：
伊琳娜·科夫勒
LifeSci 顧問有限公司
646.970.4681
ikoffler@lifesciadvisors.com
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譯文內容由第三人軟體翻譯。