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.
纽约，2024年5月13日（GLOBE NEWSWIRE）——通过新的药物递送途径开发突破性免疫调节疗法的生物技术公司Tiziana Life Sciences, Ltd.（纳斯达克股票代码：TLSA）（“Tiziana” 或 “公司”）今天宣布，它已提交美国食品药品管理局申请，要求获得用于治疗非活性继发性进行性多发性硬化的鼻内福鲁单抗的孤儿药认定（NA-SPMS）。这一要求将使foralumab成为第一种获得孤儿药称号的Na-spms疗法。我们的要求得到了Foralumab在NA-SPMS中的有效性的临床和非临床证据的支持。患病率估计值在一定程度上得到了马萨诸塞州波士顿布里格姆妇女医院的纵向研究的支持，该研究的CLIMB数据允许估计人群中的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是一种完全人源性的抗CD3单克隆抗体，是一种生物候选药物，已被证明在鼻内给药时能刺激T调节细胞。[1] 目前，一项开放标签的中等规模扩大准入（EA）计划已为10名NA-SPM患者服药，FDA最近还允许另外20名患者加入该计划。该扩大准入计划中的所有患者在使用foralumab的治疗后均已改善或稳定下来。70％的患者在接受六个月的治疗后疲劳有所改善。疲劳是许多多发性硬化症患者的一种使人衰弱的症状，由改进的疲劳冲击量表（MFIS）来衡量。所有患者的关键临床指标均未下降。此外，一项2a期随机、双盲、安慰剂对照、多中心、剂量范围试验（NCT06292923）正在研究鼻内foralumab，并计划于2025年读出数据。
"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董事长、代理首席执行官兼创始人Gabriele Cerrone评论说：“美国食品药品管理局向旨在治疗罕见疾病或病症的药物或生物制剂授予孤儿药称号，其定义是影响美国少于20万人的疾病或病症。”“如果该产品最终获得指定适应症的批准，孤儿药指定允许长达七年的上市独家经营权，并提供其他经济激励措施以协助开发的机会。因此，它为我们的公司和股东带来了巨大的价值。” 他总结道。
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.
申请提交后，将由美国食品药品管理局的孤儿药指定计划进行审查，该计划确定孤儿药指定批准的所有标准是否得到满足。孤儿药指定计划将在收到申请后的90天内对申请进行审查。
About Foralumab
关于 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 细胞在炎症过程中起着重要作用。Foralumab是唯一一种完全人源的抗CD3单克隆抗体（mAb），它与T细胞受体结合并通过调节T细胞功能来抑制炎症，从而抑制多个免疫细胞亚群中的效应器特征。这种效果已在COVID和多发性硬化症患者以及健康的正常受试者中得到证实。非活性SPMS鼻内foralumab 2期试验于2023年11月开始筛查患者。鼻腔抗CD3 mAb的免疫调节是治疗神经炎症和神经退行性人类疾病的新途径。[1]，[2]
About Tiziana Life Sciences
关于 Tiziana 生命科学
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是一家临床阶段的生物制药公司，使用变革性药物递送技术开发突破性疗法，以实现免疫疗法的替代途径。与静脉注射（IV）给药相比，Tiziana的创新鼻腔治疗有可能提高疗效、安全性和耐受性。Tiziana的主要候选药物鼻内foralumab是唯一一种全人源抗CD3单克隆抗体，迄今为止在研究中已在患者中表现出良好的安全性和临床反应。Tiziana的替代免疫疗法途径技术已获得专利，有多项应用在等待中，预计将允许广泛的在研应用。
For further inquiries:
如需进一步查询：
Tiziana Life Sciences Ltd
Paul Spencer, Business Development and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com
Tiziana 生命科学有限公司
保罗·斯宾塞，业务发展和投资者关系
+44 (0) 207 495 2379
电子邮件：info@tizianalifesciences.com
Investors:
Irina Koffler
LifeSci Advisors, LLC
646.970.4681
ikoffler@lifesciadvisors.com
投资者：
伊琳娜·科夫勒
LifeSci 顾问有限公司
646.970.4681
ikoffler@lifesciadvisors.com
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译文内容由第三方软件翻译。