Riluzole

LOS ANGELES, March 31, 2025 (GLOBE NEWSWIRE) -- Trethera Corporation (“Trethera”), a clinical stage biopharmaceutical company committed to targeting cancer and autoimmune diseases through first-in-class drugs, announced clinical results from treating a patient with ALS (amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease) with TRE-515 under the FDA Expanded Access program. TRE-515, a novel drug developed by Trethera and currently in dose escalation trials for solid tumors, targets the enzyme deoxycytidine kinase (dCK) in the nucleoside salvage pathway. Figure 1. Co-crystal 3D structure of the drug bound to the target enzyme, dCK, at the deoxycytidine binding site. The patient was diagnosed with ALS in May 2022 and had undergone more than 10 different treatments, including Riluzole, the only FDA-approved drug, without success. In the four months leading up to the trial, the patient’s forced vital capacity—a key measure of lung function—declined from 60% to 37%, despite receiving multiple drugs and experimental stem cells to fight the disease. Over a three month dosing period with TRE-515, objective measures demonstrated disease stabilization, including forced vital capacity as well as the Revised ALS Functional Rating Scale (ALSFRS-R). The patient reported gain in body weight, improved neck tone, and increased arm strength over the three month period. Importantly, no adverse events occurred over the treatment duration and the multicenter medical review group voted unanimously to increase the TRE-515 dose and extend the trial. “Our laboratory research has revealed potential to treat a range of autoimmune and inflammatory diseases, including MS, Crohn’s, and lupus. This trial marks a significant milestone as the first clinical use of TRE-515 beyond cancer,” said Dr. Ken Schultz, CEO of Trethera. “Our approach has the potential to extend survival and improve quality of life for patients facing this devastating neurodegenerative disease.” “FDA approved options to treat ALS are severely limited. Exploring alternatives with first-in-class drugs for patients lacking available therapies represents the continued advancement of science,” said Dr. Frank Diaz, Assistant Professor of Neurology at Cedars-Sinai and treating neurologist. “We are pleased to work closely with Trethera in advancing this novel therapy.” “We are particularly excited about the potential for TRE-515 as an ALS treatment given its ability to selectively modulate inflammation and its favorable safety profile,” said Dr. Dan Kelly, President of the Pacific Neuroscience Institute Foundation at Saint John’s Health Center. “We look forward to continued development of this promising therapeutic candidate for patients in need of therapies that improve the course of disease.” In collaboration with Cedars-Sinai and Providence Saint John’s Health Center, the patient enrolled under the FDA Expanded Access (EA) program. This program enables patients with life-threatening conditions, such as ALS, to access investigational therapies like TRE-515 when no satisfactory alternatives exist. EA trials allow physicians and their patients to explore promising treatments under carefully designed protocols that align with the ongoing clinical development of the drug. When integrated into the drug development process, EA trials offer several benefits — including broader patient engagement, identification of potential response-predictive biomarkers, and valuable data to inform and refine clinical research. However, future randomized clinical trials with larger numbers of patients will be necessary to prove clinical benefit. About Amyotrophic Lateral Sclerosis Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive, inflammatory neurodegenerative disease impacting nerve cells in the brain and spinal cord, reducing muscle function and control. ALS can be traced to mutations in more than 20 different genes and is often caused by a combination of multiple subtypes of the condition. Cases cannot usually be predicted, although a small percentage are inherited. ALS has a devastating impact on patients and their families. ALS patients average two to three years of life expectancy after diagnosis and there is currently no cure for the disease. Sources: Lancet.2022 Oct 15;400(10360); EurJNeurol.2020 Jul 7;27(10); SurgNeuro.2015 Nov 16;6:(171). About TretheraTrethera is a clinical stage, privately held, biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera's innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug. TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors. It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases might also respond to TRE-515 treatment. Trethera is developing TRE-515 for use as a monotherapy or in combination to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients. For more information, please visit us at trethera.com or e-mail Investor Relations at ir@trethera.com. You can also follow Trethera on Facebook and LinkedIn. Note on Forward-Looking StatementsAll statements other than statements of historical facts included in this press release that address activities, events or developments that Trethera believes or anticipates will or may occur in the future are “forward-looking statements,” which may often, but not always, be identified by the use of such words as "may," "might," "will," "will likely result," "would," "should," "estimate," "plan," "project," "forecast," "intend," "expect," "anticipate," "believe," "seek," "continue," "target" or the negative of such terms or other similar expressions. Although Trethera has a reasonable basis for the forward-looking statements contained herein, Trethera cautions that such statements are based on current expectations about future events and are subject to risks, uncertainties and factors relating to medical and scientific research, all of which are difficult to predict and many of which are beyond Trethera’s control, that may cause actual results to differ materially from those expressed or implied by the forward-looking statements in this press release. These potential risks and uncertainties include, without limitation: the extent to which development of any novel cancer therapies or therapies for autoimmune diseases succeeds; whether Trethera would obtain the necessary regulatory approvals to commence human trials or commercialize TRE-515 or any novel therapies resulting from such research; Trethera successfully implementing its growth strategy, including that relating to its disease therapies; the effects of the global Covid-19 pandemic; changes in economic conditions; competition; and risks and uncertainties applicable to the business of Trethera. The statements in this press release speak only as of the date hereof and Trethera does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise. The Company intends that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/b3b986e5-6dae-4696-9816-43ac934bb36a

3月19日，李氏大药厂控股有限公司（简称“李氏大药厂”或“集团”，股份编号：00950）发布公告：集团预期截至2024年12月31日止年度将录得股东占未审核纯利83,000,000港元至106,000,000港元，而截至2023年12月31日止年度则录得约17,000,000港元。尽管已计提南沙之土地使用权拨备及若干研究及开发项目之减值亏损，惟本集团之年度业绩大幅改善，主要受以下因素推动，其中包括(1)获纳入最新版《国家基本医疗保险、工伤保险和生育保险药品目录》产品(例如曲前列尼尔注射液(芮旎爾®)、利鲁唑口服混悬液(芮舒延®)及盐酸曲唑酮缓释片(曲特恪®))连同药品集采计划选定产品(例如那屈肝素钙注射液(立腾菁®))的销售增长于回顾年度内显著改善销售收益;(2)藉优化研究及开发组合使研究及开发费用减少;(3)本集团优化管理举措所节省成本。 本公司仍在落实本集团截至2024年12月31日止年度全年业绩。本公布所载资料仅为董事会基于目前可获得的资料(包括未经本公司核数师确认、审阅或审核之本集团截至2024年12月31日止年度未审核综合管理账目)初步评估。 本公司截至2024年12月31日止年度全年业绩公布预期将于2025年3月27日公布。 往期回顾 ◆  快讯 | 助力中国ALS循证医学发展——利鲁唑口服混悬液真实世界研究项目启动 ◆  筑安全防线，守护企业发展——“安全守护者” 线上知识竞赛 ◆  李氏大药厂创始人李小羿博士受邀参加“香港生物科技创新与转化项目启动研讨会” ◆  从38个赞到3200束光 | 巾帼风采，馥钰美丽——李氏大药厂女神节活动圆满收官 关于李氏大药厂 - 亞太地區最具創新力的上市企業 - 李氏大药厂控股有限公司（简称“李氏大药厂”或“集团”, 股份编号: 00950）成立于1994年，是一家结合了研发驱动和市场导向的生物医药集团公司，总部位于香港。李氏大药厂于2002年7月在香港联合交易所（简称：“港交所”）创业板上市，并于2010年5月成功转至港交所主板上市。李氏大药厂在中国大陆拥有两大综合性生产研发基地，即：兆科合肥基地（位于安徽省合肥市高新区）和兆科广州基地（位于广东省广州市南沙区），目前员工总数超过1100人。 李氏大药厂立足中国大陆，放眼国际，通过坚实和稳固的投入建设，全面覆盖整合了药物研发、临床研究、药政注册、生产制造、销售与市场推广等各个环节。集团集中于心血管科、妇科与产科、儿科、罕见病、肿瘤科、皮肤科、精神科等多个重要疾病领域开发不同阶段的专利新药和高端仿制药。李氏大药厂与20余家国际公司建立了广泛的合作关系，目前在中国大陆、香港、澳门及台湾地区推广及销售25种包含专利新药、仿制药物及授权引进的国外已上市进口药品。 李氏大药厂现有超过40个处于不同开发阶段的产品，既有自主研发产品，也有通过来自美国、欧洲及日本等公司通过授权引进进行研发、生产以及商业化的产品。 李氏大药厂的使命是致力于研发新药，治疗顽疾，改善生活质量，为患者带来福音，目标成为亚洲成功的生物制药集团，提供治疗疾病和改善生活质量的创新产品。 下滑查看更多 免责声明：本公众号发布或转载企业宣传资讯，仅供读者参考之用，并不构成收购、购买或认购依据，并不构成任何个人化的投资劝诱或建议。因本公布全部或任何部分内容而产生或因倚赖该等内容而引致的任何损失不承担任何责任。 文章来源：集团官网 与“李”同行 招商热线 020-39062882