BASKING RIDGE, N.J., April 09, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the "FDA") has granted Orphan Drug Designation ("ODD") to LSTA1, the Company's lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults. LSTA1 recently received Rare Pediatric Disease Designation for osteosarcoma, as announced by the Company on March 21, 2024.
"We are thrilled to have received another favorable regulatory designation from the FDA. This underscores the significant unmet medical need and demand for better treatments for patients diagnosed with osteosarcoma," stated Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata. "Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis. Receiving an ODD from the FDA is an important milestone as we plan for future clinical expansion of LSTA1, and we believe it reflects the broad clinical utility of LSTA1 for the treatment of a wide array of solid tumors."
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 United States. The designation can provide for a seven-year window of exclusive marketing rights post-approval as well as exemption from user fees and eligibility for tax credits for qualified clinical trials. In addition to the financial benefits, it also may potentially shorten clinical development due to closer collaboration with the FDA.
About LSTA1
LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., covalently bound) anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor. LSTA1 also has the potential to modify the tumor microenvironment resulting in tumors which are more susceptible to immunotherapies. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. Additionally, LSTA1 has demonstrated favorable safety, tolerability, and activity in clinical trials to enhance delivery of SoC chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively. LSTA1 has been granted ODD for pancreatic cancer in the U.S. and Europe as well as for glioblastoma multiforme and osteosarcoma in the U.S. It also received a Fast Track designation from the FDA for pancreatic cancer and, just recently, a Rare Pediatric Disease designation from the FDA for osteosarcoma.
新泽西州巴斯金里奇,2024年4月9日(GLOBE NEWSWIRE)——开发治疗晚期实体瘤和其他严重疾病创新疗法的临床阶段制药公司Lisata Therapeutics, Inc.(纳斯达克股票代码:LSTA)(“Lisata” 或 “公司”)今天宣布,美国食品药品监督管理局(“FDA”)已授予孤儿药称号(“ODD”)LSTA1 是该公司的主要候选产品,用于治疗骨肉瘤,一种罕见的癌症,可能发生在儿童、青少年和年轻人身上。正如该公司于 2024 年 3 月 21 日宣布的那样,LSTA1 最近获得了骨肉瘤罕见儿科疾病认定。
“我们很高兴获得美国食品药品管理局又一项有利的监管称号。这凸显了被诊断为骨肉瘤的患者仍存在大量未得到满足的医疗需求和对更好治疗的需求。” Lisata研发执行副总裁兼首席医学官克里斯汀·巴克医学博士说。“骨肉瘤虽然罕见,但是一种通常与高发病率、早期转移、快速进展和预后不佳相关的骨癌。在我们计划未来扩展 LSTA1 的临床过程中,获得 FDA 的 ODD 是一个重要的里程碑,我们认为这反映了 LSTA1 在治疗各种实体瘤方面的广泛临床用途。”
孤儿药认证由美国食品药品管理局授予旨在治疗罕见疾病或病症的药物或生物制剂,罕见疾病或病症的定义是影响美国少于20万人的疾病或病症。该指定可以为批准后的七年独家营销权提供期限,并免除用户费用,并有资格获得合格临床试验的税收抵免。除了经济利益外,由于与美国食品药品管理局的更紧密合作,它还可能缩短临床开发。
关于 LSTA1
LSTA1 是一种在研药物,旨在激活一种新的摄取途径,允许共同给药或联用(即共价结合)的抗癌药物更有效地穿透实体瘤。LSTA1 以肿瘤特异性方式启动这种主动转运系统,从而使全身共用的抗癌药物更有效地穿透和积聚在肿瘤中。LSTA1 还有可能改变肿瘤微环境,导致肿瘤更容易受到免疫疗法的影响。Lisata及其合作者已经积累了大量的非临床数据,这些数据表明,包括化疗、免疫疗法和基于RNA的疗法在内的一系列现有和新兴抗癌疗法的交付得到了加强。此外,LSTA1 在临床试验中表现出良好的安全性、耐受性和活性,可增强胰腺癌 SoC 化疗的提供。Lisata 正在探索 LSTA1 的潜力,它使各种治疗模式能够更有效地治疗一系列实体瘤。LSTA1 在美国和欧洲被授予胰腺癌ODD,在美国被授予用于治疗多形胶质母细胞瘤和骨肉瘤的ODD。它还获得了美国食品药品管理局的胰腺癌快速通道称号,就在最近,美国食品药品管理局授予的骨肉瘤罕见儿科疾病称号。