Two major breakthroughs in Fragile X Syndrome treatments
2024-04-16
孤儿药临床2期临床1期临床结果
来源: Pharmaceutical Technology
Fragile X Syndrome typically affects males more severely compared to females and is a significant cause of inherited intellectual disability and autism. Credit: bangoland via Shutterstock.
Spinogenix has received approval from the US Food and Drug Administration (FDA) for a Phase II clinical trial of SPG601 for Fragile X Syndrome (FXS), the same week that the European Commission gave orphan drug status to Shionogi’s zatolmilast (BPN14770).
FXS is a rare genetic condition that causes a range of developmental problems, including learning disabilities and cognitive impairment. The disorder typically affects males more severely than females and is a significant cause of inherited intellectual disability and autism. These issues arise from the silencing of the Fmr1 gene, leading to impaired BK channel function, which is essential for neuron communication.
Spinogenix’s SPG601 is a novel small molecule BK channel activatorBK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function. The Phase IIa trial will assess the neurophysiological and clinical impacts of SPG601, a once-a-day pill, in adult men diagnosed with FXS. This trial aims to determine how effective the drug is in altering disease markers and alleviating symptoms compared to a placebo.
CEO of Spinogenix Stella Sarraf emphasised the gap in effective and patient-friendly solutions for neurodevelopmental conditions such as FXS: “Like many other conditions, loss of synaptic function remains a key driver of disease, and for that reason, we are excited to launch our first US trial to address this unmet need in FXS.”
This development follows the 11 April announcement that the European Commission has granted orphan medicinal product designation for Shionogi’s FXS drug, zatolmilast, a cAMP-specific 3′,5’ cyclic phosphodiesterase 4D inhibitor. Zatolmilast is developed by Tetra Therapeutics, a Shionogi Group company. Shionogi acquired Tetra in 2020 as part of its efforts to develop medications for unmet medical needs. Zatolmilast received orphan drug designation for FXS in the US in September 2023.
See Also:
Preview
来源: Pharmaceutical Technology
NorgineNorgine seeks approval for high-risk neuroblastoma treatment
Preview
来源: Pharmaceutical Technology
In a Phase II clinical trial assessing zatolmilast, cognitive assessments using the NIH Toolbox revealed significant benefits in oral reading recognition, picture vocabulary, and cognition crystallised composite score (NCT03569631), in males aged 18 to 45 years. The drug is forecast to generate $64m in sales in 2030, according to GlobalData.
GlobalData is the parent company of Pharmaceutical Technology.
There are currently no approved treatments for FXS, and prescribed treatment consists exclusively of off-label drugs that target individual symptoms of the disease, such as SSRIs for depressive symptoms and anxiety, and stimulants that include methylphenidate for hyperactivity, inattention, and impulsivity.
According to a report on the GlobalData Pharma Intelligence Center, the FXS market across two major markets (US and Germany) was valued at $21.9m in 2020 and is projected to grow at a compound annual growth rate of 7.6% reaching $45.8m by 2030.
更多内容，请访问原始网站
文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。
立即开始免费试用!
智慧芽新药情报库是智慧芽专为生命科学人士构建的基于AI的创新药情报平台，助您全方位提升您的研发与决策效率。
立即开始数据试用!
智慧芽新药库数据也通过智慧芽数据服务平台，以API或者数据包形式对外开放，助您更加充分利用智慧芽新药情报信息。