Two major breakthroughs in Fragile X Syndrome treatments
16 Apr 2024
Orphan DrugPhase 2Phase 1Clinical Result
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Source: 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.
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Source: Pharmaceutical Technology
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Source: 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.
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