September 26, 2026 – Aspartes Pharmaceuticals Announces Completion of Phase 1 Clinical Study Evaluating ASP-001 ODT Formulations.
September 26, 2026 – Aspartes Pharmaceuticals Announces Completion of Phase 1 Clinical Study Evaluating ASP-001 ODT Formulations.
Alternating Hemiplegia of Childhood (AHC) is an ultra-rare pediatric neurodevelopmental disorder characterized by recurrent prolonged episodes of paralysis that can affect one or both sides of the body.
Estimated prevalence of AHC in the U.S.
Typical age of disease onset
Number of treatments available for AHC
AHC is most commonly caused by genetic mutations in the ATP1a3 gene, which encodes part of the Na+/K+-ATPase exchanger used by neurons to maintain normal electrical activity. These mutations lead to the exchanger to stop functioning properly and cause the brain to become vulnerable to episodes of abnormal neurological function which take the form of hemiplegic attacks, abnormal movements, seizures, and developmental difficulties.
Although ATP1a3 is the primary gene associated with AHC, other genes including ATP1a2, CACNA1A, SLC2A1, and PRRT2 have also been reported to cause AHC and AHC-like presentations in patients. These genes are involved in related neurological processes such as ion transport, calcium-channel function, and neuronal excitability.
AHC is diagnosed based on a combination of clinical history, neurological examination and the identification of key characteristic features. Genetic testing is also used to confirm the diagnosis by identifying a variant in ATP1a3, ATP1a2 or another related gene.
AHC is characterized by prolonged and refractory hemiplegic attacks that usually begin within the first 18 months of life. However, the effects of AHC are not limited to hemiplegic attacks. Almost all patients suffer from at least one non-hemiplegic comorbidity, including:
The frequency, duration, and severity of episodes vary drastically among patients and while symptoms may change with age, the condition often results in long-term neurological challenges and increased mortality.
There is currently no cure for AHC, and available treatment options are limited, highlighting a severe unmet need for patients suffering from this disease.
Aspartes is developing ASP-001, a novel pediatric orally dispersible tablet formulation of flunarizine.
Widely used off-label to help reduce the frequency, duration, and severity of hemiplegic attacks, flunarizine has the potential to become the first FDA-approved treatment specifically indicated for AHC.
