Hangzhou-based HealZen Therapeutics has announced receiving approval from the National Medical Products Administration (NMPA) to conduct a clinical study for its Category 1 drug candidate HZ-A-018 in recurrent multiple sclerosis (RMS) and neuromyelitis optica spectrum disorders (NMOSD). This marks a significant step forward in exploring new treatment options for these challenging autoimmune diseases.
Disease Background and Unmet Needs
Multiple sclerosis (MS) is an immune-mediated chronic inflammatory demyelinating disease of the central nervous system, often involving the periventricular areas, near cortex, optic nerve, spinal cord, brainstem, and cerebellum. Characterized by multiple spatial and temporal features, MS can lead to disability or even death due to its wide-ranging impact. Despite numerous approved treatments, MS remains incurable, and there is an urgent need for more effective and safer therapies.
Neuromyelitis optica spectrum disorders (NMOSD) are a group of autoimmune inflammatory demyelinating diseases primarily affecting the optic nerve and spinal cord. NMOSD is characterized by severe optic neuritis and longitudinally extensive transverse myelitis, with high recurrence and disability rates. Currently, there is a significant unmet need for effective treatments for NMOSD in China.
HZ-A-018: Mechanism and Development
HZ-A-018 is a highly selective small molecule Bruton tyrosine kinase (BTK) inhibitor with strong kinase selectivity and blood-brain barrier permeability. It can effectively inhibit the activation and differentiation of B cells in vitro and has shown promise in improving symptoms in animal models. HZ-A-018 is expected to bring new treatment options to patients with RMS and NMOSD. Previously, HZ-A-018 has been approved for clinical studies in B-cell lymphoma, relapsed/refractory primary central nervous system lymphoma (PCNSL), and chronic/persistent adult primary immune thrombocytopenia (ITP).-Fineline Info & Tech
