(RTTNews) - Alterity Therapeutics (ATHE), a biotechnology company, on Tuesday reported achieving alignment with the U.S. Food and Drug Administration (FDA) on the planned Phase 3 trial for ATH434 in treating multiple system atrophy, after a successful end-of-Phase 2 meeting.
Shares rose 10% on Tuesday.
Multiple system atrophy (MSA) is a rare, neurodegenerative disease characterized as a Parkinsonian disorder, as patients show slowed movement and rigidity, autonomic dysfunction affecting blood pressure maintenance and bladder control, and impaired balance or coordination that causes frequent falls. Pathologically, MSA is diagnosed by detecting accumulation of abnormal clumping of the protein a-synuclein within oligodendrocytes, the myelin-producing support cells of the central nervous system. There are no approved therapies for the disease.
Alterity's lead candidate, ATH434. is an oral agent developed to reduce a-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain. The drug has shown potential in treating Parkinson's disease and Parkinsonian disorders like MSA. ATH434 was previously granted Fast Track and Orphan Drug designation by the FDA and European Commission for the treatment of MSA.
The End-of-Phase 2 (EOP2) meeting resulted in the FDA aligning with the company's design for the Phase 3, including aspects like study population and treatment duration. The FDA also agreed on the primary endpoint to be measured using the Unified Multiple System Atrophy Rating Scale (UMSARS Part I), which evaluates the 11 items of orthostatic symptoms, swallowing, speech, handwriting, cutting food, dressing, hygiene, walking, falling, urinary and bowel function in the patient.
The FDA indicated that the secondary endpoints evaluated by swallowing disturbance questionnaire, the orthostatic hypotension symptom assessment, and the clinical global impression of severity were sufficient to prove efficacy. The selection of a dose regimen of 50 mg of ATH434 administered twice a day was also approved, based on results from the Phase 2 ATH434-201 study which showed a 48% slowing of disease progression in treated patients.
The company dubbed the FDA alignment an important de-risking milestone and expects the Phase 3 trial for ATH434 in treating MSA to begin at the end of 2026.
ATHE closed Tuesday at $4.29, up 10%.
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