Prnewswire·2025-05-09 16:00Core Insights - Theravance Biopharma presented analyses of its Phase 3 program for ampreloxetine, targeting symptomatic neurogenic orthostatic hypotension (nOH) at the International MSA Congress in Boston [1][2] Company Overview - Theravance Biopharma focuses on developing innovative therapies, including ampreloxetine, a selective norepinephrine reuptake inhibitor for treating nOH in patients with multiple system atrophy (MSA) [6][13] - The company has received Orphan Drug Designation for ampreloxetine in the U.S. and plans to file a New Drug Application (NDA) if ongoing studies yield positive results [6] Clinical Study Findings - A subgroup analysis from the REDWOOD 0170 study indicated that MSA patients had the highest nOH symptom burden, emphasizing the need for improved therapies [2] - Results from the studies support the ongoing registrational study (CYPRESS) evaluating ampreloxetine's efficacy in MSA patients [3] - Patients with MSA showed clinically meaningful improvement in nOH symptoms after 16 weeks of treatment with ampreloxetine, with stable symptoms in the treatment group compared to worsening in the placebo group [7][10] Study Details - The CYPRESS study is a Phase 3, multi-center, randomized withdrawal trial assessing ampreloxetine's efficacy after 20 weeks of treatment, with primary endpoints focused on changes in the Orthostatic Hypotension Symptom Assessment (OHSA) composite score [9] - The REDWOOD 0170 study included a 16-week open-label period followed by a 6-week double-blind, placebo-controlled withdrawal period, with a primary endpoint of treatment failure defined by specific worsening scores [10] Patient Impact - MSA patients experience severe symptoms affecting their quality of life due to nOH, with 70-90% of MSA patients experiencing these symptoms [11] - The clinical profile of ampreloxetine demonstrated target engagement and sustained blood pressure effects, indicating its potential as a first-in-class therapy for MSA-related nOH [6][7]