Core Insights - Theravance Biopharma has completed enrollment in the pivotal Phase 3 CYPRESS study for ampreloxetine, targeting symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA) [1][3][5] - nOH affects approximately 80% of MSA patients, leading to severe symptoms and a lack of effective treatment options [1][8] - The CYPRESS study aims to evaluate the efficacy and safety of ampreloxetine, with topline data expected in Q1 2026 [3][5] Company Overview - Theravance Biopharma focuses on developing innovative therapies, including ampreloxetine, a selective norepinephrine reuptake inhibitor for nOH in MSA patients [4][9] - The company has received Orphan Drug Designation for ampreloxetine in the U.S., highlighting the unmet medical need in this patient population [5][9] Study Details - The CYPRESS study is a global, randomized-withdrawal trial assessing ampreloxetine's impact on nOH symptoms, with a primary endpoint of change in the Orthostatic Hypotension Symptom Assessment (OHSA) composite score [2][6] - The study includes a 12-week open-label phase followed by an 8-week randomized withdrawal phase [2][6] Market Context - There are approximately 40,000 patients in the U.S. suffering from symptomatic nOH due to MSA, indicating a significant market opportunity for effective treatments [2][5] - Current therapies for nOH often fail to provide lasting relief and have safety concerns, underscoring the need for new solutions like ampreloxetine [2][3]

Core 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]

Core Insights - Theravance Biopharma announced the presentation of analyses from the initial Phase 3 program of ampreloxetine at the International MSA Congress, highlighting its potential to address neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA) [1][2] - The ongoing registrational study (CYPRESS) aims to evaluate the efficacy and durability of ampreloxetine in treating symptomatic nOH in MSA patients [4][5] Company Overview - Theravance Biopharma focuses on developing innovative therapies, with ampreloxetine being a late-stage investigational norepinephrine reuptake inhibitor for symptomatic nOH in MSA patients [3][10] - The company has received Orphan Drug Designation for ampreloxetine in the US, indicating its commitment to addressing unmet medical needs in rare diseases [3] Clinical Study Details - The CYPRESS study is a multi-center, randomized withdrawal trial evaluating ampreloxetine's efficacy after 20 weeks of treatment, with the primary endpoint being the change in the Orthostatic Hypotension Symptom Assessment (OHSA) composite score [4] - Previous studies (Study 0169 and Study 0170) assessed the efficacy and safety of ampreloxetine, with Study 0170 showing a significant benefit in MSA patients, indicated by a 72% reduction in treatment failure odds compared to placebo [5][7] Disease Context - Multiple system atrophy (MSA) is a progressive disorder affecting movement and autonomic functions, with approximately 50,000 patients in the US, of which 70-90% experience nOH symptoms [8][9] - Neurogenic orthostatic hypotension (nOH) is characterized by a significant drop in blood pressure upon standing, leading to debilitating symptoms such as dizziness and fainting [9]