Theravance Biopharma(TBPH) Prnewswire·2025-04-07 19:55Core Insights - Theravance Biopharma announced new analyses supporting the efficacy and safety of ampreloxetine for treating symptomatic neurogenic orthostatic hypotension (nOH) at the 77th Annual Meeting of the American Academy of Neurology [1][2] Group 1: Efficacy of Ampreloxetine - The Phase 3 program included two trials focusing on patients with nOH due to multiple system atrophy (MSA), Parkinson's Disease, and pure autonomic failure, with ongoing studies supporting its potential for registration [2][3] - In Study 0169, patients treated with ampreloxetine showed a mean increase of 58% in venous plasma norepinephrine levels after 4 weeks, with a 79% increase in MSA patients [6][12] - The pharmacodynamic effects demonstrated durable target engagement and a physiological effect on blood pressure, particularly in MSA patients [2][6] Group 2: Safety Profile - Analyses indicated no worsening of supine hypertension in patients treated with ampreloxetine, a significant concern with existing therapies [5][7] - Patients on ampreloxetine did not experience increased overnight supine hypertension compared to those on placebo, suggesting a favorable safety profile [7][8] - The unique mechanism of action of ampreloxetine may allow for effective treatment of nOH without the side effects associated with high blood pressure in the supine position [8][10] Group 3: Ongoing Studies and Future Prospects - The ongoing CYPRESS study aims to evaluate the efficacy and durability of ampreloxetine in MSA patients with symptomatic nOH, with a primary endpoint focused on changes in the Orthostatic Hypotension Symptom Assessment (OHSA) composite score [11][12] - The company plans to file a New Drug Application (NDA) for ampreloxetine if results from the ongoing studies are supportive [10][12] - The potential for ampreloxetine to become a first-in-class therapy for nOH in MSA patients is highlighted, addressing a significant unmet medical need [10][14]