A phase 3 study evaluating the efficacy and safety of ampreloxetine for the treatment of symptomatic neurogenic orthostatic hypotension did not meet its primary endpoint.
The multicenter, randomized, double-blind, placebo-controlled study (ClinicalTrials.gov Identifier: NCT03750552) included 195 patients aged 30 years and older with primary autonomic failures and symptomatic neurogenic orthostatic hypotension. Patients were randomly assigned to receive ampreloxetine, a long-acting norepinephrine reuptake inhibitor, or placebo orally once daily for 4 weeks.
Results showed that treatment with ampreloxetine did not meet the primary endpoint of score change from baseline on Question 1 (dizziness, lightheadedness, feeling faint, or feeling like you might blackout) of the Orthostatic Hypotension Symptom Assessment.
“These are not the results we had hoped to achieve, especially given the clear unmet need for patients suffering from symptomatic [neurogenic orthostatic hypotension] and the positive top-line 4-week results from the phase 2 study announced in 2018,” said Rick E Winningham, CEO, Theravance Biopharma. “We will continue to analyze the data to better understand the findings.”
As for safety, the majority of treatment-related adverse events were mild or moderate in severity. There was no signal for supine hypertension. Additional study results will be presented at a future scientific forum.
Theravance currently has 2 other studies (Study 0170; ClinicalTrials.gov Identifier: NCT03829657; and Study 0171; ClinicalTrials.gov Identifier: NCT04095793) evaluating ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension. Given these findings, the Company is determining next steps for these trials.
Theravance Biopharma, Inc. announces top-line results from a phase 3 study of ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension. News release. Theravance Biopharma, Inc. Accessed September 15, 2021. https://www.prnewswire.com/news-releases/theravance-biopharma-inc-announces-top-line-results-from-a-phase-3-study-of-ampreloxetine-in-patients-with-symptomatic-neurogenic-orthostatic-hypotension-301377200.html.