Theravance Biopharma to Unveil Critical Phase 3 Findings at Upcoming International MSA Congress 2025

Theravance Biopharma's Presentation at the 2025 International MSA Congress

Theravance Biopharma, Inc., a company dedicated to developing innovative therapies, has announced it will present critical analyses of the initial Phase 3 data regarding ampreloxetine at the International MSA Congress scheduled for May 9-11, 2025, in Boston, Massachusetts. The company’s studies focus on the highly selective mechanism of action of ampreloxetine and its promising potential to alleviate symptoms of neurogenic orthostatic hypotension (nOH) in patients suffering from multiple system atrophy (MSA).

What is Ampreloxetine?

Ampreloxetine is an investigational medication that acts as a norepinephrine reuptake inhibitor, aimed at treating symptomatic nOH in MSA patients. Recent studies have shown that this treatment can significantly enhance norepinephrine levels, positively impact blood pressure, and improve symptoms without increasing the risk of supine hypertension, ensuring patient safety and comfort. Importantly, Theravance Biopharma has obtained Orphan Drug Designation in the U.S. for ampreloxetine, which recognizes the medication's potential to address an unmet medical need in a rare patient population.

Upcoming Presentations

Theravance will unveil its findings through a plenary oral session and various poster presentations during the congress. Notable topics include a subgroup analysis of the Phase 3 studies of ampreloxetine and its impact on symptom burden and daily functioning for patients with alpha-synucleinopathies. Presentations will occur on May 9 and May 10, showcasing extensive research and data which indicate the benefits of ampreloxetine therapy in alleviating symptoms of nOH.
1. Oral Presentation: Norcliffe-Kaufmann L, et al. on May 9, 2025, at 9:40 AM EDT
2. Poster Presentations:
- Norcliffe-Kaufmann L, et al. on May 10, 2025, at 12:00 PM EDT
- Iodice V, et al. on May 9, 2025, at 11:45 AM EDT

The Clinical Journey

The ongoing CYPRESS study (NCT05696717) is key to evaluating the long-term efficacy and safety of ampreloxetine. This multi-center, randomized withdrawal study includes various phases to thoroughly assess the treatment’s effectiveness on nOH after 20 weeks of administration. With a focus on the Orthostatic Hypotension Symptom Assessment (OHSA) scores, this rigorous trial will contribute vital data ahead of a potential NDA filing if outcomes are favorable.

In earlier phases, particularly Study 0169 and Study 0170, Theravance examined the efficacy and safety of ampreloxetine against placebo, investigating over 195 symptomatic nOH patients. Though the broader study did not meet its primary endpoint conclusively, the subgroup analysis indicated significant improvements in MSA patients, suggesting that ampreloxetine could notably reduce treatment failure rates.

MSA and nOH Overview

Multiple System Atrophy (MSA) is a debilitating neurological disorder that affects movement, balance, and autonomic functions, leading to severe symptoms like neurogenic orthostatic hypotension. This condition often causes drastic blood pressure drops when patients stand, resulting in dizziness and fainting. Approximately 50,000 people are diagnosed with MSA in the U.S., with nOH affecting a significant proportion of these individuals. Unfortunately, many remain symptomatic despite current treatments, highlighting the urgent need for effective therapies.

Looking Ahead

Theravance Biopharma is committed to its mission of delivering impactful medicines aimed at enhancing patient lives. By presenting at the International MSA Congress, the company seeks to raise awareness of ampreloxetine and its potential role in treating nOH related to MSA. As it moves forward, Theravance continues to prioritize clinical advancements that promise to change the landscape of treatment for patients struggling with these challenging symptoms. For further details on their ongoing studies and findings, visit Theravance Biopharma’s website.

In summary, the insights gleaned from the upcoming congress will undoubtedly contribute to the medical community's understanding of nOH and MSA, paving the way for improved treatment options in the future.