Theravance Biopharma Launches Education Campaign for Rare Disease Ahead of Phase 3 Data

Theravance Biopharma has initiated a physician education campaign called "Power in the Periphery" to raise awareness about neurogenic orthostatic hypotension (nOH) associated with multiple system atrophy (MSA). This strategic move comes as the company prepares for the release of pivotal phase 3 data for its investigational drug, ampreloxetine.

Campaign Details and Disease Background

The "Power in the Periphery" campaign, launched at the International Congress of Parkinson's Disease and Movement Disorders, aims to educate healthcare professionals about nOH in MSA patients. MSA is a progressive brain disorder affecting an estimated 50,000 patients in the U.S., with 70% to 90% experiencing nOH symptoms.

nOH is characterized by drops in blood pressure upon standing, leading to symptoms such as lightheadedness, fainting, and fatigue. The campaign emphasizes that while MSA primarily affects the central nervous system, peripheral nerves remain functional and continue producing norepinephrine, a key neurotransmitter in blood pressure regulation.

Ampreloxetine Development and Clinical Trials

Theravance's ampreloxetine, a selective norepinephrine reuptake inhibitor, is designed to improve standing blood pressure and reduce nOH symptoms. The company is currently conducting a phase 3 trial focusing on nOH in MSA patients, with data expected in the first quarter of 2026.

This targeted approach follows a previous phase 3 trial that missed its primary endpoint in a broader nOH patient population. However, Theravance identified a promising efficacy signal in a prespecified subgroup of MSA patients, leading to the current, more focused study.

Market Preparation and Future Outlook

In preparation for potential market entry, Theravance has been engaging in various activities:

1. Surveying 200 neurologists and cardiologists to assess market readiness
2. Conducting disease state education and targeted stakeholder engagement
3. Initiating discussions with payers

The company plans to seek FDA approval if the upcoming phase 3 data prove positive, positioning ampreloxetine as a potential treatment option for nOH in MSA patients.