Theravance Biopharma is gearing up for a major clinical milestone. The company will host a virtual KOL investor event on December 8, 2025, led by Dr. Horacio Kaufmann from NYU Langone Health. The session aims to spotlight the clinical need, market opportunity, and upcoming data for ampreloxetine, a late-stage therapy in development for symptomatic neurogenic orthostatic hypotension (nOH) in multiple system atrophy (MSA).
Why This Event Matters?
nOH affects nearly 80% of patients with MSA. It causes sharp drops in blood pressure upon standing and severely impacts daily life. Current treatments are limited, and many patients remain symptomatic.
Ampreloxetine offers a potential precision-medicine approach with meaningful clinical benefits observed in earlier trials.
What the Event Will Cover
1. The Unmet Need in MSA-related nOH
- MSA is a rare, progressive neurodegenerative disease.
- nOH is one of its most debilitating symptoms.
- Existing therapies provide incomplete relief for many patients.
2. Clinical Rationale for Ampreloxetine
Dr. Kaufmann will discuss:
- How the drug increases norepinephrine levels.
- Its impact on blood pressure stability.
- Durable symptom improvement seen in Study 0170.
- No worsening of supine hypertension in tested patients.
Ampreloxetine has received Orphan Drug Designation in the U.S. for nOH in MSA.
3. Deep Dive Into the Phase 3 CYPRESS Study
The event will outline the strategic design of Study 0197, which:
- Is a registrational, Phase 3, randomized withdrawal trial.
- Evaluates ampreloxetine over a 20-week treatment duration.
- Uses OHSA composite score as the primary endpoint.
- Includes an open-label phase, double-blind withdrawal, and long-term extension.
Topline data are expected in Q1 2026, making this event highly anticipated.
4. Commercial Strategy and Market Opportunity
Company leadership will explain:
- Why nOH in MSA is a high-value niche with significant unmet need.
- How ampreloxetine could become the first precision therapy for this population.
- The company’s roadmap post-Phase 3 readout, including regulatory submission plans.
A live Q&A session will follow.
About Dr. Horacio Kaufmann
Dr. Kaufmann is a global authority in autonomic disorders. He leads the Dysautonomia Center at NYU Langone and has decades of clinical and research experience across:
- Familial dysautonomia
- Multiple system atrophy
- Pure autonomic failure
- Amyloidosis
- Autonomic neuropathies
His work has contributed to new diagnostic tools, therapeutic innovations, and improved patient care models.
About Multiple System Atrophy & nOH
What is MSA?
A progressive neurological disorder affecting movement, balance, and autonomic functions.
What is nOH?
A sudden drop in blood pressure within 3 minutes of standing.
Symptoms include:
- Dizziness
- Fatigue
- Blurry vision
- Syncope
- Cognitive difficulty
- Weakness
Around 70–90% of MSA patients experience nOH.
About Ampreloxetine
Ampreloxetine is:
- A once-daily, selective norepinephrine reuptake inhibitor.
- Designed to stabilize blood pressure and improve nOH symptoms.
- Being evaluated as a long-term treatment option with durable benefits.
Positive signals from earlier studies support continued late-stage development.
About Theravance Biopharma
Theravance Biopharma focuses on creating medicines that make a difference.
Key assets include:
- YUPELRI®, approved for COPD maintenance therapy.
- Ampreloxetine, now in late-stage testing for nOH in MSA.
The company combines scientific expertise with a commitment to high-impact therapies for serious conditions.
