Theravance Biopharma (TBPH) KOL Event summary

Introduction and agenda

• Event focused on ampreloxetine, an orphan drug and potential precision medicine for neurogenic orthostatic hypotension (NOH/nOH) in multiple system atrophy (MSA), with pivotal phase III Cypress study data expected Q1 2026.

• Agenda included unmet need overview, clinical evidence, phase III study details, commercial opportunity, CEO remarks, KOL-led disease and trial overview, and Q&A.

KOL background and credentials

• Dr. Horacio Kaufmann, professor at NYU and director of the Dysautonomia Center, F.B. Axelrod Professor of Neurology, has over 40 years of experience treating severe orthostatic hypotension and presented on nOH in MSA and clinical trial data.

Market insights and analysis

• NOH/nOH in MSA affects ~40,000 patients in the U.S., with high unmet need and limited effective therapies; only ~34% of patients are treated.

• Current treatments (midodrine, droxidopa, fludrocortisone) are inadequate, with 70% of MSA patients remaining symptomatic and therapies requiring frequent dosing and carrying risks like supine hypertension.

• Market is highly concentrated among a small number of specialists and centers of excellence, enabling efficient commercial targeting; only a third of MSA patients receive therapy.

• Rare disease pricing and orphan drug status position ampreloxetine for premium value and blockbuster potential, with significant opportunities in Europe and Asia.

• Market research shows high physician and payer recognition of unmet need and readiness to adopt new therapies.