Theravance Biopharma completes enrollment in pivotal Phase 3 CYPRESS study.

Theravance Biopharma has completed enrollment in the open-label portion of its Phase 3 CYPRESS study for ampreloxetine in symptomatic neurogenic orthostatic hypotension (nOH) due to multiple system atrophy (MSA). The trial aims to address the unmet need for a therapy to provide durable benefit for the 40,000 patients in the U.S. with symptomatic nOH in MSA. Topline results are anticipated in Q1 2026, and if successful, the company plans to expedite the submission of a new drug application (NDA). Ampreloxetine has Orphan Drug Designation in the U.S., highlighting the significant unmet need for symptomatic nOH treatment due to MSA.

Theravance Biopharma, Inc. has successfully completed enrollment in the open-label portion of its pivotal Phase 3 CYPRESS study for ampreloxetine, an investigational treatment for symptomatic neurogenic orthostatic hypotension (nOH) due to multiple system atrophy (MSA). The study, which aims to address the critical unmet need for a durable therapy for the 40,000 patients in the U.S. with symptomatic nOH in MSA, marks a significant milestone in the company's development pipeline [1].

The CYPRESS study, a global, randomized-withdrawal trial, enrolled patients with symptomatic nOH due to clinically diagnosed MSA at sites across four continents. The study's primary endpoint is the change in orthostatic hypotension symptom assessment (OHSA) composite score from randomized-withdrawal baseline to Week 8. Secondary outcomes include changes in Orthostatic Hypotension Daily Activity Scale (OHDAS) items 1 and 3. Topline results from the study are expected in the first quarter of 2026, with the company planning to submit an expedited New Drug Application (NDA) if the data are supportive [1].

Ampreloxetine, a once-daily, selective norepinephrine reuptake inhibitor, has shown promising results in previous studies. Study 0170 demonstrated compelling improvement in OHSA composite score without worsening supine hypertension in MSA patients. The CYPRESS study was designed with insights from Study 0170, and the company is confident that it will evaluate ampreloxetine's full potential in this patient population [1].

The completion of enrollment in the CYPRESS study underscores Theravance Biopharma's commitment to advancing high-impact catalysts. The company's strategic focus on delivering Medicines that Make a Difference® is evident in its efforts to address the significant unmet need for effective and durable treatment options in MSA. The company has been granted Orphan Drug Designation for ampreloxetine in the U.S., further highlighting the critical need for a new therapy in this area [1].

References:

[1] https://www.prnewswire.com/news-releases/theravance-biopharma-completes-enrollment-in-pivotal-phase-3-cypress-study-of-ampreloxetine-in-patients-with-symptomatic-neurogenic-orthostatic-hypotension-due-to-multiple-system-atrophy-302537380.html