Bioblast Pharma

Hope should not be rare

OPMD clinical trial

HOPEMD Phase 2 Open-Label Study

Evaluating a promising new treatment for patients with OPMD

The primary objective of the HOPEMD Phase 2 six-month open-label clinical study was to assess the safety and tolerability of trehalose 90 mg/mL IV solution. Although not powered for efficacy, secondary endpoints were included to explore if trehalose 90 mg/mL IV solution could improve or prevent worsening of OPMD disease markers.

The study enrolled 25 patients with clinical dysphagia and muscle weakness at two centers in Canada and Israel. All patients have completed 24 weeks of weekly treatment with trehalose 90 mg/mL IV solution.

Trehalose 90 mg/mL IV solution was observed to be safe and well-tolerated with no drug-related serious adverse events.

Adverse events occurring in more than 10% of patients
Adverse event % n
Elevated urine glucose* 52 13
Procedural pain 40 10
Back pain 28 7
Musculoskeletal pain 20 5
Headache 20 5
Site bruise 16 4
Fatigue 16 4
Nasopharyngitis 16 4
Abdominal pain 12 3
Influenza-like event 12 3
Urinary tract infection 12 3
Muscle fatigue 12 3
Myalgia 12 3
Pain in extremity  12 3
Cough 12 3
No patients withdrew due to adverse event.
Most adverse events were considered mild to moderate.
*Study-drug related
Serious adverse events
Adverse event n Outcome
Urinary tract infection 1 Resolved
Aspiration pneumonia** 1 Resolved
Aspiration** 1 Death
Three adverse events occurred in 2 patients.
**Same patient

Improvements versus baseline were observed in multiple secondary efficacy endpoints related to dysphagia and muscle strength and function.

Based on the positive safety and efficacy signals, Bioblast Pharma intends to enroll more than 70 patients in a Phase 2b double-blind placebo-controlled clinical trial, at up to 15 sites in the United States and Canada, to confirm the results from HOPEMD.

Find additional information about the results of the HOPEMD Phase 2 study.
Download the Bioblast Corporate Presentation