Orphan Technologies begins OT-58 phase 1/2 trial in classical homocystinuria

OT-58 clinical trial news : Swiss pharma company Orphan Technologies has dosed the first patients in a phase 1/2 trial, called CBS-HCY-01, of its modified recombinant enzyme therapy OT-58 in classical homocystinuria.

OT-58 has been designed to dramatically reduce tissue and plasma levels of homocysteine, thereby potentially preventing, delaying, and reversing clinical abnormalities, and lifting of dietary restrictions.

A rare metabolic disorder, classical homocystinuria (HCU) leads to debilitating cardiovascular, neurologic, skeletal, and ophthalmologic complications.

Danae Bartke – Executive Director of HCU Network America said: “Patients living with homocystinuria today suffer from severe side effects of the ophthalmic, skeletal, cardiovascular and neurocognitive systems yet there are no effective treatments for this disease.

“Many patients remain at risk for the life-threatening consequences of HCU. OT-58 is a potentially transformative therapy for patients with any elevated level of homocysteine.”

The CBS-HCY-01 trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical effects of OT-58 in subjects with cystathionine beta-synthase deficient homocystinuria.

The phase 1/2 trial will see the enrollment of up to 20 patients.

The primary endpoint of the phase 1 part of the study is safety while secondary endpoints are assessment of pharmacokinetic and pharmacodynamic parameters among others.

Commenting on the OT-58 clinical trial, J. Frank Glavin – CEO of Orphan Technologies, said: “Orphan Technologies is committed to reducing the burden of patients suffering from homocystinuria, who currently have no viable treatment options.

“We believe that OT-58 may be a dramatic advance for these patients and we look forward to the results of this new study. In parallel, we are conducting a comprehensive and prospective natural history study of patients with classical homocystinuria as part of our exhaustive approach to understanding and treating this underdiagnosed and underserved disease.”

In 2013, Orphan Technologies signed exclusive, worldwide licensing and collaboration agreements with The University of Colorado to develop the enzyme replacement therapy for cystathionine beta-synthase deficient homocystinuria.