OT-58

OT-58
molecule

What is OT-58?

OT-58 is a modified enzyme therapy in development for patients suffering from the rare disease classical homocystinuria. OT-58 is designed to help patients reduce their homocysteine levels, improve patient health, and restore a normal lifestyle.

How OT-58 works

OT-58 is designed to address the underlying cause of classical homocystinuria — a deficiency in the naturally occurring enzyme cystathionine beta synthase (CBS). Reduced CBS activity results in the inability to metabolize, or process, homocysteine. This can lead to dangerous elevations of homocysteine and symptoms including (please list). OT-58, is a modified enzyme therapy that may dramatically decrease tissue and plasma levels of homocysteine, and as a result may prevent, delay, and reverse clinical abnormalities, and reduce dietary restrictions in homocystinuria, and other diseases that exhibit elevated homocysteine values.

OT-58, is a modified recombinant CBS enzyme therapy that may dramatically decrease tissue and plasma levels of homocysteine.

Clinical development plan

OT-58 Phase 1/2 Enzyme Therapy Clinical Trial

Orphan Technologies has initiated the first-ever human study of OT-58, a modified version of the human enzyme that is not functioning well in patients with classical homocystinuria. The goal of this trial is to evaluate the safety and efficacy of OT-58 in patients with classical homocystinuria and identify the appropriate dose. Patients between the ages of 12 and 65 years of age with classical homocystinuria are eligible to join. If you are interested in participating, please see the trial information on ClinicalTrials.gov

Natural History Study

Orphan Technologies is conducting the first-ever prospective natural history study of patients with classical HCU to better understand how homocystinuria progresses over time and to help design new treatments for patients living with homocystinuria.

The goal of this study is to observe patients with classical HCU to learn how their disease is managed under standard care. This study does not involve any investigational medications but will provide information important to researchers hoping to develop a medication to treat the disease as well as to regulators. Patients between the ages of 5 and 65 years of age with classical HCU may be eligible to join.