Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from rare disorders. OT-58, our lead drug development candidate, has been optimized as an enzyme replacement therapy for classical homocystinuria, a genetic disease characterized by debilitating cardiovascular, skeletal, neurologic, and ophthalmologic complications. OT-58 is designed to reduce homocysteine levels via a targeted mechanism of action and may have therapeutic applications in other diseases. For more information, please visit www.orphantechnologies.com.
OT-58, Orphan Technologies’ lead drug candidate, is a modified recombinant enzyme replacement therapy in development as an enzyme replacement therapy for patients suffering from the rare disease classical homocystinuria. Classical homocystinuria is a genetic metabolic disease caused by a deficiency in the CBS enzyme leading to elevated levels of the amino acid homocysteine. OT-58 has consistently demonstrated significant reductions in homocysteine levels across multiple models of homocystinuria and has the potential to improve metabolic control, reduce or remove dietary restrictions, and positively impact clinical outcomes. OT-58 is anticipated to enter clinical evaluation in 2018 and has been granted Orphan Status by the US Food and Drug Administration and EMA. In addition, based on its mechanism of action, OT-58 has therapeutic potential in other diseases.