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Orphan Technologies Announces First Patients Treated in Phase 1/2 Trial of OT-58 in Homocystinuria

February 6, 2019

LEXINGTON, Mass.--(BUSINESS WIRE)--Feb 6, 2019--Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, today announced that the first patients have been treated in a Phase 1/2 clinical trial of OT-58 in patients suffering from the rare disease classical homocystinuria. OT-58 is a novel, recombinant enzyme therapy designed to reduce plasma and tissue homocysteine levels. Classical homocystinuria (HCU) is a rare metabolic disorder that causes debilitating cardiovascular, neurologic, skeletal, and ophthalmic side effects.

“Classical homocystinuria is a devastating genetic metabolic disease, yet the current standard of treatment for patients is inadequate and decades-old,” stated Harvey Levy, MD, Senior Physician in Medicine, Division of Genetics and Genomics’ Professor of Pediatrics, Harvard Medical School. “Biomarker and clinical data from an ongoing natural history study of classical homocystinuria demonstrate that many patients receiving the current standard of care for this disease do not receive sufficient reduction in homocysteine levels to offset the advance of this devastating disease.”

“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,” said Danae’ Bartke, Executive Director of HCU Network America. “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.”

Homocystinuria is a disease that results from significantly elevated levels of the amino acid homocysteine that can result in debilitating effects in patients including severe cardiovascular, skeletal, neurologic and ophthalmologic complications. Classical homocystinuria is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme in the conversion of the amino acid methionine to homocysteine and then to cysteine. The mainstay treatment for patients with homocystinuria is a severely protein restricted diet. Compliance with these dietary restrictions is intractable and regularly results in inadequate metabolic and disease control leading to severe medical outcomes.

“Orphan Technologies is committed to reducing the burden of patients suffering from homocystinuria, who currently have no viable treatment options,” commented J. Frank Glavin, CEO of Orphan Technologies. “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.”

About the Phase 1/2 Clinical Trial of OT-58 in Homocystinuria The CBS-HCY-01 study is a double-blind, randomized, placebo-controlled, phase 1/2 study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical effects of OT-58 in patients with cystathionine beta-synthase deficient homocystinuria. The study will enroll up to 20 patients. The primary endpoint of the Phase 1 portion of the study is safety. Secondary endpoints include evaluation of pharmacokinetic and pharmacodynamic parameters. More information on the OT-58 Clinical Study, including participation criteria, is available here.

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

About the Homocystinuria Natural History Study Orphan Technologies is conducting the first-ever prospective natural history study of patients with classical homocystinuria to understand how homocystinuria progresses over time and also to help design new treatments for patients living with the disease. The goal of this study is to observe patients over 3 years to learn how their disease is managed under standard of care. This study does not involve any investigational medications but will provide information to researchers who are currently developing a medication to treat the disease. More information about, including participation criteria for the Natural History Study of Cystathionine Beta-synthase Deficiency Homocystinuria (CBSDH), is available here.

About Orphan Technologies Orphan Technologies is dedicated to developing novel therapies to dramatically improve the lives of patients suffering from the rare disorder homocystinuria, and related diseases. OT-58, our lead drug development candidate, has been optimized to reduce plasma and tissue levels of homocysteine. Our lead indication is in 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

View source version on businesswire.com:https://www.businesswire.com/news/home/20190206005358/en/

CONTACT: Orphan Technologies

J. Frank Glavin

Glavin.f@orphantechnologies.comMedia

Cory Tromblee

617.571.7220

KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS

INDUSTRY KEYWORD: HEALTH BIOTECHNOLOGY CLINICAL TRIALS GENETICS PHARMACEUTICAL

SOURCE: Orphan Technologies

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PUB: 02/06/2019 08:00 AM/DISC: 02/06/2019 08:00 AM

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