European Medicines Agency Grants Orphan Medicinal Product Designation
to Halo Therapeutics’ HT-100
Supplements FDA Orphan Drug Status; HT-100 to Enter Phase 2 Study in 2012
Newton, Mass.—May 3, 2012—Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the European Commission (EU) has granted Orphan Medicinal Product designation to HT-100 for the treatment of Duchenne Muscular Dystrophy (DMD). HT-100, Halo Therapeutics’ proprietary formulation of halofuginone, is an orally available small molecule drug candidate that is being developed to reduce fibrosis and promote healthy muscle fiber regeneration in DMD patients. DMD is a progressive and fatal neuromuscular disorder, which afflicts approximately 1 in 3,500 boys worldwide. Orphan Medicinal Product designation follows receipt of Orphan Drug Designation in the U.S., which the company announced in January 2012. HT-100 will enter a phase 2 study in 2012.
In the EU and U.S., orphan designation is intended to promote development of therapies to treat rare diseases. It confers a range of unique benefits to the manufacturer, which in the EU include protocol assistance, reduced or eliminated registration and market authorization fees, centralized authorization for EU member countries and ten years of market exclusivity. To qualify, the drug candidate must be used to treat life-threatening or chronically debilitating conditions that affect less than 5 in 10,000 people in the EU and for which there is no satisfactory treatment.
“Finding a treatment for DMD is very much a race against time,” said Marc Blaustein, CEO of Halo Therapeutics. “Orphan designation for HT-100 in both the EU and U.S. will help accelerate our mission to deliver a safe, effective therapy for DMD to patients and their families as rapidly as possible.”
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, resulting in progressive muscle weakness. The disease manifests itself first in weakened skeletal muscles and eventually results in cardiac and pulmonary impairment. Corticosteroids are the current standard of care treatment for DMD. While this treatment delays disease progression by several years, their prolonged use is typically associated with side effects and the treatment does not alter the ultimate outcome of the disease. Young men with Duchenne typically live into their twenties or early thirties.
About Halo Therapeutics
Based in Newton, Mass., Halo Therapeutics, LLC, is a clinical-stage biopharmaceutical firm focused on developing innovative therapies for rare fibrotic diseases like Duchenne muscular dystrophy. Founded through a collaboration between patient advocacy organizations and industry veterans, the company is partnering with the clinical community and patients to transform therapeutic options and, by extension, lives. For more information, please visit www.halotherapeutics.com.
Name: Marc B. Blaustein
Company: Halo Therapeutics, LLC