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Halo Therapeutics' HT-100 Receives FDA's Orphan Designation for Duchenne Muscular Dystrophy

NEWTON, Mass., Jan. 4, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, today announced that the United States Food and Drug Administration (FDA) granted orphan drug designation for HT-100 for treatment of Duchenne muscular dystrophy (DMD). DMD is a progressive and fatal neuromuscular disorder, which afflicts approximately 1 in 3,500 boys worldwide. HT-100, also known as halofuginone, is an orally available small molecule drug candidate that is being developed to reduce fibrosis and promote healthy muscle fiber regeneration.

The FDA grants orphan designation to promote development of therapies to treat rare diseases. Once this designation is granted, the sponsor may be eligible for a range of incentives including FDA grant funding for clinical trial costs, tax credits related to development expenses, waiver of FDA user fees, and a seven-year period of marketing exclusivity in the U.S. following FDA approval.

Halo Therapeutics has an innovative business model. The company was founded and funded to date by two DMD patient foundations, Charley's Fund ( and the Nash Avery Foundation (, and executive management. The company is led by biopharma veterans Marc Blaustein as CEO and Gene Williams as Executive Chairman, and has assembled a team of experienced drug developers and DMD experts to rapidly evaluate HT-100 for DMD. Mr. Blaustein brings to Halo 15 years of experience in the biopharmaceutical industry, most recently at Dyax Corp. and Alkermes. He has directed manufacturing, commercial operations, and business development functions and led development efforts for two products that subsequently received FDA approval. Mr. Williams brings decades of industry experience, including directing the fibrotic disease portfolio at Genzyme and founding multiple life science and healthcare companies. 

"We are in a race against time to find safe and effective therapies for boys with DMD," said Benjamin Seckler, MD, President of Charley's Fund. "Our partnering with Nash Avery Foundation to establish Halo Therapeutics is the next step in the DMD patient community's tireless effort to find such a therapy. We're excited to have Gene and Marc, experienced drug development and biopharma industry business professionals, leading the company."

HT-100 works by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle fiber regeneration. The company has acquired an extensive data package on HT-100 including results of numerous animal model studies, a comprehensive toxicology package, and human clinical data from multiple trials in healthy volunteers and select disease populations.

Planning is well underway for clinical evaluation of the compound. CEO Marc Blaustein said, "We're extremely pleased that HT-100 has received orphan drug designation. This adds momentum to the significant progress we've made since our founding, and we are pressing forward to initiate a Phase II clinical trial in 2012 to evaluate HT-100 in DMD patients."About Duchenne Muscular DystrophyDuchenne 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 TherapeuticsBased 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 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

 Media Contacts Name:

Marc B. BlausteinCompany:

Halo Therapeutics, LLCPhone Number:

617.431.7250Email address:


SOURCE Halo Therapeutics, LLC
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