Patient advocacy community for Duchenne muscular dystrophy joins to accelerate development of potential therapy for DMD boys
Newton, Mass.—May 22, 2012—Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that it has received financial support totaling $1.1 million from 12 not-for-profit foundations serving the muscular dystrophy patient community. Halo will use the funds to expedite the phase 2 study of HT-100, its lead drug candidate for Duchenne muscular dystrophy (DMD). HT-100, a proprietary formulation of halofuginone, is an orally available small molecule drug candidate being developed to reduce fibrosis and promote healthy muscle fiber regeneration in DMD patients. Halo will begin its phase 2 study in the second half of 2012.
“Companies typically labor for years to attract the level of engagement we have received from such an active, dedicated patient advocacy community,” said Marc Blaustein, CEO of Halo Therapeutics. “We are both humbled and heartened by their support and believe that together we are better able to speed the delivery of new therapies for DMD to patients and their families.”
DMD is a progressive and fatal neuromuscular disorder, which afflicts approximately 1 in 3,500 boys worldwide. There is currently no treatment. Muscle fibrosis is a primary outcome and ultimately leads to early patient death: Young men with DMD typically live into their twenties or early thirties. In studies, HT-100 has demonstrated potent ability to halt and reverse existing muscle fibrosis as well as promote the growth of healthy new muscle. In an independent review, an international advisory committee of neuromuscular disease experts concluded HT-100 was “ready for the clinic.” The drug candidate has received Orphan status in both the U.S. and the EU.
“The community of muscular dystrophy foundations has rallied around our novel and aggressive approach to delivering a promising therapy to DMD boys and their families,” said Tom Wicka, founder of the Nash Avery Foundation and father of Nash, age 12. “This level of unification and engagement across the patient advocacy community for an early-stage drug candidate is extremely rare, and we are grateful for the support and vision of our funding partners.”
Added Benjamin Seckler, M.D., president of Charley’s Fund and father of Charley, age 11: “We have the ultimate drive to get this done quickly: Our children’s lives are at stake. We do not have the option of accepting the pace of typical drug development efforts. We thank our foundation partners and value their role in helping us further the development of HT-100 as rapidly as possible.”
The foundations providing support include:
The Duchenne Research Fund
Hope for Gus
Hope for Javier
Parent Project Muscular Dystrophy
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.
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