TACT believes HT-100 has potential

HT-100 for Duchenne muscular dystrophy has been reviewed by the TREAT-NMD Advisory Committee on Therapeutics (TACT), who believes the compound has potential. The disease is rare, with only around 20,000 new cases a year, and its journey to get to this stage has been an innovative case study in the emerging phenomena of the new model of do-it-yourself drug development, i.e., the joint efforts by two families, guided by expert advice, to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy. For the complete drug development story, see link

http://rareproject.org/2011/12/27/wall-street-journal-reports-do-it-yourself-drug-development-with-some-help/

Full press release is below.

Source: www.halotherapeutics.com

Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100

International Advisory Committee of Neuromuscular Disease Experts Finds HT-100 "Ready for the Clinic" in Voluntary Review

NEWTON, Mass., March 20, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism. TREAT-NMD is a global network dedicated to expediting the delivery of promising new therapies to patients with neuromuscular disease. TACT provides independent, objective guidance on the therapeutic potential of drug candidates (novel or repurposed), which are submitted for review on a voluntary basis. In its review of HT-100, TACT found the drug candidate to be "ready for the clinic."

"TACT offers the opportunity for objective review of potential new therapies to help industry, researchers and patient advocacy groups. The review provides multi-disciplinary advice on the development of the potential therapies in the context of a realistic development pathway," said Volker Straub of TREAT-NMD. "TACT evaluated the proposal from Halo Therapeutics and felt it was well prepared, and that the compound has potential based on the proposed mechanism. Overall the plan to progress to a clinical trial is realistic."

DMD is a progressive and fatal neuromuscular disorder that afflicts approximately 1 in 3,500 boys worldwide.  HT-100, Halo's proprietary formulation of halofuginone, is an orally available small molecule drug candidate intended to reduce fibrosis and promote healthy muscle fiber regeneration. HT-100 works by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle fiber regeneration. The U.S. Food and Drug Administration recently granted orphan drug designation to HT-100. The company will begin a phase 2 study in the second half of 2012.

"We submitted HT-100 for a TACT review knowing that informed, objective feedback on our pre-clinical data and development plans would be invaluable to our efforts," said Marc Blaustein, CEO of Halo Therapeutics. "We believe their recommendations for HT-100's advancement will hasten our progress toward delivering a safe and effective new therapy for DMD patients and their families."

A brief summary of TACT's review of HT-100 can be found at http://www.treat-nmd.eu/resources/tact/reviews/past/halofuginone-ht-100/.

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 TREAT-NMD 
TREAT-NMD is a neuromuscular network that has developed the infrastructure to ensure that promising therapies reach patients as quickly as possible. TREAT-NMD was funded as a network of excellence by the EU with the FP6 programme (contract number: 036825) from January 2007 – December 2011.  Since its launch the network's focus has been on the development of tools that industry, clinicians and scientists need in order to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice clinical care for neuromuscular patients worldwide.

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.

Media Contacts
Name:	Marc B. Blaustein
Company:	Halo Therapeutics, LLC
Phone Number:	617.431.7250
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