Navigation Links
Halo Therapeutics' HT-100 Receives FDA's Orphan Designation for Duchenne Muscular Dystrophy
Date:1/4/2012

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 (www.charleysfund.org) and the Nash Avery Foundation (www.nashaveryfoundation.org), 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 www.halotherapeutics.com.

 Media Contacts Name:

Marc B. BlausteinCompany:

Halo Therapeutics, LLCPhone Number:

617.431.7250Email address:

mblaustein@halotherapeutics.com


'/>"/>

SOURCE Halo Therapeutics, LLC
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Echo Therapeutics Announces $3.6 Million Warrant Exercises
2. Echo Therapeutics to Present at Biotech Showcase 2012 in San Francisco
3. Orexigen® Therapeutics Completes Public Offering of Common Stock and Warrants to Purchase Common Stock
4. AuraSense Therapeutics Secures Series B Equity Investment
5. PTC Therapeutics Antibacterial Program Receives $5 Million Award from the Wellcome Trust
6. Arecor Forms Collaboration With Lilly to Develop Advanced Formulations of Protein Therapeutics
7. Cerevast Therapeutics Receives CE Mark for Clotbust ER™ Ultrasound Stroke Treatment Device
8. Orexigen® Therapeutics Announces Proposed Public Offering of Common Stock and Warrants to Purchase Common Stock
9. Regulus Therapeutics Initiates an Orphan and Rare Disease microRNA Therapeutic Effort in Brain Cancer
10. Cell Therapeutics, Inc. Announces Institutional Investors Purchase $20 Million of Preferred Stock and Warrants
11. Pearl Therapeutics Reports Positive Phase 2b Results of PT001 in Patients With COPD
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/14/2017)... , June 14, 2017  In 2016, ... Creative Startups pitch competition and came away with ... platform is described by Forbes as "entering the life ... Medical Association as teaching "empathy to medical professionals in ... startup was recently named a finalist for the Department ...
(Date:6/9/2017)... , June 9, 2017 AirXpanders, Inc. (ASX: ... on the design, manufacture, sale and distribution of the ... the progress of its commercial roll-out in ... in more than one hundred (100) medical institutions and ... AeroForm offers a needle-free alternative for women who choose ...
(Date:6/7/2017)... , June 7, 2017 Endo International plc ... 7, 2017, the Hon. Joseph R. Goodwin , ... West Virginia , entered a case ... Pelvic Repair System Products Liability Litigation (the "MDL") that ... cases to provide expert disclosures on specific causation within ...
Breaking Medicine Technology:
(Date:6/27/2017)... , ... June 27, 2017 , ... DrugDev ... is whether they can trust the sponsor to pay them correctly and on time. ... and CROs establish payment strategies that encourage sites to work on their studies. , ...
(Date:6/27/2017)... , ... June 27, 2017 , ... The Congressional Budget ... to repeal and replace the 2010 Patient Protection and Affordable Care Act (ACA), would ... what would be expected under current law. , More than 20 million Americans have ...
(Date:6/27/2017)... ... June 27, 2017 , ... A January 18th article on ... staggering $6.81 billion by the year 2024 according to a new report. The article ... those made from titanium. Los Angeles area clinic Beverly Hills Periodontics & Dental Implant ...
(Date:6/27/2017)... York, NY (PRWEB) , ... June 27, 2017 ... ... and marketing experts to contribute to an upcoming case studies guidebook. This guidebook ... her organization. , This book will include articles, case studies and how-to’s that ...
(Date:6/27/2017)... CT (PRWEB) , ... June 27, 2017 , ... Dr. ... the recent renovation of his practice, Advanced Periodontics and Dental Implant Center of Connecticut. ... increased the administrative and waiting areas. The renovations are intended to improve patient comfort ...
Breaking Medicine News(10 mins):