Navigation Links
SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing
Date:4/24/2012

TUCSON, Ariz., April 24, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has received approval from regulatory agencies in the United Kingdom to move from laboratory to human testing, starting with healthy human volunteers.

(Logo: http://photos.prnewswire.com/prnh/20120424/DC93207LOGO)

The drug is designed to increase production of a muscle protein utrophin that can potentially compensate for the one that's missing in DMD.

The Association has been supporting the development of SMT C1100 through its MDA Venture Philanthropy drug development program, via a grant to Summit Corporation PLC of Oxford, United Kingdom.

"We're extremely pleased that SMT C1100 will now move from testing in vivo to testing in healthy humans," said Jane Larkindale, MDA's director of translational research. "We have good evidence from laboratory studies that this drug's mechanism of action is valid for slowing the progression of this degenerative muscle disease, and we hope that this first trial will show that it can be effectively and safely delivered to humans."

Summit's Chief Scientific Officer Richard Storer said: "Utrophin upregulation will be beneficial to all DMD boys, and SMT C1100 has demonstrated disease-modifying potential in nonclinical efficacy studies. This trial will evaluate if our improved formulation of SMT C1100 can produce consistent levels of the drug in the blood which, based on the preclinical studies, might be expected to have a therapeutic benefit in patients."

About SMT C1100

SMT C1100 is designed to boost production of the muscle protein utrophin. Utrophin closely resembles and can mimic some of the functions of dystrophin, the protein missing in the muscle fibers of patients with DMD.

In May 2011, an MDA-supported study showed that daily treatment with SMT C1100 dramatically reduced symptoms in dystrophin-deficient mice with a DMD-like disease. The mice showed reduced muscle abnormalities, increased overall strength and improved ability to resist exercise-related fatigue.

An earlier version of the drug, tested by California-based BioMarin Pharmaceutical, was found safe and well-tolerated in healthy human volunteers, but it failed to reach blood levels considered adequate for therapy.

The reformulated Summit PLC version of the drug is designed to reach adequate blood levels in humans following oral administration.

To learn more, read the Quest News Online article DMD Drug SMT C1100 Moves to Human Testing.

About DMD

DMD is a degenerative muscle disease — affecting boys almost exclusively — that involves progressive degeneration of voluntary and cardiac muscles, with resulting weakness and heart abnormalities. The disease manifests in early childhood, causing delayed motor milestones and, in some cases, cognitive, behavioral or language deficits. Loss of the ability to walk occurs in most children with DMD between the ages of 10 and 12 years; weakened cardiac and respiratory muscles severely limit life span.

The disease is caused by any number of mutations in the X-chromosome gene that carries instructions for the muscle protein dystrophin. Without dystrophin, muscle fibers are abnormally fragile and break down under the stress of contractions.

Corticosteroid medications, such as prednisone and its chemical cousin prednisolone, slow disease progression.

About Summit

Summit is an Oxford, U.K.-based drug discovery company with an innovative Seglin™ technology platform for the discovery of new medicines and a portfolio of drug program assets.  Summit's program portfolio consists of a number of drug programs targeting high-value areas of unmet medical need, including Duchenne muscular dystrophy and C. difficile infection. Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker symbol SUMM. Further information is available at summitplc.com.

About MDA Venture Philanthropy (MVP)

MVP is the Muscular Dystrophy Association's drug development program, which operates within MDA's translational research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

For more information, visit mda.org and follow MDA on Facebook (facebook.com/MDANational) and Twitter (@MDAnews).


'/>"/>
SOURCE Muscular Dystrophy Association
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Halo Therapeutics HT-100 Receives FDAs Orphan Designation for Duchenne Muscular Dystrophy
2. MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy
3. MDA Collaborates with AVI BioPharma on First Phase 2 Placebo-Controlled Clinical Trial of Exon-51 Skipping Drug as Potential Therapy for Duchenne Muscular Dystrophy
4. PTC Therapeutics and Parent Project Muscular Dystrophy Advance Drug Discovery Program to Improve Heart Function in Duchenne/Becker Muscular Dystrophy Patients
5. PTC Therapeutics Announces Additional Study of Ataluren in Patients with Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
6. Data Published in Muscle and Nerve Validates the Six-Minute Walk Test as an Outcome Measure in Duchenne Muscular Dystrophy
7. PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy
8. PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy
9. PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
10. PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
11. MDA Awards $1 Million to Tivorsan Pharmaceuticals for Accelerating Pivotal Pre-Clinical Work on TVN-102 as a Potential Muscular Dystrophy Treatment
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/4/2017)... Oct. 4, 2017  South Korean-based healthcare product Development ... aide "cprCUBE" on Kickstarter. The device will educate the ... arrests with better efficiency compared to the dated and ... feedback on efficacy of the compression for a more ... a goal to raise $5,000. ...
(Date:10/2/2017)... 2017  AllianceRx Walgreens Prime, the combined central specialty ... pharmacy benefit manager Prime Therapeutics LLC (Prime), today officially ... the unveiling of new signage at its headquarters in ... at a few other company-owned facilities across the country. ... some of whom will begin to see the AllianceRx ...
(Date:9/27/2017)... MIAMI , Sept. 27, 2017  Commended for their ... recent notable awards. Ranked as number one in the South ... ninth time in Inc. 5000 yearly list, the national specialty ... CEO, Armando Bardisa will soon be honored by ... Set to receive his award ...
Breaking Medicine Technology:
(Date:10/13/2017)... ... ... Apple Rehab Shelton Lakes , which specializes in the delivery of sub-acute ... of a disaster drill on October 3rd. , Apple Rehab participated with the Shelton ... well as the Connecticut Long Term Care Mutual Aid Plan (LTC-MAP). The LTC-MAP ...
(Date:10/13/2017)... New York, NY (PRWEB) , ... October 13, 2017 , ... ... most influential people in business to advocate for action towards gender equality at their ... 18,000 views from around the globe, and reached a social audience of over 3 ...
(Date:10/13/2017)... ... October 13, 2017 , ... Talented host, ... lowdown on sciatica in a new episode of "Success Files," which is an ... and innovation and investigates each subject in-depth with passion and integrity. , Sciatica ...
(Date:10/13/2017)... , ... October 13, 2017 , ... “America On The ... identity. “America On The Brink” is the creation of published author, William Nowers. ... great-grandchildren. As a WWII veteran, he spent thirty years in the Navy. ...
(Date:10/12/2017)... ... 2017 , ... Planet Fitness, one of the largest and fastest growing franchisors ... a flagship location in Covington, LA at 401 N. U.S. Highway 190, in January ... Office Depot in the Holiday Square shopping center. Its location allows it to serve ...
Breaking Medicine News(10 mins):