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:4/28/2016)... 2016 , Net Sales of $1.90 billion ... the prior year period, and an increase of 1.2% on ... EPS for the first quarter were $0.52 reported, a decrease ... an increase of 29.9% over the prior year period ... guidance for 2016 Zimmer Biomet Holdings, Inc. (NYSE ...
(Date:4/28/2016)... N.J. , April 28, 2016   Acsis ... today announced that leading IT market research and advisory ... " in the IDC MarketScape: Worldwide Pharmaceutical Track and ... 2016).  The report provides an assessment of the capabilities ... track and trace software market. Logo - ...
(Date:4/28/2016)... 28, 2016 New market ... is a report that provides an overview of ... pipelines by identifying new targets and MOAs to ... Profiles discussed in this H1 2016 Osteoarthritis Pipeline ... AbbVie Inc., Abiogen Pharma S.p.A., Ablynx NV, Achelios ...
Breaking Medicine Technology:
(Date:4/29/2016)... ... 2016 , ... Since launching its annual volunteer campaign on ... footwear industry, has broken all previous participation records in its first two weeks ... during the months of April and May, the 2016 Footwear Cares initiative is ...
(Date:4/29/2016)... ... 29, 2016 , ... Shamangelic Healing, Sedona Arizona's Premier ... Alpha BRAIN and New Mood Daily-Stress Formula for brain optimization and wellness to ... the store is just one more way Shamangelic Healing supports people’s quest for ...
(Date:4/29/2016)... (PRWEB) , ... April 29, 2016 , ... On Tuesday, ... hospitals across the Southeast, celebrated the signature of Gov. Nathan Deal on SB 258, ... Geoff Duncan (R - Cumming), offers a 70% tax credit to individuals and corporations ...
(Date:4/29/2016)... ... April 29, 2016 , ... Dr. ... of best seller "LOVE, MEDICINE and MIRACLES") addresses touchy topics related to Death ... podcasted thereafter . Dr. Bernie Siegel, author of a plethora of essential ...
(Date:4/29/2016)... ... April 29, 2016 , ... Conditions were ideal for Global ... Park on Sunday, with sunny skies, a light breeze and temperatures in the 60s. ... , The 5k Run and Walk and 1-mile walk were held to increase ...
Breaking Medicine News(10 mins):