Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



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Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



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Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD: "Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy (DMD). The preclinical data, which were presented at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, demonstrated that a single administration of SGT-001 resulted in sustained and significant microdystrophin expression and improvements in muscle function, with no observed immune response. The Company plans to initiate clinical trials for SGT-001 in the second half of 2017."



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Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Researchers correct Duchenne muscular dystrophy using gene-editing alternative: "Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

The UT Southwestern group had previously used CRISPR-Cas9, the original gene-editing system, to correct the Duchenne defect in a mouse model of the disease and in human cells. In the current work, they used a new variation of the gene-editing system to repair the defect in both a mouse model and in human cells."



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Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm - Neuromuscular Disorders

Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm - Neuromuscular Disorders: "Utrophin modulators significantly improve muscular dystrophy in the mdx diaphragm"



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Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Early treatment with heart failure drug can improve cardiac function in young boys with DMD: "Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne muscular dystrophy (DMD) and stabilize heart function in older boys with the disease."



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FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy: "The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States."



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What You Need to Know about Duchenne

What You Need to Know about Duchenne: "Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal pain/discomfort, cramps and diarrhea. Thank goodness they usually have a ‘short course,’ most lasting less than 24 hours."

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Immune system plays important role in Duchenne muscular dystrophy, research reveals

Immune system plays important role in Duchenne muscular dystrophy, research reveals: "A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches.

"The findings suggest that the immune system has an important role in the muscle disease of Duchenne muscular dystrophy," said Eric Hoffman, professor of pharmaceutical sciences and associate dean for research at Binghamton University's School of Pharmacy and Pharmaceutical Sciences."

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» GTx exploring product candidate as potential Duchenne treatment Action Duchenne

» GTx exploring product candidate as potential Duchenne treatment Action Duchenne: "GTx is exploring SARM, their lead product candidate, a selective androgen receptor modulator as potential treatment for Duchenne muscular dystrophy. 

Preclinical studies have confirmed the beneficial effects from SARMs in mice genetically altered to simulate Duchenne muscular dystrophy, compared to control groups. The Company continues to pursue a potential strategic collaboration with biopharma companies experienced in orphan drug development."

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Sarepta Therapeutics Inc. (SRPT) -Summit Therapeutics PLC (ADR) (SMMT) Deal Is "Low Risk, High Reward": Wedbush

Sarepta Therapeutics Inc. (SRPT) -Summit Therapeutics PLC (ADR) (SMMT) Deal Is "Low Risk, High Reward": Wedbush: "Sarepta Therapeutics Inc. (NASDAQ:SRPT) announced on Tuesday, a partnership deal with London-based Summit Therapeutics PLC (ADR) (NASDAQ:SMMT), for the development of drugs to treat Duchenne muscular dystrophy. As part of the deal, Sarepta will use its expertise to help develop Summit’s utrophin modulator pipeline and its key experimental DMD drug ezutromid in exchange for its European rights and a Latin America option. "



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PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to improve cardiac care and believe in the promising work Dr. Guttridge and his team at Ohio State are doing.
 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Almost all Duchenne patients develop heart problems. In order to treat the disease, new drugs will have to work by fixing both skeletal and heart muscles. In contrast to Duchenne skeletal muscles, very little is known about a dystrophic heart."



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PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to improve cardiac care and believe in the promising work Dr. Guttridge and his team at Ohio State are doing.
 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Almost all Duchenne patients develop heart problems. In order to treat the disease, new drugs will have to work by fixing both skeletal and heart muscles. In contrast to Duchenne skeletal muscles, very little is known about a dystrophic heart."



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LIF-treated muscle stem cells show promise in treatment of muscular dystrophy

LIF-treated muscle stem cells show promise in treatment of muscular dystrophy: "Satellite cells are stem cells found in skeletal muscles. While transplantation of such muscle stem cells can be a potent therapy for degenerative muscle diseases such as Duchenne muscular dystrophy, these cells tend to lose their transplantation efficiency when cultured in vitro. In a study in the current issue of the Journal of Neuromuscular Diseases, researchers treated these stem cells with leukemia inhibitory factor (LIF), which effectively maintained the undifferentiated state of the satellite cells and enhanced their transplantation efficiency.

To have enough cells for transplantation, they must be grown in vitro and prevented from differentiating before transplantation. Several growth factors, cytokines, and chemicals have been used in muscle stem cell cultures, but the optimal culture conditions required to maintain the undifferentiated state, inhibit differentiation, and enhance eventual transplantation efficiency have not yet been established."



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New Duchenne muscular dystrophy treatment 'ready in five years' - BBC News

New Duchenne muscular dystrophy treatment 'ready in five years' - BBC News: "A new treatment for a rare muscle wasting disease could be available within five years, an Oxford University researcher has said.
Duchenne muscular dystrophy (DMD) affects about 2,500 boys and men in the UK, leaving them unable to move.
Professor Kay Davies said trials of a drug to increase levels of the protein utrophin, to maintain the muscles, would start later this year.
She told Oxfordshire Science Festival it could help sufferers worldwide.
"Duchenne is horrible for these boys. They normally get diagnosed at the age of four of five, and they suffer from a progressive muscle wastage which leaves them in a wheelchair by the age of 12," she said.
"Somewhere in the next five years, we will be able to do something for these boys, to stop them from going into a wheelchair, and perhaps prolong their life and improve their quality of life."
"



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First Patient Enrolled in Summit’s PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Boys With DMD Nasdaq:SMMT

First Patient Enrolled in Summit’s PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Boys With DMD Nasdaq:SMMT: "OXFORD, United Kingdom, June 17, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, today announces that it has enrolled the first patient in PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid (formerly SMT C1100) in patients with DMD. Ezutromid dosing is expected to follow a screening period of up to 28 days.

Ezutromid is an orally administered small molecule that is designed to modulate utrophin, a protein that is structurally and functionally similar to the dystrophin protein. Dystrophin is essential for the healthy function of all muscles but is missing in patients with DMD. Utrophin modulation is a potential disease-modifying approach that could treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation."



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Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy

Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy: "Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug treatments for the disease, which currently has no cure.

Muscular dystrophy (MD) is an inherited genetic condition that gradually causes a weakening of muscles. Duchenne muscular dystrophy (DMD) is the most common, and one of the most severe types, of the disease. There are around 2,500 people in the UK living with DMD, which usually affects boys in early childhood and leads to progressively worsening disability and premature death.

In DMD, the stem cells that normally repair damaged muscle are impaired, for reasons that remain unclear. In this new study, published in Scientific Reports, researchers looked at the molecular composition of the environment within which these muscle stem cells are found, to investigate whether this could be responsible for impaired function."



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Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice: "Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper published in The Journal of Cell Biology. The study, "Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype" by Joana Capote and colleagues, adds support to the idea that osteopontin inhibitors could be used to treat patients with Duchenne muscular dystrophy (DMD).

DMD is a progressive, and ultimately fa"



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» Summit Reports Positive Interim Data from Phase 1 Testing a New Formulation of SMT C1100 in DMD Patients Action Duchenne

» Summit Reports Positive Interim Data from Phase 1 Testing a New Formulation of SMT C1100 in DMD Patients Action Duchenne: "Today, we announced preliminary interim results from an ongoing Phase 1 trial of a new formulation of SMT C1100. As you’ll see in the release pasted below, we just announced data from the first dose in patients (with up to 3 doses planned) and from healthy volunteers. The data are encouraging and the trial is proceeding to the next dose in patients. This new formulation Phase 1 trial is a separate trial to the Phase 2 PhaseOut DMD trial that we’ve been discussing recently. Some questions that you may have:"



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New Biomarkers for Utrophin Protein Levels Likely to Aid DMD Therapy Now in Clinical Testing - Muscular Dystrophy News

New Biomarkers for Utrophin Protein Levels Likely to Aid DMD Therapy Now in Clinical Testing - Muscular Dystrophy News: "Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) patients.

The study is important to the company because one of its lead products, now in clinical testing, is SMT C1100, a small molecule utrophin modulator that has been shown in a mice model of DMD to increase utrophin in skeletal and diaphragm muscle, leading to a significant decrease in disease pathology and an improved functional benefit. Utrophin up-regulation is considered a possible therapeutic option for all DMD patients, regardless of their underlying dystrophin mutation."



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