Duchenne Muscular Dystrophy: Idebenone Slows Respiratory Decline in Phase 3 Trial

NEUROLOGY TODAY

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Friday, April 24, 2015

April 24—Duchenne Muscular Dystrophy: Idebenone Slows Respiratory Decline in Phase 3 Trial

BY RICHARD ROBINSON

WASHINGTON—Idebenone slows the decline of respiratory function in boys with Duchenne muscular dystrophy (DMD), according to a yearlong, double-blind phase 3 trial in 64 boys. Lead investigator Gunnar Buyse, MD, PhD, a professor of pediatrics and child neurology at the University of Leuven in Belgium, presented the results here on Friday at the AAN Annual Meeting.

Boys receiving 900 mg per day of idebenone for 52 weeks experienced a non-significant decline in peak expiratory flow as percent predicted (PEF%p; -3%p), the primary outcome measure, compared with a significant decline (-9%p) in those receiving placebo. This represents a 66% reduction in loss of PEF%p. PEF%p is a measure of peak flow (respiratory strength) compared with height -corrected norms for the population. Other respiratory measures were also significantly different between the two groups.

“This is the first ever successful phase 3 trial in this complex disease,” Dr. Buyse told the Neurology Today Conference Reporter before the session.

The major reason behind the problem of impotence is generic viagra soft no more an issue of concern but if the problem persists for half or more times you encounter a sexual intercourse then the problem demands treatments. Causes prompting this health cialis 100mg pills issue in men include relationship issues, depression, prostate gland injury, vascular disorder, etc. sildenafil price Ammonia itself is a by-product of intensive exercise, and without the urea cycle the body would rapidly become polluted. However, cialis from india tadalafil working through such issues with a partner can help for preventing impotence condition. Idebenone is a synthetic quinone compound with both electron transfer and antioxidant effects, Dr. Buyse said. “We know that in DMD cells there is a problem with oxidative stress, as well as with energy production.” Idebenone works in the mitochondrial electron transport chain to increase energy production and reduce oxidation, he explained.

In the mdx mouse model of DMD, idebenone has been shown in blinded experiments to improve cardiac function over nine months of treatment, as well as to increase the level of voluntary exercise. A successful phase 2 trial of idebenone in DMD set the stage for the current trial.

Enrolled patients were between 10 and 18 years old and were not currently taking steroids; about 90 percent had progressed to requiring a wheelchair for mobility. Peak expiratory flow was chosen as the primary endpoint after conferring with regulators, Dr. Buyse said. It is a direct measure of strength or weakness of the expiratory muscles, unlike forced vital capacity, which can also reflect the degree of scoliosis or other comorbidities. “There is good evidence that peak flow is an earlier and more sensitive parameter than forced vital capacity,” he said.

In addition to the effect on peak expiratory flow, the benefits of treatment were seen on forced vital capacity and forced expiratory volume in one second. The researchers observed no difference in benefit between boys who had or had not previously used steroids. Treatment was safe and well tolerated. “It is really a coherent picture showing the beneficial effect of the intervention,” Dr. Buyse said.

Based on the results from this trial, the investigators plan to apply for regulatory approval in both the United States and Europe. If the drug is approved, it will be considered for use earlier in the disease than was tested in the trial. “In principle, there is no reason idebenone would not work earlier,” Dr. Buyse said. Based on both its mechanism of action and some subanalyses showing that younger patients in the trial benefited equally compared with older ones, he added, “there is every reason to think that the sooner you start it, the better it would work.”

Kathryn R. Wagner, MD, PhD, director of the Center for Genetic Muscle Disorders at the Kennedy Krieger Institute in Baltimore, MD, cautioned, however, that these results apply directly only to boys not currently taking steroids. “From this trial, we do not know if idebenone will provide benefit to boys who are receiving this standard-of-care treatment,” said Dr. Wagner, who was not involved with the study.

A further caveat is that PEF%p “is not a measure that is used clinically in DMD, and it is difficult to know its relevance,” Dr. Wagner said, adding that this could be a concern unless it can be shown to be predictive of a more clinically relevant measure, such as the need for ventilation or decreased respiratory infections.

Nonetheless, she said, “Novel drugs are desperately needed in Duchenne muscular dystrophy. We assume that drugs that improve skeletal muscle function will improve pulmonary function, but this may be an oversimplification. A trial focusing on adolescents and pulmonary function is therefore welcomed.”

Plaque Array method links blood plaque components to Alzheimer’s

Click here to read full article…

Study on Plaxgen
Alzheimer’s disease blood
diagnostic published in
peer-reviewed journal

BY LLOYD DUNLAP

SUNNYVALE, Calif.—Plaxgen Inc., a developer
of flow cytometry-based blood diagnostics for
plaque-associated illnesses such as atherosclerosis
and Alzheimer’s disease, announced that a paper
validating its Plaque Array technology for the diagnosis
of Alzheimer’s disease was published in the
peer-reviewed journal Clinica Chimica Acta.
In a study titled “Plaque Array Method and Proteomics-
based Identification of Biomarkers from
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that the Plaque Array methodology developed by
Plaxgen works well for detecting amyloid beta
(Abeta) and other particle formation in the blood
serum of subjects clinically determined to have
Alzheimer’s disease (AD).
As stated in the paper’s abstract, progressive
accumulation of amyloid plaques in the regions
of brain, carotid and cerebral arteries is the leading
cause of Alzheimer’s disease and related dementia
in affected patients. The early identification
of individuals with AD remains a challenging
task reliant on symptomatic events, and thus the
development of a biomarker-based approach will
significantly aid in the diagnosis of AD.

Results from Phase III trial of Raxone/Catena in patients with DMD published

euroPharmReview

21 April 2015  •  Author: Victoria White

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Santhera Pharmaceuticals have announced that the full results of the double-blind placebo-controlled Phase III trial (DELOS) demonstrating efficacy and safety of Raxone®/Catena® in patients with Duchenne muscular dystrophy (DMD) have been published in The Lancet.

euroPharmReview2The results of the DELOS trial demonstrated that Raxone/Catena (INN: idebenone) significantly reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted, PEF%p) by 66% compared to patients taking placebo. Other respiratory function endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume (FEV1) corroborated these results and showed a consistent pattern with treatment differences supporting efficacy of Raxone/Catena over placebo in the preservation of respiratory function. Researchers concluded that Raxone/Catena represents a new treatment option for DMD patients.

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Gunnar M. Buyse, MD, PhD, Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS trial, said, “Statistically significant and clinically relevant outcomes of primary and secondary endpoints coherently demonstrated that Raxone/Catena reduced the loss of respiratory function and that it was safe and well tolerated. I am very enthusiastic about the positive data from the trial which demonstrate that this drug represents a suitable treatment option to ameliorate a life-threatening complication of the disease.”

“With morbidity and mortality in DMD being associated with progressive restrictive lung disease and irreversible loss of lung function, these findings represent an important treatment effect and are of major clinical relevance for patients with DMD”, added Craig McDonald, MD, Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis (USA) and investigator of the DELOS trial.

“The degree of slowing of respiratory function loss demonstrated in DELOS is of major clinical relevance for patients with DMD”, commented Nicholas Coppard, PhD, SVP Development at Santhera. “Based on this benefit and its well-established safety profile, we are very excited about the prospects of Raxone/Catena as a treatment option for DMD patients and we are currently preparing the regulatory filing dossier for application of marketing authorization both in the US and Europe.”

 

EuroBiotech: More Articles of Note

EuroBiotechReportEuroBiotech: More Articles of Note

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Santhera Pharmaceuticals (SIX:SANN) picked up a fast-track designation from FDA for its Duchenne muscular dystrophy drug, Catena. Phase III data on the treatment–which is known as Raxone in Europe–is currently being prepared for a new drug application. Santhera expects to meet with the FDA to discuss the filing in the coming weeks. The stock traded up 11% following the news. Release

Santhera’s rise continues with Fast Track in DMD

BIOCENTURY

Santhera’s rise continues with Fast Track in DMD

Thursday, April 9, 2015

http://www.biocentury.com/dailynews/company/2015-04-09/santheras-rise-continues-with-fast-track-in-dmd

best pharmacy viagra A radio set helps you to stay connected in situations where the spine has become unstable because of the tumor. One of the recent famous spams is on buy cialis online Google Plus. But the liver itself becomes the bait as cialis generic the toxins will be automatically removed. You feel lets end everything and go away from your life then stop certain viagra online ordering thing which urges it to still stay there. Santhera Pharmaceuticals Holding AG (SIX:SANN) gained CHF16.95 (18%) to CHF110.50 on Thursday after FDA granted Fast Track designation to Catena idebenone to treat Duchenne muscular dystrophy (DMD). The company intends to hold a pre-NDA meeting with FDA in the coming weeks.

Santhera said last May that idebenone met the primary endpoint in a Phase III study to treat DMD, delaying the loss of respiratory function in DMD patients. Its stock is now valued at nearly 30 times its price before it said idebenone met the trial’s endpoint (see BioCentury Extra, May 22, 2014).

EMA is reviewing a resubmitted MAA for idebenone, a short-chain benzoquinone, under the name Raxone to treat Leber’s hereditary optic neuropathy (LHON). Santhera expects a decision in 1H15