SwitzerlandSwitzerland

Novartis researchers find biomarker for therapy response in Fragile X syndrome

06.01.2011

Basel – Epigenetic modifications of the FMR1 gene in Fragile X Syndrome are associated with the response to Novartis’ Phase II metabotropic glutamate receptor (mGluR5) antagonist AFQ056A, according to a study in 30 patients (Science Translat. Medicine, 5-1-2011). Fragile X is the most common known single gene cause of autism and the most common inherited cause of intellectual disability, affecting 1 in every 5,000 children worldwide. Without a cure, the syndrome is associated with the expansion of a single trinucleotide gene sequence (CGG) on the X chromosome, and results in a failure to express the protein coded by the FMR1 (Fragile X Mental Retardation-1) gene, which is required for normal neural development. According to Sébastien Jacquemont and colleagues, not all of the 30 patients treated with the symptomatic therapy showed improvement, but an analysis of those that did revealed that the promoter of the FMR1 gene in those patients was fully tagged with methyl groups. The methyl group tags signalled that the FMR1 gene was completely shut off in these patients, providing a possible signature of people with Fragile X who could benefit from mGluR5-blocking drugs. The team also ran a large set of tests designed to detect changes in behaviours, such as hyperactivity and inappropriate speech in the patients. While the new drug had no effect on behaviour as measured by the first set of tests, it did show an effect on a secondary group of tests, when compared to a group of patients given a placebo treatment. Taking a closer look, the authors found that each member of the group of patients with fully methylated FMR1 promoters showed improvement in behaviour. The group with partially methylated promoters showed no such changes. This correlation between response to treatment and methylation status of the FMR1 promoter sets the stage for future studies designed to test whether methylation can serve as a predictor of positive drug response in patients with Fragile X syndrome. Novartis runs also clinical programmes with AFQ056A in Parkinson’s disease and GERD.

SwitzerlandSwitzerland

30.03.2012

Basel/Hinxton (UK) – Two European teams have created huge repositories of cancer genomics data that help predict drug response. Swiss Novartis and the Boston-based Broad Institute have combined data obtained from large-scale...

SwitzerlandSwitzerland

27.03.2012

London/Lausanne – The global private equity firm Celtic Therapeutic L.L.L.P. believes in the power of antibody-drug conjugates (ADCs). It is investing $50m in a new Switzerland-based company called ADC Therapeutics Sarl which...

SwitzerlandSwitzerland

26.03.2012

Basel/Vienna - Following promising results from a Phase III study, Roche has submitted a regulatory application to EMA for subcutaneous Herceptin (trastuzumab) to treat HER2-positive breast cancer. Roche said subcutaneous...

SwitzerlandSwitzerland

09.03.2012

After the US Food & Drug Administration forced Swiss generics giant Sandoz AG to update its production facilities in Quebec, there are now fears that supplies of some medicines could grow tight in Canada. The regulatory authority...

SwitzerlandSwitzerland

08.03.2012

The hepatitis C space has seen a string of large deals in the last weeks that include a US$11bn buyout of Pharmasset by Gilead. Now Novartis is also trying to get in the game with the purchase of a global exclusive licence from...

SwitzerlandSwitzerland

06.03.2012

Allschwil – Swiss Actelion Ltd. has strengthened its commercial offerings by securing rights in certain countries to Auxilium’s biotech drug Xiaflex for treating tissue buildup under the skin. The US pharma is to receive US$10m...

SwitzerlandSwitzerland

06.03.2012

Zug – Life Sciences specialist HBM partners has raised €90m in the first close of its HBM BioCapital II fund, which will invest in revenue-generating companies in the pharmaceuticals, medical device and diagnostics sectors....

SwitzerlandSwitzerland

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Stans – Mondobiotech, a drug discovery company in troubled waters, could soon be under Italian ownership: The Swiss biotech announced it has entered into exclusive negotiations with Milan-based provider of pharmaceutical and...

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