5 Most Exciting Epigenetic Trials



Pharma IQ
06/07/2013

Epigenetics has been at the forefront of genetic research for over 15 years and the full potential of these druggable targets is finally being realised. Although most research has focused on cancer so far, infectious, CNS, autoimmune and inflammatory diseases also have epigenetic mechanisms, providing an a rich mine for new drugs to tap into. Dr Matt Fuchter of Imperial College London said in a recent interview with Pharma IQ “There has been a lot of interest in targeting epigenetic processes in disease, this is a growing area”. Here we have brought together a list of five exciting therapies at various stages of development which are exciting people in the field.

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Givinostat- A 2-Part phase I & II Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With Duchenne Muscular Dystrophy (DMD). DMD currently has no cure and treatments to improve quality of life are the main focus. Any developments in treatment could make a vast difference to those affected by this illness. Since it is a genetic disorder, epigenetic treatment might be an interesting route to take with treatment.

SEN0014196- An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196 (PADDINGTON) inhibits SirT1, a protein deacetylase. Biomarkers in biosamples and in neuroimaging.  Like DMD, Huntington’s Disease is an incurable genetic disease which may be amenable to epigenetic targeting.

RG2833- Repligen plans to initiate a phase I trial of its HDAC inhibitor RG 2833 for Friedreich’s Ataxia later this year. A progressive genetic neuronal disease, development of a treatment for Freidreich’s Ataxia fits in with the other diseases so far in that it can be considered an orphan disease. With companies focusing more and more on orphan diseases and epigenetics growing in popularity, now might be the time where this confluence leads to breakthroughs in rare genetic diseases. 

Vitamin B3-  As mentioned above, Friedreich’s Ataxia is a progressive genetic neuronal disease caused by a large number of repeats in the FXN gene causing a lack of expression of the frataxin protein. Dr Richard Festenstein and his team at Imperial College London along with the MRC have shown that the widely available vitamin supplement vitamin B3 could significantly raise the levels of frataxin. They are conducting an exploratory clinical trial to assess the effects of treatment with large doses of vitamin B3 and results are expected by the end of 2013.

Depression- Epigenetics have been shown to play a role in learning and memory, but an increasing focus is now being put on their role in disease. In a study which is recruiting in Taiwan, a team at the Chang Gung Memorial Hospital aims to investigate epigenetic regulation of BDNF in depression. These investigations may uncover interesting pathways to target with novel antidepressants.

What trials or drugs targeting epigenetic mechanisms do you think hold particular promise? Let us know in the comments.