Authored by Paul Foster
As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are leading the rare disease research effort. In the 2nd of the series we look at the US biopharmaceutical company Amicus Therapeutics. Amicus Therapeutics operates from both New Jersey and San Diego and currently has around 100 full-time employees.
The company is focussed on the discovery, development and commercialisation of orally administered drugs for the treatment of Lysosomal Storage Disorders (LSDs) and neurodegenerative diseases. It is focussing its research on small molecule drugs known as pharmacological chaperones. Pharmacological chaperones are designed to selectively bind to a mis-folded protein and thereby increase the proteins stability. It is hypothesised that by stabilizing the protein, pharmacological proteins may be able to restore the intended biological function of the protein. Cellular and animal studies demonstrated the ability for these drugs to increase the level of lysosomal enzyme and decrease the lysosomal substrate that is associated with diseases such as Fabry, Gaucher and Pompe.
In July 2012 GlaxoSmithKline increased their ownership of Amicus Therapeutics to 19.9% with a further investment of $18.6 million as part of their ongoing development of Migalastat HCl (Amigal) for the treatment of Fabry Disease. This clinical programme is focussed in 3 areas, monotherapy (Phase III), combination therapy with Enzyme Replacement Therapy (ERT) (Phase II) and a co-formulation of Amigal co-formulated with a proprietary recombinant human alpha-Gal A enzyme (JR-051) currently in pre-clinical development.
In December 2012, Amicus Therapeutics announced that their Stage 1, Phase III monotherapy trial failed to meet statistical significance. The stage 2 results for Amigal are expected to be published in Q2 2013. Amicus have also announced encouraging Phase II data for their combination trials. Then, in February 2013, Amicus announced the results of Study 011 which showed more encouraging data for Amigal monotherapy.
In addition, Amicus Therapeutics are developing Duvoglustat HCl (AT2220) for Pompe Disease which is recently reported positive results which established human proof-of-concept that co-administration of AT2220 just prior to infusing ERT (Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle tissue compared to ERT alone.
Amicus are also investigating the use of next generation pharmacological chaperones in neurodegenerative diseases. This programme has attracted significant attention from patient advocacy groups. Amicus have received significant grants from both the Alzheimer’s Drug Discovery Foundation and the Michael J Fox Foundation for Parkinson’s Research.
Whilst the recent Amigal results have inevitably had a significant impact on the share price it is hoped that the combination therapies and the neurodegenerative platforms Amicus Therapeutics are pursuing will have a significant impact upon the patient’s currently living with these rare diseases.
Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter