TREAT-NMD sent a set of questions to GSK, and we have posted the answers we received in this section. We hope these will help clarify some of the questions patients and families have about the DMD trials that GSK is running.
TREAT-NMD’s aim is to make it easier for all companies interested in neuromuscular diseases to run their trials – we do not have exclusive relationships with any one company, do not endorse any trials ourselves, and our services are available to all companies and researchers interested in neuromuscular treatments. We also believe it is very important that patients and families have access to as much information as possible about the trials that are going on, so that they can make informed decisions.
There is a lot to understand before deciding whether or not to take part in a clinical trial. You can read about some of the general principles behind clinical research here.
If you would like to comment on the GSK responses or ask them a follow-up question, please write to TREAT-NMD at email@example.com/site.
Below you will find the responses received from GSK. These responses were last updated by GSK on 10 February 2011.
1. Which trials is GSK currently running for DMD?
GSK is currently conducting three clinical studies to investigate its compound known as GSK2402968 (GSK’968) in Duchenne muscular dystrophy.
[TREAT-NMD additional note: these studies are all of a treatment approach known as ‘exon skipping’. You can read more about exon skipping in our DMD research overview section, in the part called ‘mutation-specific approaches’.]
The three studies currently being conducted are as follows:
|Study DMD114044 – A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy (click here to see this study on clinicaltrials.gov)|
The study is designed to assess the efficacy and safety of GSK’968 6mg/kg, once weekly, compared to placebo, for 48 weeks, in ambulant boys 5 years of age and over with DMD who have a dystrophin gene mutation amenable to an exon 51 skip
|Study DMD114117 – Phase II, double blind, exploratory study of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy (click here to see this study on clinicaltrials.gov)|
The study will assess the efficacy and safety of two different dosing regimens of GSK’968, administered over 48 weeks, in ambulant boys 5 years of age and over with DMD who have a dystrophin gene mutation amenable to an exon 51 skip
|Study DMD114118 – Phase I, double-blind, escalating dose, randomized, placebo-controlled study assessing PK, safety, tolerability in non-ambulant DMD subjects (click here to see this study on clinicaltrials.gov)|
This study will assess the pharmacokinetics, safety and tolerability of GSK’968 after a single administration, at escalating dose levels, in non-ambulatory boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip
The inclusion/exclusion criteria for these studies, as well as the endpoints and investigator locations for each study, are available on www.clinicaltrials.gov.
2. Does GSK have any additional DMD trials in the pipeline?
As mentioned above, GSK is currently conducting three studies to investigate GSK2402968 (GSK’968) in boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip. In the event that further studies with GSK’968 are to be undertaken, details will be made available as protocols are finalised.
GSK in-licensed GSK’968 (which was formerly known as PRO-051) from Prosensa, a Dutch biopharmaceutical company focused on developing treatments for DMD. Prosensa is continuing to develop early stage compounds targeting other exons, as part of its DMD exon skipping programme. GSK has exclusive options to in-license three more RNA-based compounds targeting additional DMD exons from Prosensa, upon successful completion of ‘proof of concept’ studies.
3. What is the precise nature of the relationship between GSK and Prosensa?
In October 2009, GSK & Prosensa announced that they entered into an exclusive worldwide collaboration for the development and commercialization of RNA based therapeutics for Duchenne Muscular Dystrophy (DMD). The scope of the alliance includes four RNA-based programmes, each with the goal of developing products intended to treat specific, but different, subpopulations of patients with DMD.
Under the terms of the agreement, GSK obtained an exclusive worldwide license to develop and commercialize Prosensa’s lead compound, PRO051 (now called GSK’968), intended to target DMD mutations treatable by skipping exon 51 of the dystrophin gene. GSK is progressing the clinical development of this investigational medicine, in collaboration with Prosensa. In addition, GSK has exclusive options to in-license three more RNA-based compounds targeting additional DMD exons, upon successful completion ‘proof of concept’ studies.
These questions have been translated into Japanese and are available from the Japan’s Registries website – Remudy.