MDF Magazine Newsletter Issue 56 August 2018
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Research<br />
The following five articles are from the website of Muscular Dystrophy UK.<br />
Update on Myonexus’ LGMD gene therapies<br />
By Jenny Sharpe<br />
Published 25/06/<strong>2018</strong><br />
Myonexus Therapeutics gave an<br />
update on its LGMD gene therapy<br />
programmes at Sarepta’s recent<br />
Research and Development (R&D)<br />
day. The two companies formed a<br />
partnership in May.<br />
Myonexus are currently developing<br />
gene therapies for five types of LGMD<br />
(see below). All of these share a similar<br />
design, where the desired gene is<br />
packaged into a type of adeno-associated<br />
virus (AAV). This shared design<br />
means that learnings from one study<br />
will help to inform another.<br />
LGMD 2E (MYO-101)<br />
Myonexus plan to initiate a phase<br />
1/2a trial at Nationwide Children’s<br />
Hospital, USA, testing MYO-101 in<br />
Q3 <strong>2018</strong>. This will be a randomised,<br />
placebo controlled, double blind<br />
trial recruiting nine participants.<br />
The trial will also have a ‘crossover’<br />
design, where all participants<br />
assigned to the placebo will get to<br />
have the gene therapy at some point.<br />
LGMD 2D (MYO-102)<br />
Following positive results from a<br />
small safety trial completed in 2017,<br />
Myonexus are planning to test MYO-<br />
102 in a phase 1/2a trial at Nationwide<br />
Children’s Hospital, USA. This will<br />
be a randomised, placebo-controlled,<br />
double blind trial recruiting nine participants.<br />
The trial will also have a<br />
‘crossover’ design, where all participants<br />
assigned to the placebo will get<br />
to have the gene therapy at some point.<br />
LGMD 2C (MYO-103)<br />
Myonexus are currently testing<br />
MYO-103 in preclinical safety studies<br />
using mice. The company is expecting<br />
to meet with the US Food and Drug<br />
Administration (FDA) very soon to<br />
discuss its plans for MYO-103.<br />
LGMD 2B (MYO-201)<br />
MYO-201 is currently being<br />
evaluated in a phase 1 trial at<br />
Nationwide Children’s Hospital,<br />
USA, where it was injected into the<br />
feet of six participants (intramuscular<br />
delivery). Myonexus reported that,<br />
so far, MYO-201 appears to be safe<br />
and can increase dysferlin protein<br />
levels. The company is planning a phase<br />
1/2a trial testing intravenous delivery<br />
of MYO-201(an injection into the<br />
bloodstream, rather than the muscle).<br />
LGMD 2L (MYO-301)<br />
Myonexus is currently testing MYO-<br />
301 in a mouse model of LGMD 2L.<br />
This will help to determine how well<br />
it works and whether it is safe to be<br />
tested in humans in future.<br />
Article online at: https://www.musculardystrophyuk.org/news/news/<br />
update-on-myonexus-lgmd-gene-therapies/<br />
Positive interim results from Duchenne gene therapy trial<br />
By Sofia Nnorom<br />
Published 20/06/<strong>2018</strong><br />
Sarepta Therapeutics has announced<br />
positive preliminary results from its<br />
ongoing Duchenne gene therapy trial at<br />
the company’s R&D day.<br />
28<br />
The U.S. trial is assessing AAVrh74.<br />
MHCK7.microdystrophin – an adeno-associated<br />
virus carrying a shortened<br />
version of the dystrophin gene<br />
(micro-dystrophin) into the body. The<br />
first trial participant received the therapy<br />
in January <strong>2018</strong> and since then<br />
three more boys have been treated.<br />
The company released interim<br />
results based on the first three treated<br />
children. Muscle biopsies taken 90<br />
days after the treatment showed that<br />
all three boys had a significant increase<br />
in the dystrophin protein. In addition,<br />
all of them had a significant reduction<br />
in Creatine Kinase (CK) levels – a
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