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HOME » EXPERIMENTAL TREATMENTS FOR MYASTHENIA Myasthenia
GRAVIS Gravis in
Animal Study
SEPTEMBER 12,
Myasthenia gravis (MG) is an 2019

autoimmune disease that causes muscle

Recording Eye
weakness. The disease is incurable, but Movements
several available treatments can cause May Help
MG to go into remission, sparing patients Diagnose MG
Earlier, Study
its symptoms for awhile. However, these Suggests
treatments do not work for everyone, and SEPTEMBER 10,
no approved treatments exist for rarer 2019

types of MG such as MuSK-MG . FDA Grants

Orphan Drug
Researchers continue to look for Status to Ra
Pharma’s
treatments that are
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updates effective at
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Zilucoplan as
managing the disease or ultimately
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Treatment
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curing it, with several clinical trials in the SEPTEMBER 5,
2019
pipeline.
MG Metabolic
Profile May
Investigational be Used as
Diagnostic,
Prognostic
drugs Biomarker,
Study Says
SEPTEMBER 3,
Firdapse (amifampridine phosphate) is
2019
being developed by Catalyst
Pharmaceuticals in collaboration with
BioMarin Pharmaceuticals, to treat
multiple autoimmune conditions
You May Also
including MuSK-MG. Catalyst recently
announced positive results from a proof-
Like
Chewing My
of-concept trial, and a Phase 3 trial Way
(NCT02562066) is now recruiting U.S. Through the
participants to further test Firdapse. Tough Times
⁄ MARCH 20, 2019

Prograf (tacrolimus, FK506) suppresses

the immune system. Developed by
Astrella Pharmaceuticals to prevent y Suppo t
organ transplant rejection, it’s also being System
⁄ MARCH 13, 2019
investigated in autoimmune diseases like
MG. Multiple clinical trials have assessed
its potential benefit to MG patients, and
results suggest it could be a promising G
Diagnosis
therapy suitable for long-term use.
⁄ MARCH 8, 2019

Other examples include tirasemtiv (CK-

2017357) being developed by
Cytokinetics to potentially treat
amyotrophic lateral sclerosis (ALS)  and
other diseases linked to muscle
Get the Tirasemtiv
weakness. latest updates with
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(NCT01268280). The results, published
in the scientific
journal Neurotherapeutics, showed it
produced dose-related improvements in
disease severity, was well-tolerated and
did not cause any serious adverse
events. Researchers concluded that
tirasemtiv may improve muscle function
in MG.

Biological drugs
Soliris (eculizumab) is being investigated
by Alexion Pharmaceuticals as a therapy
for drug-resistant generalized MG (gMG).
On August 21, 2017, the European
Commission (EC) approved Soliris as the
first treatment for gMG. The approval
was based on the results of a Phase 3
clinical trial called REGAIN
(NCT01997229) which suggested a Create your own user

reduction in disease severity in patients feedback survey

treated with Soliris. In January 2017, the

company also filed a supplementary
Biologics License Application (sBLA) to
the U.S. Food and Drug Administration
(FDA) to extend the Soliris indication to
include treatment for gMG.

Rozanolixizumab (UCB7665) is under

development by UCB for the treatment of
immunological disorders, including MG.
UCB is currently recruiting participants
for aGet the latest
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2 clinical trial with the Myasthenia Gravis News newsletter!
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efficacy of rozanolixizumab compared to
a placebo in patients with MG.

CFZ533 is being developed by Novartis

as an add-on therapy to standard of care
in patients with MG and is currently in
Phase 2 clinical trial (NCT02565576) to
evaluate its safety, tolerability, and
efficacy in comparison to a placebo.

Rituxan (rituximab), developed by

Genentech, is being investigated as a
therapy for MG. Multiple small pilot
studies have reported Rituxan to be a
well-tolerated and potentially effective
treatment for MG, for example in the
scientific journal Annals of Clinical and
Translational Neurology. A Phase 2 trial
(NCT02110706) called BeatMG is
expected to be completed in December
2017, and a Phase 3 trial
(NCT02950155) is currently recruiting
participants. Rituxan is approved for the
treatment of several other conditions,
including other immune diseases such
as rheumatoid arthritis and types of
ANCA vasculitis.

Other examples of biological drugs

include Orencia (abatacept) developed by
Bristol-Meyers Squibb (NCT03059888),
Hizentra (subcutaneous
immunoglobulin) developed by CSL
Behring (NCT02774239) and ARGX-113
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developed by Argenx (NCT02965573).
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Antisense drugs
Antisense drugs aim to stop production
of a protein that causes a certain
condition. Monarsen (EN101) is an
antisense drug that could potentially
treat MG. It works by binding to the
messenger RNA, the molecule used by
the protein synthesis machinery of a cell,
and stops it from being read. The aim is
to prevent the production of an enzyme
known as acetylcholinesterase (AChE),
which breaks down a chemical
messenger called acetylcholine that
carries signals between nerves
and muscles. This should increase the
amount of acetylcholine available to help
muscle activation and contraction.

Amarin bought the rights to Monarsen,

but that company has no plans to further
develop the product. Other research
groups are interested in Monarsen, but
the drug’s future is currently unknown.

Vaccines
Myasterix (CV-MG01) is a vaccine for MG
being developed by CuraVac in
collaboration with several European
companies, with funding from the
European Commission. Myasterix aims
to improve or cure
Get the latest symptoms
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is currently being tested
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 efficacy in people with MG. That trial is
now recruiting participants.

Note: Myasthenia Gravis News is strictly

a news and information website about
the disease. It does not provide medical
advice, diagnosis, or treatment. This
content is not intended to be a substitute
for professional medical advice,
diagnosis or treatment. Always seek the
advice of your physician or other qualified
health provider with any questions you
may have regarding a medical condition.
Never disregard professional medical
advice or delay in seeking it because of
something you have read on this
website. 

Laura
Flavell,
PhD

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Myasthenia latest updates
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