Category Archives: Hunter Syndrome Trial Blog

Armagen Presents Data for Treatment of Hunter Syndrome MPS 2

In a recent press release, Armagen Inc. presented data from the first chorot of their Phase 1/2a sutyd of AGT-182, an investigational Enzyme Replacement Therapy that has received orphan drug designation from the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of Hunter Syndrome aka MPS 2.

 

Using ArmaGen’s proprietary technology, AGT-182 is designed to take advantage of the body’s natural system for transporting products across the Blood-Brain Barrier (BBB) by binding to the same receptor that delivers insulin to the brain. AGT-182 is engineered by the fusion of the replacement IDS enzyme to an antibody that is attracted to a receptor on the BBB. The IDS enzyme is designed to travel through the BBB attached to that antibody.

 

To view the full press release, please click and download it below:
Armagen Press Release July 14, 2016

 

The favorable data from Cohort 1 informed the decision to proceed to  Cohort 2, in which up to four adult (age 18 and above) patients are to receive weekly IV infusion starting  at a dose of 3.0 mg/kg. If you are interested in participating in the Breaking Barriers Hunter Syndrome Trial, please sign up here.

 

Hunter syndrome, also known as mucopolysaccharidosis type II, or MPS 2, is a lysosomal storage disorder caused by inadequate activity of the enzyme iduronate-2-sulfate (IDS), which is needed to break down complex sugars produced in the body. Currently available treatment of Hunter Syndrome do not cross the BBB in clinically relevant amounts and therefore do not address the progressive neurological complications of the disease.  Dr. James Callaway, chief executive officer of Armagen says “We are making steady progress in developing a viable therapeutic for Hunter Syndrome that crosses the BBB. Favorable data from the first cohort of patients suggests AGT-182 can safely address somatic symptoms of the disease. We are grateful for the investigators and patients who have participated in the Break Barriers trial to date, and we look forward to beginning the second cohort as recommended by the data monitoring committee in an effort to confirm these preliminary data.”

 

Inside MPS II or Hunter Syndrome – Types and Treatments

 

Mucopolysaccharidosis type II ( MPS II ) disease—more commonly known as Hunter Syndrome—is a genetically acquired disease, which is characterized by an inadequate production of an enzyme known as iduronate sulfatase (IDS). This production of IDS is essential in breaking down complex sugars (also known as GAGs), which are responsible for the repair and creation of cells, tissues, and tendons to support the normal functions of a human body. The absence of IDS destroys the process of breaking down these complex GAGs.

When GAGs are not properly broken down into simpler forms they lock themselves inside the enzyme. These stored GAGs increase and eventually block other GAGs from coming out, causing cell malfunctions inside the body. Stored GAGs ultimately damage many parts of the body, as the undigested sugars build up within the cells.

Types of MPS II

Effects of Hunter Syndrome

MPS II usually results in round-neck bone deformities. It also coarsens facial features by thickening the lips, tongue, and nostrils. Other side effects of Hunter Syndrome include dwarfism, deafness, cardiovascular disorders, and the enlargement of the liver and spleen. In the most severe of cases, Hunter Syndrome can also lead to premature death.

Treatment

MPS II Treatments

While there is currently no cure for MPS II, there are treatments that can help to slow the progression of the disease and lessen the severity of the syndrome. For the families who have a Hunter Syndrome patient at home, it is essential for you seek advice from your doctors on how to properly treat your child. There are also support groups that help spread the awareness and further knowledge of the syndrome.

It is not easy to suffer such condition, for both family and patients, but with the proper guidance and help of people around you, life will be that much easier to manage.

MPS 2 DiseaseMap

We’ve joined the MPS 2 DiseaseMap made by diseasemaps.org to help promote greater communication and awareness for Hunter Syndrome! If you haven’t yet added yourself to the map, do so now! This is a great way to stay in touch with people all over the world who are connected to MPS 2. Just click the “Join the Map” button at the bottom of the map!

Hunter Syndrome Treatments

 

Hunter Syndrome Treatments

Catching Hunter Syndrome at an early stage is crucial for treatment. A child can undergo a bone marrow transplant where bone marrow is taken from the hip of a donor and transplanted into a child. This treatment may help lessen breathing difficulties or problems with mobility, as well as improve heart, liver, and spleen functions. It can also prevent a child’s mental regression.

 

Another possible treatment option is enzyme therapy. This therapy replaces the missing or defective enzymes of a child with man-made or genetically engineered enzymes, which eases many symptoms of Hunter Syndrome. Unfortunately, this treatment comes with some risks and has side effects to the body including headache, fever, skin reactions, and high blood pressure.

 

A third option is gene therapy, which effectively replaces the chromosome for producing the missing enzyme of the body.

 

What are the complications of Hunter Syndrome treatments?

There are several things to consider when choosing the right therapy for a child as each comes with considerable complications. For example, relief for respiratory complications the tonsils and adenoids are removed, but as the disease progresses the tissues thicken and respiratory complications could return. Addressing heart complications could result in surgery to replace heart valves, which comes with its own set of dangers to be aware of.

 

Other complications arise when treating connective tissue problems, managing behavioral problems, and addressing sleep difficulties. Physical therapy will improve joint flexibility, but it will not stop the progression of joint motion. Behavioral problems or issues with sleep can be managed through medication, but these medications could in fact lead to other complications with the disease.

 

How can we manage Hunter Syndrome?

Talk to your doctor, first. There are genetic tests that are available if you believe you could be a carrier of Hunter Syndrome. Second, be positive! A child suffering from Hunter Syndrome needs encouragement and love. Be sure to keep the child active so that the joints remain flexible and mobile. And finally, educate and learn. Nothing is more satisfying to a child than learning, so teach them as much as you can while the brain is still functioning properly. Learn for yourself, as well. There are plenty of online communities available to you and your family to help educate you on Hunter Syndrome.

This is only a brief overview of currently-approved and available hunter syndrome treatments. Info from Breaking Barriers Hunter Syndrome Trial.

 

What Is Hunter Syndrome?

 

What is Hunter Syndrome? Hunter Syndrome (also known as mucopolysaccharidosis II or MPS II) is a disorder that occurs when an enzyme the body needs is either missing or it does not work properly. If the body does not have enough enzymes, it cannot break down the kind of sugar that builds bones, skin, tendon, and other tissue. Hunter syndrome eventually causes permanent, progressive damage affecting the appearance, mental development, organ function, and physical abilities of a child.

 

This rare genetic disorder affects mostly males as young as 18 months and has no known cure. The current treatment for Hunter Syndrome involves the management of symptoms, but it has many complications.

What are the symptoms of Hunter Syndrome?

The symptoms of Hunter Syndrome vary person to person. It’s not always present at birth, but indications of this syndrome are known to show up between 2 and 4 years of age. If the symptoms occur at an early age it is usually more severe.

what is Hunter Syndrome InfoGraphic

What causes MPS II?

Children who suffer from Hunter Syndrome inherit the disease from their parents. When a chromosome is missing or malfunctioning, it is usually because the mother was carrying MPS II. However, a father suffering from Hunter Syndrome can also pass it to his daughter who will eventually pass it to her son.

 

 

Hunter Syndrome Trial Update 3-18-2016

 

The Breaking Barriers Hunter Syndrome Trial has successfully completed enrollment for the first cohort/group of four patients! We are now enrolling for the second cohort. Armagen plans to enroll 12 patients that are 18 years and older into this study. If you would like to view the full study update, you can download it below:
Study Update from Armagen 3-18-16

 

Armagen is developing therapies to non-invasively deliver drugs across the blood-brain barrier, a filter that protect the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. Available medications for Hunter Syndrome/MPS II do not cross the blood-brain barrier in clinically relevant amounts and therefore do not address the progress neurological complications of the disease.

 

If you would like to enroll in the Breaking Barriers Hunter Syndrome Trial, you may sign up here.

Hunter Syndrome Clinical Trial Underway!

 

The Breaking Barriers Hunter Syndrome Trial is taking place at Emory University in Atlanta, GA and at many other locations world-wide. Take a look at the research behind this clinical study of Hunter Syndrome.

 

If you would like to sign up for the Breaking Barriers Hunter Syndrome trial, you can do so here.

Breaking Barriers Hunter Syndrome Trial

 

 

Researchers are conducting a Hunter Syndrome trial of AGT-182, an investigational treatment for people with mucopolysaccharidosis type II or MPS 2.

 

AGT-182 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of Hunter syndrome.

 

Currently approved treatments for Hunter syndrome (or MPS 2) are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies for MPS disease do not address many of the severe and progressive neurological complications of the Hunter Syndrome. AGT-182 is designed to cross the BBB in the same way insulin does.

 

To sign up for the Breaking Barriers Hunter Syndrome trial, sign up here.