About NEXVIAZYME

An enzyme replacement monotherapy* for
LOPD

The NEX chapter in POMPE treatment for LOPD patients

NEXVIAZYME is a monotherapy*. This means you only have to take a single medicine to treat your LOPD.* It’s also an M6P-enriched treatment. Understand the difference that could make, and see the pivotal study results below.

*Not including premedication or pretreatment your doctor may prescribe.

What is M6P?

M6P (Mannose 6-Phosphate) is a part of the molecule that binds to certain muscle cell
receptors and helps enzyme replacement therapy reach your muscle cells.

NEXVIAZYME has ~15x MORE M6P than the current standard of care, alglucosidase
alfa (Lumizyme), and was made to improve uptake of the enzyme into muscle cells.

What is POMPE disease?

How does NEXVIAZYME work?

Pompe is a degenerative muscle disease caused by a deficiency in the GAA enzyme.
In a normal muscle cell, GAA enzyme breaks down glycogen, a type of sugar, so the cell can it use it for energy. This happens in an area called the lysosome. To get there, GAA needs the help of M6P, which is a part of a molecule within the cell.
People with Pompe disease have very little GAA or none at all, which is why the body can’t break down glycogen in the muscle as it should. The glycogen builds up, causing ongoing muscle damage.
NEXVIAZYME is an M6P-enriched enzyme replacement therapy for late-onset Pompe disease (LOPD). It helps replace GAA enzyme for those whose bodies don’t produce enough. NEXVIAZYME is scientifically designed to be absorbed effectively by muscles.

NEX move: better breathing, greater endurance

NEXVIAZYME vs alglucosidase alfa

NEXVIAZYME showed meaningful improvement for breathing and walking when compared to alglucosidase alfa in a pivotal study.

Everyone (100 patients aged 16-78 years of age with LOPD) in the study had late-onset Pompe disease, but none of them had received any prior treatment. Some were given NEXVIAZYME, and others were given alglucosidase alfa. All 100 people stayed in the study for 49 weeks.

The 2 tests used to measure how people were doing in the study are tests you may be familiar with if you’ve been living with LOPD:

  • Forced vital capacity (FVC) measures how much air you can force out in a deep breath
  • 6-minute walk test (6MWT) measures the distance you can walk within that time frame

The tests were given at the beginning and again at the end of the study.

After 49 weeks on NEXVIAZYME, people were able to:

person breathing
Improve the breathing by averange of 2.9 percentage points in a breathing test

*Compared to when they began treatment.

Those who took alglucosidase alfa improved their breathing by an average of 0.5 percentage points.

This resulted in an average of 2.4 percentage points measurable improvement for people treated with NEXVIAZYME compared with people taking alglucosidase alfa, although NEXVIAZYME was not statistically superior.

person walking
Improve the walking distance by an average of 106 feet during a 6mwt

*Compared to when they began treatment.

Those who took alglucosidase alfa improved their walking distance by an average of 7.2 feet.

People taking NEXVIAZYME walked an average of 98 feet further than those who were taking alglucosidase alfa.
This measurement was not tested to determine statistical superiority of NEXVIAZYME to alglucosidase alfa.

What was learned about safety

In a clinical trial, NEXVIAZYME demonstrated similar safety and risks to alglucosidase alfa. Over the 49-week study, people taking NEXVIAZYME had fewer serious side effects than those taking alglucosidase alfa. No one had to stop taking NEXVIAZYME because of side effects, while 4 people had to stop taking alglucosidase alfa.

COMMON SIDE EFFECTS OF NEXVIAZYME INCLUDE:

  • headache
  • fatigue
  • dizziness
  • nausea
  • diarrhea
  • vomiting
  • joint pain
  • muscle pain
  • itching
  • shortness of breath
  • rash
  • “pins-and-needles” sensation
  • hives
  • headache
  • fatigue
  • dizziness
  • nausea
  • diarrhea
  • vomiting
  • joint pain
  • muscle pain
  • itching
  • shortness of breath
  • rash
  • “pins-and-needles” sensation
  • hives

These are the most common side effects, but there are other possible side effects. You should always tell your doctor about any changes in the way you feel, even if it’s not one of the listed side effects. You can also report side effects at
1-800-FDA-1088 or www.fda.gov/medwatch

When antibodies form, they can interfere with some medicines. There is a potential to develop antibodies, but for the patients in the trial it didn’t affect their efficacy. Patients in the clinical trial who did have high titers for antibodies had a higher risk of experiencing infusion-associated reactions.

NEXVIAZYME has a demonstrated safety profile with nearly 7 years of clinical data from 4 clinical trials, which includes people who switched from alglucosidase alfa.

The Comet trial was not designed to demonstrate a statistically significant difference in the incidence of adverse reactions between NEXVIAZYME and alglucosidase alfa. In addition, 13 out of 51 people (25%) receiving NEXVIAZYME experienced mild to moderate infusion-associated reactions. These reactions included headache, diarrhea, itching, hives, and rash. No one experienced a severe infusion-associated reaction

WHAT
MOVES
YOU?

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