In the clinical trial measuring DUVYZAT plus steroids versus steroids alone, participants taking DUVYZAT completed a 6-stair climb 1.78 seconds faster on average at month 18. Additional signals included maintaining motor function abilities longer (based on NSAA score decline) and maintaining more ability to bend an elbow or straighten a knee (muscle strength measures). Improvements in NSAA scores and flexibility were observed, though this analysis was not able to confirm they were due to treatment. DUVYZAT also reduced how much fat was in the muscle (a measure of muscle loss), but again the analysis could not prove this was due to treatment.

What is HDAC inhibition?

DUVYZAT is a histone deacetylase (HDAC) inhibitor. The page includes a video titled “A deeper dive into HDAC inhibition” explaining how HDAC inhibition may help people living with Duchenne.

DUVYZAT protects from Duchenne progression

The clinical trial measured whether DUVYZAT + steroids more effectively preserved muscle strength and function than steroids alone (placebo). See more about the trial >

MAIN MEASURE

Participants taking DUVYZAT

Completed 4-stair climb 1.78 seconds faster

than participants taking steroids alone, demonstrating significant protection.*

*This was measured by the average time it took participants in both groups to climb 4 stairs at the end of 18 months.

Ethan, taking DUVYZAT for 4+ years. — Ethan, taking DUVYZAT for 5+ years.

Additional measurements of protection

After 18 months, participants taking DUVYZAT + steroids:

Maintained more ability to bend their elbow or straighten their knee, a measure of muscle strength
Maintained motor function abilities longer compared with those who took placebo, based on North Star Ambulatory Assessment (NSAA) scores
- The NSAA is a 17-item test that looks at everyday movements, such as ability to stand up from the floor, walk, climb stairs, balance, and jump
- Those who took DUVYZAT + steroids saw an average of 1.91 fewer points of decline on the NSAA than those who took only steroids
DUVYZAT also reduced how much new fat was in the muscle, a measurement of muscle loss
- Those who took DUVYZAT + steroids had 30% less new fat build-up in their muscles than those who were on steroids alone

Researchers use statistics to determine whether improvements are truly due to a treatment or could have happened by chance. In this case, even though the results looked positive, the analysis couldn’t confirm that the difference was due to the treatment alone.

DUVYZAT reduced how much new fat was in the muscle

In another follow-up analysis of the clinical trial (meaning it was not one of the main measures planned when the study began), researchers looked at individual people to see how much new fat was in the muscle. The images below represent results from 2 individual people from the study after 18 months. These results may not be the same for everyone.

How to read these images:

Magnetic resonance spectroscopy (MRS) images can show the difference between muscle and fat in the body
By taking images of the same patients 18 months apart, researchers can track the amount of new fat that is building up in the muscles
The darker areas of the images represent muscle tissue and the white areas represent fat

At the beginning of the clinical trial	After 18 months
		Participant taking steroids alone
		Participant taking DUVYZAT + steroids

Dive image — Ryan, taking DUVYZAT for 5+ years, and friend.

How DUVYZAT was studied

The clinical trial was 18 months long

Participants received steroids along with either DUVYZAT or placebo

During the trial, the DUVYZAT dose was based on weight and was reduced if certain side effects occurred

The clinical trial included 179 participants^† with a wide range of genetic variants of Duchenne:

The effectiveness of DUVYZAT was studied in 120 participants
The safety of DUVYZAT was studied in 179 participants

^†The median age was 9.8 years, meaning that half of the participants were younger than 9.8 years, and the other half were 9.8 years or older.

A clinical trial is a research study that tests new medicines to make sure they are safe and effective.

SEE WHAT THE RESEARCHERS LOOKED AT

4-stair climb

How fast participants could climb 4 stairs before and after 18 months of using DUVYZAT and steroids compared with steroids alone

NSAA

Participants’ mobility and muscle function across multiple muscle groups as measured by the NSAA, a test that measures how well people with Duchenne can move and do daily activities

Muscle strength

A muscle strength test that included how well participants could bend their elbow and straighten their knee measured with a small handheld device called a myometer

Fat tissue

Measurement of how much fat was in the muscle of the upper leg to understand muscle loss. This was assessed by MRS, an imaging test

Ready to talk about what these results could mean for you? Use the DUVYZAT Discussion Guide to start a conversation with your healthcare provider.

Science Behind DUVYZAT Key Safety Information

DUVYZAT protects from Duchenne progression

Completed 4-stair climb 1.78 seconds faster

Additional measurements of protection