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Pivotal clinical trial

DUVYZAT was studied in EPIDYS, one of the most inclusive phase 3 DMD studies to date1,*

EPIDYS was a global, 18-month, double-blind, randomized, placebo-controlled trial that included patients with a broad range of genetic mutations.1,2

Taking DUVYZAT

Ethan, an actual DUVYZAT patient for 4+ years.

Patients were:

  • Ambulant boys aged ≥6 years with genetically confirmed DMD (median age of 9.8 years)1,2
  • No restrictions on genetic mutations, but these were generally balanced across both groups1
  • All patients completed two 4SC assessments with a mean of 8 seconds or less, were ambulatory, and had a time-to-rise of at least 3 seconds but less than 10 seconds1
  • Patients were on a stable dose and regimen of corticosteroids, which was continued throughout the study1

Efficacy was assessed in 120 patients using multiple measures, including 4SC,† NSAA (including time-to-rise from the floor), 6-minute walking test, change in VLFF, knee extension and elbow flexion, and a muscle strength assessment.1

Safety was assessed in 179 patients (Group A + Group B).1

Recruitment was prespecified in 2 groups1:

Efficacy and safety analysis group, n=120

Baseline VLFF >5% but ≤30%


Composed of patients unlikely to lose mobility suddenly but expected to show measurable decline in function, strength, and fat fraction when on placebo + corticosteroids.

Safety analysis only, n=59

Baseline VLFF ≤5% or >30%


Additional subjects recruited to assess the safety of DUVYZAT + corticosteroids in a broader population of patients with DMD.

4SC, 4-stair climb; DMD, Duchenne muscular dystrophy; NSAA, North Star Ambulatory Assessment; VLFF, vastus lateralis fat fraction.

* EPIDYS is an acronym for Epigenetic Rescue of Dystrophin Dysfunction trial.1

†The 4SC is a measure of muscle function that tests the time it takes to climb 4 stairs.

Taking DUVYZAT

Ethan, an actual DUVYZAT patient for 4+ years.

4-STAIR CLIMB
NSAA
VLFF

PROTECTION against DMD progression with DUVYZAT2

Patients treated with DUVYZAT were able to complete 4SC faster than patients taking placebo2

PRIMARY ENDPOINT: Change in time to perform 4SC vs placebo

Estimates compared across multiple real-world and clinical trial data sources indicate that change ≥1.3 seconds to complete the 4SC is clinically meaningful.3

DUVYZAT demonstrated greater preservation of motor function vs placebo as shown by NSAA1,§

SECONDARY ENDPOINT: Change from baseline in total NSAA item score over 18 months1,||

§ Nominally significant but not statistically significant based on the prespecified multiplicity adjustment.1

II The NSAA is a DMD-specific assessment scale measuring lower limb function in ambulant children with DMD, comprising 17 items scored on a scale of 0 to 2. A score of 2 indicates the activity is performed without difficulty; 1 indicates the activity is performed with some compensation; 0 indicates the activity cannot be performed independently.1,2,5

Post hoc analysis: fewer failed NSAA test items for patients on DUVYZAT vs placebo1,2

Loss of NSAA items at 18 months—target population1,2

% of patients with failure in NSAA items

DUVYZAT + corticosteroids
Placebo + corticosteroids
% of patients with failure in NSAA items

Compared with placebo at 18 months,

DUVYZAT reduced new fat infiltration in key muscle groups required for ambulation1,2,7,§

SECONDARY ENDPOINT: Changes in mean VLFF over 18 months from baseline1,7

At 18 months, for the patients with VLFF baseline in the range of >5% to ≤30%, the mean increase (absolute difference from baseline levels) of VLFF was 7.48% in the DUVYZAT-treated patients compared to a 10.89% increase in patients who received placebo.2

§ Nominally significant but not statistically significant based on the prespecified multiplicity adjustment.1

Exploratory analysis: DUVYZAT reduced new fat infiltration in the thigh muscle8

Baseline
18 months
Patient taking placebo + corticosteroids
Patient taking placebo baseline
Patient taking placebo 18 months
Patient taking DUVYZAT + corticosteroids
Patient taking Duvyzat baseline
Patient taking Duvyzat 18 months
Baseline
18 months

MRS imaging showing increase in fat fraction from baseline after 18 months.7

Images are from single individuals and may not be representative of all patients' results.

MRS, magnetic resonance spectroscopy.

DUVYZAT safety

DUVYZAT safety is based on the pivotal EPIDYS 18-month study in a total of 179 ambulant DMD patients aged 6 years and older on concomitant corticosteroid treatment.2 95% of patients completed the EPIDYS clinical trial.1

Adverse reactions reported in >5% of DUVYZAT-treated patients

DUVYZAT + corticosteroids (n=118)

%

 Placebo + corticosteroids (n=61)

%
Diarrhea 37 20
Abdominal pain 34 25
Thrombocytopenia‡ 33 0
Nausea/vomiting 32 18
Hypertriglyceridemia 23 7
Pyrexia 13 8
Myalgia 9 3
Rash 9 2
Arthralgia 8 2
Fatigue 8 0
Constipation 7 2
Decreased appetite 7 0

‡ Thrombocytopenia includes platelet count decreased and thrombocytopenia.

Diarrhea, the most common adverse reaction, usually occurred within the first few weeks of treatment and resolved with continued dosing.2,4

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References: 1. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al; EPIDYS Study Group. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23(4):393-403. 2. DUVYZAT. Prescribing information. ITF Therapeutics; 2024. 3. Muntoni F, Signorovitch J, Sajeev G, et al. Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): a multi-center study. PLoS One. 2024;19(7):e0304984. 4. Data on file, ITF Therapeutics. 5. Ayyar Gupta V, Pitchforth JM, Domingos J, et al. Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy. PLoS One. 2023;18(4):e0283669. 6. Mercuri E, Brogna C, Mah JK, et al. Givinostat in DMD: results of the Epidys study with particular attention to NSAA. Poster presented at: 2023 World Muscle Society Conference; October 2023; Charleston, SC. 7. Vandenborne K. Givinostat in DMD: results of the Epidys study with particular attention to MR measures of muscle fat fraction. Oral presentation at Muscular Dystrophy Association Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

  • Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen.
  • Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment.
  • Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist.
  • QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation.

Adverse Reactions

The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%).

Drug Interactions

Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities.

Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms.

INDICATION

DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.

To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for additional safety information.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

  • Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen.
  • Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment.
  • Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist.
  • QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation.

Adverse Reactions

The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%).

Drug Interactions

Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities.

Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms.

INDICATION

DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.

To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for additional safety information.