ITF Therapeutics to Unveil Extensive DUVYZAT Data at MDA Conference

CONCORD, Mass. – February 23, 2026 – Nearly two years after its landmark approval, new long-term data on the Duchenne muscular dystrophy (DMD) treatment DUVYZAT (givinostat) is set to take center stage. ITF Therapeutics LLC has announced it will present a substantial body of research—encompassing ten separate abstracts—at the upcoming Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida, from March 8-11.

The presentations promise to provide the most comprehensive update yet on the drug's long-term safety and efficacy, offering critical insights for clinicians and families navigating the complexities of this progressive muscle-wasting disease. The data trove includes an oral presentation and nine posters, delving into DUVYZAT's impact on disease progression, muscle integrity, and flexible dosing strategies.

DUVYZAT received FDA approval in March 2024, celebrated by the Duchenne community as the first non-steroidal treatment approved to treat patients regardless of their specific genetic mutation. As a histone deacetylase (HDAC) inhibitor, it targets a different biological pathway than the gene-specific exon-skipping drugs or the corticosteroids that have long been the standard of care. This upcoming data release is poised to further define its role in the modern DMD treatment paradigm.

Bolstering the Clinical Case with Long-Term Evidence

A key focus of the new presentations will be the long-term performance of DUVYZAT. For a chronic condition like Duchenne, initial trial results are only the beginning of the story. The medical community has been eagerly awaiting data that demonstrates sustained safety and a lasting impact on the disease's relentless progression.

ITF Therapeutics appears ready to deliver on that front. One of the de novo posters, presented by Dr. John F. Brandsema of the Children's Hospital of Philadelphia, will share interim results from an open-label extension study on the long-term safety of givinostat. This type of long-term follow-up is crucial for building confidence among prescribers and patients who are considering a lifelong therapy.

Perhaps most compelling for the Duchenne community are the findings related to slowing the physical decline. A presentation by Italfarmaco's Sara Cazzaniga will detail how givinostat was shown to reduce the decline of muscle contractile area and decrease fat infiltration. In DMD, healthy muscle is progressively replaced by fat and fibrotic scar tissue. This analysis, which goes beyond functional tests to look at the underlying muscle quality, provides tangible evidence that givinostat may be preserving muscle structure.

Further building on this theme, a poster from Italfarmaco's Chief Medical Officer, Dr. Paolo Bettica, will present an analysis suggesting a potential delay in the age at which patients lose the ability to walk. The loss of ambulation is one of the most devastating milestones in DMD, and therapies that can postpone it are considered highly valuable. These findings, if robust, could significantly influence treatment decisions for ambulatory boys.

A Patient-Centric Approach to Dosing and Safety

Beyond demonstrating efficacy, ITF Therapeutics is also focusing on the practical, real-world aspects of using DUVYZAT. This reflects a deep understanding of the day-to-day challenges faced by families managing Duchenne.

An encore oral presentation by Dr. Aravindhan Veerapandiyan, a respected pediatric neurologist from the University of Arkansas for Medical Sciences, will address the rationale and efficacy of weight-based flexible dosing. This is a critical topic for a pediatric population, where a child's growth can impact drug exposure and side effects. The analysis, based on the pivotal Phase 3 EPIDYS trial, will examine functional outcomes like the 4-stair climb test, providing clinicians with data-driven guidance for tailoring treatment to individual patients.

"Since the launch of ITF Therapeutics just over two years ago, our progress has been shaped by the voices and experiences of people living with Duchenne and their families whom we are privileged to serve," said Matt Trudeau, President of ITF Therapeutics, in the company's press release. "At this year's meeting, we look forward to presenting new data... that reflect our ongoing dialogue with the Duchenne community."

This dialogue is evident in the slate of poster presentations dedicated to characterizing and managing the drug's known side effects. Multiple posters presented by experts like Dr. Brandsema and Dr. Bettica will offer detailed analyses of thrombocytopenia (low platelet counts) and gastrointestinal issues, which are among the most common adverse reactions. Providing this level of detail helps clinicians anticipate, monitor, and manage these side effects effectively, improving the overall treatment experience. The company will also present data on givinostat's cardiac safety profile, another critical consideration in DMD care.

To further bridge the gap between clinical data and lived experience, ITF is sponsoring an industry forum featuring a caregiver who will share their personal journey with DUVYZAT. This direct-from-the-source perspective underscores the company's strategy of engaging with and learning from the community it serves.

Defining a Role in a Crowded Treatment Landscape

The comprehensive data release at the MDA conference represents a strategic move by ITF Therapeutics to solidify DUVYZAT's position in an increasingly complex and competitive DMD treatment landscape. Unlike mutation-specific therapies such as Sarepta's exon-skippers or its gene therapy Elevidys, DUVYZAT offers a broad-spectrum approach.

As an HDAC inhibitor, it works by "reprogramming" cellular machinery to reduce the chronic inflammation and fibrosis that destroy muscle in all Duchenne patients, regardless of their specific dystrophin gene mutation. This positions it as a foundational therapy that can be used by a wide patient population (ages six and up) and potentially in combination with other treatments. Its non-steroidal nature also makes it an attractive alternative or adjunct to corticosteroids, which, while effective, carry a heavy burden of long-term side effects.

By presenting a wealth of data covering everything from molecular action to long-term ambulation and practical dosing, Italfarmaco and its U.S. affiliate are building a robust evidence package. This strategy is designed to arm clinicians with the confidence to integrate DUVYZAT into their treatment plans and to provide clear answers to the questions that matter most to patients and their families. As the neuromuscular community gathers in Orlando, all eyes will be on these presentations to see how the long-term promise of givinostat continues to unfold.