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Long-term treatment with Duvyzat (givinostat) delays the loss of key motor skills in people with Duchenne muscular dystrophy (DMD), published results from an open-label extension study show.

“The sustained benefit observed across functional outcomes reinforces the potential of Duvyzat to meaningfully alter the course of the disease,” Scott Baver, PhD, head of U.S. medical affairs at ITF Therapeutics, said in an interview with Muscular Dystrophy News Today.

The results were published in the Annals of Clinical and Translational Neurology, in the study, “Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.”

“These new findings show that treatment with [Duvyzat] continues to have a positive impact by addressing symptoms that patients are most concerned about, and that these long-term benefits are achieved over several years while treatment remains safe and tolerable,” Craig M. McDonald, MD, a study investigator, and chair and professor at the University of California Davis Health, said in a company press release.

What is Duvyzat?

Duvyzat is approved for DMD in people, ages 6 and older. It blocks the activity of histone deacetylases, enzymes that regulate gene activity. Available data indicate it helps reduce inflammation, scarring, and fat infiltration into muscles. The drug’s approval was mainly based on data from the Phase 3 EPIDYS clinical trial (NCT02851797), which tested it against a placebo in 179 boys with DMD who could walk and were on corticosteroids. They were later able to enroll in a long-term extension study (NCT03373968) where all the participants are taking Duvyzat.

The extension study also includes those given Duvyzat in a Phase 1/2 trial (NCT03238235) and some who were screened, but weren’t assigned to treatment or the placebo in EPIDYS. The published analysis includes 194 patients treated for a mean of 559.6 days, or about 1.5 years. The maximum treatment duration was more than eight years, including Duvyzat’s use in prior studies.

Comparing the findings from the long-term study against natural history studies that assessed the disease trajectory without treatment (except for corticosteroids), Duvyzat delayed the loss of patients’ ability to walk, climb a short set of stairs, and rise from the floor.

“These data suggest that treatment with Duvyzat may delay the loss of motor function, further supporting the potential of the drug to provide clinical benefit to patients with Duchenne,” Baver said.

The motor function gap that favors Duvyzat over the natural history group started around age 10. According to Baver, “maintaining muscle function can mean patients can live with higher levels of independence and spend more time doing everyday activities.”

Measuring respiratory function

The extension study also evaluated changes in respiratory function, using forced vital capacity (FVC) and peak expiratory flow (PEF) as measures. FVC tracks how much air can be exhaled in a forceful breath, while PEF measures how quickly air is exhaled.

Over the follow-up, FVC percent declined, but PEF percent remained largely unchanged. “This suggests that while lung capacity declined over time, patients’ ability to exhale forcefully did not show a significant decrease during the study,” said Baver, who noted that, compared with “matched historical controls, the decline in FVC was less pronounced in participants treated with Duvyzat over the same time period.” The existing data are still too limited to know Duvyzat’s effect on later-stage disease milestones, such as the need for a tracheostomy, wherein a small opening is made in the neck so a tube can be inserted directly into the windpipe, he said.

As shown at the Muscular Dystrophy Association Clinical & Scientific conference, two years after patients lost their ability to walk, the average FVC score was higher for those given Duvyzat than with the natural history study.

Studying Duvyzat

Most participants reported at least one adverse event, mostly mild to moderate in severity, without unexpected safety issues.

The extension study also included nonambulatory patients who completed the EPIDYS trial, but the results here varied. To better understand Duvyzat’s effects in this patient population, Italfarmaco (which includes ITF Therapeutics) is running the Phase 3 study ULYSSES trial (NCT05933057) in boys with DMD, ages 9-17. Its primary goal is to assess the efficacy of Duvyzat at reducing muscle decline, as measured by Performance of the Upper Limb 2.0. The therapy’s safety and tolerability are also being evaluated, along with its efficacy, including with respiratory function.

“We hope that the ULYSSES trial will provide more insights regarding how Duvyzat can help slow disease progression, specifically in the nonambulant population, which is a patient group that has historically had fewer research-driven treatment options,” Baver said.

Also, the observational PROVIDUS study (NCT07127978) will evaluate safety and efficacy in DMD patients who are starting Duvyzat or have initiated it recently. The study will also include participants in gene therapy and exon-skipping therapies.

“The goal of this real-world evidence study is to complement findings from our clinical trials and provide a more comprehensive understanding of Duvyzat’s real-world performance and the overall patient experience,” said Baver, who noted that although the data so far reinforce the drug’s safety and efficacy, ITF will continue working with “members of the patient, caregiver, and advocacy communities to advance research that can provide more insights on the impact of treatment with Duvyzat.”

Sept. 11, 2025 — Noise is annoying, whether you’re trying to sleep or exploit the laws of quantum physics. Although noise from environmental disturbances will always be with us, a team including scientists at the National Institute of Standards and Technology (NIST) may have found a new way of dealing with it at the microscopic scales where quantum physics reigns. Addressing this noise could make possible the best sensors ever made, with applications ranging from health care to mineral exploration.

By taking advantage of quantum phenomena known as superposition and entanglement, researchers can measure subtle changes in the environment useful for everything from geology to GPS. But to do this, they must be able to see through the noise caused by environmental sources such as stray magnetic fields to detect, for example, an important signal from the brain.

New findings, detailed today in Physical Review Letters, would enable interlinked groups of quantum objects such as atoms to better sense the environment in the presence of noise. A horde of unlinked quantum objects can already outperform a conventional sensor. Linking them through the process of quantum entanglement can make them perform better still. However, entangling the group can make it vulnerable to environmental noise that causes errors, making the group lose its additional sensing advantage.

The team’s theoretical solution specifies how to design in advance a group of quantum objects called qubits — also known as the units of data in quantum computers — before they are used for the task of sensing. Rather than preparing the group in a way that will correct all the errors the interlinked qubits experience, the team has found that if it corrects only some of the errors, the sensor becomes more robust in the face of noise. The group of entangled qubits loses a bit of sensitivity in the process, but the trade is worthwhile, because the sensor still outperforms unentangled qubits.

“Usually in quantum error correction, you want to correct the error perfectly,” said Cheng-Ju (Jacob) Lin, one of the paper’s authors and a former Institute for Robust Quantum Simulation (RQS) postdoctoral fellow at the Joint Center for Quantum Information and Computer Science (QuICS), a joint institute of NIST and the University of Maryland. “But because we are using it for sensing, we only need to correct it approximately rather than exactly. As long as you prepare your entangled sensor the way we discovered, it will protect your sensor.”

Qubits can exist in multiple energy states, such as high and low, but they also can exist in a “superposition” of those states, acting as if they are simultaneously in all these states. This superposition is not only the property a quantum computer would harness to solve problems conventional computers find intractable, but it also makes the qubit highly sensitive to minute changes in its environment, such as the presence of a faint magnetic field, which would measurably affect the qubit’s energy state. Quantum-enhanced measurement techniques like this allow for higher precision than conventional sensing methods, making them useful in applications such as navigation.

But qubits can get even more sensitive with the use of an additional quantum property called entanglement, a phenomenon where multiple objects have interlinked quantum states. When a group of qubits is entangled, each qubit senses the signal not only directly but also through its links with other qubits, thereby amplifying the signal — making the group even better than unentangled qubits at sensing slight changes.

Increasing the number of entangled qubits dramatically improves the group’s capabilities as well. For example, compared to a single qubit in superposition, 100 unentangled qubits would be 10 times more sensitive, but 100 entangled qubits would be 100 times as sensitive.

The trouble is that entangling qubits generally requires complete isolation from environmental changes such as mechanical vibrations or temperature shifts. Such disturbances create what quantum technology designers call noise — a perennial problem for quantum computers and quantum sensing alike.

The team’s approach designs the group of entangled qubits so they can tolerate some noise-related errors. Building on observations seen in others’ experiments, the team applies techniques for correcting corrupted data in quantum computers — known as quantum error correction codes — to make the group robust in the face of noise.

“In analyzing these error correction codes, we found that there is a family of codes that protects entangled sensors,” Lin said. “One type of error correction code enables entangled qubits to detect magnetic fields with higher precision than unentangled qubits, even if some of the entangled qubits become corrupted with errors.”

While other teams’ experiments suggested the result, Lin said that his group’s paper places the findings on a more mathematically rigorous footing.

“It may take time for technologists to create sensors that take advantage of the findings,” he said. “However, the community’s understanding of quantum mechanics is good enough that we think the results will hold up under experiment, which we invite others to test in the lab.”

 

Paper: C.-J. Lin, Z.-W. Liu, V.V. Albert and A.V. Gorshkov. Covariant Quantum Error-Correcting Codes with Metrological Entanglement Advantage. Physical Review Letters. Published online Sept. 10, 2025. DOI: 10.1103/dttc-ksdn

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Source: NIST