Capricor Therapeutics has announced positive three-year data from its Phase II open-label expansion study. Image credit: Shutterstock / Hailshadow.

Capricor Therapeutics has announced its deramiocel cell therapy continues to be safe and effective after three years for Duchenne muscular dystrophy (DMD).

The Phase II (NCT03406780) three-year open-label extension (OLE) study showed that deramiocel led to a 47% slowing of disease in skeletal muscle in patients with DMD.

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The trial, involving 12 patients on deramiocel, also showed improved measures of cardiac function, especially in patients that had higher ejection fractions (>45%) at the beginning of the randomised portion of the trial.

All patients encountered adverse events (AEs), with the majority being low grade, with 38.5% of events being Grade 3. There were no Grade 4 or Grade 5 events. This remains consistent with previous studies of deramiocel.

Capricor says that the data supports the need for early intervention to maintain function and potentially slow the progression of cardiomyopathy, which is one of the leading causes of death in patients with DMD.

Capricor CEO Dr Linda Marbán said: “The results of the open-label study are tremendously important for DMD patients, as they showed sustained skeletal and cardiac benefits after three years of continuous treatment with deramiocel, which underscores the potential long-term benefits this therapy can offer patients with DMD.

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Based on this data and prior results, Capricor adds that it plans to speak with the US Food and Drug Administration (FDA) to progress the biologics licence application (BLA) filing, with a meeting scheduled for Q3 2024.

The company is investigating deramiocel in a Phase III, pivotal study (NCT05126758), with topline data expected in Q4 2024.

Deramiocel is a cell therapy which uses endogenous stromal cells obtained from donated healthy human hearts. The cells act by decreasing infarct size in myocardial infarction and ischemic left ventricular dysfunction.

DMD landscape

On June 12, Pfizer announced its DMD therapy fordadistrogene movaparvovec failed to meet the primary endpoint of a Phase III study (NCT04281485). This comes as the Phase III CIFFREO trial is subject to a dosing pause due to a patient death reported in the Phase II DAYLIGHT trial.

Meanwhile, Sarepta has secured a label expansion from the FDA for its DMD therapy Elevidys to be used in males with DMD aged four years and above.

There are currently no approved treatments for DMD cardiomyopathy, highlighting the critical need for therapies in this space.

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