Canada-based Satellos Bioscience will be dosing the first patient in a Phase I trial of its Duchenne muscular dystrophy (DMD) drug after receiving a rare pediatric disease designation (RPDD) from the US Food and Drug Administration (FDA).
CEO Frank Gleeson told the Clinical Trials Arena that he hopes the first patient will be dosed in the trial next month. The first part of the trial will be on 72 healthy volunteers to evaluate the safety and pharmacokinetics (PK) of the drug.
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The second part of the Phase I trial will evaluate safety and PK in adult patients with DMD to ensure the same profiles are replicated in the patient population.
After the Phase I study, the company aims to initiate a Phase II trial in the target patient population of children aged between seven and 12 years with DMD. The trial will enrol around 50 patients. The trial endpoints will evaluate efficacy signals through serum markers, functional markers, and MRI scans.
The company also received orphan drug designation from the FDA earlier this year.
“Obtaining the RPDD and orphan drug designations for SAT-3247 from the FDA are important milestones for Satellos as we continue to progressively build value in our DMD programme,” said Frank Gleeson, CEO, and co-founder of Satellos.
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By GlobalData“The RPDD for SAT-3247 highlights the continued need for new disease-modifying therapeutic options for paediatric patients with DMD, a need which we believe SAT-3247 has the potential to address.”
SAT-3247 is a first-in-class oral small molecule therapeutic designed to restore the innate muscle regeneration and repair process, independent of dystrophin and regardless of exon mutation status. The drug candidate operates by inhibiting adaptor-associated kinase 1 (AAK1) to modulate muscle stem cell polarity leading to the repair and regeneration of functional muscle tissue.
DMD landscape
DMD is a severe type of muscular dystrophy predominantly affecting boys. The onset of muscle weakness typically begins around the age of four, with rapid progression.
On 12 June, Pfizer announced its DMD therapy fordadistrogene movaparvovec failed to meet the primary endpoint of a Phase III study (NCT04281485). This came following the company’s announcement of 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.
