Avidity Biosciences has reported positive findings from the Phase I/II EXPLORE44 clinical trial of AOC 1044 in healthy subjects to potentially treat Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44). 

The placebo-controlled, randomised, double-blind trial is designed to analyse the tolerability, safety, pharmacokinetics and pharmacodynamic effects of single and multiple ascending intravenous doses. 

The trial is expected to enrol 40 healthy subjects and 24 DMD44 participants aged seven to 27 years. 

It will also analyse exon skipping and dystrophin protein levels in the participants.

According to the findings, AOC 1044 offered phosphorodiamidate morpholino oligomers (PMO) concentrations in skeletal muscle. The single-dose treatment resulted in up to 50 times improved PMO concentrations in skeletal muscle versus peptide-conjugated PMOs in healthy subjects. 

Furthermore, AOC 1044 was found to be well tolerated with up to 1.5% exon 44 skipping following a single 10mg/kg dose versus placebo in healthy subjects. The treatment also resulted in higher exon skipping in all subjects.

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By GlobalData

Avidity plans to report the initial AOC 1044 data from the trial in the second half of next year.

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug designation for the therapy, which also received fast track designation from the FDA. 

Mild to moderately severe treatment-emergent adverse events were reported in participants who received AOC 1044.

Avidity Biosciences president and CEO Sarah Boyce said: “We are excited with the early data set of AOC 1044 demonstrating unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers. 

“We are rapidly advancing AOC 1044 and look forward to sharing data in people living with DMD44 in 2024. Data from our clinical programs continue to reinforce the broad and disruptive potential of our AOC platform for the treatment of high burden muscle diseases like DMD, DM1 and FSHD.”

The company is also analysing AOC 1001 in the MARINA open-label extension (MARINA-OLE) trial in myotonic dystrophy type 1 (DM1) patients.

Another asset, AOC 1020, is being analysed in the Phase I/II FORTITUDE trial to treat facioscapulohumeral muscular dystrophy (FSHD).