Avidity Biosciences’ Duchenne muscular dystrophy (DMD) therapy, del-zota, demonstrated reversal of disease progression in a Phase I/II and open-label extension trial.
In the EXPLORE44 (NCT05670730) and EXPLORE44-OLE (NCT06244082) trials, del-zota, also known as delpacibart zotadirsen, demonstrated up to a 25% increase in normal dystrophin production and restored total dystrophin up to 58% of normal.
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Creatine kinase (CK) levels rapidly reduced by more than 80% and were sustained at near normal levels for up to 16 months. 50% of patients had CK levels within the normal range at one year of treatment.
As well as these endpoints, in the 4-Stair Climb (4SC), patients improved by 2.1 seconds while the natural history group declined by 2.7 seconds. In the 10m Walk/Run Test (10mWRT), patients improved by 0.7 seconds compared to a decline of 1.5 seconds in the natural history group.
In Time to Rise from Floor (TTR), patients improved by 3.2 seconds while the natural history group declined by 1.6 seconds and in Performance of Upper Limb (PUL2), patients treated improved by 1.5 points. In contrast, the natural history group declined from baseline by 0.7 points.
In the North Star Ambulatory Assessment (NSAA), patients who received del-zota remained stable; meanwhile, the natural history group declined from baseline by 2.4 points.
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By GlobalDataDel-zota continued to demonstrate a favourable long-term safety and tolerability profile, with just one patient discontinuing treatment in the EXPLORE44-OLE following an event of hypersensitivity.
The recent success will support Avidity’s biologics licence application (BLA) to the US Food and Drug Administration (FDA) before the end of this year for accelerated approval of the DMD drug.
Avidity CEO Sarah Boyce said: “For the first time, we have data showing that sustained muscle protection leads to meaningful improvements across multiple key functional measures in DMD. These unprecedented data underscore the impact of our revolutionary targeted approach to deliver RNA directly to muscle.”
Avidity Biosciences’ stock is up nearly 5% on the readout, from a 9 September market close of $46.62 to a 10 September market high of $48.93 at the time of writing. The company has a market cap of $6.3bn.
Del-zota acts by delivering phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin mRNA and enable production of functional, near-full-length dystrophin. Approximately 7% of the DMD population presents with this mutation, meaning that there is a gap in the market for this specific variety of exon-skipping therapy.
Earlier this month, Capricor released its reply to the FDA after it received a complete response letter (CRL) for deramiocel, another DMD candidate. The agency published the CRL as part of a transparency push; however, Capricor has hit back, stating that the FDA interpreted the data incorrectly.
Research by GlobalData estimates that across the seven major markets (7MM: US, France, Germany, Italy, Spain, the UK, and Japan), the market for DMD treatments is expected to rise from sales of $2.3bn in 2023 to $5.2bn by 2033. This is mostly driven by Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec) and Santhera Pharmaceuticals’ Agamree (vamorolone).
GlobalData is the parent company of Clinical Trials Arena.
