Wave Life Sciences is eyeing an accelerated approval for its Duchenne muscular dystrophy (DMD) drug, with the company’s stock price soaring by more than 50% after Phase II trial data was unveiled.

Patients in the Phase II FORWARD-53 trial (NCT04906460) benefitted from substantial dystrophin expression and the WVE-N531, an exon skipping oligonucleotide, was safe and well tolerated.

Off the back of the positive data, the company is planning to meet with the US Food and Drug Administration (FDA) to push for accelerated approval, with hopes to hear back from the agency before the end of Q1 2025.

The company’s stock rose by 53.37% from $5.34 at close on 23 September to $8.19 at close yesterday (24 September).

Chief development officer, Anne-Marie Li-Kwai-Cheung said: “The high and consistent dystrophin levels at this interim timepoint are compelling and speak to the potential of WVE-N531 for boys amenable to exon 53 skipping, where better therapeutic options are urgently needed.”

The open-label trial is investigating WVE-N531 in 11 boys, aged five to 11 with DMD who are amenable to exon 53 skipping.

There was a mean absolute muscle content-adjusted dystrophin expression of 9%. The mean absolute unadjusted dystrophin expression was 5.5%, as measured by Western Blot. Dystrophin expression was quantified from two isoforms consistent with those observed in Becker muscular dystrophy patients who display milder disease. Patients also showed multiple indicators of improvement in muscle health.

Mean exon skipping was 57% as measured by reverse transcription polymerase chain reaction (RT-PCR).

Complete 48-week data from the FORWARD-53 trial is also expected to be announced in Q1 2025.

The company also plans to announce proof-of-mechanism data for the WVE-006 programme in Alpha-1 antitrypsin deficiency before the end of 2024. In Q1 2025, the company hopes to initiate a trial for WVE-007 in obesity.

DMD landscape

There have many developments in the DMD landscape.

On 24 September, Capricor Therapeutics announced it will file a Biologics Licence Application (BLA) to the FDA for deramiocel after positive data from the HOPE-3 trial (NCT05126758).

In August 2024, Pfizer discontinued its gene therapy fordadistrogene movaparvovec after a Phase III trial (NCT04281485) did not meet its primary endpoint and a patient died in a Phase II trial (NCT05429372).

Meanwhile, Sarepta Therapeutics was handed a label expansion for Elevidys (delandistrogene moxeparvovec-rokl), to be used in ambulatory and non-ambulatory patients aged four and over with DMD back in June. The drug was first granted approval in 2023.

Also in March 2024, the FDA approved Duvyzat (givinostat), an oral medication for patients aged six and older.