Wave rides DMD success to regulators, boosting stock

Wave Life Sciences has achieved a significant milestone in its study of Duchenne muscular dystrophy (DMD), a severe genetic disorder characterized by muscle degeneration. The company is now in a position to discuss accelerated approval with regulatory authorities, even as it continues to monitor patients through the completion of the trial. Shares in the company surged by 39%, opening at $7.40.

In its recent study, Wave Life Sciences reported a mean absolute unadjusted dystrophin level of 5.5% among nine ambulatory boys with exon 53 DMD after 24 weeks of treatment with its oligonucleotide therapy, WVE-N531. The biotech firm had set a target expression level above 5% prior to the data readout. This figure excludes one non-ambulatory patient with significantly lower dystrophin levels post-treatment.

The results align closely with data that supported the accelerated approval of NS Pharma’s exon 53 DMD drug, Viltepso. NS Pharma reported mean dystrophin levels of 5.9% of normal by Week 25, reflecting a 5.3% increase from baseline. However, NS Pharma's mean results were influenced by two outliers, with dystrophin levels exceeding 10% of normal, while the remaining patients exhibited lower levels, with three showing less than 3.1%.

Wave's study showed dystrophin levels exceeding 5% of normal in six patients. In the other three ambulatory patients, levels ranged from 3.3% to 4.8%. The single non-ambulatory patient had a dystrophin level of just 1% of normal. Additionally, Wave provided data on muscle content-adjusted dystrophin, a crucial factor given the muscle’s central role in DMD-related health challenges. The mean muscle content-adjusted dystrophin expression was reported at 9%.

The results demonstrated a consistent performance across the cohort, with expression levels ranging from 6% to 13.9% in the eight ambulatory patients, and one outlier showing an expression level of 1.2%. Only one ambulatory patient had an expression level below Wave’s 5% target, at 4.6%.

The study administered WVE-N531 every two weeks, mirroring the dosing schedule of Viltepso. However, Wave Life Sciences believes its data supports the feasibility of monthly dosing. According to CEO Paul Bolno, M.D., the drug shows a high level of muscle exposure with a half-life of 61 days, suggesting that monthly dosing would not only maintain efficacy but also preserve the production range of dystrophin.

“The muscle contains a substantial amount of the drug, which should facilitate the continuous production of dystrophin,” Bolno said in a call with analysts. He emphasized the necessity of monthly dosing for families and patients, highlighting the challenges of frequent weekly IV infusions.

As a result, Wave is transitioning patients in the trial to a monthly dosing regimen. The company anticipates reporting 48-week data in the first quarter of 2025 and expects regulatory feedback on the pathway to accelerated approval around the same time.

On the announcement of these promising results, shares in Wave Life Sciences opened up 39% at $7.40, reflecting investor optimism about the future prospects of the company's DMD therapy.