Wave Life And Capricor Surge Forward With Hope - Are These Stocks In Your Portfolio?

(RTTNews) - Duchenne muscular dystrophy, or DMD, is a severe genetic disorder, typically affecting males, and is characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Patients suffering from DMD lack a muscle protein called dystrophin, and they typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. There are about 200,000 DMD patients worldwide.

Although there is no cure for this childhood form of muscular dystrophy, there are several approved drugs to treat this condition. The approved drugs include Sarepta Therapeutics' Exondys 51, Vyondys 53, Amondys 45, and Elevidys; PTC Therapeutics' Emflaza; NS Pharma's Viltepso; and Italfarmaco Group's Duvyzat.

Tuesday (Sep.24, 2024), the stocks of two companies operating in the DMD landscape reached a new 52-week high, driven by encouraging news about their investigational DMD therapies.

Wave Life Sciences Ltd. (WVE)

Wave Life Sciences' phase II clinical trial of WVE-N531 in boys with DMD amenable to exon-53 skipping, dubbed FORWARD-53, has yielded positive interim data. The interim analysis was conducted after 24 weeks of 10 mg/kg dosing every two weeks.

In the FORWARD-53 trial, treatment with WVE-N531 resulted in mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, with high consistency across participants. "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," said Anne-Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences.

The company expects to complete the FORWARD-53 trial and receive feedback from regulators on a pathway to accelerated approval in the first quarter of 2025.

We alerted readers to WVE on Sep.18, 2024, when it was trading at around $5.70. The stock touched a 52-week high of $8.35 during intraday trading Tuesday, before closing at $8.19, up 53%.

Capricor Therapeutics Inc. (CAPR)

Capricor intends to file a Biologics License Application for its drug candidate Deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy cardiomyopathy based on existing cardiac and natural history data following recent meetings with the FDA.

The BLA filing is expected to commence next month with full submission expected by year-end 2024. The company noted that the BLA filing will be based on existing cardiac data from the phase II HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children's Hospital Medical Center.

A phase III trial of CAP-1002 in patients with DMD and impaired skeletal muscle function, dubbed HOPE-3, is underway. The study is comprised of two cohorts - A and B.

Capricor plans to combine Cohorts A and B of the phase III HOPE-3 clinical trial to serve as a post-approval study to support potential label expansion to treat DMD skeletal muscle myopathy. The company does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.

We alerted readers to CAPR on Aug.8, 2024, when it was trading at around $4.02. The stock touched a 52-week high of $9.24 during intraday trading Tuesday, before closing at $9.10, up 52%.