Duchenne muscular dystrophy (DMD) is a serious genetic disorder, mainly affecting males, characterized by a gradual weakening and chronic inflammation of the skeletal, heart, and respiratory muscles. DMD patients lack the muscle protein dystrophin, leading to a loss of walking ability during adolescence and typically resulting in cardiac or respiratory complications by age 30. The global patient population is approximately 200,000.
Despite the lack of a cure for this childhood-onset muscular dystrophy, several approved drugs are available for its treatment. These include Sarepta Therapeutics' Exondys 51, Vyondys 53, Amondys 45, and Elevidys; PTC Therapeutics' Emflaza; NS Pharma's Viltepso; and Italfarmaco Group's Duvyzat.
On Tuesday, September 24, 2024, stocks of two companies in the DMD field reached new 52-week highs, fueled by promising news concerning their investigational therapies.
### Wave Life Sciences Ltd. (WVE)
Wave Life Sciences reported positive interim data from its phase II clinical trial of WVE-N531 for boys with DMD amenable to exon-53 skipping, designated FORWARD-53. This interim analysis followed 24 weeks of administering 10 mg/kg every two weeks.
The FORWARD-53 trial showed that WVE-N531 treatment resulted in a mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, demonstrating high consistency among participants. “The consistently high dystrophin levels at this interim stage are very encouraging and highlight the potential of WVE-N531 for boys amenable to exon 53 skipping, where better therapies are critically needed,” stated Anne-Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences.
The company aims to complete the FORWARD-53 trial and gain regulatory feedback on a pathway to accelerated approval by Q1 2025.
We informed our readers about WVE on September 18, 2024, when it was trading at about $5.70. The stock hit a 52-week high of $8.35 during intraday trading Tuesday, closing at $8.19, marking a 53% increase.
### Capricor Therapeutics Inc. (CAPR)
Capricor Therapeutics plans to file a Biologics License Application (BLA) for its candidate, Deramiocel, to treat all DMD patients diagnosed with cardiomyopathy based on existing cardiac and natural history data, following FDA meetings.
The BLA filing will commence next month, with full submission anticipated by the end of 2024. The application will leverage cardiac data from the phase II HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, along with natural history data from Vanderbilt University Medical Center and Cincinnati Children's Hospital Medical Center.
A phase III trial of CAP-1002 in DMD patients with impaired skeletal muscle function, named HOPE-3, is ongoing, consisting of two cohorts—A and B.
Capricor plans to combine Cohorts A and B in the phase III HOPE-3 trial to support a potential label expansion to treat DMD skeletal muscle myopathy post-approval. The company will not unblind Cohort A at this time, a step initially expected in Q4 2024.
We alerted our readers to CAPR on August 8, 2024, when it was trading at approximately $4.02. The stock peaked at a 52-week high of $9.24 during intraday trading Tuesday, closing at $9.10, representing a 52% gain.