Entrada given go-ahead to test DMD therapy after two-year hold
Here is a summary of the key information about Entrada Therapeutics' DMD therapy trial:
FDA Removes Clinical Hold on ENTR-601-44
The FDA has lifted the clinical hold on Entrada Therapeutics' investigational therapy ENTR-601-44 for Duchenne muscular dystrophy (DMD)2. This allows Entrada to initiate the ELEVATE-44-102 Phase 1b multiple ascending dose clinical study.
Key details:
- The study will evaluate ENTR-601-44 in approximately 32 adult DMD patients amenable to exon 44 skipping2
- It will include both ambulatory and non-ambulatory patients2
- Dosing will be administered every 6 weeks across 4 cohorts, ranging from 0.16 mg/kg to 1.28 mg/kg2
- Enrollment is planned to begin in the first half of 20262
Significance of the Trial
- This study targets an underserved population of adult DMD patients, who represent nearly 50% of those amenable to exon 44 skipping2
- It addresses a gap in current DMD trials, which often focus on pediatric patients2
- The inclusion of both ambulatory and non-ambulatory patients allows for comprehensive evaluation2
Background on DMD and Exon Skipping
DMD is caused by mutations in the DMD gene that lead to a lack of dystrophin protein3. Exon skipping therapies aim to produce a shortened but functional dystrophin protein5.
This trial approval marks an important step forward for Entrada Therapeutics in developing a potential treatment for DMD, especially for adult patients who have limited therapeutic options.
Sources:
2. https://www.stocktitan.net/news/TRDA/entrada-therapeutics-announces-fda-removal-of-clinical-hold-on-entr-l44jfp0s3zfa.html
3. https://musculardystrophynews.com/news/pfizer-stops-dmd-gene-therapy-development-trial-failure/
5. https://musculardystrophynews.com/news/phase-1-2-trial-exon-44-skipping-therapy-dmd-cleared-uk/