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Expert Interview

Slingshot members are talking to an expert! The topic is:

Exploring Sarepta Therapeutics' Elevidys Gene Therapy for Duchenne Muscular Dystrophy ahead of June 21 PDUFA

Ticker(s): SRPT

Who's the expert?

Institution: Duke University

  • Assistant Professor in the Department of Pediatrics, Division of Neurology at Duke University. Co-Director of Duke Children's Neuromuscular Program
  • Manages 20 patients with Becker Muscular Dystrophy
  • Research interest in Duchenne Muscular Dystrophy, spinal muscular dystrophy, and other rare forms of neuromuscular disease including Becker Muscular Dystrophy.

Interview Questions
Q1.

The EMBARK trial showed an increase in NSAA total score in Elevidys-treated patients compared to placebo at 52 weeks, although the primary endpoint was not met. Can you explain the clinical relevance of this score and how even a modest increase might impact patients with Duchenne muscular dystrophy?

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Q2.

Elevidys demonstrated robust, statistically significant results on all key pre-specified secondary endpoints, including time to rise and the 10-meter walk test. How do these endpoints reflect the overall effectiveness of the treatment, and what do they suggest about the potential long-term benefits for patients?

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Q3.

The positive effects of Elevidys were consistent across all age groups in the trial. Why is it important to see this consistency, and what implications does it have for treating different age cohorts within the DMD population?

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Q4.

Time to rise was highlighted as a strong predictor of early loss of ambulation. Can you discuss the importance of this measure in the context of DMD progression and how Elevidys' ability to improve this metric might influence disease management?

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Q5.

No new safety signals were observed in the EMBARK study. How do the safety profiles of Elevidys compare to other current DMD treatments, and what do these results indicate about its potential for long-term use?

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Q6.

The study mentioned significant improvements in stride velocity 95th centile using a community wearable activity monitor. How does this novel measure provide insight into the everyday mobility of DMD patients, and what advantages does it offer over traditional clinical assessments?

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Q7.

The FDA has indicated openness to reviewing data for label expansion based on the totality of evidence from EMBARK. What are the potential benefits and challenges of expanding Elevidys' indication to treat all patients with DMD?

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Q8.

Given the promising results from the EMBARK trial, what are the next steps in the development of Elevidys? Are there plans to investigate its efficacy in other neuromuscular disorders or broader patient populations within DMD?

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