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Expert Interview

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

Exploring the potential of PRAX-222 in patients with SCN2A gain-of-function developmental epilepsies.

Ticker(s): PRAX

Who's the expert?

Institution: CHU Sainte- Justine

  • Child neurologist and clinical neurophysiologist
  • Treats pediatric patients with epilepsy including SCN2A GOF related epilepsy
  • Clinical and research interests are relation to predictors of epilepsy surgery outcomes in children.

Interview Questions
Q1.

Could you elaborate on how PRAX-222 functions as a novel antisense oligonucleotide in targeting SCN2A gain-of-function mutations? How does this approach differ from traditional treatments for neurological disorders?

  • Based on the results from the EMBRAVE clinical trial, how does PRAX-222's efficacy in managing early-onset SCN2A developmental and epileptic encephalopathy compare to existing treatments?

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Q2.

What insights can you provide regarding the safety profile of PRAX-222, particularly in pediatric patients? Were there any notable side effects or concerns highlighted in the trial?

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Q3.

In light of the data presented, how does PRAX-222 potentially impact the quality of life and developmental outcomes for patients with SCN2A gain-of-function mutations?

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Q4.

Considering the data on PRAX-628, a next-generation small molecule for focal epilepsy, how does PRAX-222 compare in terms of potency and potential as a best-in-class treatment?

  • Based on the novel method to define an EEG composite for detecting drug effects, how does PRAX-222 influence EEG patterns in patients, and what does this imply for its therapeutic efficacy?

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Q5.

How does the novel translational concordance framework aid in understanding PRAX-222's effectiveness across different seizure models, and what does this mean for its application in both focal and generalized epilepsies?

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Q6.

Given the current and emerging therapies in the field, where do you see PRAX-222 fitting into the broader treatment paradigm for neurological disorders linked to SCN2A mutations?

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Q7.

Considering the unique properties of PRAX-222 as an antisense oligonucleotide, can you provide insights into its potential scalability and production capabilities for widespread clinical use?

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Q8.

Can you describe the process of developing the qEEG composite using machine learning algorithms? How did you ensure its accuracy and sensitivity in predicting the treatment effect of next-generation small molecules in epilepsy?

  • The abstract presented shows qEEG composite can distinguish pharmacodynamic effects of novel agents from placebo with high accuracy. How do you envision this method impacting the future of drug development in epilepsy, particularly in accelerating the process and improving the reliability of clinical trials?

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Q9.

Traditional EEG methods have limitations in sensitivity and power, especially with high inter-subject variability. How does your novel qEEG composite address these challenges, and what advantages does it offer over conventional EEG techniques in detecting pharmacodynamic effects?

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Q10.

  • In the abstract they presented a developed a unified translational scoring matrix to assess the concordance between preclinical seizure models and clinical epilepsy types. Could you elaborate on how this scoring matrix was constructed and how it helps in determining the predictive validity of these models for both focal and generalized epilepsies

  • Given the findings presented in the abstracts, which identified models like MES, mouse audiogenic, and mouse 6-Hz (32mA) as having high predictive validity, how do you foresee this impacting the development and testing of new anti-seizure medications? Will this lead to a more targeted and efficient approach in preclinical testing?

Added By: sara_admin

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