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Expert Interview

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

Angelman Syndrome Pipeline: Recent Updates on GTX-102 (Ultragenyx & GeneTx), ION582 (Ionis Pharmaceuticals), and rugonersen.

Ticker(s): OAK HILL BIO, IONS, RARE

Who's the expert?

Institution: University of California San Diego

  • Dysmorphologist and clinical geneticist at Rady Children's, and a professor of clinical pediatrics at UC San Diego.
  • Medical director of Rady Children's multidisciplinary Prader-Willi Syndrome Clinic and co-founder and medical administrator of Rady Children's Down Syndrome Center.
  • Research focuses on Angelman syndrome, and she is the principal investigator in San Diego of a multi-site consortium that conducts a natural history study and clinical trials. 

Interview Questions
Q1.

Roughly how many patients with Angelman syndrome do you currently manage?

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Q2.

What are your thoughts on the recent data presented on ION582?

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Q3.

What are your thoughts on the recent data presented on GTX-102?

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Q4.

What are the main challenges in diagnosing Angelman syndrome, and how does insurance coverage impact this?

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Q5.

What are your thoughts on the safety and efficacy of ION582 compared to other ASOs for Angelman syndrome, and what might explain the discontinuation of Roche's rugonersen despite promising data?

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Q6.

Is there room for more than one approved ASO for AS?

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Q7.

All the companies have generated EEG biomarker data. Any thoughts on what this shows?

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Q8.

What are your thoughts on the data that Roche presented at ASF and FAST?

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Q9.

Anecdotally, how would you describe the responses you've seen to the different drugs?

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Q10.

Have you seen any work done nonclinically for how the ASOs compare?

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Q11.

how confident are you in ultragenyx's ability to manage safety? they saw toxicity at lower doses than ionis and roche.

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Q12.

were there differences in how the tests / assessments were performed for the different trials? did all use the same standards for vineland? bayley?

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Q13.

how would you evaluate the trade-off in safety and efficacy? would you generally want to push the dose to get to greater efficacy, even if it increases some of the side effects seen, like vomiting and pyrexia?

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Q14.

what are your plans for participation in the phase 3 trials?

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Q15.

assuming an ASO is approved, any feeling for what percent of your patients would be interested in treatment?

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