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Expert Interview

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

A 3rd View: Discussing GBT's recent Sickle Cell Phase 3 HOPE Results for Voxelotor (Previously GBT440)

Ticker(s): GBT, BLUE

Who's the expert?

Name: Dr Nirmish Shah - MD

Institution:  Duke

  • Assistant Professor in Medicine and Pediatrics and Director of Clinical Research in Benign Hematology.
  • Manages 350 out of the 900 patients with sickle cell disease at Duke University and 10 patients with beta thalassemia.
  • Director of Duke’s sickle cell transition program where he has led multiple trials in sickle cell disease and is 75% research focused.

Interview Questions
Q1.

Please describe your background and experience with Sickle Cell Disease and any work with Voxelotor.

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Q2.

What is your high level opinion of today's top-line HOPE trial data?

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Q3.

Investors were particularly concerned about the PRO secondary endpoint result. How concerning are these for you? What do you think of the company's attribution to the lack of a statistically significant result on low-baseline scores.

  • "The patient reported outcomes (PRO) data were difficult to interpret due to low baseline symptom scores and high inter-subject and intra-subject variability. Given this, GBT does not plan to utilize the PRO as a key secondary endpoint." ~ Link

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Q4.

What additional questions do you have for the full data presentation. Does the magnitude of effect matter for the measures below if they are statistically significant and dose dependent?

  • Hemoglobin 
  • Reticulocytes 
  • Bilirubin

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Q5.

Do you think the company has a strong argument for accelerated approve based on this top-line data? 

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Q6.

On a scale of 1-10 how excited about this program are you for patients?

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Q7.

What do you think needs to be demonstrated for approval of the drug? Keeping in mind the primary endpoint is Hb increase of >1g/dL, secondary of VOC and PRO outcomes.

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Q8.

What is your opinion on the PRO result?  Does the lack of a stat-sig outcome surprise you or concern you? What do you make of the company's explanation? 

  • "There were numerically fewer VOC episodes in both voxelotor groups than in the placebo group, which as anticipated did not reach statistical significance due to limited patient follow-up."

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Q9.

What is your opinion on the theoretical safety risk including ischemia, stroke, peripheral hypoxia, and off-target aldehyde mediated binding to protein N-termini? Do you think the risk is more prominent in patients treated with hydroxyurea due to higher O2 affinity of HbF?

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Q10.

What is your opinion on other treatments in clinical trials for SCD? Specifically selectin-class and bluebird bio's gene-related approaches?

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Q11.

Previously investors were concerned about the impact of background use of hydroxyurea. Do you think this result puts that question to rest?

  • "Improvements in these clinical measures of anemia and hemolysis were similar in patients with or without background use of hydroxyurea. Approximately 64 percent of patients enrolled in Part A are on background use of hydroxyurea"

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Q12.

To what extent is voxelotor's market potential limited by gene therapy approaches to SCD? 

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Q13.

How long would a patient take Voxelotor before you would consider it a safe drug to take indefinitely?  Do you have safety concerns about this drug?

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Q14.

How familiar are you with Voxelotor? What is your opinion of it considering all of the publicly known data?

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Q15.

The company seems to have hinted on their conference call that the drug may decrease TCD velocities. If that proves out would you consider this a meaningful clinical benefit? Would you prescribe the drug based on just the Hb + retic + bilirubin data or would you need to see a clinical benefit first?

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