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Expert Interview

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

A 2nd Look: Discussing this week's Sickle Cell Phase 3 HOPE Results for Voxelotor (Previously GBT440)

Ticker(s): GBT, BLUE

Who's the expert?

Name: Dr Lewis Hsu - MD/ PhD

Institution: University of Illinois

  • Professor of Pediatric Hematology-Oncology and Director of Pediatric Sickle Cell.
  • Currently manages 170 patients with sickle cell; Active investigator in multicenter clinical trials of potential therapies for sickle cell sponsored by pharmaceutical companies and by NIH
  • Research experience with mouse models and human studies on vascular adhesion of blood cells and inflammation as contributors to sickle vaso-occlusive pain, partial chimeric stem cell transplant for cure, sickle acute chest syndrome, priapism, and nitric oxide therapy

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?

Added By: bin2gray

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