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Expert Interview

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

A 4th 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 Richard Drachtman - MD

Institution: Rutgers Medical School

  • Interim Chief, Pediatric Hematology/Oncology and Director, Comprehensive Sickle Cell Center at Rutgers Cancer Institute of New Jersey; Vice Chair, IRB at Rutgers Biomedical and Health Sciences; and Vice Chair, NCI Pediatric Central IRB.
  • Currently manages 400 patients with sickle cell disease.
  • Research focuses on Hodgkin Disease, Pediatric oncology/ hematology, and Sickle cell disease.

Interview Questions

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

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

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

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

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

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

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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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In their recent call, the company alluded to the fact that while measuring the number of VOCs has been/is an easily quantifiable endpoint it may not be the best assessment for long-term outcomes with correction of hemoglobin being a better (more important?) factor in determining efficacy in terms of overall survival.  Do you feel this is a valid argument based on your clinical experience?

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