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Expert Interview

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FLASH CALL: Discussing this morning's Sickle Cell Phase 3 HOPE Results for Voxelotor (Previously GBT440)

Ticker(s): GBT, BLUE

Who's the expert?

Name: Dr Cage Johnson - MD

Institution: University of Southern California

  • Among the world’s leading authorities on sickle cell disease; former Director of the Sickle Cell Center at Los Angeles County and USC Medical Center and Professor Emeritus of Medicine & of Physiology & Biophysics.
  • Currently manages 125 patients with sickle cell disease.
  • Has published nearly 100 papers on sickle cell disease and other hemoglobinopathies in peer reviewed journals and has presented with the National Institutes of Health, the National Science Foundation, the Food and Drug Administration and the Sickle Cell Foundation.

Interview Questions

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

Added By: joe_mccann

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

Added By: joe_mccann

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

Added By: joe_mccann

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

Added By: joe_mccann

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

Added By: joe_mccann

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

Added By: joe_mccann

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.

Added By: joe_mccann

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."

Added By: joe_mccann

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?

Added By: joe_mccann

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"

Added By: joe_mccann

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

Added By: joe_mccann

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

Added By: joe_mccann

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?

Added By: joe_mccann

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

Added By: joe_mccann

Could side effects related to hydroxyurea be a factor in patient's symptoms and in your opinion could Voxelotor work better if administered without hydroxyurea say after first few weeks?

Added By: xsdenied

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