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A 4th View: Discussing GBT's recent Sickle Cell Phase 3 HOPE Results for Voxelotor (Previously GBT440)Ticker(s): GBT, BLUE
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.
Please describe your background and experience with Sickle Cell Disease and any work with Voxelotor.Added By: c_admin
What is your high level opinion of today's top-line HOPE trial data?Added By: c_admin
On a scale of 1-10 how excited about this program are you for patients?Added By: c_admin
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
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?
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: c_admin
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."
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: c_admin
What is your opinion on other treatments in clinical trials for SCD? Specifically selectin-class and bluebird bio's gene-related approaches?
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"
To what extent is voxelotor's market potential limited by gene therapy approaches to SCD?Added By: c_admin
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?
How familiar are you with Voxelotor? What is your opinion of it considering all of the publicly known data?Added By: c_admin
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: c_admin
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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