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Discussing the Sickle Cell Landscape and Emerging Treatment Options Including Voxelotor, Rivipansel, and LentiGlobin.

Ticker(s): GBT, PFE, GLYC, BLUE

Who's the expert?

Name: Dr Cindy Neunert- MD

Institution: Columbia University

  • Pediatric Hematologist & Associate Professor of Pediatrics at Columbia University.
  • Currently treats 10-20 patients per week with sickle-cell disease in a practice with over 300 SCD patients.
  • Served as an investigator for the RESET clinical trial for SDC, and research interests are focused on understanding and evaluating patient-related outcomes and shared-decision making in benign hematology.   

Interview Questions

Questions from Damian Garde to come! 

Added By: joe_mccann

Right so, the good news is, there are more than 15 therapies in the pipeline that might improve the lives of these patients. Let’s start with the most advanced ones.

Added By: damiangarde

Another small-molecule therapy in development is rivipansel, which was invented by a company called GlycoMimetics and is being developed by Pfizer. So this one has a somewhat similar mechanism to the Novartis antibody, and we’re expecting to get top-line results from a late-stage trial any day now. But i wanted to talk about the Phase 2 data. The companies looked at rivipansel in sickle cell patients hospitalized with VOCs to see how quickly the drug could resolve them and get patients out of the hospital. That study did not meet either endpoint. What do you think about rivipansel's prospects? [would there be a place for a therapy like this if Novartis’s antibody is approved to resolve VOCs?]

Added By: damiangarde

Now let’s talk about gene therapy. Bluebird Bio has a gene therapy called Lentiglobin, now in clinical trials, that could be a one-time therapy for sickle cell. We’ve seen data from a handful of patients, followed for a handful of months. What do you think of the safety and efficacy so far?

Added By: damiangarde

Crizanlizumab is a monoclonal antibody, developed by Novartis, that could win approval in the U.S. within a year. What did you make of the clinical data we’ve seen to date on this antibody? [SUSTAIN showed that crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3% compared to placebo (1.63 vs 2.98, P=0.010) in patients with or without hydroxyurea therapy. The study also demonstrated that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo (35.8% vs 16.9%, P=0.010) during treatment4.]

Added By: damiangarde

Now let’s talk about voxelotor. That’s a once-a-day pill developed by a company called Global Blood Therapeutics. What’s interesting me is that while Novartis has data on those vaso-occlusive crises that are so important, GBT is seeking FDA approval based on their drug’s effect on hemoglobin. What do you think about that -- what is the relationship between hemoglobin production and VOCs? [can we infer an effect?]

Added By: damiangarde

This seems like a natural time to zoom out. We’re looking at a potential future in which sickle patients will have a choice between a monthly antibody injection, a daily pill, or a one-time gene therapy. How would you approach that? Are there situations where you’d reach for one over the others?

Added By: damiangarde

GBT has stated that its trial was not powered to show statistical significance in reduction of VOC events, but that the trial nevertheless showed a meaningful reduction in VOC events . Related to Q 11, do you think that doctors treating SCD will prescribe this drug for its impact on hemoglobin levels and its positive trend on VOC events or do you think they will wait to see further evidence of reduced occurrence of VOC events before prescribing?

Added By: gkiernan

So we’re going to get into the treatment landscape and the potential of various investigational medicines, but first I was thinking Dr. Neunert it might be helpful for everyone on the call if you could briefly describe your practice and your exposure to and work in sickle cell disease.

Added By: damiangarde

How much of a factor is cost? [we know gene therapy costs millions of dollars, but also monoclonal antibody costs add up over time]

Added By: damiangarde

Further down the pipeline we have a couple of approaches that use genome editing to modify stem cells and get them to produce healthy hemoglobin. What do you think of those approaches? [Would they be practical if there’s already a gene therapy? How much better would they need to be?]

Added By: damiangarde

Let’s get into the details of the disease. How prevalent is sickle cell, and what is the treatment algorithm for patients once they’re diagnosed?

Added By: damiangarde

Medication adherence is a common concern, especially as patients transition from pediatric to adult care. How important a consideration will adherence be for doctors as therapeutic options (with highly variable dosing strategies, as stated in Q6) increase?

Added By: jperry

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