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Expert Interview

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

Analyzing the Previously Released Data on Mast Therapeutics' (MSTX) Vepoloxamer in Sickle Cell Disease and What It Can Tell Us About The Drug's Chance For Success In The Phase 3 EPIC Trial (Interview 1 of 3)

Ticker(s): MSTX, PFE, 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

Can you describe your experience treating patients with a vaso-occlusive crisis from sickle cell disease? How would you describe the safety and efficacy of narcotic analgesics currently used in treatment? What other methods are used to treat a vaso-occlusive crisis associated with sickle cell disease?

Added By: pjloria

Looking at the Company's arguments in support of EPIC potentially succeeding on a high level how strong do you think they are? 

Added By: pjloria

Vepoloxamer demonstrated a statistically significant reduction in the duration of vaso-occlusive crises in patients on hydroxyurea and pediatric patients in the 2001 trial.  Does the selection of these subgroups make sense to you from a mechanistic or clinical perspective?

Added By: pjloria

The company has changed the endpoint from the failed 2001 trial to the current EPIC trial.  How important are these changes?

Added By: pjloria

The company powered the trial based on the assumption that 96 hours was the average duration of a crisis with a standard deviation of 51 hours.  How well established is this average? The previous trial seemed much longer for both treatment and placebo patients.

Added By: pjloria

Given the standard deviation of 51 hours, how clinically meaningful would a 16 hour reduction really be?  From a statistical standpoint does it sound like this trial is powered to detect the reduction with this high a standard deviation?

Added By: pjloria

Does the endpoint of: duration from randomization to last dose of parenteral opioid have any validation in other studies? How does this endpoint strike you from a clinical perspective. Does it solve in your mind the issues with the earlier trials?

Added By: pjloria

Interim EPIC study results provided by the company in September 2015 indicated that after 250 patients treated, the mean duration is 79 hours, w/ SD at 47 hours. The study was built on the expectation that patients on placebo would result in a mean of 96 hours and SD of 51 hours. If the placebo estimate of 96 hours is reliable, that would mean the treatment arm experienced a mean crisis duration of 62 hours, or twice the expected/hoped for 16 hour duration. Are these conclusions valid? It appears much depends on the placebo crisis duration to determine whether or not the treatment arm delivers results that are statistically significant.

Added By: user746b570e

Will EPIC study have to deliver on Primary End Point AND Secondary End Points to be considered successful, and what, in your opinion, will the FDA require for Vepoloxamer to be approved?

Added By: user746b570e

How logical is the mechanism of action for Vepoloxamer? Does the company's argument that it could be synergistic with Hydroxyurea plausible to you? How?

Added By: joemac84

If the drug has success in this more pediatric population do you think it will be used widely in all patients?

Added By: pjloria

There has been concern about this drug in a world where BlueBird and other gene therapy companies succeed in Sickle Cell.  Can you talk about those programs and specifically what patient overlap there is?  I know BlueBird's LentiGlobin BB305 drug is focused on patients with transfusion-dependent β-thalassemia. How much impact would success in this program have on Vepoloxamer's addressable market?

Added By: joemac84

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