Auspex SD-809 Presentation-HDSA 2013

SD-809 for the Treatment of Chorea
Associated with Huntington’s Disease
David Stamler, M.D.
Chief Medical Officer
Auspex Pharmaceuticals, Inc.
Jacksonville, FL
June 22, 2013
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sessions and any other educational
presentation made as part of the 2013
HDSA Convention program is for
informational use only.
HDSA encourages all attendees to consult
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mentioned as part of any presentation.
Presenter Disclosures
David Stamler
The following personal financial relationships with
commercial interests relevant to this presentation
existed during the past 12 months:
Employee of Auspex Pharmaceuticals
Outline
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Who is Auspex?
What is SD-809?
Why is Auspex developing SD-809?
What clinical trials have been conducted with
SD-809?
Key findings from the clinical trials
Ongoing and planned patient studies?
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What is SD-809?
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An investigational, oral medication for treating
chorea associated with HD that has started
Phase 3 patient studies
SD-809 has the same mechanism as
tetrabenazine
SD-809 is broken down in the body more slowly
than tetrabenazine
We are partnering with the Huntington Study
Group to develop SD-809
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Why is Auspex developing SD-809?
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Although effective for many HD patients with
chorea, tetrabenazine has a variety of limitations
We want to develop a treatment for chorea that
offers improvements over tetrabenazine
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Can be dosed once or twice a day
Has smoother blood levels with potential for fewer side
effects
Can be used more easily by you and your doctor
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How is SD-809 Different from Tetrabenazine?
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SD-809 contains deuterium, which is a naturally
occurring, non-toxic form of Hydrogen
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We all have about 1-2 gm of deuterium in our bodies
SD-809 is broken down more slowly in the body than
tetrabenazine, with the following advantages
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Given less often
Need lower doses
Smoother blood levels
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Clinical Trials with SD-809
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We have completed 6 clinical studies in healthy
volunteers
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A total of 130 persons have been exposed to SD-809
No serious side effects have been seen with SD-809
These studies have helped us understand
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How SD-809 is absorbed by the body
How the body breaks down SD-809
How SD-809 interacts with other drugs
We have reviewed the results from these clinical studies with
the FDA and they agreed we are OK to study SD-809 in
chorea associated with HD
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Smoother Blood Levels with SD-809 Dosing
TBZ and SD-809 have the same amount of drug in the body over 24 hours
Plasma Concentration (ng/mL)
45
40
Tetrabenazine
35
Peak/Trough
30
25
SD-­‐809
3.26
TBZ
9.72
SD-809
20
15
10
SD-­‐809 9mg bid
5
TBZ 12.5mg tid
0
0
6
12
18
24
Time (hours)
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Ongoing Clinical Trial with SD-809
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First time Use of SD-809 in HD
Partnering with the Huntington Study Group under the
leadership of Samuel Frank, MD, Principal Investigator,
(Boston University School of Medicine) and Claudia
Testa, MD, PhD, co-Principal Investigator (Virginia
Commonwealth University)
More information at: www.huntington-study-group.org
We are planning an open label extension study
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First-HD Sites
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Summary
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SD-809 is an investigational drug for treating
chorea associated with HD
SD-809 is broken down more slowly in the body
than tetrabenazine, allowing
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Less frequent dosing
Lower doses to be administered
Smoother blood levels to be achieved
These improvements have the potential to make
treatment more convenient and to improve
patient outcomes
The First-HD trial has started
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