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A Novel Technology Platform for
Quantification of Peptide Biomarkers in
Complex Biological Samples
Marko Poglitsch
APEIRON Biologics AG
Non-confidential presentation, June 2012
Page 1
Apeiron Biologics: Snapshot
§  Focus
-  Biological / immunological treatments
of cancer and related conditions
§  Company
-  Operational since 2006 in Austria
-  25 employees
-  Financed by several private investors
and one Big Pharma partner
§  Pipeline
-  5 clinical and 2 preclinical projects
-  Technology platform
Non-confidential presentation, June 2012
Page 2
§  Network
Apeiron-Biologics: Snapshot
Discovery
Pre-clinical
Phase I
Phase II
Phase III
Indication(s)
Partner
APN01 (rhACE2)
Acute Respiratory
Distress Syndrome
GSK*
APN201 (rhSOD)
Cancer-related
skin conditions
Polymun**
Neuroblastoma
Merck, NCI (COG)**
Melanoma
NCI**
APN311 (ch14.18)
Neuroblastoma
SIOPEN / CCRI**
APN401 (Cbl-b/siRNA)
Various cancers
-
APN411 (Cbl-b/LMW)
Various cancers
-
APN301 (hu14.18-IL2)
RAS-Fingerprint™
High sensitivity peptide quantification (LC-MS/MS)
spin-off
a"# quant#
diagnos-cs#
Non-confidential presentation, June 2012
Page 3
Quantification of peptides in
complex biological samples
Peptide Quantification in Blood – The Challenge
Blood
Proteins and peptides (60-80 mg/ml)
Salts, acids, bases
Lipids
Sugars
Metabolites
Peptide hormones (<100 pg/ml)
Plasma
Erythrocytes
Leukocytes
Platelets
Blood
Cells
Non-confidential presentation, June 2012
Page 4
Plasma proteins
:
Peptide hormones
7.000.000.000
:
1
APEIRON Biologics, Vienna, Aus
Technology Overview – The Solution
Results a
nsin-System is a peptide hormone system which is critically involved in the regulation of blood pressure via the
Angiotensin II (Ang 1-8) and Angiotensin 1-7. The Angiotensins are liberated from the pre-hormone
y proteolytic cleavages. Multiple Angiotensin metabolites are reportedPeptide
to exert physiologic
activity via binding to
Signal Intensity
n various cell types and tissues. Therefore, the enzymes involved in Angiotensin metabolism represent attractive
In-House
procedure
(pat.
velopment especially in the field of antihypertensive therapy. Caused by
the sequence
similarity
ofpending)
the effector
Standard
very low physiologic concentrations (<10 pg/ml), antibody based quantification methods have been struggling
ms regarding specificity and sensitivity, leading to surprisingly variable values for physiologic Angiotensin peptide
ong the literature. We have developed a highly sensitive and standardized procedure for simultaneous absolute
è
p to 10 Angiotensin peptides in one single sample. RAS-Fingerprinting is not only able to provide reliable effector
ions, but furthermore delivers a comprehensive picture of the RAS allowing conclusions about enzyme activities
iotensin peptide metabolism.
System dynam
Sensitivity
ased RAS-Fingerprinting
1-5
Sample Stabilization
C
3-8
Pharmacodyn
Matrix
Depletion
3-7
Peptide
Enrichment
2-8
2-7
LC-MS/MS-Analysis
Non-confidential presentation, June 2012
Page 5
Physiologic Plasma Conc. N=21 [pg/ml]
1-7
1-9
1-8
è Selectivity
1-10
Example: The Renin-Angiotensin-System (RAS)
The Renin-Angiotensin-System (RAS)
§ 
§ 
§ 
Peptide hormone system
Fluid balance / Hypertension
Imbalance: Cardiovascular diseases
(CKD, heart failure, stroke,…)
Tool: The RAS-FingerprintTM
è RAS-Blockers
Problems
§ 
§ 
§ 
Drug side effects
Lack of response / Drug tolerance
Duration to treatment efficacy
è  Patient specific factors
Hypothesis
§ 
§ 
Characterize biochemical hardware of individual patients
Apply personalized treatment schemes for cardiovascular diseases
è  Decrease the number of cardiovascular events
Non-confidential presentation, June 2012
Page 6
Pharmacodynamic Analysis of RAS-Blockers
ACE-Inhibitor
Non-confidential presentation, June 2012
Page 7
Renin-Inhibitor
è  Drug development
è  Companion diagnostics
ARB
Individuality of RAS-Fingerprints
Donor A
Donor B
Donor C
è Personalized treatment schemes targeting the RAS
Non-confidential presentation, June 2012
è Biomarker discovery
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Attoquant Diagnostics: Activities
Collaborations
Fee-for-service
RAS-FingerprintTM
§ 
§ 
§ 
Clinical science
Basic science
Drug development
LC-MS/MS Assay Development
§ 
§ 
Neuropeptides, Natriuretic peptides, …
Customized analytic solutions
Non-confidential presentation, June 2012
Page 9
Partnering options
§ 
§ 
§ 
Non-exclusive licenses
Co-developments
Biomarker discovery (RAS)
Acknowledgements
Prof. Dr. Stephan Krähenbühl
Dr. Manuel Haschke
Department of Pharmaceutical Sciences
Clinical Pharmacology and Toxicology
a"# quant#
Non-confidential presentation,
diagnos-cs# June 2012
Page 10
Contact Details
§  Contact us:
-  [email protected] (Designated CEO, Attoquant)
-  [email protected] (CEO, Apeiron)
-  [email protected] (COO, Apeiron)
-  [email protected] (CFO, Apeiron)
§  Meet us at:
-  BCVS 2012, July 23-26, New Orleans
-  HBPR 2012, September 19-22, Washington
-  AHA Scientific Sessions 2012, November 3-7, Los Angeles
§  Visit us:
-  online:
www.apeiron-biologics.com
-  at our premises:
APEIRON Biologics AG
Campus-Vienna-Biocenter 5
1030 Vienna, Austria
Non-confidential presentation, June 2012
Page 11