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Combating diseases with small molecule
compounds
C3BIO GmbH
C3
Bio
Confidential
20.07.2015
Company concept: rational drug design

Patents and exclusive world licenses on
families of compounds (purines and
pyrazolopyrimidines)

Extensive network of cooperation partners

Progress made in 3 indication areas:
Rheumatoid arthritis, restenosis and cancer
C3
Bio
Confidential
20.07.2015
Pyrazolopyrimidines
HN
H2 N
H
N
N
N
N
H
Roscovitine
N
1407
C3
Bio
Confidential
20.07.2015
Cancer

Recognized hallmarks of cancers
 Uncontrolled proliferation
 Evasion of apoptosis
 Sustained angiogenesis
 Genomic instability

Common target: cyclin-dependent kinases
(CDKs)
C3
Bio
Confidential
20.07.2015
Cancer

Identified compounds have increased:
 Activity in biochemical and cellular assays
 Potency in vivo
 Metabolic stability

Tumour cell migration as well as endothelial
migration are specifically being investigated to
prevent cancer metastasis-a number of
compounds show promising results
C3
Bio
Confidential
20.07.2015
Restenosis

Focus is on identification of new drug
candidates interfering with vascular smooth
muscle cells (VSMC) proliferation

Identified compounds may be useful for antirestenotic therapy-some of the work is
published
C3
Bio
Confidential
20.07.2015
Rheumatoid Arthritis

Concentrate on the 30-40% nonresponding RA patients

New targets-neutrophils and synovial
fibroblasts

Patients at early stage of disease
progression
C3
Bio
Confidential
20.07.2015
Rheumatoid Arthritis: LGR1407 induces
neutrophil apoptosis
120
Apoptotic cells (%)
100
80
LGR1407
60
R-roscovitine
40
20
0
0
0.01
0.1
1
10
50
100
Concentration (M)
C3
Bio
Confidential
20.07.2015
Rheumatoid Arthritis: LGR1407 inhibits
TNF-induced secretion of IL-8 in
neutrophils
C3
Bio
Confidential
20.07.2015
Proliferation (% control)
Rheumatoid Arthritis: LGR1407 inhibits
proliferation of synovial fibroblasts from
RA patients
120
100
80
60
40
20
0
control
0.1uM
0.5uM
1.0uM
5.0uM
10uM
LGR 1407 concentration
C3
Bio
Confidential
20.07.2015
Rheumatoid Arthritis

Molecular target had been identified

Compounds are orally available

Toxicology and pharmacokinetic studies
indicate a useful therapeutic window

in vivo studies successfully carried out
C3
Bio
20.07.2015
Combating diseases with small molecule compounds
For more information contact
Dr. Paul Pechan, CEO
Email:[email protected]
Tel: 49 89 67847951
C3
Bio
Confidential
20.07.2015