Tr a n s l a t i n g p r e - c l i n i c a l f i n d i n g s
i n t o s u c c e s s f u l c l i n i c a l t r i a l s
Wahid Awad, Ph.D, Chief Business Officer
ARO 2021
CILcare is the leading external innovation R&D partner in
hearing disorders
2
Paris,
France
Montpellier, France
Headquarter
PK/PD
Boston, MA, USA
GLP facilities
Copenhagen,
Denmark
Pharmaceutical
Nutraceutical
Veterinary
Medical devices
+120Studies
+60Sponsors
2Labs
Established in France, US & Denmark, CILcare operates worldwide since its inception in 2014
A CATALYST of the hearing disorders area!
3
• Strongly involved in patient
associations
• Collaborating with a global
network of preclinical & clinical
CRO partners & consultants
• Frequently publishing in
international peer review journals
SPONSORS
• Working with internationally
recognized KOL from industry &
academia
• Continuously building strategic
industrial partnerships
• Speaking in international
scientific conferences
ARO
Patients
Think clinical development
Portfolio Screening
Design TPP
+
R&D Plans
Pre-formulation
+
PK
Preclinical
Efficacy
Ototoxicitytesting
&
GLP Tox
IND Phase I Phase II
I n d i c a t i o n ● r o u t e o f a d m i n i s t r a t i o n ● e n d p o i n t s
4
In preclinical studies, select the best:
MODEL DRUG REGIMEN READ-OUTS
Clinical protocol
Models for auditory research & development
5
COCHLEAR
EXPLANTS
ZEBRAFISHOTIC
CELL LINE
SCREENING PHASE
PK TOLERANCE GLP-OTOTOXICITYEFFICACY
RODENTS & LARGE SPECIES
Mice Rats Guinea pigs Chinchillas Mini pigs Sheep
Select your model to target your clinical indication
6
• Age-
related
hearing
loss
• Salicylate-
induced
tinnitus
• Noise-
induced
tinnitus
• Noise-
induced
hearing
loss
• Otitis
media
• Drug-
induced
hearing
loss
• Gene
therapy
PRESBYCUSIS TINNITUS HEARING LOSS OTOTOXICITY GENETIC DEAFNESS OTITIS MEDIA
MICESAMP8
RATSLong Evans
RATSWistar
GUINEA PIGSHartley
RATSWistar
MICECBA
RODENTS
MINI PIGS
SHEEP
CHINCHILLAS
Bastard F1N1
Design efficacy study mimicking clinical situation
Formulation
Administration regimen
Read-outs
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Differentiate POC studies from efficacy studies in terms of:
Preventive versus
curative / regenerative treatment
For slow / Sustained release
Translational read-outs
Select translational and complementary measures
MEMRI
BEHAVIORAL MEASURES
GPIAS(Gap-Prepulse Inhibition of the
Acoustic Startle reflex)
OTOSCOPY
COCHLEOGRAM
HISTOLOGY
Non invasive & recognized read-outs Complementary measurements
DPOAE(Distorsion Product
OtoAcoustic Emissions)
RIBBON
SYNAPSES COUNT
GROSS
ASSESSMENT
OF THE MIDDLE
EAR
CLINICAL SIGNS
GENE & PROTEIN
ARRAYSBiochemical biomarkers
ABR(Auditory Brainstem
Responses)
WAVE I
ANALYSIS
TYMPANOMETRY
SEM(Scanning Electron
Microscopy)
8
Expert
team
Reproducible
data over
time
Working on robust and reproducible models
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Experiments
conducted in a
high-quality
acoustic
environment
High
throughput
measures
Cisplatin 13mg/kg L+R
Frequency (Hz)
0 5000 10000 15000 20000 25000 30000 35000
AB
R t
hre
sh
old
s (
dB
SP
L)
Me
an
± S
EM
10
20
30
40
50
60
70
80 Baseline (n=50)
T+3days (n=50)
l l ll l l
www.cilcare.com l [email protected]
Thank you for your attention !Feel free to contact me:
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