O4CP, Vincent Grek
O4CP, Vincent Grek
O4CP, Vincent Grek
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Company Profile<br />
• Only for Children Pharmaceuticals was founded in 2007.<br />
• « Etablissement Pharmaceutique » under the French<br />
law (Q1-2011).<br />
• 10 FTE (5 FTE in CDI, 1 FTE Consultants, 4 FTE Trainees)<br />
• Privately owned SAS backed by ESSEC venture (5%).<br />
• Office in Paris: Institut Villemin, 10 Avenue de Verdun,<br />
Paris 10ème.<br />
• Financed via FP7 EU and French grants so far (21<br />
milllions euros of project).<br />
• Establish in the US<br />
2 Copyright <strong>O4CP</strong> 2011 -<br />
Confidential
A Global Pediatric Speciality<br />
Pharma<br />
3 Copyright <strong>O4CP</strong> 2011 -<br />
Confidential
<strong>O4CP</strong> ACHIEVEMENTS FROM 2007 UP TO 2011<br />
2<br />
Product<br />
in clinical<br />
trial<br />
4<br />
FP7/FUI<br />
Projects<br />
5/9<br />
Orphan<br />
Labels/SA<br />
11<br />
Products<br />
Pipeline<br />
21<br />
Million €<br />
Grants<br />
25<br />
R&D<br />
Partners<br />
Copyright <strong>O4CP</strong> 2011 - Confidential 4
<strong>O4CP</strong> Ecosystem<br />
5 Copyright <strong>O4CP</strong> 2011 -<br />
Confidential
Un environnement réglementaire<br />
positif!<br />
6
Les voies d’enregistrement<br />
Médicaments pédiatriques à base de<br />
molécule tombée dans le domaine publique
Voie d’enregistrement<br />
Molécule hors brevet (off-patent)<br />
Générique<br />
Hybride<br />
+/-Orphelin<br />
+/- PIP<br />
+/-<br />
SA<br />
+/-<br />
SA<br />
Mixte<br />
+/-Orphelin<br />
PIP<br />
PUMA<br />
PUMA<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 8
La source des molécules (API)<br />
Médicaments pédiatriques à base de<br />
molécule tombée dans le domaine publique
Difficulté de source API<br />
• Des sources ne sont plus produites en Europe, par<br />
exemple l’Isoniazide.<br />
• Besoin de très petites quantités entrainant des<br />
difficulté d’assurer la continuité d’approvisionnement<br />
sur la durée, par exemple la bumetanide.<br />
• Besoin de très petites quantités entrainant des refus de<br />
commande, par exemple les antibiotiques.<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 10
Acute Lymphoblastic Leukemia<br />
• LOULLA&PHILLA FP7: HEALTH-2007-4.2-1:<br />
Adapting off-patent medicines to the specific needs<br />
of paediatric populations<br />
• Coordinator: <strong>Vincent</strong> <strong>Grek</strong> (<strong>O4CP</strong>)<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 11
FP7 LOULLA&PHILLA<br />
Creation<br />
<strong>O4CP</strong> EMA<br />
with Orphan<br />
10.000 euros Designation<br />
Idea!<br />
ALL<br />
MTX<br />
6MP<br />
SME<br />
FP7<br />
Application<br />
ESR<br />
15/15<br />
NEGO<br />
NEGO<br />
EMEA Scientifc Advices<br />
Guan<br />
ranty<br />
Patent<br />
on device<br />
Study<br />
LOULLA001<br />
FSI<br />
LSO<br />
2007 2008 2009 2010 2011<br />
2012<br />
3.3 millions 36 months<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 12
Consortium<br />
• Assistance Publique – Hôpitaux de Paris, Leverger<br />
• Assistance Publique – Hôpitaux de Paris, Baruchel & Jacqz-Aigrain<br />
• University of Lyon- Bertrand<br />
• University of Nantes<br />
• University of Lille<br />
• University of Kiel, Schrape<br />
• University of Copenhague, Schiemegelow<br />
• University of Düsseldorf, Breitkreutz<br />
• Laboratoires Philippe Davioud, Davioud<br />
• FEI, Olivier Degrand<br />
• Cardinal Systems (CRO), Antoine Perrier<br />
• <strong>O4CP</strong>, <strong>Vincent</strong> <strong>Grek</strong><br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 13
Acute Lymphoblastic Leukemia<br />
• Most common cancer in infants 1<br />
• Orphan : 4000 new cases per year in Europe 2<br />
• ALL is characterized by anemia, thrombocytopenia and leukocytemia<br />
• Current treatment consists in induction phase followed by maintenance phase<br />
(2 years)<br />
1<br />
Pui et al. (1999)<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 14<br />
² Eurostat, Statistiques de la Population, 2005, page 56 14
Cystostic Preparation<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong><br />
15
The Shaker<br />
SAFE<br />
ACCURATE<br />
Liquid vehicle<br />
6 ml<br />
Prototype 2007 Patented Device 2009<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong><br />
16
Pharmaceutical development<br />
Stab Done<br />
Stab Done<br />
Stab Done<br />
CTA Approved<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong><br />
17<br />
17
Loulla & Philla<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 18
Non clinical development<br />
Animal model<br />
for mucosal<br />
toxicity: no<br />
toxicity<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong><br />
19
Clinical development<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong><br />
20
Epilepsie du nouveau-né
Neonatal seizure<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 22
1970: Loop diuretic<br />
Bumetanide<br />
Furosemide<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 23
1996: Bumetanide in infants<br />
1987: NKC Receptors<br />
1978: Ototoxicity<br />
1970: Bumetanide<br />
1970 1980 1990 2000 2010 2020<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 24
2005: Seizure mechanism<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 25
2008: NKCC1-Bumetanide<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 26
<strong>O4CP</strong> Bumetanide for neonates<br />
benzyl alcohol free<br />
22/05/2012 <strong>O4CP</strong> Confidential - Copyright 2011
Consortium<br />
• University College London, Ronit Pressler, Helen Cross,<br />
• University College London, Neil Marlow, Janet Rennie<br />
• University College Cork, Geraldine Boylan<br />
• INSERM U663, University Rene Descartes, Catherine Chiron<br />
• Assistance Publique – Hopitaux de Paris, Gerard Pons<br />
• INMED, INSERM U29, Yehezkel Ben-Ari<br />
• Helsinki University Central Hospital, Sampsa Vanhatalo<br />
• Uppsala University Hospital, Linda De Vries<br />
• University Medical Centre Utrecht, Lena Hellstrom-Westas<br />
• Karolinska University Hospital Huddinge, Mats Blennow<br />
• Duke Clinical Research Institute, Barry Mangum<br />
• University of Leeds, Malcolm Levene<br />
• <strong>O4CP</strong>, <strong>Vincent</strong> <strong>Grek</strong><br />
• ClinInfo S.A, Patrick Chevarier<br />
• GABO:mi, Judith Millecker<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 28
International,multi-centre<br />
SPONSOR<br />
CO-SPONSOR<br />
CHIEF INVESTIGATOR<br />
Dr Ronit Pressler<br />
CORK<br />
HELSINKI<br />
LEEDS<br />
LONDON<br />
PARIS<br />
STOCKHOLM<br />
UPPSALA<br />
UTRECHT<br />
IRE<br />
FIN<br />
UK<br />
UK<br />
FRA<br />
SWE<br />
SWE<br />
NL<br />
PI<br />
PI<br />
PI<br />
PI<br />
PI<br />
PI<br />
PI<br />
PI<br />
Dr<br />
Dr<br />
Dr<br />
Prof<br />
Prof<br />
Prof<br />
Dr<br />
Prof<br />
Brendan<br />
Marjo<br />
Malcolm<br />
Neil<br />
Alexandre<br />
Mats<br />
Lena<br />
Linda<br />
Murphy<br />
Metsäranta<br />
Levene<br />
Marlow<br />
Lapillonne<br />
Blenow<br />
Hellström-<br />
Westas<br />
DeVries<br />
TRIAL TEAMS<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 29
Clinical development<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 30
Amélioration des PUMAs<br />
# Problème Amélioration<br />
1 Différence d’appréciation entre la CE et<br />
EMA des projets FP7<br />
2 Différence d’appréciation au sein de<br />
l’EMA (CHMP, Avis Scientifique, )<br />
3 Le PUMA est un incitatif « faible »<br />
notamment au niveau du prix<br />
Evaluation par le PedCO des<br />
propositions FP7<br />
• Meilleure communication<br />
• Guidelines (Paraben)<br />
• Au niveau national, faire des<br />
recommendations sur l’évaluation<br />
des prix des médicaments<br />
pediatriques (voir accord LEEM et<br />
Agence Française), organisation<br />
spécifique pour l’évaluation des<br />
médicaments pédiatriques<br />
• Au niveau Européen, donner des<br />
recommendations au niveau des<br />
prix (orphan)<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 31
Merci!<br />
<strong>O4CP</strong> 2011-Dr <strong>Vincent</strong> <strong>Grek</strong> 32
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