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A Clinical Phase IIB trial with 2OHOA in patients with newly-diagnosed malignant glioma.

Objective

"Glioma is a rare brain cancer with one of the highest mortality rates. It is considered an orphan disease due to its low prevalence (less than 0.5 cases per 10,000 inhabitants in the EU) and the lack of plausible therapies. Based on the discovery of the lipid proliferation switch (high membrane PE-to-SM molar ratio that enables recruitment of cell growth transducers to the membrane), the SME Lipopharma (leading this application) defined a novel anticancer drug target, the tumor repressor protein sphingomyelin synthase 1 (SMS1). An innovative SMS1 activator, 2OHOA, was designed and showed safety and efficacy in preclinical GLP and non-GLP studies. A first-in-man clinical trial I/IIa (ClinicalTrials.gov identifier #NCT01792310) further demonstrated its safety and efficacy in humans. The European Medicines Agency (EMA) designated 2OHOA orphan drug for the treatment of glioma and approximately half of the patients with glioma submitted to >2 months of treatment showed positive response. The present project aims to perform a clinical phase IIB study to demonstrate 2OHOA’s efficacy against glioblastoma multiforme, the most aggressive form of glioma. In this context, a written formal report from the EMA after scientific advice and protocol assistance (SA/PA-EMA/CHMP/SAWP773534/2014) indicates that 2OHOA would obtain Conditional Marketing Authorisation if this phase-IIB study further demonstrates statistically significant efficacy. In addition this project will further investigate 2OHOA’s safety, mechanism of action and biomarkers for glioblastoma diagnosis, prognosis and response to 2OHOA treatment. These studies will let us (i) know the molecular basis underlying the response to 2OHOA treatment, (ii) define new biomarkers, (iii) design more efficacious personalized treatments and (iv) investigate therapeutic alternatives in patients who do not respond to treatment."

Call for proposal

H2020-SC1-2016-2017

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Sub call

H2020-SC1-2017-Two-Stage-RTD

Coordinator

LAMINAR PHARMACEUTICALS SA
Net EU contribution
€ 2 985 285,09
Address
Ctra Valledemossa Km.7,4 C/ Issac Newton, S/n Block A, Planta 1, Puerta 3, Parc Bit Edificio Norte
07121 Palma De Mallorca
Spain

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Region
Este Illes Balears Mallorca
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00

Participants (12)

PRAXIS PHARMACEUTICAL SA

Participation ended

Spain
Net EU contribution
€ 618 655,78
Address
Hermanos Lumiere 5
01510 Minano Mayor

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Region
Noreste País Vasco Araba/Álava
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

BIOKERALTY RESEARCH INSTITUTE AIE

Participation ended

Spain
Net EU contribution
€ 82 059,13
Address
Entidad Arcaute 005 Arcaute
01192 Arcaute Alava

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Region
Noreste País Vasco Araba/Álava
Activity type
Other
Non-EU contribution
€ 0,00
SPECIALIZED MEDICAL SERVICES-ONCOLOGY BV

Participation ended

Netherlands
Net EU contribution
€ 1 212 500,00
Address
Walaardt Sacrestraat 401
1117 BM Schiphol

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
West-Nederland Noord-Holland Groot-Amsterdam
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
Net EU contribution
€ 238 750,00
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne

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Region
North East (England) Northumberland and Tyne and Wear Tyneside
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
Italy
Net EU contribution
€ 117 000,00
Address
Via Giovanni Celoria 11
20133 Milano

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Region
Nord-Ovest Lombardia Milano
Activity type
Research Organisations
Non-EU contribution
€ 0,00
RESEARCH FUND OF THE HADASSAH MEDICAL ORGANIZATION (R.A)
Israel
Net EU contribution
€ 222 500,00
Address
Kiryat Hadassah Ein Karem
91120 Jerusalem

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Activity type
Research Organisations
Non-EU contribution
€ 0,00
UNIVERSITAT DE LES ILLES BALEARS
Spain
Net EU contribution
€ 195 500,00
Address
Carretera De Valldemossa Km 7.5
07122 Palma De Mallorca

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Region
Este Illes Balears Mallorca
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSITA DEGLI STUDI DI SALERNO
Italy
Net EU contribution
€ 101 875,00
Address
Via Giovanni Paolo Ii 132
84084 Fisciano Sa

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Region
Sud Campania Salerno
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
LANA MANAGEMENT AND BUSINESS RESEARCH INTERNATIONAL LLC
United States
Net EU contribution
€ 50 000,00
Address
360 Massachusetts Avenue Suite 203
01720 Acton, Massachusetts

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
THE ROYAL MARSDEN NATIONAL HEALTH SERVICE TRUST
United Kingdom
Net EU contribution
€ 117 000,00
Non-EU contribution
€ 0,00
INSTITUT GUSTAVE ROUSSY
France
Net EU contribution
€ 117 000,00
Address
Rue Camille Desmoulins 39
94805 Villejuif

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Region
Ile-de-France Ile-de-France Val-de-Marne
Activity type
Research Organisations
Non-EU contribution
€ 0,00
LIPODOM KUTATO FEJLESZTO ES TANACSADO KORLATOLT FELELOSSEGU TARSASAG
Hungary
Net EU contribution
€ 97 000,00
Address
35-37 Dorottya Utca
6726 Szeged

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Alföld és Észak Dél-Alföld Csongrád
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00