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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."

Coordinator

LIPOPHARMA THERAPEUTICS SL

Address

Ctra Valldemossa Km 7,4 Parcbit, Edificio 17, Incu Empresas, 2a Planta, Modulo C - 8
07121 Palma De Mallorca

Spain

Activity type

Private for-profit entities (excluding Higher or Secondary Education Establishments)

EU Contribution

€ 2 582 500

Participants (11)

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PRAXIS PHARMACEUTICAL SA

Spain

EU Contribution

€ 1 103 500

SPECIALIZED MEDICAL SERVICES-ONCOLOGY BV

Netherlands

EU Contribution

€ 1 212 500

UNIVERSITY OF NEWCASTLE UPON TYNE

United Kingdom

EU Contribution

€ 238 750

FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA

Italy

EU Contribution

€ 117 000

RESEARCH FUND OF THE HADASSAH MEDICAL ORGANIZATION (R.A)

Israel

EU Contribution

€ 222 500

UNIVERSITAT DE LES ILLES BALEARS

Spain

EU Contribution

€ 195 500

UNIVERSITA DEGLI STUDI DI SALERNO

Italy

EU Contribution

€ 101 875

LANA MANAGEMENT AND BUSINESS RESEARCH INTERNATIONAL LLC

United States

EU Contribution

€ 50 000

THE ROYAL MARSDEN NATIONAL HEALTH SERVICE TRUST

United Kingdom

EU Contribution

€ 117 000

INSTITUT GUSTAVE ROUSSY

France

EU Contribution

€ 117 000

LIPODOM KUTATO FEJLESZTO ES TANACSADO KORLATOLT FELELOSSEGU TARSASAG

Hungary

EU Contribution

€ 97 000

Project information

Grant agreement ID: 755179

Status

Ongoing project

  • Start date

    1 December 2017

  • End date

    30 November 2020

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 6 155 125

  • EU contribution

    € 6 155 125

Coordinated by:

LIPOPHARMA THERAPEUTICS SL

Spain