MLT-based Minerval: a breakthrough for the treatment of glioma

Transcription

1 ML-based Minerval: a breakthrough for the treatment of glioma Vicenç ur. CEO, Co-Founder Lp-pmglci

2 Lipopharma [2007] is an pioneering science-driven biopharmaceutical company based in Palma de Mallorca (Spain) that focuses on the discovery, rational design and development of a new generation medicines on the basis of a novel therapeutic approach: the Membrane Lipid herapy (ML) 2

3 Novel echnology: Membrane-Lipid herapy (ML) Normal (non-tumor) cell membrane OU IN OU IN OU IN OU IN Cancer cell membrane BEFOE EAMEN AFE EAMEN BEFOE EAMEN AFE EAMEN Escribá (2006) rends in Molecular Medicine 12:34-43 ML drugs are designed to influence and regulate lipid organization in cell membranes based on structure function principles, inducing a concomitant modulation of membrane protein localization and activity, which finally induces changes in cell signaling and gene expression. In the case of Minerval, it induces a rise in the levels of SM (yellow) and DAG (red) and decreases of PE (green). hese changes only affect cancer but not normal cells, and cause an specific inactivation (normalization) of signalling (over-activated in most cancer cells) and related signalling pathways (e.g., MAP) in many types of cancer (glioma, lung cancer, leukaemia) 3

4 Effect of SMS1 genetic alterations on glioma patients survival (Differential SGMS1 Gene Expression & Copy Number Analysis for SGMS1) SGSM1 up-regulated (n = 1) SGMS1 Intermediate (n = 258) All Glioma average (n=454) SGSM1 down-regulated (n = 84) SGSM1 Amplified (n = 73) All Glioma average (n=454) SGSM1 Deleted (n = 278) National Cancer Institute EMBAND < Accessed October

5 Minerval, a new class of lipid regulator multi-target anticancer drug for tumors with sphingomyelin metabolism alterations Specific activator of sphingomyelin synthase (SMS) [1] Selective regulation of lipid composition in cancer cell membranes: SM; DAG; PE; PC [2] PC/Cyclin CD egulation [4] traslocation MAP PI3/Akt pathway normalization [3] Cell cycle arrest [ DHF] & differentiation [4] selective Autophagy [5] 5

6 Anti-cancer effect of Minerval in animal models of cancer Effect of Minerval in animal models of human brain tumours (GLIOMA) compared with temozolomide and control group 6

7 Next steps: PI/II Clinical Study with Minerval A phase I/IIa open-label dose escalation study of Minerval in subjects with advanced solid tumors including malignant glioma. op leading European OL and investigational sites involved: Johann de Bono (oyal Marsden Hospital, London), oger Stupp (University Hospital, Zurich), Jordi odon (Vall d'hebron Institute of Oncology, Barcelona), Herbie Newell and uth Plummer (Northern Institute for Cancer esearch, Newcastle) Part A. Dose escalating study. Up to 30 patients. 3 week treatments. Glioma and other solid tumors (lung, pancreas,) Part B. Exploratory study. Up to 20 patients in two groups. 3 week treatments. 1st group with glioma patients. 2nd group with biopsiable solid-tumors patients for biomarker evaluation. Biomarkers: SM, DHF and GFAP (glioma only) + Imaging (FL/PE, MS), mina,... 7

8 Lipopharma & Minerval today: Clinical development (PI studies) started in Q in the U PI/II trials in glioma & solid tumors scheduled for Q Orphan Drug status granted in EU (glioma) 6.5+ M Euro already raised in equity / grants ( ) Sound scientific, development & management team on place (15 direct, 40+ indirect & specialized consultants) World class strategic partners & collaborators network Looking for additional collaborations/investment for PII studies in glioma & other solid tumors (up to 10M Eur.) 8

9 hank you! Lipopharma Ctra. Valldemossa, m. 7,4. ParcBI. Edif. Disset. 2º C-8. E07121 Palma de Mallorca. Spain