Investor Presentation. Winter 2010

Transcription

1 Investor Presentation Winter 2010

2 In a few sentences... Glycoregimmune is developing sulfatide, a first-in-class therapeutic, for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig s Disease Sulfatide, a self-glycolipid that broadly activates type II NKT cells, can ameliorate disease in multiple models of autoimmunity ALS is a progressive, fatal, neurodegenerative disease caused by the degeneration and eventual death of motor neurons, with high unmet need for treatment options Market size: $150M in 2006, combined with limited competition, rapid uptake after launch and orphan drug status make ALS a compelling indication

3 Management Team Marc Hertz, PhD CEO, co-founder Biotech executive with 12 years experience with immunotherapeutics from bench to Phase 3. Operations, Investor Relations, BD, fundraising, early clinical Pharmexa Inc (CEO); Pharmexa A/S (SVP); MultimericBio (startup CEO) Albert Agro, PhD CMO, co-founder Biotech/pharma/consulting executive for 15 years Global clinical development (phase I-IV), Global Medical Marketing, Healthcare PR TransTech Pharma (SVP); NATIONAL Public Relations, Axon Clinical Research; Boehringer Ingleheim GmBH, Bayer Inc. Vipin Kumar, PhD Director, scientific co-founder Inventor of technology and world recognized leader of type II NK T cell biology PhD Biochemistry Indian Institute of Science; further training at Harvard and California Institute of Technology Member, Torrey Pines Institute for Molecular Studies, La Jolla Institute for Allergy & Immunology, UCLA

4 The Opportunity in ALS The ALS Market ALS market $150MM 2006 (with minimal treatment options) ALS occurs throughout the world with no racial, ethnic, or socioeconomic boundaries Approx. 5,600per year; 30,000 cases in the U.S. Average life span is 2-5yrs from time of diagnosis Riluzole the only approved (`95) drug for ALS The benefits are modest - prolonging survival in ALS patients for several months, but has no significant effect on measures of function ALS community Captive patient audience and KOLs trying to advance therapies Orphan Drug Status indication

5 The Problem Complex disease Multiple organ/body systems Lethal (median survival 3.5 yrs) Pathology not fully understood Difficult to diagnose Differential diagnosis No biomarkers Poor correlation between efficacy in animal models and disease SOD1 mutation not a positively predictive model Limited therapeutic options One approved agent with minimal effectiveness

6 The Solution Sulfatide Novel therapeutic approach Potential disease modification Predicted to increase survival and QofL Exclusive worldwide license for all fields of use License from Torrey Pines Institute for Molecular Studies Patents pending worldwide

7 Type II NKT cells NKT cells posses features of both NK and T cells, and play an important role in autoimmunity, anti-tumor immunity and host defenses to foreign pathogens Dominant NKT cell population in humans Unique specificity and biology compared to inkt cells Type II NKT cells are regulatory cells Tolerize Dendritic cells, microglia, T cells and inkt cells Regulate the expression of key cytokines during disease Type II NKT cells can modulate the phenotype of inflammatory cells from pro-inflammatory/ cytotoxic to anti-inflammatory/protective

8 Sulfatide ameliorates disease in multiple models of autoimmunity Efficacious in... Multiple Sclerosis Asthma Type I Diabetes Autoimmune hepatitis Systemic Lupus Erythematosus

9 ALS pathology and rational for type II NKT ALS Pathology Neurodegenerative disease of the voluntary motor system Multifaceted neuroinflammation (astrocytes, microglia, T cells and other inflammatory cells) Novel therapies must target several events/cell types to be effective Type II NKT cells Sulfatide-activation leads to novel immunoregulatory pathway Tolerization of dendritic cells, microglia, type I NKT cells and T cells Can modulate the phenotype of inflammatory cells from proinflammatory/cytotoxic to anti-inflammatory/protective

10 The Competition - ALS DRUG COMPANY STATUS MOA / Comments Rilutek (Riluzole) Rocephin (Ceftriaxone) ALS-02 (Creatine) Radicut (Edaravone / MCI-186) E0302 (Mecobalamin) Sanofi Aventis M Approved in 1995 Hoffman-La Roche III Marketed antibiotic; increase astrocyte glutamate transport Avicena Group III neuroprotective Mitsubishi Tanabe Pharma Corp Eisai III II/III Free radical scavenger Vitamin B-12 coenzyme

11 Sulfatide Development Plan-ALS Yr 1 Yr 2 Yr 3 Yr 4 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q $1.75MM IND $250k GMP manufacturing $1.5m IND-enabling studies -dose ranging tox - single dose tox -7-day tox -28-day tox $1. 5MM $1.5m Phase 1a SAD $1.75MM $1.75m Phase 1b MAD $1.5MM $1.5M 6-Month Tox Program $4MM $4M Phase 2a POC Study $2.5 M $4.5 M $2.5 M $5 M The ALS development plan calls for approx. $14.5 M( including overhead) over 4 yrs to advance a Sulfatide through a Phase 2a trial POC in ALS

12 Sulfatide Development $14.5MM to develop Sulfatide for ALS thru POC prove safety & efficacy Orphan Drug Status Limited Competition Proactive community Follow-on indications compelling markets Ischemic Reperfusion Injury* Multiple Sclerosis* Alzheimer s Disease * Pre-clinical evaluation in relevant disease models performed

13 Contact Albert Agro cmo Vipin Kumar co-founder Marc Hertz ceo