EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles

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1 EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles

2 BrainDTech Team Dr. Pietro Conti, MD, MBA President Medicine & MBA Founder/CEO several startups Director Health Int l Board Member Dr. Fabio Bianco, PhD Chief Executive Officer Medical Biotech, Pharmacology +10yr in Biotech/Pharma Board Member Dr. Ennio Ongini, PhD Chief Scientific Officer Pharmacology 20yrs R&D Director Schering Plough 15 yrs VP Research Nicox SA +200 peer reviewed papers Dr. Noemi Tonna, PhD Lab Manager Medical Biotech, Pharmacology Lab Manager@NeuroZone +10yr in drug discovery Dr. Carlo Frati, MBA Board Member Economics - Bocconi Univ; MBA, INSEAD McKinsey & Co. ( ): Partner Capital Shuttle S.p.A. (2016): CEO Banca Consulia (2017-present): CFO Dr. Laura Ferro, MD, MBA Board Member Medicine & Psychiatry Bocconi Univ., Milano Top 10 innovative women managers in Italy Founder /CEO several biotechs (including Gentium, IPO at Nasdaq Dr. Elisabetta Borello, PhD Board Member Economics-Bocconi Univ., Milano +30 yrs in Healthcare Information Management Systems Founder /Board member many biotechs

3 The issue (need) Currently, diagnosis of brain diseases is carried out observing clinical symptoms, which occur when most of the neurodegeneration has already occured years Brain INFLAMMATION Brain DEGENERATION CLINICAL SYMPTOMS

4 BrainDTech proposal BrainDTech represents a disruptive approach to brain disease diagnosis because it detecs a signal (MicroRNA in microvesicles) released by microglia before clinical manifestation. Brain INFLAMMATION MICROVESICLES FROM MICROGLIA Bianco et al EMBO J 2009 Neuroinflammation activates MICROGLIA Activated microglia produce MICROVESICLES Microvesicles contain microrna Microvesicles can be found in LIQUID BIOPSY

5 BrainDTech added value MICROVESICLES can be isolated from liquid biopsies Clinical data show that there are more Microvesicles in the Cerebrospinal Fluid of Alzheimer s Disease patients with respect to pre-alzheimer, or Mild Cognitive Impaired patients (Agosta et al ann Neurol 2014) mirnas can be analyzed in MICROVESICLES We have observed that in different in vitro models, mirna from microglial microvesicles are differently up (green) or down (orange) regulated, creating pathology-specific patterns. mirna patterns are SPECIFIC for each PATHOLOGY We have observed that in each pattern there are specific mirna which are exclusively up or down regulated in a pathological model, thus creating pathology specific patterns, which we have patented.

6 BrainDTech IP position PATENTS 2015/11 - Italian patent submission (AD, PD, ischemia) 2016/07 - PCT patent extension 2016/11 - Claim extension (+20 pathologies) PATENT STRATEGY PATHOLOGY-SPECIFIC PATTERNS DEVELOPMENT NOVEL THERAPEUTIC TARGETS IDENTIFICATION EARLY DIAGNOSIS COMPANION DIAGNOSTICS Pathology specific patterns to be outlicenced for early diagnosis or companion diagnostics in drug development Since mirna control several biological processes, we plan to study the mirna patterns obtained and identifty novel therapeutical targets

7 MARKET APPLICATIONS The market: Diagnostics in brain diseases BIOMARKER MARKET FIGURES GLOBAL BIOMARKERS MARKET $24 Billion in 2015 $45,55 Billion by 2020 GLOBAL mirna MARKET $1 Billion by 2019 CURRENT mirna FOCUS AREAS CANCER CARDIO The market for mirna diagnostics is at a starting point, currently only focused on cancer and cardiovascular diseases, but expected to grow exponentially in the upcoming years. BEFORE CLINICAL MANIFESTATION CURRENT STATE OF ART AFTER CLINICAL MANIFESTATION Lipid test for AD in blood Nature Methods 2014 Acquisition of AD test from Memory Dx: Enables differentiationad vs dementia $18 million in equity shares Development milestone after phase 2 9% royalties on sales Acquisition of Avid Radiopharma: imaging agent for Abeta plaque detection $300 million cash Up to $500 million in milestones Recent market highlights in the field (Alzheimer as an example), refer to diagnostics that help to characterize the patient once the symptoms appear. BrainDTech fulfills an unmet need of pre-symptomatic diagnosis, as well as specific companion diagnostic in drug development

8 The market: competition CANCER Few innovative players in cancer diagnosis. Brain solutions are currently focusing on circulating mirna in plasma for detection of AD BRAIN The main issue with circulating mirna based approaches is lack of specificity, given mirna can be produced by many different cell types BrainDTech approach is revolutionary because we isolate intact microvesicle from a specific cell type (microglia) and then analyze the content only of these organelles, thus significantly increasing specificity. BrainDTech approach is potentially breakthrough because recent scientific evidence (Plog et al J.Neurosci 2015) suggest that these vesicles could be present also in easily accessible biopsies such as plasma (this hypothesis is currently being validated in our lab).

9 BrainDTech Development plan Clinical PoC PoC AD vs MS AD clinical study Cerebrospinal Fluid Microvesicle identification Microvesicle isolation mirna analysis Blood Revenue stream 1 Outlicencing of non-core know how (microvesicle handling) 2 Pathology specific licencing 3 Access to DATABASE

10 BrainDTech info E. Ongini F. Bianco N. Tonna BrainDTech srl OpenZone Via Ariosto Bresso MI Italy Tel: Brain DTech srl Registration date: 21/7/2016 VAT number: Share capital: 576 k info@braindtech.com