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

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Elvin Lawson
6 years ago
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1 EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles

(President and CSO) CEO of BrainDTech Director of Sanipedia Business Nursery >10 yr in Biotech/Pharma with

Pietro Conti, MD Chief Financial Officer Degree in Medicine & MBA Director @several Health International

Ennio Ongini, PhD Chief Scientific Officer PhD in Pharmacology 20 yrs Research Director Schering Plough 15

Noemi Tonna, PhD Lab Manager PhD in Pharmacology Degree in Medical Biotechnology Lab Manager in NeuroZone

Elisabetta Borello, MBA Board Member Degree in Economics @ Bocconi Univ.

2 BrainDTech team Dr. Fabio Bianco, PhD Chief Executive Officier PhD in Pharmacology, Degree in Biotech Founder of NeuroZone (President and CSO) CEO of BrainDTech Director of Sanipedia Business Nursery >10 yr in Biotech/Pharma with executive roles Dr. Pietro Conti, MD Chief Financial Officer Degree in Medicine & MBA Health International Projects in UN agencies (UNDP, WHO, UNICEF) Founder and CEO of several startups Dr. Ennio Ongini, PhD Chief Scientific Officer PhD in Pharmacology 20 yrs Research Director Schering Plough 15 yrs VP Research Nicox SA Member of different evaluation Commitees >200 peer reviewed papers, H-index 38 Dr. Noemi Tonna, PhD Lab Manager PhD in Pharmacology Degree in Medical Biotechnology Lab Manager in NeuroZone >10 yr expertise in cell culturing/ in vitro drug discovery Dr. Elisabetta Borello, MBA Board Member Degree in Bocconi Univ., Milano >30 years expertise in Healthcare Information Management Systems Founder & Board member of many biotechs Dr. Filippo Martinelli Boneschi, MD Scientific Advisor Degree Medicine, Residency Neurology Master Epidemiology & Columbia University PhD in HSR Neurologist, Head of Laboratory of HSR Institute > 120 peer-reviewed papers, PI of National and EU funded grants

The issue (need) 10 15 years Brain INFLAMMATION Brain DEGENERATION CLINICAL SYMPTOMS Currently, diagnosis of brain diseases is carried out observing clinical symptoms,

For example, Parkinson s Disease symptoms (tremor) manifest when over 70% of dopaminergic neurons are dead (Parkinson Disease Foundation 2013).

3 The issue (need) years Brain INFLAMMATION Brain DEGENERATION CLINICAL SYMPTOMS Currently, diagnosis of brain diseases is carried out observing clinical symptoms, which occur when most of the neurodegeneration has already occurred. For example, Parkinson s Disease symptoms (tremor) manifest when over 70% of dopaminergic neurons are dead (Parkinson Disease Foundation 2013). Before neuronal degeneration, there is a sustained inflammatory brain activity, which results in exacerbated neuroinflammation leading to cell death. The onset of this inflammatory reaction can occur even years before the actual symptoms manifest (Lull et al Neurotherapeutics 2010).

in LIQUID BIOPSY BrainDTech represents a disruptive approach to brain disease diagnosis because it detects a signal

4 BrainDTech proposal 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 BrainDTech represents a disruptive approach to brain disease diagnosis because it detects a signal (MicroRNA) contained in microvesicles released by microglia (the immune cells of the brain) years before any clinical manifestation.

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

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.

BrainDTech IP position We have submitted the following patent: "Methods

neurodegenerative and inflammation based diseases via mirna contained in

102015000074820) (AD, PD, ischemia) 2016/07 - PCT patent extension

patterns to be patented and outlicenced for early diagnosis or companion

6 BrainDTech IP position We have submitted the following patent: "Methods for diagnosis, prognosis and treatment monitoring of neurological, neurodegenerative and inflammation based diseases via mirna contained in microglial microvesicles" 2015/11 - Italian patent submission (n ) (AD, PD, ischemia) 2016/07 - PCT patent extension (PCT/EP2016/061940) 2016/11 - Claim extension (Epilepsy, MS, Tourette Syndrome.+20 pathologies) Our strategy is to : Develop pathology specific patterns to be patented and outlicenced for early diagnosis or companion diagnostics in drug development Since mirna control several biochemical processes, we plan to study the mirna patterns obtained and identifty novel therapeutical targets

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

grow exponentially in the upcoming years.

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

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

The market: competition CANCER Few innovative players in cancer diagnosis.

on circulating mirna in plasma for detection of AD BRAIN The main issue with circulating mirna based

approach is revolutionary because we isolate intact microvesicle from a specific cell type (microglia) and

BrainDTech approach is potentially breakthrough because recent scientific evidence (Plog et al J.

8 The market: competition CANCER Few innovative players in cancer diagnosis. One player (Diamir) in brain diagnosis, still in R&D (reached a research agreement with Janssen) focused 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 R&D We are currently running the proof of concept in humans, testing AD patients in order to confirm data so far obtained in in vitro models. Then we will compare 2 indications (AD and PD), then we will proceed in stratifying patients. In parallel, we are testing the possibility of isolating microglial microvesicles from plasma. CSF plasma PoC human MV identification PoC AD vs PD MV isolation AD vs PD clinical study mirna analysis Revenue stream BrainDTech expects to obtain first revenue from pathology specific pattern licencing starting from In the meantime, drug discovery service activity to third parties with proprietary know how on microvesiclce isolation can be provided Services on MV isolation Support to R&D Pathology specific licencing

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

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

EARLY DIAGNOSIS OF BRAIN DISEASES via liquid biopsy, based on mirna patterns in microglial microvesicles BrainDTech Team Dr. Pietro Conti, MD, MBA President Medicine & MBA Founder/CEO several startups