Deborah Rathjen CEO & Managing Director Safe Harbor Statement - - PowerPoint PPT Presentation

Creating and developing innovative therapies

Deborah Rathjen

CEO & Managing Director

Safe Harbor Statement

Factors Affecting Future Performance This presentation contains "forward-looking" statements within the meaning of the United States’ Private Securities Litigation Reform Act of 1995. Any statements contained in this presentation that relate to prospective events or developments, including, without limitation, statements made regarding Bionomics’ development candidates BNC105, BNC210 (IW-2143), BNC101(ET101) and BNC375, its licensing agreements with Ironwood Pharmaceuticals and Merck & Co, its acquisition of Eclipse Therapeutics, drug discovery programs and pending patent applications are deemed to be forward-looking

• statements. Words such as "believes," "anticipates," "plans," "expects," "projects,"

"forecasts," "will" and similar expressions are intended to identify forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by these forward-looking statements, including risks related to our available funds or existing funding arrangements, a downturn in our customers' markets, our failure to introduce new products or technologies in a timely manner, regulatory changes, risks related to our international operations, our inability to integrate acquired businesses and technologies into our existing business and to our competitive advantages, as well as other factors. Results of studies performed on competitors products may vary from those reported when tested in different settings. Subject to the requirements of any applicable legislation or the listing rules of any stock exchange on which our securities are quoted, we disclaim any intention or obligation to update any forward-looking statements as a result of developments occurring after the date of this presentation.

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Bionomics Snapshot

KEY STATI STI CS ( July 2 0 1 3 ) ASX code BNO Share price ( 2 6 / 7 / 1 3 ) A$ 0 .4 0 5 2 w eek high A$ 0 .4 5 5 5 2 w eek low A$ 0 .2 4 5 Shares on issue 4 1 1 .1 M Market capitalisation A$ 1 6 4 M Cash 3 0 / 6 / 1 3 A$ 2 2 .5 M MANAGEMENT Deborah Rathjen CEO & MD José I glesias CMO Melanie Young CFO & Com pany Secretary 3 BOARD Graem e Kaufm an Chairm an Deborah Rathjen CEO & MD Trevor Tappenden Non-exec Director Errol DeSouza Non-exec Director Jonathan Lim Non-exec Director 6 MONTH SHARE PRI CE PERFORMANCE

• Emerging biotech with global operations in Australia (ASX:BNO), Europe and the

United States

• World class discovery and development capabilities targeting multi-billion dollar

market opportunities in solid tumour oncology, CNS, and immune diseases

• Broad and deep portfolio of clinical and preclinical drug candidates supported by

proprietary technology platforms:

• BNC1 0 5 solid tumour targeting agent in Phase II trials for renal and ovarian cancer with key data in

early 2014

• BNC2 1 0 (IW-2143) targeting anxiety and depression partnered with Ironwood Pharmaceuticals
• US$345 million in upfront, research and milestone payments, plus royalties
• BNC1 0 1 and other Cancer Stem Cell (CSC) targeting antibodies from the acquisition of Eclipse

Therapeutics; BNC1 0 1 entering clinic in 2014

• Nav1 .7 program targeting chronic and neuropathic pain partnered with Merck & Co (MSD)
• US$172 million in option exercise fees, development and regulatory milestone payments, plus

royalties

• Partnerable Kv1 .3 program and BNC3 7 5 drug candidate anticipated to generate additional value

inflection points in CY2013

Strategy to partner our compounds at key value points to crystallise value, improve rate of success and manage risk

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Bionomics Investment Highlights

Bionomics Business Model

Partnering is key to managing execution risk, validating our compounds and ensuring our compounds reach their markets

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Drug Discovery Drug Development Partnering

• Lay off risk with

experienced partners

• Generate revenue

streams to support discovery programs

• Platform for

delivering new drug candidates

• Propagates pipeline

with multiple shots

• n goal
• Targeted clinical

trials

DRUG CANDI DATE/ PROGRAM DI SCOVERY PRE- CLI NI CAL PHASE I PHASE I I LI CENSEE/ PARTNER

CANCER

BNC1 0 5 – Solid Tumours, renal, ovarian, mesothelioma BNC1 0 1 – Cancer stem cells, Solid Tumours, colon, breast, pancreatic BNC1 0 2 – Cancer stem cells, Solid Tumours VEGFR3 – Solid Tumours, melanoma, breast RET – lung and thyroid Cancers

CENTRAL NERVOUS SYSTEM

BNC2 1 0 ( I W -2 1 4 3 ) – Anxiety/Depression BNC3 7 5 – Cognitive impairment, Alzheimer's Disease, ADHD, schizophrenia GABA-A – Epilepsy NaV1 .7 - Pain

I MMUNE DI SEASE

Kv1 .3 –Psoriasis, Multiple Sclerosis, Rheumatoid Arthritis

Drug Candidate Portfolio Leverages Core Strengths

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CORE STRENGTHS

Targeted licensing point

Nav 1.7: $172M Pain Partnership with Merck & Co

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COMMERCI AL ELEMENTS

• Option and license agreement with Merck & Co
• US$ 1 7 2 m in option exercise fees, development/regulatory milestone

payments, plus royalties THERAPEUTI C AREA

• Treatment of chronic pain, including neuropathic pain

MARKET SI ZE & POSI TI ONI NG

• Pain Market totalled US$22 billion in sales in 2010
• Neuropathic pain segment of market expected to grow from US$2.4

billion in 2010 to US$3.6 billion by 2020

• Current medications have limited effectiveness:
• estimated only 1 in 4 patients with neuropathic pain achieve

>50% reduction in pain levels

• side-effects include drowziness, somnolence and dizziness

VALI DATI ON

• Nav1.7 as a target has been validated through genetic studies
• Deal further validates ionX & MultiCore drug discovery platforms
• Value creation through strategic partnering business model
• Future success-based revenue streams & royalties secured

BNC210 (IW-2143): A next generation compound with potential in the treatment of anxiety and depression

TREATMENT Anxiety and Depression MODE OF ACTI ON

• Modulates novel pathway, to promote anti-anxiety activity and neurite
• utgrowth in vitro.

CLI NI CAL

• Four Phase I trials completed, including a trial assessing panic attack

symptoms:

• 59 subjects enrolled in double-blinded placebo controlled trial;15

subjects classified as having a panic attack upon CCK-4 administration

• Statistically significant decrease in both number & intensity of

symptoms (p<0.05)

• BNC210 treated subjects returned to normal emotional status within

10 minutes, compared to 60 minutes on placebo

• This trend correlated with the statistically significant reduction in

panic symptoms by BNC210

• BNC210 has been administered to 108 healthy subjects to date with

excellent safety profile BENEFI TS

• BNC210-related changes in human brain activity indicative of efficacy
• Reduced panic symptoms
• No evidence of sedation or addiction to date

MARKETS

• Anxiety – global sales of US$5-7bn annually
• Depression – global sales US$11bn
• Partnered with Ironwood (NASDAQ:IRWD) US$345 million in upfront,

research and milestone payments, plus royalties

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COMPETI TI VE ADVANTAGES OF BNC2 1 0 * DRUG NO SEDATI ON NO W I THDRAW AL SYNDROME NO MEMORY I MPAI RMENT FAST ACTI NG NO DRUG/ DRUG I NTERACTI ONS ONCE-A- DAY DOSI NG BNC2 1 0

     

VALI UM

     

PROZAC

     

BUSPAR

     

* Based on preclinical data and results of Phase I trial comparing BNC210 with Lorazepam

BNC210 (IW-2143): Fewer side-effects expected to be a key product differentiator

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• 7 NAChR receptor activation improves attention, working memory

and recognition memory

• Following late-stage development failures by drugs targeting β-

amyloid (Lilly, Pfizer, J&J), 7 NAChR approaches have come to the forefront; Pharma hungry for AD drugs

• Market opportunity for drugs modulating 7 NAChR activity includes

many neurodegenerative and psychiatric disorders:

• Alzheimer’s Disease (AD), Parkinson’s Disease, Schizophrenia, ADHD and

mood and anxiety disorders Prevalence Estim ated Global sales Alzheimer’s Disease 9.7 Million $10.2 Billion (2012) Cognitive Dysfunction in Schizophrenia 3.4 Million No approved products ADHD 44.9 Million $4.9 Billion Parkinson’s Disease 7.0 Million $3.5 Billion

BNC375: More than Alzheimer’s Disease

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BNC375: Competitive advantages*

Characteristics Bionom ics BNC3 7 5 Com peting Agents+ Potent   Rapid onset of action   Potentiates endogenous receptor ligand   Preserve the normal signalling patterns of the receptor   Do not cause receptor desensitization   No potential for development of tolerance   Selectivity over other nicotinic receptors  

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*Based on data from preclinical animal studies +Published information and Bionomics’ in-house data

BNC105: “Best in Class” vascular targeting agent with unique dual mechanism of action

TREATMENT

• Solid Cancers: New paradigm w ith blockbuster potential through rational combination

with molecular targeted therapies that benefit from induction of tumour hypoxia MARKETS

• The current market size in treatment of all solid tumours is >US$10b (Avastin,

Genentech/Roche sales >US$5b in 2011)

• Renal cancer market size >US$2.5bn; include TKI Sutent, Pfizer; Nexavar, Bayer/ Onyx;

Afinitor, Novartis

• Ovarian cancer market size US$2.2bn in 2011

CLI NCAL

• Phase II Renal cell cancer (US, Australia, Singapore)
• Trial design: randomized 2-arm; Afinitor+/- BNC105; enrolment of 139 patients

completed; patients previously failed TKI therapy; Primary endpoint 6 month PFS; data anticipated 2H, 2013

• Phase I results: combination BNC105 + Afinitor safe and well tolerated; 12 patients

enrolled across 4 BNC105 dose levels (4-16mg/m2); 8 patients achieved stable disease; 1 patient still on study having received 24 treatment cycles (72 weeks, 18 months)

• Phase II Mesothelioma (Australia): single arm; BNC105 following Alimta + cisplatin failure;

30 patients enrolled; 1 patient with durable & sustained partial response with corresponding reduction in mesothelin levels; 13 patients with stable disease

• Phase I/II Ovarian cancer (Australia, New Zealand, US)
• Phase I enrolment completed; carboplatin and gemcitabine +/- BNC105; population

partially sensitive to platinum-based therapy BENEFI TS

• Dual Action – selectively targets both tumour blood vessels and cancer cells
• Rapid, Potent Action – works rapidly to shut down tumour blood vessels, potent anti-

tumour action as a single agent, tumour less likely to escape treatment

• Enhances Effectiveness of Other Cancer Treatm ents – delivers synergistic anti-cancer

effects in numerous combinations (e.g., anti-VEGF) 12

BNC1 0 5 treated untreated

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By selectively shutting dow n tum our blood vessels, BNC1 0 5 rapidly inhibits tum our grow th

BNC105: Targeting solid tumours

BNC105: Renal Cancer

• Phase II Trial Enrolment now

complete

• 139 patients
• On pathway to partnership
• Potential to be a new

treatment paradigm for patients with renal cancer

• Encouraging initial results

from US renal cancer trial

• Combination of Afinitor &

BNC105 safe & well tolerated

• As effective as Sutent in

reducing tumour size in animal model

• Sutent (Pfizer) is the current

market leader with 2010 global sales US$1b

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Right Kidney Tum our Burden

Globally, Renal Cancer is 7th most common form of cancer resulting in over 100,000 deaths p.a.

Cancer Stem Cell Therapeutics

• The missing link in

treating solid tumours

• CSCs are resistant to

conventional therapies

• Eradicating CSCs may

prevent tumour recurrence

• Complements the

BNC105 program by targeting the “seeds”

• f cancer

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BNC101: Targeting cancer stem cells in solid tumours

TREATMENT Solid tumours; colon, breast, pancreatic

MOA

• Humanised monoclonal antibody
• BNC101 binds selectively to LGR5; LGR5 marks

tumour-initiating cells in colon and gastric cancer

• LGR5 is highly overexpressed in colon, ovarian, liver,

breast, lung and other solid tumours

• Ligand: R-spondin, forms part of LGR5 – LRP6 – Wnt –

Fzd signaling complex

• High expression of LGR5 in colon cancer has been

linked to tumour recurrence and poor prognosis MARKETS Market for cancer stem cell therapeutics has been estimated to be a $8B market opportunity by 2018 DEVELOPMENT STAGE Late preclinical; GMP manufacture by Lonza; IND enabling studies commenced in US; Anticipated IND filing 2014 BENEFI TS First-in-class therapeutic antibody against validated CSC target Specific targeting of cancer stem cells

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Market Opportunity: CSCs Next Significant Advance in Cancer Treatment

Com pany Technology Financials OncoMed

• CSC antibodies targeting cancer

gene/signalling pathways (Wnt & Notch)

• Strong preclinical trial results
• Clinical trials ongoing

Strategic alliance: Bayer

• Total: >$1,975M
• Upfront: $40M

Strategic alliance: GSK

• Total:>$1,400M
• Upfront: undisclosed
• IPO July 2013, raised $85M,

priced $17 per share, market cap $500M

Stem line Therapeutics

• Discovery Platform
• Phase I/II clinical trials
• IPO Jan 2013

(NASDAQ:STML)

• Market cap  $330M

Verastem

• Screening platform CSC-like cells
• Preclinical Wnt program
• Clinical focal adhesion kinase (FAK) program
• IPO 2012 (NASDAQ:VYST)
• Market cap~$330M

Boston Biom edical

• Advanced clinical development programs

(Phase I, II, planning Phase III) Acquired by Dainippon for >$2.6B in 2012 ($200M upfront)

Roche ( Arius)

• Pre-IND anti-CD44 antibody

Acquired for $184M cash by Roche

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R&D Outlook & Anticipated Milestones: Partnership Focus

Key Program Milestone Anticipated Tim ing ( CY) BNC1 0 5 : All Solid Tum ours Complete Phase II renal cancer trial enrolment 1H, 2013 Results from renal cancer trial 1Q, 2014 Complete ovarian Phase I trial enrolment 1H,2013 New data presentations at AACR, ASCO-GU & ASCO 1H, 2013 BNC1 0 1 ( form erly ET1 0 1 ) : Cancer Stem Cells Initiation of IND enabling studies & GMP manufacture 1H, 2013  BNC3 7 5 / Alpha 7 : Alzheim er’s Disease/ Cognitive Enhancem ent Drug candidate selection 2H, 2012  Initiation of IND enabling studies & GMP manufacture 1H, 2013 Kv1 .3 : Autoim m une Disease Partnership 2013

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 Nav1.7 partnership with Merck & Co ‐ potential for up to US$172 million in option fees, regulatory and development milestones, plus royalties

BNO Investment Proposition

• Bionomics has world class discovery & development capabilities targeting multi-billion

dollar market opportunities in cancer, CNS & immune diseases

• Bionomics’ technology platforms deliver diversified product pipeline, while corporate

partnering manages execution risk

• Clinical trial success of BNC105 in renal cancer should lead to a lucrative licensing

transaction

• Licensing of anxiety drug BNC210 to Ironwood has potential to deliver significant

shareholder returns via milestone payments and royalties

• Latest partnership with Merck & Co (MSD) further demonstrates Bionomics’ business

model

• Advancing range of additional pipeline drug candidates including BNC375 Alzheimer’s

candidate & Kv1.3 program with strong partnering potential

• Acquisition of Eclipse Therapeutics (now Bionomics Inc) has positioned BNO as a

leading player in cancer stem cells, adding high value antibody therapeutics & a US platform

• Bionomics well positioned to execute its plan to build sustainable revenue streams

from multiple drug candidates

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ASX:BNO

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