BBY Life Sciences Conference Regeneus (ASX:RGS) John Martin - - PowerPoint PPT Presentation

BBY Life Sciences Conference Regeneus (ASX:RGS)

“Improving quality of life through innovative cellular therapies”

John Martin Executive Chairman Sydney/Melbourne November 2013

Important Notice

No offer to sell, issue or recommend securities This document does not constitute or form part of any offer or invitation to sell or issue, or any solicitation of any offer to purchase or subscribe for, any shares in the Company, nor shall any part of it nor the fact of its distribution form part of or be relied on in connection with any contract or investment decision relating thereto, nor does it constitute a recommendation regarding the securities of the Company. Forward-Looking Statements This document may contain forward-looking statements, forecasts and estimates made by the Company (forward-looking statements), including the anticipated future performance of the Company and the market in which it operates. These forward-looking statements are based on certain key expectations and assumptions of known and unknown risks, uncertainties and other factors, which where deemed reasonable when made but may or may not prove correct. Actual events are difficult to predict and may depend upon factors that are beyond the Company’s

• control. Therefore, actual results, the financial condition, performance or achievements of the

Company, or industry results, may turn out to be materially different from any future results, performance or achievements express or implied by such forward-looking statements. Forward- looking statements only speak as of the date of this document and no representations are made as to the accuracy or fairness of such forward-looking statements. The Company disclaims any

• bligation to update any forward-looking statements, to reflect any change in the Company’s

expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement is based.

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Overview – emerging leader in cell-based regenerative medicine

• Emerging leader in cell-based regenerative medicine

– developing and commercialising innovative cell therapies and complimentary biologicals – for unmet medical needs in human and veterinary health markets

• Focus on 4 major disease areas in human and vet health:

– Osteoarthritis and other inflammatory musculoskeletal conditions – Pain management – Inflammatory skin conditions – Oncology

• Underpinned by 4 proprietary technology platforms:

– autologous (your cells) adipose (fat) mesenchymal stem cells (MSCs) - HiQCell – allogeneic (donor cells) adipose MSCs – CryoShot – allogeneic adipose MSC secretions - Secretions – autologous cancer vaccine - Kvax

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Competitive edge

• Adipose tissue provides competitive advantage

– rich and accessible source of MSCs – 500-1000 times more MSCs than bone marrow per gram –

• nly source of MSCs to provide a therapeutic dose without need to culture
• Low risk nature of autologous adipose MSC technology enables

– accelerated product development and commercialisation pathways for vet and human markets – accelerated path to pioneering clinical data and engagement with clinicians – flexibility in treatment applications drives medical innovation

• Veterinary cell therapies provide

– accelerated product and clinical development and market feedback eg Cryoshot – leverages development of human cell therapies – eg Human OTS and Kvax

• Strategic IP portfolio

– understanding of mechanism of action of adipose regenerative cells: what cells secrete vs what they differentiate into; network effect of regenerative cells; activity in vivo – measurement of biomarkers to monitor impact of cell therapies

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Strong price performance since IPO

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• Incorporated in 2007
• ASX Listing Date - 19

September 2013

• Raised A$10.5m at $0.25c (mkt

cap $45m)

• 183.8m shares on issue (26%

subject to escrow)

• First ASX biotech IPO since

2011

• Share price up 96% since listing
• $0.49c (mkt cap $90m) at

17.11.13

RGS share price since listing on 19 September 2013

HiQCell – autologous cell therapy for OA

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• Cell-based therapy using autologous adipose derived

MSCs to treat human osteoarthritis and other musculoskeletal conditions

• Simple, low risk, same day procedure performed by

licensed medical specialist (exempt biological under TGA regulatory framework)

• Small amount of adipose

tissue is harvested via liposuction

• 1. Tissue

Harvest

• Regeneus isolates

regenerative cells from adipose tissue at point of care and creates cell suspension for injection

• 2. Cell

Processing

• Medical specialist injects

cell suspension into the joint

• Multiple joints can be

done at the same time

• Cells can be stored for

future injections

• 3. Cell

Injection

HiQCell – commercial roll out

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• Early Commercialisation

– Over 350 procedures across 800 joints since commercial launch in FY12 – Available through sports med and orthopaedic specialists in Sydney, Melbourne and Gold Coast – expand to other major cities – Look to expand into select foreign markets – Singapore, EU – Cryostorage option for future injection of cells – “off-the-shelf” – Proprietary inflammatory biomarkers (MIF and CTXII) to measure impact

• f therapy and timing of reinjection

– Supported by RCT and registry data (>230 patients and growing)

• Focused on treatment gap

– Between pain medication and joint replacement – OA is # 1 chronic disease in Australia – no cure – OA affects ~4m people and estimated to grow to 7m by 2050 – >80k joint replacements (knee and hip) per year and growing – Expand applications to pain management

CryoShot – Off-the-Shelf Cell Therapy

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• Fat from donor animals during desex

> 40k vials from single donor

• Genuine off-the-shelf product

– delivered in frozen 2ml vials to maximise the flexibility in terms of use, shelf life and viability – in-house manufacturing, distribution and handling capabilities

• > 2000 vials administered for the

treatment of canine and equine musculoskeletal conditions since introduction in early 2012 (APVMA permit 7250) – > 70 vet practices have taken up the trial product

Canine CryoShot – seeking registration

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• Extensive clinical data shows significant

reduction in pain and improvement in mobility within 10 days of treatment with no need for ongoing surgery or drug

• Seeking registration for canine OA in:
• Australia (APVMA),
• US (FDA),– opened INADA with FDA on

28 June ’13

• Commence US efficacy trial in 2014
• EU (EMA)
• Seeking to enter markets with pre-

registration access schemes eg some EU countries

Initiating Human CryoShot

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• Experiments demonstrate >500k units of product from one

donor

• Product uses same manufacturing spec as veterinary

CryoShot

• Ethics approval for safety study in 2014
• Opportunity to explore new accelerated cell therapy

approval process in Japan – limited safety and efficacy data

• New Japanese regs proposing 5 year licence with data

reporting requirements and up to 70% reimbursement

Novel Autologous Cancer Vaccine

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• Cancer vaccine technology that uses dog’s tumour cells as

source of therapy

• Developed at Kolling Institute of Medical Research – RNSH/

Usyd

• Exclusive worldwide commercial rights for vet and option
• ver human applications

Compelling Pre-Clinical and Safety Data

Page 12 25 50 75 100 125 150 175 25 50 75 100 Controls Vaccine 2 doses *P=0.0165

B

Days Percent survival 25 50 75 100 125 150 175 25 50 75 100 Controls Tumour rechallenge *P=0.0056

E

Days Percent survival

• Studies in rats in a glioma model

– analogous tumour to human glioma (brain tumour)

• Very significant increase in survival

rates (30-60%)

• Re-challenge with glioma did not

result in tumour formation

• Safety study in 40 dogs (all comers)

did not elicit any safety concerns – 84% outlived expected survival times for cancers – liver, bone and melanoma

• Research to be published in early

2014

Accelerated Path to US Vet Market

• Center for Veterinary Biologicals (USDA) green light for commercial use in

USA – November ‘13

• US marketing trial planned with KOL for Q1 2014 for nominated aggressive

cancer vs expected survival times

• Opportunity to fast track launch in US market
• Exploring other major vet markets for accelerated market entry
• Plan to fast track vaccine into human safety study
• Potential for vaccine to have accelerated pathway for humans in

Australia as “exempt biological”

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Stem Cell Cream for inflammatory skin conditions

• Stem cells secrete cytokines and growth factors that modulate the

immune system to:

• decrease inflammation
• stimulate repair and regeneration
• reduce scarring
• Secretions based cream has demonstrated promising results for:
• acne
• psoriasis
• dermatitis
• insect stings and bites
• burns
• Finalising manufacture scale up and commercialisation pathways
• Complete safety study in FY14

Accelerated healing

• Liquid nitrogen burns

1 week

After 1 day blistered Treatment begun on right leg burn at 1 hr timepoint No treatment used on left leg burn Considerable reduction in pain on the treated burn

1 month 28 months Treated Untreated

Product Pipeline

Indication Product Pre-clinical Clinical Market Human

Osteoarthritis/ Musculoskeletal/Pain HiQCell

Exempt biological

OA / MSK CryoShot Human Oncology Kvax Human

Exempt biological

Acne/inflammatory skin conditions Secretions

Regulated and non-regulated

Animal

OA / MSK CryoShot Canine/ Equine

Permit 7250 (Aus)

Oncology Kvax Canine

autologous adipose MSCs allogeneic adipose MSCs autologous tumour cells

Outlook for next 12 months

• HiQCell – Autologous point-of-care

– Expand treatment footprint and procedure rates in Australia and launch in foreign market – Explore commercial partner opportunities – Grow clinical data through Registry – Extend to new musculoskeletal applications – pain management

• CryoShot – Canine OTS

– Enter into manufacturing agreement – FDA sign off on product for trial and trial design – Continue to gather field trial data in Australia

• Cancer Vaccine – Vet and Human autologous

– Commence US marketing trial in canines – Prepare for market launch – Seek safety trial in humans

• CryoShot – Human OTS

– Finalise product manufacture – Approval for safety trial

• Secretions Cream-OTS

– Safety study for topical applications for inflammatory skin conditions – Explore accelerated market entry

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Questions

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