ASCEND BIOPHARMACEUTICALS LIMITED
Ascend Biopharmaceuticals LimitedCorporate Overview
October 2016
ASCEND BIOPHARMACEUTICALS LIMITED 2
Disclaimer
This presentation is being provided for the sole purpose of providing the recipients with background information about Ascend
Biopharmaceuticals Limited’s (“Ascend”) business. This presentation, including the information contained in this disclaimer, does not
constitute an offer, invitation or recommendation to subscribe for or purchase any security, and neither the presentation, disclaimer nor
anything contained in them forms the basis of any contract or commitment. This presentation does not purport to summarize all
information that an investor should consider when making an investment decision. Before making an investment decision you should
consider whether it is suitable for you in light of your own investment profile and objectives and financial circumstances and the merits and
risk involved.
No representation, express or implied, is made as to the fairness, accuracy, completeness or correctness of information, opinions and
conclusions contained in this presentation, including the accuracy, likelihood of achievement or reasonableness of any forecasts,
prospects, returns or statements in relation to future matters contained in the presentation (“forward-looking statements”). Such forward-
looking statements are by their nature subject to significant uncertainties and contingencies and are based on a number of estimates and
assumptions that are subject to change (and in many cases are outside the control of Ascend and its Directors) which may cause the
actual results or performance of Ascend to be materially different from any future results or performance expressed or implied by such
forward-looking statements. Forward-looking statements are provided as a general guide only and should not be relied upon as an
indication or guarantee of future performance.
To the maximum extent permitted by law, neither Ascend nor its related corporations, directors, employees or agents, nor any other
person, accepts any liability, including, without limitation, any liability arising from fault or negligence, for any loss arising from the use of
this presentation or its contents or otherwise arising in connection with it.
You represent and confirm by attending and/or retaining this presentation, that you accept the above conditions. This presentation does
not constitute an offer to sell or a solicitation of an offer to buy securities in the United States.
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Ascend Company Highlights
Ascend is an immuno-oncology company targeting regulated cell death and immune pathways with:
a high value lead opportunity: lead candidate addresses a major high-value clinical need in nodular BCC patients considered to be poor candidates for surgery
a clinically de-risked lead with prospects of an expedited pivotal program: lead candidate has good safety and clinical benefit results in 3 different skin cancers
phase 2 data available shortly: current Phase 2 study in nodular BCC will have top-line interim data in November 2016 and final data in 1H, 2017
lead candidate with multiple follow-on opportunities: potential to address multiple high-value follow-on indications in recurrent ovarian cancer and recurrent cutaneous squamous cell carcinoma
*Based on regulatory precedence for Aldara in Superficial BCC patients who were not good surgery candidates
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Board & Management
Dr Clement Leong, PhD,
MBA
CEO
• Over 20 years of biotech, pharma and venture capital experience in Australia and United States
• Commercial and development roles with Schering Plough, Entelos, SciVentures; lead investor on multiple
private/public biotech and med. tech transactions
• PhD from University of Western Australia; MBA from the Australian Graduate School of Management
Dr. Richard Stead, MD.
Non-executive Director
• Over 25 years directing clinical trials in Industry and licensing transactions
• Formerly Med. Director at Amgen & VP Clinical Res at Immunex; involved in approval of Epogen & Neupogen
• MD from Stanford University; Postdoc training at the Brigham and Woman’s Hospital, Harvard Medical School
Mr George Tsiamis
Non-executive Director
• Over 30 years’ experience in funds management
• Formerly Head of Operations for ANZ Asset Management, $15B in Funds under Management.
• Formerly Chief Financial Officer of the publicly listed McMillan Shakespeare Ltd (ASX: MMS).
Dr. Paul Weiden MD
Chief Medical Officer
• Over 30 years as medical oncologists and directing/assisting with clinical trials
• Formerly a member of the University of Washington, Fred Hutchinson and Virginia Mason Medical Center
• MD from Harvard Medical School.
Dr. Satish Menon, PhD
CMC Director
• Over 26-years of experience in protein based therapeutics R&D and manufacture
• Directed process and bioprocess development for Schering Plough, DNAX, Genitope and Allergan
• Ph.D. from the Indian Institute of Science, Bangalore and Postdoc at Harvard Medical School.
Dr Geoffrey Pietersz, PhD
Director of Technology
Development
• Over 30 years as an organic chemist working in cancer & infectious diseases
• Formerly a Professor at the Burnet Institute, expert in bio-conjugation of therapeutics (e.g., vaccine; antibody
drug conjugates) and inventor of the ASN-004 technology
• Ph.D. from the University of Melbourne.
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Pipeline
CBCL Cutaneous B cell lymphoma; BCC Basal Cell Carcinoma; H&N SCC Head and Neck Squamous Cell
Carcinoma; RCD induction of multiple regulated cell death pathways; IT Immunotherapy; OC Ovarian Cancer;
PC Peritoneal Cancer
Product Composition Indication
ASN-002 ASN-002 Monotherapy Cutaneous BCL Completed
Bladder ASN-002 ASN-002 Monotherapy Nodular BCC H1, 2017 complete
Cutaneo ASN-002/small molecule ASN-002 Chemo-immuno therapy Head & Neck SCC H2 2017 start
ASN-002/small molecule ASN-002 Chemo-immuno therapy Recurrent OC H2, 2017/2018 start
ASN-002/small molecule PlannedASN-002 Chemo-immuno therapy Nevoid BCC H2, 2017/2018 start
ASN-006 Mannan-genetic adjuvant Peritoneal Cancer H2, 2017
Breast ASN-008 Replicon vector - RCD genes* Refractory cancers on-going
Replicon vector - Neoantigens Refractory cancers on-going
Preclinical Phase 1 Phase 2 Phase 3
Addressing high-value clinical opportunities
* Preclinical work performed in collaboration with Fox Chase Cancer Centre in Philadelphia, USA
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The Opportunity: nodular Basal Cell Carcinoma
Basal cell carcinoma is the most common cancer
worldwide arising in ~3 Million patients annually (~2
million with nodular BCC and ~1 million with superficial
BCC)
Prognosis is good if excised but if untreated can cause
significant morbidity/disfigurement
Surgical excision is the main treatment option for nBCC
It is estimated that around 25-30% of nBCC patients
(over 500,000 patients annually) can be considered poor
candidates for surgery
A significant clinical and commercial opportunity in high-risk nBCC
Most nBCC lesions occur on the face, head and neck, making excision and wound closure more difficult
If untreated BCCs become locally invasive and can cause extensive disfigurement
Excision margins of 2 to 5 mm (all around the whole of the outer tumour mass) are appropriate for most BCC resections
The risk of recurrence is determined by the patients age, immune status, and the location and subtype (histology) of the tumour
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Current Non-surgical Treatment Options
Existing options have major disadvantages or are clinically inadequate
Telfer NR Br J Dermatol 2008 159(1) 35-48 Guidelines for the management of basal cell carcinoma; Arch Dermatol August 2012
Small Lesion Large Lesion Comments
Photodynamic Therapy Generally a poor choice Generally a poor choice Poor efficacy for High-risk nBCC
5-FU Poor choice Poor choice Poor efficacy for High-risk nBCC
Radiotherapy Generally good clinical
clearance
Generally good clinical
clearance
Risk of radiation induced secondary
cancers
Aldara Generally a poor choice Should not be used Poor efficacy for High-risk nBCC
Cryosurgery Generally fair choice Generally a poor choice Poor efficacy for High-risk nBCC
Vismodegib/Sonidigib
(Smoothened
inhibitors)
Not recommended Recommended for Locally
advanced basal cell skin
cancer patients with residual
disease in whom surgery and
radiation are contraindicated
Major adverse effects (hair loss,
muscle cramps, taste disturbance)
Secondary resistance
High-risk nBCC
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Rationale for ASN-002 in Nodular BCC
Strong Biological Rationale for the use of Interferon in BCC:
Interferons can be effective in up to 100%* of nBCC patients
o Recombinant Interferon therapy requires multiple daily injections (e.g., schedules of 9-12 administrations)
A single injection of ASN-002 can result in sustained local expression of Interferon for up to 2 weeks
Favorable pathway to registration
• Availability of nodular BCC patients in Australia and US – many productive sites available
Histological clearance an FDA approvable endpoint (prospects for shorter on study time)
No registered therapeutics approved for nodular BCC in target patient setting^
o First mover advantage in high-risk BCC indication possible
o Vehicle-controlled Phase 3 possible (lower hurdle for demonstrating clinical superiority)
*Libby Edwards et al, J Am Acad Dermatol 1990;22:496-500; Stephen B. Tucker et al, J Am Acad Dermatol 2006;54:1033-8.
^Different target patient population to Vismodegib
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ASN-002: Mechanism of Action
A proven and well understood mechanism of action
ASN-002 is an adenovirus (a type of cold-virus) engineered to produce Interferon-g
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Previous ASN-002 Clinical Experience
CR PR
Total
Patients
Patient
Responses
16% 25% 32 41%
53% 32% 19 84%
23% 16% 13 39%
Patients Evaluated 64
Advanced Melanoma
Cutaneous T Cell Lymphoma
Cutaneous B cell Lymphoma
Good safety and clinical outcome data in three types of skin cancers
*For the Advanced melanoma trial – ASN-002 was administered in combination with T cell therapy
*
Advanced cutaneous melanoma patient treated over 4 months
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Milestone and Time-table
Treatment
Follow-up
o Cohort 1. 5x1010 virus particles per injection once a
week for three weeks – up to 6 patients
o Cohort 2 1.5x1011 virus particles per injection once
a week for three weeks – up to 6 patients
o Cohort 4* 3.0x1011 virus particles per injection
once a week for 3 weeks – up to 6 patients
o Cohort 5 2.25x1011 virus particles per injection
once a week for 3 weeks – up to 6 patients (Cohort
5 is only recruited if AEs found in Cohort 4)
17 weeks
Screening (Day -15 to -1)
Week 1 Baseline
Week 2
Week 3
Week 8
Week 4
Week 12
Week 17 (Exit visit)
Dosing: weekly injections for 3 weeks
Interim results expected in Q4, 2016 *In a protocol amendment, cohort 3 was superseded and replaced by cohort 4
Current 18 patient Phase 2 nodular BCC trial with approval for 36 patients
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Registration Strategy
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Next Steps and Development Time-table
Major clinical activities that are planned
- A Type B FDA meeting in 1H, 2017
- Phase 2b/3 pivotal study in BCCNS/Nevoid BCC patients (40-60 patients)
- Phase 1/2a in recurrent locally advanced head and neck cSCC (~24 patients) and chemotherapy resistant locally
advanced ovarian cancer (~24 patients)
- Phase 2b trial in nodular BCC (64 patients)
Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2
2016 2017 2018 2019 2020
Phase 1/2a nBCC
Phase 2b/3 BCCNS (~40-60 patients)
Phase 1/2a H&N cSCC (~24 patients)
Phase 1/2a Ovarian Cancer (~24 patients)
Phase 2b nBCC (~64 patients)
Phase 3 nBCC (~320-400 patients)
*Pre-IND Mtn FDA
*Open IND
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Estimated Addressable Population by Geography
Significant and high value clinical opportunity
Assumes pricing of $800 and $1600 for small and large lesions in Australia/Europe/ROW and *$1800 and $3000 for the US
High Risk nBCC
'difficult surgeries' Small lesion
Large
lesion Small lesion Large lesion
Australia 70,000 59,500 10,500 $47,600,000 $16,800,000
USA* 240,000 204,000 36,000 $367,200,000 $108,000,000
EU 200,000 170,000 30,000 $136,000,000 $48,000,000
RoW 15,000 12,750 2,250 $10,200,000 $3,600,000
525,000 446,250 78,750 $561,000,000 $176,400,000
Market Size AU$
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Transactions
Transactions of comparable products
Potential revenues
• nBCC opportunity could potentially generate over $7 billion in worldwide revenue from 2022 to 2031
• LA OC and LA H&N SCC could generate worldwide revenues of $7.4B and $12.7B over a similar period
*The assumptions and models used to generate these revenue figures are available upon request
Company Candidate Value Date
Completed
Stage Indications
Biovex OncoVec US 425M in cash
and US 575M in
milestones
Jan-11 Phase 3 on-
going
Metastatic
melanoma
Peplin PEP005 US 287.5M Sep-09 Phase 3 on-
going
Actinic Keratosis
Ceptaris
Therapeutics
Valchlor US 25M and US
250M on approval
Aug-12 Phase 3
completed
Cutaneous T cell
Lymphoma
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Additional Slides
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Pipeline inducing Multiple Cell Death Programs
Neo-antigens
Chemokine(s)recruiting cross-presenting dendritic
cells
Immunogenic cell death
DAMPS
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DNA launched SRIPs (Single Round Infectious Particles)
SRIP DNA vectors can be conjugated to targeting moieties (e.g., antibody)
Transfected SRIP DNA vectors produce infectious particles that can transduce cells within the TME
SRIP RNA encode a self-amplifying replicon RNA leading to high levels of gene expression
SRIP RNA can encode immunotherapy payloads such as cytokines, chemokines, antibodies
SRIPs are viral vectors produced within the tumour microenvironment launched from a DNA plasmid
Delivery to stromal or neo-vasculature within the TME SRIPs can encode immuno-oncology genes
SRIP DNA can be conjugated to various targeting moieties
ASN-008 is being developed to induce highly immunogenic cell death
ASCEND BIOPHARMACEUTICALS LIMITED
Ascend Biopharmaceuticals Ltd
Level 1, 159 Dorcas St, South Melbourne VIC 3205, Australia
ACN 106 265 098
Ph: +61 3 8606 3488
Fax: +61 3 9686 9866
Email: [email protected]