H1 2005

1
H1 2005 August 2005
ANSWERS for CANCER
2
Contents

• H1 2005 financials and results
• Expectations for 2005
• Highlights
• Product portfolio

3
H1 2005 financials and results

4
H1 2005 financials and results
5
2005 Expectations

• Expectations for 2005
• Research and development expenses DKK 115
  million to DKK 125 million
• Administrative expenses and
• marketing and sales costs DKK 35 million to
  DKK 40 million
• Loss DKK 70 million to DKK 90 million
• Unchanged and as forecast in the Prospectus from
  9th June 2005

6
Highlights

• TopoTarget an integrated oncology product
  development company
• Based in Copenhagen (HQ), Oxford and Frankfurt
• Focussed on the highly unmet clinical need in
  cancer
• Early revenues from specialist products
• Value growth from major products
• Clear path to profitability
• Critical mass
• Experienced management and partners

2006 gt2007
gt2008
PXD 101
BACECA
SAVENE
PEAC
AVUGANE
Topotect
7
Highlights key milestones achieved post IPO

• Milestone highlights
• IPO in June 2005
• SAVENETM EU application for marketing approval
  submitted to EMEA
• PXD101 Extension of Phase II clinical trials to
  US centres
• PEAC Granting of orphan drug designation by the
  FDA in the US
• Further patent rights granted for TopoTarget
  products
• SAVENETM (Russia, China)
• BACECA (Australia)
• HDAC inhibitors (Europe)

Jun 2002
Jun 2004
2002/2003
Aug 2004
Oct 2000
Apr 2004
Feb 2005
April 2005
June 2005
May 2004
Established
CuraGen deal
Chiron deal
IPO
Acquisition of Prolifix
Extravasation technology
NeuroSearch deal
Brain metastases
Acquisition of G2M
NCI collabor.
8
Strong foundations in clinical oncology
Drug platform based on key cancer enzyme targets
Proliferation
Single cell
Tumour
Exploit
Control
HDACi
Topo II
Commercial partners
Acquisitions
Strong science
Clinical Networks



HDACi
Topo IIi

• Triggers cancer cell death
• Substantial Big Pharma interest
• PXD101 in Phase II trials

• Refines topo II therapy
• SAVENE inhibits activity and controls toxicity

9
TopoTargets clinical pipeline
In-house programmes
Technology
Name
Indication
Pipeline status
Next milestone
Anticipated timing
Partner
SAVENE
Topo IIi
Extravasation
Phase II/III
Approval
1H 2006
-
SAVENE
PXD 101
HDACi
Muliple myeloma
Phase II
PII data
2H 2006
CuraGen
AVUGANE
HDACi
Acne vulgaris
Phase II
PII data
2H 2005
-
BACECA
HDACi
Basal cell carcinoma
Phase I/II
PII data
1H 2006
-
PEAC
HDACi
FAP
Phase I/II
PII data
2H 2006
-
PEAC
Topotect
Topo IIi
Brain metastases
Phase I
PI data
2H 2006
RH / RAFA
In-licensed programmes
Endovion
VRACi
Brain cancer
Phase I
PII data
gt2006
NeuroSearch
ZEMAB
Anti-HER2
Breast cancer Headneck cancer
Phase I
PII data
gt2006
-
10
SAVENE for extravasation

• Clinical need costly complication
• No effective therapy
• Affects 1 in 100 cancer patients
• TopoTarget solution SAVENE
• Must be used within 6 hours
• Kit must be kept at the ready
• Product supply secured
• Priced based on pharmacoeconomics

Extravasation
Current therapy
Treatment with SAVENE
11
SAVENE for extravasation

• Phase II/III clinical trials
• Patients 71 patients recruited, 51 eligible
• Multicentre trials successfully closed
• Primary end points prevention of necrosis and
  surgeries
• Randomised control trials unworkable for ethical
  reasons
• Phase II/III data are convincing
• 98 success - only one patient failure
• FDA and EMEA Orphan Drug Designation in EU and
  US
• Anticipated market launch
• EU Late 2006
• EU application for marketing approval filed
• US Late 2006
• US application for marketing expected 2H 2005

• n

• Necrosis

• SUCCESS
• RATE

51
1
98
TT01 P value lt 0.025
TT02 P value lt 0.025
12
TopoTargets European sales force

• Clearly defined and addressable target market
• SAVENE sales team to cover Western Europe niche
  markets for specialist products
• Leverage SAVENE sales team for future niche
  products (PEAC)

TopoTargets sales and marketing team has
substantial pharma experience to drive an
effective product to market strategy
13
PXD 101 high potential product

• Phase II trials started in multiple myeloma
• Ideal target for PXD 101 prevent cancer cell
  growth
• Trials active in Europe and USA
• Velcade efficacy but no cure
• First indication more in process
• PXD 101 rationally developed HDACi
• HDACi proof-of-concept demonstrated SAHA
• PXD 101 highly potent, low tox, broad range HDACi
• Phase I trial using i.v. PXD 101 completed
• Safely administered to cancer patients

Complete data from MM Phase II trial anticipated
H2 2006
14
PXD 101 high potential product

• PXD 101 extension to other indications
• Substantial clinical trials program lymphoma,
  colon and ovarian cancer
• Further 11 clinical trials to be sponsored by the
  NCI (USA)
• Partnered with CuraGen (NASD CRGN)
• A good partner financially, strategically and
  operationally
• TopoTarget retains European sales rights CuraGen
  for US and RoW

15
BACECA for basal cell carcinoma (BCC)

• Clinical need BCC is the most common of all
  cancers 5 million
• patients annually - usually treated by surgery
• TOPO solution BACECA provide an effective
  topical treatment
• BACECA is a proprietary novel use of the HDACi
  valproic acid (VPA)
• Commercialization strategy
• BACECA will be partnered

16
BACECA for basal cell carcinoma (BCC)

• Clinical efficacy in pilot study
• Patients 10 included 7 with history of
  failed therapy
• Treatment BACECA in combination with
  retinoids
• Phase IIb trials start in Q3 2005
• Patients Two double-blind, randomised
  trials, each with 45 patients
• Treatment Retinoid /- BACECA

Clinical efficacy data

• n

• Full response

• Partial response

• No response

• 4

• 0

• 10

• 6

6 weeks BACECA retinoid
Before treatment
Pictures shown for illustrative purposes
17
AVUGANE for acne vulgaris overview

• Clinical need ineffective therapies and adverse
  effects
• TOPO solution provide an effective topical
  treatment
• Develop VPA and A-vitamin into AVUGANE for
  topical application
• Phase II clinical trials quick and cost
  effective - ongoing
• Substantial market approval anticipated for gt2008

18
Topotect for brain metastases (BM)

• Topotect

• Clinical need lethal malignancy with 0.3m
  cases/year
• Problem normal doses of chemotherapy will not
  kill cancer cells in the brain because of the
  blood brain barrier
• TOPO solution high dose chemo without toxicity
• Comprises two compounds
• Etoposide targets dividing tumour cells, not
  found in normal brain tissue
• Topotect inhibits etoposide, does not cross
  blood-brain barrier (BBB)
• Extensively demonstrated in animal models

Cytotoxic agent
Protective agent

• Protects body
• Crosses BBB poorly
• Allows a higher dose of cytotoxic

• Kills cancer cells
• Needs high doses to work in brain

• Treat in tandem

• Brain receives therapeutic dose
• Body protected from overdose

19
Topotect for BM clinical development

• Preliminary Phase I clinical trials completed
• 10 patients no effect on etoposide
  pharmacokinetics
• Compound well tolerated when used with etoposide
• Full Phase I clinical trials ongoing
• Define most suitable schedule of high dose
  etoposide and Topotect
• Two multi-centre trials underway

20
PEAC for FAP and colon cancer

• Clinical problem predisposition to colon cancer
• Current therapy is surgery removal of the colon
• TopoTarget solution HDACi to target polyp growth
• Phase II pivotal clinical trial
• Orphan status in EU and USA
• Marketing strategy leverage SAVENETM channel
• FAP patients are well organised
• Successful PEAC can be applied to colon cancer

21
Summary
ANSWERS for CANCER

• Early cash flow from niche products
• High upside from potential blockbuster products
• Growing into integrated oncology company with own
  sales