1. ProInvestor Life Science Seminar
Anders Vadsholt (CFO) and Lars Damstrup (Medical Director)
Topotarget A/S – March 27, 2012

2. Safe harbor statement
This presentation may contain forward-looking statements, including statements about our expectations of the
progression of our pre-clinical and clinical pipeline including the timing for commencement and completion of
clinical trials and with respect to cash burn guidance. Such statements are based on management's current
expectations and are subject to a number of risks and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements. Topotarget cautions investors that there
can be no assurance that actual results or business conditions will not differ materially from those projected or
suggested in such forward-looking statements as a result of various factors, including, but not limited to, the
following: The risk that any one or more of the drug development programs of Topotarget A/S will not proceed
as planned for technical, scientific, or commercial reasons or due to patient enrollment issues or based on new
information from non-clinical or clinical studies or from other sources; the success of competing products and
technologies; technological uncertainty and product development risks; uncertainty of additional funding;
Topotarget's history of incurring losses and the uncertainty of achieving profitability; Topotarget's stage of
development as a biopharmaceutical company; government regulation; patent infringement claims against
Topotarget's products, processes, and technologies; the ability to protect Topotarget's patents and proprietary
rights; uncertainties relating to commercialization rights; and product liability exposure. We disclaim any
intention or obligation to update or revise any forward-looking statements, whether as a result of new
information, future events, or otherwise, unless required by law.
2

3. Topotarget corporate presentation
• Executive summary
• Belinostat supercharging chemotherapy
• Financial overview
3

4. Topotarget at a glance (1)
An international Scandinavian-based biopharmaceutical company
dedicated to develop and market cancer therapies
• Headquartered in Copenhagen, Denmark (Medicon Valley Alliance) with ~24 employees with a
lean cost structure pursued
• Focused on development and commercialization of belinostat, a molecular targeted cancer therapy
• Divestment of Topotarget USA, Inc. and Totect to Apricus Biosciences, Inc. closed on December 30,
2011
Listing NASDAQ OMX Copenhagen
Symbol TOPO
Market capitalization
DKK 400m
(as of March 22, 2012)
No. of shares
132,652,050
(as of March 22, 2012)
4

5. Topotarget at a glance (2)
• In collaboration with Spectrum Pharmaceuticals, Inc. (SPPI) and the NCI we are developing and
commercializing belinostat in the US
• Based on current plans and without taking potential SPPI milestones into account, cash resources
will take us into 2013
Shareholder Ownership Geographic split of shareholders*
The 10 largest shareholders Other
+ 30%
combined 20%
HealthCap funds + 13%
Denmark
Avanza Pension + 6% 40%
UK
8%
* Estimated
Switzerland
10%
Sweden
22%
5

6. Topotarget is making a difference to
cancer patients …
Creating shareholder value
Lead Strong
Unmet
Track record development
TOPOTARGET Partnerships
– STRONG INVESTMENT CASE belinostat
market need
candidate profile
Proven track Novel cancer drug Strong Solid tumor Promising
record only 7 target: partnerships: preliminary
years from idea to Hematological clinical efficacy
launch: HDACi Spectrum diseases
Pharmaceuticals, I Robust safety
Totect® nc. Solid commercial
Savene® potential Strong IP
National Cancer
Institute Large database
Rigshospitalet (DK) Mono- and
combination
therapy
6

7. … with a clear-cut focus on belinostat
2010 2012 
Belinostat
Early-stage acquisitions • Optimized organization • PTCL***
• Prioritized late-stage pipeline • Cancer of unknown primary
Savene®/Totect®
• Experienced development team • MDS/AML***
Unfocused early-stage pipeline • US partner • Non-small cell lung cancer
• Research facility closed down • Bladder cancer
Discovery platform
• Enhancement of BoD and • Hepatocellular cancer
High burn rate, 100+ employees management team • Ovarian cancer
• Establishment of GOAB* and
Several cancer targets disease-specific advisory boards *) Global Oncology Advisory Board
**) Clinical Study Report
• Completion of CSRs** ***) Peripheral T-cell lymphoma, myelodys-
Belinostat plastic syndromes, acute myeloid
leukemia
Take-off 2011
7

8. Partnerships are key to a successful
commercialization of belinostat
North Asia
Canada
Europe
USA
China
Mexico India
Africa
South
America
Australia
China: First right of negotiation
to Spectrum Pharmaceuticals, Inc.
8

9. Belinostat partner agreement with
Deal terms
• Agreement, February 2, 2010
• Total deal value USD 350 million, USD 30 million cash upfront, double-digit royalties
• Milestone includes 1 million SPPI shares and cash milestone at acceptance of PTCL NDA filing
• SPPI funds PTCL BELIEF trial and Topotarget funds randomized phase II CUP study
• Co-development in additional indications, cost split 70/30 (SPPI/Topotarget)
• Joint development and commercialization committees set-up
• Territory: North Ameri-ca and India, with a first right of negotiation to China
• SPPI is responsible for submitting an NDA on belinostat for PTCL
9

10. Overall strategy for 2012 and beyond (1)
belinostat
Continue to establish belinostat as one of the most
successful HDAC inhibitors in selected indications by:
– Analyzing late-stage PTCL and CUP study results
– Submitting NDA to the FDA in PTCL through US partner
Spectrum Pharmaceuticals (SPPI)
– Exploring commercial opportunities outside the US to
maximize commercial value
– Unlocking full potential by initiating further clinical studies in
the most promising indications within hematology and solid
tumors
10

11. Strategy for 2012 and beyond (2)
• Pre-market and launch PTCL in ex-US countries
– Timely and complete submission of NDA (led by SPPI)
– Leverage FDA approval for launches in Latin America, Eastern Europe (non-EMA), and
Asia/Pacific (led by Topotarget A/S)
• Identify partners and/or distributors for ex-US markets
• Develop life-cycle plan and prepare markets for future indications
– Liquid tumors – pursue at least one hematology indication
– Solid tumors – leverage CUP data into randomized trials, e.g. bladder cancer
– Initiate investigator-driven clinical trials
• Develop aggressive communication plan to drive customer value and
differentiation vs. competition
• Strengthen customer collaborations and engagements
– KOLs, oncology organizations, and advocacy groups
11

12. Topotarget corporate presentation
• Executive summary
• Belinostat supercharging chemotherapy
• Financial overview
12

13. Histone DeACetylase inhibitors (HDACi)
Bypassing natural apoptosis is a hallmark of the cancer disease
Main characteristics of belinostat
• ”Turns on” suppressor genes
‒ Inhibiting HDACs activate silenced genes
‒ Some of these are apoptotic (cell death) genes
‒ Activation causes selective cancer cell death
• ”Turns off” oncogenes
‒ Results in inhibitions of cancer cell growth
Other mechanisms of action
• Inhibition of the growth and development of new blood vessels, in effect starving cancer cells
• Induction of immune system to target cancer cells
• Interacts with for example tubulin, thus synergizing with various chemotherapies and potentially
overcoming drug resistance, which is the main reason for failure of cancer treatment
13

14. Belinostat is supercharging chemotherapy
Cancer cells can over time develop resistance to the given chemotherapy – a
resistance that HDACi’s may overcome
HDACi’s work in synergy with chemotherapy by supercharging the effects of
cytostatics
Belinostat is a novel pan-HDACi with the ability of regulation of multiple class I
and II HDAC’s
Belinostat in combination with chemotherapy increases efficacy with only a minor
or moderate increase in toxicity
14 14

15. Belinostat has a compelling clinical profile
FLEXIBLE ADMINISTRATION
Option of multiple administration and formulation modes
(IV, CIV, and oral)
ABILITY TO COMBINE
Combined with main established chemotherapies will lead to a maximized
commercial potential
ENCOURAGING SAFETY PROFILE
Shown to be well tolerated in the clinical use (≈900 pts.), with an excellent safety
including cardiac toxicity profile and minimal bone marrow toxicity
PROMISING EFFICACY DATA
Preliminary clinical efficacy in solid and hematological malignancies
Synergistic pre-clinical effect with established therapies
15

16. Preliminary safety data confirms
belinostat’s differentiation potential
Belinostat preliminary safety data shows lower incidence of grade 3-4 Adverse Events within most
common related AEs (nausea, fatigue, diarrhea, vomiting) when compared to marketed drugs
within same class and within same primary pursued PTCL indication
Istodax® Zolinza® Folotyn®
1) 2) 3) 4)
Belinostat Romidepsin Vorinostat Pralatrexate
# of patients: 68 363 74 111
% of patients with
grade 3-4 related
Adverse Events: % % % %
Nausea 1 4 4 4
Fatigue 3 11 5 6
Diarrhea 0 2 0 2
Vomiting 0 5 0 2
Please note that for this slide, the comparison data is not generated in a head-to-head study. The analysis is carried out based on
available published study data on the respective marketed drugs approved for CTCL and PTCL.
1) Source: Safety population from belinostat clinical studies: TT-30 and CLN-6, I.V.
2) Source: Prescribing information Romidepsin, FDA label 2011-09-30, approved for CTCL and PTCL, I.V.
3) Source: Olsen et al., Phase IIb Multicenter Trial of Vorinostat …, Journal of Clinical Oncology 2007; 25 (21), approved in CTCL, Oral
4) Source: Prescribing information Pralatrexate, FDA label 2011-07-01, approved for PTCL , I.V.
16

17. Overview of belinostat
clinical trials 2012
Randomized
Target Enrollment Time
Indication Study Sponsor Phase I Phase II phase II or Milestone
# status frame
pivotal
BELIEF 100- Top-line results
PTCL SPPI Completed 2012
(CLN-19) 120 NDA filing
H1
CUP CLN-17 TT 88 Completed Top-line results
2012
Solid Scientific
CLN-9 TT 92 Completed 2012
tumors publication
Results stage I
Phase I
completed H1
Solid + STS CLN-14 TT 55 LPFV stage I in
Phase II 2012
phase II
recruiting
Drug-Drug
CLN-20 SPPI/TT 39 Recruiting Top-line results 2012
interaction
SPI-1014 Recruitment
NSCLC SPPI/TT 35 Recruiting n/a
Bel completed
17

18. PTCL is an orphan indication with a high
unmet medical need ...
Key facts for PTCL PTCL characteristics
• Incidence: 15,500 new cases per annum • Peripheral T-cell lymphoma (PTCL) is a
(US, Japan, and EU27) subtype of non-Hodgkin’s lymphoma
• Prevalence: 41,000 patients (US, EU) • 10-15% of non-Hodgkin’s lymphoma
patients are PTCL patients
• Niche market: World-wide market size • Aggressive, high-grade cancer
estimated to be USD 100-130 million
• Generally with a poor prognosis and
limited treatment options
• Average age of patients with PTCL:
65 years
18

19. ... and there are promising data
from initial PTCL clinical trial
Pre-
Left
Study CLN-6 axillary Case report PTCL (monotherapy)
node
• Phase IIa trial with patients who had
refractory CTCL (28) or PTCL (25)
• Efficacy in 19 evaluable PTCL patients
‒ CR: 2, PR: 4, SD: 4
‒ Response rate: carinal
node Pre-treatment On treatment
6/19 = 32% [CI: 16-45%] -
‒ Duration of
a) Response: +268 days • 61-
•
b) Stable disease: +133 days
19

20. Pivotal PTCL study BELIEF
successfully enrolled
FDA Interactions Study facts
BELIEF
Special Protocol Assessment (SPA) • BELinostat In patients with relapsed or
• Pivotal trial BELIEF in place with an rEFractory peripheral T-cell lymphoma
ORR of at least 20%
Protocol design
Orphan drug • Open-labelled, multi-center,
• Designation granted prospective, phase IIb pivotal trial
• Enrollment of 129 patients completed
Fast track • Dosing
• Designation granted – I.V., 1000mg/m2, days 1-5 every
three weeks
NDA submission
• Planned for 2012 DMC outcome
• Positive interim analysis in March 2011
– Safety and futility – based on <5
responses in 45 evaluable patients
20

21. Update on BELIEF study (pivotal PTCL) (1)
• Registrational and pivotal phase IIb study – 129 patients enrolled
• Sponsored, conducted, and finalized by SPPI (initiated by Topotarget A/S)
• Top-line data expected to be announced by SPPI in H2 2012
• NDA submission to the FDA expected by end 2012 with estimated approval
in 2013
• Expected milestone payments from SPPI:
– Following FDA acceptance of NDA: 1 million shares of common stock in SPPI and a double-
digit million USD cash payment
– Upon FDA approval: Double-digit million USD cash payment
21

22. Update on BELIEF study (pivotal PTCL) (2)
• Following US launch, Topotarget A/S will receive double-digit royalty
payments from SPPI
• Possibility of subsequent drug approval in emerging markets for PTCL
indication (provided FDA approval) and led by Topotarget A/S
• Topotarget A/S pursues partnership opportunities in Europe,
Asia/Pacific, Latin America, and in the rest of the world
• Potential value of belinostat may exist in other liquid tumor indications
as well, e.g. MDS
22

23. Update on CUP clinical study
• Randomized, controlled study with 89 patients enrolled
• Fully conducted and sponsored by Topotarget A/S
• Announcement of top-line phase II data expected by end of H1 2012
• Based on the design and modest powering of the CUP study, the
study will not serve as a registration study
• However, an obtained PFS improvement rate of 20-40% will be viewed
as a positive trend warranting further studies in this indication
23

24. Topotarget corporate presentation
• Executive summary
• Belinostat supercharging chemotherapy
• Financial overview
24

25. Specific Action Plan to secure financial
capability for 2012 and onwards
• ‘Special Plan’ initiated in December 2011 to
best secure financial capability for belinostat:
– Direct investment efforts into finalization of PTCL
pivotal study and subsequent NDA filing with SPPI
– Finalize randomized phase II CUP study
– Continue clinical development in solid tumors and
hematological cancer
– Topotarget USA, Inc. and all Totect® related activities
divested to Apricus Biosciences, Inc.
– Hiring freeze in place and a reduction of the number
of employees in Denmark by around 40%
– Reduction of cash burn rate by approx. 15% incl. cash
preserving plan
25

26. Successful divestment of Totect and
Topotarget USA, Inc.
• Transaction completed in
December 2011
• Apricus Biosciences, Inc. acquired
the rights to Totect in North and
South America as well as
Topotarget USA, Inc.
• Improved financial implications:
– Upfront: USD 2 million
– Milestone: USD 2 million
26

27. Triggers expected to increase the value*
– a data-driven process
PTCL top-line data PTCL NDA filing
CUP top-line Ovarian and CUP Potential orphan drug FDA approval of NDA filing
data read-out designation for PTCL EU (SPPI milestone)
H1 2012 H2 2012 H1 2013
Divestment of Initiation of new belinostat SPPI milestone upon
Totect clinical trials** acceptance of NDA filing
Partner Europe
and Asia/Pac
* Timelines are illustrative only
** Preparation (i.e. contracts and regulatory)
27 27

28. Financial highlights
DKK ' 000 2011 2010
Revenue 65.598 107.826
Production costs (1.840) (5.442)
Research and development costs (54.345) (71.608)
Write down of research and development projects - (189.541)
Administrative expenses (40.765) (38.778)
Net loss from continued operations (29.012) (84.785)
Net (loss)/profit from discontinued operations (3.999) 29.095
Net loss for the year (33.011) (55.689)
Basic and diluted EPS (DKK) continued and discontinued operations (0,25) (0,42)
Dec 31, 2011 Dec 31, 2010
Cash flow from operating activities (88.847) 40.101
Cash flow from investing activities (1.919) 34.686
Cash flow from financing activities - 138
28 28

29. Outlook
• Topotarget A/S expects an estimated pre-tax loss in the range
of DKK 75-95 million for the full year financial result of 2012
• The expected net cash and cash equivalents will be around DKK
35-55 million at year-end 2012
29 29

30. Contact information
Topotarget A/S
Fruebjergvej 3
DK-2100 Copenhagen
Tel: +45 39178392
Email: enquiries@topotarget.com
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31. Q&A
31 31