ProInvestor Life Science SeminarAnders Vadsholt (CFO) and Lars Damstrup (Medical Director)

Topotarget A/S – March 27, 2012

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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.

Safe harbor statement

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• Executive summary• Belinostat supercharging chemotherapy• Financial overview

Topotarget corporate presentation

Topotarget at a glance (1)

• 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

An international Scandinavian-based biopharmaceutical companydedicated to develop and market cancer therapies

Listing NASDAQ OMX Copenhagen

Symbol TOPO

Market capitalization(as of March 22, 2012) DKK 400m

No. of shares (as of March 22, 2012) 132,652,050

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Shareholder Ownership The 10 largest shareholders combined + 30%

HealthCap funds + 13%

Avanza Pension + 6%

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

* Estimated

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Denmark40%

Sweden22%

Switzerland10%

UK8%

Other20%

Geographic split of shareholders*

Topotarget is making a difference to cancer patients …

TOPOTARGET – STRONG INVESTMENT CASE

Proven track record only 7

years from idea to launch:

Totect®

Savene®

Track record

Novel cancer drug target:

HDACi

Lead development

candidate

Strong partnerships:

Spectrum Pharmaceuticals,

Inc.

National Cancer Institute

Rigshospitalet (DK)

Partnerships

Solid tumor

Hematological diseases

Solid commercialpotential

Unmet market need

Promising preliminary

clinical efficacy

Robust safety

Strong IP

Large database

Mono- and combination

therapy

Strong belinostat

profile

Creating shareholder value

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… with a clear-cut focus on belinostat

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2010 2012

Take-off 2011

Early-stage acquisitions

Savene®/Totect®

Unfocused early-stage pipeline

Discovery platform

High burn rate, 100+ employees

Several cancer targets

Belinostat

• Optimized organization

• Prioritized late-stage pipeline

• Experienced development team

• US partner

• Research facility closed down

• Enhancement of BoD and management team

• Establishment of GOAB* and disease-specific advisory boards

• Completion of CSRs**

• PTCL***

• Cancer of unknown primary

• MDS/AML***

• Non-small cell lung cancer

• Bladder cancer

• Hepatocellular cancer

• Ovarian cancer

*) Global Oncology Advisory Board**) Clinical Study Report***) Peripheral T-cell lymphoma, myelodys-

plastic syndromes, acute myeloid leukemia

Belinostat

NorthAmerica

East Asia

Canada

USA

South America

Africa

Europe

North Asia

China

IndiaMexico

Australia

China: First right of negotiationto Spectrum Pharmaceuticals, Inc.

Partnerships are key to a successfulcommercialization of belinostat

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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

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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

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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

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• Executive summary• Belinostat supercharging chemotherapy• Financial overview

Topotarget corporate presentation

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

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Belinostat is supercharging chemotherapy

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HDACi’s work in synergy with chemotherapy by supercharging the effects of cytostatics

Belinostat in combination with chemotherapy increases efficacy with only a minor or moderate increase in toxicity

Belinostat is a novel pan-HDACi with the ability of regulation of multiple class I and II HDAC’s

Cancer cells can over time develop resistance to the given chemotherapy – a resistance that HDACi’s may overcome

Belinostat has a compelling clinical profile

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ABILITY TO COMBINECombined with main established chemotherapies will lead to a maximized

commercial potential

ENCOURAGING SAFETY PROFILEShown 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 DATAPreliminary clinical efficacy in solid and hematological malignancies

Synergistic pre-clinical effect with established therapies

FLEXIBLE ADMINISTRATIONOption of multiple administration and formulation modes

(IV, CIV, and oral)

Preliminary safety data confirms belinostat’s differentiation potential

Istodax® Zolinza® Folotyn®

Belinostat 1)

Romidepsin2)

Vorinostat3)

Pralatrexate4)

# 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

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

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, Oral4) Source: Prescribing information Pralatrexate, FDA label 2011-07-01, approved for PTCL , I.V.

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.

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Overview of belinostat clinical trials 2012

Indication Study Sponsor Phase I Phase IIRandomized phase II or

pivotalTarget

#Enrollment

status Milestone Time frame

PTCL BELIEF (CLN-19) SPPI 100-

120 Completed Top-line resultsNDA filing 2012

CUP CLN-17 TT 88 Completed Top-line results H1 2012

Solid tumors CLN-9 TT 92 Completed Scientific

publication 2012

Solid + STS CLN-14 TT 55Phase I completedPhase II recruiting

Results stage I

LPFV stage I in phase II

H1 2012

Drug-Drug interaction CLN-20 SPPI/TT 39 Recruiting Top-line results 2012

NSCLC SPI-1014Bel SPPI/TT 35 Recruiting Recruitment

completed n/a

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PTCL is an orphan indication with a high unmet medical need ...

• Incidence: 15,500 new cases per annum(US, Japan, and EU27)

• Prevalence: 41,000 patients (US, EU)

• Niche market: World-wide market size estimated to be USD 100-130 million

• Peripheral T-cell lymphoma (PTCL) is a subtype of non-Hodgkin’s lymphoma

• 10-15% of non-Hodgkin’s lymphoma patients are PTCL patients

• Aggressive, high-grade cancer

• Generally with a poor prognosis and limited treatment options

• Average age of patients with PTCL: 65 years

PTCL characteristics Key facts for PTCL

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... and there are promising datafrom initial PTCL clinical trial

Pre-

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Study CLN-6• 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:

6/19 = 32% [CI: 16-45%]‒ Duration of

a) Response: +268 daysb) Stable disease: +133 days

axillarynode

carinalnode

• 61-•

Pre-treatment On treatment

Left

Case report PTCL (monotherapy)

-

Pivotal PTCL study BELIEFsuccessfully enrolled

Special Protocol Assessment (SPA) Pivotal trial BELIEF in place with an ORR

of at least 20%

Orphan drug Designation granted

Fast trackDesignation granted

NDA submissionPlanned for 2012

FDA Interactions

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BELIEF• BELinostat In patients with relapsed or

rEFractory peripheral T-cell lymphoma

Protocol design Open-labelled, multi-center, prospective,

phase IIb pivotal trialEnrollment of 129 patients completedDosing– I.V., 1000mg/m2, days 1-5 every

three weeks

DMC outcomePositive interim analysis in March 2011– Safety and futility – based on <5

responses in 45 evaluable patients

Study facts

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

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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

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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

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• Executive summary• Belinostat supercharging chemotherapy• Financial overview

Topotarget corporate presentation

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

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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

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H1 2012

H2 2012

H1 2013

CUP top-line data

Ovarian and CUP read-out

FDA approval of NDA filing (SPPI milestone)

Divestment of Totect

Partner Europe and Asia/Pac

PTCL top-line data

Initiation of new belinostat clinical trials**

PTCL NDA filing

Triggers expected to increase the value* – a data-driven process

Potential orphan drug designation for PTCL EU

* Timelines are illustrative only** Preparation (i.e. contracts and regulatory)

SPPI milestone upon acceptance of NDA filing

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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

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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

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Contact information

Topotarget A/SFruebjergvej 3

DK-2100 Copenhagen

Tel: +45 39178392Email: enquiries@topotarget.com

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Q&A

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