Topotarget A/S Rodman & Renshaw Annual Global Investment Conference 2013 Safe harbor statement

This announcement may contain forward-looking statements, including statements about Topotarget A/S’ expectations to the progression of Topotarget A/S’ clinical pipeline and with respect to cash burn guidance. Such statements are subject to risks and uncertainties of which many are outside the control of Topotarget A/S, and which could cause actual results to differ materially from those described. Topotarget A/S disclaims 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 Danish law.

2 Topotarget at a glance

A Scandinavian-based biopharmaceutical company dedicated to develop and market therapies • Situated in , with ~14 employees and a lean cost structure • Focused on the development and commercialization of , a molecular targeted cancer therapy

NASDAQ OMX Listing Copenhagen 22% Symbol TOPO Denmark 45% Market UK capitalization EUR 48 million Sweden (as of Aug 28, 2013) 20% ROW

No. of shares 143,317,114 8% 4%

3 Building a strong commercial profile for belinostat

Large sales potential in solid First registration filing tumors (CRC, bladder, expected Q4 2013 ovarian, NSCLC)

Partnership with Spectrum Outstanding safety profile. Pharmaceuticals, Inc. Belinostat tested in more Pursuing partnerships in EU than 1,100 patients and Asia-Pac

Belinostat – a pan-HDACi - Orphan indications (PTCL, effective in cancer patients thymoma, MDS, HCC, STS) both as mono-/combo therapy

Strong synergies with gold standard

HDACi: inhibitor STS: Soft tissue sarcoma PTCL: Peripheral T-cell NSCLC: Non-small cell 4 MDS: Myelodysplastic syndrome CRC: Colorectal cancer HCC: Hepatocellular carcinoma What is a histone deacetylase inhibitor?

5 Strong clinical profile

Flexible administration Ability to combine Combination with vital, Enables various established chemotherapy administration and treatments entails a formulation forms maximized commercial (IV, CIV, and oral) potential

Promising effect data Excellent safety profile Preliminary clinical effect in hematological and solid Demonstrated to be well-tolerated cancer types. in clinical use in more than 1,100 Synergistic, preclinical effect patients with excellent safety with established therapies (including heart toxicity profile and minimal bone marrow toxicity)

6 Clinical trials

Target Enrollment Indication Study Sponsor Phase I Phase II Pivotal Milestone Time frame # status

BELIEF PTCL SPPI 100 Completed NDA filing Q4 2013 (CLN-19)

SPI-1014 Recruitment NSCLC SPPI/TT 35 Recruiting n/a Bel completed

BelCHOP Recruitment PTCL* (SPI-BEL- SPPI/TT 28 Recruiting 2014 completed 12-104)

Drug-drug CLN-20 SPPI/TT 39 Recruiting Top-line results 2013 interaction

*Part one of confirmatory study 7 BELIEF study*

• ORR 26% in all PTCL patients • ORR 28 % in patients with platelet counts >100,000/μL • Median overall duration of respons of 13.6 months according to International Working Group criteria • Favorable safety profile • >98% dose intensity → new combination therapy

Belinostat’s special strengths: Strong safety profile Possibility of combination treatment

* Data presentet at ASCO 2013 8 Value inflection*

Submission of NDA FDA approval of NDA Initiation of new studies

Q4 2013 2014

*Estimated timelines 9 Partnerships are key to a successful commercialization of belinostat

Asia Canada US China

Mexico India

Africa

South America

Australia

China: First right of negotiation to Spectrum Pharmaceuticals, Inc.

10 Financial highlights – H1 2013

• Topotarget recognized revenues of DKK 0.8 million during the period (DKK 2.2 million in the same period in 2012) • Research and development costs were DKK 13.4 million during the period (DKK 25.9 million in the same period in 2012) • Administration expenses were DKK 9.2 million during the period (DKK 17.7 million in the same period in 2012) • The net financials were a net loss of DKK 0.6 million during the period (net income of DKK 0.7 million in the same period in 2012) • The net loss from continued operations before tax for the period was DKK 23.0 million (net loss of DKK 41.3 million for the same period) • The Group’s net cash and cash equivalents as of June 30, 2013 totaled DKK 43.3 million (DKK 41.5 million at year-end 2012)

11 Belinostat and the future

• Submission of NDA for belinostat in PTCL • Milestone payments from Spectrum Pharmaceuticals • Confirmatory phase III study of PTCL → potential entry into Europe • Explore possible (orphan drug) indications for belinostat

12 Q&A Contact information

Topotarget A/S Fruebjergvej 3 DK-2100 Copenhagen

Tel: +45 3917 8392 Fax: +45 3917 9492 Email: [email protected] or [email protected]

Company website: www.topotarget.com

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