TopoTarget Announces Encouraging Clinical Results from Phase II Trial of Topical BACECA®

TopoTarget A/S
Symbion
Fruebjergvej 3
DK 2100 Copenhagen Ø
Denmark
Tel: +45 39 17 83 92
Fax: +45 39 17 94 92
CVR-nr: 25695771
www.topotarget.com


To the OMX
Announcement No. 40-07 / Copenhagen, 29 November 2007


TopoTarget Announces Encouraging Clinical Results from Phase II Trial of
Topical BACECA® in Combination with Retinoid for the Treatment of the Skin
Cancer Basal Cell Carcinoma 

-	69% clinical and pathological remission obtained at three months after end of
16 weeks with combination treatment in Danish trial  - 

-	Clear anti-cancer activity observed in Russian study when BACECA® used alone -

Copenhagen, Denmark - 29 November 2007 - TopoTarget A/S (OMX: TOPO) announced
today results from a Phase II Danish clinical trial to evaluate the HDAC
inhibitor BACECA® as a new topical treatment for the most common skin cancer,
Basal Cell Carcinoma (BCC). The trial was performed as a double-blind,
randomised and placebo-controlled study to evaluate the efficacy and
tolerability of BACECA® in combination with the retinoid tazarotene. The
results showed a 69% clinical and pathological complete remission (CR) obtained
at three months after the end of the 16 weeks treatment period with the
combination treatment. This response rate confirmed the positive results of the
Italian pilot study with this combination, carried out in cooperation with G2M
now TopoTarget Germany AG. Furthermore, TopoTarget announced results from an
additional eight week Russian-based trial exploring the use of BACECA® in
combination with a different retinoid (Isotrex®). This trial showed clear
anti-cancer efficacy when administered alone, with a response rate of
approximately 50% with 20% histological CR. 

Trial designs and objectives
The Danish BACECA® Phase II trial enrolled 50 patients in a 2-arm trial design
as a topical treatment of superficial and nodular types of BCC in combination
with a retinoid (tazarotene) (Study arm 1: Placebo plus retinoid, study arm 2:
BACECA® plus retinoid, both for eight weeks followed by eight weeks of BACECA®
plus retinoid for both groups). This study was conducted as a double-blinded,
randomised and placebo-controlled study. The efficacy evaluation was based on
histological examination of biopsy samples, and clinical endpoints including
evaluation of affected skin area, assessment of the clinical appearance, and
photo documentation. 
Study results
After 16 weeks of treatment (eight weeks plus eight weeks), biopsy samples were
taken from areas treated and a histological CR rate of approximately 50% was
observed, thus confirming the results seen in the pilot study carried out in
Italy, where six out of ten patients obtained clinical tumour clearance after
treatment with BACECA® plus the retinoid tazarotene. As in the earlier pilot
study, tumour regressions were seen within the first eight weeks of treatment
and they continued during the following treatment. For the 37 patients seen at
the three months follow-up a 69% clinical and pathological CR rate was observed
in the group treated for 16 weeks with BACECA® compared to a clinical and
pathological CR rate of 52% for the group treated for just eight weeks with
BACECA®. 
It is of considerable interest that the clinical improvement was continuously
ongoing also after the end of treatment. Importantly, for further developments
these data strongly suggest that a most effective assessment of the full
clinical potency of BACECA® requires careful post treatment follow-up
assessments to obtain the exact response rates. 
The medication was generally well-accepted, with side effects mainly consisting
of mild conditions localised to the treatment area, such as itching, local
discomfort, peeling or reddening. 
In the Russian BCC study, also performed as a randomized, placebo controlled,
blinded study, the activity of BACECA® was tested in combination with a
different retinoid, Isotrex®. In this eight week trial, 33 patients with
superficial BCC were included in Russian study centres. The randomization
groups were BACECA® alone, BACECA® plus retinoid and retinoid alone. Supporting
the previously reported antineoplastic effect of valproic acid, BACECA® showed
clear anti-cancer efficacy when administered alone, with a response rate of
approximately 50% with 20% histological CR. Since the retinoid employed in this
study did not lead to further improvement of the clinical activity of BACECA®
this retinoid combination will not be further explored. 

As in the Danish trial, the treatment was well tolerated with only mild
localised discomfort experienced by patients. 

Principal Trial Investigator, Professor Hans Christian Wulf, at Bispebjerg
University Hospital, Copenhagen, Denmark commented, “The treatment has
generally been easy to use for the patients and with few problems. Therefore it
is encouraging to experience that the cure rate has been reasonably high
compared to other cream based treatments of basal cell carcinoma. This also
opens for exploration of its use in related diseases.” 

“These data supports our belief in valproic acid as an important new player in
the treatment of various skin disorders. In 2005 we acquired the company G2M
based on their impressive pilot results in BCC and now these data are confirmed
- this is very encouraging and we believe BACECA® represents a new treatment
opportunity for BCC patients,” said Professor Peter Buhl Jensen, CEO of
TopoTarget. 

Today's news does not change TopoTarget's full-year financial guidance for 2007.


TopoTarget A/S

	
For further information, please contact:

Dr. Peter Buhl Jensen	Telephone	+45 39 17 83 41
Chief Executive Officer	Mobile	+45 21 60 89 22

Ulla Hald Buhl 	Telephone	+45 39 17 83 92
Director IR & Communications	Mobile	+45 21 70 10 49




Background information

About BACECA®
BACECA® is a novel proprietary formulation of valproic acid as a topical
gel-based therapeutic drug. VPA targets HDAC enzymes, which are involved in
excessive cell proliferation and tumour formation. 

Valproic acid has been widely used for the treatment of epilepsy. Therefore, a
large body of evidence about the compound's systemic toxicity profile is
available. As BACECA® is applied topically (to the skin), thus, patients
undergoing therapy will be exposed to significantly lower systemic levels of
the active compound as have been studied both in vitro and in vivo. 

TopoTargets holds patent rights granted in, e.g., the US and the EU, for the
use of valproic acid for the treatment of various types of cancers, including
skin cancer. 
 
About Basal Cell Carcinoma (BCC)
Basal Cell Carcinoma (BCC) is a common form of skin cancer, arising as the name
suggests in the basal cells located at the bottom of the epidermis, the outer
layer of the skin. The malignancy is locally invasive, but rarely spreads to
distant locations. BCC mostly affects those areas of the skin exposed to the
sun, thus mainly head and neck parts. However, a fair number show up on parts
of the body such as the abdomen, leg, and scalp exposed to little or no
sunlight. BCC is the most common type of skin cancer with an estimated 5
million cases per year globally. 

About TopoTarget
TopoTarget (OMX: TOPO) is a biotech company, headquartered in Denmark and with
subsidiaries in the US, Switzerland, Germany and the UK, dedicated to finding
''Answers for Cancer'' and developing improved cancer therapies. TopoTarget is
founded and run by clinical cancer specialists and combines years of hands-on
clinical experience with in-depth understanding of the molecular mechanisms of
cancer. Focus lies on highly predictive cancer models and key cancer targets
(including HDACi, NAD+, mTOR, FasLigand and topoisomerase II inhibitors) and a
strong development foundation has been built. TopoTarget has a broad portfolio
of small molecule pre-clinical drug candidates and nine drugs (both small
molecules and protein based) are in clinical development, including both novel
anti-cancer therapeutics and new cancer indications for existing drugs.
Savene™/Totect™ was approved by EMEA in 2006 and the FDA in 2007 and is
TopoTarget's first product on the market. For more information, please refer to
www.topotarget.com. 

TopoTarget Safe Harbour Statement
This announcement may contain forward-looking statements, including statements
about our expectations of the progression of our preclinical 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 will not proceed as planned for technical, scientific or commercial
reasons or due to patient enrolment 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 expo-sure; 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.