FDA and EMEA Grant Orphan Drug Designation for Antisense Pharma's Investigational Drug Trabedersen in Pancreatic Carcinoma

Orphan Drug Designation Ensures Market Exclusivity for Seven to Ten Years
Following Market Approval

REGENSBURG, Germany--(Business Wire)--
The biopharmaceutical company Antisense Pharma GmbH has announced today that it
has received orphan drug designation from both the European Medicines Agency
EMEA and the US Food and Drug Administration FDA for its investigational drug
trabedersen in the treatment of pancreatic carcinoma. Trabedersen has already
been granted orphan drug designation by both authorities in the treatment of
high-grade gliomas in 2002. This underlines the high potential of trabedersen to
treat various aggressive tumors.

Orphan drug designation can be applied for if the disease is life-threatening or
chronically debilitating and affects not more than five in 10,000 persons in the
European Community (equals around 250,000 people) and fewer than 200,000 people
in the United States, respectively, in case that no other satisfactory therapy
exists or the medicinal product is expected to provide significant benefit over
existing therapies.

The orphan drug designation is meant to encourage pharmaceutical companies to
develop drugs for diseases that meet the above criteria by providing them
scientific advice, reduction or waiver of registration fees and market
exclusivity in addition to patent protection.

This is another important milestone in the development of trabedersen, a
first-in-class, targeted compound based on antisense technology for the
treatment of various aggressive tumors.

Highly promising preliminary efficacy data
In an ongoing clinical Phase I/II study, trabedersen has shown a good safety and
tolerability profile and encouraging survival data in patients with advanced
pancreatic carcinoma. "The preliminary clinical data are quite impressive"
states the Committee on Orphan Medicinal Products (COMP) of the EMEA in its
report.

23 patients received single agent trabedersen intravenously as second-, third-,
or fourth-line treatment either in a 7-day on/7-day off or 4-day on/10-day off
schedule.

Median overall survival (mOS) for patients in the first schedule was 6.8 months
(status Aug 2009). Moreover, one patient with recurrent advanced pancreatic
cancer (after surgical resection and three chemotherapies) and liver metastases
had a complete response and is still alive 45.6 months after receiving
trabedersen therapy (as of Feb 2009).

The current mOS for pancreatic carcinoma patients in the first cohort of five
patients in the second schedule is 13.4 months (as of Aug 2009). One patient is
still alive 19 months after start of study treatment (as of April 2009).

In the same study good safety, tolerability and encouraging first efficacy data
for trabedersen was observed also in patients with advanced malignant melanoma
or colorectal carcinoma. Of five malignant melanoma patients treated with
trabedersen, one from the first schedule showed stable disease and lived for
13.8 months.

Incentives for development and competitive advantages
Orphan drugs generally follow the same regulatory development path as any other
pharmaceutical product. However, incentives such as scientific advice and
reduction or waiver of registration fees may be given in an effort to maintain
development momentum. In addition, Antisense Pharma may sell trabedersen without
competition for seven years in the US and for ten years in the EU following
market approval, in respect of a medicinal product containing a similar active
substance for the same indication; unless a similar product would demonstrate
superior therapeutic benefit.

"We are delighted to have received the orphan drug status from the EMEA and FDA
for the treatment of pancreatic carcinoma. This further accelerates our efforts
to make trabdersen available to those who need it as quickly as possible" says
Dr. Karl-Hermann Schlingensiepen, Chief Executive Officer of Antisense Pharma.
"Pancreatic carcinoma is one of the most aggressive and devastating cancers.
Despite various therapeutic approaches including surgery, radio- and
chemotherapy, the prognosis for the patients remains poor. Based on the results
of several clinical studies, we expect trabedersen to significantly improve the
therapeutic outcome not only of patients with pancreatic carcinoma but also of
patients suffering from high-grade gliomas, malignant melanoma or colorectal
carcinoma. Indeed, with its unique mode of action, we believe that trabedersen
has the potential to lead to a paradigm shift towards tackling malignant tumors
at their roots while providing a better quality of life for patients."

Additional Information

About pancreatic carcinoma
Pancreatic carcinoma is one of the most aggressive cancers with high unmet
medical need. It has a dismal prognosis, with one of the highest mortality
rates:

Worldwide, pancreatic cancer causes 227,000 deaths annually and is the eighth
most common cause of death from cancer.1

In Europe, cancer of the pancreas is the 10th most frequent cancer, accounting
for about 65,000 deaths each year.2 European data show that the incidence rate
of pancreatic cancer is approximately 5 to 9 (female/male) per 100,000 of the
population per year.2

The American Cancer Society estimated for the US that of about 1.5 million new
cases of cancer diagnosed in 2009, 44,380 people of both men and women will have
pancreatic cancer and that 33,740 would die of the disease, making this type of
cancer the fourth leading cause of cancer death in the US.3

Current therapies comprise surgery, radiation and/or chemotherapy. Despite
recent advances, the prognosis for these patients is still poor, with a high
proportion dying within one year after initial diagnosis.

How trabedersen works
Trabedersen is a first-in-class, targeted, antisense compound (a
phosphorothioate oligodeoxynucleotide) designed to selectively downregulate the
production of a protein known as transforming growth factor-beta 2 (TGF-β2) at
the translational level.4,5
Various aggressive tumors such as high-grade gliomas, advanced pancreatic
cancer, malignant melanoma and advanced colorectal cancer cells produce an
excessive amount of TGF-β2, which plays a critical role in tumor progression
(proliferation, angiogenesis and metastasis) and acts as a shield that protects
the tumor from the body`s immune system.4,5,6
By inhibiting TGF-β2, trabedersen has multiple antitumoral effects: it
hinders tumor progression, angiogenesis and metastasis.4,7 In addition,
trabedersen restores the body`s immune system, by breaking down the protective
shield so that the immune system can recognize and destroy the tumor cells.

Targeted therapies drive market growth
Unlike non-specific therapies, e.g. chemotherapy or radiotherapy, targeted
therapies act specifically at the molecular roots of the disease. Commanding up
to 80% of the growing oncology market, the targeted therapies like trabedersen
substantially drive the growth of the pharmaceutical market8. A marketing
authorization would make trabedersen the first TGF-beta targeting drug for the
treatment of cancer.

Clinical studies

For more information on the clinical Phase I/II study in advanced pancreatic
carcinoma, malignant melanoma or colorectal carcinoma please visit the website
www.krebsstudien.info (only in German).

For more information on the clinical Phase III SAPPHIRE trial in recurrent or
refractory anaplastic astrocytoma please visit the website www.anticancer.de.

About Antisense Pharma GmbH
Antisense Pharma is a biopharmaceutical company located in Regensburg, Germany.
The company focuses on targeted gene silencing therapies for malignant tumors
and is dedicated to discovering and developing drugs based on antisense
technology for worldwide commercialization. The medications specifically block
the synthesis of key cancer proteins. Antisense Pharma has currently clinical
trials running that involve patients with brain tumors, advanced pancreatic
carcinoma, malignant melanoma and colorectal carcinoma. Therapies for other
indications are under preclinical development. The company has been honored with
the Bavarian Innovation Award and the German Founder`s Award and was awarded one
of the 100 most innovative medium-sized companies in Germany in 2009.

References

1. Parkin DM (2005) CA Cancer. J Clin 55(2):74-108
2. Cascinu S et al. (2009) Pancreatic Cancer ESMO Clinical Recommendations. Ann
Oncol 20 (Suppl. 4): iv37-iv40
3. American Cancer Society. http://www.cancer.org last accessed 08/2009.
4. Schlingensiepen KH et al. (2006) Cytokine Growth Factor Rev 17(1-2):129-139
5. Tsamandas, AC, Kardamakis, D et al. (2004) The potential role of TGFbeta1,
TGFbeta2 and TGFbeta3 protein expression in colorectal carcinomas. Correlation
with classic histopathologic factors and patient survival. Strahlenther Onkol
180(4):201-8
6. Kouvidou, C, Latoufis, C et al. (2006) Expression of Smad4 and TGF-beta2 in
colorectal carcinoma. Anticancer Res 26(4B):2901-7
7. Schlingensiepen R et al. (2005) Oligonucleotides 15(2):94-104
8. IMS Health

Antisense Pharma GmbH
Dr. Alexis Katechakis / Public Relations
Phone: +49 (0) 941 920 13 - 104
Fax: +49 (0) 941 920 13 - 29
E-Mail: pr@antisense-pharma.com
Web: www.antisense-pharma.com
or
Edelman
Annie Tiranti
Phone: +44 (0) 203 047 2103
E-Mail: Annie.Tiranti@edelman.com

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