Pharmexa will start phase II trial in liver cancer with GV1001

May 31, 2006

Summary: The application to initiate the Heptovax phase II trial has now been submitted. The trial is planned to start in the third quarter of 2006 and will include up to 41 liver cancer patients.
The Heptovax trial is a phase II, open-label trial evaluating the safety and efficacy of GV1001 in advanced hepatocellular carcinoma (“HCC” or “liver cancer”). The trial will enrol patients from 3 centres in Spain, France and Germany. The application has been submitted in France and will be submitted in Spain shortly.

Dr. Jordi Bruix from the Liver Unit at Hospital Clinic, Barcelona, investigator in the Heptovax study says: “The use of new agents such as GV1001 represents interesting pathways for developing better treatments of liver cancer. If GV1001 lives up to its promise, it may become a cornerstone in the future treatment of this dreaded disease.”

Professor Michel Beaugrand from Bondy, Paris, investigator in the Heptovax study says: “GV1001 is an exciting new option for the treatment of liver cancer. Its mode of action is new and it appears to be without significant side effects.”

Trial design
Up to 41 patients with advanced stage liver cancer will receive a single pre-treatment dose of the chemotherapeutic drug cyclophosphamide three days prior to the start of immunotherapy, followed by doses of GV1001 plus GM-CSF three times in the first week, and once weekly in week 2,3,4 and 6. Thereafter, GV1001 plus GM-CSF will be given once a month. All patients will be treated for a minimum of 6 months unless they show symptomatic progression, in which case patients will be discontinued from the trial.

The primary endpoint of the trial is efficacy, measured by objective tumor response (modified RECIST). Secondary endpoints include the safety and immunogenecity of the vaccine. Approximately half of the patients with advance stage liver cancer die within a year and survival benefits in the trial will also be measured.

The trial will stop 6 months after inclusion of the last patient. Accordingly the observation time for patients recruited late in the trial may be truncated, i.e. at 6 months in order to be able to report the results of the trial in a reasonable timeframe. Depending on the speed at which patients can be recruited, results from the trial will therefore be available late 2007/early 2008. If the results are positive, Pharmexa plan to initiate a pivotal phase III trial of GV1001 in liver cancer.

Liver cancer represents a large unmet need
HCC is the fifth most common cancer in the world. More than 600,000 new cases of hepatocellular carcinoma occur worldwide each year with almost as many deaths. In Europe alone, more than 50,000 new cases occur each year. The incidence of HCC, however,

varies a great deal between different countries and regions, occurring significantly more often in Japan than in Europe. Moreover, accumulating evidence indicates that the incidence of liver cancer is rising. HCC is twice as common in men as in women and has a strong association with hepatitis B and hepatitis C infection as well as with cirrhosis. Early and medium stage liver cancers are sub-optimally treated with surgery, radiofrequency ablation and chemoembolization while no treatment options really exist for the disease in its advanced stage. Current chemotherapy treatments are limited by the site of the cancer - the cirrhotic liver - because of dose limiting liver toxicity concerns with these agents.

GV1001: A cancer vaccine targeting telomerase
GV1001 is a peptide vaccine that activates the immune system - primarily the immune system’s T-cells - to recognize and kill cancer cells. GV1001 targets an enzyme called telomerase. Telomerase is seldom found in normal cell types but is over expressed in most cancer cells. In scientific circles, telomerase activity is considered a key factor in the process whereby cancer cells lose their normal mortality, which is a common feature for all cancers. In theory, GV1001 could therefore turn out to be a universal cancer vaccine and Pharmexa’s development strategy for GV1001 reflects this.

In addition to the Heptovax phase II trial, Pharmexa has started phase III trials with GV1001 in pancreatic cancer based on promising data in this indication. More than 1,600 patients are expected to be enrolled in this program.


Hørsholm, May 31, 2006


Jakob Schmidt
Chief Executive Officer


Additional information: Jakob Schmidt, Chief Executive Officer, telephone +45 4516 2525

Note to editors: Pharmexa A/S is a leading company in the field of active immunotherapy and vaccines for the treatment of cancer, serious chronic and infectious diseases. Pharmexa’s proprietary technology platforms are broadly applicable, allowing the company to address critical targets in cancer, rheumatoid arthritis, bone degeneration and Alzheimer’s disease, as well as serious infectious diseases such as HIV, influenza, hepatitis and malaria. Its leading programs are GV1001, a peptide vaccine that has entered phase III trials in pancreatic cancer, HER-2 AutoVac Protein, a recombinant protein vaccine in phase II against breast cancer, and HIV and hepatitis vaccines in phase I/II. Collaborative agreements include H. Lundbeck, Innogenetics, IDM Pharma and Bavarian Nordic. With operations in Denmark, Norway and USA, Pharmexa employs approximately 100 people and is listed on the Copenhagen Stock Exchange under the trading symbol PHARMX.

Contact Information

Jakob Schmidt

Chief Executive Officer

Pharmexa A/S

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