Cancer is the second largest cause of death worldwide with approximately 7.9 million fatalities (around 13% of all deaths) in 2007 and a projected increase of 45% from 2007 to 2030 (from 7.9 million to 11.5 million deaths) due in part to an increasing and aging global population.

Although the pharmaceutical industry has expended tremendous efforts in trying to translate the ever-increasing scientific insight and knowledge regarding the causes of cancer into novel therapeutics, the outlook for many cancer patients has not improved significantly in past decades. As such, cancer remains one of the greatest unmet medical needs of our time, where novel, well tolerated, effective treatments are desperately needed.

LNA-based drugs offer the means to specifically inhibit mRNA and microRNA targets in many different tissues and organs, providing the key to effective and well tolerated treatments. As such, the Santaris Pharma A/S LNA Drug Platform is ideally suited to develop novel drugs to target many of the genes that are known to be associated with the onset, progression and spread of various types of cancers.

EZN-2968: a novel treatment for solid tumors and lymphomas
Our partner, Enzon Pharmaceuticals, is developing EZN-2968, a specific inhibitor of HIF-1α , which is an oxygen sensitive transcription factor that regulates a host of genes involved in crucial aspects of malignant cell behavior, such as angiogenesis, cell survival, glucose metabolism and invasion. Up-regulation of HIF-1α is commonly found in a range of solid tumors, is associated with unfavorable prognosis and has been linked to poor response to radiotherapy and chemotherapy. EZN-2968 thus has the potential to be used in the treatment of a variety of human cancers and could potentially re-sensitize resistant tumors to contemporary treatments.

Data from a Phase 1 clinical study evaluating EZN-2968 in patients with different types of solid tumors and lymphomas showed that the compound was well tolerated. Moreover, early signs of clinical benefit, scored as prolonged stable disease, were observed in several patients.

EZN-3042: a novel treatment for solid tumors and lymphomas
Our partner, Enzon Pharmaceuticals, is developing EZN-3042, a specific inhibitor of Survivin, which is a cellular inhibitor of apoptosis and a key regulator of normal mitotic progression and cell division. Survivin is heavily over-expressed in many cancers but almost absent in normal adult differentiated tissue. Clinically, Survivin expression is generally associated with poor prognosis, increased cancer recurrence and resistance to therapy. EZN-3042 thus has the potential to become a broadly applicable and highly cancer-specific drug.

In preclinical studies, EZN-3042 potently inhibited Survivin expression and potentiated the antitumor activity of Taxol, an approved therapeutic drug for the treatment of cancer. EZN-3042 recently commenced a phase 1 clinical trial in patients with many different solid tumors and lymphomas. The trial will evaluate EZN-3042 as a single-agent and in combination with Taxol.