Basilea initiates phase 1/2a oncology study with oral formulation of tumor checkpoint controller BAL101553

Basilea Pharmaceutica AG / Basilea initiates phase 1/2a oncology study with oral formulation of tumor checkpoint controller BAL101553 . Processed and transmitted by NASDAQ OMX Corporate Solutions. The issuer is solely responsible for the content of this announcement.

Basel, Switzerland, June 25, 2015 - Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today the start of a clinical phase 1/2a study with an oral formulation of its anti-cancer drug candidate BAL101553. BAL101553 is a microtubule-destabilizing small molecule, acting as tumor checkpoint controller (TCC) as it promotes tumor cell death through activation of an important checkpoint in cell proliferation.

The primary objective of the phase 1 study is to determine the maximum tolerated dose (MTD) in adult patients with advanced or recurrent solid tumors who have failed standard therapy or for whom no effective standard therapy is available. BAL101553 capsules will be administered once daily. A subsequent phase 2a extension of the study is planned to further evaluate the safety, tolerability and the pharmacokinetic profile of daily oral BAL101553 at the MTD, and to assess its anti-tumor activity. Furthermore, biomarkers will be explored in both the phase 1 and phase 2a parts of the study to determine their potential utility in identifying patients who are most likely to respond to treatment.

An intravenous (i.v.) formulation of BAL101553 is currently being investigated in a separate phase 2a clinical study in cancer patients with advanced solid tumors.

"Oral bioavailability of our anti-cancer compound BAL101553 may provide advantages complementary to the i.v. formulation," said Prof. Achim Kaufhold, Basilea's Chief Medical Officer. "The availability of an oral formulation of BAL101553 offers increased flexibility with regard to both single agent and drug combination treatment strategies across different tumor types and thus could potentially broaden the options for the clinical development of this anti-cancer drug candidate."

About tumor checkpoint controller BAL101553

BAL101553 is a water-soluble prodrug of Basilea's small molecule oncology drug candidate BAL27862. The prodrug is available in oral and intravenous (i.v.) formulations. The investigational drug is being developed for the treatment of diverse cancers, including tumor types unresponsive or resistant to standard therapeutics. BAL101553 showed first evidence of clinical anti-tumor activity in the phase 1 part of an ongoing phase 1/2a i.v. study in solid tumor cancer patients.[1]