June 04, 2013

Basilea presents first phase 1 data from its novel anti-cancer drug BAL101553 at ASCO conference




Basel, Switzerland, June 4, 2013 - Basilea Pharmaceutica Ltd. (SIX: BSLN) reported today the presentation of interim data of its oncology drug BAL101553 from its ongoing phase 1 study in patients with advanced solid tumors at the Annual Meeting of the American Society of Clinical Oncology (ASCO), held in Chicago, Illinois (USA) from May 31 to June 4.

The presented data demonstrated that intravenously administered BAL101553 was well tolerated at the doses studied thus far and provided first evidence of anti-tumor activity. Among the 18 patients treated, one patient experienced a partial response (decrease in tumor lesion size) and an additional five patients reported stable disease of which two patients showed stable disease for more than 16 weeks. The main side effects observed were nausea/vomiting and transient blood pressure elevations which were well manageable. Dose escalation continues to determine the maximum tolerated dose.

Pharmacokinetic profiles from the study indicated dose-proportionality; initial pharmacodynamic analyses showed vascular disrupting and anti-proliferative effects in post-treatment tumor biopsies.

Prof. Achim Kaufhold, Basilea's Chief Medical Officer, commented: "Resistance to currently available anti-cancer drugs remains a major challenge in the treatment of cancer patients. These interim results from our investigational drug BAL101553 are encouraging, with first evidence of anti-tumor activity in patients who failed to respond to standard treatment. In addition, the observed safety profile of BAL101553 is promising."

Dr. Heidi Lane, Head of Cancer Biology Basilea, added: "Once the maximum tolerated dose is established, the study will be extended to enlarge patient numbers and identify tumor types likely to respond to this novel anti-cancer compound. In parallel we are actively exploring novel biomarkers for patient stratification to determine which patients will potentially benefit most from treatment."

BAL101553 is a highly water-soluble pro-drug of Basilea's small molecule BAL27862, a novel anti-cancer drug targeting the intracellular microtubule network critical for tumor cell proliferation. It was previously shown that the drug has a dual mode of action directly attacking drug-refractory tumor cells as well as disrupting tumor blood supply.

Poster on BAL101553
A first-in-human (FIH) dose-escalation study of the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of intravenous BAL101553, a novel microtubule inhibitor, in adult patients with advanced solid tumors - A.H. CALVERT, M. GONZALEZ, S. GANGULI, M. NG, S. BENAFIF, M. CAPELAN, R. GOLDSTEIN, K. SHAH, C. JARVIS, M. FLYNN, M. FORSTER, S. ANDERSON, A. SCHMITT-HOFFMANN, H. LANE, M. ENGELHARDT, A.L. HANNAH, A. TZANKOV, F. BACHMANN, L. R. MOLIFE, R. KRISTELEIT; A 2566

For further information please visit chicago2013.asco.org.

About Basilea

Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland, and listed on the SIX Swiss Exchange (SIX: BSLN). Through the fully integrated research and development operations of its Swiss subsidiary Basilea Pharmaceutica International Ltd., the Company focuses on innovative pharmaceutical products in the therapeutic areas of bacterial infections, fungal infections and oncology, targeting the medical challenge of rising resistance and non-response to current treatment options.


This communication expressly or implicitly contains certain forward-looking statements concerning Basilea Pharmaceutica Ltd. and its business. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Basilea Pharmaceutica Ltd. to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd. is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

For further information, please contact:

Media RelationsInvestor Relations
Peer Nils Schröder, PhD
Head Public Relations &
Corporate Communications
+41 61 606 1102
Barbara Zink, PhD, MBA
Head Corporate Development

+41 61 606 1233

This press release can be downloaded from www.basilea.com