September 06, 2016

Basilea starts continuous infusion phase 1/2a clinical study with oncology drug candidate BAL101553




Basel, Switzerland, September 6, 2016 - Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today that the first patient has been dosed in a new phase 1/2a continuous infusion study with its tumor checkpoint controller BAL101553 in patients with advanced solid cancers. Continuous infusion with portable pumps is an established mode of drug administration used for the treatment of certain cancers.

Prof. Achim Kaufhold, Basilea's Chief Medical Officer, commented: "We are excited to start this phase 1/2a study with our novel tumor checkpoint controller BAL101553. Continuous infusion could provide additional administration flexibility beyond daily oral administration and weekly 2-hour intravenous (i.v.) infusion. In cancer therapy, it is important to offer maximum administration flexibility in order to optimize treatment for specific tumor types and to synchronize treatment schedules in combination with other cancer therapies."

The open-label, multicenter study is being conducted in Switzerland. It includes adult patients with advanced solid tumors who failed standard therapy or for whom no effective standard therapy is available. Study participants receive i.v. BAL101553 administered as 48-hour continuous infusions.

About BAL101553
Basilea's small molecule oncology drug candidate BAL101553 (the prodrug of BAL27862)1 is being developed as a potential therapy for diverse cancers, including tumor types unresponsive to standard therapeutics. BAL101553 is currently undergoing clinical phase 1/2a evaluation in patients with advanced solid tumors. It has shown evidence of clinical anti-tumor activity in a phase 1/2a study with weekly 2-hour i.v. dosing, during which the maximum tolerated dose and the recommended phase 2 dose were established. In addition to the newly initiated 48-hour continuous intravenous infusion phase 1/2a study, BAL101553 is also currently being investigated in a dose-escalation phase 1/2a study with an once-daily oral formulation. In preclinical studies, the drug candidate demonstrated in-vitro and in-vivo activity against diverse treatment-resistant cancer models, including tumors refractory to conventional approved therapeutics and radiotherapy.2, 3, 4 BAL101553 efficiently distributes to tumors and to the brain, with anticancer activity in glioblastoma (brain cancer) models.5, 6 The active moiety BAL27862 binds the colchicine site of tubulin with distinct effects on microtubule organization7, resulting in the formation of the "spindle assembly checkpoint" which promotes tumor cell death.8 Basilea's approach to oncology includes the early evaluation of potential biomarkers, which are already being tested in phase 1/2a clinical studies in order to optimize dose selection and identify cancer patient groups more likely to respond.

About Basilea
Basilea Pharmaceutica Ltd. is a biopharmaceutical company developing products that address increasing resistance and non-response to current treatment options in the therapeutic areas of bacterial infections, fungal infections and cancer. The company uses the integrated research, development and commercial operations of its subsidiary Basilea Pharmaceutica International Ltd. to discover, develop and commercialize innovative pharmaceutical products to meet the medical needs of patients with serious and potentially life-threatening conditions. Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN). Additional information can be found at Basilea's website

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:

Peer Nils Schröder, PhD
Head of Corporate Communications & Investor Relations
+41 61 606 1102

This press release can be downloaded from


1       J. Pohlmann et al. BAL101553: An optimized prodrug of the microtubule destabilizer BAL27862 with superior antitumor activity. American Association for Cancer Research (AACR) annual meeting 2011, abstract 1347; Cancer Research 2011, 71 (8 Supplement)
2 A. Broggini-Tenzer et al. The novel microtubule-destabilizing drug BAL101553 (prodrug of BAL27862) sensitizes a treatment refractory tumor model to ionizing radiation. EORTC-NCI-AACR symposium 2014, abstract 202
3 G. E. Duran et al. In vitro activity of the novel tubulin active agent BAL27862 in MDR1(+) and MDR1(-) human breast and ovarian cancer variants selected for resistance to taxanes. American Association for Cancer Research (AACR) annual meeting 2010, abstract 4412
4 F. Bachmann et al. BAL101553 (prodrug of BAL27862): A unique microtubule destabilizer active against drug refractory breast cancers alone and in combination with trastuzumab. American Association for Cancer Research (AACR) annual meeting 2014, abstract 831
5 A. Schmitt-Hoffmann et al. BAL27862: a unique microtubule-targeted agent with a potential for the treatment of human brain tumors. AACR-NCI-EORTC conference 2009, abstract C233; Molecular Cancer Therapeutics 2009, 8 (12 Supplement)
6 A. C. Mladek et al. The novel tubulin-binding 'tumor checkpoint controller' BAL101553 has anti-cancer activity alone and in combination treatments across a panel of GBM patient-derived xenografts. American Association for Cancer Research (AACR) annual meeting 2016, abstract 4781
7 A. E. Prota et al. The novel microtubule-destabilizing drug BAL27862 binds to the colchicine site of tubulin with distinct effects on microtubule organization. Journal of Molecular Biology 2014 (426), 1848-1860
8 F. Bachmann et al. BAL101553 (prodrug of BAL27862): the spindle assembly checkpoint is required for anticancer activity. American Association for Cancer Research (AACR) annual meeting 2015, abstract 3789