December 02, 2016

Basilea expands oncology drug candidate BAL101553 clinical phase 1/2a oral study to include glioblastoma patients




Basel, Switzerland, December 02, 2016 - Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today the expansion of its ongoing oncology drug candidate BAL101553 clinical phase 1/2a oral formulation study. The first patient has been dosed in an additional arm containing adult patients with recurrent or progressive glioblastoma (brain cancer) after prior radiotherapy with or without chemotherapy.

Prof. Achim Kaufhold, Basilea's Chief Medical Officer, commented: "We are excited to explore BAL101553 in a separate glioblastoma study arm to our ongoing phase 1/2a clinical study. Glioblastoma is a cancer indication associated with high medical need. There are limited treatment options for glioblastoma patients due in part to the challenge of getting medication into the brain through the blood brain barrier. BAL101553 has been shown to enter the brain and has demonstrated anticancer activity with oral dosing in various preclinical models of glioblastoma, including models refractory to or with reduced sensitivity to standard therapies."

In addition to the activity in glioblastoma tumor lines, BAL101553 was shown to have potent anticancer activity against glioblastoma stem-like cells in a pre-clinical model as reported in a recent publication co-authored by Basilea and the research group of Prof. Diane Braguer of Aix-Marseille University, France. Tumor stem-like cells contribute to glioblastoma regrowth as well as brain invasion, a phenomenon which also occurs in the pre-clinical model used. The publication further reported the observation that BAL101553 promoted the loss of stem-cell properties. These published data further support the potential of BAL101553 to target glioblastoma, a tumor often associated with poor prognosis for patients.1

Glioblastoma is the most common primary brain tumor and one of the most lethal types of cancer. The incidence of glioblastoma is approximately 3 patients per 100,000 in the United States.2 Median survival of about 15 months from diagnosis has been reported for adult glioblastoma patients receiving standard-of-care treatment,3 with a 5-year survival rate of 5%.2

The ongoing phase 1/2a study includes patients with advanced or recurrent solid tumors who have failed standard therapy or for whom no effective standard therapy was available. Phase 1 dose escalation to determine the maximum tolerated dose (MTD) of daily oral dosing is currently ongoing. A subsequent phase 2a extension of the study is planned to further evaluate the safety, tolerability and the pharmacokinetic profile of oral BAL101553 at the MTD, and to assess its anti-tumor activity. Furthermore, biomarkers are assessed in both the phase 1 and phase 2a parts of the study to determine their utility in identifying patients who are most likely to respond to treatment, including biomarkers with potential relevance to glioblastoma.

About BAL101553

Basilea's small molecule oncology drug candidate BAL101553 (the prodrug of BAL27862)4 is being developed as a potential therapy for diverse cancers. BAL101553 is currently undergoing clinical phase 1/2a evaluation (oral and continuous infusion) in patients with advanced solid tumors. 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.5, 6, 7 BAL101553 efficiently distributes to the brain, with anticancer activity in glioblastoma (brain cancer) models.1, 8, 9 The active moiety BAL27862 binds the colchicine site of tubulin with distinct effects on microtubule organization,10 resulting in the formation of the "spindle assembly checkpoint" which promotes tumor cell death.11

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

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1 R. Bergès et al. The novel tubulin-binding checkpoint activator BAL101553 inhibits EB1-dependent migration and invasion and promotes differentiation of glioblastoma stem-like cells. Molecular Cancer Therapeutics 2016 (15), 2740-2749. Published November 2016
2 Q.  T. Ostrom et al. CBTRUS statistical report: primary brain and central nervous system tumors diagnosed in the United States in 2007-2011. Neuro-Oncology 2014 (16, Suppl 4), iv1-iv63
3 R. Stupp et al. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. New England Journal of Medicine 2005 (352), 987-996
4 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)
5 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
6 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
7 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
8 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)
9 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
10 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 
11 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