Basilea Pharmaceutica Ltd. Logo
 

Corporate information

 
 
 

Basilea Pharmaceutica Ltd. is a commercial stage biopharmaceutical company, focusing on the development of products that address the medical challenges in the therapeutic areas of oncology and anti-infectives. With two commercialized drugs, the company is committed to discovering, developing and commercializing innovative pharmaceutical products to meet the medical needs of patients with serious and life-threatening conditions. Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN).

Portfolio 

Basilea has currently two products on the market.

  • Isavuconazole is an intravenous (i.v.) and oral azole antifungal and the active agent of the prodrug isavuconazonium sulfate. It received marketing authorization in Europe for the treatment of adult patients with invasive aspergillosis and for the treatment of adult patients with mucormycosis for whom amphotericin B is inappropriate.1 It is approved in the United States for patients 18 years of age and older in the treatment of invasive aspergillosis and invasive mucormycosis.2 In Switzerland, isavuconazole is approved for the treatment of adult patients with invasive aspergillosis and for the treatment of mucormycosis in adult patients who are resistant to or intolerant of amphotericin B and in adult patients with moderate to severe renal impairment.3 It is also approved in several additional countries. Isavuconazole has orphan drug designation for the approved indications in Europe and the U.S. and was designated a Qualified Infectious Disease Product (QIDP) by the U.S. Food and Drug Administration (FDA) under the Generating Antibiotics Incentives Now (GAIN) Act. Basilea has entered into several license and distribution agreements for isavuconazole in the U.S., Europe, China, Japan, Latin America, Asia-Pacific, the Middle East and North Africa region, Canada, Russia, Turkey and Israel4. Outside the U.S. and the EU, isavuconazole is currently not approved for commercial use.
  • Ceftobiprole is a cephalosporin antibiotic for intravenous administration with rapid bactericidal activity against a wide range of Gram-positive and Gram-negative bacteria, including methicillin-susceptible and resistant Staphylococcus aureus (MSSA, MRSA) and susceptible Pseudomonas spp.4 Ceftobiprole is approved in major European countries and several non-European countries for the treatment of adult patients with community-acquired pneumonia (CAP) and hospital-acquired pneumonia (HAP), excluding ventilator-associated pneumonia (VAP).4 Basilea has entered into license and distribution agreements for the brand in Europe, Latin America, China, Canada, Peru, Israel, and the Middle East and North Africa (MENA) region. Basilea is conducting two cross-supportive phase 3 studies with ceftobiprole with the goal to gain regulatory approval for the brand in the U.S. The U.S. market is an estimated 80% of the global market for branded hospital antibiotics based on value and therefore it plays a critical role in Basilea's commercial strategy for ceftobiprole. Top line results from the first study, in acute bacterial skin and skin structure infections (ABSSSI), are expected in the second half of 2019. The second study, in Staphylococcus aureus bacteremia (SAB), was initiated in August 2018 and is expected to take two to three years to complete recruitment. Both studies are conducted under Special Protocol Assessments (SPAs) agreed with the FDA. The phase 3 program for ceftobiprole is funded in part (up to USD 128 million, which is approximately 70% of the total estimated program costs) with Federal funds from the U.S. Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under Contract No. HHSO100201600002C.

Basilea's pipeline includes three early-stage oncology drug candidates:

  • Derazantinib is an investigational orally administered small molecule inhibitor of the fibroblast growth factor receptor (FGFR) family of kinases with strong activity against FGFR 1, 2, and 3. Therefore, it is called a panFGFR kinase inhibitor. FGFR kinases are key drivers of cell proliferation, differentiation and migration. FGFR alterations, e.g. gene fusions, overexpression or mutations, have been identified as potentially important therapeutic targets for various cancers, including intrahepatic cholangiocarcinoma (iCCA), bladder, breast, gastric and lung cancers.5 Current scientific literature suggests that FGFR alterations exist in a range of 5% to 30% in these cancers.6 In iCCA, FGFR2 gene fusions have been reported in 13-22% of the cases7, 8 and FGFR gene mutations have been reported in up to 5% of the cases.9 Basilea in-licensed derazantinib from ArQule Inc. in April 2018. The drug candidate has demonstrated favorable clinical data in previous clinical studies, including a biomarker-driven Phase 1/2 study in iCCA patients.9 A registrational phase 2 study in patients with FGFR2 gene fusion-expressing iCCA is ongoing. Derazantinib is currently not approved in any jurisdiction.
  • BAL101553 is a clinical stage small-molecule tumor checkpoint controller being developed as a potential therapy for diverse cancers. The drug candidate is currently in phase 1/2a clinical evaluation. In Switzerland, a phase 2a expansion study is exploring the drug in recurrent glioblastoma and platinum-resistant ovarian cancer patients using weekly 48-hour infusion. In the UK, phase 1 dose escalation is ongoing in recurrent or progressive glioblastoma patients with daily oral administration. In the U.S., an additional phase 1 study is evaluating oral BAL101553 in combination with standard radiotherapy in patients with newly-diagnosed glioblastoma which have a reduced sensitivity to standard chemotherapy. 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. 10, 11, 12 BAL101553 efficiently distributes to the brain, with anticancer activity in glioblastoma models.13, 14, 15 The active moiety BAL27862 binds the colchicine site of tubulin with distinct effects on microtubule organization,8 resulting in the activation of the "spindle assembly checkpoint" which promotes tumor cell death.9
  • BAL3833 is an orally available small-molecule oncology drug candidate. It interferes with the transmission of kinase-mediated growth and proliferation signals. If deregulated, these signaling pathways may lead to uncontrolled growth, i.e. cancer, and also to the development of resistance to current therapies. BAL3833 is called a panRAF/SRC kinase inhibitor because it not only blocks the BRAF and CRAF kinases but also inhibits the SRC kinase family. In particular, melanoma, the most aggressive type of skin cancer, is often linked to a mutated BRAF kinase. BAL3833 demonstrated activity in preclinical studies in a range of patient-derived melanoma models with intrinsic or acquired resistance to selective BRAF inhibitors, as well as in tumor models derived from colorectal, pancreatic and lung cancers associated with genetic changes resulting in activation of the RAF pathway.16 The compound originates from The Institute of Cancer Research (ICR) in London, where it was developed by scientists funded by Cancer Research UK and the Wellcome Trust. In 2018, Basilea’s partners completed a first-in-human phase 1 dose-escalation study. A maximum tolerated dose was not defined. Pre-clinical activities to explore alternative formulations are planned.

 

Footnotes

1. European Public Assessment Report (EPAR) Cresemba

2. Cresemba U.S. prescribing information

3. Full indication in: Swissmedic-approved information for healthcare professionals as of August 2017

4. U.K. Summary of Product Characteristics (SPC)

5. R. Porta, R. Borea, A. Coelho et al. FGFR a promising druggable target in cancer: Molecular biology and new drugs. Critical Reviews in Oncology/Hematology 2017 (113), 256-267

6. T. Helsten, S. Elkin, E. Arthur et al. The FGFR landscape in cancer: Analysis of 4,853 tumors by next-generation sequencing. Clinical Cancer Research 2016 (22), 259-267

7. R. P. Graham, E. G. Barr Fritcher, E. Pestova et al. Fibroblast growth factor receptor 2 translocations in intrahepatic cholangiocarcinoma. Human Pathology 2014 (45), 1630-1638

8. A. Jain, M. J. Borad, R. K. Kelley et al. Cholangiocarcinoma with FGFR genetic abberations: a unique clinical phenotype. JCO Precision Oncology 2018 (2), 1-12

9. V. Mazzaferro, B. F. El-Rayes, M. Droz dit Busset et al. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. British Journal of Cancer. Published online on November 13, 2018. https://doi.org/10.1038/s41416-018-0334-0

10. 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)

11. 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

12. 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

13. 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

14. 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

15. 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)

16. G. Saturno et al. Therapeutic efficacy of the paradox-breaking panRAF and SRC drug CCT3833/BAL3833 in KRAS­driven cancer models. American Association for Cancer Research (AACR) annual meeting 2016, abstract LB­212

History 

Basilea was founded in 2000 and went public on the SIX Swiss Exchange in 2004.


 
Info