October 12, 2020

Basilea reports pooled efficacy data for derazantinib in iCCA patients with FGFR2 gene mutations and amplifications presented at ESMO MAP Virtual Congress 2020



Basel, Switzerland, October 12, 2020

Basilea Pharmaceutica Ltd. (SIX: BSLN) today reported that its fibroblast growth factor receptor (FGFR) inhibitor, derazantinib, demonstrated antitumor efficacy in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 gene mutations or amplifications. The analysis was presented at the Molecular Analysis for Precision Oncology (MAP) Virtual Congress 2020, organized by the European Society for Medical Oncology (ESMO), which took place from 9-10 October, 2020. It is based on pooled data from 23 patients treated with derazantinib in two clinical studies1, 2 as well as from the early access3 and compassionate use programs.

The median progression free survival was 7.2 months and patients received treatment for a median of 8.2 months. This is consistent with results previously reported for derazantinib-treated iCCA patients with FGFR2 gene fusions.4, 5 Derazantinib showed a manageable safety profile with a low incidence of nail toxicity, retinal events, hand-foot syndrome and stomatitis (inflammation of the mouth).

Dr. Marc Engelhardt, Chief Medical Officer, said: “Our development strategy for derazantinib is focused on strengthening the clinical evidence on its differentiation versus other FGFR inhibitors based on its unique kinase inhibition spectrum and safety and tolerability profile. FGFR inhibitors, including derazantinib, have demonstrated clinical antitumor activity in patients with FGFR2 gene fusion-positive iCCA. However, to date there is limited clinical evidence for the benefit of FGFR inhibitors in iCCA patients with FGFR2 gene mutations and amplifications. The data presented at the MAP congress show that derazantinib is active in this group of patients and underscore the broad therapeutic potential of derazantinib in FGFR2-positive iCCA.“

ICCA is a cancer originating from the biliary system. Patients are often diagnosed with advanced or metastatic disease, that cannot be surgically removed and the prognosis for these patients is poor.

Apart from iCCA, Basilea is also exploring derazantinib in two phase 1/2 studies, as monotherapy and in combinations with other cancer treatments, in patients with advanced urothelial cancer (FIDES-02), or advanced gastric cancer (FIDES-03), with FGFR genetic aberrations.

The following e-poster was presented at the ESMO MAP Virtual Congress 2020:
Presentation #Authors/title
45PM. Droz dit Busset, W. L. Shaib, W. P. Harris, N. Damjanov, M. J. Borad, A. Vogel, J. Bridgewater, L. Sellmann, V. Dadduzio, M. Borner, J. Snider, F. Cantero, M. Saulay, S. Braun, V. Mazzaferro, M. M. Javle

Efficacy of derazantinib in intrahepatic cholangiocarcinoma patients with FGFR2 mutations or amplifications: Pooled analysis of clinical trials and early access programs.

For further information, please visit www.esmo.org/meetings/map-virtual-2020/programme

About derazantinib

Derazantinib is an investigational orally administered small-molecule FGFR inhibitor with strong activity against FGFR1, 2, and 3.6 FGFR kinases are key drivers of cell proliferation, differentiation and migration. FGFR genetic aberrations, e.g. gene fusions, mutations or amplifications, have been identified as potentially important therapeutic targets for various cancers, including intrahepatic cholangiocarcinoma (iCCA), urothelial, breast, gastric and lung cancers.7 In these cancers, FGFR genetic aberrations are found in a range of 5% to 30%.8
Derazantinib also inhibits the colony-stimulating-factor-1-receptor kinase (CSF1R).6, 9 CSF1R-mediated signaling is important for the maintenance of tumor-promoting macrophages and therefore has been identified as a potential target for anti-cancer drugs.10 Preclinical data has shown that tumor macrophage depletion through CSF1R blockade renders tumors more responsive to T-cell checkpoint immunotherapy, including approaches targeting PD-L1/PD-1.11, 12
Derazantinib has demonstrated antitumor activity and a manageable safety profile in a previous biomarker-driven phase 1/2 study in iCCA patients,4 and has received U.S. and EU orphan drug designation for iCCA. Basilea is currently conducting three clinical studies with derazantinib. The first study, FIDES-01, is a registrational phase 2 study in patients with inoperable or advanced iCCA. It comprises one cohort of patients with FGFR2 gene fusions and another cohort of patients with mutations or amplifications.2 The second study, FIDES-02, is a phase 1/2 study evaluating derazantinib alone and in combination with Roche's PD-L1-blocking immune-checkpoint inhibitor, atezolizumab, in patients with advanced urothelial cancer, including metastatic, or recurrent surgically unresectable disease, expressing FGFR genetic aberrations.13 The third study, FIDES-03, is a phase 1/2 study evaluating derazantinib alone and in combination with other cancer treatments, for instance with Roche’s PD-L1 checkpoint inhibitor, atezolizumab, in patients with advanced gastric cancer with FGFR genetic aberrations. Basilea in-licensed derazantinib from ArQule Inc, a wholly-owned subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A.

About Basilea

Basilea Pharmaceutica Ltd. is a commercial-stage biopharmaceutical company, focused on the development of products that address the medical challenges in the therapeutic areas of oncology and infectious diseases. 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). Additional information can be found at Basilea's website www.basilea.com.


This communication expressly or implicitly contains certain forward-looking statements, such as "believe", "assume", "expect", "forecast", "project", "may", "could", "might", "will" or similar expressions concerning Basilea Pharmaceutica Ltd. and its business, including with respect to the progress, timing and completion of research, development and clinical studies for product candidates. 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. Derazantinib and its uses are investigational and have not been approved by a regulatory authority for any use. Efficacy and safety have not been established. The information presented should not be construed as a recommendation for use. The relevance of findings in nonclinical/preclinical studies to humans is currently being evaluated.

For further information, please contact:

Peer Nils Schröder, PhD

Head of Corporate Communications & Investor Relations
Phone+41 61 606 1102

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


  1. ClinicalTrials.gov identifier: NCT01752920
  2. FIDES-01: ClinicalTrials.gov identifier: NCT03230318
  3. Early access program: ClinicalTrials.gov identifier: NCT04087876
  4. 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 2019 (120), 165-171. ClinicalTrials.gov identifier: NCT01752920
  5. M. Droz Dit Busset, S. Braun, B. El-Rayes et al. Annales of Oncology 2019 (30) suppl_5, v253–v324.
  6. T. G. Hall, Y. Yu, S. Eathiraj et al. Preclinical activity of ARQ 087, a novel inhibitor targeting FGFR dysregulation. PLoS ONE 2016, 11 (9), e0162594
  7. 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
  8. 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
  9. P. McSheehy, F. Bachmann, N. Forster-Gross et al. Derazantinib (DZB): A dual FGFR/CSF1R-inhibitor active in PDX-models of urothelial cancer. Molecular Cancer Therapeutics 2019 (18), 12 supplement, pp. LB-C12
  10. M. A. Cannarile, M. Weisser, W. Jacob et al. Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy. Journal for ImmunoTherapy of Cancer 2017, 5:53
  11. Y. Zhu, B. L. Knolhoff, M. A. Meyer et al. CSF1/CSF1R Blockade reprograms tumor-infiltrating macrophages and improves response to T cell checkpoint immunotherapy in pancreatic cancer models. Cancer Research 2014 (74), 5057-5069
  12. E. Peranzoni, J. Lemoine, L. Vimeux et al. Macrophages impede CD8 T cells from reaching tumor cells and limit the efficacy of anti–PD-1 treatment. Proceedings of the National Academy of Science of the United States of America 2018 (115), E4041-E4050
  13. FIDES-02: ClinicalTrials.gov identifier: NCT04045613 


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