Press Release: Basilea reports pooled efficacy data for derazantinib in iCCA patients with FGFR2 gene mutations and amplifications presented at ESMO MAP Virt...

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 studies(1, 2) as well as from the early access(3) 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

#

45P M. 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

https://www.globenewswire.com/Tracker?data=n1NJYi9lyiNmYq3qPmQHDTno2Sp2861vT5-MqDntumb-IhJaBPE2BgUO6-Q55Fgr53hGuEqkmCrblANeVW4_XnzyHS1f9i6JSl6QEwaFneKvKBTA3oWc49We3duD0kP53C2bJVCQYWOJOKtdllXi_O2ImZBZvxWmiUft-l0PUuc=

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

https://www.globenewswire.com/Tracker?data=n1NJYi9lyiNmYq3qPmQHDWgSvQnjbmc-ffmoog8c41E55a06SL6hdoZg53ltosGfuDnJyV7wj7Uj7LGNSnXpQw==

www.basilea.com.

Disclaimer

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

E-mail mailto:media_relations@basilea.com media_relations@basilea.com

mailto:investor_relations@basilea.com investor_relations@basilea.com

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www.basilea.com.

References

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

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October 12, 2020 01:15 ET (05:15 GMT)