Press Release: Basilea provides corporate update

-- Key oncology clinical studies with derazantinib and lisavanbulin remain

on track

-- FDA approves protocol amendment for the phase 3 ERADICATE bacteremia

study with ceftobiprole to include a broader spectrum of severely ill

patients

-- No negative COVID-19 impact expected on global prescriptions of

Cresemba(R) and Zevtera(R)

-- Early R&D portfolio prioritization

Basel, Switzerland, May 28, 2020

Basilea Pharmaceutica Ltd. (SIX: BSLN) provided a general corporate

update today. The company does not expect a material impact on the

timelines of ongoing or planned oncology studies with the FGFR kinase

inhibitor derazantinib and the planned phase 2a biomarker driven study

with its tumor checkpoint controller, lisavanbulin, due to the

coronavirus pandemic. The impact on the ongoing ceftobiprole phase 3

study remains limited, with patient enrolment timelines potentially

extended by up to a quarter.

David Veitch, Chief Executive Officer of Basilea, said: "We have

assessed the potential impact of the coronavirus pandemic on our

business based on the information available to date. We are pleased to

report that we do not currently anticipate an impact on our key oncology

clinical studies with our most advanced compounds derazantinib and

lisavanbulin. As the global healthcare community is prioritizing

measures against coronavirus infections, we expect a limited impact of

up to a quarter on the timelines for our ceftobiprole phase 3 study. As

previously reported, we have no indication from our commercial partners

of any negative impact on global prescriptions for both Cresemba and

Zevtera, our two marketed brands. The continued strong market demand is

also reflected in the 30 percent year-on-year growth in U.S. Cresemba

sales as reported by our license partner Astellas, mid-May, with sales

of 155 million U.S. dollars for the period April 2019 to March 2020."

For Basilea's antibiotic ceftobiprole, the U.S. Food & Drug

Administration (FDA) has recently approved a protocol amendment for the

phase 3 study ERADICATE, to progress the study to the pre-planned second

cohort and extend the maximum treatment duration from four to up to six

weeks. ERADICATE explores intravenous ceftobiprole for the treatment of

patients with Staphylococcus aureus bacteremia (SAB), a type of

bacterial bloodstream infection, in comparison to intravenous daptomycin,

with or without intravenous aztreonam.(1, 2) The overall target patient

enrolment number in the study remains unchanged.

Dr. Marc Engelhardt, Chief Medical Officer of Basilea, said: "We are

very satisfied that the study progresses as planned to its next stage.

The possibility for an extended treatment duration is important as it

enables us now to expand enrolment to patients with more

difficult-to-treat infections, including those with complications such

as osteomyelitis and epidural or cerebral abscess."

In its continued effort to optimize resource allocation across its

portfolio, Basilea has taken several decisions with respect to its

earlier stage R&D portfolio. Basilea has prioritized two potential

first-in-class oncology programs, these are expected to potentially

enter pre-clinical, IND-enabling studies in the next 12 months. At the

same time, it will discontinue the development of the panRAF/SRC kinase

inhibitor BAL3833, which was developed by scientists at The Institute of

Cancer Research (ICR) in London, funded by Cancer Research UK and the

Wellcome Trust, and in-licensed by Basilea in 2015. In 2018, a

first-in-human phase 1 dose-escalation study of BAL3833 was completed by

the ICR in conjunction with The Christie and Royal Marsden NHS

Foundation Trusts and The Cancer Research UK Manchester Institute at The

University of Manchester. Basilea had been conducting pre-clinical

activities to explore alternative formulations of BAL3833. In addition,

it has decided to discontinue one other, externally sourced,

pre-clinical oncology project.

About derazantinib

Derazantinib is an investigational orally administered small-molecule

FGFR kinase inhibitor with strong activity against FGFR1, 2, and 3.(3)

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.(4) In these cancers, FGFR genetic aberrations are found in a

range of 5% to 30%.(5)

Derazantinib also inhibits the colony-stimulating-factor-1-receptor

kinase (CSF1R).(3, 6) 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.(7) Pre-clinical

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.(8, 9) Derazantinib has

demonstrated antitumor activity and a manageable safety profile in

previous clinical studies, including a biomarker-driven phase 1/2 study

in iCCA patients,(10) and has received U.S. and EU orphan drug

designation for iCCA. Basilea is currently conducting two 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.(11) 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 (Tecentriq(R) ) in patients with advanced

urothelial cancer, including metastatic, or recurrent surgically

unresectable disease, expressing FGFR genetic aberrations.(12) Basilea

in-licensed derazantinib from ArQule Inc, a wholly-owned subsidiary of

Merck & Co., Inc., Kenilworth, N.J., U.S.A.

About lisavanbulin (BAL101553)

Basilea's oncology drug candidate lisavanbulin,BAL101553, (the prodrug

of BAL27862)(13) is being developed as a potential therapy for diverse

cancers.(14, 15, 16) In pre-clinical studies, lisavanbulin demonstrated

in-vitro and in-vivo activity against diverse treatment-resistant cancer

models, including tumors refractory to conventional approved

therapeutics and radiotherapy.(17,) (18, 19) Lisavanbulin efficiently

distributes to the brain, with anticancer activity in glioblastoma

models.(20, 21, 22) In pre-clinical studies, end-binding protein 1 (EB1)

was identified as a potential response-predictive biomarker in

glioblastoma models.(22) The active moiety BAL27862 binds to the

colchicine site of tubulin, with distinct effects on microtubule

organization,(23) resulting in the activation of the "spindle assembly

checkpoint" which promotes tumor cell death.(24)

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.

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.

For further information, please contact:

Peer Nils Schröder, PhD

Head of Corporate Communications & Investor Relations

Phone +41 61 606 1102

E-mail media_relations@basilea.com

investor_relations@basilea.com

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

References

1. ClinicalTrials.gov identifier: NCT03138733. The ceftobiprole phase 3

program 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.

2. K. Hamed, M. Engelhardt, M. E. Jones et al. Ceftobiprole versus

daptomycin in Staphylococcus aureus bacteremia: a novel protocol for a

double-blind, Phase III trial. Future Microbiology 2020 (15), 35-48

3. 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

4. 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

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