Press Release: Basilea initiates targeted biomarker-driven phase 2 study with lisavanbulin in patients with brain cancer

Basel, Switzerland, September 29, 2020

Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today that it has

initiated a phase 2 expansion study with its tumor checkpoint controller,

lisavanbulin, in patients with recurrent glioblastoma multiforme (GBM),

whose tumor has tested positive for the potential response-predictive

biomarker EB1 (end-binding protein 1).(1)

Glioblastoma is the most common type of primary brain cancer and one of

the most lethal types of cancer.(2) In the open-label study, patients

will receive once daily oral lisavanbulin. To identify patients with

EB1-positive glioblastoma, a tissue screening program has been

implemented using a CE-marked immunohistochemistry clinical trial assay

developed for the lisavanbulin program.

Dr. Marc Engelhardt, Chief Medical Officer, said: "The initiation of the

phase 2 study is an important step for us to validate our hypothesis

that lisavanbulin may be developed for a targeted patient population

based on a patient-selection biomarker. We expect interim results in the

first half of 2021 and the outcome of this study will define the next

development steps for lisavanbulin, including potentially expanding into

other tumor types using a biomarker-driven approach."

EB1 was selected by Basilea as a potential response-predictive biomarker

for lisavanbulin based on preclinical studies in glioblastoma models and

initial clinical signals from earlier clinical studies. One glioblastoma

patient in the phase 1 portion of the current study, whose tumor tissue

was strongly positive for EB1, was reported as an exceptional

long-lasting responder.(3) This patient continues on treatment for more

than two years now and shows a more than 80% area reduction of the brain

tumor.

About lisavanbulin (BAL101553)

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

of BAL27862)(4) is being developed as a potential therapy for diverse

cancers.(1, 5, 6) In preclinical studies, lisavanbulin demonstrated

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

models, including tumors refractory to conventional approved

therapeutics and radiotherapy.(7,) (8, 9) Lisavanbulin efficiently

distributes to the brain, with anticancer activity in glioblastoma

models.(10, 11, 12) In preclinical studies, end-binding protein 1 (EB1)

was identified as a potential response-predictive biomarker in

glioblastoma models.(12) The active moiety BAL27862 binds to the

colchicine site of tubulin, with distinct effects on microtubule

organization,(13) resulting in the activation of the "spindle assembly

checkpoint" which promotes tumor cell death.(14)

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: NCT02490800

2. B. M. Alexander, T. F. Cloughesy. Adult Glioblastoma. Journal of Clinical

Oncology 2017 (35), 2402-2409

3. J. S. Lopez, R. Kristeleit, R. Rulach et al. Phase 1/2a study of once

daily oral BAL101553, a novel tumor checkpoint controller (TCC), in adult

patients with progressive or recurrent glioblastoma (GBM) or high-grade

glioma. Journal of Clinical Oncology 2019, 37, 15 supplement, 2025

4. J. Pohlmann, F. Bachmann, A. Schmitt-Hoffmann 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. ClinicalTrials.gov identifier: NCT03250299

6. ClinicalTrials.gov identifier: NCT02895360

7. A. Sharmq, A. Broggini-Tenzer, V. Vuong et al. The novel microtubule

targeting agent BAL101553 in combination with radiotherapy in

treatment-refractory tumor models. Radiotherapy Oncology 2017 (124),

433-438

8. G. E. Duran, H. Lane, F. Bachmann 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; Cancer Research 2010, 70 (8 supplement)

9. F. Bachmann, K. Burger, G. E. Duran 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; Cancer Research 2014, 74 (19 supplement)

10. A. Schmitt-Hoffmann, D. Klauer, K. Gebhardt 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)

11. A. C. Mladek, J. L. Pokorny, H. Lane 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; Cancer Research 2016, 76 (14 supplement)

12. R. Bergès, A. Tchoghandjian, S. Honoré 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

13. A. E. Prota, F. Danel, F. Bachmann 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

14. F. Bachmann, K. Burger, H. Lane. BAL101553 (prodrug of BAL27862): the

spindle assembly checkpoint is required for anticancer activity. American

Association for Cancer Research (AACR) annual meeting 2015, abstract

3789; Cancer Research 2015, 75 (15 supplement)

Attachment

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(END) Dow Jones Newswires

September 29, 2020 01:15 ET (05:15 GMT)