Basel, Switzerland, June 07, 2021 
 
   Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today that data on the 
prevalence of end-binding protein 1 (EB1) in glioblastoma and other 
tumor types is being presented at the American Society of Clinical 
Oncology (ASCO) Annual Meeting that takes place online from June 4 to 8, 
2021. 
 
   EB1 plays a pivotal role in the regulation of microtubule dynamics 
during cell division and has been shown to interact with 
microtubule-targeting agents, such as lisavanbulin, inhibiting tumor 
growth.(1) EB1 has been identified as a response-predictive biomarker 
for Basilea's tumor checkpoint controller lisavanbulin in pre-clinical 
glioblastoma models.(2) In the previously reported phase 1 portion of 
the ongoing phase 1/2 clinical study, long-lasting clinical benefit was 
observed in two patients with recurrent glioblastoma whose tumor tissues 
show EB1-positive staining. Both patients are ongoing in the study for 
more than 2 years.(3) 
 
   The prevalence assessment of EB1-positivity or strong EB1-staining using 
immunohistochemistry methods presented at ASCO was based on 565 patient 
tissue samples from 14 different tumor types, including more than 100 
glioblastoma samples. Approximately 5% of glioblastoma tissue samples 
were found to be EB1-positive. The strongest expression of EB1 in 
non-glioblastoma tumors was detected in tissue samples from 
medulloblastomas and neuroblastomas, which are cancers that occur 
predominantly in the pediatric population. 
 
   EB1-positive staining was also found in tissue samples from metastatic 
melanoma (skin cancer). Other tumors expressing slightly lower levels of 
EB1 staining include non-small cell lung cancer, colorectal cancer and 
triple-negative breast cancer.(4) 
 
   Dr. Marc Engelhardt, Chief Medical Officer, commented: "We are very 
pleased that two patients with EB1-positive recurrent glioblastoma have 
obtained long-lasting clinical benefit in our phase 1 study with 
lisavanbulin. We are looking forward to the interim results in our phase 
2 study, which is enrolling patients with recurrent glioblastoma that is 
EB1-positive, towards the end of 2021. The new prevalence data presented 
at ASCO are consistent with our initial frequency estimates of 
EB1-positive glioblastoma. A clinical proof-of-concept in glioblastoma 
based on positive interim results would support exploring the selection 
of patients based on EB1-positivity in other tumor types as well, such 
as melanoma, breast, colorectal and lung cancers or rare cancer types 
such as medulloblastomas or neuroblastomas." 
 
   The rationale and study design of the ongoing phase 2 study with 
lisavanbulin in patients with EB1-positive recurrent glioblastoma is 
being presented in a second poster at the ASCO Annual meeting.(5) 
 
 
 
 
The following posters are presented at the 2021 ASCO 
 Annual Meeting: 
Abstract #  Title 
3118        Expression of end-binding protein 1 (EB1), a potential 
             response-predictive biomarker for lisavanbulin, in 
             glioblastoma and various other solid tumor types 
             Authors: Magdalena Skowronska, Crescens Tiu, Alexandar 
             Tzankov, Fatima König, Joanne Lewis, Igor Vivanco, 
             Malte Kleinschmidt, Kirk Beebe, Stephanie Anderson, 
             Felix Bachmann, Marc Engelhardt, Heidi A. Lane, Thomas 
             Kaindl, Alexandru C Stan, Elizabeth Ruth Plummer, 
             T. R. Jeffry Evans, Inti Zlobec, Juanita Suzanne Lopez 
TPS2068     The potential utility of end-binding protein 1 (EB1) 
             as response-predictive biomarker for lisavanbulin: 
             A phase 2 study of lisavanbulin (BAL101553) in adult 
             patients with recurrent glioblastoma 
             Authors: Crescens Tiu, Sarah Derby, Noor Md. Haris, 
             Liam Welsh, Anna Stansfeld, Thomas Hundsberger, Patrick 
             Roth, Fatima König, Joel Robert Eisner, Malte 
             Kleinschmidt, Stephanie Anderson, Felix Bachmann, 
             Heidi A. Lane, Marc Engelhardt, Thomas Kaindl, Karine 
             Litherland, Alexandru C. Stan, T. R. Jeffry Evans, 
             Elizabeth Ruth Plummer, Juanita Suzanne Lopez 
 
 
   For further information, please visit 
https://conferences.asco.org/am/abstracts-posters 
 
   About lisavanbulin (BAL101553) 
 
   Basilea's oncology drug candidate lisavanbulin (BAL101553, the prodrug 
of BAL27862)(6) is being developed as a potential therapy for diverse 
cancers.(1) (,) (7, 8) 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.(9, 10, 11) 
 
   Lisavanbulin efficiently distributes to the brain, with anticancer 
activity in glioblastoma models.(12, 13) In preclinical studies, 
end-binding protein 1 (EB1) was identified as a potential 
response-predictive biomarker in glioblastoma models.(2) The active 
moiety BAL27862 binds to the colchicine site of tubulin, with distinct 
effects on microtubule organization,(14) resulting in the activation of 
the "spindle assembly checkpoint" which promotes tumor cell death.(15) 
 
   About Basilea 
 
   Basilea is a commercial-stage biopharmaceutical company founded in 2000 
and headquartered in Switzerland. We are committed to discovering, 
developing and commercializing innovative drugs to meet the medical 
needs of patients with cancer and infectious diseases. We have 
successfully launched two hospital brands, Cresemba for the treatment of 
invasive fungal infections and Zevtera for the treatment of severe 
bacterial infections. We are conducting clinical studies with two 
targeted drug candidates for the treatment of a range of cancers and 
have a number of preclinical assets in both cancer and infectious 
diseases in our portfolio. Basilea is listed on the SIX Swiss Exchange 
(SIX: BSLN). Please visit 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. A. Nehlig, A. Molina, S. Rodrigues-Ferreira et al. Regulation of 
      end-binding protein EB1 in the control of microtubule dynamics. Cellular 
      and Molecular Life Sciences 2017 (74), 2381-2393 
 
   2. 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 
 
   3. ClinicalTrials.gov identifier: NCT02490800. Phase 1 results: C. Tiu, A. 
      Tzankov, R. Plummer et al. The potential utility of end-binding protein 1 
      (EB1) as response-predictive biomarker for lisavanbulin: Final results 
      from a phase I study of lisavanbulin (BAL101553) in adult patients with 
      recurrent glioblastoma (GBM). Annals of Oncology 2020 (31) supplement 4, 
      S396-S408 
 
   4. M. Skowronska, C. Tiu, A. Tzankov, et al. Expression of end-binding 
      protein 1 (EB1), a potential response-predictive biomarker for 
      lisavanbulin, in glioblastoma and various other solid tumor types. J Clin 
      Oncol 39, 2021 (suppl 15; abstr 3118) 
 
   5. C. Tiu, S. Derby, N. Haris et al. The potential utility of end-binding 
      protein 1 (EB1) as response-predictive biomarker for lisavanbulin: A 
      phase 2 study of lisavanbulin (BAL101553) in adult patients with 
      recurrent glioblastoma. J Clin Oncol 39, 2021 (suppl 15; abstr TPS2068) 
 
   6. 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) 
 
   7. ClinicalTrials.gov identifier: NCT03250299 
 
   8. ClinicalTrials.gov identifier: NCT02895360 
 
   9. 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 
 
  10. 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) 
 
  11. 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.

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June 07, 2021 01:15 ET (05:15 GMT)