Race Initiates Extramedullary AML Preclinical Study
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ASX Announcement
Race Initiates Extramedullary AML Preclinical Study
Race enters a collaborative preclinical research program with The University of Newcastle
to support the use of Bisantrene in the treatment of extramedullary acute myeloid
leukaemia (AML)
Research designed to support the use of Bisantrene to treat this form of AML with
substantial unmet clinical needs
The work will aid the clinical progress of Bisantrene under Pillar 3 of Race’s Three Pillar
strategy
30th March 2021 – Race Oncology Limited (“Race”) is pleased to announce that it has entered
into a new collaborative preclinical research program with The University of Newcastle. This
work will be led by the eminent cancer researcher, Associate Professor Nikki Verrills, who
successfully ran Race’s preclinical breast and ovarian program (ASX announcements: 24
November 2020, 23 February 2021, 9 March 2021).
The aim of this project is to support the clinical use of Bisantrene as a novel treatment for
extramedullary AML, a difficult to treat form of AML, using an extramedullary mouse model
developed by A/Prof Verrills’ team. Extramedullary AML occurs when the leukaemia spreads
from the bone marrow and forms solid tumours in tissues such as the skin, breast, kidney,
brain, or other organs1. A 2020 prospective positron imaging trial identified that up to 22% of
AML patients have extramedullary AML2.
In a recent Sheba Medical Centre Phase II clinical trial conducted in relapsed and refractory
(R/R) AML patients (ASX announcement: 16 June 2020), Bisantrene was observed to be highly
effective in patients with extramedullary AML, with all patients with this subtype (4/4) showing
a clinical response3. Patients with extramedullary AML currently have limited treatment choices
with no approved, and very limited experimental treatment options4.
Race is pursuing Bisantrene therapies targeting AML, as part of its Three Pillar strategy (ASX
announcement: 30 Nov 2020). This new program could lead to AML treatments with improved
safety and efficacy for patients with extramedullary AML.
In addition, this study will be used to provide supportive data for a pivotal (Phase II/III) trial of
Bisantrene in extramedullary AML patients with the aim of providing a rapid path to FDA
approval for Bisantrene as an orphan drug under the 505(b)(2) track. Orphan drug designation
provides for seven years post approval marketing exclusivity in the USA and potentially 10
years in the EU/UK, as well as other tax and regulatory benefits.
Chief Scientific Officer, Dr Daniel Tillett said: “This is a key project for Race using Associate
Professor Verrills’ extramedullary AML mouse model. Recent clinical evidence has identified
Bisantrene as an effective treatment option for patients with the difficult-to-treat extramedullary
Race Oncology Ltd ABN 61 149 318 749
Registered office: L36, 1 Macquarie Place, Sydney NSW 2000
www.raceoncology.com
form of AML. We believe that we have identified a low-risk pathway to rapid approval of
Bisantrene via this indication that offers significant upside for Race in a crowded clinical space.”
This project is to start immediately with the pre-clinical results expected to be reported over
the coming 12 months.
1. Solh, M., Solomon, S., Morris, L., Holland, K., & Bashey, A. (2016). Extramedullary acute myelogenous leukemia.
Blood Reviews, 30(5), 333–339.
2. Stölzel, F., Lüer, T., Löck, S., Parmentier, S., Kuithan, F., Kramer, M., et al. (2020). The prevalence of extramedullary
acute myeloid leukemia detected by 18FDG-PET/CT: final results from the prospective PETAML trial.
Haematologica, 105(6), 1552–1558.
3. Canaani, J., Danylesko, I., Shemtov, N., Zlotnick, M., Lozinsky, K., Benjamini, O., et al. (2021). A phase II study of
bisantrene in patients with relapsed/refractory acute myeloid leukemia. European Journal of Haematology, 106(2),
260–266.
4. Cunningham, I., Hamele Bena, D., Guo, Y., Shiomi, T., Papp, A. C., Chakravarti, B., et al. (2019). Extramedullary
leukemia behaving as solid cancer: clinical, histologic, and genetic clues to chemoresistance in organ sites.
American Journal of Hematology, 94(11), 1200–1207.
-ENDSAbout Associate Professor Nikki Verrills
Since completing her PhD in 2005 on chemotherapy resistance in childhood leukaemia,
Associate Professor Verrills was awarded a Peter Doherty Postdoctoral Fellowship from the
National Health and Medical Research Council in 2006. In the same year she was the inaugural
recipient of a Hunter Medical Research Foundation grant for young cancer researchers. Since
then she has established an innovative research lab at the University of Newcastle studying
the differences between cancer cells that respond well to drug treatments and those that do
not.
Prof Verrills is currently supported by a fellowship from the Australian Research Council and
project funding from the National Health and Medical Research Council. She has published
over 60 journal articles with an H-index of 24.
About Race Oncology (ASX: RAC)
Race Oncology is an ASX listed precision oncology company with a Phase II/III cancer drug
called Bisantrene.
Bisantrene is a potent inhibitor of the Fat mass and obesity associated (FTO) protein. Overexpression of FTO has been shown to be the genetic driver of a diverse range of cancers. Race
is exploring the use of Bisantrene as a new therapy for melanoma and clear cell renal cell
carcinoma, which are both frequent FTO over-expressing cancers. The Company also has
compelling clinical data for the use of Bisantrene as a chemotherapeutic agent with reduced
Race Oncology Ltd ABN 61 149 318 749
Registered office: L36, 1 Macquarie Place, Sydney NSW 2000
www.raceoncology.com
cardiotoxicity in Acute Myeloid Leukaemia (AML), breast and ovarian cancers and is
investigating its use in these areas.
Race is pursuing outsized commercial returns for shareholders via its ‘Three Pillar’ strategy for
the clinical development of Bisantrene.
See more at www.raceoncology.com.
Release authorised by:
Media contact:
Phil Lynch, CEO/MD on behalf
Jane Lowe
of the Race Board of Directors
+61 411 117 774
phillip.lynch@raceoncology.com
jane.lowe@irdepartment.com.au
Race Oncology Ltd ABN 61 149 318 749
Registered office: L36, 1 Macquarie Place, Sydney NSW 2000
www.raceoncology.com
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