Announcement PDF ASX Announcement Race Oncology Phase II Bisantrene AML trial webinar 17 June 2020 – A group investor briefing will be held to discuss the significance of the trial results in more detail on Wednesday 17 June 2020 at 10:30am Australian Eastern Time (8:30am Western Australian/Hong Kong time; 8:30pm New York time and 5:30pm San Francisco time on Tuesday 16 June 2020). Participants will need to pre-register for the call, using the following link: https://us02web.zoom.us/webinar/register/WN_UZqeygRqSwq41YoH3PBWEw - ENDS - About Race Oncology (RAC: ASX) Race Oncology (RAC) is a drug development biotech with a Phase II/III cancer drug called Bisantrene. RAC has compelling clinical data for Bisantrene in acute myeloid leukaemia (AML) as well as breast and ovarian cancer. RAC is pursuing an exciting ‘5-Path’ clinical development strategy that involves parallel US and Australian clinical trials in AML, breast and ovarian with clinical trials to begin in 2020. Release authorised by: John Cullity, Executive Chairman John.cullity@raceoncology.com Media contact: Henry Jordan, Six Degrees Investor Relations +61 431 271 538 henry.jordan@sdir.com.au Daniel Tillett, Chief Scientific Officer & Director daniel.tillett@raceoncology.com Race Oncology Ltd ABN 61 149 318 749 Registered office: L40, 140 William St, Melbourne VIC 3004 www.raceoncology.com Bisantrene Phase II Data Presentation June 2020 Trial highlights | Chair Clinical Advisory Board, Professor Borje Andersson June 2020 About Bisantrene Bisantrene is a cancer chemotherapy that was previously approved for AML in France 1988 and then shelved. Race Oncology is returning this drug back into clinical development Bisantrene (Xantrene®) is an anthracene with anthracycline-like activity and was shown in earlier clinical trials to be an effective salvage therapy in R/R AML with little or no discernible cardiotoxicity Since then, Race: • Has successfully manufactured Bisantrene • Built a strong patent position (3 granted US patents) • US Orphan Drug designation (7 years exclusivity) • Secured a Rare Paediatric Disease designation with the potential to receive a Priority Review Voucher (PRV) Bisantrene remains an excellent cancer drug with great potential in modern cancer treatment for AML and other cancers Current AML Treatment Generalized overview Complete AML Intensive Chemotherapy (Induction and consolidation) Response (CR) Stem Cell Transplant (HSCT) This trial Salvage chemotherapy Bisantrene IV 250mg/m2 x 7 days Cure This trial Relapsed +/or Refractory AML Management of R/R AML represents a significant therapeutic challenge with no established standard of care Phase II Bisantrene monotherapy in relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) Bisantrene is active, safe and effective in Adult R/R AML after multiple prior therapies Bisantrene ORR = 40% (4/10 patients * 4 Responses reported (1 CR + 3 PR) 7 day (single IV course) treatment Favourable safety and tolerability 10 patients enrolled 10 Evaluated for safety and efficacy *4/4 responses in high-risk extramed. disease Study design - Phase II open-label single arm single center study (NCT03820908) - Site. Chaim Sheba Medical Center, Tel Aviv, Israel - Principal investigator. Professor Arnon Nagler Study design Criteria Key inclusion Key exclusion Patients with Relapsed/Refractory AML Active pulmonary disease, diffusing capacity for carbon monoxide(DCFO) < 30% Aged 18 years or older Symptomatic heart failure (NYHA Grade 2 or above) Eastern Cooperative Oncology Group (ECOG) performance score 0 or 1 Bilirubin > 3.0 mg/dl Adequate cardiac function; left ventricular ejection fraction (LVEF) ≥ 40% Transaminase levels more than three times the upper limit of normal (ULN) Adequate birth control in potentially fertile patients Creatinine > 2.0 mg/dl Active central nervous system (CNS) disease# Grade III-IV graft-versus-host disease (GVHD) # Two CNS patients enrolled with a waiver approved by local IRB; NYHA - New York Heart Association [NYHA] Class III to IV Study design Treatments and assessments Treatment Bisantrene 250mg/m2 over 2-hour, one infusion daily for 7 days Salvage therapy - predominantly as 4th line therapy Endpoints Primary à composite of complete remission (CR) and complete remission with incomplete hematologic recovery (CRi) Secondary à survival (defined as the time from study entry to death from any cause) remission duration, type incidence and severity of AEs gene mutational analyses Study design Assessments ? Safety • • • • Vital signs & physical Clinical lab tests and bone marrow examination ECG Adverse Events per CTCAE v5.0 Evaluable patients: all that took one course of bisantrene (n=10) $ Response • • Blast cells in bone marrow and in blood According to 2003 International Working Group (IWG) standardized response criteria for AML Evaluable patients: all completed bisantrene treatment (n=10) Disease mutations • Next generation sequencing (NGS) studies on patient bone marrow samples pre-treatment Evaluable patients: all that provided a sample (n=7) Patient Demographics Characteristic N=10 Median age Characteristic N=10 MRC Cytogenetic risk Years (range) 43 (22-80) Favourable Intermediate Advanced Gender Male Female 6 4 ELN 2017 risk category ECOG performance status at screening 0 1 9 (90%) 1 (10%) Secondary disease (acute antecedent myeloid disorder) 3 (30%) Extramedullary disease Prior system therapy regimens (lines) 4 (40%) 1 2 >3 1 (10%) 3 (30%) 6 (60%) Favourable Intermediate Advanced 2 (20%) 5 (50%) 3 (30%) NPM1wt NPM1mut 8 (80%) 2 (20%) FLT3wt FLT3-ITD 6 (60%) 4 (40%) NPMI status Disease diagnosis Prior allogenic transplant 0 7 (70%) 3 (30%) FLT3-ITD status 7 (70%) “Hard to treat” disease setting Safety Treatment Related Adverse Events (TRAEs) in 10 patients Any grade > Grade 3 1 (10%) 2 (20%) 3 (30%) 1 (10%) 2 (20%) 3 (30%) Most frequently reported serious adverse events: • thrombocytopaenia (low blood platelets) • mucositis (mouth ulcers) Haematologic Anemia Neutropenia Thrombocytopenia Both of these are expected side effects with chemotherapeutics of this kind. Non-Haematologic Fever Headache Chills Dyspnea Myocardial infarction Mucositis Acute Kidney Injury Sepsis Blood bilirubin increased Confusion Hypernatremia Ejection fraction decreased Atrial fibrillation Abdominal pain Diarrhea Vomiting Rash Upper gastrointestinal bleeding 6 (60%) 1 (10%) 1 (10%) 1 (10%) 2 (20%) 7 (70%) 1 (10%) 3 (30%) 2 (20%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 1 (10%) 2 (20%) 6 (60%) 1 (10%) 2 (20%) 1 (10%) 1 (10%) 0 1 (10%) Death (unrelated) # Cardiac arrest# 1 (10%) Deemed due to pre-transplant chemotherapy conditioning regimen Outcomes - Of the 10 patients, one patient (10%) achieved a complete remission and three patients achieved a partial remission → Overall Response Rate of 40% All 4 responders had high-risk extramedullary disease: • Patient with leukaemia cutis (skin) achieved CR and bridged to allogeneic stem cell transplantation • Patient with breast chloromas achieved high reduction in sites of disease deemed as partial response • Patients with CNS disease both achieved transient clearance of peripheral blood blasts with one resulting in partial remission of ocular disease and other partial response of CNS disease Conclusion Established safety and tolerability of current Bisantrene formulation Confirmed previous anti-leukeamic activity Dose of (250mg/m2) maintains effectiveness in R/R AML Bisantrene is safe and its effectiveness maintained. Positioned to be investigated with complementary anti-leukemic therapy to re-enter the modern AML therapy landscape So Where, Henceforth ……….. 17/6/20 B.S. Andersson Other Activities | CSO Daniel Tillett June 2020 The MRD opportunity 2-year survival and residual disease status at transplant 100 Up to 80% of AML patients who are fit enough for induction chemotherapy (3+7) with enter remission (CR) and may then be candidates for a human stem cell transplant (HSCT) ● MRD(+) patients (those with MRD) have less than 25% two- year survival time ● MRD(-) patients have a 80% survival post transplant = potential cure! As yet, there are no approved treatments that can change MRD status from (+) to (-) for AML ● Bisantrene is potentially the answer 80 % 2 year survival Whether the transplant results in long term survival depends largely on the patient’s MRD (Measurable Residual Disease) status at the time of transplant 90 70 60 50 40 30 20 10 0 MRD(+) MRD(-) Bisantrene in AML treatment for MRD Bisantrene could transform cure rates by changing MRD status MRD(+) Fit patients Newly Diagnosed AML Unfit patients 3+7 Induction Chemotherapy Complete Response (CR) MRD(-) Bisantrene MRD(-) Stem Cell Transplant (HSCT) Nonresponders Various salvage combination +/or targeted therapies Relapsed +/or Refractory AML Cure 1 Phase II MRD trial Phase II study of Bisantrene treatment after (7+3) induction chemotherapy to change MRD status Aim to run trial in USA and/or Israel in partnership with a leading US cancer center Eligibility Transplant eligible MRD(+) patients in CR after induction chemotherapy – potential for paediatric study too Study Design Open label 7-day Bisantrene 250mg/m2/day treatment in 28 MRD(+) patients Endpoints MRD status post-Bisantrene Treatment post-transplant survival Goal Early FDA approval of Bisantrene for MRD(+) patients Breast cancer combination trial 4 Phase I/II proof-of-concept (POC) trial in breast cancer Will use drug combinations which preclinical data show synergise with Bisantrene (preclinical studies underway with U. Newcastle) Use optimal dosing, administration and combination of Bisantrene Historical breast cancer trials used sub-optimal dosing and administration of Bisantrene (but still showed good activity!) Goal Opens up much larger cancer market than AML (2 million cases each year) Show equivalent efficacy to existing treatments but with fewer serious side effects (less damage to the heart) Displace current anthracyclines used in breast cancer treatment Ovarian cancer combination trial 5 Phase I/II proof of concept (POC) trial in ovarian or other cancer Preclinical trials to be performed to identify those cancers that respond most to Bisantrene and which drug combinations show synergy Use optimal dosing, administration & combinations of Bisantrene Historical non-AML cancer trials all used sub-optimal dosing and did not use combinations, but still showed activity for Bisantrene Goal Open up much larger cancer market than AML (200,000 cases each year) POC trial to attract pharmaceutical partner for approval trials L40 140 William St, Melbourne, VIC 3000 info@raceoncology.com +61 3 9098 8750