Kazia Therapeutics Annual Report 2021

GBM AGILE – KEY FACTS • An ‘adaptive trial’ that only recruits the number of patients needed to reach an answer • Substantially faster and more cost-effective than conventional approaches • Up to 200 patients on paxalisib, with the potential to conclude much earlier, depending on emergent data • Operational in >40 hospitals across the United States, with expansion to Canada, Europe, and China in FY2022 A ‘PIVOTAL’ STUDY OR A ‘PHASE III’ STUDY? Clinical drug development has traditionally been divided into three phases, denoted by Roman numerals. However, the terminology has become old- fashioned. Regulatory agencies increasingly differentiate between studies which are exploratory in nature, and those which are intended to secure product registration. The latter are described as ‘pivotal’ studies, or ‘registration’ studies, and Kazia generally uses that language in relation to GBM AGILE. Much of the work that Kazia has been doing with paxalisib this year has been to support GBM AGILE, and to facilitate the regulatory submission which hopefully follows conclusion of the study. One manifestation of these efforts has been a slew of ‘special designations’ from FDA in August 2020, comprising orphan drug designation, rare pediatric disease designation, and fast track designation. Collectively, these regulatory milestones very much enhance and deepen the discussion between Kazia and FDA, and help us to best position the drug for success. IN GLIOBLASTOMA, NO DRUG THIS CENTURY HAS SHOWN CONVINCING EVIDENCE OF AN ABILITY TO IMPROVE OS. IF OUR PARTICIPATION IN GBM AGILE IS A SUCCESS, THEN PAXALISIB MAY BE THE FIRST NEW THERAPY, AT LEAST FOR NEWLY DIAGNOSED PATIENTS, IN TWENTY YEARS. Kazia has primarily been focused on FDA, the US Food and Drug Administration, for much of paxalisib’s development. Following our partnership with Simcere Pharmaceutical in China, however, an almost equal amount of work is going into preparing paxalisib for consideration by NMPA, the Chinese regulatory agency. The Kazia and Simcere teams have spent many hundreds of hours preparing for the very specific requirements set out by NMPA, with an objective of launching GBM AGILE in China by the end of CY2021. Our partnership with Simcere is not only of great operational value – by itself, Kazia would never be able to optimally navigate the Chinese regulatory environment – but it also represents another important milestone for the drug. Our licensing agreement with Simcere in March 2021 provided gross proceeds of US$ 11 million upfront, plus up to $281 million in contingent milestones and mid-teen royalties on net sales. These terms represent one of the richer transactions ever executed in China for a drug of this type and have, in effect, provided the first revenue for paxalisib. As the drug proceeds through development, further milestone payments will be reinvested by Kazia in the global paxalisib program. Although all eyes are rightly focused on product registration, the reality is that paxalisib has already begun to generate value for Kazia and its shareholders. Meanwhile, a broad and growing portfolio of clinical trials in other forms of brain cancer will help us in due course to expand the potential commercial opportunity for paxalisib. Although glioblastoma represents, on a conservative assessment, a US$ 1.5 billion annual commercial market, paxalisib has the potential to provide benefit in a much wider range of diseases. The clinical program that has been deployed will help to identify new opportunities. To be clear, it is possible that not all of these trials will be successful. However, if even one of them suggests an additional use for paxalisib, it will substantially increase the commercial value of the product. Kazia Therapeutics Limited Annual Report 2021 11 2021 AT A GLANCE CHAIRMAN’S LETTER CEO’S REPORT KEY MILESTONES PIPELINE REVIEW PARTNER FOR SUCCESS WORK WITH THE BEST #2 IN THE KAZIA STORY FINANCIAL REPORTS