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ANNUAL REPORT 2022 CONTENTS 2022 at a Glance 2 Chairman s Letter 4 CEO s Report 6 Key Milestones 8 Pipeline Review 10
Environment, Society & Governance 14 Our Collaborators 16 Financial Reports 20
The journey of a young biotech company is often circuitous, but we have nevertheless continued to make great progress in the past year.
We have two first-class drug candidates in clinical development, with a diverse portfolio of trials that have the potential to open up very substantial commercial markets. We have an experienced and capable team, and an international network of
supportive partners and collaborators. In almost every important respect, the fundamentals of Kazia have never been stronger. kaziatherapeutics.com
2022 AT A GLANCE GLOBAL COLLABORATION OUR CLINICAL RESEARCH Kazia Therapeutics is a late clinical stage 400+ oncology company. hospitals
We work alongside 6 involved in clinicians, researchers, scientific conference paxalisib and partners to bring presentations of clinical trial paxalisib data in impactful new therapies 1H CY2022 program to patients with cancer. OUR PATIENTS
Paxalisib has shown evidence of activity in multiple forms of brain cancer 4 3 distinct indications special designations in phase II studies awarded to paxalisib with paxalisib by FDA Fast Track designation, Orphan Drug designation, glioblastoma,
DIPG, PCNSL, Rare Pediatric Disease brain metastases designation OUR BUSINESS Kazia has grown Indicative Analyst rapidly, driven Valuation (US$) 82% by progress in its world-class pipeline Jan 2020 $93M of operating Dec 2020 $174M cashflows of
cancer drug invested in Apr 2021 $247M candidates R&D Oct 2021 $277M Mar 2022 $294M Edison Research for FY2022
2H CY Phase I 6 2023 8 study of EVT801 final data underway countries involved in anticipated ongoing clinical development in France
of paxalisib from pivotal clinical trials study of with paxalisib United States, Canada, paxalisib in Spain, France, Switzerland, 2H CY2023 United Kingdom as at 30 June 2022 Most 200k Multiple common potential approximate patients indications
cause of 2 per annum treated with whole brain for EVT801, cancer death radiotherapy in United childhood brain States including lung in children is cancers under investigation with cancer, bowel brain cancer paxalisib cancer, and No FDA-approved kidney cancer therapies for DIPG Brown et al. (2018). J Clin CBTRUS or AT/RT Oncol. 36(5):483-491 84-94% 150+ $4.2m GBM
forecast adoption of new equity capital of paxalisib in US, years of raised through if approved by FDA 4 aggregate drug financing in FY2022 licensing development partnerships experience in place among Triangle Insights research project, management
commission by Kazia team Therapeutics AU$ Kazia Theraputics Limited Annual Report 2022
ADVANCING THE PIPELINE Dear Shareholder, Kazia has continued to make real progress during the 2022 fiscal year, despite the headwinds
resulting from an enormously challenging financial environment for listed biotech companies. Notwithstanding, I am pleased to be able to review here some of key developments in Kazia over the past year. PIPELINE PROGRESS The company s
pipeline remains understandably dominated by paxalisib, our late-stage asset for brain cancer, however, during the financial year, Kazia has also made very good progress with EVT801, our second asset, which was licensed from Evotec SE in April 2021.
That drug is now well-advanced in a phase I clinical trial, and we anticipate some initial results in 1H CY2023. With the benefit of an additional year, we ve had the chance to further understand, discuss, and chart its potential, and I should
say that we are even more excited by EVT801 now than we were at the time of its licensing. For paxalisib, the confidence of some of our investors has understandably been shaken by the news, post period, that the drug would not enrol the second stage
of the GBM AGILE pivotal trial. The implications of this development are discussed elsewhere in this annual report, and so I will not recapitulate them here. Suffice to say, however, that the first stage of the study remains ongoing, having achieved
full recruitment, and will report final data in 2H CY2023. There remains every chance that paxalisib will yet achieve approval in glioblastoma on the basis of this substantial data set, and all involved with its development
continue to push forward on that basis with all the resources and energy at their command. The news demonstrates the inherent unpredictability of drug development. Thankfully, Kazia s strategy has always been to diversify its activities as
broadly as possible, so that the company and its shareholders are insulated from adverse developments. GBM AGILE is only one of eight ongoing clinical trials of paxalisib, covering four distinct disease areas. The news from GBM AGILE was bookended
by very positive outcomes in two studies of brain metastases and was preceded by exceptionally promising preclinical data in two forms of childhood brain cancer. We believe that paxalisib has great promise, across a wide range of brain cancers, and
we remain resolute in the task of demonstrating its potential. FINANCIAL PERFORMANCE We concluded FY2022 with a cash balance at 30 June 2022 of $7.4 million, versus $27.6 million at 30 June 2021. Our total assets were
$35 million, compared to $58 million at 30 June 2021. During FY2022, we deployed $22 million to move forward the company s pipeline, representing over 80% of our total expenditure for the year. We should acknowledge that we
are reporting these results when the financial markets are characterised by extremely negative sentiment towards the biotech sector. Between 30 June 2021 and 30 June 2022, XBI, the de facto NASDAQ small cap biotech index, lost more
than 45% of its value, and there is evidence that smaller companies such as ours have, on average, been even more adversely affected. In recent months, there have at times been more than 200 listed biotech companies on NASDAQ
whose market capitalisation is less than their cash balance. Meanwhile, the number of IPOs and secondary offerings has fallen to a trickle, with many institutional investors reserving capital to support their existing portfolio investments. For
Kazia, there is no doubt that these dynamics have complicated the natural cadence of our funding cycle. Our strategy has always been to fund the company to milestones, taking only what we need in each financing round to move the pipeline to its next
stage of development, thereby minimising both risk and dilution for our investors. Given the current state of the market, we have in some respects taken this strategy one step further by making the decision to implement an at-the-market (ATM) facility to provide interim access to capital through the market downturn. ATMs are a common financing instrument for small companies,
particularly on NASDAQ. In brief, the tool allows us to place stock directly into the market, allowing us to periodically raise modest amounts of capital at no discount to market, with no requirement for warrants or options, and with very modest
banking fees. For a company such as Kazia, which runs exceptionally lean, the ATM can be an excellent device to manage cashflows. Indeed, in our case it has enabled us in recent months to fully support the company s ongoing activities, in a way
that has spared our shareholders the very deleterious terms that would have inevitably accompanied a more conventional transaction.
We believe that paxalisib has great promise, across a wide range of brain cancers, and we remain resolute in the task of demonstrating
its potential. However, the ATM is not a permanent solution and to fulfil its potential, Kazia will need to continue to bring good quality, long-term, fundamentally-driven healthcare investors onto its register. As and when the market re opens, we
will waste no opportunity to put the company s compelling story in front of the widest range of investors we can. In the meantime, however, the access that we have been able to secure to cost-effective and minimally dilutive capital has been
vital to our business. CONCLUSION The journey of any biotech company is often circuitous, but we have nevertheless continued to make great progress in the past year. We have two first-class drug candidates in clinical development, with a diverse
portfolio of trials that have the potential to open up very substantial commercial markets. We have an experienced and capable team, and an international network of supportive partners and collaborators. In almost every important respect, the
fundamentals of Kazia have never been stronger. I would like to thank, once again, my fellow directors and our management team, led by our CEO, James Garner, for their dedication to the company s work. And, as always, we
remain grateful for the ongoing support of our many shareholders, whose faith in the company makes possible everything that we do. Iain Ross Chairman of the Board Kazia Theraputics Limited Annual Report 2022
A DIVERSE PORTFOLIO Dear Shareholder, The past twelve months have been an extremely fertile period for Kazia s research and
development efforts, particularly in respect of our lead program, paxalisib. We have commenced two new clinical trials: one at Weill Cornell Medicine investigating paxalisib in combination with a low-insulin
state for glioblastoma, and one in collaboration with the Pacific Pediatric Neuro-Oncology Consortium, examining paxalisib in combination with another drug for the treatment of diffuse midline gliomas (DMGs), a highly-aggressive group of childhood
brain cancers. The work that we start is, in a sense, an investment whose return is the data we receive a year or two hence. Several of the studies that we began in the past few years have reported important milestones during FY2022. Our own phase
II study of paxalisib has completed, with very encouraging results in the final efficacy data. The phase II study in brain metastases, run by the Alliance for Clinical Trials in Oncology, has graduated to a second stage in patients with breast
cancer brain metastases. And, in early August, we saw extremely positive signals from a study of paxalisib in combination with radiotherapy for brain metastases, in which every evaluable patient demonstrated radiological response. The ever-growing
body of data around paxalisib, derived from a very broad range of clinical trials and laboratory studies, helps to provide both confidence in its activity and breadth in its commercial opportunity. While clinical trials
naturally more readily capture the imagination, we have also reported this year some very promising preclinical data in childhood brain cancer. A team from Johns Hopkins Medical School reported data in atypical teratoid / rhabdoid tumours (AT/ RT)
at the AACR Conference in April 2022, and Professor Matt Dun of the University of Newcastle presented data on DIPG to the ISPNO Conference in June 2022. Together, these presentations, from leading scientists at first-rate institutions, have expanded
our thinking in relation to the opportunity for paxalisib in childhood brain cancer. We see this as an increasingly important plank in paxalisib s overall development. We have secured orphan designation and rare pediatric disease designation in
both AT/RT and DIPG, and these achievements help to greatly facilitate our regulatory strategy in childhood brain cancer. If paxalisib is approved in either disease, we may be eligible to receive a pediatric priority review voucher (pPRV), which can
be sold to other companies and which typically commands a price in excess of one hundred million dollars. No doubt, however, these important and exciting developments are coloured to some extent by the news we received at the end of July, that
paxalisib would not graduate to the second stage of the GBM AGILE pivotal study. It is important to be clear what this development may or may not mean for the drug s further development. The
two-stage design of GBM AGILE was designed primarily to increase the statistical power of the study. A drug which successfully clears both stages of the trial may be considered almost unimpeachable in terms of
the statistical confidence that accompanies its data. However, this approach sets a high bar for any drug participating in the study, and it is very far from certain that failure to complete both stages is incompatible with an eventual product
approval. GBM AGILE will likely provide for the evaluation of paxalisib a more substantial number of patients than were available to support the approval of temozolomide, the existing standard of care in glioblastoma, and the study remains ongoing.
As is almost invariably the case in drug development, we will need to wait and see the data before we understand our position. We continue to anticipate that data in 2H CY2023 and, until then, all Kazia personnel remain blinded to
efficacy and safety. In the meantime, the patients who have enrolled in GBM AGILE continue to receive treatment and to undergo follow-up, per protocol, and will continue to provide data for analysis. As the
data matures, we will no doubt learn a great deal more about paxalisib and will be much better placed to understand its potential benefit to patients with glioblastoma.
The ever-growing body of data around paxalisib, derived from very broad range clinical trials and laboratory studies, helps to provide
both confidence in its activity and breadth its commercial opportunity. It is entirely understandable that this development has caused uncertainty in the minds of some of our investors. In truth, however, there is almost nothing concrete that we
have learned from GBM AGILE to date, for better or for worse, that we did not know this time last year. The study is scarcely half-complete. And ultimately, in a disease such as glioblastoma, which is characterised by an overwhelming unmet medical
need, there may be an inclination on the part of regulators and clinicians to accommodate a drug which can show any degree of meaningful benefit. Meanwhile, the other seven clinical trials of paxalisib continue to progress well in general, with
multiple positive read-outs in recent months and a great deal more data to come. And EVT801, which joined our pipeline last year, is now well-advanced in a phase I study in Europe, with initial data anticipated in 1H CY2023. Regardless of the
eventual outcome of GBM AGILE, both of our outstanding drug candidates are blessed with many opportunities to succeed. To that end, all of us in the Kazia team continue to apply ourselves wholeheartedly to the task of finding how best to use our
drug candidates to help patients. I am grateful to my colleagues on the Board and in the Management Team for their perseverance and their professionalism, and to our shareholders for their ongoing support. Dr James Garner Chief Executive Officer
Kazia Theraputics Limited Annual Report 2022
KEY MILESTONES HIGHLIGHTS 2021/2022 December 2021 Kazia releases
top-line final data from phase II study of paxalisib in glioblastoma, showing meaningful signals of efficacy. Kazia expands April 2022 management team November 2021 with two senior US- Preclinical data in AT/RT, EVT801 phase I study based appointments: a rare childhood brain commences recruitment Dr John Friend as Chief cancer is presented at September 2021 at Oncopole Hospital in Medical
Officer, and the AACR conference. EVT801 is granted Toulouse, France. The Karen Krumeich as This data expands the approval by ANSM, the biomarker-enhanced study Chief Financial Officer. opportunity for paxalisib French regulatory agency, is intended
to provide These appointments in childhood brain to commence a phase I safety and dosing data bring, in aggregate, cancer, positioning it clinical trial, less than six but also to demonstrate more than 50 years of as a substantial area months after
the asset the pharmacological biotech experience to of focus for the drug s was licensed by Kazia. activity of EVT801. the management team. development. November 2021 February 2022 May 2022 Paxalisib commences A phase II
study of GBM AGILE pivotal recruitment to a phase paxalisib in combination study of paxalisib II adaptive study in with a ketogenic diet in glioblastoma DIPG run by the Pacific for the treatment of expands to Europe. Pediatric Neuro-Oncology
glioblastoma commences Consortium (PNOC). recruitment at Weill This study administers Cornell Medicine. This paxalisib with ONC201, study is informed by a combination which world-class research has shown evidence of from Professor Lew activity in
preclinical data Cantley, who discovered and compassionate use. the PI3K pathway that paxalisib targets. GBM AGILE pivotal study of paxalisib in glioblastoma expands to Canada.
June 2022 Final data from the phase II study of paxalisib in glioblastoma is presented at ASCO. Preclinical data examining the
combination of paxalisib with ONC201 for treatment of DIPG is presented at the ISPNO conference. The data provides powerful support for the ongoing PNOC study, which commenced recruitment in November 2021. The Alliance study of paxalisib in brain
metastases moves into an expansion cohort in breast cancer brain metastases, having seen positive signals in the initial exploratory cohort. Further cohorts continue to examine brain metastases from lung cancer and other primary tumours. Paxalisib
receives orphan drug designation from FDA for the treatment of AT/RT, providing additional market exclusivity, waiver of PDUFA fees, and access to grant opportunities.
PIPELINE REVIEW A BROAD CLINICAL PIPELINE PAXALISIB Although glioblastoma remains very much the lead indication, childhood brain cancer
has emerged as a very important second element in the paxalisib story. Brain cancer is the most common cause of cancer death in children, and it remains terribly poorly treated. Both diffuse intrinsic pontine glioma (DIPG) and atypical teratoid /
rhabdoid tumours (AT/ RT), two diseases which have been a strong focus for Kazia in the past year, have no FDA-approved drug treatments and, as a consequence, the prognosis is very poor. The second quarter of
CY2022 saw important preclinical data presented at international conferences in this area. Professor Jeffrey Rubens and colleagues at Johns Hopkins Medical School presented very positive data for paxalisib in AT/RT at the American Association of
Cancer Research (AACR) Annual Meeting in April 2022. This data enabled paxalisib to receive Orphan Drug Designation (ODD) for this disease in June 2022. Kazia is currently in discussion about potential opportunities to translate this very promising
work into a clinical trial. In June 2022, Associate Professor Matt Dun from the Hunter Medical Research Institute at the University of Newcastle, Australia, presented very powerful results from his research in the combination of paxalisib with a
drug called ONC201 (manufactured by Chimerix, Inc) in the treatment of DIPG. This data has already enabled a clinical trial of the combination in this disease, which began recruitment in November 2021. Professor Dun s presentation also included
several very promising case studies from compassionate use experience with the two drugs in combination. Kazia s pipeline is remarkable for its diversity. Paxalisib, the lead program, is in clinical trials for multiple forms of brain
cancer. EVT801 has potential applications in a wide range of solid tumours. Together, they give Kazia an extensive breadth of opportunity for a company of its size. Another element of the paxalisib program that has been
emerging as a very promising opportunity has been brain metastases, a collective term for cancer which spreads to the brain from other parts of the body. More than 200,000 patients each year develop brain metastases in the United States alone, and
treatment options are limited. Three clinical trials have been examining paxalisib as a potential treatment for these patients. One of these studies, run by the Alliance for Clinical Trials in Oncology, has already seen positive data for paxalisib
in patients with breast cancer brain metastases and, on that basis, has moved the drug into an expansion phase. The study remains in an exploratory phase for patients with lung cancer brain metastases, and for patients with brain metastases from
other primary tumours. A second study, at Memorial Sloan Kettering Cancer Center, has similarly moved into an expansion phase, with initial data from the first part of the trial accepted for a prestigious oral presentation at a specialist scientific
conference on brain metastases organised jointly by the Society for Neuro-Oncology (SNO) and the American Society for Clinical Oncology (ASCO). Excitingly, this data showed all evaluable patients responding to the combination of paxalisib with whole
brain radiotherapy, suggesting the potential for our drug to play an important role in augmenting the efficacy of this ubiquitous therapy. In addition, paxalisib is also the subject of a clinical trial in primary CNS lymphoma, a less common form of
brain cancer that remains very challenging to treat. Paxalisib belongs to a class of medicines known as PI3K inhibitors, and four
KEY GLOBAL REGULATORY AGENCIES of the five PI3K inhibitors that have been approved by FDA have been approved for types of lymphoma.
Since none of these drugs cross the blood-brain barrier, they are far from ideal to treat lymphoma in the brain, but paxalisib is very well suited to this patient group. Meanwhile, paxalisib is now some eighteen months into GBM AGILE, the pivotal
clinical trial for registration in glioblastoma. Completion of a pivotal clinical trial is one of the most critical landmarks in the development of a new medicine. We learned at the end of July that the drug would not graduate to the