Full Press Release Details
Contents 2 4 6 Chairman and Key About GBM CEO s Letter Milestones 8 12 Environment, Financial Society & Reports Governance
ii ASX:KZA | NASDAQ:KZIA
Chairman and CEO s The journey of a young biotech Letter company is often circuitous, but we have nevertheless continued to make
great progress in the past year. We Key have two first-class drug candidates Milestones in clinical development, with a diverse portfolio of trials that have the potential to open up very substantial commercial About markets. GBM We have an
experienced and capable team, and an international network of supportive partners and collaborators. In almost every important respect, the Environment, Society fundamentals of Kazia have never been and stronger. Governance
kaziatherapeutics.com Financial Reports Kazia Theraputics Limited Annual Report 2024 1
Chairman and CEO s Letter PROGRESSING TREATMENT AREAS Dear fellow shareholder, We are pleased to present Kazia Therapeutic s
Annual Report for the period ending 30 June 2024. This has been a year of transformation for our company, marked by significant milestones in the development of our two drug candidates, paxalisib and
EVT-801, as we make important progress in bringing these new cancer therapies to patients worldwide. The healthcare landscape has continued to evolve at a rapid pace, and Kazia has remained agile, navigating
challenges and opportunities with resilience. Our core mission is clear: to develop innovative treatments that address unmet needs in cancer care. In this spirit, we have focused our efforts on advancing our lead asset, paxalisib across three key
indications (GBM, DIPG and brain metastases), while completing the phase I clinical study for EVT-801. Our strategy to best position Kazia to deliver on the clinical plans for these two drug candidates
ultimately led to our decision to de-list from the ASX in October 2023 to focus on NASDAQ, the largest biotech equity market in the world. Whilst this was a big decision, and one we did not take lightly, we
believe that it was the right decision for our shareholders and for the future of the company. As a result of our decision to delist from the ASX, we have been able to reduce our operating costs and administrative burden while enabling long term
access to the largest capital market on better terms from a more diverse and much larger investor base. This access is critical to ensuring we can raise appropriate growth capital to deliver on our clinical development plans. PIPELINE PROGRESS
Paxalisib Paxalisib has continued to deliver promising clinical results in FY24, with strong data being released from a number of trials. In July 2023 we were delighted to receive Fast Track Designation (FTD) from the US Food & Drug
Administration (FDA) for paxalisib for the treatment of solid tumour brain metastases harbouring PI3K pathway mutations in combination with radiation therapy. Following on from the FTD, Kazia announced in February 2024 the early conclusion of Part
II of this two-part Phase 1 investigator-initiated trial following positive safety data and promising clinical response findings. After reviewing the Part II patient data, the three lead investigators
determined that the primary endpoint of the study had been reached. The data from this clinical trial was presented at the American Society for Radiation Oncology 66th Annual Meeting on 1 October 2024.
Chairman and CEO s In November 2023, we were pleased to provide a Recruitment also commenced for the phase II Letter preliminary
update from the ongoing investigator- clinical collaboration with the Australian and New initiated Phase 2 clinical trial at Dana-Farber Zealand Children s Haematology / Oncology Cancer Institute in Boston, evaluating paxalisib Group (ANZCHOG)
to investigate paxalisib in as a monotherapy treatment in patients with children with advanced solid tumours. OPTIMISE, relapsed/refractory primary central nervous as the study is known, will be the first clinical trial system lymphoma. We were
encouraged by of paxalisib led out of Australia and will enrol up to the clinical activity observed and look forward 24 children with PI3K pathway mutation cancers. to sharing additional updates in FY2025. As we have outlined above, we now have
three Key Also in the same month, we were invited to present clinical trial data sets across three different data from our ongoing Phase II study of paxalisib as cancerous conditions: brain metastases in patients an investigational drug for the
treatment of diffuse with underlying solid cancers, in combination with Milestones intrinsic pontine glioma (DIPG) and other diffuse midline radiation for children with DPIG and lastly in adult gliomas (DMGs), sponsored by the Pacific Pediatric
patients with glioblastoma. The combination of these Neuro-Oncology Consortium (PNOC), at the 2023 data provides supporting evidence that paxalisib is Society for Neuro-Oncology (SNO) Annual Meeting. an active anti-cancer agent and further encourage
us as we embark on our interactions with the FDA Promising additional data from this study was also regarding the next steps in development of this agent. presented in June at the 2024 International Symposium on Pediatric Neuro-Oncology (ISPNO.
Highlights On the commercial front, we entered into an exclusive of the data included median overall survival of 13.2 licensing agreement with Sovargen to develop, months in Cohort 1 (newly diagnosed, enrolled
pre- manufacture and commercialise paxalisib as a potential About radiation n=33), 15.8 months in Cohort 2 (newly treatment of intractable epilepsy in rare diseases such GBM diagnosed, enrolled post-radiation
n=69) and 8.8 as focal cortical dysplasia type 2 (FCD T2) and tuberous months in Cohort 3 (relapsed patients, enrolled sclerosis complex (TSC) disease. The underlying cause after progression n=30). The Cohort 1 and 3 data for both these diseases can
be traced back to somatic are encouraging and provides further evidence mutations in the PI3K/Akt/mTOR pathway or mutations supporting the anti-cancer activity of paxalisib. in TSC1 or TSC2 genes, which lead to the overactivation of the mTOR
pathway. Under the agreement we Immediately post reporting period we announced received an upfront payment of U$1.5M, with potential results from GBM-AGILE the Phase II/III study by the milestone
payments of up to $19M, and a percentage of Global Coalition for Adaptive Research (GCAR) that sub-licensing revenues and royalties. While our primary included the evaluation of paxalisib versus standard of
focus is oncology, this agreement allows us to explore care for patients with glioblastoma. Encouragingly, the the impact of paxalisib in other areas where there is trial data showed clinically meaningful improvement a substantial patient need and
market opportunity.Environment, (3.8 months) in a prespecified secondary analysis for overall survival in newly diagnosed, unmethylated Our collaboration with QIMR Berghofer Medical Society patients being treated with paxalisib. These results
Research Institute in Brisbane, Australia to explore are very consistent with our previous Phase II study paxalisib in combination with immuno-therapies and and we now look forward to engaging with the FDA for advanced breast cancer, such as Triple
Negative to discuss various regulatory approaches for the Breast Cancer , a type of breast cancer that doesn t approval of paxalisib, including accelerated approval. have estrogen receptors, progesterone receptors and HER2, three receptors that
are usually found on the Governance surface of cancer cells. The research collaboration with QIMR continues to evolve and we look forward to providing updates on the program later this year. Financial Reports Kazia Theraputics Limited Annual Report
Chairman and CEO s Letter EVT801 FY24 saw the successful completion of stage 1 of our Phase 1 clinical trial evaluating EVT801 in
patients with advanced solid tumours, having successfully met the primary and secondary endpoints. The signals of clinical activity, especially in patients with advanced ovarian cancer, were highly encouraging and we are more committed than ever to
continue progressing the clinical development program for EVT801 as a potential first-in-class VEGFR3 inhibitor targeted therapeutic. Preliminary biomarker and clinical
data from the study was presented in April 2024 at the Annual Meeting for the American Association of Cancer Research (AACR). This included the recommended dose of EVT801 for subsequent Phase II trials (if approved), as well as preliminary biomarker
and clinical data focused on the advanced ovarian cancer patients enrolled in the study. Post reporting period in September, we were invited to present data highlighting promising clinical activity of EVT801 in high grade serous (HGS) Ovarian Cancer
at the 15th Biennial Ovarian Cancer Research Symposium. This event was co-presented by American Association of Cancer Research (AACR) and the Rivkin Center for Ovarian Cancer Research in Seattle Washington
,and was attended by clinicians and ovarian cancer researchers from around the globe. The evolution of our clinical program for both paxalisib and EVT801, and the data reported in FY24, gives us continued confidence in their potential to improve
patient outcomes in devastating disease areas. We anticipate further milestones in the year ahead, including the finalisation of late-stage clinical trials and potential regulatory filings if supported by the FDA. BOARD AND MANAGEMENT TRANSITIONS
During FY24, Kazia has undergone several key changes within the executive team, aimed at strengthening our leadership and positioning the company for future growth. In August 2023, Dr John Friend joined the Board of Directors as Managing Director,
having assumed the role of Chief Executive Offer earlier in the year. Kazia also welcomed Mr Bryce Carmine as Chairman in January 2024, following the resignation of Mr Iain Ross as Non-Executive Chairman in
August 2023. Mr Carmine has served on the Kazia Board since 2015 and has extensive experience in drug development and global healthcare leadership. At the same time, we welcomed Mr Robert Apple to the Board as a
Non-Executive Director. Mr Apple, bringing more than 25 years of senior leadership experience in the pharmaceutical industry to Kazia. These leadership transitions are reflective of our commitment to
ensuring we have the right team in place to drive the next phase of Kazia s growth, as we advance our clinical programs and pursue new strategic initiatives.
FINANCIAL PERFORMANCE & OUTLOOK Kazia s cash balance at 30 June 2024 was $1.7m versus $5.2m at 30 June 2023. Our
total assets were $21.6m, compared to $28.1m at 30 June 2023. Financially, Kazia remains well-positioned to execute its strategic initiatives. In December 2023 we announced a $2m registered direct offering upon entering into a definitive
agreement for the purchase and sale of up to 4,44,445 of our American Depository Shares (ADSs). As mentioned earlier, we were pleased to enter into an exclusive licensing agreement with Sovargen, receiving an upfront payment of U$1.5M, with
potential milestone payments of up to $19M. This was followed in April 2024 with a purchase agreement with Alumni Capital to sell up to $15m of American Depository Shares (ADSs). In addition to these agreements, Kazia has demonstrated a judicious
use of our at-the-market (ATM) offering to maximise capital raised while minimising dilution for our existing shareholders, reflecting a careful balance between growth
and shareholder value. While challenges remain in the global healthcare environment, we are optimistic about the future. We will continue to take a disciplined approach to capital management and the strength of our clinical data supports our belief
that we are on the right path toward delivering meaningful new treatments to cancer patients. LOOKING AHEAD As we move forward into FY25, our priorities remain clear. We will continue to advance paxalisib and EVT801 through their clinical
development and explore opportunities to broaden our pipeline through strategic collaborations. Our overarching goal is to create a world where cancer patients have access to better, more effective treatments, and we are unwavering in our commitment
to this vision. We would like to thank our fellow board, management team, shareholders, partners, and employees for their continued support and dedication. Together, we are making tremendous progress, and we are excited for what lies ahead. Mr Bryce
Carmine Dr John Friend Chairman Chief Executive Officer Chairman and CEO s Letter Key Milestones About GBM Environment, Society and Governance Financial Reports 5 Kazia Theraputics Limited Annual Report 2024
key Milestones HIGHLIGHTS 2023/2024 23 2323 24 23 23 JAN JUL AUG SEP OCT NOV Kazia s lead drug Dr John Friend, A
late-breaking Kazia announced Kazia voluntarily Kazia announced asset paxalisib Kazia s Chief oral presentation its intention delisted from the the resignation was awarded Executive Officer, at the 2023 to voluntarily ASX to become of Chief
Financial Fast Track was appointed Society for de-list from solely traded Officer Karen Designation Managing Neuro-Oncology the Australian on NASDAQ. Krumeich. (FTD) by the Director to (SNO) Annual Securities
Concurrently, United States the Board of Meeting was Exchange (ASX) Kazia shared Non-Executive Food and Drug Directors. In awarded to and remain on key highlights Director Bryce Administration other changes,
Kazia, presenting the NASDAQ from the clinical Carmine was (FDA) for the Mr Iain Ross data from the Exchange and preclinical appointed as treatment of resigned as ongoing phase II to reduce paxalisib-related the Chairman to solid tumour Non-Executive study of paxalisib administrative presentations Kazia board, and brain metastases Chairman and for the treatment costs and more given by key Mr Robert Apple harbouring CEO Dr John of diffuse intrinsic
readily access thought leaders was appointed as PI3K pathway Friend assumed pontine glioma capital from a at the Society Non-Executive mutations in the role of Interim (DIPG) and other wider investor of Neuro-
Director. combination with Chairman. diffuse midline base to deliver Oncology 2023 radiation therapy. gliomas (DMGs), on clinical Annual Meeting, sponsored by the development including highly Pacific Paediatric plans. Kazia was encouraging
Neuro-Oncology officially delisted preliminary Consortium from the ASX in overall survival (PNOC). November 2023. data from the PNOC022 clinical Data from the trial study with company s diffuse midline ongoing phase gliomas patients. I clinical
trial evaluating EVT801 in patients with advanced solid tumours was presented at the European Society of Medical Oncology Congress 2023. 6
Chairman and CEO s Letter 24 24 24 24 24 24 REPORTING FEB MAR MAY JUN JUNPOSTPERIOD JUL Key Kazia announced Kazia announced Kazia
announced Kazia announced Jeffrey Kazia announced Milestones the early the presentation that the Safety the presentation Bonacorda was the results from conclusion of of new data for Review Team of new data from appointed as the GBM-AGILE, an investigator- both its pipeline (SRT) of the its lead program, Vice President a phase II/ initiated trial molecules, EVT801 phase paxalisib, at the of Finance and III study. Data evaluating the
paxalisib and I clinical trial 21st International Controller, showed clinically use of paxalisib EVT801, at the concluded that Symposium starting July meaningful with radiation Annual Meeting the primary on Paediatric 1, 2024. Mr. improvement
in therapy, for the of the American and secondary Neuro-Oncology Bonacorda a prespecified treatment of Association of objectives (ISPNO 2024). brings over secondary About patients with Cancer Research of stage 1 of Concurrently, thirty years of
analysis for GBM PI3K pathway (AACR). the trial had Kazia announced experience in the overall survival in mutation brain successfully the publication pharmaceutical, paxalisib-treated, metastases from Kazia entered been met. of an article in
consumer newly diagnosed solid tumours. into an exclusive the European products unmethylated The clinical trial licensing Journal of and service patients with was observed to agreement Cancer, which industries. glioblastoma. reach its primary with
Sovargen highlighted Based on the endpoint. to develop, the need for totality of the manufacture and evaluating GBM clinical commercialise mutation- data generated paxalisib as specific, CNS to date, Kazia a potential penetrant, is planning on
Environment, treatment of inhibitors to meeting with the intractable treat paediatric FDA to discuss Society epilepsy in focal patients with the data and and cortical dysplasia Diffuse Midline a regulatory type 2 (FCD T2) Glioma (DMG). path forward.
and tuberous sclerosis complex Governance (TSC) disease. Financial Reports Kazia Theraputics Limited Annual Report 2024 7
About GBM WHAT IS GLIOBLASTOMA? Glioblastoma (glioblastoma PAXALISIB AS A POTENTIAL TREATMENT multiforme or GBM) is the most
Glioblastoma is the lead indication for paxalisib, Kazia s lead drug assets. Paxalisib is a unique PI3K common and most aggressive form inhibitor that was invented by Genentech, Inc. PI3K of brain cancer. is a critical control mechanism in
growth and cell division, which is activated in many forms of cancer. Originating in the adult brain, glioblastoma tumours PI3K is an attractive target for glioblastoma because comprise cancer cells that can rapidly grow and 85% of the patients have
activation of this pathway. multiply. In rare cases, glioblastoma can spread into PI3K is a well-validated target for cancer drugs, with other areas of the brain and spinal cord. There is no five FDA-approved
therapies in this class: Zydelig clear cause, or strong risk factors for glioblastoma. It (idelalisib), Aliqopa (copanlisib), Copiktra (duvelisib), can develop at any age, but most commonly in people Piqray (alpelisib), and
Ukoniq (umbralasib). One in their 60s. It is estimated that there are 133,000 of the most distinguishing features of paxalisib is its people are diagnosed with glioblastoma each year, with ability to cross the blood-brain barrier (BBB).
Ordinarily, an average life expectancy of approximately 15 weeks1. the BBB prevents many drugs from reaching brain In the last 20 years, there has been very little tissue, and is a challenge for the treatment of any improvement in prognosis for
patients with disease in the central nervous system. Paxalisib s glioblastoma. The current standard of care, also ability to cross the BBB is very unique in this class known as Stupp s regimen, consists of debulking of medicines and
differentiates the drug from the surgery where possible, followed by six weeks of approved products in the PI3K inhibitor class. radiotherapy combined with temozolomide treatment. PAXALISIB AND GMB AGILE TRIAL Then after a four-week interval,
patients are back on temozolomide maintenance therapy for six 28- On 10 July 2024, Kazia announced results from day cycles. Additional treatment approaches are GBM AGILE, a phase II/III study that
included an considered for older patients who are less tolerant to evaluation of paxalisib versus standard of care Stupp s regimen, such as Perry s modification (3 weeks (SOC) for patients with glioblastoma. GBM AGILE of chemoradiotherapy)
or by chemotherapy alone2. is sponsored by the Global Coalition for Adaptive Research, a US-based, not-for-profit organisation
consisting of some of the world s foremost clinical, Even a few months increase in translational and basic science researchers. In 2019, Kazia announced its inclusion in GBM overall survival makes a huge AGILE and the first patient was
enrolled in 2021 difference for my patients, so into the paxalisib arm of the study. Paxalisib was evaluated in Newly Diagnosed Unmethylated (NDU) efficacy of an approved therapeutic patients as well as recurrent GBM patients. makes the largest
impact. Paxalisib is the third drug candidate to complete its evaluation in the study and was evaluated A US Neuro-Oncologist as a treatment in two patient signatures NDU (N=54) and recurrent disease (N=100). Data on file.
Chairman and CEO s The NDU control arm of GBM AGILE was the current SOC (Stupp protocol, comprising 6 weeks of chemoradiation
Letter followed by adjuvant TMZ). As GBM AGILE is an ongoing adaptive study, where control patients are continuously enrolled, two cohorts of control patients could be used in statistical analyses concurrent controls and cumulative controls.
These are defined as follows: Paxalisib Paxalisib stage 1 stage 2 0 50 100 150 200 Key Evaluate for graduation Milestones Evaluate for futility Paxalib Study enrolment completed stops 12 months after Time adjusted controls Concurrent controls
last patient enrolled (20 months) (14 months) July 2019 March 2021 May 2022 May 2023 About GBM Cumulative control arm Concurrent controls: patients randomized to 3.8-month, approximately 33%
improvement in the control arm from the date of inclusion of patient overall survival comparing NDU patients paxalisib [April 2021] onto the study until the receiving paxalisib to the concurrent SOC group. date the last patient randomized to
paxalisib. For the primary analysis the median OS was 14.77 Concurrent control patients are censored one year months for paxalisib-treated NDU patients (n=54) versus after last patient is enrolled in Paxalisib arm. 13.84 months for cumulative SOC