Full Press Release Details
KAZIA CORPORATE UPDATE AND
CORPORATE PRESENTATION
Sydney, 26 June 2023 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development
company, is pleased to provide an update on key corporate and clinical initiatives and release a new investor presentation.
Following his appointment as CEO on 1 May 2023, Dr John Friend has completed his transition into the role. He commented, In the weeks since my
appointment, I ve worked with the team to review the full set of assets and clinical trials within the Kazia portfolio and the process has strongly reinforced my conviction in the opportunities ahead of us.
We believe substantial potential exists across our pipeline and in particular, across three key pillars of development for paxalisib adult brain
cancer, paediatric brain cancer and brain metastases. We are keen to explore paxalisib s use in patient populations that may have the greatest benefit from a PI3K/AKT/mTOR targeted therapeutic candidate.
As we move toward the second half of 2023, critical milestones are approaching for both paxalisib and EVT-801.
We look forward to keeping investors updated across our progress on these important programs.
Clinical programs update
11 clinical trials are currently in progress or in planning for Kazia s two assets, paxalisib and EVT-801, and
Kazia continues to execute across all programs. Current program highlights are set out in brief below. Refer to the appended corporate presentation for more detail:
Kazia s lead program is paxalisib,
an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in
various brain cancers.
GBM AGILE Pivotal study
Final data are expected from the GBM AGILE pivotal study of paxalisib in glioblastoma in 2H CY2023. Depending on the results of the study, Kazia may use such
data to support submission of a new drug application for marketing authorisation to the FDA.
PNOC022 phase II study
Paxalisib is being studied as an investigational drug for the treatment of the paediatric brain cancers DIPG and other DMGs in a study sponsored by the Pacific
Pediatric Neuro-Oncology Consortium (PNOC).
The study team at PNOC has been diligently collecting and preparing the data for interim analysis. At this
point, Kazia expects that the data will be available in July or August 2023.
LUMOS2 phase II study
On 8 June 2023, Kazia announced that it is supporting the University of Sydney on a molecularly-guided phase II clinical study evaluating paxalisib in
adult patients with recurrent/progressive isocitrate dehydrogenase (IDH) mutant grade 2 and 3 gliomas (G2/3 gliomas).
The study, named LUMOS2, will be
sponsored by the University of Sydney, and coordinated by NHMRC Clinical Trials Centre, University of Sydney, in collaboration with COGNO (CoOperative Trials Group for Neuro-Oncology). LUMOS2, an umbrella study with multiple arms, is expected to
enroll up to 76 patients and will be a multi-centre study at several Australian sites, with the potential to expand internationally. We anticipate enrollment to commence in 4Q23.
MSKCC phase I clinical study
Paxalisib is the subject of an ongoing phase I clinical study in combination with radiotherapy for the treatment of brain metastases, sponsored by Memorial
Sloan Kettering Cancer Center in New York, NY.
Kazia is pleased to confirm that the phase I expansion cohort is enrolling. Two world-renowned cancer
centres have joined MSKCC in this study: Miami Cancer Institute and Fred Hutch Cancer Center in Seattle, WA. Preliminary data from the expansion cohort is anticipated by 1Q24.
Kazia is also developing EVT801, a
small-molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021. Preclinical data showed EVT801 to be active against a broad range of tumour types and has shown evidence of synergy with immuno-oncology agents. A phase I study
commenced recruitment in November 2021.
EVT-801 continues to enroll in the phase I dose finding study. Kazia
anticipates stage 1 data in 2H23, which we believe will enable identification of the recommended dose for subsequent phase II trials. Kazia also anticipates reporting preliminary biomarker data focused on high-grade, serous ovarian cancer as part of
About Kazia Therapeutics Limited
Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA) is an oncology-focused drug development company, based in Sydney, Australia.
Our lead program is paxalisib, a brain-penetrant inhibitor candidate of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of
brain cancer. Licensed from Genentech in late 2016, Paxalisib is or has been the subject of ten clinical trials in various brain cancers. A completed phase II study in glioblastoma reported promising signals of clinical activity in 2021, and a
potentially pivotal study for registration, GBM AGILE, is ongoing, with final data expected in CY2023. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported
encouraging interim data.
Paxalisib was granted Orphan Drug Designation for glioblastoma by the US FDA in February 2018, and Fast Track Designation for
glioblastoma by the US FDA in August 2020. In addition, Paxalisib was granted Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020, and for atypical teratoid / rhabdoid tumours (AT/RT) in June 2022 and July
Kazia is also developing EVT801, a small-molecule inhibitor candidate of VEGFR3, which was licensed from Evotec SE in April 2021.
Preclinical data has shown EVT801 to be active against a broad range of tumour types and has shown evidence of synergy with immuno-oncology agents. A phase I study commenced recruitment in November 2021.
This document was authorized for release to the ASX by John Friend, Chief Executive Officer.
Forward-Looking Statements
This announcement may contain
forward-looking statements, which can generally be identified as such by the use of words such as may, will, estimate, future, forward, anticipate, plan,
expect, potential, explore, or other similar words. Any statement describing Kazia s future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not
historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for interim or final results and data related to Kazia s clinical and preclinical trials, or third-party trials evaluating
Kazia s product candidates, timing and plans with respect to enrolment of patients in Kazia s clinical and preclinical trials and Kazia s strategy and plans with respect to its programs, including paxalisib and EVT-801. Such statements are based on Kazia s current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could
cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, related
to Kazia s executive leadership changes, and related to the impact of global economic conditions, including disruptions in the banking industry. These and other risks and uncertainties are described more fully in Kazia s Annual Report,
filed on form 20-F with the SEC, and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events,
or otherwise, except as required under applicable law. You should not place undue reliance on these forward looking statements, which apply only as of the date of this announcement.
A Diversified Oncology Drug Development Company June 2023 ASX: KZA |
NASDAQ: KZIA | Twitter: @KaziaTx
Forward-Looking Statements This presentation contains forward-looking
statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," "plan,"
"expect," "explore," "potential" or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not
historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for interim or final results and data related to Kazia's clinical and preclinical trials, or third-party trials evaluating
Kazia's product candidates, timing and plans with respect to enrolment of patients in Kazia's clinical and preclinical programs and Kazia's strategy and plans with respect to its programs, including Paxalisib and EVT-801. Such
statements are based on Kazia's expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those
anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, risks related to Kazia's executive leadership changes,
and the related to the impact of global economic conditions, including disruptions in the banking industry. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the SEC, and in
subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place
undue reliance on these forward-looking statements, which apply only as of the date of this presentation. 1
Company Overview A late-clinical-stage oncology drug development company
Paxalisib EVT801 Company is dual-listed on NASDAQ (KZIA) and ASX (KZA) with market cap around US$ 27 million Brain-penetrant pan-PI3K / mTOR inhibitor Selective VEGFR3 inhibitor Well-validated class with five FDA-approved therapies
Designed to avoid off-target toxicity of older Only brain-penetrant PI3K inhibitor in development angiokinase inhibitors Primarily targets lymphangiogenesis Licensing-driven business model, with programs sourced from Genentech
(paxalisib) and Sanofi / Evotec In development for multiple brain cancers Currently in phase I for advanced solid tumors (EVT801) Clinical trials ongoing in brain metastases, childhood Adaptive, biomarker study ongoing at 2 leading
cancer brain cancer, glioblastoma, IDH-mutant glioma, and sites in France primary CNS lymphoma Unique asset being evaluated in multiple trials Cash runway into 4Q23, with potential Potential use in many solid tumors opportunities for non-dilutive
income via Multiple signals of clinical activity across several Potential indications include: ovarian cancer, renal cell additional partnering activity cancer types carcinoma, liver cancer, colon cancer, and sarcoma Fast
Track, Orphan Drug, and Rare Pediatric Disease Designations from US FDA Rich potential commercial opportunity Potential combination with immunotherapy Lean virtual pharma model, with ~75% Glioblastoma alone sized at US$ 1.5 billion per
annum Strong evidence of synergy in preclinical data supports of cashflows applied directly to clinical Commercial licensee in place for China potential of monotherapy or combination use trials Final Phase III Data: Anticipated 2H
CY2023 Initial Phase I Data: Anticipated 2HCY2023 2
Pipeline - Two Differentiated Assets Lead asset provides a
pipeline in a molecule' licensed from: Paxalisib Investigational, small molecule, potent, brain-penetrant inhibitor of PI3K / mTOR Preclinical PHASE 1 PHASE 2 PHASE 3 MARKET Anticipated Final Data Glioblastoma & IDH-mutant glioma 3
studies 2H CY23 Common primary brain cancer Initial Data DIPG/Advanced Solid Tumors 3 studies 3Q CY23 Childhood brain cancer Further Data AT/RT 1H CY23 Childhood brain cancer Further Data Brain Metastases 3 studies CY23 Cancer that spreads to brain
from elsewhere Initial Data Primary CNS Lymphoma 1 study CY23 Form of non-Hodgkin's lymphoma Further Data Cancers outside the CNS CY23 Melanoma; TNBC, Ovarian licensed from: EVT801 Investigational, small molecule, highly specific inhibitor of
VEGFR3 Initial Data Advanced Solid Tumors 2H CY23 Patients w/ highly treatment-resistant cancer IDH: Isocitrate dehydrogenase, DIPG: Diffuse Intrinsic Pontine Glioma, AT/RT: Atypical Teratoid Rhabdoid Tumor, CNS: central nervous system, TNBC: triple
negative breast cancer, VEGFR3: vascular endothelial growth factor receptor 3 3
Paxalisib History Asset licensed from Genentech in 2016 Kazia commences
phase I clinical Kazia commences phase II Positive final data from phase II trial in diffuse midline glioma clinical trials in brain metastases glioblastoma study Partnership with St Jude Partnerships with leading US Basis
for launch of pivotal GBM Children's Research Hospital specialist cancer centres AGILE trial in Jan 2021 2016 2017 2018 2019 2020 2021 2022 Kazia licenses paxalisib from Kazia commences phase II Greater China rights licensed to Multiple
positive data read -outs Genentech, Inc clinical trial in glioblastoma Simcere Pharmaceutical in various cancers $5 million upfront payment 6 leading US based cancer $11 million upfront payment 2 brain mets studies
graduate Royalties on commercial sales centres Up to $281 million in milestone to expansion stages Asset licensed after strategic Focuses development on payments Compelling preclinical DIPG review by Genentech
refocused newly-diagnosed patients, a Mid- to high -teen royalties on data in combination with R&D on immuno-oncology and larger commercial opportunity net sales in greater China ONC201 presented tumors outside the brain and more
treatment - Positive preclinical data in responsive population AR/RT Positive preclinical data in melanoma Collaborations with Leading Brain Cancer Research Centres 4
CY2023 Milestones and Newsflow Multiple catalysts across two clinical
programs Preclinical data for paxalisib in melanoma presented at AACR (2023) 1H CY2023 Preclinical data for paxalisib in DMG/DIPG & AT/RT presented at AACR (2023) 1H CY2023 Preliminary data from Kazia's EVT801 phase I trial
presented AACR 2023 1H CY2023 Initiate OPTIMISE clinical study in pediatric PI3K/mTOR activated cancers 1H CY2023 Initiate LUMOS2 clinical study in adult IDH -mutant glioma patients w/PI3K/ mTOR mutations 1H CY2023 Initial
interim data from paxalisib phase II DIPG study with PNOC 3Q CY2023 EVT801 Stage 1 completion 2H CY2023 QIMR preclinical data and provisional patent update 2H CY2023 Initial interim data from paxalisib phase II PCNSL study at Dana -Farber 2H CY2023
Further interim data from paxalisib brain metastases trials 2H CY2023 Final data from GBM AGILE pivotal study of paxalisib 2H CY2023 Paxalisib + radiotherapy Stage 2 data phase I brain mets study at Memorial Sloan-Kettering 1Q CY2024 Note: all