A Diversified Oncology
Drug Development Company
Kazia Corporate Overview
April 2024
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
PaxalisibEVT801
Since delisting from Australian Securities Exchange (ASX) in November 2023, the Company has
Brain-penetrantpan-PI3K / mTOR inhibitor
- Well-validatedclass with five FDA-approved therapies
- Only brain-penetrant PI3K inhibitor in development
In development for multiple brain cancers
- Clinical trials ongoing in brain metastases, childhood brain cancer, glioblastoma, IDH-mutant glioma, and primary CNS lymphoma
Unique asset being evaluated in multiple trials
- Multiple signals of clinical activity across several cancer types
- Fast Track, Orphan Drug, and Rare Pediatric Disease Designations from US FDA
Rich potential commercial opportunity
- Glioblastoma alone sized at US$ 1.5 billion per annum
- Commercial licensee in place for China
- Licensee for intractable seizures in rare CNS diseases
Top Line Ph. 3 Data: Anticipated 2Q CY2024
Selective VEGFR3 inhibitor
- Designed to avoid off-target toxicity of older, non- selective angiokinase inhibitors
- Primarily targets lymphangiogenesis
Currently in phase I for advanced solid tumors
- Adaptive, biomarker study ongoing at 2 leading cancer sites in France
Potential use in many solid tumors
- Potential indications include: ovarian cancer, renal cell carcinoma, liver cancer, colon cancer, and sarcoma
Potential combination with immunotherapy
- Strong evidence of synergy in preclinical data supports potential of monotherapy or combination use
Phase 1 Data: Anticipated 3Q CY2024
been solely listed on NASDAQ (KZIA)
Licensing-driven business model, with programs sourced from Genentech (Paxalisib) and Sanofi / Evotec (EVT801)
Potential opportunities for non-dilutive
income via additional partnering
activity
Lean virtual pharma model, with
~75% of cashflows applied directly to
clinical trials
2
Pipeline - Two Differentiated Assets
CY2024 anticipated data releases has the potential to be transformative
Paxalisib
Investigational, small molecule, potent, brain-penetrant inhibitor of PI3K / mTOR
licensed from:
Preclinical PHASE 1 PHASE 2 PHASE 3 MARKET
Glioblastoma & IDH-mutant glioma | |||
3 studies | |||
Common primary brain cancer | |||
DIPG/Advanced Solid Tumors | |||
3 studies | |||
Childhood brain cancer | |||
AT/RT | |||
Childhood brain cancer | |||
Brain Metastases | |||
3 studies | |||
Cancer that spreads to brain from elsewhere | |||
Primary CNS Lymphoma | |||
1 study | |||
Form of non-Hodgkin's lymphoma | |||
Cancers outside the CNS | |||
TNBC, Ovarian | |||
EVT801
Investigational, small molecule, highly specific inhibitor of VEGFR3
Advanced Solid Tumors
Patients w/ highly treatment-resistant cancer
Anticipated
Phase 3 Data
2Q CY24
PNOC022
data
2Q CY24
Further Data
2Q CY24
Further Data
CY24
Initial Data
CY24
Further Data
CY24
licensed from:
Initial Data
3Q CY24
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
4
Paxalisib Mechanism of Action
Only brain-penetrant drug in development within the PI3K inhibitor class
1 The PI3K pathway is activated in | 2 Five PI3K inhibitors have already | 3 Paxalisib is the only brain- | ||
many forms of cancer | been approved by FDA | penetrant PI3K inhibitor | ||
Glioblastoma | 90% | • | Chronic lymphocytic | Only 2% of small-molecule |
leukemia | ||||
• | Follicular lymphoma | drugs are brain-penetrant | ||
Breast | 80% | • | Follicular lymphoma | |
Lung | 75% | • | Chronic lymphocytic | |
leukemia |
- Follicular lymphoma
Endometrial 60%
• Breast cancer
Ovarian 60%
• Follicular lymphoma | Not able to cross blood-brain barrier |
Able to cross blood-brain barrier |
Prostate 45%
Source: Data on file
5
Glioblastoma
6
Paxalisib in Glioblastoma
High unmet need, especially in 'MGMT unmethylated' patients
Standard | |||||||
of Care | Debulking surgery | Radiotherapy + | Temozolomide | ||||
('Stupp | where possible | temozolomide | maintenance therapy | ||||
Regimen') | |||||||
6 weeks | 4w | 6 x 28-day cycles | |||||
Methylated MGMT Status |
~35% of patients respond to temozolomide
Extends overall survival from 15 to 22 months
Unmethylated MGMT Status
~65% of patients don't respond to temozolomide
Extends overall survival from 12 to 13 months
Source: ME Hegi, A-C Diserens, T Gorlia, et al. (2005). N Engl J Med 352:997-1003
Paxalisib is being developed primarily for the ~65% of newly-diagnosed GBM patients who will not respond to existing chemotherapy with temozolomide
For these patients, there is no effective pharmacological treatment currently available
Note: Temozolomide is only approved therapy for newly-diagnosed patients; Avastin (bevacizumab) is approved for use in recurrent setting
MGMT: O6-methylguanine-DNA methyltransferase
7
Paxalisib in Glioblastoma
Phase II study suggests encouraging PFS and OS in context of current treatment landscape
Progression-Free Survival (PFS) | Overall Survival (OS) | |
(n=30) | (n=30) |
Median PFS: 8.6 months (6.6-11.0) | Median OS: 15.7 months (11.1-19.1) | |
Comparator figure for existing therapy: 5.3 months
(Hegi et al. 2005)
Comparator figure for existing therapy: 12.7 months
(Hegi et al. 2005)
Note: Figures for existing therapy are for temozolomide, per Hegi et al. (2005); No head-to-head studies have been published
8
Paxalisib in Glioblastoma
Safety profile in Phase II study is favorable for a drug in advanced cancer
Number of Patients at Any Dose (n=30) Experiencing AEs 'Possibly' or 'Likely' Related to Paxalisib (affecting ≥10% of patients)
Term | Gr 1 | Gr 2 | Gr 3 | Gr 4 | Total (%) |
Fatigue | 3 | 13 | 2 | 18 (60%) | |
Stomatitis | 4 | 7 | 3 | 14 (47%) | |
Decreased appetite | 6 | 6 | 1 | 13 (43%) | |
Hyperglycemia | 3 | 1 | 6 | 2 | 12 (40%) |
Nausea | 4 | 6 | 1 | 11 (37%) | |
Rash, maculo-popular | 1 | 1 | 7 | 9 (30%) | |
Diarrhea | 7 | 1 | 8 (27%) | ||
Vomiting | 4 | 2 | 1 | 7 (23%) | |
Rash | 2 | 4 | 1 | 7 (23%) | |
Neutrophils decreased | 3 | 3 | 1 | 7 (23%) | |
Platelets decreased | 6 | 1 | 7 (23%) | ||
Weight decreased | 5 | 2 | 7 (23%) | ||
Lymphocytes decreased | 2 | 3 | 5 (17%) | ||
Dehydration | 4 | 1 | 5 (17%) | ||
Dysgeusia | 4 | 4 (13%) | |||
Cholesterol increased | 4 | 4 (13%) | |||
ALT increased | 1 | 2 | 3 (10%) | ||
Triglycerides increased | 1 | 2 | 3 (10%) | ||
Malaise | 2 | 1 | 3 (10%) | ||
9
Attachments
- Original Link
- Original Document
- Permalink
Disclaimer
Kazia Therapeutics Limited published this content on 12 April 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 12 April 2024 00:42:02 UTC.