GLOBAL RESEARCH ARCHIVE
Lighting Up the Doppler: Paxalisib is an Oral, Once-daily, Brain-penetrant Pan-PI3K/mTOR inhibitor with Early Efficacy Signals in TNBC
Research evidence excerpt
Lighting Up the Doppler: Paxalisib is an Oral, Once-daily, Brain-penetrant Pan-PI3K/mTOR inhibitor with Early Efficacy Signals in TNBC
Investment Thesis
Paxalisib is not simply a broader PI3K inhibitor, but a differentiated biology modulator with the potential to reprogram tumor
signaling through both PI3K/mTOR pathway inhibition and epigenetic as well as inflammatory mechanisms. Early Phase 1b Arm B
data in TNBC are compelling, with 2 evaluable patients achieving partial responses and an expanded access case demonstrating a
complete metabolic response, providing an encouraging early clinical signal in a high unmet need setting. We view the expansion of
Arm B target enrollment from 12 to 36 patients as an important next step that should provide a more meaningful read on response,
durability, and biomarker-driven patient selection. With TNBC as the primary value driver and additional optionality in GBM and
pediatric brain cancers, we believe paxalisib can continue to build on early efficacy and support a projected ~$500M peak salesBIOTECHNOLOGY opportunity. Kazia has approximately $46M in cash, providing runway for continued pipeline development.
Upcoming Catalysts Price Performance
■ 2H26: Additional clinical update from paxalisib TNBC Ph1b study $18EQUITY ■ 2H26: Start of NDL2 and MSETC IND-enabling studies $16
■ 2H26: Updates from neuro-oncology focused clinical trials $14
$12
$10
$8RESEARCH Base Case Assumptions: $34 Price Target $6
We believe Paxalisib’s differentiated profile supports a broader commercial $4
opportunity than traditional PI3K inhibitors, reflecting its brain-penetrant pan-PI3K/ Aug-25 Sep-25 Oct-25 Nov-25 Dec-25 Jan-26 Feb-26 Mar-26 Apr-26 May-26 Jun-26 Jul-26 mTOR activity and potential to favorably alter tumor biology and improve immune
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