Proactive Article - Kazia Therapeutics lays solid foundation for...

Proactive Article - Kazia Therapeutics lays solid foundation for further development of pipeline in 2021
5 January 2021

The company is developing innovative, high-impact drugs for cancer and has one of the leading assets in the global glioblastoma pipeline with potential to address a US$1.5 billion market.

Kazia is listed on ASX and NASDAQ with a market capitalisation of approximately US$125 million.

Kazia Therapeutics Ltd (ASX:KZA) (NASDAQ:KZIA) has laid a solid foundation for further development in 2021 of a pipeline that includes two clinical-stage drug development candidates.

The lead program is paxalisib (formerly GDC-0084), a small molecule inhibitor of the PI3K/AKT/mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults.

Kazia is moving confidently along a path towards commercialisation supported by encouraging progress in 2020 and a number of upcoming milestones.

Another candidate is TRX-E-002-1 (Cantrixil), a third-generation benzopyran molecule with activity against cancer stem cells, which is being developed to treat ovarian cancer.

Key objectives
CEO James Garner will outline in a presentation today to the HC Wainwright & Co BIOCONNECT conference that one of Kazia’s key 2021 objectives is to execute GBM AGILE, a platform study designed by leading brain cancer experts to expedite the approval of new drugs for glioblastoma– the deadliest brain cancer.

The study is currently operational at around 31 sites in the United States and Canada, with steps taken to expand to Europe and China in 2021.

Cutting-edge ‘adaptive design’ ensures that the study will only recruit the number of patients needed to reach an answer (up to 200 on paxalisib), avoiding redundancy and ensuring the fastest possible path to market.

GBM AGILE has received substantial grant funding thus reducing the cost of participation for companies such as Kazia.

Simultaneously, the company also plans to drive data from the broader paxalisib program while intensifying partnering activity for the candidate and is also planning to start rolling new drug application (NDA) filing activities with authorities.

Milestones and newsflow
In October and November of 2020, the company executed a definitive agreement with Global Coalition for Adaptive Research (GCAR), a not-for-profit entity based in the United States, for the GBM AGILE pivotal study while furthering interim data from the phase II glioblastoma trial.

The company also received initial interim data in November from the phase I DIPG trial at St Jude and is hoping to receive interim data from a phase II BCBM trial at Dana-Farber in the first half of 2021.

Kazia plans to start recruitment during the current quarter to the GBM AGILE pivotal study in glioblastoma, to the PNOC combination study in DIPG and to the phase II PCNSL study at Dana-Farber.

In the first half of this year, the company expects to receive initial interim data from a phase II brain mets study by Alliance Group along with initial interim data from a phase I brain mets study at Sloan-Kettering.

During the same period, it is also hoping to complete the final data from Kazia phase II glioblastoma trial.

Lead candidate paxalisib
Kazia’s lead candidate paxalisib was licensed from Genentech in late 2016 and has a well-proven mechanism of action with the unique differentiating factor of brain penetration along with a strong scientific rationale for development in brain cancer.

Recent regulatory achievements position paxalisib well as it moves towards commercialisation.

Paxalisib entered GBM AGILE, a pivotal study in glioblastoma, in October 2020 and 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.

Besides, paxalisib was granted Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020.

The company is encouraged by clinical data emerging from US-based phase II study with potentially best-in-class toxicity profile.

The segment represents around US$1.5 billion in commercial opportunities along with high unmet medical needs as temozolomide, the only FDA-approved drug for GBM, is ineffective in around 65% of cases.

Paxalisib is being developed for those GBM patients who are not responding to existing treatment.

Ovarian cancer candidate
Kazia’s other lead candidate is TRX-E-002-1 (Cantrixil), a third-generation benzopyran molecule with activity against cancer stem cells, is being developed to treat ovarian cancer.

It has completed a phase I clinical trial in Australia and the United States.

Cantrixil was granted orphan designation for ovarian cancer by the US FDA in April 2015.

**promotion blocked**.au/companies/news/937629/kazia-therapeutics-lays-solid-foundation-for-further-development-of-pipeline-in-2021-937629.html

looking forward to 2021 as the interim data is revealed.

Regards.