Kazia Therapeutics is a late-stage clinical pharmaceutical company with lead asset paxalisib (a PI3K inhibitor that can cross the BBB, licensed from Genentech), in a pivotal study for GBM. It also recently in-licensed the Phase I drug EVT801, an inhibitor of lymphangiogenesis in tumors.
Investment summary
In an encouraging development for Kazia Therapeutics’ efforts towards combating pediatric brain cancers, its lead drug paxalisib has received orphan drug designation (ODD) from the FDA for the treatment of atypical teratoid/rhabdoid tumors (AT/RT). AT/RT is a rare and aggressive childhood brain cancer with a five-year survival rate of c 32%. Approximately 600 people are living with the cancer in the United States, with around 60 new cases reported each year. The ODD accords seven years of market exclusivity in the US on approval, in addition to possible grant funding and tax credits. Kazia is undertaking preclinical studies in AT/RT and has recently presented encouraging data from combination studies in xenograft models. As a reminder, paxalisib already has ODD in malignant gliomas, including glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG). Our valuation is unchanged at US$294m or US$22.28/ADR. Please see our Deep dive into childhood brain cancer note, published on 24 May.
GBM is the most common primary cancer of the brain with c 12,500–13,000 new cases reported in the United States per year. There are very limited treatment options for GBM and there is a very low survival rate. Paxalisib is currently being developed for use in the adjuvant setting after initial resection and radiation treatment. EVT801 will target the multi billion dollar angiogenesis cancer market.
