Kazia Therapeutics - Paxalisib receives ODD for AT/RT

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.