Kazia Therapeutics - Paxalisib awarded RPDD in AT/RT

Kazia Therapeutics has announced that the company’s brain-penetrant kinase inhibitor, paxalisib, has been granted Rare Pediatric Disease Designation (RPDD) for the treatment of atypical teratoid/rhabdoid tumors (AT/RT) by the Food and Drug Agency (FDA). The designation entitles Kazia to receive a pediatric priority review voucher (pPRV), should paxalisib be approved, which could expedite the FDA approval process for another asset to six months (usually c 10 months). pPRVs are transferable and are often valued at over $100m. Previously, paxalisib has also been awarded an orphan drug designation, granting a potential seven-year market exclusivity from approval. We view continued regulator recognition of paxalisib’s utility in AT/RT as encouraging support for Kazia’s development program. For further detail on paxalisib and the company’s strategy, see our Deep dive into childhood brain cancer. Our valuation is unchanged at US$294m or US$22.28/ADR.