Core Viewpoint - Kazia Therapeutics Limited plans to hold a follow-up Type C meeting with the U.S. FDA to discuss overall survival findings in newly diagnosed glioblastoma patients treated with paxalisib and to seek feedback on a potential regulatory pathway aligned with the FDA's Project FrontRunner initiative [1][2]. Regulatory Strategy - The company aims to engage the FDA to determine if the overall survival data from newly diagnosed glioblastoma patients treated with paxalisib can support a conditional approval pathway [2]. - Kazia intends to propose the initiation of a post-approval, randomized Phase 3 confirmatory study prior to submitting the New Drug Application (NDA), ensuring alignment with the FDA's emphasis on overall survival as a primary endpoint [2][5]. Project FrontRunner Initiative - Project FrontRunner is an FDA initiative encouraging earlier approval of cancer drugs for advanced or metastatic diseases, rather than only for patients who have exhausted treatment options [3]. - Kazia plans to reference Project FrontRunner principles in its Type C briefing package, including an overall survival-driven confirmatory study plan for newly diagnosed glioblastoma [4]. Clinical Data - In a prespecified secondary analysis, the median overall survival for newly diagnosed unmethylated glioblastoma patients treated with paxalisib was reported as 15.54 months, compared to 11.89 months for the standard of care [4]. - The company emphasizes the importance of overall survival as a meaningful endpoint for patients and clinicians in the context of glioblastoma treatment [2][5]. Company Background - Kazia Therapeutics is an oncology-focused drug development company based in Sydney, Australia, with paxalisib as its lead program, targeting multiple forms of cancer [7]. - Paxalisib has undergone ten clinical trials and has received various designations from the FDA, including Orphan Drug Designation and Fast Track Designation for glioblastoma [7][8].

Core Insights - Immutep Limited has received positive feedback from the FDA regarding the clinical development of its MHC Class II agonist, eftilagimod alfa (efti), for first-line treatment of recurrent/metastatic head and neck squamous cell carcinoma (HNSCC) patients with PD-L1 expression below 1 [1][2][4] Clinical Development - The FDA acknowledged the potential of efti in combination with Merck's KEYTRUDA (pembrolizumab) for patients with CPS <1, indicating support for further development of this combination therapy [2][4] - Future clinical development paths may include a randomized registrational trial comparing efti with KEYTRUDA against standard-of-care therapy or a smaller single-arm study focusing on safety and response rates, followed by a confirmatory randomized study [3][6] Market Need - Patients with CPS <1 in 1L HNSCC represent a significant unmet medical need, with up to 20% of these patients not benefiting from current anti-PD-1 therapies, which are only approved for those with PD-L1 expression (CPS >1) [7] - Current treatment options for patients with PD-L1 CPS <1 are limited to chemotherapy [7] Company Focus - The primary focus of Immutep remains on the pivotal TACTI-004 Phase III trial evaluating efti as first-line therapy for non-small cell lung cancer, with positive feedback from physicians regarding its progress [5] - The company is considering future collaborative clinical development paths for head and neck cancer based on FDA feedback [4][5] About Immutep - Immutep is a late-stage biotechnology company specializing in immunotherapies for cancer and autoimmune diseases, particularly focusing on Lymphocyte Activation Gene-3 (LAG-3) [8] - The company aims to leverage its expertise to provide innovative treatment options and maximize shareholder value [8]