On November 15, 2023 Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, reported that the Society of Neuro-Oncology 2023 Annual Meeting late breaking abstract entitled "PNOC022: a combination therapy trial using an adaptive platform design for patients with diffuse midline gliomas (DMGs) at initial diagnosis, post-radiation therapy and at time of therapy" was released by the conference organizers on 10 November 2023 (Press release, Kazia Therapeutics, NOV 15, 2023, View Source [SID1234637717]). The preliminary results from a single cohort in this Pacific Pediatric Neuro-Oncology Consortium (PNOC) sponsored cooperative group clinical study will be presented on Sunday, 19 November 2023 in Vancouver, Canada. Sixty-eight children and young adults with DMG who completed standard of care radiation treatment received paxalisib (an investigational PI3K-mTOR dual inhibitor) and ONC201 (an investigational dopamine receptor D2 (DRD2) and ClpP agonist). The abstract reported that median overall survival (OS) from time of diagnosis was 16.5 months.
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"We are very excited to see the OS preliminary results of 16.5 months from this cohort of patients, keeping in mind there are two other cohorts of patients in this study for which data is anticipated in 2024. The average (median) OS rate for children with DMG receiving standard of care is less than 1 year, generally ranging from 8-11 months," stated John Friend, MD, CEO Kazia Therapeutics. "The PNOC022 study has exceeded expectations in terms of enrolment rates and has partnered with 29 leading children’s cancer centers and physicians across the globe."
Key Points from the Abstract
Sixty-eight patients with biopsy-proven DMG were enrolled between November 2021 and June 2023 (median age 9 years [range 3-37], n=41 female [60%])
Median OS from time of diagnosis was 16.5 months (lower 95% confidence interval (CI) 11.6 months) with a median follow-up time of 9.9 months (95% CI: 8.5, 11.4)
Most common grade 3 and above treatment-related adverse events were decreased neutrophil count (n=4); mucositis (n=3); and, colitis, drug reaction with eosinophilia and systemic symptoms, decreased lymphocyte count, hyperglycemia, and hypokalemia (n=2)
About the PNOC022 Phase 2 study
The PNOC DMG Adaptive Combination Trial (PNOC022) Phase 2 study is an adaptive platform study that is examining paxalisib in combination with ONC201, an experimental DRD2 antagonist developed by Chimerix, Inc. (Durham, NC). PNOC022 is enrolling children and young adults with DMG, a category of brain tumours that includes diffuse intrinsic pontine glioma (DIPG). The study includes three cohorts comprising of newly diagnosed patients, patients who have completed initial radiotherapy, and patients who have experienced disease progression after treatment. The primary endpoint will be the proportion of patients who are progression-free at six months for newly diagnosed patients, and OS for recurrent patients.
Rare Pediatric Disease Designation
In 2020, the United States Food and Drug Administration (FDA) awarded Rare Pediatric Disease Designation (RPDD) to paxalisib for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG). With RPDD granted, Kazia may now be eligible to receive a ‘priority review voucher’ (PRV) if paxalisib is first approved for DIPG. A PRV grants the holder an expedited six-month review of a new drug application by FDA. PRVs can be sold to other companies and have historically commanded prices in excess of US$100 million.
This announcement was authorized for release by Dr. John Friend, CEO.