Introduction to GDC-0084
GDC-0084, now known as paxalisib, is a novel drug candidate developed by Kazia Therapeutics Limited, an Australian biotech company, for the treatment of glioblastoma multiforme (GBM), the most aggressive and common form of primary brain cancer. Here’s a detailed update on its development and market projections.
Development History of GDC-0084
Initial Development and Licensing
GDC-0084 was initially developed by Genentech and later in-licensed by Kazia Therapeutics in 2016 following a successful Phase I safety study that also showed signs of efficacy. This drug is distinguished by its ability to cross the blood-brain barrier, a significant challenge for many brain cancer treatments[1][3][4].
Phase I and Early Clinical Data
The Phase I study for advanced solid tumors demonstrated the drug's safety and preliminary efficacy. This positive data paved the way for further clinical trials focused specifically on brain cancers[3].
Phase II Clinical Trials
In March 2018, Kazia initiated a Phase II clinical trial for GDC-0084 in patients with glioblastoma. The trial is designed to evaluate the drug's efficacy in newly diagnosed patients who are resistant to the standard treatment, temozolomide. Recruitment began at the Stephenson Cancer Center at the University of Oklahoma, with additional sites in the US and Australia expected to follow[1][4].
Recent Clinical Updates
As of 2024, the GBM-AGILE Phase II/III trial has shown clinically meaningful improvements in overall survival for newly diagnosed, unmethylated patients with glioblastoma. This adaptive study design allows for multiple drugs to be evaluated in parallel, expediting the approval process and avoiding redundant recruitment[3].
Regulatory Designations
Orphan Drug Designation
GDC-0084 was granted Orphan Drug Designation by the US FDA in August 2019, recognizing its potential as a treatment for a rare disease with high unmet patient need[1][3].
Breakthrough Designation
In August 2020, the drug was granted Breakthrough Designation by the US FDA for glioblastoma, further highlighting its promising clinical data and potential to address a significant unmet medical need[3].
Market Potential and Projections
High-Grade Glioma Market
The high-grade glioma market, which includes glioblastoma, is expected to grow significantly. As per DelveInsight's analysis, the market size was USD 1.4 billion in 2022 and is projected to grow at a significant CAGR from 2019 to 2032. This growth is driven by factors such as an aging population, advancing research, and the development of innovative systemic therapies[2].
Glioblastoma Market Size
The glioblastoma market alone is estimated to be valued at approximately USD 1.5 billion per annum. With GDC-0084 showing promising clinical activity, it stands to capture a significant share of this market if approved[3].
Competitive Landscape
The high-grade glioma market is witnessing an upsurge in growth due to emerging pipeline candidates from various companies, including Kazia Therapeutics, BioMimetix, Bayer, and others. GDC-0084's unique ability to cross the blood-brain barrier and its specific design for brain cancer position it as a strong contender in this market[2].
Clinical and Commercial Opportunities
Multiple Indications
GDC-0084 is being evaluated for multiple brain cancers, including glioblastoma, brain metastases, childhood brain cancer, IDH-mutant glioma, and primary CNS lymphoma. It also shows potential in various solid tumor types, such as ovarian cancer, renal cell carcinoma, and liver cancer[3].
Combination Therapies
There is strong evidence of synergy between GDC-0084 and immunotherapy in preclinical data, suggesting potential for both monotherapy and combination use. This versatility further enhances its commercial opportunity[3].
Licensing and Partnerships
Kazia Therapeutics has a licensing-driven business model, with a commercial licensee in place for China and potential opportunities for non-dilutive income through additional partnering activities[3].
Key Takeaways
- Development Milestones: GDC-0084 has progressed through Phase I and is currently in Phase II/III clinical trials, showing promising efficacy in glioblastoma patients.
- Regulatory Recognition: The drug has received Orphan Drug and Breakthrough Designations from the US FDA.
- Market Potential: The high-grade glioma market is expected to grow significantly, with GDC-0084 poised to capture a substantial share due to its unique properties and clinical data.
- Clinical Versatility: GDC-0084 is being evaluated for multiple cancer types and shows potential for combination therapies.
FAQs
What is GDC-0084, and how does it differ from other brain cancer treatments?
GDC-0084, now known as paxalisib, is a drug candidate developed to treat glioblastoma. It is distinguished by its ability to cross the blood-brain barrier, a critical feature for effective brain cancer treatment.
What is the current stage of clinical trials for GDC-0084?
GDC-0084 is currently in Phase II/III clinical trials, with ongoing studies evaluating its efficacy in newly diagnosed and recurrent glioblastoma patients.
What regulatory designations has GDC-0084 received?
GDC-0084 has received Orphan Drug Designation and Breakthrough Designation from the US FDA, recognizing its potential to address a significant unmet medical need.
What is the projected market size for the high-grade glioma market?
The high-grade glioma market is expected to grow significantly, with a market size of USD 1.4 billion in 2022 and projected growth at a significant CAGR from 2019 to 2032.
What other cancer types is GDC-0084 being evaluated for?
In addition to glioblastoma, GDC-0084 is being evaluated for brain metastases, childhood brain cancer, IDH-mutant glioma, primary CNS lymphoma, and various solid tumor types such as ovarian cancer and renal cell carcinoma.
How does GDC-0084's ability to cross the blood-brain barrier impact its efficacy?
GDC-0084's ability to cross the blood-brain barrier allows it to effectively target brain tumors, which is a significant advantage over many other cancer drugs that are unable to penetrate this barrier.
Sources
- Kazia Therapeutics. Novel Australian drug GDC-0084 for glioblastoma brain cancer starts Phase II clinical trial in US. Retrieved from https://www.kaziatherapeutics.com/site/blog/novel-australian-drug-gdc-0084-for-glioblastoma-brain-cancer-starts-phase-ii-clinical-trial-in-us
- PR Newswire. High-grade Glioma Market to Witness Upsurge in Growth by 2032, Predicts DelveInsight. Retrieved from https://www.prnewswire.com/news-releases/high-grade-glioma-market-to-witness-upsurge-in-growth-by-2032-predicts-delveinsight---companies-to-watch-out---biomimetix-bayer-medimmune-dnatrix-chimerix-bristol-myers-squibb-laminar-immunomic-inovio-301966820.html
- Kazia Therapeutics. A Diversified Oncology Drug Development Company. Retrieved from https://www.kaziatherapeutics.com/site/pdf/ebcc5b2e-29a6-410c-ab9a-c3e722413615/Kazia-Corporate-Presentation-November-2024.pdf
- Kazia Therapeutics. GDC-0084 and glioblastoma multiforme. Retrieved from https://www.kaziatherapeutics.com/site/blog/gdc-0084-and-glioblastoma-multiforme
- Coherent Market Insights. Recurrent Glioblastoma Multiforme Treatment Market. Retrieved from https://www.coherentmarketinsights.com/market-insight/glioblastoma-multiforme-market-682