Our Science
Targeting the molecular drivers of GIST to develop more effective and durable therapies.
Understanding GIST
Gastrointestinal Stromal Tumors (GIST) are rare cancers that arise in the gastrointestinal (GI) tract. The majority of GISTs are driven by mutations in two key genes: KIT and PDGFRA. These mutations lead to the over-activation of proteins called receptor tyrosine kinases, which signal cancer cells to grow and divide uncontrollably.
While the development of tyrosine kinase inhibitors (TKIs) like imatinib revolutionized GIST treatment, many patients eventually develop resistance to these therapies. This resistance is often caused by secondary mutations in the KIT or PDGFRA genes, creating a significant ongoing challenge in the long-term management of the disease.
Our Approach: Precision and Innovation
Existing treatments can be effective initially, but drug resistance due to new mutations remains a major hurdle for long-term patient survival.
We are developing a next-generation kinase inhibitor which overcomes the limitations of current therapies by potently targeting a wide spectrum of primary and resistance mutations.
Our goal is to provide a durable, effective treatment that can improve and extend the lives of GIST patients, including those who have failed previous lines of therapy.
The von Pfeffel Pharma Difference
The molecule at the heart of our research binds to the ATP pocket of the KIT and PDGFRA kinases with high affinity, even in the presence of mutations that confer resistance to approved and experimental TKIs. Preclinical studies have demonstrated its potential to potently inhibit a broad range of clinically relevant mutations.
By focusing on a differentiated mechanism of action, we aim to address the critical unmet need for therapies that can provide lasting benefits for GIST patients. Our scientific journey is driven by a deep understanding of GIST biology and a relentless commitment to translating that knowledge into a life-changing medicine.