QED Therapeutics

The company's core business revolves around developing and commercializing infigratinib for various medical conditions. The business model involves forming strategic partnerships to advance its clinical programs and commercialize its treatments globally. For instance, QED entered into a collaboration with Helsinn Group to co-develop and co-commercialize infigratinib for oncology indications in the U.S. and other regions, with BridgeBio retaining full rights for skeletal dysplasia applications. Another key agreement was made with Kyowa Kirin to develop and commercialize the drug for skeletal dysplasias in Japan, involving a $100 million upfront payment and potential future royalties and milestones.

Infigratinib, also known as Truseltiq, is an orally administered tyrosine kinase inhibitor designed to selectively target FGFR1, FGFR2, and FGFR3. Overactivity in these receptors is a known driver in several cancers and genetic disorders. The drug works by blocking the abnormal protein signals that cause cancer cells to multiply, thereby slowing the spread of the disease. Initially, infigratinib received accelerated approval from the U.S. FDA in May 2021 for treating adults with a specific type of bile duct cancer (cholangiocarcinoma) who have an FGFR2 gene fusion. However, it is no longer marketed for this indication in the U.S.

QED Therapeutics is now primarily focused on evaluating infigratinib for pediatric skeletal dysplasias, specifically achondroplasia, the most common form of disproportionate short stature. The company is conducting clinical trials to assess the potential of low doses of the drug to correct pathological hallmarks of achondroplasia by targeting the condition at its source—the overactivity of the FGFR3 gene. Preclinical studies have shown promising results in mouse models, and the FDA has granted the drug breakthrough therapy designation for this investigational use.