BridgeBio Pharma has launched its latest biotech startup: a company built around an abandoned Novartis cancer drug candidate that is now backed by $65 million in fresh capital.
The new company, QED Therapeutics, aims to pick up where Novartis (NYSE: NVS) left off. Novartis took the drug, infigratinib, as far as mid-stage clinical trials as a treatment for bile duct cancer. But in addition to cancer, QED also aims to develop the compound to treat a rare pediatric bone disorder characterized by dwarfism.
BridgeBio, which like QED is based in Palo Alto, CA, develops drugs by forming companies around compounds whose rights it acquires. These lean companies share BridgeBio’s resources in a model it describes as “hub and spoke.” Last year, BridgeBio raised $135 million to support its efforts. Today, its subsidiaries include companies developing drugs for cancer, heart conditions, and skin disorders.
Infigratinib blocks proteins from binding to fibroblast growth factor receptors (FGFR), which have a role in helping cancers grow. QED notes that this receptor has been linked to multiple cancers. Novartis studied the drug in patients whose cholangiocarcinoma, or bile duct cancer, failed chemotherapy. There is no standard treatment for cancers that have failed first-line treatment with chemo.
Novartis took infigratinib into Phase 2 studies targeting FGFR2. Trial results published in the Journal of Clinical Oncology showed that the compound had activity against the bile duct cancer. Nonetheless, Novartis decided to stop work on the drug.
QED says that FGFR also plays a role in forms of pediatric growth disorders. One of those disorders, achondroplasia, prevents cartilage from changing to bone, particularly in the arms and the legs. According to the National Organization for Rare Disorders, the condition has been linked to mutations of the FGFR3 gene. Frequency of achondroplasia ranges from one in 15,000 to one in 30,000 births, NORD says.
The research on targeting FGFR to treat achondroplasia is still early. According to a study published in the Journal of Clinical Investigation, mice injected with a low dose of infigratinib showed skeletal improvements. More research is needed to advance the compound into human testing. Neil Kumar, CEO of BridgeBio, said in a prepared statement that in addition to the opportunity to develop a new treatment for bile duct cancer, the drug offers a “potential best-in-class therapy to treat achondroplasia at its source.”
BridgeBio is not disclosing specific financial details of its licensing deal with Novartis. The company says it has committed $65 million to support QED, which includes an upfront payment to Novartis and equity in QED. If the compound hits development and commercialization targets, Novartis would receive milestone payments and royalties from the drug’s sales.