Dive Brief:
- Californian company BridgeBio Pharma has snapped up Novartis' sidelined cancer drug infigratinib, which targets the tyrosine kinase receptor FGFR. The newly-launched BridgeBio subsidiary QED Therapeutics will develop infigratinib, supported by a $65 million financial commitment from its parent company.
- The payment includes "a substantial upfront payment to Novartis," as well as equity in QED, and BridgeBio will also pay out on development and sales milestones, as well as royalties. No further financial details have been disclosed.
- According to BridgeBio co-founder and investor Frank McCormick, "Infigratinib embodies the crux of what we set out to do at BridgeBio: develop targeted therapies for genetically-driven tumors and monogenic disorders."
Dive Insight:
BridgeBio's latest acquisition, Novartis' sidelined FGFR inhibitor, is currently in a Phase 2 clinical trial in patients with chemotherapy-refractory bile duct cancer (cholangiocarcinoma) containing FGFR2 fusions, and has shown some activity. This form of cancer affects between 6,000 and 8,000 people across the U.S. each year, and treatment choices are limited.
"We are committed to moving this compound forward in late-stage development and further proving the strong efficacy in cancer that has already been demonstrated across multiple trials," said Daniel Hoth, CMO of QED.
What is perhaps more interesting, though, is the drug's potential in skeletal dysplasias, including achondroplasia, the most common form of dwarfism. This disorder is a result of a mutation in the FGFR3 gene, which regulates bone formation. The mutation causes more than just short stature, however. People with achondroplasia may also have problems with sleep, ear infections, skeletal problems, obesity and may experience chronic pain. Their lives may be shortened, and there is no current treatment that is approved by the Food and Drug Administration.
QED isn't the only company targeting FGFR3 in dwarfism. In Phase 2 proof-of-concept studies, BioMarin's vosoritide, an analog of C-type natriuretic peptide (CNP), increased growth velocity by 50% in children. The company is recruiting children for a Phase 3 study. This is expected to complete in February 2025.
Danish biopharma Ascendis Pharma A/S is developing a once-weekly formulation of CNP, delivered using its TransCon technology. Ascendis is preparing for a Phase 1 trial in healthy volunteers in Australia, due to begin during the second quarter of 2018. Topline data announcements are planned for fourth quarter of this year.
Therachon AG's TA-46, which has U.S. and European orphan drug designation, modulates FGFR3, preventing excessive activation, and is in preclinical development. And also in preclinical development, BioClin Therapeutics, Inc. is working on an anti-FGFR3 antibody, B-701, for achondroplasia. B-701 is also in Phase 2 trials for bladder cancer, and the safety data generated in these studies may speed its development in dwarfism.
Despite early stage signals that showed infigratinib could be effective in certain cancers, the data wasn't enough for Novartis and the big pharma dumped the drug last year. Novartis, like many of its peers, has been trying to monetize assets that it isn't pursuing and right-sizing its portfolio to concentrate on ares of strength.