Kyowa Kirin pays $100M to partner with BridgeBio on potential dwarfism drug

With an upfront payment of $100 million, Kyowa Kirin has bought into a partnership with BridgeBio to develop and commercialize Truseltiq (infigratinib) in Japan.

Besides the sizable upfront installment, Kyowa Kirin agreed to fork over undisclosed payments if the drug reaches certain milestones, plus pay royalties up to the “high-twenties" in percentage terms.

The once-daily oral therapy was approved by the FDA in 2021 to treat bile duct cancer (cholangiocarcinoma), but the partnership is more about the potential of infigratinib in other indications. The FGFR-3 tyrosine kinase inhibitor has demonstrated potentially best-in-class efficacy in a phase 2 study in children with achondroplasia, a genetic condition which hinders bone plate growth.

“By partnering with Kyowa Kirin, we hope to significantly accelerate the development of infigratinib to potentially provide options for children with achondroplasia, hypochondroplasia and eventually skeletal dysplasias in Japan,” Justin To, CEO of BridgeBio’s affiliate QED Therapeutics, said in a release.

BridgeBio CEO Neil Kumar added that the company was encouraged to partner with Kyowa Kirin in part because of the Japanese company’s success in the development and commercialization of rare disease drugs.

By 2025, Kyowa Kirin hopes to initiate a registrational trial of infigratinib in achondroplasia. BridgeBio has already kicked off its PROPEL 3 study in the indication, dosing its first patient two months ago.

If infigratinib eventually reaches the Japanese market to treat achondroplasia, it would go up against BioMarin's Voxzogo. In 2022, that drug scored an approval in Japan to treat children with the disease whose growth plates are not closed. Voxzogo is a key growth driver at BioMarin, generating $324 million in the first 9 months of last year.

For BridgeBio, the deal helps the company focus on its heart disease treatment acoramidis. On Monday, the nine-year old California biotech revealed that the FDA has accepted its application to for the medicine treat transthyretin amyloid cardiomyopathy (ATTR-CM) and assigned a Nov. 29 decision date.

Less than a year ago, BridgeBio was the subject of buyout rumors from large pharma companies, according to a report in Bloomberg. Rare disease companies have become hot M&A targets. An example is Horizon Therapeutics which was sold last year to Amgen in a $27.8 billion deal.