BridgeBio Pharma, Inc. and Kyowa Kirin Co., Ltd have announced a partnership wherein BridgeBio’s affiliate, QED Therapeutics, grants Kyowa Kirin an exclusive license to develop and commercialise infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan. In exchange, BridgeBio will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments. Infigratinib is an oral small molecule designed to inhibit FGFR3 and thus target FGFR3-driven skeletal dysplasias at their source, including achondroplasia and hypochondroplasia.

“We are pleased to partner with Kyowa Kirin given their community-oriented core mission to bring smiles to faces by providing innovative medicines with life-changing value, which has seen success with their commercialisation of Crysvita and other rare disease drugs. The BridgeBio vision is to help bring people access to novel treatments and trials, and so we are pleased that this collaboration will ensure the development of infigratinib for children living with achondroplasia, and eventually for other skeletal dysplasias in Japan,” said Neil Kumar, Ph.D., BridgeBio’s CEO and founder.

“We are thrilled to announce that we have signed the License Agreement with BridgeBio,” said Yasuo Fujii, MBA., Chief Strategy Officer, Managing Executive Officer, Vice President, Head of Strategy Division at Kyowa Kirin. “It is important for us to strengthen our portfolio by introducing pipelines in the fields of bone & mineral disorders, including achondroplasia. Based on the results from the latest clinical trials, we believe BridgeBio’s infigratinib has high potential for treating achondroplasia. We will steadily advance the development in Japan and aim to deliver life-changing value to people with skeletal dysplasias including achondroplasia.”

In December 2023, BridgeBio dosed the first child in PROPEL 3, a one-year, 2:1 randomised, placebo-controlled Phase 3 pivotal trial, evaluating the efficacy and safety of infigratinib in children with achondroplasia aged 3 to <18 years with open growth plates. The Phase 3 builds on the success of PROPEL 2, a Phase 2 trial of infigratinib in achondroplasia which demonstrated a +3.38cm/yr increase in annualised height velocity, the strongest clinical result published to date. In Japan, Kyowa Kirin will start to discuss with Pharmaceuticals and Medical Devices Agency (PMDA) of Japan in 2024, with the aim of initiating a Japanese registrational trial in 2025.