Wednesday, February 07, 2024
BridgeBio Pharma, Inc. (Nasdaq: BBIO) and Kyowa Kirin Co., Ltd have announced a significant partnership. Under this agreement, BridgeBio's affiliate, QED Therapeutics, has granted Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan. The deal includes an upfront payment of USD 100 million to BridgeBio, along with royalties up to the high twenties percent on sales of infigratinib in Japan, as well as potential milestone-based payments. Infigratinib, an oral small molecule designed to inhibit FGFR3, targets FGFR3-driven skeletal dysplasias, including achondroplasia and hypochondroplasia.
BridgeBio, expressed satisfaction with the partnership, citing Kyowa Kirin's commitment to providing innovative medicines with life-changing value. He highlighted BridgeBio's mission to provide access to novel treatments and trials, ensuring the development of infigratinib for children with achondroplasia and other skeletal dysplasias in Japan.
QED Therapeutics, emphasized their goal of accelerating the development of infigratinib in Japan to cater to the needs of children with achondroplasia and hypochondroplasia. He expressed eagerness to begin trials in Japan at a later date.
Kyowa Kirin, stressed the importance of strengthening their portfolio in bone and mineral disorders, including achondroplasia. He expressed confidence in infigratinib's potential based on the latest clinical trial results and reaffirmed their commitment to advancing development in Japan.
The partnership was positively received by Eihaku Itooka, founder and president of GLORY TO ACHONDROPLASIA, a Japanese bone dysplasia patient advocacy organization. Itooka highlighted the responsibility to improve treatment options and social recognition for children with achondroplasia.
BridgeBio recently dosed the first child in PROPEL 3, a Phase 3 pivotal trial evaluating infigratinib's efficacy and safety in children with achondroplasia. Kyowa Kirin aims to initiate a Japanese registrational trial in 2025. Additionally, BridgeBio plans to commence ACCEL, an observational lead-in study for infigratinib in hypochondroplasia, in the first half of 2024.
Achondroplasia affects approximately 55,000 people in the U.S. and EU, and 6,000 in Japan. It is uniformly caused by an activating mutation in FGFR3, leading to medical complications such as obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis.