BridgeBio lands $100M from Kyowa for Japanese rights to skeletal dysplasia drug
07 Feb 2024
Phase 3License out/inClinical ResultDrug Approval
BridgeBio Pharma announced it has granted Kyowa Kirin exclusive rights in Japan to develop and sell infigratinib for achondroplasia and related bone growth disorders. As part of the deal announced Wednesday, Kyowa Kirin will pay BridgeBio $100 million upfront plus unspecified milestones, along with up to high-20s percent royalties on potential future sales.
Infigratinib is a selective oral ATP-competitive inhibitor of fibroblast growth factor receptors designed to target FGFR3-driven skeletal dysplasias such as achondroplasia and hypochondroplasia at their source. In December, BridgeBio dosed the first patient in the Phase III PROPEL 3 trial evaluating infigratinib in children with achondroplasia after a mid-stage study showed that it significantly increased annual height velocity by an average +3.38 cm/year through to six months.
Providing a "once-daily oral treatment option" is a key goal for the programme based on patient community needs, according to Justin To, chief executive of QED Therapeutics, BridgeBio’s skeletal dysplasia-focused subsidiary.
Japanese trial in 2025
Kyowa Kirin aims to begin a pivotal trial in Japan next year for infigratinib, which adds to its portfolio in bone disorders and rare diseases. The Japanese drugmaker previously partnered with Ultragenyx Pharmaceutical to develop injectable Crysvita (burosumab-twza), a monoclonal antibody that blocks FGF23 to treat X-linked hypophosphataemia as well as tumour-induced osteomalacia.
If approved, infigratinib could compete against BioMarin Pharmaceutical's once-daily injectable treatment Voxzogo (vosoritide), which Japan’s Ministry of Health, Labor and Welfare greenlight in 2022 to treat achondroplasia in children of all ages, whose growth plates are not closed.
BridgeBio continues deal spree
The arrangement with Kyowa Kirin adds to the tally of recent deals done by BridgeBio to help fund its burgeoning pipeline of drugs focused on addressing genetically well-defined conditions amid stiff competition.
Earlier this year it reached a royalty agreement with Blue Owl and CPP Investments that will see it pocket a $500-million milestone payment if another pipeline drug, acoramidis, scores an approval in the US for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The total financing from the two funds could climb to as much as $1.25 billion. Acoramidis, which is currently under review by the FDA with a decision expected by November 29, is expected to compete against Pfizer's ATTR-CM treatment Vyndamax (tafamidis).
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