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BridgeBio sells partial rights to dwarfism drug for $100M

Dive Brief:

  • BridgeBio Pharma is selling partial rights to an experimental drug for a common form of dwarfism to Japanese pharmaceutical company Kyowa Kirin, the companies announced Wednesday.
  • Per deal terms, BridgeBio affiliate QED Therapeutics will grant Kyowa Kirin rights in Japan to a therapy known as infigratinib that is in Phase 3 testing. BridgeBio will get $100 million upfront, and is eligible for milestone payments as well as sales royalties if the drug reaches the market.
  • In December, BridgeBio dosed the first patient in a Phase 3 trial testing its therapy in achondroplasia, a genetic condition that causes dwarfism. The drug is being evaluated in another skeletal abnormality as well.

Dive Insight:

Achondroplasia is the most common form of dwarfism and is caused by alterations to a gene called FGFR3, which makes a protein implicated in the growth of bones. The condition disrupts the body’s ability to turn cartilage into bone, causing disproportionately short stature and health problems such as sleep apnea and spinal stenosis.

Like a similar medicine from BioMarin Pharmaceutical that’s sold as Voxzogo, BridgeBio’s medicine is designed to block a protein coded by that gene. BioMarin’s therapy is injectable, while BrigeBio’s is a pill.

The therapy has shown promise in mid-stage testing, with results last year suggesting the medicine might help children with the condition grow faster. The drug was well-tolerated, too, a key consideration as analysts have noted one of the key selling points of Voxzogo is its safety profile.

The Phase 3 study, which is enrolling children between three and 18 years old, will provide more definitive answers. Results could come next year, according to a federal database. BridgeBio is planning to begin another trial this year as well, while Kyowa Kirin is discussing a Japan-based trial with regulators there. 

The deal gives BridgeBio more financial bandwidth at a time when the company is preparing to launch a closely watched medicine for the rare genetic disease transthyretin amyloidosis cardiomyopathy. That drug, acoramidis, could be approved by U.S. regulators in November. The company recently secured up to $1.25 billion ahead of that decision by selling drug royalties and raising a credit line.