‘Highly Impressive’ Data Propels BridgeBio’s Achondroplasia Drug Forward

 

Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.

BridgeBio Pharma’s investigational FGFR3 inhibitor infigratinib significantly improved yearly growth in a late-stage study of achondroplasia, the most common form of dwarfism, paving the way for an approval application later this year.

Writing to investors Thursday morning, analysts at Truist Securities called the results “highly impressive,” noting that infigratinib “meaningfully exceeded even the most optimistic expectations” for its efficacy and safety.

In the Phase 3 PROPEL 3 study, infigratinib accelerated growth by 2.1 cm per year on average as compared with placebo, a treatment effect that met the bar for statistical significance, according to a Thursday release. The drug also hit key secondary endpoints, including body proportionality.

There were no serious toxicities related to infigratinib, nor were there dropouts due to side effects, BridgeBio noted in its news release. The biotech documented three episodes of elevated phosphate levels, though all were mild, transient and asymptomatic, and did not require dose changes or discontinuation.

BridgeBio is trading at $84.22 before the opening bell on Thursday, a 15% increase from its closing price of $73.33 on Wednesday.

Mizuho Securities was also bullish on PROPEL 3’s findings, writing in its own note that the study met its “best case scenario,” with data that are “incredibly clean on both efficacy/safety.” In particular, the analysts pointed to the body proportionality outcome of the trial.

The proportionality point, Mizuho continued, “could potentially allow for premium pricing.” BridgeBio’s decision to enroll a broad age group in PROPEL 3 could also set infigratinib up to be “the easiest, best-in-indication solution for physicians and patients across the indication.” PROPEL 3 included patients aged 3 to less than 18 years.

With these data, BridgeBio now plans to meet with the FDA to discuss a potential regulatory road for infigratinib and expects to file a new drug application in the second half of this year. The company is enrolling patients in the ACCEL program, an observational lead-in study for the drug’s Phase 3 study in hypochondroplasia. BridgeBio is also testing infigratinib in newborn infants and toddlers up to 3 years of age with achondroplasia.

If approved, BridgeBio will enter the the same arena as BioMarin, which owns Voxzogo, currently the only commercial therapy for achondroplasia. Another circling competitor is Ascendis Pharma, which is expecting an FDA decision for TransCon CNP by Feb. 28.

https://www.biospace.com/drug-development/highly-impressive-data-propels-bridgebios-achondroplasia-drug-forward