BridgeBio Pharma (NASDAQ:BBIO) saw its shares rise more than 5% to about $77 on Thursday after reporting positive Phase 3 topline results for oral infigratinib in achondroplasia, with Jefferies analysts describing the data as “compelling.”
Achondroplasia is the most common form of dwarfism, a genetic condition that affects bone growth and results in short stature and disproportionate limb development.
BridgeBio’s PROPEL 3 trial met its primary endpoint, showing a statistically significant increase in annualized height velocity at Week 52 compared with baseline.
The mean treatment difference against placebo was +2.10 centimetres per year (cm/year), with a least-squares (LS) mean of +1.74 cm/year.
In a pre-specified exploratory analysis of a key secondary endpoint, the treatment achieved the first statistically significant improvement in body proportionality in children younger than 8 years old, with an LS mean difference of -0.05 versus placebo.
The study also met the secondary endpoint of change from baseline in height Z-score relative to an achondroplasia reference population at Week 52, with an LS mean increase of +0.41 SD on the treatment arm.
BridgeBio said oral infigratinib was well tolerated, with no discontinuations or serious adverse events related to the drug. Three cases (4%) of mild, transient hyperphosphatemia were reported, with none requiring dose adjustment. No adverse events linked to FGFR1 or FGFR2 inhibition, such as retinal or corneal complications, were observed.
The company plans to submit a New Drug Application (NDA) and Marketing Authorization Application (MAA) in the second half of 2026.
Infigratinib holds Breakthrough Therapy Designation from the US Food and Drug Administration, making it the only therapeutic in development for achondroplasia with that status.
Given the strength of the data, BridgeBio said it will accelerate development of infigratinib for hypochondroplasia and has begun enrolling the observational run-in for the related Phase 3 trial.
Following the results, Jefferies reiterated a ‘Buy’ rating on BridgeBio with a price target of $85, adding that the Phase 3 data could drive a 10% to 20% stock move.
“Infigratinib raises the bar in achondroplasia,” Jefferies wrote, highlighting that the placebo-adjusted annualized height velocity of +1.74 cm/year (LS mean) and +2.10 cm/year (unadjusted) appears stronger than BioMarin’s Voxzogo and Ascendis Pharma’s TransCon CNP in cross-trial comparisons.
The analysts also pointed to the statistically significant improvement in body proportionality in children under 8 years old as a key differentiator, noting that proportionality data “trumps annualized height velocity” in their view.
Jefferies said safety “should be well received by the Street,” citing only 4% Grade 1 hyperphosphatemia, no ocular or renal toxicity, and no drug-related discontinuations or serious adverse events.
The firm said it expects BridgeBio to file an NDA in the second half of 2026 and sees a 90% probability of approval following the 52-week PROPEL 3 data.
The analysts added that BridgeBio is positioned to launch four growth products in parallel by 2027, including BBP-418 for LGMD2I/R9, encaleret for ADH1, and infigratinib for achondroplasia, with additional Phase 2 data in hypochondroplasia expected later this year.
Jefferies estimated $450 million in peak sales for infigratinib in achondroplasia and hypochondroplasia, calling that figure conservative given the drug’s oral dosing, differentiation on proportionality, and what it described as significant untreated patient “whitespace” globally.