BridgeBio data shows lasting benefits for achondroplasia drug

BridgeBio Pharma has reported promising results from a mid-stage clinical trial of its experimental drug infigratinib, aimed at treating individuals with achondroplasia, a common cause of disproportionate short stature. Data from the trial revealed that the drug significantly increased the annual growth rate in height among participants and improved body proportionality without serious side effects.

The trial involved a dozen individuals who received the highest tested dose of infigratinib. Over 18 months, the drug boosted the annualized height velocity by 2.5 centimeters, a notable improvement from the baseline measurements. These results follow previous data released a year ago, which had already placed infigratinib in the spotlight due to its promising six-month performance. At that time, the drug had surpassed the growth increase benchmark set by Voxzogo, a competing treatment from BioMarin Pharmaceutical, which is a known player in the achondroplasia treatment landscape.

BridgeBio has since initiated a Phase 3 clinical trial of infigratinib, aiming to complete participant enrollment by the end of the year. The latest findings have raised anticipation for the upcoming Phase 3 trial results. The recorded growth rate increase of 2.5 centimeters per year, although slightly less than the 3.38 centimeters per year reported at six months, still exceeds the expectations of both the company and analysts.

Safety is a key concern in drug development, and BridgeBio reported no serious adverse effects or treatment discontinuations due to side effects. Importantly, no high phosphate levels were observed, a potential issue flagged in earlier cancer studies involving infigratinib.

Analysts are keeping a close watch on the developments. Paul Matteis from Stifel noted that infigratinib appears to be a strong competitor to Voxzogo, though he pointed out that it is too early to definitively determine if it outperforms Voxzogo. He emphasized that the real test for infigratinib will be its safety profile in Phase 3 trials.

Other analysts, particularly from Mizuho Securities and Raymond James, are more optimistic, suggesting that BridgeBio's drug might indeed prove superior. Voxzogo's Phase 3 trial had shown a 1.57 centimeters annualized height velocity increase over placebo after one year, setting a benchmark for comparison.

In addition to achondroplasia, BridgeBio is expanding the development of infigratinib to treat hypochondroplasia, another skeletal dysplasia with varied symptoms. The company estimates that there are between 14,000 and 25,000 children in the United States and Europe with either condition who could benefit from such treatments.

These developments signal a potentially groundbreaking advancement in the treatment of growth disorders associated with achondroplasia and hypochondroplasia. The progress of infigratinib through clinical trials will be closely monitored by the medical community, patients, and investors alike, as it holds the potential to offer a new, effective treatment option for those affected by these conditions.