BridgeBio Pharma (BBIO) shattered Wall Street expectations in a study of children with dwarfism, leading BBIO stock to explode in massive volume.
The company tested a drug called infigratinib in children with achondroplasia, a genetic condition that slows bone growth. At six months, eight out of 10 children have responded to treatment. The group saw an improvement in annualized growth velocity – how much they could be expected to grow in a year – by 3.03 centimeters.
Analysts had expected an improvement of 1.52 centimeters, Mizuho Securities analyst Salim Syed said in a report. In comparison, a competing treatment of BioMarin Pharmaceutical (BMRN) called Voxzogo showed an improvement of 2.01 centimeters per year in annualized height velocity in a similar test. Ascendant Pharma (ASND) is also studying a treatment for achondroplasia.
“To say the data (was) a win might be an understatement,” he said. “We are now seeing an average change in annual height velocity from baseline and in absolute terms into a whole new level.”
In morning trading in the stock market today, BBIO stock jumped 61.2% to 17.52. The shares came out of a long consolidation with a buy point at 12.74, according to MarketSmith.com. BioMarin stock fell 6.7% near 94.38. Ascendis shares fell 1.2% near 112.79.
BBIO stock: 99th percentile growth
Achondroplasia affects a protein called fibroblast growth factor receptor, or FGFR. In children with this disorder, the protein works abnormally and slows bone growth. BridgeBio’s infigratinib blocks a form of this FGFR protein.
In this study, BridgeBio tested its treatment in 12 children. Two did not respond and two have not yet reached the six-month follow-up time. At the median, children who received infigratinib had an annualized height rate of 7.6 centimeters per year. This is beyond the 99th percentile for children with this disorder.
The two patients who had not yet reached a six-month follow-up had an annualized height velocity of 8.8 centimeters per year based on three months of data.
Mizuho’s Syed estimates that BBIO stock could reach a market capitalization of $3 billion on the drug alone. The company’s market capitalization hit $2.63 billion by mid-morning, up from $1.65 billion before the market opened. It has a buy rating and target price of 23 on BridgeBio stock.
No data security issues
Importantly, the drug had no safety issues. None of the patients had elevated blood phosphate levels – a key safety signal for this treatment. A high phosphate content can lead to kidney problems.
There was a case of elevated blood phosphate in an earlier study. But it was a mild case resolved by dose adjustment.
“These data clearly (position) infigratinib as BridgeBio’s second highly de-risked late-stage asset and we expect the shares to trade into the teens today,” said SVB Leerink analyst Mani Foroohar. , in a report. It has an outperform rating on BBIO shares and raised its price target from 20 to 26.
Mizuho’s Syed expects BridgeBio to conduct a similar clinical study for infigratinib to BioMarin’s Voxzogo. In phase 3 testing, BioMarin enrolled 120 patients.
“Management noted that there were already around 60 attendee slots already requested, which reflects a high level of interest which should result in a high registration rate,” he said.
The news sent BridgeBio shares to their highest level since December 2021. BBIO stock has a relative strength rating of 93, which puts the shares in the top 7% of all stocks when it comes to performance. over 12 months, according to IBD Digital.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
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