BridgeBio Pharma surges on positive trial data, BioMarin Pharma slides

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BridgeBio Pharma (NASDAQ:BBIO) shares surged Monday morning after the company announced positive results in its achondroplasia trial.

The biopharmaceutical company’s share price is up 62% at the time of writing.

The PROPEL2 Phase 2 trial is testing the investigational therapy infigratinib in children with achondroplasia. The company revealed that 80% of the 10 children in the study with six-month visits were responders, with a change from baseline annualized height velocity of at least 25%.

BioMarin Pharmaceutical Inc (NASDAQ:BMRN), which has a competing treatment named Voxzogo, has seen its share price fall over 5% so far in Monday’s session.

Reacting to the news, RBC Capital Markets analysts told investors in a note that it is early days, but BBIO’s data was “impressive.”

“Small n (n=10) in a single arm trial, but BBIO +3.03 cm/yr vs baseline is numerically superior to Voxzogo label (+1.57 cm/yr, albeit vs placebo). Even if we compare vs the initial Phase II single arm data (arguably more apples-to-apples), the +3.03 cm/yr shown by BBIO is numerically superior to the +2.01 cm/yr showed by Voxzogo,” wrote the analysts.

“While this has no short-term impact to Voxzogo estimates, it may cap the longer-term commercial potential should the data be replicated, especially in the context of an oral daily pill vs a daily injectable,” they added.

Raymond James analysts maintained an Outperform rating on BBIO but upped the firm’s price target on the stock to $29 from $17 per share.

“Though a small study, the topline number is striking. Of the 10 patients (of 12) that reached 6-months follow-up, AHV change from baseline was 3.03 cm/year, substantially higher than what has previously been reported for competitors at the 6 or 12-month mark (~2 cm/yr),” said the analysts. “These data top our expectation and somewhat assuage the concern of a potential decline in AHV benefit from month 6 to 12.”

Stifel analysts were more cautious, stating that the main question with infigratinib continues to be safety.

“BBIO’s press release, though it displays a bar chart with patient level efficacy data, does not include an adverse event table, and high level conveys no drug-related AEs/discontinuations,” they argued.

Evercore ISI lowered its price target on BMRN to $115 from $130 but kept an Outperform rating on the stock, telling investors in a note that they are reducing their Voxzogo outer year estimates.

“Peak #s fall from $1.5B to $1.2B and raising our discount rate to 9.5% to reflect the fact that our estimates are still at risk if infigratinib continues to show this compelling profile,” revealed Evercore analysts.