Ultragenyx Shares Slide After Positive, But Noisy, Angelman Data >RARE

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By Colin Kellaher

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Shares of Ultragenyx Pharmaceutical Inc. fell sharply Tuesday despite an update from the biopharmaceutical company on its Angelman syndrome program that Wall Street analysts generally viewed as positive.

The Novato, Calif., company said interim data from a dose-escalating Phase 1/2 study of GTX-102 in pediatric patients with the genetic disorder was encouraging, and that it had exercised its option to buy GeneTx Biotherapeutics LLC, its partner in the program.

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Ultragenyx also said the interim data supported a protocol amendment to the study that UK and Canadian health authorities approved in May to initiate additional, new cohorts of patients at higher monthly loading doses.

Analysts on Wall Street in general agreed that while the data from the much-anticipated update was a bit “noisy,” the overall takeaway was positive.

In a research note, Citi analysts said the Angelman program “is still very much a work in progress,” but that the study update was a solid step forward and an important de-risking step. Citi has a buy rating and $146 price target on Ultragenyx shares, with the Angelman program contributing about $35 to that target.

Analysts at Truist, meanwhile, said they viewed the efficacy trend reported from the UK and Canada as an incremental positive, and that Ultragenyx’s move to buy GeneTx implies conviction.

Truist also noted that while the study safety was good, the volatility in Ultragenyx shares could stem from continued debate around efficacy due to lack of a clear dose response and numerically inferior effect size compared to the initial three cohorts.

But Truist, with a buy rating and $185 price target on Ultragenyx shares, said it would use that volatility and buy the dip.

Analysts at Stifel said the update checks off important safety and efficacy boxes en route to more derisking of GTX-102, and that the acquisition of GeneTx underscores Ultragenyx’s commitment and confidence in the drug.

Ultragenyx shares were recently changing hands at $52.62, down nearly 14%.

Write to Colin Kellaher at [email protected]

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Credit: www.marketwatch.com /

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