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FDA Approves New Drug Application For Ultragenyx’s mRNA Therapy; Shares Drop

Ultragenyx Pharmaceutical announced that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for UX053, an experimental mRNA therapy designed for the treatment of Glycogen Storage Disease Type III (GSDIII). Shares of the biopharmaceutical company closed at $115 on March 8, down almost 3%.

Following the FDA clearance, Ultragenyx (RARE) expects the two-part Phase 1/2 clinical trial in adults to begin in the second half of 2021. These clinical trials will evaluate the efficacy of UX053.

GSDIII is a disease caused by the deficiency of glycogen debranching enzyme (AGL), which results in glycogen accumulation in the liver and muscle. This disease affects more than 10,000 patients globally. (See Ultragenyx stock analysis on TipRanks)

Ultragenyx is developing UX053 along with several other mRNA therapies in the preclinical stage for undisclosed indications with partner Arcturus Therapeutics (ARCT), a clinical-stage mRNA medicines name, the company said.

On March 3, Stifel Nicolaus analyst Dae Gon Ha resumed coverage of the stock with a Buy rating and a price target of $179 (55.7% upside potential).

The analyst said, “Ultragenyx, which has received three drug approvals since its IPO in 2014, has been and continues to be relentless in developing meaningful therapies from a science-driven, patient-centric approach… The company’s prospects remain strong, with five clinical-stage assets poised to generate meaningful catalysts in 2021 and beyond.”

The rest of the Street is cautiously optimistic about the stock with a Moderate Buy consensus rating. That’s based on 7 analysts suggesting a Buy and 5 analysts recommending a Hold. The average analyst price target of $163.33 implies a 42% upside potential to current levels. Shares have increased about 50.7% over the past six months.

TipRanks’ Hedge Fund Trading Activity tool shows that confidence in Ultragenyx is currently Positive as 2 hedge funds increased their cumulative holdings of the stock by 9,300 shares in the last quarter.

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