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Ultragenyx's (RARE) Portfolio of Marketed Drugs Aids Growth

Zacks Equity Research
·3-min read

Ultragenyx Pharmaceutical Inc. RARE is seeing a steady uptake of its portfolio of marketed drugs that are approved for the treatment of rare and ultra-rare diseases.

The company currently markets three drugs, namely Crysvita, Mepsevii and Dojolvi.

Crysvita is approved for treating X-linked hypophosphatemia, an inherited disorder and tumor-induced osteomalacia, an ultra-rare disease. Mepsevii is approved to treat Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. Dojolvi was approved in June 2020 for all forms of long-chain fatty acid oxidation disorders (LC-FAOD).

All these drugs have witnessed a strong uptake so far with Crysvita contributing a majority to the top line. The drug has also been approved by the FDA for additional indications, which is likely to further boost sales for the company.

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Shares of Ultragenyx have declined 45.8% so far this year compared with the industry’s fall of 17.3%.

 

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Ultragenyx has several products and some gene therapy candidates in its pipeline.

The company is developing DTX301, an adeno-associated virus 8 (AAV8) for the treatment of patients with Ornithine transcarbamylase deficiency. DTX401, an AAV8 gene therapy candidate, is currently being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa). Ultragenyx is developing UX143 (setrusumab) in a phase II/III Orbit study for treating Osteogenesis Imperfecta (OI).

Ultragenyx is developing GTX-102 for treating patients with Angelman syndrome.

Apart from this, Ultragenyx has made some promising licensing and collaboration deals.

The successful development and commercialization of the candidates are likely to boost the company’s revenues.

In January 2022, Ultragenyx announced a license and collaboration agreement with Regeneron REGN.

Per the deal, Ultragenyx obtained the rights to develop, commercialize and distribute Evkeeza (evinacumab) outside the United States. The collaboration with REGN for Evkeeza gives Ultragenyx a fourth approved product that can add to its revenues beside Crysvita, Mepsevii and Dojolvi. Regeneron is responsible for the development and distribution of Evkeeza in the United States.

Such deals are a source of generating funds for Ultragenyx.

However, Ultragenyx has had its share of pipeline setbacks. In October 2020, Ultragenyx and GeneTx announced positive interim data from the phase I/II study of GTX-102 for treating Angelman Syndrome. After reaching substantially higher doses per protocol, all five patients experienced a serious adverse event of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing.

Similar development or regulatory setbacks are likely to hurt the company’s stock in the days ahead.

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are ASLAN Pharmaceuticals Limited ASLN and Immunocore Holdings plc IMCR, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Loss per share estimates for ASLAN Pharmaceuticals have narrowed 6.1% for 2022 and 5.7% for 2023 in the past 60 days.

Earnings of ASLAN Pharmaceuticals surpassed estimates in two of the trailing four quarters and missed on the remaining two occasions. ASLN witnessed an earnings surprise of 1.64% on average.

Loss per share estimates for Immunocore have narrowed 39.7% for 2022 and 39.4% for 2023 in the past 60 days.

Earnings of Immunocore surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. IMCR witnessed an earnings surprise of 68.34% on average.

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Regeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report

Ultragenyx Pharmaceutical Inc. (RARE) : Free Stock Analysis Report

ASLAN Pharmaceuticals Ltd. (ASLN) : Free Stock Analysis Report

Immunocore Holdings PLC Sponsored ADR (IMCR) : Free Stock Analysis Report

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