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Ultragenyx (RARE) Presents Preliminary 2020 Revenues & Guidance

Ultragenyx Pharmaceutical Inc. RARE reported preliminary 2020 Crysvita revenues and provided 2021 revenue guidance for the same.

Crysvita revenues in Ultragenyx territories for 2020 were in the range of $137-$139 million.

Cash, cash equivalents and available-for-sale investments were about $1.2 billion as of Dec 31, 2020.

For 2021, the company expects Crysvita revenues between $180 million and $190 million.

Shares of the company have surged 146.5% in the past year compared with the industry’s growth of 9.7%.

The company released positive long-term data from the phase I/II study onits adeno-associated virus 8 (AAV8) gene therapy candidate, DTX401, which is being evaluated for the treatment of glycogen storage disease type Ia (GSDIa). The company expects to initiate a phase III study in the first half of 2021. It held Scientific Advice and end of phase II meetings with the European Medicines Agency (EMA) and the FDA, respectively. This feedback is being incorporated into the phase III design, and finalization is pending.

Another AAV8 candidate, DTX301, is being evaluated in a phase I/II study for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency. In the phase I/II study, all six responders in the first three cohorts demonstrated durable metabolic control, including greater than two-year sustained responses in the earliest treated patients. A phase III study is expected to begin in the second half of 2021.

The company is evaluating an experimental antisense oligonucleotide, GTX-102, for the treatment of Angelman syndrome (AS) in a phase I/II study. All five patients in the phase I/II study demonstrated improvements in at least 3 disease domains. All five patients had a grade 1 or 2 serious adverse event (SAE) of lower extremity weakness associated with local inflammation in the region of intrathecal administration in the lower back at the higher doses of GTX-102. Dosing was paused after the first SAE onset was observed and the study is on clinical hold. The study is expected to resume enrollment and dosing in the first half of 2021, following the resolution of FDA requests and approval to proceed.

Ultragenyx has submitted an Investigational New Drug (IND) application for UX701, an AAV9 gene therapy, for the treatment of Wilson Disease. The company expects to initiate a seamless, single-protocol phase I/II/III study in the first half of 2021.

It entered into a collaboration and license agreement with MereoBioPharma for setrusumab, a monoclonal antibody for the treatment of Osteogenesis Imperfecta (OI) in December 2020. 

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare sector include Alkermes Plc. ALKS, Halozyme Therapeutics Inc. HALO and Aerpio Pharmaceuticals Ltd. ARPO. While Halozyme carries a Zacks Rank #1 (Strong Buy), Alkermes and Aerpio carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alkermes’ earnings per share estimates have increased from 48 cents to 52 cents for 2021 over the past 60 days. Shares of the company have increased 12.9% in the past year.

Halozyme’s earnings per share estimates have increased from 85 cents to 92 cents for 2020 and from $1.66 to $1.72 for 2021 over the past 60 days.

Aerpio’s loss per share estimates have narrowed from 15 cents to 14 cents for 2020 and from 63 cents to 57 cents for 2021 over the past 60 days.

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