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Ultragenyx (RARE) Focuses on Pipeline Amid Low Revenues

Zacks Equity Research

We issued an updated report on Ultragenyx Pharmaceutical Inc. RARE on Aug 22.

The company has two approved therapies in its portfolio. Mepsevii (vestronidase alfa) was approved in the United States, in November 2017, for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. The drug is under review in the EU for the same indication. Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 (FGF23), is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. The drug received European conditional marketing authorization, in February 2018, for the treatment of XLH with radiographic evidence of bone disease in children one year of age or older, and adolescents with growing skeletons. A filing for adults with XLH is also planned in Europe.

Shares of the company increased 31.1% year to date against the industry’s decline of 0.4%.

The company is also looking to expand the label of Crysvita. The drug is also being developed for the treatment of tumor-induced osteomalacia (TIO). The company is in discussions with the FDA regarding the regulatory pathway for the same. It is also developing the drug to treat children with XLH. The company released longer-term data from the phase III pediatric study of Crysvita for the treatment of XLH. The study showed superior efficacy of the drug compared with conventional oral phosphate treatments.

Ultragenyx is developing a synthetic triglyceride, UX007, for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). In August 2019, the company submitted to the FDA a new drug application (NDA) for the same. It expects to hear back from the agency on submission acceptance and review designation within 60 days. Additionally, discussions are progressing with the European Medicines Agency regarding a potential conditional marketing authorization in Europe. 

The company also has some gene-therapy candidates in its pipeline. These include DTX301, which is an adeno-associated virus 8 (AAV8) being evaluated for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency. It reported data from the first two dose cohorts of DTX301 gene therapy in the same indication. DTX301 phase I/II data from the third dose cohort are expected in the third quarter of 2019.

DTX401 is an AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa). 

The company is developing DTX201 in its FVIII gene-therapy program for the treatment of hemophilia A in collaboration with Bayer BAYRY. The investigational new drug (IND) application was accepted by the FDA e in the second quarter of 2018.

All of Ultragenyx’s current clinical product candidates are in-licensed from academic institutions or derived from partnerships with other pharmaceutical companies.

In August 2019, the company inked a deal with GeneTx Biotherapeutics LLC to develop the latter’s GTX-102, an antisense oligonucleotide (ASO), for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder. GTX-102 is currently in late-preclinical development, with an IND application expected to be filed with the FDA in the first half of 2020.

In June 2019, Ultragenyx and Arcturus Therapeutics Holdings Inc. ARCT expanded their existing collaboration to discover and develop mRNA, DNA and siRNA therapeutics for up to 12 rare disease targets.

In October 2018, the company exercised its option with REGENXBIO Inc. RGNX to develop a gene therapy to treat patients with CDD, using REGENXBIO’s adeno-associated virus (AAV) vectors like AAV9.

However, being a new commercial company with lower revenues, development or regulatory setbacks could result in higher operating expenses and the need for additional capital.


Ultragenyx Pharmaceutical Inc. Price


Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. price | Ultragenyx Pharmaceutical Inc. Quote

Ultragenyx currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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