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Ultragenyx's (RARE) IND for Wilson Disease Cleared by FDA

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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced that the FDA has cleared the Investigational New Drug (IND) application for UX701, being evaluated for the treatment of Wilson Disease. Wilson disease is a rare inherited disorder, in which excess copper builds up in the body. Its symptoms are typically related to the brain and liver.

Shares of the company have decreased 3.9% in the past year against the industry’s 9.7% growth.

UX701 is an investigational AAV type 9 gene therapy designed to deliver a stable expression of the ATP7B copper transporter following a single intravenous infusion. Data from the preclinical studies showed that the candidatenormalizes copper trafficking and excretion from the body. 

UX701 will be studied in a seamless, single-protocol phase I/II/III study.

In the first stage of the study, the safety and efficacy of three dose levels of UX701 will be evaluated in 27 patients, randomized 2-to-1 (gene therapy versus placebo). The dose cohorts will be enrolled sequentially using ascending doses. The patients will be followed for 52 weeks before transitioning to long-term follow-up and selecting a pivotal dose.

The second and pivotal stage will use the dose selected from Stage 1 and enroll an additional 63 patients, randomized 2-to-1 (gene therapy versus placebo). The co-primary endpoints in Stage 2 will evaluate the effect of UX701 on copper regulation based on 24-hour urinary copper concentration and percent reduction in the current standard of care (SOC) at Week 52.

All patients in Stage 1 and Stage 2 who are randomized to receive placebo may be eligible to receive UX701 at the Stage 2 dose. Patients receiving UX701 will continue being monitored for long-term safety and durability of response.

Enrolment in a seamless single-protocol phase I/II/III is expected to begin in the first half of 2021.

The available treatment for Wilson diseaseinvolves complicated use of medications that block the absorption of copper from the diet or remove copper by chelation. However, UX701 is designed to directly address the underlying cause of the disease by restoring normal copper metabolism in the liver. Thus, this one-time treatment has the potential tobetter addressmany serious effects of this disease.

Last month, the FDA granted an Orphan Drug designation to UX701.

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 - Free Report) , Acorda Therapeutics Inc. (ACOR - Free Report) and Alexion Pharmaceuticals Inc. (ALXN - Free Report) . While Alkermes and Acordacarry a Zacks Rank #1 (Strong Buy), Alexion carries 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.

Acorda’s loss per share estimates have narrowed from $9.66 to $8.48 for 2020 and from $9.60 to $7.33 for 2021 over the past 60 days.

Alexion’s earnings per share estimates have increased from $12.04 to $12.06 for 2020 and from $12.77 to $12.78 for 2021 over the past 60 days.

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