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AstraZeneca's (AZN) Rare Disease Study Meets Primary Goal

AstraZeneca's (AZN) phase III study evaluating ALXN1840 for Wilson disease achieves primary endpoint. The company also receives approval in Japan for...

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This story originally appeared on Zacks

AstraZeneca AZN announced that the phase III pivotal FoCus study evaluating its oral candidate, ALXN1840, for Wilson disease met its primary endpoint of improvement in copper mobilisation from tissues when compared with standard-of-care (“SoC”) treatments.

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Per the study, the company assessed this copper mobilisation by measuring the daily mean Area Under the Effect Curve for directly measured non-ceruloplasmin-bound copper over 48 weeks. Data from the study demonstrated that ALXN1840 achieved approximately three times greater copper mobilisation from tissues than SoC.

The company’s stock has rallied 17.2% so far this year in comparison with the industry’s 16.9% increase.

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The FoCuS study compared the efficacy and safety between ALXN1840 and SOC in patients with Wilson disease aged 12 years and older. The study enrolled 214 patients in two cohorts consisting of treatment-experienced patients and treatment-naïve patients. While the first cohort enrolled 161 patients who received SoC for more than 28 days, the second cohort enrolled 53 patients who were treatment naïve or had received SoC for 28 days or less.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised. Over time, this build-up of excess copper can cause damage and greatly impact a patient’s life. While presently available medicines remove copper from the blood, the latest data shows ALXN1840’s significant impact on mobilising copper from tissues.

Please note that ALXN1840 is one of the many pipeline products acquired by the company since its purchase of Alexion Pharmaceuticals, which closed last month. Following the acquisition, the company set up Alexion as a separate unit to focus on drugs for rare diseases.

In a separate press release, AstraZeneca announced that its SGLT2 inhibitor, Forxiga, has received approval from health regulators in Japan for the treatment of chronic kidney disease (“CKD”) in adults with and without type-II diabetes. CKD is a serious, progressive condition defined by decreased kidney function.

This approval is based on data from the DAPA-CKD phase III study, which demonstrated that Forxiga reduced the relative risk of worsening renal function and cardiovascular or renal death by 39% in comparison with SoC treatment, and the onset of end-stage kidney disease.

The approval in Japan is one of the many approvals received by AstraZeneca for Forxiga that will help boost the drug’s revenue prospects. Please note that the drug received approval for the CKD indication in the United States and Europe in April 2021 and August 2021, respectively.

Zacks Rank & Stocks to Consider

AstraZeneca currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector include Horizon Therapeutics HZNP, Regeneron Pharmaceuticals REGN and Vertex Pharmaceuticals VRTX, each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Horizon’s earnings per share estimates for 2021 have increased from $3.62 to $4.46 in the past 30 days. The same for 2022 has risen from $5.18 to $5.84 over the same period. The stock has rallied 49.1% in the year so far.

Regeneron’s earnings per share estimates for 2021 have increased from $49.96 to $54.15 in the past 30 days. The same for 2022 has risen from $40.91 to $44.11 over the same period. The stock has rallied 38.5% in the year so far.

Vertex’s earnings per share estimates for 2021 have increased from $11.22 to $12.37 in the past 30 days. The same for 2022 has risen from $12.24 to $13.13 over the same period.



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