AstraZeneca Advances Rare Bone Disease Pipeline While Enhertu Expands In Europe

On Sunday, AstraZeneca Plc (NYSE: AZN ) reported positive late-stage data for investigational therapy efzimfotase alfa in children with hypophosphatasia (HPP), while separately securing European Union approval for cancer drug Enhertu in a broad group of HER2-positive solid tumors, marking two significant milestones across its rare disease and oncology portfolios.

Efzimfotase Alfa Meets Primary And Secondary Endpoints In HPP Trial The company said data from the Phase 3 MULBERRY trial showed efzimfotase alfa, also known as ALXN1850, significantly improved bone health in children aged 2 to under 12 years with HPP who had not previously received Strensiq (asfotase alfa).

Patients treated with efzimfotase alfa achieved a median Radiographic Global Impression of Change (RGI-C) score of 1.67 at week 25, compared with 0 in the placebo group, representing a statistically significant difference (p=0.0003).

The trial also met its key secondary endpoint, with patients receiving the therapy showing significant improvement in Rickets Severity Score versus placebo.

Beyond bone health, efzimfotase alfa demonstrated improvements in measures of physical function and quality of life, including gains in Pediatric Outcomes Data Collection Instrument scores and clinically meaningful improvement in the Six-Minute Walk Test.

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AstraZeneca said efzimfotase alfa was generally well-tolerated across all three Phase 3 studies.

A pooled analysis from the MULBERRY and HICKORY studies showed the annualized injection-site reaction rate was five times lower with efzimfotase alfa than that observed with Strensiq in registrational studies.

Patients treated with efzimfotase alfa also recorded a median of 98.8% injection-site-reaction-free days during treatment.

Enhertu Receives EU Tumor-Agnostic Approval Separately, AstraZeneca and Daiichi Sankyo announced that Enhertu (trastuzumab deruxtecan) received approval from the European Commission as a monotherapy for adults with unresectable or metastatic HER2-positive solid tumors who have previously received treatment and lack satisfactory treatment options.

The approval was based on data from the Phase 2 DESTINY-PanTumor02, DESTINY-Lung01, and DESTINY-CRC02 trials, which demonstrated confirmed objective response rates ranging from 46.9% to 52.9% across multiple tumor types.

Following the approval, AstraZeneca will make a $25 million milestone payment to Daiichi Sankyo tied to the tumor-agnostic indication.

AZN Stock Price Activity: AstraZeneca shares were up 0.64% at $189.62 at the time of publication on Monday, according to Pro data.

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