AstraZeneca Plc 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. These developments mark significant milestones across the company's rare disease and oncology portfolios. The Enhertu approval triggers a $25 million milestone payment to partner Daiichi Sankyo.

Efzimfotase Alfa Meets Primary And Secondary Endpoints

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.

Safety Profile And Injection-Site Reactions

In the Phase 3 CHESTNUT trial, children who switched from Strensiq to efzimfotase alfa experienced treatment-emergent adverse events at rates comparable to those who remained on Strensiq, while maintaining bone health outcomes through week 25. 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.

AstraZeneca and YMCA of the USA (Y-USA) have formed a five-year partnership to address gaps in cancer care by strengthening education in screening and early detection. The collaboration aims to reach 175,000 people across 75 communities nationwide over the first two years. This initiative seeks to increase awareness of cancer screening and support survivors through evidence-based programs.

The partnership was announced at the Aspen Ideas: Health conference. It leverages Y-USA’s 175-year legacy in community health and AstraZeneca’s ambition to eliminate cancer as a cause of death. The initial focus is on tailored, community-driven solutions to improve early detection rates and support individuals navigating their cancer journey.

Key Objectives and Reach

The collaboration targets significant disparities in cancer screening rates across different demographics. By combining AstraZeneca’s global expertise in life-changing innovations with the YMCA’s expansive community reach, the partners aim to transform the nation’s approach to cancer care.

Leadership Perspectives

Mohit Manrao, Senior Vice President, Head of US Oncology, AstraZeneca and President, AstraZeneca Foundation, emphasized the need for grassroots intervention. "We are in the golden era of cancer care, yet cancer screening and early detection rates for many types of cancer remain low, with significant disparities across zip codes," Manrao said. "Our partnership with Y-USA can directly reach millions of Americans at scale and at the grassroots level because every patient, regardless of zip code, deserves support throughout their cancer journey."

Suzanne McCormick, President and CEO, Y-USA, highlighted the complementary strengths of the two organizations. "We are bringing together AstraZeneca’s global expertise in life-changing innovations and the YMCA’s expansive community reach to transform our nation’s approach to cancer care," McCormick stated. "Over the next five years, we will expand screening, early detection, and survivorship programs to reach more people in the places they live and help them lead longer, healthier lives."

Context and Data

New data indicates that while people are living longer after a cancer diagnosis due to advances in treatment, more people are being diagnosed with the disease. A 2025 survey from the Prevent Cancer Foundation reveals that 51% of US adults aged 21 and older reported having a routine medical appointment or cancer screening in the last year, a 10% drop compared to its 2024 survey. However, 73% of those surveyed were more likely to schedule routine cancer screenings after learning about the benefits of early detection. The five-year survival rate for many types of cancer is almost 90% when found early.