Alexion Presents Promising Data on Ultomiris and Soliris at 2025 AAN Annual Meeting

On March 27, 2025, Alexion, AstraZeneca Rare Disease, announced it will present 21 abstracts—including three oral presentations—at the upcoming American Academy of Neurology (AAN) Annual Meeting in San Diego (April 5–9, 2025). The presentations focus on long-term safety and efficacy data for **Ultomiris (ravulizumab)** and **Soliris (eculizumab)** in treating **neuromyelitis optica spectrum disorder (NMOSD)** and **generalised myasthenia gravis (gMG)**.

Highlights include:

- **CHAMPION-NMOSD trial** data showing that **no relapses occurred** among Ultomiris-treated patients during a median follow-up of over 170 weeks.
- Real-world data from Japan confirming Soliris' effectiveness in gMG patients, with sustained benefits across severity levels and for those switching to Ultomiris.
- Registry-based evidence demonstrating decreased corticosteroid use and fewer hospitalisations among gMG patients on C5 inhibitors.
- Biomarker research suggesting that glial fibrillary acidic protein (GFAP) and neurofilament light chain (NfL) levels may provide insights into disease activity in NMOSD, though not predictive of relapse risk.
- Early findings from a smartphone-based study (ME&MGopen™) show potential for digital tracking of daily symptom impact in gMG patients.
- Ongoing trials, including a Phase III study of Ultomiris in paediatric gMG and the PREVAIL trial of the investigational drug **gefurulimab**, were also featured.
- Project ASPIRE results shed light on health disparities in gMG, emphasizing the need for equitable access to care for people of color.

These findings further support the safety and long-term benefit of Ultomiris and Soliris, reinforcing Alexion's leadership in rare neurological disease care and its commitment to advancing treatment equity and patient-focused innovation.

AstraZeneca Reinforces Tagrisso as Backbone Therapy for EGFR-Mutated Lung Cancer

On March 25, 2025, AstraZeneca presented new clinical trial results at the European Lung Cancer Congress, highlighting the continued efficacy of Tagrisso (osimertinib) across various stages and treatment settings of EGFR-mutated non-small cell lung cancer (NSCLC). The findings reinforce Tagrisso’s role as a foundational therapy, both as a standalone treatment and in combination with other targeted therapies.

In the Phase III LAURA trial, Tagrisso demonstrated a trend toward improved overall survival in patients with unresectable Stage III EGFRm NSCLC following chemoradiotherapy. Median overall survival reached 58.8 months for patients treated with Tagrisso compared to 54.1 months for those on placebo.

Phase II results from the SAVANNAH and ORCHARD trials explored combination treatments for patients who experienced disease progression on first-line Tagrisso. SAVANNAH showed that combining Tagrisso with Orpathys (savolitinib) produced a 56% objective response rate in patients with high MET expression or amplification. Similarly, Tagrisso plus Datroway (datopotamab deruxtecan) in the ORCHARD trial demonstrated encouraging response rates and progression-free survival, especially at the higher dose.

Additionally, the FLAURA2 trial confirmed a strong progression-free survival benefit when Tagrisso was combined with chemotherapy in the first-line treatment of advanced EGFRm NSCLC, with median PFS exceeding two years regardless of pemetrexed maintenance duration.

These findings underscore Tagrisso’s clinical value across the treatment spectrum, from early-stage to advanced disease, and support AstraZeneca’s broader strategy of developing innovative, well-tolerated therapies to meet significant unmet needs in lung cancer care.

On March 21, 2025, AstraZeneca announced a $2.5 billion investment in Beijing to establish its sixth global strategic R&D centre and expand partnerships with Chinese biotech firms. The new R&D centre—AstraZeneca’s second in China after Shanghai—will focus on early-stage research and clinical development, supported by a new AI and data science lab within Beijing’s BioPark. The initiative is part of a broader partnership with the Beijing Municipal Government and includes agreements with Harbour BioMed (multi-specific antibodies), Syneron Bio (macro-cyclic peptides), and a joint venture with BioKangtai to develop and manufacture vaccines. AstraZeneca’s Beijing workforce is expected to grow to 1,700 employees.

Harbour BioMed Enters into Global Strategic Collaboration with AstraZeneca to Discover and Develop Next-Generation Therapeutic Antibodies

SOURCE: PR NEWSWIRE

AstraZeneca announced that eneboparatide (AZP-3601), an investigational parathyroid hormone receptor 1 agonist, met the primary endpoint in the CALYPSO Phase III trial for chronic hypoparathyroidism (HypoPT). The treatment demonstrated statistical significance in normalizing serum calcium levels and eliminating the need for active vitamin D and oral calcium therapy after 24 weeks.

HypoPT is a rare endocrine disorder affecting over 200,000 people in the US and EU, with 80% of cases occurring in women. It results in impaired calcium and phosphate regulation, leading to complications such as hypercalciuria, osteopenia, and osteoporosis.

Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease, highlighted the potential of eneboparatide as a new treatment option and emphasized the importance of completing the 52-week trial to fully assess its risk-benefit profile. The trial's extension period will continue until week 52, after which AstraZeneca plans to present full safety and efficacy data to global health authorities.

Eneboparatide has received fast-track and orphan drug designation from the US FDA and orphan designation from the European Medicines Agency. The treatment aims to restore parathyroid hormone function while preserving kidney function and bone health.

For more information, visit www.alexion.us or www.astrazeneca-us.com.

AstraZeneca has canceled its planned £450 million investment in a vaccine manufacturing plant in Speke, Liverpool, citing protracted discussions with the UK government and a reduction in state funding. The decision marks a setback for the UK’s ambitions to strengthen domestic vaccine production. The planned facility was intended to expand AstraZeneca’s flu vaccine manufacturing capacity, but the company stated that the current site will continue operations. Despite the cancellation, AstraZeneca is shifting its focus to international investments, including a £460 million expansion in Canada to support scientific innovation and job creation. The move has raised concerns about the UK’s ability to attract and retain major pharmaceutical investments.

AstraZeneca announced a C$820 million (US$570 million) investment in Canada, creating over 700 high-skilled jobs and supporting a move to a state-of-the-art facility in the Greater Toronto Area. This investment aligns with AstraZeneca's global goals to achieve US$80 billion in revenue and deliver 20 new medicines by 2030, with eight already introduced and seven Phase III clinical trial readouts expected in 2025.

AstraZeneca has been a leading R&D investor in Canada, contributing over C$230 million in 2023, supporting more than 210 global clinical studies. Including this new funding, AstraZeneca's investments in Canada since 2023 exceed C$1.3 billion, creating 1,200 jobs. In 2024, AstraZeneca also completed a C$3 billion acquisition of Fusion Pharmaceuticals in Hamilton, Ontario, further boosting its cancer radiotherapy research.

CEO Pascal Soriot emphasized Canada’s potential as a global hub for life sciences, citing its diverse talent pool and collaboration with Ontario’s government. Premier Doug Ford highlighted the government's support of C$16.1 million via Invest Ontario to attract jobs and innovation to the province.

AstraZeneca's Canadian R&D and rare disease hubs are leading clinical studies in oncology, respiratory, and rare diseases, solidifying the company’s role in advancing global healthcare innovation.

AstraZeneca announced the US FDA approval of Calquence (acalabrutinib) combined with bendamustine and rituximab for adult patients with previously untreated mantle cell lymphoma (MCL) who are ineligible for stem cell transplantation. This marks the first BTK inhibitor approved for first-line treatment of MCL in the US. The decision was based on the ECHO Phase III trial, which showed significant improvement in progression-free survival compared to chemoimmunotherapy alone, extending it by more than 16 months.

The trial highlighted the combination's ability to reduce the risk of disease progression or death by 27% compared to standard treatment, with a median progression-free survival of 66.4 months versus 49.6 months. The safety profile of Calquence was consistent with previous data, and no new safety concerns were identified.

This approval also converted Calquence’s prior accelerated approval for MCL to full approval. Regulatory submissions for the combination therapy are under review in other countries, including Australia, Canada, Switzerland, the EU, and Japan. Calquence, a selective BTK inhibitor, has been used to treat over 85,000 patients globally and is approved for various B-cell blood cancers. This milestone reinforces AstraZeneca’s commitment to advancing cancer treatment.

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