Following the presentation of detailed Phase III data last week, Alnylam Pharmaceuticals Inc.’s Amvuttra seems likely to become the first gene silencer approved for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). But perhaps not the last: Ionis Pharmaceuticals, Inc. and AstraZeneca PLC are awaiting pivotal data on Wainua (eplontersen) in the disease, and their decision to enroll huge numbers of patients could give their product an edge.
AstraZeneca Believes Size Matters In Amyloidosis
Following the pivotal data on Alnylam’s Amvuttra, all eyes turn to the next amyloidosis readout – that of AstraZeneca and Ionis’ Wainua. And the UK major’s ambitions do not stop there.

More from Clinical Trials
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
The biotech is banking on itolizumab’s longer-term efficacy as well as unmet need in frontline acute graft-versus-host disease as it prepares to meet with the FDA.
The head of the Spanish medical dermatology specialist told Scrip that maintaining the status quo will only result in the continent’s life sciences sector slipping further behind the US and China.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
More from Leadership
Stakeholders are pleading for newly confirmed FDA Commissioner Martin Makary to stand up for the agency's high scientific standards and staff as he begins his term.
The Danish firm is spending nearly $580m to repurchase up to 2.2 million shares
Heads of Novartis Biomedical, World Economic Forum and Indian majors like Sun Pharma’s SPARC, among others, discuss generative AI in drug discovery along with pointers for India to leapfrog the R&D process