News

Ionis Pharmaceuticals Inc announced positive data on 2 September from two Phase 3 trials for its RNA-targeted medicine olezarsen (Tryngolza) in patients at risk of acute pancreatitis events. Tryngolza has already been approved in the US for the treatment of familial chylomicronaemia syndrome, a genetic disease that prevents the body from breaking down triglyceride fats. The company is now seeking to expand the drug’s use for people with severe hypertriglyceridaemia. This disorder occurs when the levels of triglycerides are significantly elevated, increasing the risk of acute pancreatitis.

Cyted Health, a UK diagnostics company with a focus on gastrointestinal health, has raised $44 million to expand its business in the US, taking advantage of a Food and Drug Administration approval of its lead product and experience using the device for patients in the UK. Announced on 2 September, the Series B funding is being led by the private equity group EQT Life Sciences and co-led by Advent Life Sciences and the British Business Bank. It follows FDA approval of the product EndoSign in 2024. 

Wegovy (semaglutide), the weight-loss drug developed and now marketed by Novo Nordisk A/S, is able to cut the risk of heart attack, stroke or death by 57% compared with tirzepatide (Zepbound), the competing drug developed by Eli Lilly and Co, according to the Danish pharma company. Novo presented the comparative data, derived from a real-world study of the two drugs’ benefits, at the annual European Society of Cardiology meeting in Madrid on 31 August.

Roche and Alnylam Pharmaceuticals Inc are to advance their jointly developed RNA interference therapy for hypertension into a Phase 3 trial following clinical data showing that the drug, zilebesiran, could have a significant impact on patients with uncontrolled hypertension. The data comes from three Phase 2 trials which tracked the performance of the drug in patients with hypertension and high cardiovascular risk.

AstraZeneca Plc disclosed the full results of its Phase 3 trial of baxdrostat, a drug for hypertension, on 30 August, confirming the therapy’s ability to reduce systolic blood pressure in patients with hard-to-control hypertension. In a presentation at the European Society of Cardiology Congress in Madrid, the company said that baxdrostat, a small molecule drug that inhibits aldosterone synthase, met all primary and secondary endpoints, delivering sustained blood pressure reductions. 

Novartis Pharma AG has entered into an option and collaboration agreement with BioArctic AB to develop an antibody treatment for a neurodegenerative disorder that takes advantage of the Swedish company’s technology for helping drugs cross the blood-brain barrier. The target for the prospective treatment has not been disclosed. But the transporter technology is public and is being used by BioArctic in multiple projects. It is based on the transferrin receptor, a protein responsible for the delivery of iron to cells. This can also enable antibody drugs to cross the barrier.

Susan Monarez was dismissed as director of the US Centers for Disease Control and Prevention (CDC) on 28 August in a surprise move that also led to the resignations of other top officials including Debra Houry, the agency’s chief medical officer. Robert F Kennedy Jr, secretary of the Department of Health and Human Services (HHS), announced Dr Monarez’s departure on the social media site X. This was subsequently confirmed by the White House.

The US Food and Drug Administration has suspended the licence of a vaccine developed by Valneva SE to prevent chikungunya virus infection following 32 serious adverse event reports, including 21 hospitalisations and three deaths. The vaccine, Ixchiq (chikungunya vaccine, life), was given an accelerated approval by the FDA in November 2023 and since then has been approved in several other countries, some of which are also reviewing patient data. Valneva is expected to start a post-marketing study of the vaccine on 1 October to further assess the risks.

Efpia, the European federation of pharmaceutical industries and associations, has criticised a new US-EU trade agreement as potentially harmful to patients and the European pharma industry and called for exemptions for innovative medicines. The Efpia statement was issued on 21 August shortly after the European Commission published details of the framework agreement setting a maximum, all-inclusive tariff of 15% for the majority of EU exports to the US, including pharmaceuticals. The exact pharma rate is still to be determined but it would not exceed the 15% ceiling.

Anocca AB has raised SEK 440 million ($46 million) from Mellby Gård, a Swedish investment group, and existing shareholders to conduct a programme of trials of T cell immunotherapies for pancreatic cancer. The trials will investigate multiple approaches for targeting mutations in the KRAS gene which is involved in the regulation of cell division. KRAS mutations are drivers of pancreatic cancer, as well as lung and colorectal cancers.