In this edition
Monday: UK NICE pricing warning; AstraZeneca hypertension win
Tuesday: CapVest-Stada buyout; Trump COVID-vaccine challenge
Wednesday: Novartis-Arrowhead Parkinson's; Merck oral PCSK9; Vertex-Enlaza
Thursday: Novartis-Argo China deal; RFK resignation calls; FDA ultra-rare pathway
Monday 1 September
The chief executive of NICE warned that meeting the drug industry's pricing demands would force cuts to NHS hospital care.
Sam Roberts, who runs the body that decides which medicines the NHS in England funds, told The Times that with the health budget fixed, any rise in what the NHS pays for drugs would have to come from the rest of the system: fewer A&E staff, fewer nursing posts, fewer operations such as hip replacements. Her comments sharpen a standoff between ministers and global manufacturers over UK pricing, with the companies pressing for higher prices and warning about investment.
Sources:
AstraZeneca's baxdrostat cut blood pressure in patients whose hypertension resists existing drugs, in full Phase III results presented in Madrid.
At the European Society of Cardiology congress on 30 August, and in the New England Journal of Medicine the same day, the company reported that baxdrostat lowered seated systolic blood pressure at 12 weeks by 9.8 mmHg at the 2 mg dose and 8.7 mmHg at 1 mg more than placebo, on top of standard treatment, in the BaxHTN trial. The drug, an aldosterone synthase inhibitor, met the primary and all secondary endpoints and was generally well tolerated, with the effect holding across age, sex and kidney function.
AstraZeneca said the treatment could cut the risk of stroke and heart attack for as many as ten million people in Britain and could reach patients in a little over a year, according to The Sunday Times.
Sources:
Baxdrostat delivered a statistically significant reduction in systolic blood pressure in the BaxHTN Phase III trial - AstraZeneca
AstraZeneca drug cuts heart attack and stroke risk - The Sunday Times
Tuesday 2 September
CapVest agreed to buy a majority stake in the German drug and consumer-health group Stada, in a deal reported to value the company at about 10 billion euros.
The London-based private equity firm said on 1 September it would take control of Stada from Bain Capital and Cinven, which each keep a minority holding, with completion expected in early 2026. Stada, a maker of generics and over-the-counter brands, reported 2024 sales of about 4 billion euros. Bain and Cinven bought it in 2017 for 5.3 billion euros and had explored a relisting before turning to a sale.
Including debt, the transaction values Stada at around 10 billion euros, Europe's largest healthcare deal of the year, according to the Financial Times.
Sources:
CapVest to acquire majority stake in STADA from Bain Capital and Cinven - Stada
Irish-led CapVest buys German drugmaker Stada in 10bn euro private equity deal - Financial Times
President Trump publicly challenged drug companies to prove their COVID-19 vaccines and treatments work.
In a Truth Social post on 1 September, Trump wrote that it was "very important that the Drug Companies justify the success of their various Covid Drugs," said the CDC was "being ripped apart over this question," and demanded an answer "NOW." He added that he had been shown extraordinary information from Pfizer and others that had never been made public. The post landed days after he removed CDC director Susan Monarez, which triggered a wave of senior resignations at the agency.
Sources:
Truth Social post on Covid drugs - Donald J. Trump, Truth Social
Wednesday 3 September
Novartis paid Arrowhead $200m up front to license an experimental RNA therapy for Parkinson's disease.
Announced on 2 September, the global agreement gives Novartis exclusive worldwide rights to ARO-SNCA, a preclinical small interfering RNA candidate that targets alpha-synuclein, the protein that accumulates in Parkinson's and related synucleinopathies. Arrowhead is eligible for up to $2bn in milestone payments plus royalties, and the deal extends Novartis's access to further central nervous system targets through Arrowhead's TRiM delivery platform.
Sources:
Arrowhead Pharmaceuticals and Novartis enter into a global license and collaboration agreement - Arrowhead Pharmaceuticals
Merck's oral cholesterol drug enlicitide passed another Phase III trial, moving it toward becoming the first PCSK9 inhibitor available as a pill.
On 2 September the company said enlicitide decanoate met all primary and key secondary endpoints in CORALreef Lipids, its largest completed late-stage study, cutting LDL cholesterol by about 56% more than placebo at 24 weeks in adults with high cholesterol, with safety comparable to placebo. PCSK9 inhibitors are potent cholesterol-lowering drugs given today only by injection, so an oral version would change how they are prescribed and taken.
Sources:
Enlicitide decanoate met all primary and key secondary endpoints in the pivotal CORALreef Lipids study - Merck & Co
Vertex signed a drug-discovery collaboration with Enlaza Therapeutics worth more than $2bn in potential payments.
Announced on 2 September, the deal pairs Enlaza's War-Lock platform, which builds protein drugs that bind their targets covalently, with Vertex's work in certain autoimmune diseases and in improved conditioning for sickle cell disease and beta thalassemia. Enlaza receives $45m up front, including an equity investment, and is eligible for more than $2bn in research, development, regulatory and commercial milestones plus royalties.
Sources:
Enlaza Therapeutics announces strategic collaboration with Vertex Pharmaceuticals - Enlaza Therapeutics
Thursday 4 September
Novartis committed up to $5.2bn for rights to cardiovascular drug candidates from the Chinese biotech Argo Biopharma.
Argo said on 3 September it would receive $160m up front, with milestone and option payments that could reach $5.2bn plus royalties, across several RNA-based heart-disease programmes, including candidates for severe hypertriglyceridaemia and mixed dyslipidaemia. It is the third transaction between the two companies and adds to a run of licensing deals that have left large drugmakers increasingly reliant on Chinese-discovered molecules to refill their pipelines.
Sources:
Twenty medical and public health organizations called on US Health Secretary Robert F. Kennedy Jr. to resign.
In a joint statement on 3 September, groups including the American Society for Microbiology and the Infectious Diseases Society of America said Kennedy was endangering the nation's health, citing his role in the firing of CDC director Susan Monarez, the resignations of senior CDC leaders, and the appointment of vaccine critics to federal advisory roles. They said his resignation was needed to restore the agency's scientific integrity.
Sources:
Joint statement calling for Secretary Kennedy resignation - American Society for Microbiology
The FDA set out a new pathway that lets treatments for ultra-rare genetic diseases win approval on a single trial.
Announced on 3 September, the Rare Disease Evidence Principles cover conditions driven by a known genetic defect that generally affect fewer than 1,000 people in the United States. For eligible drugs, the agency said one adequate and well-controlled study, which may be a single-arm trial, backed by confirmatory evidence such as data from a relevant disease model, can meet the standard for effectiveness. Sponsors can apply before starting a pivotal trial.
Sources:
Friday 5 September
Kennedy drew bipartisan criticism at a Senate Finance Committee hearing, with Republicans joining Democrats to challenge his handling of vaccines and the CDC.
At the 4 September hearing, titled "The President's 2026 Health Care Agenda," Republican senators including Bill Cassidy, John Barrasso and Thom Tillis pressed Kennedy over the removal of CDC director Susan Monarez, cuts to mRNA vaccine funding and tighter COVID-19 vaccine recommendations. The exchanges grew heated over roughly three hours, with Kennedy defending the firing and standing by his record on vaccines.
Sources:
Hearing: The President's 2026 Health Care Agenda - United States Senate Committee on Finance
The FDA said it would publish drug rejection letters as it issues them, and released 89 tied to applications that were never approved.
In a 4 September announcement, the agency said it would post complete response letters, the notices it sends when it declines to approve a drug or biologic, in real time going forward, rather than only after an eventual approval. The batch of 89 covers pending or withdrawn applications, the first time the agency has released letters for submissions still in play. The letters are redacted for trade secrets and personal information.
Sources:
ALETH · OPEN ACCESS RESEARCH