News

GSK Plc is to expand its portfolio of products for the treatment of liver disease with the acquisition of an asset to treat and prevent the progression of steatotic liver disease (SLD), formerly known as fatty liver disease. Announced on 14 May, the deal features an upfront payment of $1.2 billion for the asset with potential for additional success-based milestones of $800 million. The liver disease affects a significant percentage of the global population and is mainly driven by obesity, type 2 diabetes, and alcohol intake, according to a 2024 review in The Lancet. 

CRISPR base editing has delivered a landmark n-of-1 gene therapy for an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, in a clinical milestone that may redefine how rare genetic diseases are treated. CPS1 deficiency is a mitochondrial disorder caused by a loss-of-function mutation in the CPS1 gene that impairs the urea cycle and leads to life-threatening hyperammonaemia.

Lars Fruergaard Jørgensen, chief executive of Novo Nordisk A/S, who has led the Danish company for eight years including a period of strong growth with the launch of two obesity drugs, is to step down. The management change was apparently taken under pressure from the Novo Nordisk Foundation which controls a majority of the company’s shares. It follows a steep decline in the company’s shares after Phase 3 data for a new obesity compound failed to meet investor expectations. It did however meet the trial’s primary endpoint.

A consortium led by Jeito Capital of France has raised €132 million in new funds for Azafaros BV of the Netherlands enabling it to advance an experimental compound for rare metabolic disorders into Phase 3. Co-leading the Series B round was Forbion, whose operating partner Carlo Incerti is chair of the company’s board of directors. Joining the board as a new member will be Audrey Cacaly, a principal at Forbion Growth, which is a fund supporting late-stage assets.

TILT Biotherapeutics Ltd has raised €22.6 million from the European Innovation Council and other investors to bring its oncolytic virus therapy for patients with ovarian cancer into Phase 2. The funding will also support earlier studies for the product, TILT-123, in other indications.  TILT-123 is an oncolytic adenovirus encoding tumour necrosis factor alpha and interleukin 2. It is designed to work alongside T cell therapies and immune checkpoint inhibitors to target and kill cancer cells.

Efpia, the federation that represents pharmaceutical companies in Europe, has deferred comment on President Donald Trump’s executive order on 12 May which is intended to lower the prices of prescription drugs in the US. The order would introduce the Most Favoured Nation policy to drugs. This is a principle of the World Trade Organization under which all nations treat their trading partners equally. In the context of the Trump order, it is being interpreted to mean that US drug prices would be tied to those set in other countries.

Galapagos NV is to revisit its plan to separate into two companies by creating a spin-out focused on innovation, and leaving the parent company to develop cell therapies. The revaluation of the company’s strategy was announced on 13 May together with the appointment of a new chief executive officer who will succeed Paul Stoffels, a former Johnson & Johnson Inc manager who has led Galapagos since 1 April 2022. The new CEO is Henry Gosebruch, most recently head of Neumora Therapeutics Inc, a US-based neuroscience drug developer.

A new nonsteroidal treatment for Duchenne muscular dystrophy (DMD) has been recommended in Europe for a conditional marketing authorisation to treat patients from the age of six. The drug, Duvyzat (givinostat), was approved by the US Food and Drug Administration in 2024. It was given a positive opinion by the European Medicines Agency on 25 April. Duvyzat is an oral suspension to be taken at the same time as corticosteroid, the current standard of care. The EMA is recommending the drug for patients who can still walk.

Novartis achieved a double digit increase in operating profit during the first quarter as sales rose strongly and expenses declined. Even taking upcoming generic competition for three products later in the year, the company is forecasting a high single-digit rise in sales for 2025 and a low double-digit increase in core operating profit. The core operating profit excludes the amortisation and impairment of intangible assets.

Novartis is to pay up to $1.7 billion to buy Regulus Therapeutics Inc of San Diego, US, in a deal that will strengthen its portfolio of treatments for kidney disease. The deal also puts a spotlight on microRNA, a drug target that affects gene expression without coding for a protein. Regulus has an early clinical stage product for autosomal dominant polycystic kidney disease, a serious genetic disorder that can lead to end-stage renal disease. The product, farabursen, is an oligonucleotide designed to inhibit a microRNA known as miR-17.