Gilead Sciences has announced that it will be expanding its liver disease pipeline by acquiring CymaBay Therapeutics for $4.3bn.

The deal gives the company access to CymaBay’s lead experimental candidate, seladelpar, which is currently being evaluated as a treatment for primary biliary cholangitis (PBC).

Affecting approximately 130,000 people in the US, PBC is a rare cholestatic liver disease mainly affecting women.

According to results from the late-stage RESPONSE trial, seladelpar achieved statistical significance over placebo across primary composite endpoints of biochemical response, normalisation of alkaline phosphatase at 12 months, as well as statistically significant improvement in pruritus at six months that was sustained to month 12 among patients experiencing moderate-to-severe itch.

The candidate has already been accepted for priority review by the US Food and Drug Administration, with a Prescription Drug User Fee Act target action date of 14 August set.

Under the terms of the agreement, which is expected to close during the first quarter of this year, Gilead will acquire CymaBay for $32.50 per share in cash, a 27% premium to the company’s closing price on 9 February.

Novo Nordisk and Neomorph have entered into a collaboration and licensing agreement aimed at discovering and developing molecular glue degraders (MGDs) for cardiometabolic and rare diseases, with the deal potentially worth up to $1.46bn.

MGDs are believed to open up opportunities to eliminate therapeutically relevant proteins that have previously been considered ‘undruggable’ by other approaches.

In exchange for the use of its proprietary glue discovery platform, Neomorph will receive upfront and near-term milestone payments, research and development funding from Novo and will be eligible for future clinical, commercial and sales milestone payments plus tiered royalties.

Neomorph will lead the discovery and preclinical activities against selected targets, after which Novo will have the option to exclusively pursue further clinical development and commercialisation of the compounds.

The partnership comes just one week after Novo gave Almirall exclusive rights to develop its IL-21-blocking monoclonal antibody for immune inflammatory dermatological diseases.

Novo’s NN-8828 candidate has the potential to block the activation of the downstream signalling pathways of IL-21 and inhibit the pathophysiological functions induced by cytokines in several immune cells.

The European Commission (EC) has granted conditional marketing authorisation to Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy for use in transfusion-dependent beta thalassaemia (TDT) and severe sickle cell disease (SCD) patients aged 12 years and over.

Casgevy (exagamglogene autotemcel) is now the first treatment approved in the EU that uses the gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

SCD and beta-thalassaemia are both genetic diseases caused by errors in the genes for haemoglobin.

For those with SCD, this genetic error can lead to organ damage, a shortened lifespan and vaso-occlusive crises (VOCs).

In beta-thalassaemia, the error can lead to anaemia, with patients affected by the most severe form of the disease requiring regular blood transfusions and iron chelation therapy throughout their lives.

Casgevy is designed to edit the faulty gene in patients’ bone marrow stem cells so that the body produces functioning haemoglobin.

The therapy has been specifically authorised by the EC for use in SCD patients with recurrent VOCs or those with TDT for whom haematopoietic stem cell transplantation is appropriate but not available.