News

AstraZeneca Plc is to expand its pipeline of cell and gene therapies with the acquisition of the China-based company Gracell Biotechnologies Inc which has a chimeric antigen receptor (CAR) T cell therapy in development for multiple myeloma. The deal value is $1.2 billion which includes an upfront cash payment as well as potential contingent value payments linked to the achievement of an undisclosed regulatory milestone. The transaction is expected to close in the first quarter of 2024.

A chimeric antigen receptor (CAR) T cell therapy approved by the US Food and Drug Administration in 2022 for multiple myeloma has been given a boxed warning by the agency after patients treated with the drug developed secondary malignancies. The product, Carvykti (ciltacabtagene autoleucel), consists of a patient’s T cells that have been engineered to attach to the B cell maturation antigen (BCMA) on multiple myeloma calls and kill them. The FDA approved the therapy on the basis of deep and durable responses by patients taking the therapy.

Bristol Myers Squibb Co is to acquire the Boston, US, based company Karuna Therapeutics Inc for $14 billion in order boost its presence in neuroscience. Karuna has a candidate product for schizophrenia in registration at the US Food and Drug Administration which is expected to come up for a decision on 26 September 2024. If approved, it would provide new revenue for BMS whose other neuroscience products are currently in Phase 1.

Sanofi has stopped a global programme evaluating the clinical effectiveness of tusamitamab ravtansine, an antibody-drug conjugate (ADC), after a Phase 3 trial of the drug failed to improve progression-free survival in patients with a type of non-small cell lung cancer. Tusamitamab ravtansine was targeting a transmembrane cell adhesion molecule – carcinoembryonic antigen-related cell adhesion molecule 5 – which is associated with a number of cancers. 

argenx SE reported the second failure of a trial involving its antibody fragment efgartigimod alfa on 20 December following unexpectedly positive results from the placebo arm of the study. Called ADDRESS, the Phase 3 trial was investigating a subcutaneous formulation of efgartigimod (efgartigimod alfa and hyaluronidase) in adults with two types of pemphigus, an autoimmune disease that affects the skin and mucous membranes. 

A small molecule drug in development by Vertex Pharmaceuticals Inc has reduced pain in patients with diabetic peripheral neuropathy, a type of nerve damage that can occur with the disease. A Phase 2 trial of VX-548 which inhibits NaV1.8, a voltage-gated sodium channel, showed a statistically significant reduction in a measure of pain, Vertex announced on 13 December 2023. Patients with diabetic peripheral neuropathy are often treated with anti-seizure drugs or antidepressants, according to the Mayo Clinic. Opioid analgesics can also be used as second or third-line agents.

France-based Sofinnova Partners is to take a direct role in creating new biotech companies in Europe in order to accelerate the development of novel medicines for patients. The strategy, announced on 13 December, follows similar moves by other venture capitalists who are seeking to capture the value of healthcare innovations early and guide development to a successful conclusion. Sofinnova did not disclose the size of its investment in the project which is called Biovelocita.

However it did say that the strategy has already been successful in Italy.

The US Food and Drug Administration has approved a new drug for the treatment of desmoid tumours, a rare subtype of soft tissue sarcoma. Desmoid tumours are non-cancerous and typically treated with surgery, but not all patients are eligible for this procedure. And even when surgery is an option there is a risk that the tumours will return, affecting a person’s quality of life, according to the FDA.

Switzerland-based BC Platforms AG is to increase its capacity as a provider of healthcare data to the pharmaceutical industry with the acquisition of Medexprim SAS of France. The financial terms of the transaction, which was announced on 7 December 2023, were not disclosed.

AstraZeneca Plc is to acquire Icosavax Inc of Seattle, US, a developer of virus-like particle (VLP) vaccines in a bid to strengthen its late-stage pipeline for candidate vaccines and immune therapies. The deal, valued at up to $1.1 billion if all contingent requirements are met, will build on the UK company’s experience developing two products for the prevention of respiratory syncytial virus (RSV), a major cause of acute respiratory infection in individuals of all age groups.