Company News

A gene therapy developed to treat hearing loss has been given an accelerated approved by the US Food and Drug Administration following the results of an ongoing single-arm trial. Developed by Regeneron Pharmaceuticals Inc, Otarmeni (lunsotogene parvec) was approved on 23 April and is the first gene therapy authorised for a genetic hearing loss. The disorder, OTOF-related hearing loss, is an ultra-rare condition affecting about 50 children born every year in the US. It is caused by variants in the OTOF gene and the lack of a functional otoferlin protein.

Baxfendy (baxdrostat), a small molecule drug in-licensed and developed by AstraZeneca Plc, has been approved by the US Food and Drug Administration to treat adults with hypertension – a disease estimated to affect 1.4 billion people worldwide and a leading risk factor for cardiovascular disease and premature death. Announced on 18 May, the approval is for the use of Baxfendy in combination with other antihypertensive medicines to lower blood pressure in adults who are not adequately controlled.

Eli Lilly and Co’s agreement on 20 April to acquire Kelonia Therapeutics Inc in a deal worth up to $7 billion puts the company in place to exploit the emerging field of in vivo CAR-T therapy which has been described by analysts as a way to achieve therapeutic value without a complex production step. This compares with the traditional ex vivo approach in which T cells are extracted from a patient, modified in a laboratory, and then reinfused into the patient.

AstraZeneca Plc has reported trial data for a new combination therapy directed against muscle-invasive bladder cancer which showed improvements in event-free survival for the patients concerned. The muscle-invasive disease is estimated to affect one in four people with bladder cancer and arises when a tumour invades the bladder’s muscle wall. The standard treatment for these patients is surgery, with or without an adjuvant treatment.

Bristol Myers Squibb Co announced a drug discovery and development agreement with Jiangsu Hengrui Pharmaceuticals Co Ltd on 12 May which is expected to yield 13 new products across the oncology, haematology, and immunology sectors and be commercialised across the globe. The deal comes as Hengrui is expanding its market presence outside China and BMS is taking steps to generate new revenue in response to a loss of patent protection on some important medicines.

A panel of life science investors raised concerns at the AngloNordic conference in London on 23 April that early-stage biotech companies are struggling to raise finance. Stephen Hansen, director of biopharma intelligence at BioCentury, cited data showing pre-clinical companies made up 41-42% of financings in 2021/22, but just 25% in 2025. He worried that a vital part of the biotech sector was being neglected in favour of clinical stage ventures, saying: "Somebody's got to be making companies....

Novartis continued to navigate the patent cliff in the first quarter of 2026 as sales of leading drugs such as Entresto for heart failure faced stiff competition from generics. Net sales for the company as a whole fell by 5% to $13 billion at constant exchange rates and by 1% at actual rates. Operating income contracted by an even larger amount as the company increased spending on research and development. The allocation for R&D in the quarter was $2.7 billion compared with $2.4 billion a year earlier.

With Luke Miels in place as chief executive, GSK Plc is taking steps to simplify operations and identify new opportunities in its late-stage pipeline. A portfolio review is underway to identify 50 promising assets that could be the focus of drug development in the coming years. Mr Miels became CEO on 1 January, succeeding Emma Walmsley. He had previously been the company’s chief commercial officer having joined GSK in 2017.

UCB SA announced on 3 May that it has signed an agreement to acquire Candid Therapeutics Inc of San Diego, US, broadening its portfolio to include biologics targeting autoimmune and inflammatory diseases. The Belgium-based biopharma company already has marketed products for epilepsy, plaque psoriasis, and Crohn’s disease. The acquisition will add preclinical and early clinical-stage products to UCB’s portfolio, the most advanced of which are bispecific T cell engagers. These are antibodies that can bind T cells to tumour cells to kill cancers.

AstraZeneca Plc expects revenue this year to grow by a mid-to-high single-digit percentage as label expansions for existing drugs and new molecular entities progress towards market entry.  At a briefing for journalists on 29 April, Pascal Soriot, the chief executive, confirmed the company’s ambition to achieve revenue of $80 billion by 2030. He said the company is advancing through a “catalyst-rich period” with multiple positive Phase 3 readouts and regulatory submissions underway.