Abivax SA has confirmed plans to submit a new drug application for obefazimod, its candidate product for ulcerative colitis, to the US Food and Drug Administration late in the fourth quarter. This follows results from a Phase 3 trial that showed strong efficacy. The small molecule drug upregulates a microRNA known as miR124 in order to stabilise the immune response in patients with ulcerative colitis, a disease that affects the colon.
Les Laboratoires Servier SAS of France has extended its reach into the rare disease space with the acquisition of the muscular dystrophy business of Edgewise Therapeutics Inc of Boulder Colorado, US. The lead asset is sevasemten, a small molecule drug that is being developed for both Becker muscular dystrophy and Duchene muscular dystrophy. The two disorders are caused by mutations in the DMD gene which encodes for the dystrophin protein.
Eli Lilly and Co has announced plans to invest up to $3.83 billion to buy three companies developing candidate vaccines, effectively charting new territory in the field of infectious disease. Announced on 26 May, the acquisitions include Curevo Inc and Vaccine Company Inc of the US, and LimmaTech Biologics AG of Switzerland.
Isomorphic Labs Ltd, with headquarters in London and global business offices in Lausanne, Switzerland and Cambridge, US, announced funding of $2.1 billion on 12 May, said to be the second largest equity funding round for a company since the start of the biotechnology industry. The Series B round comes only a year after the company raised $600 million for its activities, the company’s first external financing since its launch in 2021.
Bristol Myers Squibb Co has signalled a commitment to artificial intelligence with a strategic agreement with Anthropic PBC to deploy the AI tool Claude across the company’s research, clinical development, manufacturing, commercial and corporate functions. Announced on 20 May, the agreement will make the technology available to more than 30,000 BMS employees. The financial terms of the agreement were not disclosed.
A UK company with an ambition to develop a new class of therapeutics based on laboratory grown red blood cells has raised £3.2 million in seed funding. The financing was announced on 7 May and awarded to Scarlet Therapeutics Ltd which was founded by the University of Bristol, UK, professors Ash Toye and Jan Frayne. The company’s technology platform is based on engineered red blood cells with a potential for delivering therapeutic proteins in vivo to humans. The proteins remain inside the red blood cell membrane and therefore are not recognised by the immune system.
ERS Genomics Ltd, which manages licensing agreements for the CRISPR/Cas9 gene editing tool, is to supply the technology to Aurigene Pharmaceutical Services Ltd in India, enabling the company to integrate gene editing into its drug discovery activities. Aurigene is a global contract research, development and manufacturing organisation. The deal was announced on 13 May.
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.