News

Sarepta Therapeutics Inc, developer of the gene therapy Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy, faced a further setback on 18 July after  the US Food and Drug Administration was reportedly poised to stop all shipments of the therapy. This followed the deaths of three patients with the disease. Two of the deaths were reported early in the year and involved young people. The third, disclosed on 18 July, involved a 51-year-old man, according to the Bloomberg news service.

Two Norwegian oncology companies announced a merger on 30 June that will generate cash for drug development and advance a joint portfolio of radiopharmaceuticals. The companies are BerGenBio ASA and Oncoinvent ASA. The merger follows BerGenBio’s decision to discontinue its lead cancer product, bemcentinib, and join forces with Oncoinvent to continue Norway’s innovation in radiopharmaceuticals.

US-based Inovio Pharmaceuticals Inc has strengthened its financial position with a public share offering that is expected to raise $25 million. Announced on 3 July, the proceeds will advance an early clinical pipeline of medicines directed against human papillomavirus-related diseases, cancer, and infectious diseases.

Cosentyx (secukinumab), one of Novartis’ top-selling drugs, has failed to show efficacy in a Phase 3 trial of giant cell arteritis, an autoimmune disease affecting the blood vessels. The trial results were announced on 3 July and represent an unusual setback for a drug that has already been approved for seven immune-mediated inflammatory conditions. Cosentyx inhibits interleukin-17A and was first approved in the US in 2015 for plaque psoriasis. The name of the recent trial was GCAptAIN.

DeepMind Technologies Ltd, also known as Google DeepMind, has unveiled a new artificial intelligence tool that has demonstrated an ability to predict how mutations in human DNA sequences can impact the processes that regulate genes. The tool was described for the first time in the 25 June edition of Nature. Google DeepMind is also the developer of AlphaFold, the AI system for predicting the three dimensional structures of proteins.

Novartis has entered into a four-year partnership with ProFound Therapeutics Inc of the US to explore the vast domain of proteins contained in the human proteome in order to find new targets for cardiovascular medicines. Announced on 26 June, the collaboration will entail an upfront payment of $25 million with potential near-term milestone payments of $750 million for each target selected. ProFound was founded in 2020 by Flagship Pioneering, a Cambridge, US-based venture capital group.

A consortium led by the Danish biotech company, Adaptvac ApS, has received $12.4 million from CEPI (the Coalition for Epidemic Preparedness Innovations) and the EU to design and test a candidate vaccine that could offer broad protection against several filoviruses. Filoviruses are a family of viruses known to cause severe haemorrhagic fever in humans. The vaccine would be used to protect high risk populations, such as healthcare workers, in areas where filovirus outbreaks are most prevalent. These are primarily in Central and East Africa.

A destructive cellular process long dismissed as the end of the biological road may in fact be central to the onset of human ageing and chronic disease, according to a new paper published in Nature Oncogene. The paper - co-authored by a global consortium of clinicians and scientists, including teams from LinkGevity, Mayo Clinic, Mass General Brigham, the European Space Agency, and University College London - argues that necrosis, a chaotic form of cell death, plays an active and targetable role in age-related degeneration. 

Thermosome GmbH, a clinical-stage German drug developer, reported positive early data on 24 June from a thermosensitive, liposomal version of a commonly used chemotherapy drug that produced meaningful clinical activity in pre-treated patients with soft tissue sarcomas. The median progression-free survival for patients after treatment reached 4.5 months across two dose levels, a result that exceeded typical survival figures for patients receiving first-line chemotherapy. 

A new clinical-stage company with plans to develop therapies for neuropsychiatric disorders was launched in the UK on 18 June on the back of $140 million in Series A financing led by Access Biotechnology, the investment arm of Access Industries. The company, Draig Therapeutics Ltd, is an academic spin-out which capitalises on scientific discoveries made by two professors at Cardiff University in Wales.