A new blood test for Alzheimer’s disease has received a ‘breakthrough device designation’ from the US Food and Drug Administration for its promise as a way of identifying the disease early, and distinguishing it from other neurodegenerative disorders. The device, Elecsys pTau217 is an in vitro immunoassay intended for the quantitative determination of the protein phospho-tau (217P) in human plasma.
The European Medicines Agency has issued a positive opinion for Fabhalta (iptacopan), a monotherapy for a rare genetic blood disorder which has shown superior activity to the standard of care. The disorder is paroxysmal nocturnal haemoglobinuria (PNH) which causes the premature breakdown of red blood cells in the body leading to fatigue, shortness of breath and often requiring blood cell transfusions.
A group of medicines approved to treat type 2 diabetes and obesity, which include semaglutide, have passed a safety review by the European Medicines Agency. The review was triggered by reports linking use of the drugs, which are glucagon-like peptide-1 receptor agonists (GLP-1), to suicidal and self-harm thoughts and behaviour. On 12 April, the agency’s pharmacovigilance risk assessment committee (PRAC) said an investigation could not establish a causal association between the medicines and the health risks.
Genentech Inc and its Swiss parent, Roche, have ended a cell therapy partnership with Adaptimmune Therapeutics Plc which was focused on developing allogeneic T cell therapies for cancer. The reason for the termination, announced on 10 April, wasn’t given. However it comes at a time when other large companies are making staff redundant or divesting early-stage biotech projects. On 11 April, Syncona, the investment group, announced plans to sell a cell therapy portfolio company to Century Therapeutics Inc. This was to prioritise capital allocation on later-stage assets.
Sumagen Co Ltd, a Korean-Canadian company developing an experimental vaccine for HIV-1, is to start manufacturing the product for use in clinical trials under a new partnership with Naobios SAS of France. Naobios is a contract development and manufacturing organisation. Announced on 15 April, the companies did not disclose the financial terms. There are currently no approved vaccines for HIV, a disease that is estimated to affect 39 million people worldwide. The Sumagen product is based on recombinant genetically modified virus technology.
An experimental gene therapy for the rare neurological disorder, Canavan disease, has been given a regenerative medicine advanced therapy (RMAT) designation by the US Food and Drug Administration. RMAT designation is a regulatory incentive awarded by the agency to cell or gene therapy products which show promise for treating or reversing the course of a serious disease.
Two small European companies have announced a collaboration under which they plan to repurpose an approved medicine for chemotherapy induced nausea to be used in patients with lung cancer. The business deal was announced on 9 April and involves PlusVitech of Spain and Nanoform Finland Plc of Finland. It focuses on progressing a small Phase 2 study in patients with non-small cell lung cancer refractory to standard treatment. The current formulation of the drug involves the administration of potentially dozens of capsules per day to patients who are frail and may have dysphagia.
Citing a need to conserve capital, the Syncona investment group, has divested its portfolio company Clade Therapeutics Inc to Century Therapeutics Inc of the US. Syncona is to sell Clade for up to $45 million. This includes an upfront consideration of $35 million in a combination of cash and shares in Century, along with potential milestone payments of $10 million.
Vertex Pharmaceuticals Inc is to expand its portfolio of drugs for genetic diseases with the acquisition of Alpine Immune Sciences Inc, giving the Boston, US-based company a clinical-stage product for IgA nephropathy. The acquisition, announced on 10 April, has been priced at $65 per share for a total deal value of $4.9 billion. The transaction is expected to close before the end of June.
The cardiovascular medicines field, which recently saw Novo Nordisk A/S acquire Cardior Pharmaceuticals of Germany, has also witnessed a new deal in medical technology. On 5 April, Johnson & Johnson Inc announced an agreement to acquire Shockwave Medical Inc, a US company with a catheter-based technology for treating the build-up of calcium deposits in the body. J&J is to pay $335 per share in cash for Shockwave, giving an enterprise value of $13.1 billion.