News

Capstan Therapeutics Inc, a US biotech company with proprietary lipid nanoparticle technology, closed an oversubscribed Series B financing round on 20 March to advance an in vivo cell therapy product for autoimmune disorders. The round was led by RA Capital Management with participation from five new investors including Forbion, Johnson & Johnson Innovation, and Sofinnova Investments. The proceeds will be used to advance CPTX2309, an in vivo engineered chimeric antigen receptor (CAR) T cell therapy and to further develop the company’s pipeline.

The US Food and Drug Administration approved a new therapy for a rare genetic disease on 18 March, indicating a widening of the scope of regulatory authorisations where there is a serious unmet medical need. The product, Lenmeldy (atidarsagene autotemcel), is a gene therapy intended as a one-time treatment for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare genetic disease that affects the brain and nervous system.

A drug for the treatment of patients with Duchenne muscular dystrophy (DMA) was approved by the US Food and Drug Administration on 22 March. Duvyzat (givinostat) is the first nonsteroidal drug for patients with all genetic variants of the disease. DMD is a rare neurological disorder which causes progressive muscle weakness due to a lack of dystrophin, a muscle protein. Over time, individuals with the disease experience muscle weakness that can lead to problems with walking, breathing and an early death.

The US Food and Drug Administration has approved Rezdiffra (resmetirom), a small molecule agonist of the thyroid hormone receptor beta (THR-beta), to treat non-alcoholic steatohepatitis (NASH) in adults with moderate to advanced liver scarring. The developer is Madrigal Pharmaceuticals Inc. The approval, announced on 14 March, is the first for a product that directly addresses liver scarring and is to be used alongside diet and exercise.

Privately-owned Tubulis GmbH has raised €128 million in an upsized venture round in order to advance its lead antibody-drug conjugate (ADC) products into the clinic. Announced on 14 March, the Series B2 round was co-led by the private equity group EQT Life Sciences and Nextech Invest Ltd of Switzerland. Several US investors participated including Frazier Life Sciences and Deep Track Capital. The financing follows a Series B round of €60 million in 2022.

The obesity drug, Wegovy (semaglutide), first approved in 2021 for weight control, has now been authorised in the US to reduce the risk of heart attack and stroke in adults with cardiovascular disease and either obesity or excessive weight. It is the first drug to receive Food and Drug Administration approval for both indications as evidence of the linkage between excessive weight and cardiovascular events becomes clearer.

A confirmatory Phase 3 trial of a drug for patients with amyotrophic lateral sclerosis (ALS) failed to meet its primary and secondary endpoints, raising the possibility that the therapy, originally approved in 2022, could be withdrawn from the market. The developer, Amylyx Pharmaceuticals Inc, announced the trial results on 8 March, saying that a voluntary market withdrawal had not been ruled out. The therapy, AMX0035, is a combination of two small molecules, sodium phenylbutyrate and taurursodiol, which each target different neurodegenerative pathways.

NodThera Ltd said that its small molecule inhibitor of the NLRP3 inflammasome has shown evidence of reducing neuroinflammatory and inflammatory biomarkers in Parkinson’s disease. Data from a Phase 1b/2a a trial, disclosed on 7 March, showed that the drug, NT-0796, delivered mean reductions of these markers in the cerebrospinal fluid of elderly patients, bringing them to levels approximating those of healthy elderly controls.

The US Food and Drug Administration has approved two biosimilar drugs for the treatment of bone-related disorders – the first follow-on products for medicines developed by Amgen Inc. The new biosimilars, Wyost and Jubbonti, have been produced by Sandoz Group AG, a recent spin-out of Novartis. The active ingredient in both medicines, denosumab, is a human monoclonal antibody that binds to the RANKL protein, an activator of osteoclasts. Osteoclasts are cells that are involved in breaking down bone tissue.

Novo Nordisk A/S’s semaglutide (Ozempic), currently approved to reduce the risk of adverse cardiovascular events in adults with type 2 diabetes and cardiovascular disease, has achieved another milestone. This was evidence that the drug reduced the risk of kidney disease-related events by 24% in people with type 2 diabetes and chronic kidney disease. The data comes from the Phase 3 FLOW trial, which was stopped early in October 2023 due to efficacy. The headline results from this trial were released on 5 March.