Clinical Research

Efgartigimod, the antibody treatment being developed by argenx SE for a spectrum of autoimmune diseases, leapt over another hurdle on 17 August with news that it had met the primary endpoint in a trial of two rare muscle diseases. The Phase 3 study showed that the antibody fragment, in combination with hyaluronidase enzymes, was able to restore the strength and muscle function in patients with two types of myositis: immune-mediated necrotising myopathy (IMNM) and dermatomyositis (DM). Both diseases cause muscle weakness leading potentially to long-term disability.

Switzerland-based Vaderis Therapeutics AG has raised $152 million from a Series B financing round to support development of a small molecule drug, engasertib, for a rare vascular disease for which no treatments are currently approved. The disease is hereditary haemorrhagic telangiectasia (HHT), a genetic disorder affecting the vasculature, which manifests in nose bleeds, anaemia, and visceral arteriovenous malformations (AVMs) AVMs are tangles of blood vessels that can cause internal bleeding and organ damage.

Netherlands-based AdJane Holding BV has reported the first clinical data for a new vaccine platform that is being positioned as a technology for use in preparing for future pandemics. The early data showed that outer membrane vesicles, which are vesicles released from the outer membranes of Gram-negative bacteria, were safe and well-tolerated in 40 healthy adults. The vaccine, based on outer membrane vesicles, was administered intranasally and combined with a SARS-CoV-2 spike protein- the structural component of the virus that caused the Covid-19 pandemic.

GSK Plc has stopped development of a small molecule drug for chronic cough following only limited efficacy for the product in a Phase 3 programme. However the drug will continue to be evaluated for irritable bowel syndrome. The drug, camlipixant, entered GSK’s portfolio with the acquisition of the Canadian company, BELLUS Health Inc in 2023. At the time, the acquisition was valued at $2 billion. Camlipixant is an antagonist of the P2X3 receptor, a type of receptor primarily found in sensory neurons.

The US Food and Drug Administration issued new draft guidance on 29 May about how companies can reduce the use of animals in drug testing – this time for cancer medicines. Entitled, Oncology Pharmaceuticals: Streamlined Nonclinical Safety Studies for Biologics and Conjugated Products, the guidance recommends new criteria for general toxicology studies, including when animal testing may be unnecessary because there is no binding or pharmacologic activity.

The European Medicines Agency, in coordination with the African Medicines Agency and national regulators in Africa, started talks in early June about organising clinical trials to evaluate possible vaccines and therapies against Ebola disease caused by the Bundibugyo virus. The virus is currently circulating in the Democratic Republic of Congo and Uganda and was declared a public health emergency of international concern by the World Health Organization on 17 May. As of 18 June, the WHO had reported just over 900 cases of the disease and approximately 235 deaths.

uniQure NV has reached an agreement with the US Food and Drug Administration to proceed with a marketing authorisation application for its gene therapy for Huntington’s disease, a rare, inherited neurodegenerative disorder. The application is expected to be filed in the third quarter of this year, the company announced on 17 June.

Ona Therapeutics SL, a 2019 spin-out from a Spanish research institute and foundation, has raised $86.6 million from venture capitalists to advance a portfolio of early-stage antibody-drug conjugates (ADCs) for refractory cancers. The Series B round was co-led by Columbus Venture Partners of Spain and Mérieux Equity Partners of France. They were joined by the existing Spain-based investors Alta Life Sciences and Asabys Partners and Bpifrance, the French public investment bank.

Collaborative networks have been a driving force for innovation across the life sciences for decades. They connect universities, governments and venture capitalists to create conditions for the launch of new companies. The ecosystem around the Massachusetts Institute of Technology in Cambridge, US, is probably the best-known incubator of new companies.  Less common are ecosystems focused on a single therapeutic area. This is the unique feature of a new cluster in France called The Hive and Campus Grand Parc, located in outer Paris.

US-based POP Biotechnologies Inc has announced that the Coalition for Epidemic Preparedness Innovations (CEPI) is to provide up to $9.7 million in additional funding to help advance the company’s SNAP (Spontaneous Nanoliposome Antigen Particle) protein vaccine platform into a Phase 1 clinical trial. The deal builds on an initial $1.5 million investment from CEPI announced in July 2025, which supported early-stage research into the technology.