News

France-based ophthalmology company Nicox SA announced an extension of its cash runway on 2 September ahead of regulatory reviews in both the US and China for its lead product NCX 470 (bimatoprost grenod) for two ocular conditions. As of 31 August, the company’s estimated cash and cash equivalents was €8.4 million compared with €4.1 million on 31 December 2025. The company said the cash position will carry it through the regulatory decision period which, if successful, would generate new revenue.

Patients with polycythaemia vera, a rare blood disorder that causes the body to make too many red blood cells, will be eligible for treatment with a new peptide therapeutic approved by the US Food and Drug Administration on 28 August. Mimrylo (rusfertide) is a hepcidin mimetic that works by mimicking the action of hepcidin, a hormone that regulates the body’s use of iron. Too little iron can lead to iron-deficiency anaemia while too much can be toxic.

The US Food and Drug Administration approved a new treatment for dermatomyositis on 27 August – the first oral drug for a disease that can cause progressive damage to the muscles, skin and lungs and an impaired quality of life. Existing treatments include combinations of chronic steroids and antirheumatic drugs which have not been effective in controlling the disorder. The new treatment, Lisraya (brepocitinib), works as a Janus kinase inhibitor, blocking pathways that play a key role in the body’s immune responses.

A new small molecule drug has won approval from the US Food and Drug Administration to treat patients with metastatic pancreatic ductal adenocarcinoma – an aggressive cancer with a poor prognosis for survival. The drug, Rasonque (daraxonrasib), has been authorised for adults who have received at least one prior systemic therapy for their cancer, or who are not candidates for multiagent systemic therapy.

US-based ReCode Therapeutics Inc has strengthened its ties with the Cystic Fibrosis Foundation as it progresses an experimental messenger RNA (mRNA) therapy for the disease through the clinic. Simultaneously, it promoted Heather Clark on 1 July to the role of chief executive from vice president and head of the company’s cystic fibrosis franchise. Ms Clark joined ReCode in 2022. She has nearly 30 years of experience in rare disease drug development of which more than half were spent at Vertex Pharmaceuticals Inc, developer of five marketed cystic fibrosis products.

The US Food and Drug Administration granted an accelerated approval on 19 August for a new gene therapy for patients with an inherited disorder caused by the shortage of an enzyme needed to maintain stable blood sugar levels in the body. The therapy, Genglycos (pariglasgene brecaparvovec), is the first treatment to be authorised for the disorder – glycogen storage disease type la. Also called von Gierke disease, the disorder results in the  build-up of glycogen, a complex sugar, in the body’s cells.

A messenger RNA (mRNA)-based therapy designed for cancer has delivered positive Phase 3 results in patients with resected, advanced myeloma representing a first of its kind treatment in oncology. Announced on 19 August, the trial, INTerpath-001, delivered recurrence-free survival and distant metastasis-free survival for patients who had tumours removed but were still at risk of disease. The trial enrolled 1,137 patients who were randomised to receive intismeran, the mRNA therapy, and Keytruda (pembrolizumab) or Keytruda alone.

AstraZeneca Plc reported two positive outcomes and one negative outcome for three Phase 3 lung cancer trials on 17 August. The trials were all testing treatments for non-small cell lung cancer, the most prevalent type, which makes up about 85% of all lung cancers globally, according to the World Health Organization. The largest trial, which was testing a dual checkpoint inhibitor bispecific antibody, failed to show efficacy. However the two other studies, targeting cancers with specific genetic mutations, achieved statistically significant and clinically meaningful results.

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.