Clinical Research

Artios Pharma Ltd, a UK clinical-stage company with products to kill cancer cells by blocking their ability to repair damaged DNA, has raised $115 million in an oversubscribed Series D financing. The round was co led by founding investor SV Health Investors and new investor RA Capital Management. The funds will be used to advance two small molecule drugs against large cancers. The first, alnodesertib, is an inhibitor of the ATR protein which is involved in DNA repair, and the second is ART6043, an inhibitor of the DNA repair enzyme DNA polymerase theta (Pol theta).

A trial for adults with major depressive disorder failed to significantly reduce the severity of the disorder compared with a placebo, according to the sponsor Neurocrine Biosciences Inc of San Diego, US. The Phase 2 study enrolled 73 adults with a diagnosis of major depressive disorder and an inadequate response to at least one antidepressant in their regular treatment paradigm. The product, NBI-1070770, is a negative allosteric modulator of a subunit of a receptor that plays a role in the working memory. Participants received the drug or a placebo for four weeks.

Germany-based biotech InflaRx NV, has published positive Phase 2a data for a new small molecule drug for the treatment of two skin diseases: hidradenitis suppurativa and chronic spontaneous urticaria. The drug, INF904, is an inhibitor of the C5a receptor which modulates the body’s inflammatory responses. In a basket study of patients with the two diseases, the drug achieved rapid disease control. In the case of hidradenitis suppurativa this involved decreases in measures of nervous system changes.

An antibody-drug conjugate, made with a toxic mushroom rather than chemotherapy, has shown promising results in a Phase 1/2a trial in patients with multiple myeloma. The drug, HDP-101 (pamlectabart tismanitin), is being developed by Heidelberg Pharma AG of Germany. In October it received a ‘fast track’ designation from the US Food and Drug Administration for its potential as a treatment for severely ill and heavily pretreated patients. 

A candidate radio-diagnostic has received ‘fast track’ designation from the US Food and Drug Administration for its protential use in combination with a therapeutic to identify and treat clear cell renal cell carcinoma, the most common type of kidney cancer. ITM-94 is a radiolabeled gallium 68 imaging agent which is in a Phase 1/2 trial in patients with locally advanced or metastatic solid tumours. It is being developed with ITM-91, an investigational radioparmaceutical. The combination is known as a theranostic.

A candidate treatment for malaria developed by Novartis has reached the primary endpoint in a Phase 3 trial, paving the way for regulatory submission. If approved, the drug would be a new approach for tackling resistance to current treatments across Africa and beyond. The drug, KLU156, combines a novel compound, ganaplacide, with a new once-daily formulation of the existing antimalarial lumefantrine. It was developed in collaboration with the Swiss non-profit organisation Medicines for Malaria Venture and received funding from several European governments.

Baxdrostat, a small molecule inhibitor of the hormone, aldosterone, achieved a statistically significant reduction in systolic blood pressure in a Phase 3 trial, confirming its efficacy as a treatment for patients with hard-to-control hypertension. The data were presented on 9 November at the American Heart Association meeting in New Orleans, US. The developer, AstraZeneca Plc, is expected to start regulatory submissions by the end of the year.

A gene therapy intended as a one-time treatment for Huntington’s disease significantly slowed progression of the disorder in a pivotal Phase 1/2 trial, paving the way for a regulatory submission in the first quarter of 2026. The developer, uniQure NV, showed that it is possible to safely impact the disease by targeting both the disease-causing huntingtin protein and the normal protein using a vector-based gene therapy and a gene encoding a microRNA (miRNA).

The European Medicines Agency has issued a positive opinion for a new indication of an antibody therapeutic that targets B cells in order to treat immunoglobulin G4 related diseases. These are autoimmune disorders caused when the body’s own defence system attacks normal tissues. This can lead to fibrosis and inflammation in one or multiple organs of the body. The therapy, Uplizna (inebilizumab), has already been approved for the treatment of neuromyelitis optica spectrum disorder, an inflammatory disorder of the central nervous system.

A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.