News

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 novel antibody that has shown it can delay the onset of Type 1 diabetes received a positive opinion from the European Medicines Agency on 14 November. Teizeild (teplizumab) has been developed for the most advanced stage of disease onset when most pancreatic beta cells have been destroyed, insulin production is down, and blood sugar levels are rising. The same product, known as Tzield, was approved by the US Food and Drug Administration in 2022.

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 gene therapy for Wiskott-Aldrich syndrome, whose development was supported by the Italian non-profit organisation, Fondazione Telethon ETS, received a positive opinion from the European Medicines Agency on 14 November. Wiskott-Aldrich syndrome is a rare genetic disorder that keeps a child’s immune system from functionally normally. It mainly affects young boys. The treatment, Waskyra (etuvetidigene autotemcel) is the first gene therapy to be evaluated for this disease.

The US Food and Drug Administration has updated a warning about the safety of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), and restricted its use. This follows reports earlier this year of fatal acute liver failure in certain non-ambulatory, male, paediatric patients taking the drug. Following the incidents, the developer, Sarepta Therapeutics Inc, voluntarily paused distribution of Elevidys to this patient group. This has now been formalised in a notice issued on 14 November.

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.

argenx SE achieved sales of $1.13 billion in the three months ended 30 September, a quarterly record for the company which has successfully exploited the therapeutic potential of a single biologic asset. Announced on 30 October, the quarterly sales were nearly double the $573 million generated a year earlier. They were helped by high demand for a prefilled syringe formulation of the lead product Vyvgart (efgartigimod). Vyvgart binds to the neonatal Fc receptor (FcRn), which is responsible for maintaining the circulation of immunoglobuline G (IgG) in the body.

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.