Real-world data from the University of Catania, Italy, suggests that Pepaxti, a treatment for relapsed, refractory multiple myeloma (RRMM), is effective and well tolerated in heavily pretreated patients.

The retrospective analysis included eight triple-class refractory RRMM patients who had received a median of 3.5 prior lines of therapy. Some had high-risk features, including extramedullary disease, yet Pepaxti, combined with dexamethasone, achieved an overall response rate of 37.5%. Partial responses were observed even in patients with advanced disease.

Dr Etta Conticello, from the Division of Haematology at Azienda Policlinico-S. Marco, University of Catania, noted: “Our experience adds to the growing body of real-world evidence on melflufen in relapsed and refractory myeloma. Even in a high-risk patient cohort, we observed encouraging disease control with a manageable safety profile.”

The findings align with results from the HORIZON trial, further supporting Pepaxti’s role in treating patients with limited therapeutic options.

Oncopeptides’ chief medical officer, Stefan Norin, commented: “These real-world findings provide further support for Pepaxti’s potential benefit in heavily pretreated patients, including those with aggressive disease phenotypes.”

The treatment is approved in Europe for RRMM patients who have received at least three prior lines of therapy and whose disease has progressed on or after their last treatment.

Bruno Bolognese, head of European medical affairs at Oncopeptides, said: “It is truly encouraging to see how real-world clinical experience continues to confirm the potential of Pepaxti in treating patients with advanced multiple myeloma. Close collaboration with the medical community is essential as we work to address the significant unmet needs of patients with limited treatment options.”

The US Food and Drug Administration (FDA) has approved Click Therapeutics’ CT-132 as the first prescription digital therapeutic for the prevention of episodic migraine.

CT-132, which can now be prescribed for patients aged 18 years and older, is intended for adjunctive use alongside acute and/or other preventive treatments.

More than 37 million adults in the US are affected by migraine, a condition characterised by episodes of moderate-to-severe headache, nausea, and increased sensitivity to light and sound.

Despite the availability of preventive migraine medications, many patients continue to experience frequent and debilitating attacks, which can negatively impact key aspects of life such as employment, education and relationships.

CT-132 is built on Click’s artificial intelligence-enabled platform and “combines scientifically proven therapies with proprietary mechanisms of action to deliver clinically meaningful interventions” for patients with episodic migraine, the company outlined.

The FDA’s decision was based on results from the pivotal ReMMi-D trial, in which CT-132 on top of background acute and preventive migraine medications demonstrated significant reductions in monthly migraine days compared to a sham digital control.

Data from the ReMMiD-C bridging study, which showed that CT-132 performed similarly in patients being treated with calcitonin gene-related peptide inhibitors, also supported the approval.

Click’s Chief Medical and Scientific Officer, Shaheen Lakhan, said: “As a groundbreaking digital therapeutic for migraine prevention, CT-132 offers eligible patients a new path to reducing the burden caused by migraine, one they can access anywhere via an evidence-based mobile application on their smartphone, significantly improving accessibility and expanding care to patients.”