Joana Carvalho, PhD,  managing science editor—

Sevuparin, Modus Therapeutics' lead therapeutic candidate for managing painful vaso-occlusive crises (VOCs), failed to show clinically meaningful improvements in patients with sickle cell disease (SCD), Phase 2 clinical trial results show. Modus Therapeutics is part of Karolinska Development’s portfolio of pharmaceutical companies. Karolinska Development is a Sweden-based investment company focused on identifying and supporting breakthrough medical innovations developed by other companies. Sevuparin is an investigational drug with anti-adhesive and anti-inflammatory properties that has been developed to prevent the obstruction of blood vessels and restore normal blood flow, reducing the risks of VOCs, one of the most common and painful complications of SCD. The multicenter, randomized, double-blind, placebo-controlled, Phase 2 trial (NCT02515838) was designed to assess the safety and effectiveness of continuous intravenous infusion of sevuparin for the management of painful VOCs in SCD patients over the course of two to seven days, compared to a placebo. The trial's primary outcome was to assess the time of VOCs resolution after administering sevuparin. Secondary outcomes included treatment safety assessments, drug pharmacokinetic properties, mean change in pain intensity, duration of the most severe pain, and cumulative dose of opioids. (Pharmacokinetics is the study of how a drug is absorbed, distributed, metabolized, and eliminated from the body.) The study enrolled 144 SCD patients from multiple sites across Europe, the Middle East and the Caribbean. Unfortunately, according to statements from Karolinska Development, findings from the trial failed to show clinical benefits associated with the use of sevuparin. "It is disappointing that sevuparin did not demonstrate a meaningful clinical benefit in the Phase 2 study conducted by Modus Therapeutics, particularly as patients with sickle cell disease have no treatment options that can reduce the length of their vaso-occlusive crises or manage their pain, other than opioids. Modus will now consider options for further development of sevuparin in other indications where its multimodal action may have utility," Viktor Drvota, CEO of Karolinska Development, said in a press release. Karolinska said in it announcement that it will return with updated information as soon as Modus Therapeutics makes a final decision regarding the future direction of its development project involving sevuparin.

A study of sickle cell disease (SCD) at the molecular level found abnormal hemoglobin molecules assemble much faster and less efficiently than previously thought — findings that could lead to the development of new treatments. The researchers said better understanding of the assembly process is the first step toward…

Abnormal sickle-shaped red blood cells characteristic of sickle cell disease (SCD) change the structure and dynamics of blood clots’ formations in veins, a study in mice and human blood shows. Such changes may explain why SCD patients are at higher risk for venous thromboembolisms. The study with those findings, “…

Alterations in the lungs’ network of blood vessels, caused by sickle cell disease (SCD), reflect disease burden and should be carefully monitored over time, a recent study says. The study with that finding, “Clinical relevance of pulmonary vasculature involvement in sickle cell disease,” was published in the…

Oral morphine treatment is a valid therapy option for patients with sickle cell disease (SCD) who are undergoing a vaso-occlusive crisis (VOC), researchers suggest. It was found to significantly reduce the rate of hospitalizations and the number of intravenous treatments patients must undergo to control these crisis. The…

Bariatric surgery  may help reduce the rates of vaso-occlusive crises (VOCs) in morbidly obese patients with sickle cell disease (SCD) without increasing the risk of mortality or other adverse events, a study says. The study, “Impact of Bariatric Surgery on Outcomes of Patients with Sickle Cell Disease: a…

Brain oxygen supply is different in different regions of the brain of sickle cell disease (SCD) patients who are affected by anemia, which may explain the incidence of silent strokes, a recent study says. The findings of the study, “White matter has impaired resting oxygen delivery in sickle…

The American Thoracic Society (ATS) released a set of new guidelines for home oxygen therapy for children with chronic health conditions that cause a reduction in blood oxygen levels, such as sickle cell disease. Other conditions include cystic fibrosis, bronchopulmonary dysplasia, sleep disturbed breathing, pulmonary…

Crizanlizumab (SEG101), Novartis‘ investigational compound for sickle cell disease (SCD), received breakthrough therapy designation from the U.S. Food and Drug Administration for its potential to prevent vaso-occlusive crises (VOCs). The FDA designation was granted based on promising data from the company’s multicenter, randomized, double-blind, 52-week, Phase…

Sancilio Pharmaceuticals‘ investigational oral compound Altemia (SC411) shows promising results in treating children with sickle cell disease (SCD), a Phase 2 clinical trial reveals. The study, “Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial),” was published in…