Sevuparin

– potential best-in-class treatment

in Sickle Cell Disease

Sickle cell

Dilaforette’s lead drug candidate, sevuparin, is currently being tested in a multi-center, double-blind, placebo-controlled phase II study, enrolling hospitalized Sickle-Cell Disease (SCD) patients experiencing vaso-occlusive crises (VOC).

SCD is an orphan disease with disabling and potentially fatal outcomes and with a large unmet medical need in both the developed and developing world. In the US and in Europe, it is estimated that close to 100,000 and 35,000 patients, respectively, are diagnosed with this hereditary disease. There is also an even larger patient pool in the Middle East and North Africa (MENA) region. SCD patients undergo on average one VOC per year. This acute complication is caused by sickled blood cells obstructing the blood flow to vital organs leading to ischemia and often severe pain. Long-term, SCD patients are at risk of organ damage and premature death.

Sevuparin is Dilaforette’s innovative, disease-modifying polysaccharide drug, with extensive world-wide IP protection. Dilaforette has received Orphan Drug Designation (ODD) in both US and EU for sevuparin in SCD.

Sevuparin acts like an anti-adhesive, blocking the key adhesion receptors involved in the events leading up to a VOC. Based on its mechanism of action and excellent tolerability, sevuparin has the potential to become the best-in-class treatment of painful crisis in SCD patients.

Both preclinical data and clinical studies support that sevuparin can have rapid and clinically relevant effects on prevention and resolution of the microvascular obstructions. Dilaforette intends to initially develop sevuparin for the acute treatment of Vaso-Occlusive Crises (VOCs) starting with the ongoing phase II study in hospitalized SCD patients, where sevuparin is administrated intravenously. In parallel, a subcutaneous formulation is being developed for early self-treatment in the home setting. Such an early treatment has the potential to decrease the number of patients going into full-blown VOCs and requiring hospital care. Subcutaneous sevuparin could thus be key in normalizing the SCD patients’ daily lifes.

There is a large need for new therapies addressing the cause and not just the symptoms of vaso-occlusion in SCD patients, as there currently is none available. The only treatment approved for SCD patients is hydroxyurea (a.k.a. hydroxycarbamide), indicated for reducing the frequency of VOCs in SCD patients with recurrent VOCs. However, treatment with hydroxyurea has no effect on neither the pain intensity nor the severity of the acute VOC for the patient.

Based on its mechanism of action and excellent tolerability, sevuparin has the potential to become the best-in-class treatment for SCD. Ultimately, it is believed that sevuparin can be self-administered to abort developing VOCs.

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  • News

    • Dilaforette poster presentation at EHA Congress

      2016-06-09 08:00:19 STOCKHOLM – 9 JUNE, 2016. Dilaforette AB, a Karolinska Development (STO:KDEV) portfolio company focused on innovative treatments for patients with Sickle Cell Disease, announces it will have a poster presentation at the European Hematology Association Congress in Copenhagen. Dilaforette’s submission is entitled: "Sevuparin Demonstrates Binding to Key Adhesion Receptors Involved in Pathogenesis of Sickle-Cell Disease" under session for non-malignant hematopoetic disorders. The 21 Congress of the European Hematology Association is to take place on 9-12 June 2016 at The Bella Center, Center Boulevard

    • Dilaforette signs collaboration agreement with Arabian Gulf University (Bahrain) for Phase 2 clinical development of sevuparin for Sickle Cell Disease

      2016-02-16 07:48:23 STOCKHOLM, MANAMA – 16th February, 2016. Dilaforette AB, a Karolinska Development (STO:KDEV) portfolio company focused on innovative treatments for patients with sickle-cell disease, and Arabian Gulf University (AGU), based in Bahrain, today announced that they have signed a Clinical Collaboration agreement for the Phase 2 proof of concept trial of sevuparin in patients with Sickle Cell Disease (SCD) experiencing acute Vaso-Occlusive Crisis (VOC).   Under the scope of the agreement, Dilaforette and AGU’s collaboration will facilitate the development of Dilaforette’s lead candidate,

    • Dilaforette appoints new VP of Development

      2016-02-08 07:52:49 STOCKHOLM – 8th February, 2016. Dilaforette AB, a Karolinska Development (STO:KDEV) portfolio company focused on innovative treatments for patients with sickle-cell disease, announces the appointment of Lena Jendeberg as its new Vice President of Development.   Lena Jendeberg joins Dilaforette from Swedish Orphan Biovitrum AB (Sobi), where she was Global Program Director, Project & Portfolio Management and Strategic Operations from 2010. In this role she was primarily responsible for Life Cycle Management of one of Sobi’s key products Kineret (anakinra) – a