– 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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