SuppreMol GmbH today announced that the European Commission has granted orphan medicinal product designation for Suppre-Mol‘s lead product SM101, a recombinant human soluble Fc-gamma receptor IIb, for the treatment of idiopathic thrombocytopenic purpurea (ITP). SM101 is in preclinical development and scheduled to enter Phase I clinical studies in the second half of 2008.
„The orphan drug designation is an important recognition of our approach, offering increased opportunity to engage the EMEA in discussions concerning its further clinical development,“said Peter Buckel, CEO of SuppreMol.
The EMEA's orphan drug program is designed to promote the development of drugs to treat rare and life-threatening or very serious conditions (prevalence: < 5 in 10,000 people in the European Union). The designation provides EU market exclusivity for up to ten years in the given indication. Other potential benefits include a reduction in fees associated with various aspects of the regulatory process, including the application for marketing approval as well as EMEA guidance in preparing protocols concerning studies relevant for approval.
Notes to Editors
About SuppreMol
SuppreMol is a privately held biopharmaceutical company developing novel therapeutics for the treatment of autoimmune diseases. The Company is pioneering the development of soluble Fcã-Receptors (sFcRs), which are recombinant autologous therapeutic proteins with a proven strong immunosuppressive potential. The Company plans to develop sFcRs for the treatment of idiopathic thrombocytopenic purpurea (ITP), systemic lupus erythematodes, rheumatoid arthritis and other autoimmune conditions.SuppreMol was founded in 2002 as a spin-off from the laboratory of Prof. Dr Robert Huber, Nobel Prize for Chemistry in 1988, at the Max Planck Institute for Biochemistry in Martinsried, Germany. The Company has raised EUR 4 million in a Series A round in May 2006 and received a EUR 1,75 million grant from the BMBF in March 2007.
About sFcRs
Fc receptors (FcRs) control the interactions of antibodies with effector cells, an important mechanism linking cellular and humoral immunity. As an example, members of the FcR family modulate the activation of immune cells by immune complexes and/or antigens and are involved in generating both protective immune responses and pathological inflammatory reactions. Soluble FcRs (sFcRs) are recombinant soluble versions of the Fc receptors that compete with the cellular Fc receptor. As a result they prevent the binding of immune complexes to immune cells. Using this approach, multiple types of cellular receptors can be barred from being activated.
About idiopathic thrombocytopenic purpurea (ITP)
Idiopathic Thrombocytopenic Purpura (ITP) is a bleeding condition characterized by defective blood clotting due to a decreased number of platelet cells. ITP has an incidence of about 30,000 adult patients per year. While children developing ITP often experience complete spontaneous remission, adults often develop chronic ITP, a disease characterized by persistent moderate to severe thrombocytopenia that puts them at risk for bleeding with trauma and can also result in spontaneous hemorrhage of variable severity. Standard treatment options include steroids, but about 70% of patients do not adequately respond to steroids and are candidates for splenectomy. However, about half of splenectomized patients continue to have thrombocytopenia. For those refractory patients, treatment options usually include immunosuppression, but clinical experience is limited and no comparative trials are available so far.
„The orphan drug designation is an important recognition of our approach, offering increased opportunity to engage the EMEA in discussions concerning its further clinical development,“said Peter Buckel, CEO of SuppreMol.
The EMEA's orphan drug program is designed to promote the development of drugs to treat rare and life-threatening or very serious conditions (prevalence: < 5 in 10,000 people in the European Union). The designation provides EU market exclusivity for up to ten years in the given indication. Other potential benefits include a reduction in fees associated with various aspects of the regulatory process, including the application for marketing approval as well as EMEA guidance in preparing protocols concerning studies relevant for approval.
Notes to Editors
About SuppreMol
SuppreMol is a privately held biopharmaceutical company developing novel therapeutics for the treatment of autoimmune diseases. The Company is pioneering the development of soluble Fcã-Receptors (sFcRs), which are recombinant autologous therapeutic proteins with a proven strong immunosuppressive potential. The Company plans to develop sFcRs for the treatment of idiopathic thrombocytopenic purpurea (ITP), systemic lupus erythematodes, rheumatoid arthritis and other autoimmune conditions.SuppreMol was founded in 2002 as a spin-off from the laboratory of Prof. Dr Robert Huber, Nobel Prize for Chemistry in 1988, at the Max Planck Institute for Biochemistry in Martinsried, Germany. The Company has raised EUR 4 million in a Series A round in May 2006 and received a EUR 1,75 million grant from the BMBF in March 2007.
About sFcRs
Fc receptors (FcRs) control the interactions of antibodies with effector cells, an important mechanism linking cellular and humoral immunity. As an example, members of the FcR family modulate the activation of immune cells by immune complexes and/or antigens and are involved in generating both protective immune responses and pathological inflammatory reactions. Soluble FcRs (sFcRs) are recombinant soluble versions of the Fc receptors that compete with the cellular Fc receptor. As a result they prevent the binding of immune complexes to immune cells. Using this approach, multiple types of cellular receptors can be barred from being activated.
About idiopathic thrombocytopenic purpurea (ITP)
Idiopathic Thrombocytopenic Purpura (ITP) is a bleeding condition characterized by defective blood clotting due to a decreased number of platelet cells. ITP has an incidence of about 30,000 adult patients per year. While children developing ITP often experience complete spontaneous remission, adults often develop chronic ITP, a disease characterized by persistent moderate to severe thrombocytopenia that puts them at risk for bleeding with trauma and can also result in spontaneous hemorrhage of variable severity. Standard treatment options include steroids, but about 70% of patients do not adequately respond to steroids and are candidates for splenectomy. However, about half of splenectomized patients continue to have thrombocytopenia. For those refractory patients, treatment options usually include immunosuppression, but clinical experience is limited and no comparative trials are available so far.
