CAMBRIDGE, MA, USA I June 24, 2017 I Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement mediated diseases, today announced the presentation of scientific data at the 22nd Congress of the European Hematology Association (EHA), June 22-25, 2017 in Madrid, Spain.
“The data presented at EHA showcase a novel class of orally bioavailable small molecules that bind to C5 with high affinity and inhibit its cleavage into C5a and C5b,” said Alonso Ricardo, Senior Vice President and Head of Research at Ra Pharma. “These findings demonstrate the feasibility of an orally-administered therapy for complement-mediated disorders and support the continued advancement of these molecules.”
Inhibition of C5 is a clinically-validated approach for the therapeutic treatment of complement-mediated disorders. Ra Pharma’s portfolio includes drug candidates for several such disorders, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and lupus nephritis (LN). The Company is pursuing the identification of orally-available inhibitors of C5 with the goal of providing additional options for patients diagnosed with these and other complement-mediated disorders.
“We are encouraged by our scientists’ ability to discover and characterize a small molecule inhibitor of complement C5,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “These data show that C5 inhibition can be accomplished with an orally-bioavailable small molecule, and we hope that compounds from this class can be advanced to provide a new treatment option for a broader spectrum of patients.”
The presentation is available at http://rapharma.com/science/presentations-and-publications/. A summary of the data presented by Ra Pharma at the conference is as follows:
Title: Discovery of Orally Bioavailable Small Molecules for Inhibition of Complement C5
Session Title: Bone marrow failure and PNH (oral presentation)
Presenter: Alonso Ricardo, PhD, Senior Vice President and Head of Research, Ra Pharma
Date/Time: Saturday, June 24, 5:00- 5:15 PM CEST
Location: Room N104
Abstract Code: S500
Summary: These initial studies describe a series of first-in-class, orally bioavailable small molecules that bind to C5, inhibiting the protein’s cleavage into C5a and C5b. These molecules possess favorable pharmacokinetic properties, exhibiting up to 50% oral availability in preclinical species. Molecules from this class were found to bind to a unique site on complement C5 with a 1:1 stoichiometry. Notably, binding to this site also inhibited the cleavage of C5 containing the R885H polymorphism, a mutation that confers resistance to eculizumab, today’s standard of care in PNH. The extensive in vitro and in vivo data, along with a high-resolution crystal structure, support the feasibility of developing alternatives to injectable treatment of disorders of complement regulation.
About Ra Pharmaceuticals
Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com.
SOURCE: Ra Pharmaceuticals
Post Views: 103
CAMBRIDGE, MA, USA I June 24, 2017 I Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement mediated diseases, today announced the presentation of scientific data at the 22nd Congress of the European Hematology Association (EHA), June 22-25, 2017 in Madrid, Spain.
“The data presented at EHA showcase a novel class of orally bioavailable small molecules that bind to C5 with high affinity and inhibit its cleavage into C5a and C5b,” said Alonso Ricardo, Senior Vice President and Head of Research at Ra Pharma. “These findings demonstrate the feasibility of an orally-administered therapy for complement-mediated disorders and support the continued advancement of these molecules.”
Inhibition of C5 is a clinically-validated approach for the therapeutic treatment of complement-mediated disorders. Ra Pharma’s portfolio includes drug candidates for several such disorders, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and lupus nephritis (LN). The Company is pursuing the identification of orally-available inhibitors of C5 with the goal of providing additional options for patients diagnosed with these and other complement-mediated disorders.
“We are encouraged by our scientists’ ability to discover and characterize a small molecule inhibitor of complement C5,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “These data show that C5 inhibition can be accomplished with an orally-bioavailable small molecule, and we hope that compounds from this class can be advanced to provide a new treatment option for a broader spectrum of patients.”
The presentation is available at http://rapharma.com/science/presentations-and-publications/. A summary of the data presented by Ra Pharma at the conference is as follows:
Title: Discovery of Orally Bioavailable Small Molecules for Inhibition of Complement C5
Session Title: Bone marrow failure and PNH (oral presentation)
Presenter: Alonso Ricardo, PhD, Senior Vice President and Head of Research, Ra Pharma
Date/Time: Saturday, June 24, 5:00- 5:15 PM CEST
Location: Room N104
Abstract Code: S500
Summary: These initial studies describe a series of first-in-class, orally bioavailable small molecules that bind to C5, inhibiting the protein’s cleavage into C5a and C5b. These molecules possess favorable pharmacokinetic properties, exhibiting up to 50% oral availability in preclinical species. Molecules from this class were found to bind to a unique site on complement C5 with a 1:1 stoichiometry. Notably, binding to this site also inhibited the cleavage of C5 containing the R885H polymorphism, a mutation that confers resistance to eculizumab, today’s standard of care in PNH. The extensive in vitro and in vivo data, along with a high-resolution crystal structure, support the feasibility of developing alternatives to injectable treatment of disorders of complement regulation.
About Ra Pharmaceuticals
Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com.
SOURCE: Ra Pharmaceuticals
Post Views: 103
