Initial Data from Global Phase 2 Program Expected Mid-2017
CAMBRIDGE, MA, USA I April 26, 2017 I Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it has initiated dosing in the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel and potent subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Inhibition of C5 is a clinically validated approach for the control of complement-induced hemolysis, the process responsible for the rupture and destruction of red blood cells (RBCs) in patients with PNH.
“Commencement of dosing in our Phase 2 clinical program for RA101495 in PNH is an important step in bringing a potential new therapy to patients with this rare and life-threatening blood disorder,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Building on our promising Phase 1 data, our Phase 2 program is designed to demonstrate RA101495’s safety and preliminary efficacy in PNH patients. RA101495 is a convenient, once daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH. We look forward to sharing initial data from our Phase 2 program in mid-2017 and additional data by year-end.”
The global Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The program is comprised of two open-label Phase 2 studies, one conducted outside the U.S. and one conducted in the U.S.
- Outside of the U.S., the study will evaluate RA101495 in two cohorts. Cohort A is expected to include 8-12 eculizumab-naïve patients and Cohort B is expected to include 6-8 patients switching from eculizumab to RA101495.
- The U.S. study will evaluate RA101495 in approximately 6-8 patients who are currently treated with eculizumab but have evidence of an inadequate response. An inadequate response is defined as a lactate dehydrogenase (LDH) level equal to or greater than 1.5-fold the upper limit of normal, indicating ongoing hemolysis. LDH is a measure of red blood cell hemolysis and a key disease biomarker.
All patients enrolled in the Phase 2 studies will receive a single, 0.3 mg/kg SC loading dose of RA101495, followed by 0.1 mg/kg SC daily thereafter. Following two weeks of treatment and based on a review of safety and efficacy data, patients will continue with 0.1 mg/kg daily or, if needed, the dose can be increased to 0.3 mg/kg daily to achieve adequate control of hemolysis. Patients in all three cohorts will be eligible for a long-term extension study following the completion of the initial 12-week studies. The primary efficacy endpoint is change in LDH from baseline.
About RA101495
Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, once daily SC self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement C5 with sub-nanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. By binding to a region of C5 corresponding to C5b, RA101495 also disrupts the interaction between C5b and C6 and prevents assembly of the membrane attack complex (MAC). This activity defines an additional, novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.
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 Pharmaceuticsls
Post Views: 34
Initial Data from Global Phase 2 Program Expected Mid-2017
CAMBRIDGE, MA, USA I April 26, 2017 I Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it has initiated dosing in the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel and potent subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Inhibition of C5 is a clinically validated approach for the control of complement-induced hemolysis, the process responsible for the rupture and destruction of red blood cells (RBCs) in patients with PNH.
“Commencement of dosing in our Phase 2 clinical program for RA101495 in PNH is an important step in bringing a potential new therapy to patients with this rare and life-threatening blood disorder,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Building on our promising Phase 1 data, our Phase 2 program is designed to demonstrate RA101495’s safety and preliminary efficacy in PNH patients. RA101495 is a convenient, once daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH. We look forward to sharing initial data from our Phase 2 program in mid-2017 and additional data by year-end.”
The global Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The program is comprised of two open-label Phase 2 studies, one conducted outside the U.S. and one conducted in the U.S.
- Outside of the U.S., the study will evaluate RA101495 in two cohorts. Cohort A is expected to include 8-12 eculizumab-naïve patients and Cohort B is expected to include 6-8 patients switching from eculizumab to RA101495.
- The U.S. study will evaluate RA101495 in approximately 6-8 patients who are currently treated with eculizumab but have evidence of an inadequate response. An inadequate response is defined as a lactate dehydrogenase (LDH) level equal to or greater than 1.5-fold the upper limit of normal, indicating ongoing hemolysis. LDH is a measure of red blood cell hemolysis and a key disease biomarker.
All patients enrolled in the Phase 2 studies will receive a single, 0.3 mg/kg SC loading dose of RA101495, followed by 0.1 mg/kg SC daily thereafter. Following two weeks of treatment and based on a review of safety and efficacy data, patients will continue with 0.1 mg/kg daily or, if needed, the dose can be increased to 0.3 mg/kg daily to achieve adequate control of hemolysis. Patients in all three cohorts will be eligible for a long-term extension study following the completion of the initial 12-week studies. The primary efficacy endpoint is change in LDH from baseline.
About RA101495
Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, once daily SC self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement C5 with sub-nanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. By binding to a region of C5 corresponding to C5b, RA101495 also disrupts the interaction between C5b and C6 and prevents assembly of the membrane attack complex (MAC). This activity defines an additional, novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.
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 Pharmaceuticsls
Post Views: 34
