NEWARK, CA, USA I April 23, 2015 I CymaBay Therapeutics, Inc. (CBAY), a clinical-stage biopharmaceutical company developing therapies to treat metabolic diseases with high unmet medical need, today announced the initiation of a Phase 2 study of MBX-8025 in patients with homozygous familial hypercholesterolemia (HoFH). MBX-8025 is an orally administered potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist.
HoFH is a rare, life-threatening, autosomal genetic disease characterized by loss-of-function mutations in both alleles of the LDL receptor (LDL-R) gene. The accompanying loss of LDL-R activity results in marked elevations in the plasma levels of LDL cholesterol (LDL-C), causing premature cardiovascular disease that often presents during the first decades of life and which can result in myocardial infarction, ischemic stroke and premature death. The Phase 2 trial is an open label, dose-escalation study that will target enrollment of 8 patients at sites in Europe and North America. Following enrollment, patients will initially receive a 50 mg dose of MBX-8025 once daily that will be increased first to 100 mg and eventually 200 mg of MBX-8025 over the course of 3 months.
“HoFH is a rare and serious disease associated with early morbidity and mortality,” said Harold Van Wart, president and chief executive officer of CymaBay. “Because patients with HoFH do not have fully functional LDL receptors, conventional lipid lowering therapy is minimally effective. Moreover, recently approved therapies for HoFH that reduce LDL-C by mechanisms independent of the LDL-R have significant limitations and the unmet need for this disease remains high. Clinical and preclinical results for MBX-8025 indicate that it offers the potential to significantly lower LDL-C in patients with HoFH while exhibiting markedly improved tolerability over current therapies. We look forward to the outcome of this study around the end of this year.”
In a clinical study conducted in patients with mixed dyslipidemia, MBX-8025 was shown to reduce LDL-C. Data from a preclinical model of human HoFH indicate that MBX-8025 lowers LDL-C in the absence of a fully functional LDL-R. The U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for MBX-8025 for the treatment of HoFH. MBX-8025 has also received orphan designation for Fredrickson types I and V hyperlipoproteinemia.
About MBX-8025
MBX-8025 is a potent and selective agonist of PPARδ, a nuclear receptor important for lipid transport, storage and metabolism in liver and muscle. MBX-8025 has shown favorable effects on lipid and metabolic parameters in a Phase 2 study in patients with mixed dyslipidemia. Treatment effects observed include lowering of LDL-C with selective depletion of pro-atherogenic dense LDL-C particles, decreases in triglycerides and increases in high density lipoprotein, as well as decreases in hsCRP, a biomarker of cardiovascular inflammation. CymaBay has initiated a pilot clinical study evaluating the activity of MBX-8025 in patients with homozygous familial hypercholesterolemia. The U.S. Food and Drug Administration (FDA) has granted CymaBay orphan drug designation for MBX-8025 as a treatment for homozygous familial hypercholesterolemia (HoFH) and Fredrickson types I and V hyperlipoproteinemia.
About CymaBay
CymaBay Therapeutics, Inc. (CBAY) is a clinical-stage biopharmaceutical company developing therapies to treat metabolic diseases with high unmet medical need, including serious rare and orphan disorders. Arhalofenate, the company’s lead product candidate, has shown two therapeutic actions in a single drug in multiple Phase 2 gout studies. In gout patients, arhalofenate is intended to prevent painful flares in joints while at the same time promoting excretion of uric acid by the kidney, thereby addressing both the signs and symptoms of gout and the hyperuricemia that is the root cause of the disease. CymaBay’s second product candidate, MBX-8025 is a potent, selective, orally active PPARδ agonist. A Phase 2 study of MBX-8025 in patients with mixed dyslipidemia established that it has an anti-atherogenic lipid profile. CymaBay has initiated a pilot study of MBX-8025 in patients with homozygous familial hypercholesterolemia.
SOURCE: CymaBay Therapeutics
Post Views: 440
NEWARK, CA, USA I April 23, 2015 I CymaBay Therapeutics, Inc. (CBAY), a clinical-stage biopharmaceutical company developing therapies to treat metabolic diseases with high unmet medical need, today announced the initiation of a Phase 2 study of MBX-8025 in patients with homozygous familial hypercholesterolemia (HoFH). MBX-8025 is an orally administered potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist.
HoFH is a rare, life-threatening, autosomal genetic disease characterized by loss-of-function mutations in both alleles of the LDL receptor (LDL-R) gene. The accompanying loss of LDL-R activity results in marked elevations in the plasma levels of LDL cholesterol (LDL-C), causing premature cardiovascular disease that often presents during the first decades of life and which can result in myocardial infarction, ischemic stroke and premature death. The Phase 2 trial is an open label, dose-escalation study that will target enrollment of 8 patients at sites in Europe and North America. Following enrollment, patients will initially receive a 50 mg dose of MBX-8025 once daily that will be increased first to 100 mg and eventually 200 mg of MBX-8025 over the course of 3 months.
“HoFH is a rare and serious disease associated with early morbidity and mortality,” said Harold Van Wart, president and chief executive officer of CymaBay. “Because patients with HoFH do not have fully functional LDL receptors, conventional lipid lowering therapy is minimally effective. Moreover, recently approved therapies for HoFH that reduce LDL-C by mechanisms independent of the LDL-R have significant limitations and the unmet need for this disease remains high. Clinical and preclinical results for MBX-8025 indicate that it offers the potential to significantly lower LDL-C in patients with HoFH while exhibiting markedly improved tolerability over current therapies. We look forward to the outcome of this study around the end of this year.”
In a clinical study conducted in patients with mixed dyslipidemia, MBX-8025 was shown to reduce LDL-C. Data from a preclinical model of human HoFH indicate that MBX-8025 lowers LDL-C in the absence of a fully functional LDL-R. The U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for MBX-8025 for the treatment of HoFH. MBX-8025 has also received orphan designation for Fredrickson types I and V hyperlipoproteinemia.
About MBX-8025
MBX-8025 is a potent and selective agonist of PPARδ, a nuclear receptor important for lipid transport, storage and metabolism in liver and muscle. MBX-8025 has shown favorable effects on lipid and metabolic parameters in a Phase 2 study in patients with mixed dyslipidemia. Treatment effects observed include lowering of LDL-C with selective depletion of pro-atherogenic dense LDL-C particles, decreases in triglycerides and increases in high density lipoprotein, as well as decreases in hsCRP, a biomarker of cardiovascular inflammation. CymaBay has initiated a pilot clinical study evaluating the activity of MBX-8025 in patients with homozygous familial hypercholesterolemia. The U.S. Food and Drug Administration (FDA) has granted CymaBay orphan drug designation for MBX-8025 as a treatment for homozygous familial hypercholesterolemia (HoFH) and Fredrickson types I and V hyperlipoproteinemia.
About CymaBay
CymaBay Therapeutics, Inc. (CBAY) is a clinical-stage biopharmaceutical company developing therapies to treat metabolic diseases with high unmet medical need, including serious rare and orphan disorders. Arhalofenate, the company’s lead product candidate, has shown two therapeutic actions in a single drug in multiple Phase 2 gout studies. In gout patients, arhalofenate is intended to prevent painful flares in joints while at the same time promoting excretion of uric acid by the kidney, thereby addressing both the signs and symptoms of gout and the hyperuricemia that is the root cause of the disease. CymaBay’s second product candidate, MBX-8025 is a potent, selective, orally active PPARδ agonist. A Phase 2 study of MBX-8025 in patients with mixed dyslipidemia established that it has an anti-atherogenic lipid profile. CymaBay has initiated a pilot study of MBX-8025 in patients with homozygous familial hypercholesterolemia.
SOURCE: CymaBay Therapeutics
Post Views: 440
