Shifa Biomedical Corporation (Shifa) is a drug discovery company in Malvern Pennsylvania. It is a privately held biopharmaceutical company dedicated to the cost-effective development of drugs for the treatment of cardiovascular disease. Incorporated in 2006, Shifa has recognized expertise for the discovery of small molecule drugs, including pharmaceutical experience guided by biochemical, cell-based, and virtual screening. Shifa scientists exploit molecular biology, biochemistry, crystallography and medicinal, computational and combinatorial chemistry. Shifa is led by a highly-experienced team of scientists with extensive expertise in early stage drug discovery.
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SHIFA’S CANDIDATE TO TREAT CHOLESTEROL IMBALANCE
A Novel, Oral Small Molecule that Significantly Lowers “Bad” Cholesterol
Shifa’s most advanced compound for addressing cardiovascular disease is for the treatment of dyslipidemia, which refers to the presence of an abnormal amount of lipids, cholesterol and fat, in the blood. Such lipid disorders are the major contributor of premature morbidity and mortality from heart disease. Intervention with drugs to reduce “bad” cholesterol (also known as low density lipoprotein-cholesterol; LDL-C) has proven to decrease the risk of subsequent cardiovascular events, including mortality. Based on the wealth of clinical data, it is widely agreed that virtually all patients with coronary artery disease should be treated with lipid lowering drugs to reduce the risk of subsequent events.
UNMET NEED
Although there have been considerable advancements in developing therapies to lower “bad” cholesterol in the past 25 years, a significant number of patients are either unresponsive or experience concerning side effects with current treatment options. To address these concerns, more aggressive and selective targeted therapies are needed.
CONTROLLING LDL RECEPTOR DEGRADATION
One promising avenue for developing specific LDL-C lowering agents is the LDL receptor (LDLR) degradation pathway, which is primarily mediated by the critical regulatory protein known as PCSK9. PCSK9 controls the degradation of the LDLR in the liver and thereby contributes to cholesterol homeostasis.
We have pursued proprietary approaches and identified an orally bioavailable, small molecule lead compound, P-21, that interferes with the function of PCSK9.
TARGET PCSK9
Several strategies have been tried for targeting PCSK9. Most have focused on the development of injectable biologics. The most successful approach is the injection of monoclonal antibodies (mAb) that interfere with the function of PCSK9. A few years ago, Sanofi and Amgen launched mAbs therapy against PCSK9; these are injectable drugs. To date, no orally bioavailable anti-PCSK9 drug has advanced to the clinic. Therefore, Shifa’s approach will result in a first-in-class small molecule, orally bioavailable drug that regulates PCSK9.
LOWER CHOLESTEROL
Shifa’s oral small molecule lead compound significantly lowers LDL-C; it is as potent as the injectable monoclonal antibody.
UNMET NEED
Although there have been considerable advancements in developing therapies to lower “bad” cholesterol in the past 25 years, a significant number of patients are either unresponsive or experience concerning side effects with current treatment options. To address these concerns, more aggressive and selective targeted therapies are needed.
CONTROLLING LDL RECEPTOR DEGRADATION
One promising avenue for developing specific LDL-C lowering agents is the LDL receptor (LDLR) degradation pathway, which is primarily mediated by the critical regulatory protein known as PCSK9. PCSK9 controls the degradation of the LDLR in the liver and thereby contributes to cholesterol homeostasis.
We have pursued proprietary approaches and identified an orally bioavailable, small molecule lead compound, P-21, that interferes with the function of PCSK9.
TARGET PCSK9
Several strategies have been tried for targeting PCSK9. Most have focused on the development of injectable biologics. The most successful approach is the injection of monoclonal antibodies (mAb) that interfere with the function of PCSK9. A few years ago, Sanofi and Amgen launched mAbs therapy against PCSK9; these are injectable drugs. To date, no orally bioavailable anti-PCSK9 drug has advanced to the clinic. Therefore, Shifa’s approach will result in a first-in-class small molecule, orally bioavailable drug that regulates PCSK9.
LOWER CHOLESTEROL
Shifa’s oral small molecule lead compound significantly lowers LDL-C; it is as potent as the injectable monoclonal antibody.