MyFatDog
Phase II
Phase II Pipeline Drugs
AMG-145 Being developed by Amgen. AMG-145 is an antibody to the protein PCSK9 that is being
developed as a treatment for elevated LDL levels. The drug is currently being tested in patients
with familial hypercholesterolemia, in patients that don't tolerate statins and for its effectiveness
when given in combination with a statin.
CER-001 (Cerenis-001) Being developed by Cerenis Therapeutics. This drug is a synthesic HDL
made from apoA-I mimetic peptide complexed with phospholipid that is designed to increase
reverse cholesterol transport. This drug is also now being considered for use in rare disorders of
lipoprotein metabolism such as homozygous FH in addition to patients with acute coronary
syndrome.
CSL-112 Being developed by CSL Limited. CSL-112 is a reconstituted HDL that is being
developed as a treatment to increase HDL levels and stimulate cholesterol removal from tissues
(reverse cholesterol transport). CSL-111, a compound similar to CSL-112, had progressed to
phase II studies but was apparently discontinued following a study that showed that the drug was
no better than placebo in reducing atherosclerosis. CSL-112 is a different formulation that is
reported to be more potent than CSL-111. A phase II study testing safety and pharmacokinetics
in patients with acute coronary syndrome will begin in January 2012.
DRL-17822 Being developed by Dr. Reddy's Labs. DRL-17822 is a cholesteryl ester transfer
protein inhibitor (CETP inhibitor) that is being developed as a treatment to increase HDL levels.
Results from phase I indicate that the drug is able to increase HDL from 51-111% and reduce LDL
from 25-56%. A phase II study testing the safety and effectiveness of the drug in patients with
high LDL cholesterol levels is underway.
ETC-1002 (ESP-55016) Being developed by Esperion Therapeutics. The mechanism by which
ECT-1002 affects plasma lipids is by inhibiting lipid synthesis and enhancing lipid oxidation in liver.
The drug has been tested for its effectiveness in reducing LDL cholesterol levels although the
results have yet to be reported.
GFT505 Being developed by Genfit. GFT505 is a pan PPAR alpha/gamma/delta activator. The
drug is being developed as a treatment designed to decrease triglyceride levels and increase HDL
levels. The drug appears to be safe and effective alone and when used in combination with a statin
(simvastatin). Initial reports suggest beneficial effects on triglyceride and HDL levels at higher (100
mg and 60 mg vs. 30 mg). The drug has been reformulated and is now being tested as a 60 mg
pill rather than a 20 mg pill. The new formulation is being tested for safety and effectiveness in
doses up to 300 mg in subjects with obesity and type 2 diabetes. A summary of studies with GFT-
505 can be found here.
GSK-256073 Being developed by GlaxoSmithKline. GSK-256073 is a niacin receptor activator
that is being developed to reduce plasma triglyceride levels and increase HDL levels. Phase I clinical
trials have been conducted to see if this drug can minimize niacin-induced flushing when given in
combination with niacin. The drug is currently being tested to determine if it is able to reduce
glucose levels in patients with type 2 diabetes that are also being treated with conventional
antidiabetic therapy (metformin).
JTT-130 Being developed by Akros Pharma, a subsidiary of Japan Tobacco. JTT-705 is an
intestine-specific MTP inhibitor. This drug is being developed to reduce the production of
chylomicrons, particles that carry dietary fat into blood. This drug is being developed to reduce
chylomicron, VLDL and LDL levels. A study testing the safety and effectiveness in patients with
diabetes was completed in July 2010.
JTT-302 Being developed by Akros Pharma, a subsidiary of Japan Tobacco. JTT-302 is a
cholesteryl ester transfer protein inhibitor that is being developed as a treatment for dyslipidemia.
Evacetrapib (LY2484595) Being developed by Eli Lilly and Co. LY2484595 is a cholesteryl
ester transfer protein inhibitor that is being developed as a treatment for dyslipidemia. Results
from a study that examined the effectiveness of the drug alone or in patients being treated with
statins was published in 2011. The drug seems to have been reformulated and the reformulated
version is currently undergoing phase I testing.
MBX-8025 Being developed by Metabolex. MBX-8025 (formerly RWJ-800025) is a PPAR delta
activator. It is being developed as a treatment to reduce LDL cholesterol levels and to increase
HDL cholesterol levels. The drug has been reported to be safe when used alone and in combination
with atorvastatin. Treatment resulted in triglyceride and LDL lowering and an increase in HDL. It
was also reported that the drug was effective in reducing insulin and blood glucose. There has
been no news on this compound since 2008 so its development status is unclear.
MLDL1278A (BI-204; anti oxLDL) Being co-developed by Genentech and BioInvent. This
compound is an antibody to oxidized LDL. This drug is being developed to reduce levels of
oxidized LDL in plasma and has completed a successful phase I trial. A phase II ("proof of
activity") trial that examines the change in atherosclerotic plaque inflammation started in December
2010 and is expected to be completed in November 2012.
1-MNA (TRIA-662) Being developed by Pharmena. 1-MNA is a niacin derivative that is reported
to be "flush-free" meaning that the drug does not cause the subcutaneous flushing that is
associated with niacin or extended release niacin use. The drug is being developed as a treatment
of dyslipidemia (high triglyceride levels, high LDL cholesterol levels, low HDL cholesterol levels. The
company reported that the drug is effective in lowering triglycerides and CRP as well as effective in
raising HDl cholesterol levels.
REGN727 Being developed by Regeneron. REGN727 is an antibody that binds to PCSK9. When
administered this will interfere with PCSK9 and allow more LDL to be cleared from the blood
resulting in LDL lowering. The drug has been evaluated using subcutaneous (injection) and
intravenous (infusion) administration. A phase I study showed that LDL could be lowered by up to
60% for more than 1 month in patients taking statins after a single dose of the drug. Phase II
studies are currently underway in patients with hypercholesterolemia.
Rilapladib Being developed by GlaxoSmithKline. Rilapladib is an inhibitor of lipoprotein-associated
phospholipase A2 (LP-PLA2 inhibitor) which is being developed as a treatment to reduce the
formation of pro-atherogenic LDL. It has also been tested for its effects on platelet aggregation.
A study testing the effect of the drug on the stability of atherosclerotic plaque has been completed
although the results have not been announced. The drug is also being tested to see if it improves
cognitive function in patients with Alzheimer's disease.
RVX-208 (RVX000222) Being developed by Resverlogix. The exact structure of RVX-208 has
not been disclosed although it is described as being a member of the quinazoline family. A 13
week phase II study, ASSERT, did not meet the primary and secondary endpoints of increasing
apoA-I and HDL. There was also a significant increase in liver enzymes and a trend towards an
increase in creatinine which is used to monitor kidney function. As a result, a second study
ASSURE will proceed in Q1 of 2011 after having its enrollment procedure reassessed. The primary
endpoint of the ASSURE study is regression of atherosclerotic plaque volume with secondary
endpoints being change in apoA-I, HDL and HDL subclass. An exploratory endpoint is the change
in plaque composition which has been associated with a beneficial change in plaque stability in
other studies. The ASSURE study is currently underway and is expected to be completed in
January 2013.
SLx4090 Being developed by Nano Terra (formerly Surface Logix). SLx4090 is an intestinal-
specific MTP inhibitor. This drug is being developed to reduce the production of chylomicrons,
particles that carry dietary fat into blood. This drug is being developed to reduce chylomicron,
VLDL and LDL levels. The drug has been reported to be safe and effective although no additional
studies appear to be underway.
AMG-145 Being developed by Amgen. AMG-145 is an antibody to the protein PCSK9 that is being
developed as a treatment for elevated LDL levels. The drug is currently being tested in patients
with familial hypercholesterolemia, in patients that don't tolerate statins and for its effectiveness
when given in combination with a statin.
CER-001 (Cerenis-001) Being developed by Cerenis Therapeutics. This drug is a synthesic HDL
made from apoA-I mimetic peptide complexed with phospholipid that is designed to increase
reverse cholesterol transport. This drug is also now being considered for use in rare disorders of
lipoprotein metabolism such as homozygous FH in addition to patients with acute coronary
syndrome.
CSL-112 Being developed by CSL Limited. CSL-112 is a reconstituted HDL that is being
developed as a treatment to increase HDL levels and stimulate cholesterol removal from tissues
(reverse cholesterol transport). CSL-111, a compound similar to CSL-112, had progressed to
phase II studies but was apparently discontinued following a study that showed that the drug was
no better than placebo in reducing atherosclerosis. CSL-112 is a different formulation that is
reported to be more potent than CSL-111. A phase II study testing safety and pharmacokinetics
in patients with acute coronary syndrome will begin in January 2012.
DRL-17822 Being developed by Dr. Reddy's Labs. DRL-17822 is a cholesteryl ester transfer
protein inhibitor (CETP inhibitor) that is being developed as a treatment to increase HDL levels.
Results from phase I indicate that the drug is able to increase HDL from 51-111% and reduce LDL
from 25-56%. A phase II study testing the safety and effectiveness of the drug in patients with
high LDL cholesterol levels is underway.
ETC-1002 (ESP-55016) Being developed by Esperion Therapeutics. The mechanism by which
ECT-1002 affects plasma lipids is by inhibiting lipid synthesis and enhancing lipid oxidation in liver.
The drug has been tested for its effectiveness in reducing LDL cholesterol levels although the
results have yet to be reported.
GFT505 Being developed by Genfit. GFT505 is a pan PPAR alpha/gamma/delta activator. The
drug is being developed as a treatment designed to decrease triglyceride levels and increase HDL
levels. The drug appears to be safe and effective alone and when used in combination with a statin
(simvastatin). Initial reports suggest beneficial effects on triglyceride and HDL levels at higher (100
mg and 60 mg vs. 30 mg). The drug has been reformulated and is now being tested as a 60 mg
pill rather than a 20 mg pill. The new formulation is being tested for safety and effectiveness in
doses up to 300 mg in subjects with obesity and type 2 diabetes. A summary of studies with GFT-
505 can be found here.
GSK-256073 Being developed by GlaxoSmithKline. GSK-256073 is a niacin receptor activator
that is being developed to reduce plasma triglyceride levels and increase HDL levels. Phase I clinical
trials have been conducted to see if this drug can minimize niacin-induced flushing when given in
combination with niacin. The drug is currently being tested to determine if it is able to reduce
glucose levels in patients with type 2 diabetes that are also being treated with conventional
antidiabetic therapy (metformin).
JTT-130 Being developed by Akros Pharma, a subsidiary of Japan Tobacco. JTT-705 is an
intestine-specific MTP inhibitor. This drug is being developed to reduce the production of
chylomicrons, particles that carry dietary fat into blood. This drug is being developed to reduce
chylomicron, VLDL and LDL levels. A study testing the safety and effectiveness in patients with
diabetes was completed in July 2010.
JTT-302 Being developed by Akros Pharma, a subsidiary of Japan Tobacco. JTT-302 is a
cholesteryl ester transfer protein inhibitor that is being developed as a treatment for dyslipidemia.
Evacetrapib (LY2484595) Being developed by Eli Lilly and Co. LY2484595 is a cholesteryl
ester transfer protein inhibitor that is being developed as a treatment for dyslipidemia. Results
from a study that examined the effectiveness of the drug alone or in patients being treated with
statins was published in 2011. The drug seems to have been reformulated and the reformulated
version is currently undergoing phase I testing.
MBX-8025 Being developed by Metabolex. MBX-8025 (formerly RWJ-800025) is a PPAR delta
activator. It is being developed as a treatment to reduce LDL cholesterol levels and to increase
HDL cholesterol levels. The drug has been reported to be safe when used alone and in combination
with atorvastatin. Treatment resulted in triglyceride and LDL lowering and an increase in HDL. It
was also reported that the drug was effective in reducing insulin and blood glucose. There has
been no news on this compound since 2008 so its development status is unclear.
MLDL1278A (BI-204; anti oxLDL) Being co-developed by Genentech and BioInvent. This
compound is an antibody to oxidized LDL. This drug is being developed to reduce levels of
oxidized LDL in plasma and has completed a successful phase I trial. A phase II ("proof of
activity") trial that examines the change in atherosclerotic plaque inflammation started in December
2010 and is expected to be completed in November 2012.
1-MNA (TRIA-662) Being developed by Pharmena. 1-MNA is a niacin derivative that is reported
to be "flush-free" meaning that the drug does not cause the subcutaneous flushing that is
associated with niacin or extended release niacin use. The drug is being developed as a treatment
of dyslipidemia (high triglyceride levels, high LDL cholesterol levels, low HDL cholesterol levels. The
company reported that the drug is effective in lowering triglycerides and CRP as well as effective in
raising HDl cholesterol levels.
REGN727 Being developed by Regeneron. REGN727 is an antibody that binds to PCSK9. When
administered this will interfere with PCSK9 and allow more LDL to be cleared from the blood
resulting in LDL lowering. The drug has been evaluated using subcutaneous (injection) and
intravenous (infusion) administration. A phase I study showed that LDL could be lowered by up to
60% for more than 1 month in patients taking statins after a single dose of the drug. Phase II
studies are currently underway in patients with hypercholesterolemia.
Rilapladib Being developed by GlaxoSmithKline. Rilapladib is an inhibitor of lipoprotein-associated
phospholipase A2 (LP-PLA2 inhibitor) which is being developed as a treatment to reduce the
formation of pro-atherogenic LDL. It has also been tested for its effects on platelet aggregation.
A study testing the effect of the drug on the stability of atherosclerotic plaque has been completed
although the results have not been announced. The drug is also being tested to see if it improves
cognitive function in patients with Alzheimer's disease.
RVX-208 (RVX000222) Being developed by Resverlogix. The exact structure of RVX-208 has
not been disclosed although it is described as being a member of the quinazoline family. A 13
week phase II study, ASSERT, did not meet the primary and secondary endpoints of increasing
apoA-I and HDL. There was also a significant increase in liver enzymes and a trend towards an
increase in creatinine which is used to monitor kidney function. As a result, a second study
ASSURE will proceed in Q1 of 2011 after having its enrollment procedure reassessed. The primary
endpoint of the ASSURE study is regression of atherosclerotic plaque volume with secondary
endpoints being change in apoA-I, HDL and HDL subclass. An exploratory endpoint is the change
in plaque composition which has been associated with a beneficial change in plaque stability in
other studies. The ASSURE study is currently underway and is expected to be completed in
January 2013.
SLx4090 Being developed by Nano Terra (formerly Surface Logix). SLx4090 is an intestinal-
specific MTP inhibitor. This drug is being developed to reduce the production of chylomicrons,
particles that carry dietary fat into blood. This drug is being developed to reduce chylomicron,
VLDL and LDL levels. The drug has been reported to be safe and effective although no additional
studies appear to be underway.
Phase II If a drug is found to be safe in phase I studies it can, following approval of the FDA,
move to phase II. Phase II studies involve treatment of patients with the disease or condition that
the drug is intended for. Phase II studies usually involve more patients (about 100) than phase I
studies and last for a longer duration at 1-2 months. The objective of phase II is to demonstrate
both safety and clinical benefit.
move to phase II. Phase II studies involve treatment of patients with the disease or condition that
the drug is intended for. Phase II studies usually involve more patients (about 100) than phase I
studies and last for a longer duration at 1-2 months. The objective of phase II is to demonstrate
both safety and clinical benefit.
Copyright © 2008-2012 John Millar. All rights reserved.