MyFatDog
Phase I
Phase I Pipeline Drugs
ALN-PCS02 Being developed by Alnylam Pharmaceuticals. ALN-PCS02 is a drug that reduces
plasma levels of PCSK9. PCSK9 prevents LDL from being cleared from blood so lowering PCSK9
enhances LDL clearance from blood resulting in lower LDL levels. The drug is targeting patients
with severe hypercholesterolemia that are no adequately controlled with existing treatments, a
population estimated to be 500,000.
ARI-3037MO Being developed by Arisaph. ARI-3037MO is a niacin-like drug developed to have
the lipid-altering benefits of niacin without the flushing side-effects. In March 2011 the US FDA
accepted the Investigational New Drug application that allows the company to proceed with
human trials.
BMS-844421(BMS-PCSK9Rx) Being co-developed by Bristol-Myers Squibb and Isis
Pharmaceuticals. PCSK9 antisense is a drug that reduces plasma levels of PCSK9, a protein that
targets the LDL receptor for degradation. This drug is being developed as a treatment to lower
LDL levels. A phase I study was terminated so the status of this drug is currently unknown.
DB959Na Being developed by Dara Biosciences. DB959Na is a dual PPAR gamma/delta
activator. The drug is being developed as a way to improve insulin sensitivity in diabetics as well
as a way of improving the plasma lipoprotein profile in these patients. A phase I study has been
completed and was reported to be safe at a dose that should allow once daily administration.
DRL-21995 Being developed by Dr. Reddy's Labs. DRL-21995 is being developed for the
treatment of dyslipidemia. A phase I study has been completed and the results of this study are
being analyzed. DRL-21994 is apparently a similar compound that is a backup compound for
DRL-21995.
IMO-3100 Being developed by Idera Pharmaceuticals. IMO-3100 is a compound that blocks the
action of two receptors, Toll-like receptors (TLR) 7 and 9, that turn on inflammatory pathways in
cells. Although initially developed to treat inflammation, IMO-3100 has been shown to effectively
reduce LDL in preclinical studies in mice and may prove to be of benefit in improving diseases with
generalized inflammation such as the metabolic syndrome. The drug was well tolerated in phase I
studies and the company plans to move forward into phase II development.
MDCO-216 (formerly ETC-216) (The Medicines Company under license from Pfizer).
Commonly referred to as "Drano for the heart" since it was shown to rapidly reduced
atheroclerotic plaque in an earlier clinical study, this is a synthetic HDL containing recombinant
apoA-I (Milano). Clinical studies with MDCO-216 are expected to begin in 2011.
MP-136 Being developed by Mitsubishi Tanabe Pharma. MP-136 is a PPAR alpha activator being
developed as a treatment for dyslipidemia.
NCX6560 Being developed by NicOx. NCX6560 is a new statin drug that also has vasodilatory
properties. This drug is being developed to lower LDL levels and to enhance vasodilation. A
phase Ib study showed that the highest dose was able to lower LDL levels by 57% which was
similar to what was achieved with atorvastatin. The company is currently seeking partners to
assist with future development.
SPC4955 Being developed by Santaris Pharma. SPC4955 is a compound designed to lower LDL
levels by reducing apoB100, the main protein on LDL. A phase I ascending dose study was
completed in November 2011.
SPC5001 Being developed by Santaris Pharma. SPC5001 is a compound designed to lower LDL
levels by reducing PCSK9. PCSK9 prevents LDL from being cleared from blood so lowering
PCSK9 enhances LDL clearance from blood resulting in lower LDL levels. A phase I ascending
dose study was reported as being terminated in November 2011 so future development is
uncertain.
TA-8995 Being developed by Mitsubishi Tanabe Pharma. TA-8995 is a cholesteryl ester transfer
protein inhibitor (CETP inhibitor) that is being developed as a treatment to increase HDL and
reduce LDL levels.
VIA3196 Being developed by Madigral Pharmaceuticals under license from Roche. This drug is a
thyroid receptor beta activator being developed as a treatment to reduce LDL cholesterol and
triglyceride levels in combination with a statin.
XL014/XL652 Being developed by Exelixis under license to Bristol-Myers Squibb. XL 041/ and
XL 652 are LXR activators that are being developed to increase reverse cholesterol transport.
ALN-PCS02 Being developed by Alnylam Pharmaceuticals. ALN-PCS02 is a drug that reduces
plasma levels of PCSK9. PCSK9 prevents LDL from being cleared from blood so lowering PCSK9
enhances LDL clearance from blood resulting in lower LDL levels. The drug is targeting patients
with severe hypercholesterolemia that are no adequately controlled with existing treatments, a
population estimated to be 500,000.
ARI-3037MO Being developed by Arisaph. ARI-3037MO is a niacin-like drug developed to have
the lipid-altering benefits of niacin without the flushing side-effects. In March 2011 the US FDA
accepted the Investigational New Drug application that allows the company to proceed with
human trials.
BMS-844421(BMS-PCSK9Rx) Being co-developed by Bristol-Myers Squibb and Isis
Pharmaceuticals. PCSK9 antisense is a drug that reduces plasma levels of PCSK9, a protein that
targets the LDL receptor for degradation. This drug is being developed as a treatment to lower
LDL levels. A phase I study was terminated so the status of this drug is currently unknown.
DB959Na Being developed by Dara Biosciences. DB959Na is a dual PPAR gamma/delta
activator. The drug is being developed as a way to improve insulin sensitivity in diabetics as well
as a way of improving the plasma lipoprotein profile in these patients. A phase I study has been
completed and was reported to be safe at a dose that should allow once daily administration.
DRL-21995 Being developed by Dr. Reddy's Labs. DRL-21995 is being developed for the
treatment of dyslipidemia. A phase I study has been completed and the results of this study are
being analyzed. DRL-21994 is apparently a similar compound that is a backup compound for
DRL-21995.
IMO-3100 Being developed by Idera Pharmaceuticals. IMO-3100 is a compound that blocks the
action of two receptors, Toll-like receptors (TLR) 7 and 9, that turn on inflammatory pathways in
cells. Although initially developed to treat inflammation, IMO-3100 has been shown to effectively
reduce LDL in preclinical studies in mice and may prove to be of benefit in improving diseases with
generalized inflammation such as the metabolic syndrome. The drug was well tolerated in phase I
studies and the company plans to move forward into phase II development.
MDCO-216 (formerly ETC-216) (The Medicines Company under license from Pfizer).
Commonly referred to as "Drano for the heart" since it was shown to rapidly reduced
atheroclerotic plaque in an earlier clinical study, this is a synthetic HDL containing recombinant
apoA-I (Milano). Clinical studies with MDCO-216 are expected to begin in 2011.
MP-136 Being developed by Mitsubishi Tanabe Pharma. MP-136 is a PPAR alpha activator being
developed as a treatment for dyslipidemia.
NCX6560 Being developed by NicOx. NCX6560 is a new statin drug that also has vasodilatory
properties. This drug is being developed to lower LDL levels and to enhance vasodilation. A
phase Ib study showed that the highest dose was able to lower LDL levels by 57% which was
similar to what was achieved with atorvastatin. The company is currently seeking partners to
assist with future development.
SPC4955 Being developed by Santaris Pharma. SPC4955 is a compound designed to lower LDL
levels by reducing apoB100, the main protein on LDL. A phase I ascending dose study was
completed in November 2011.
SPC5001 Being developed by Santaris Pharma. SPC5001 is a compound designed to lower LDL
levels by reducing PCSK9. PCSK9 prevents LDL from being cleared from blood so lowering
PCSK9 enhances LDL clearance from blood resulting in lower LDL levels. A phase I ascending
dose study was reported as being terminated in November 2011 so future development is
uncertain.
TA-8995 Being developed by Mitsubishi Tanabe Pharma. TA-8995 is a cholesteryl ester transfer
protein inhibitor (CETP inhibitor) that is being developed as a treatment to increase HDL and
reduce LDL levels.
VIA3196 Being developed by Madigral Pharmaceuticals under license from Roche. This drug is a
thyroid receptor beta activator being developed as a treatment to reduce LDL cholesterol and
triglyceride levels in combination with a statin.
XL014/XL652 Being developed by Exelixis under license to Bristol-Myers Squibb. XL 041/ and
XL 652 are LXR activators that are being developed to increase reverse cholesterol transport.
Phase I Once a new drug has demonstrated that it is safe and effective in animals the company
can file an Investigational New Drug (IND) application with the FDA. This allows for limited testing
in humans. Initial testing is usually done in a small number (about 20) of normal volunteers. The
drug is first tested at a very low dose and then the dose is increased to a dose that is thought to
be effective for treating disease. The duration of treatment in phase I is fairly short at 1-2 weeks.
can file an Investigational New Drug (IND) application with the FDA. This allows for limited testing
in humans. Initial testing is usually done in a small number (about 20) of normal volunteers. The
drug is first tested at a very low dose and then the dose is increased to a dose that is thought to
be effective for treating disease. The duration of treatment in phase I is fairly short at 1-2 weeks.
Copyright © 2008-2012 John Millar. All rights reserved.