Oral Presentation at ECTRIMS Congress Describes Dramatic Upregulation of Genes in Signaling Pathways Related to Myelin Sheath Production

Stuart SchlossmanMultiple Sclerosis, Myelin Repair, Stem Cell Related

FOR IMMEDIATE RELEASE

ENDECE Neural’s NDC-1308 Potently Enhances
Remyelination in Experimental Models of Multiple Sclerosis (MS)

Oral Presentation at ECTRIMS Congress
Describes Dramatic Upregulation of Genes in Signaling Pathways Related to
Myelin Sheath Production

Mequon, Wisc., October 4, 2013 – 
Scientists from ENDECE Neural presented preclinical data today showing
that the company’s lead compound, NDC-1308, addresses one of the root causes of
multiple sclerosis (MS)  by significantly
inducing remyelination in nerves that have been damaged by MS. In an oral
presentation at the 29th Congress of the European Committee for
Treatment and Research in Multiple Sclerosis (ECTRIMS) in Copenhagen, Denmark, the
ENDECE Neural researchers also reported a dramatic upregulation of genes in
signaling pathways involved in myelin sheath production.

NDC-1308 works
by inducing differentiation of oligodendrocyte progenitor cells (OPCs) into
mature oligodendrocytes, cells that synthesize and maintain the myelin sheath
that covers nerves in the brain and spinal cord, the researchers noted. By
contrast, the female hormones estradiol and estriol do not exert that effect.

“Repair of
the myelin sheath, called remyelination, has long been an elusive goal in the
treatment of multiple sclerosis,” explained Steven Nye, Ph.D., Vice President
of Discovery at ENDECE Neural. “The synthesis of NDC-1308, an estradiol analog,
was inspired by observations that pregnant women typically do not experience
the symptoms of MS during the third trimester. In our experiments, NDC-1308
induces remyelination in animal models of demyelination, compared to estradiol
and estriol which appear to be neuroprotective but do not induce OPC
differentiation.”

In his
presentation, Dr. Nye described how he and his colleagues synthesized more than
40 proprietary estradiol analogs in which the core structure of estradiol had
been modified, and assessed how subsets of those modifications changed the
hormone’s biological activity. The researchers identified NDC-1308 as the most
potent of several proprietary analogs having the ability to directly induce
differentiation of OPCs into mature oligodendrocytes. NDC-1308 derives its
novel biological activity from the addition of a specific alkoxyalklyl moiety
to the C-6 position on the estradiol B-ring.

Dr. Nye
reported the following findings:

·        
A
20% increase in remyelination (compared to vehicle control) of hippocampal
regions of the brain (P<0.01) was
associated with a 2-week course of NDC-1308 (50 mg/Kg once daily) in a mouse
model of demyelination, in which the neurotoxicant cuprizone was used to remove
the myelin sheath from the axons (nerve fibers) of mice.

·        
NDC-1308
caused a dramatic upregulation of key genes (5- to 75-fold) in signaling
pathways involved in OPC differentiation and myelin sheath production.

·        
NDC-1308
significantly induced OPCs to differentiate into mature oligodendrocytes (P<0.05).

·        
NDC-1308
(3 μM/day for 4 days) enhanced remyelination in demyelinated rat brain slices
visualized by staining for myelin basic protein, which was consistent with the
mouse cuprizone data.

·        
Prophylactic
administration of NDC-1308 (10 mg/kg/day for 10 days) delayed the onset of MS
symptoms and reduced the severity of MS in a mouse model of
experimental
allergic encephalitis (EAE) induced by myelin oligodendrocyte glycoprotein
(MOG), suggesting that the compound may
play a dual remyelination/anti-inflammatory role in treating MS.

“Despite its
structural similarity to estradiol and estriol, NDC-1308 differs from those two
female hormones by virtue of its potent induction of remyelination, as
demonstrated in animal models of MS,” commented James G. Yarger, Ph.D., chief
executive officer and co-founder of ENDECE Neural. “Unlike estradiol and
estriol, NDC-1308 induces OPC differentiation and maturation of
oligodendrocytes. We envision administration of NDC-1308 either alone or in
combination with current therapeutics that target the immune response and/or
inflammation associated with MS. NDC-1308 may thus fill an unmet medical need
for a remyelinating therapy in MS, one that may help improve the lives of
patients living with this devastating neurological disease.”                                   
About NDC-1308
NDC-1308 is a
novel chemical entity designed to address one of the root causes of MS, and is
being developed for potential use either alone or in combination with other MS
therapeutics that slow the progression of the disease. By controlling key genes
in pathways leading to myelin synthesis, NDC-1308 appears to induce restoration
of the lost myelin sheath that is believed to cause the devastating symptoms of
MS. NDC-1308 is a small molecule that readily crosses the blood-brain barrier,
allowing it to reach the tissues in the brain and spinal cord where promoting
myelin production is needed. ENDECE Neural discovered NDC-1308, and owns the
intellectual property surrounding the compound.
About ENDECE Neural
ENDECE Neural
is a private biotechnology company at the forefront of developing therapies to
repair and potentially reverse damage caused by devastating neurological
diseases such as MS. A wholly owned subsidiary of ENDECE LLC, ENDECE Neural was
founded in 2011 to focus on the development of what could be the first drug
capable of inducing remyelination of damaged nerves in patients with MS. The
company is leveraging decades of accumulated knowledge about how activation of
estrogen receptors in a specific manner affects gene regulation. Researchers at
ENDECE Neural have identified small-molecule compounds that upregulate key
genes in pathways involved in promoting myelin sheath synthesis. ENDECE Neural is
developing NDC-1308, which appears to directly induce OPCs to differentiate
into mature oligodendrocytes that restore the depleted myelin sheath in rodent
models of MS.  ENDECE Neural discovered
and owns the intellectual property surrounding its compounds, and the company’s
management team has a track record of successfully taking products from the
laboratory through FDA approval and commercial release.

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