targeting a leupeptin analogue into muscle cells to inhibit calpain in order

[Guest guest]

(NOTE: While this news is about a type of muscular dystrophy, DMD, I

am posting this for the information of this new drug that can help

preserve muscle, a calpain inhibitor. I am wondering if once trials

are conducted, it may be a way one day also of helping preserve CMT

muscles ~ Gretchen)

Duchenne Muscular Dystrophy, NEJM Substantiates Myodur's Development

Initiatives

23 Jun 2005 Medical News Today

A recent New England Journal of Medicine (June 9, 2005 issue) lead

review article, " Mechanisms of Disease: Calpains and Disease "

implicates calpain as a primary protease that, when up-regulated,

initiates accelerated tissue breakdown in several neuromuscular and

neurodegenerative diseases. This includes Duchenne muscular dystrophy

(DMD), a genetic disease which affects 1 out of every 3,500 male

children rendering them wheelchair dependent by about the age of 12

with death common in late adolescence. The article substantiates

CepTor's drug development initiatives of MYODUR to treat DMD by

targeting a leupeptin analogue into the muscle cells to inhibit

calpain in order to preserve muscle.

According to Norman Barton, M.D., PhD, Executive Vice President and

Chief Medical Officer of CepTor, " This is a very important article as

it is further validation that our research and development strategy

for MYODUR is on the right track. We have been focused on calpain

inhibition for DMD, and as a result of extensive research we believe

that we have developed a unique, proprietary, safe and efficient,

targeted calpain inhibitor. " Dr. Barton continued, " Currently, we are

preparing to file an investigational new drug application and move

MYODUR into phase I/II clinical trials for DMD in children with this

devastating disease. "

ABOUT MYODUR:

MYODUR targets muscle cells by using a carnitine carrier molecule and

inhibits calpain by attaching a leupeptin analogue onto the carrier.

Leupeptin has been studied extensively in a variety of animal models

and was shown to be an effective means of slowing or delaying muscle

wasting. Carnitine is a compound present in skeletal muscle involved

in the transfer of fatty acids across mitochondrial membranes.

CepTor's technology uses the carnitine carrier molecule to direct the

leupeptin analogue into targeted cells where it inhibits the up-

regulation of calpain. Therefore, by targeting a calpain inhibitor to

muscle cells, it is believed that MYODUR will prevent the degradation

and promote the preservation of functional muscle.

ABOUT CALPAIN: Calpain exists in every cell of the body and is a

protease that degrades cellular proteins naturally in a normal

metabolic process. When calpain is abnormally up regulated, the

accelerated degradation process breaks down cells and tissues faster

than they can be restored, resulting in several serious neuromuscular

and neurodegenerative diseases.

About CepTor

CepTor Corporation is a development-stage biopharmaceutical company

engaged in the discovery, development, and commercialization of

proprietary, cell-targeted therapeutic products for the treatment of

neuromuscular and neurodegenerative diseases with a focus on orphan

diseases. The Company's mission is to increase the quality and

quantity of life of people suffering with these diseases. An orphan

disease is defined in the United States as a serious or life-

threatening disease that affects less than 200,000 people and for

which no definitive therapy currently exists. CepTor Corporation

seeks to create an efficient orphan drug platform by taking advantage

of the legislative, regulatory and commercial opportunities common to

these rare diseases. CepTor's primary efforts are currently being

focused on moving its lead product, MYODUR, into phase I/II clinical

trials for Duchenne's muscular dystrophy. The Company's broad

platform technology also includes the development of products for

multiple sclerosis, retinal degeneration and epilepsy.

This news release contains forward-looking statements. Such

statements are valid only as of today, and we disclaim any obligation

to update this information. These statements are subject to known and

unknown risks and uncertainties that may cause actual future

experience and results to differ materially from the statements made.

These statements are based on our current beliefs and expectations as

to such future outcomes. Drug discovery and development involve a

high degree of risk. Factors that might cause such a material

difference include, among others, uncertainties related to the

ability to attract and retain partners for our technologies, the

identification of lead compounds, the successful preclinical

development thereof, the completion of clinical trials, the FDA

review process and other government regulation, our pharmaceutical

collaborator's ability to successfully develop and commercialize drug

candidates, competition from other pharmaceutical companies, product

pricing and third party reimbursement.

http://www.ceptorcorp.com