MDA Supports Development of 'Dissociative Glucocorticoid' for DMD

Muscular Dystrophy Association has awarded ReveraGen BioPharma $1.5 million to develop drug

TUCSON, Ariz., May 3, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association announced today that it has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, to develop and test a "dissociative glucocorticoid" as a potential treatment for Duchenne muscular dystrophy (DMD).

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MDA has partnered with the U.S. National Institutes of Health Therapeutics for Rare and Neglected Diseases (TRND) program in supporting development of this drug. The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program.

Glucocorticoid (also known as "corticosteroid") medications — such as prednisone and prednisolone — are widely prescribed in DMD, where they slow the progression of the disease, probably by reducing muscle inflammation, as well as other effects. However, glucocorticoids have many side effects, such as weight gain, reduced bone density, hypertension and psychological distress, which limit their usefulness and highlight the need for alternative therapies.

The side effects are believed to be caused mostly by the so-called "genomic" actions of glucocorticoids, while most of their benefits appear to be due to their nongenomic actions.

ReveraGen (formerly known as Validus BioPharma) says its experimental compound VB15 is designed to eliminate the genomic effects of glucocorticoids while retaining their nongenomic effects.

"We're really excited about this project," said Jane Larkindale, MDA's director of translational research. "A medication that provides the benefits of glucocorticoids without the side effects would be most welcome by the Duchenne dystrophy community. Such a drug could be used alone or in combination with other types of therapies now in development."

Larkindale added that this type of drug could, down the road, have application to many other diseases besides DMD that are now treated with glucocorticoids. Examples are myasthenia gravis (MG), dermatomyositis (DM), polymyositis (PM), asthma and rheumatoid arthritis.

ReveraGen has tested VB15 in mice with a DMD-like disease and has found it improved muscle function and reduced muscle inflammation.

"It is highly encouraging to see significant beneficial effects with VBP15 in mdx [DMD-like] mice," said Kanneboyina Nagaraju, co-founder of ReveraGen.

Edward Connor, interim CEO and president of ReveraGen, said, "VB15 is moving well through the drug development process. We are preparing for our initial regulatory interactions for DMD, and preclinical evaluation of other indications is ongoing."

To learn more, read the Quest News Online article MDA Funds Development of Gentler Glucocorticoid for DMD.

About DMD

DMD is a degenerative muscle disease — affecting boys almost exclusively — that involves progressive degeneration of voluntary and cardiac muscles, with resulting weakness and heart abnormalities. The disease manifests in early childhood. Loss of the ability to walk occurs in most children with DMD between the ages of 10 and 12 years; weakened cardiac and respiratory muscles severely limit life span.

Glucocorticoid medications, such as prednisone and its chemical cousin prednisolone, slow disease progression and prolong walking ability.

About MDA Venture Philanthropy (MVP)

MVP is the Muscular Dystrophy Association's drug development program, which operates within MDA's translational research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

For more information, visit mda.org and follow MDA on Facebook (facebook.com/MDANational) and Twitter (@MDAnews).

About ReveraGen

ReveraGen is a privately held company focused on novel drug development for neuromuscular and chronic inflammatory disease. Its initial lead compound, VBP15, is a dissociative steroid that separates efficacy-associated nongenomic activities from the genomic properties associated with side effects.

About the NIH TRND program

The Therapeutics for Rare and Neglected Diseases (TRND) program, administered by the U.S. National Institutes of Health (NIH), encourages the development of new drugs for rare and neglected diseases. The program is intended to stimulate drug discovery and development collaborations between NIH and other entities, including academic scientists, nonprofit organizations and industry.

SOURCE Muscular Dystrophy Association



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