MDA has awarded $1 million to Tivorsan Pharmaceuticals for development of TVN-102, an experimental treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

The award was made through MDA's Venture Philanthropy (MVP) arm, part of MDA's translational research program. Tivorsan is a biotechnology company located in Providence, R.I.

TVN-102 is an experimental drug based on biglycan, a naturally occurring protein found in the membrane surrounding each muscle fiber. Biglycan is part of a cluster of proteins located at intervals in the membrane. These shock-absorbing clusters appear to protect the membrane as muscle fibers contract and relax.

Tivorsan hopes to have completed animal testing and other FDA-required studies of TVN-102 and to request permission for human trials of the drug by late 2012. The company hopes to begin human testing of TVN-102, probably in healthy volunteers without DMD or BMD, in 2012 or 2013.

If the preliminary studies show the drug is safe and well-tolerated and that it has the potential to be effective in treating DMD or BMD, tests in people with one of these diseases will likely follow.

MDA launched its innovative Bridge-to-Industry (B2I) program with a $180,000 grant over three years to postdoctoral fellow Archi Joardar at the University of Arizona in Tucson, to develop two promising drug candidates for the treatment of amyotrophic lateral sclerosis (ALS).

MDA’s Bridge-to-Industry, or B2I, is a pilot project that trains promising researchers in translational research by providing experience both in academia and the biopharmaceutical industry.    

Through the B2I program, MDA is able to fund investigators to conduct drug development research under the guidance of two mentors: one experienced in academic research and the other experienced in the industrial side of drug development.

Daniela Zarnescu, associate professor in neuroscience and molecular & cellular biology at the College of Science, University of Arizona (UA), will provide academic mentoring to Joardar. Zarnescu currently has an active MDA research grant for her work on gene and drug discovery research in a fruit fly model that carries a mutation in the ALS-associated TDP43 gene.

Joardar will receive mentoring on the industry side from Chris Hulme, co-director of the BIO5 Institute in Oro Valley, Ariz. Hulme is an expert in small-molecule drug design and the development of chemical-based methods to hasten the drug discovery process.

Mentorship under Zarnescu and Hulme will provide Joardar with a unique environment in which to train while furthering the development of two promising ALS drug candidates identified in Zarnescu's work. Joardar will evaluate the ability of each drug to reduce neurotoxicity caused by ALS-associated mutations in the TDP43 gene..

The Muscular Dystrophy Association has committed $750,000 to help support a phase 2 clinical trial assessing the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and quality of life in people with amyotrophic lateral sclerosis (ALS).

Developed by Synapse Biomedical in Oberlin, Ohio, the DPS received approval from the U.S. Food and Drug Administration (FDA) on Sept. 29, 2011, as a “humanitarian use device” (HUD) for the treatment of chronic hypoventilation (inadequate breathing) in ALS. The surgically implanted device stimulates the movement of the diaphragm, the main muscle used in breathing.

In people with ALS who have chronic breathing problems and whose diaphragms are still able to respond to electrical stimulation, the DPS may forestall or negate the need for invasive ventilation.

Investigators will compare respiratory function and quality of life in people with ALS who use the DPS and those who receive the current standard treatment.

The Muscular Dystrophy Association has awarded $120,000 to Cambridge, Mass.-based Catabasis Pharmaceuticals. The award is part of a strategic partnership under which the pharmaceutical company will test two compounds — CAT-1004 and CAT-1040 — in the mdx research mouse model of Duchenne muscular dystrophy (DMD).

The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program.

Catabasis plans to demonstrate in the new four-month study that CAT-1004 reduces inflammation in muscle tissue and improves muscle function. It will compare CAT-1004, CAT-1040 and prednisolone to identify the most effective compound. If favorable results are obtained, the company plans to begin clinical trials in people with DMD.

Catabasis has completed a safety, tolerability and pharmacokinetics (what the body does to a drug) study of CAT-1004 in healthy human volunteers over an eight-week trial period. It has an open Investigational New Drug application for the compound with the U.S. Food and Drug Administration, which allows it to conduct clinical trials of the investigational drug in the United States.

The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular dystrophy (DMD).

The goal is to develop a drug that provides the benefits of currently available glucocorticoids (such as prednisone and deflazacort), but that doesn't cause the negative side effects associated with these medications.

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

ReveraGen BioPharma (formerly known as Validus BioPharma) has developed an experimental compound called VBP15, which may offer people with DMD the beneficial effects of prednisone and related drugs without the side effects.

The unwanted side effects, ReveraGen scientists have found, are largely due to the genomic mechanism of action in glucocorticoids. VBP15 does not appear to have this genomic mechanism of action, the scientists say, but it appears to retain the beneficial effects of glucocorticoids' nongenomic mechanisms, which are those that combat inflammation and stabilize cell membranes.

ReveraGen says mice with a DMD-like muscle disease benefited from treatment with this drug, showing improved muscle function, increased activity and reduced muscle inflammation.

The Muscular Dystrophy Association has given a $300,000 grant to the Center for Genetic Medicine Research (CGMR) at Children’s National Medical Center in Washington, D.C., to continue standardized and reliable preclinical studies of neuromuscular diseases.

The MDA Translational Research Grant was awarded to CGMR's Kanneboyina Nagaraju, who directs the Murine Functional Testing Core Facility. It's the largest preclinical drug testing facility for neuromuscular disease models.

The facility helps advance drug development by testing drugs and other experimental treatments in mouse models of neuromuscular disease, allowing researchers to obtain information about how a treatment is likely to influence a disease course.

The U.S. Food and Drug Administration (FDA) generally require this type of preclinical study before it allows new substances to be tested in humans.

Several strains of mice with various neuromuscular diseases are maintained at the facility, including some with rare forms of muscular dystrophy that are not widely available.