Startup Duchenne muscular dystrophy (DMD) drug developer DMD Therapeutics said today it will advance its first candidate into the clinic after winning a commitment of $400,000 from three foundations.
Ryan's Quest, Michael's Cause, and Pietro's Fight have committed the $400,000 in seed capital toward developing DMD-813, which has been shown in preclinical studies to reduce damage and inflammation in muscle leading to “markedly” increased muscle strength in the standard mouse model of DMD, the company said.
Additional studies have shown significant increases in the ability of animals treated with DMD-813 to walk long distances, DMD Therapeutics added.
“The preclinical results with DMD-813 therapy are striking, and its primary mechanism of action affects one of the molecular mechanisms responsible for muscle weakness in DMD,” Carlo Rago, Ph.D., CSO and co-founder of DMD Therapeutics, said in a statement. “There are few if any other drug candidates that demonstrate the same level of efficacy in preventing muscle damage and restoring muscle strength in mdx mice.”
Another co-founder of the company, Ron Berenson, M.D., added that DMD-813 has demonstrated its therapeutic effects in skeletal muscle, including the diaphragm, a major cause of pulmonary failure that often leads to death in DMD.
Based in Seattle, DMD Therapeutics was founded last year to develop novel therapeutics for DMD and other muscular dystrophies. In August, the company disclosed plans for a $600,000 investment offering, according to a Form D filed with the U.S. Securities and Exchange Commission on August 11, 2016.
DMD Therapeutics said today Drs. Berenson and Rago are being joined by experts in biotechnology with “extensive experience in all aspects of drug development, including animal studies, manufacturing, toxicology, clinical trials, and regulatory affairs.”
“We are confident that this management team will drive this promising agent through development and clinical trials. If the preclinical results hold up in patients, we should end up with a highly effective therapy for DMD,” stated Dave Schultz, co-founder with his wife Maria Schultz of Ryan's Quest, named for the couple’s son Ryan.
“When Ryan was diagnosed with DMD, we were forced to face our worst nightmare—that our child had a fatal disorder for which there is no treatment or cure. Although our dreams for Ryan's lifelong future were stolen by this news, hope was left in its place,” the couple says on the Ryan’s Quest website. “We have made the choice to fight this disease for our son by gaining support and funding for DMD research.”
Ryan's Quest, Michael's Cause, and Pietro's Fight are all 501(c)3 nonprofit organizations focusing on funding preclinical, IND-enabling, and clinical studies for DMD therapy candidates deemed to be the most promising.