After showing some early promise in treating European patients, Ann Arbor, MI-based Phrixus Pharmaceuticals, a startup developing drugs to treat cardiac and respiratory problems in young males with Duchenne muscular dystrophy, is preparing to launch U.S. clinical trials early next year.
During the first quarter of 2018, Phrixus will dose the first patient with its lead compound, Carmeseal-MD, in a clinical study at Cincinnati Children’s Hospital. The single-center, open-label Phase 2 trial will enroll eight non-ambulatory boys and young men over age 12 with early heart failure and respiratory dysfunction.
Duchenne muscular dystrophy affects between 15,000 to 20,000 boys in the United States, and is usually fatal by the time a patient reaches his 20s. The genetic disease, the most common form of muscular dystrophy, is marked by progressive muscle degeneration.
Thomas Collet, president and CEO of Phrixus, co-founded the company, which was spun out of the University of Michigan, in 2006. Unlike many other Duchenne drugs that focus on extending the time that patients can walk, Carmeseal-MD works to delay potentially fatal damage to the diaphragm and heart muscles.
Described by Phrixus as “a molecular band-aid,” Carmeseal was initially developed in the 1990s to treat heart attacks. Repurposed as Carmeseal-MD, the drug is infused into the bloodstream, where it finds microscopic tears in the heart muscle and binds to them. Collet says this can stop the leakage of calcium into heart cells, which if left unchecked can prevent the heart from delivering sufficient oxygen to vital organs.
Each patient in the phase 2 trial will be on Carmeseal-MD for 12 months, with drug product provided by the National Heart, Lung, and Blood Institute. Charley’s Fund and Team Joseph led the funding of the trial, with support from Harrison’s Fund, Coalition Duchenne, Hope for Gus, JB’s Keys to DMD, Little Hercules Foundation, and Michael’s Cause.
“We have enough capital to cover the minimum scope of the trial, and we’re looking to talk to corporate partners for our next phase of testing,” Collet says. “The FDA has already approved a protocol for 120 boys at 20 to 25 sites.” Carmeseal-MD, he notes, has received an orphan drug designation from the FDA.
In September, Phrixus announced results from the first patient receiving Carmeseal-MD as an unlicensed special in Europe. The patient underwent 15 months of treatment without any apparent adverse reactions. In a press release, Phrixus said the drug was “well tolerated, with benefits including reductions in muscle damage markers such as creatine kinase and cardiac troponin.”
The company will continue to make Carmeseal-MD available outside the U.S. as an unlicensed special through its expanded access program, an initiative done in partnership with Ethicor Pharma; click here for more information.
