Chondrial Therapeutics Inc., the Bala Cynwyd-based biopharmaceutical company, has raised $25.8 million to develop treatment for mitochondrial disorders.
The firm is targeting Freidreich’s Ataxia (FA), a genetic disorder that affects about 5,000 people in the United States and 10,000 in Europe.
FA attacks multiple body systems, particularly the brain and heart, and is caused by a deficiency of a key protein, frataxin, which is normally in the mitochondria of all tissues. Mitochondria convert nutrients into energy within cells. Affected children and young adults gradually lose their coordination, along with the ability to speak and walk. Their hearts become thick and weak, and can fail. There is no cure for FA, according to the company.
Chondrial’s premise is that by replacing the deficient protein, the mitochondria will resume normal energy-making functions, patients’ symptoms may be minimized, and disease progression may be curtailed.
The treatment will administered by subcutaneous injection, said CEO Carole Ben-Maimon. “We don’t know how frequently it will be, but it will be lifetime therapy. That’s to be seen in clinical trials.”
Chondria’s FA therapy has the much-coveted orphan drug designation from the FDA. Chondrial will file an Investigational New Drug application in the next couple of months and go into trials “almost immediately,” she said.
“There is really no other company doing what we’re doing,” Ben-Maimon said. “There are a bunch of trials going on, but there is no one replacing the frataxin molecule. Some work around the problem. There are some doing gene therapy for FA.”
Ben-Maimon said that in addition to Chondrial’s FA research project, the company has a technology that allows it to carry various types of proteins across intracellular organelles.
She said the 11-employee company soon will look for a second round of financing. Most of Chondria’s money is from Deerfield Management, which invests broadly in public health-care securities. The company has no revenues yet.
Ben-Maimon said she hopes that Chondrial follows the success of other biotechs launched in University City.