Most start-up drug companies need to raise significant funds to pay for all the tests necessary just to get permission to try their medicine in humans.
But Indianapolis-based Chondrial Therapeutics LLC might have found a better way: it got the federal government to do the testing for it.
Chondrial, which is developing a drug for a rare condition known as Friedreich’s ataxia, will announce Wednesday that it has been accepted into a program run by the National Institutes of Health in which the NIH’s own researchers conduct tests on Chondrial’s drug and pay for outside contractors as needed.
Those services are worth at least $5 million and possibly twice that much, said Steve Plump, CEO of Chondrial.
“It helps validate our scientific platform. They’ve looked at everything from our [intellectual property] to our manufacturing,” said Plump, who was formerly chief marketing officer at Indianapolis-based Eli Lilly and Co. “For Chondrial, it’s a dramatic expansion of our drug development expertise. [NIH researchers] actually are an extension of our company now.”
Chondrial’s goal is to apply with the U.S. Food and Drug Administration as early April to get approval for human testing of its drug.
Chondrial’s drug, if successful, would be among the first to treat Friedreich’s ataxia, a genetic condition that affects only 5,000 Americans and no more than 20,000 people worldwide.
The disease leads to loss of coordination, loss of ability to walk and, ultimately, to fatal heart disease.
The NIH program also allows Chondrial to do its own work, separate from the NIH, to help advance testing on its drug, known as TAT-Frataxin. Plump and Chondrial founder Dr. Mark Payne, a pediatric cardiologist at Indiana University Health’s Riley Hospital for Children, think their drug could also prove useful for other rare diseases that, like Friedreich’s ataxia, involve problems in the mitochondria of the body’s cells.
“If it works here, then this strategy and this technique can walk into other rare diseases,” Payne said. “That becomes important, as the rare disease space is actually very lucrative to other large pharma companies.”
Chondrial estimates that its drug, if effective, could garner more than $1 billion in annual sales.
To date, Chondrial has raised $1.1 million from New York angel investor Tom Hamilton. It has used that money to manufacture small quantities of the drug for animal tests, verify that the drug diffuses into heart and brain tissues in mice, and zero in on an effective dose.
Hamilton, who has a daughter with Friedreich’s ataxia, said he expects to contribute more money in the next few months to move Chondrial’s research along
“Speed is important to me,” said Hamilton. “Hopefully kids who have this disease now will have a far better outcome than kids who were diagnosed 10 or 20 years ago.”
The NIH program, known as Therapeutics for Rare and Neglected Diseases, has already produced one success story. A company called Vtesse licensed from the NIH rights to a drug to treat a rare disease called Niemann-Pick Type C. That drug has been granted breakthrough status by the FDA and is now in late-stage testing.
Anton Simeonov, director of the division of preclinical innovation at the NIH’s the National Center for Advancing Translational Sciences, said, “NCATS seeks to develop new technologies and more efficient paradigms for translation, in the context of important unmet medical needs.”