BioPontis, Reborn as Nonprofit, Takes On Rare Disease Drugs

Of the more than 7,000 rare diseases that each affect just a sliver of the population, an estimated 95 percent of them have no treatment. The lack of treatments is an economic problem, not a scientific one, says Barbara Handelin, CEO of the BioPontis Alliance for Rare Diseases.

Large pharmaceutical companies see little financial gain from the massive investments required to develop a drug for a small patient population, says Handelin. That’s because such research entails higher up-front financial risk, too high perhaps, even for a treatment with the potential for a stronger return. But Handelin says a nonprofit model can unlock promising academic drug research in rare diseases.

“The philanthropic donation is looking for a return in one way—to make as many tries as possible in this rare disease that affects somebody I love,” she says. “Their return on capital is trying, and hopefully, being successful if you make enough tries.”

BioPontis, which was initially formed as a for-profit venture in 2009, has now relaunched as BioPontis Alliance for Rare Diseases, a Raleigh, NC-based nonprofit organization focused on advancing academic research in rare diseases. BioPontis plans to partner with with patient groups and universities around the world to identify drug technology and bring it to a point where it can be licensed by companies to develop, test, and if all goes well, commercialize.

BioPontis is announcing its first patient group partnership on Monday, a joint venture with the Hereditary Neuropathy Foundation. The New York City-based foundation supports research on treatments for Charcot-Marie-Tooth Disease (CMT), an inherited neurological disorder that affects motor and sensory nerves. The disease causes muscle weakness in the feet and lower legs. CMT is not fatal but as it progresses, motor skills deteriorate and patients often lose the ability to walk. An estimated 2.6 million people worldwide have CMT, which currently has neither a cure nor any treatments.

The partnership with the Hereditary Neuropathy Foundation demonstrates the kind of university research that BioPontis wants to unlock. The genetic cause for the most common form of CMT was first identified in 1991. But screening technology to search for a small molecule drug to treat the disease was not developed until 2012.

Allison Moore, CEO of the foundation, says her organization has provided $1.1 million in funding to universities researching CMT treatments, including the University of Southern California and the University of California, Los Angeles. With BioPontis bringing support and expertise to the universities, Moore says, her foundation can operate more efficiently and devote resources to finding patients who could participate in future clinical trials.

“The most important thing is to get patients that are suffering, treatments,” says Moore, who was diagnosed with CMT in 2001. “What’s the fastest way to get there? This partnership, we believe.”

Read more at the main source.  Article by Frank Vinluan.