The nonprofit CureDuchenne is applying venture capital thinking to early-stage research funding with a new initiative that will support development of treatments for the most common form of lethal muscular dystrophy, Duchenne.
The California non-profit said yesterday that it’s launching CureDuchenne Ventures to identify and develop a robust pipeline of therapies to treat the rare genetic disease. It will invest in early-stage projects of biotechnology and pharmaceutical companies and academic researchers, inviting philanthropists and investors to participate alongside, said Debra Miller, CD’s founder and CEO.
“Proof of concept clinical studies will require substantial effort and funds beyond the reach of traditional philanthropy,” Miller said in an email. “CureDuchenne Ventures was established to raise funds from venture philanthropists, foundations, donors and also offers an opportunity for the more traditional investor.”
CureDuchenne Ventures has already made its first investment in Lexicon Pharmaceuticals. Woodlands, Texas-based Lexicon is running clinical trials of an experimental drug, LX2931, for rheumatoid arthritis but is apparently also exploring the drug’s potential to initiate muscle regeneration in mouse models of Duchenne. CD says it plans to raise $5 million to support clinical development of LX2931 for Duchenne, with the hope of initiating clinical studies as early as next year.
Duchenne is a progressive muscle-wasting disease that results from a defective gene that encodes dystrophin, a key protein for muscle development. It occurs in young boys and is usually diagnosed before the age of 5. Most boys with the condition and are in a wheelchair by age 12 and don’t survive past their mid-20s.
No cure currently exists. Sarepta Therapeutics is pursuing regulatory approval for eteplirsen, which in a small clinical study showed increased production of dystrophin in boys with Duchenne along with an improved ability to walk further. Another Duchenne drug, drisapersen, recently failed to meet its primary endpoint in a Phase III clinical study.
CD provided funding to both of those projects and five others that have progressed to clinical trials.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
With the new initiative, it will take a blanketed approach to funding, supporting research in a spectrum of approaches and pathways including protein replacement strategies, treatments aimed at slowing progression, repositioned drugs and treatments for cardiac issues that accompany the disease.
“CureDuchenne Ventures will power our ability moving forward to support the most promising therapies and ensure that multiple avenues of research are explored,” said CureDuchenne Chief Scientific Officer Michael Kelly in a statement.
Venture philanthropy applies venture capital strategies like board involvement and long-term investment and applies them to achieving philanthropic goals. In healthcare, it’s being employed by other groups like the American Heart Association and the CureAlzheimer’s Fund to fill gaps in early-stage funding and de-risk investments for institutional investors.
