Pinnacle Hill Project Aims to Treat Angelman by Changing Gene Activity

Pinnacle Hill will start a second project aimed at the development of a treatment for Angelman syndrome based on changing gene activity, the company announced.

Pinnacle Hill is the result of a partnership between the University of North Carolina (UNC) at Chapel Hill and Deerfield Management, an investment management firm. Launched with an investment of up to $65 million from Deerfield in October 2018, Pinnacle Hill’s goal is to accelerate the development of innovative medicines for diseases with unmet medical needs.

Angelman is caused by the loss or malfunction of the maternal copy of the UBE3A gene in neurons within specific brain regions. Replacing the faulty UBE3A gene or inducing its reactivation in these nerve cells could hold therapeutic potential.

The research project, led by Ben Philpot an associate director of the UNC Neuroscience Center in the UNC School of Medicine and supported by an interdisciplinary team of scientists, aims to explore new compounds that can manipulate gene activity in Angelman’s patients.

“The project with Dr. Philpot is another great example of innovative research at UNC which greatly benefits from the funding and drug development expertise and capabilities offered through Pinnacle Hill,” Jon Collins, the chief scientific officer of Pinnacle Hill, said in a press release.

“I can’t envision a more qualified team of academic and drug development scientists to work hand-in-hand on delivering a new medicine for Angelman’s Syndrome patients,” he added.

This is Pinnacle’s Hill second funded research. The first project aimed to develop new therapies for multiple myeloma, a type of cancer that develops in the bone marrow. Lindsey James, an assistant professor at the UNC Eshelman School of Pharmacy’s Division of Chemical Biology and Medicinal Chemistry, leads that line of research.

A steering committee composed of members from both UNC-Chapel Hill and Deerfield leadership teams approve and oversee all of Pinnacle Hill’s funded projects. Projects are selected based on their potential to advance investigational therapeutic candidates.

“The goal of our alliance with UNC-Chapel Hill is to identify and advance the most important discoveries that could improve the lives of patients and their families,” James E. Flynn, a managing partner at Deerfield, said in another press release.