Pinnacle Hill builds momentum with Angelman Syndrome, multiple myeloma projects
Finding new therapies and medicines for some of the world’s most perplexing and deadly diseases won’t happen with a same-old, same-old mindset. And that’s why, in late 2018, UNC-Chapel Hill and Deerfield Management set out to build something new. By bringing together Carolina’s world-class life sciences research expertise with Deerfield’s funding resources and deep industry experience in drug development, the two organizations launched Pinnacle Hill. After launching less than two years ago, the company has been busy scoping, supporting and launching new research projects and engaging with faculty in discussions about new potential treatments that they envision in the not-so-distant future.
Since its launch, Pinnacle Hill has set the foundation for identifying potential faculty research projects and working with faculty to accelerate the development of their ideas and technologies into new medicines that address the significant unmet medical needs of our times. Answers to two of these pressing medical needs are being pursued by the first faculty projects currently funded by Pinnacle Hill: multiple myeloma, a devastating cancer that develops in bone marrow, and Angelman syndrome, a neuro-genetic disorder characterized by developmental delays, abnormal brain activity and severe seizures. Neither of these conditions currently has a cure.
With such serious medical challenges comes the need for resources and expertise that can expedite treatments and therapies. As part of Deerfield’s involvement with Pinnacle Hill, it committed up to $65 million of targeted funding to support projects that cover a wide range of therapeutic areas. Yet, the value that Deerfield brings to the partnership far exceeds the monetary support alone. It also supports faculty with expert drug development guidance, project management and business strategy insights that help move projects ahead and allow faculty to concentrate on their core mission – advancing the research itself.
In April 2019, the University hired Jon Collins, a seasoned pharmaceutical executive from GlaxoSmithKline, as its chief scientific officer to lead Pinnacle Hill’s drug discovery and development initiatives. Since joining, Collins has been busy connecting with Carolina researchers and getting the first Pinnacle Hill projects up, running and quickly moving forward.
“It has been a tremendously rewarding experience meeting with investigators to learn about the cutting-edge research being pursued across campus,” said Collins. “Pinnacle Hill has funded two exciting projects thus far, which bring together expert Carolina researchers with veteran drug development scientists to work hand-in-hand to progress the projects. There is no doubt that the funding and resources provided to these projects through Pinnacle Hill will not only progress the basic research, but will also improve the probability of successfully delivering new medicines to patients.”
On a mission to treat multiple myeloma
After Collins’ arrival in 2019, Pinnacle Hill announced its first project agreement, which supports the work of Lindsey James, an assistant professor in the Eshelman School of Pharmacy’s chemical biology and medicinal chemistry division. James’ work focuses on multiple myeloma, a cancer that forms in a type of white blood cell called a plasma cell and can damage the bones, immune system, kidneys and red blood cells. The American Cancer Society estimates that approximately 32,000 people in the U.S. will be diagnosed with multiple myeloma in 2020 alone.
“The funding from Deerfield has brought us closer to thinking about how what we do in the lab can be truly translational and ultimately have an impact on human health,” said James. “Deerfield has assembled an outstanding team of scientists who are invested in the project, provide unique insights and want to help the project succeed. Working in this collaborative environment has been extremely fruitful and rewarding.”
Before projects like James’ begin working as part of Pinnacle Hill, they are reviewed and approved by a joint steering committee comprised of members from UNC-Chapel Hill and Deerfield leadership teams. Projects selected for support through Pinnacle Hill receive a development plan complete with targeted milestones. They also receive funding to support further research across the UNC-Chapel Hill campus. If projects generate profits, these will be shared by UNC-Chapel Hill and Deerfield.
At the intersection of academia and industry
It’s this kind of academic-industry collaboration that draws enthusiasm from Michael Kline, who works closely with Collins on Pinnacle Hill initiatives and serves as the director of outreach and technology development in the UNC Office of Technology Commercialization.
“This is a unique and special opportunity for faculty to advance their therapeutic research programs to patients. From day one, Deerfield Management has been a true partner and we have all enjoyed working closely with them to identify, evaluate and advance opportunities at UNC-Chapel Hill,” said Kline. “They not only bring financial resources, but also a breadth of knowledge of therapeutic development and the evolving investment and market landscapes. I am excited to see projects advance and new ones come on board.”
Core to Pinnacle Hill’s success is creating opportunities for ongoing interactions and opportunities to learn about emerging research being pursued by UNC-Chapel Hill faculty. Collins and Kline continually engage with faculty to understand their research and identify any potential fits within the Pinnacle Hill model. Senior leaders from Deerfield regularly hold group information sessions with Carolina faculty to provide updates on opportunities with Pinnacle Hill, while also offering targeted talks led by external industry experts on a variety of topics, such as a session on antibody therapy development held in 2019.
Beyond the large-group sessions, senior leaders at Deerfield also schedule more personal, one-on-one discussions with Carolina faculty to gain an in-depth understanding of their specific research projects and technologies. By doing so, the Pinnacle Hill team can continually gain the most up-to-date insights on emerging research and curate potential projects that may soon be ready to submit an application.
Angelman syndrome advancements
Finding new ways to bridge the scientific expertise of Carolina researchers and the product development know-how of Deerfield is key for approaching complicated, multi-faceted diseases like Angelman syndrome. Developing and bringing a new medicine to Angelman’s patients is the goal of Ben Philpot, Kenan Distinguished Professor and associate director of the UNC Neuroscience Center in the UNC School of Medicine. Philpot’s research on the disease became the second project funded by Pinnacle Hill.
Announced in June 2020, Philpot’s work on Angelman syndrome in conjunction with Pinnacle Hill represents new hope for the more than 15,000 people in the U.S. and 500,000 people worldwide suffering from the disease. The symptoms that affect children with the syndrome – such as problems with intellectual development, speech, movement and balance – continue to affect these individuals throughout their lives. Genetic therapies explored by Philpot and team signal hope for a treatment.
“Our lab has made great strides in identifying small molecules that could potentially treat Angelman syndrome, but we reached a stage where medicinal chemistry was clearly needed to optimize compounds. Thus, we could not be more thrilled to partner with Pinnacle Hill and Deerfield Management, a team with proven expertise in this area,” said Philpot. “The Pinnacle Hill team is working intimately with my lab, as well as with the labs of Jeff Aube at the Eshelman School of Pharmacy and Stormy Chamberlain at the University of Connecticut, to gain deeper mechanistic insights into candidate compounds and to help advance these safely towards eventual therapeutic application. While we are still in the early stages of this partnership, it is clear that we have chosen the best team to shepherd us towards a potentially transformative small molecule treatment for Angelman syndrome.”
As Philpot’s work on Angelman’s syndrome and James’ research on multiple myeloma move ahead, Pinnacle Hill looks forward to not only supporting these critical initiatives but also continuing to meet with faculty and working with senior leaders in order to identify the next round of Pinnacle Hill-funded projects. Carolina faculty who are interested in learning about Pinnacle Hill are encouraged to reach out directly to Jon Collins and Michael Kline for more information.
“Given the research I have seen thus far, and the many researchers who I have yet to meet, I am highly confident that there are many more ongoing projects on campus which could be funded through Pinnacle Hill,” Collins said.