HEALTHCARE
HEALTHCARE
Advancing Therapies for Rare and Devastating Diseases
Biotech CEO is Keynote Speaker at the 96th Annual CIANJ Luncheon
To understand how Princeton , New Jerseybased Amicus Therapeutics ( NASDAQ Symbol : FOLD ) started requires a bit of an explanation ; a story so unique that it even inspired a Hollywood movie .
“ My introduction to rare disease was more than 25 years ago as a concerned friend ,” relates Bradley Campbell , President and CEO of Amicus Therapeutics . “ My friend John and his wife Aileen had two children born with a rare and fatal condition known as Pompe disease and I wanted to help them raise money to fund research . My involvement really started from a personal place out of concern for the diagnosis of John ’ s two kids .”
Named after Joannes Cassianus Pompe in 1932 , the ailment is a form of muscular dystrophy that causes the accumulation of glycogen in muscle tissue . Patients with Pompe disease develop weak skeletal muscles and life-threatening enlarged organs . At the time John Crowley ’ s children were diagnosed , no treatment existed and his children were not expected to live past the age of five .
“ Over time , Pompe disease robs patients of the ability to walk and to breathe on their own ,” says Campbell . “ In the 1990s , Pompe was often a fatal disease because there were no treatments available .” Campbell first met Crowley when the young Campbell served as a consultant following his undergraduate studies at Duke University .
Determined to save his children , Crowley began prolific fundraising efforts , and in 1999 , he co-founded a biotech startup called Novazyme to find a treatment for Pompe . Novazyme ’ s promising research into an enzyme replacement treatment ( ERT ) for Pompe led to its acquisition by Genzyme in 2001 . A couple of years later , the first-ever treatment for Pompe disease received FDA approval and saved the Crowley children ’ s lives . Crowley ’ s remarkable journey sparked the creation of the Hollywood film , Extraordinary Measures , starring Brendan Frasier and Harrison Ford in 2010 .
Crowley ’ s search for a treatment did not end there . In 2005 , he co-founded Amicus Therapeutics to focus on finding treatments for Pompe and other rare diseases , a company Bradley Campbell leads with the same entrepreneurial and patient-focused spirit .
“ John had this vision of building the next great biotechnology company specifically focused on rare diseases with the idea that we could be the most patient-centric company in the space ,” notes Campbell . “ By keeping patients at the center of what we do , we could improve our efforts at creating medicines for those patients .”
Campbell was asked to join Amicus in 2006 with that patient-centric vision in mind . Seventeen years later , he has taken over the role of President and Chief Executive Officer , having also served as Chief Operating Officer of the company . Crowley now currently serves as Executive Chairman .
Campbell ’ s leadership role comes at a watershed moment in the company ’ s success . “ We are about to enter the second chapter of our journey as we bring a second therapy to patients .
“ This is an evolution , not a revolution ,” he says . “ In my mind , the next chapter requires that we continue to expand access to our medicines while maintaining our focus as passionate entrepreneurs . We must continue to drive performance while staying true to our patient-centric mission . We have to keep patients at the center of what we do .”
With this milestone , the company has become a more important developer and player in this space ,” Campbell explains .
A Real-Life New Jersey Incubator Success Story
Amicus ’ first home was an incubator space provided by the NJEDA on Route 1 . “ We are a bornand-raised-in-New Jersey story ,” jokes Campbell .
The company ’ s first technology , developed out of Mount Sinai School of Medicine , is a pharmacological chaperone . “ Chaperone technology led to our first medicine , Galafold ® for treating Fabry Disease ,” Campbell explains .
Bradley Campbell , President and CEO of Amicus Therapeutics .
“ Our second therapy is the first two-component therapy approved for Pompe disease .”
Next-Level Thinking
Campbell is quick to acknowledge his respect for Amicus ’ founder and how his relationship and the mentorship he received from Crowley shaped his future . “ John has handed me the baton to bring us to the next chapter of our growth ,” he shares .
Campbell ’ s leadership style is inclusive , yet decisive . “ This is an evolution , not a revolution ,” he says . “ In my mind , the next chapter requires that we continue to expand access to our medicines while maintaining our focus as passionate entrepreneurs . We must continue to drive performance while staying true to our patient-centric mission . We have to keep patients at the center of what we do .”
With the transition from an early start-up to a successful , multiproduct firm , Amicus is now positioned to earn what Campbell calls the “ right to do more .” This means having the ability to self-fund future development investments with revenue from the current product lineup .
Continued On Page 8
Photo : Russ DeSantis Photography and Video
6 COMMERCE www . commercemagnj . com