Dallas, Texas-based biotech company AveXis Inc. has announced the resignation of its CEO John Carbona as part of a succession plan designed to take the company to the next phase of development for its lead experimental spinal muscular atrophy (SMA) therapy. Mr. Carbona will remain as a director of the company, as AveXis will continue to focus on the clinical development of its ChariSMA gene therapy.
The company’s Board of Directors is implementing a reformulation of the management team to enhance AveXis’ growth and accelerate the development of the clinical program that is expected to result in the eventual approval of its novel therapy for SMA. The current chairman of the Board, John D. Harkey, will become the interim Executive Chairman, as AveXis will start searching for a new CEO through an executive recruiting firm.
“We are fortunate to have had John lead our firm through a very dynamic growth period,” stated Harkey, Jr. “John has provided tremendous efforts in establishing AveXis as a premier gene therapy company both in the US and abroad. We would like to thank John for his tireless commitment to AveXis which has advanced the goal of helping families with children with SMA.”
In his time as CEO, Carbona presided over several advancements for AveXis, including the launch of its current, ongoing clinical trials for SMA Type 1 patients, securing of $10 million in new financing for the company, and the establishment of AveXis subsidiaries in Europe to facilitate the approval process with EMC. In a personal gesture, Carbona also launched the “Mo” The Monkey campaign to ensure every child in the AveXis clinical trial received a plush toy — a symbolic gesture of hope that was inspired by the former CEO’s late mother.
“I am proud of the team we built here in Dallas and the accomplishments to date at AveXis,” said Carbona. “I will forever be indebted to the SMA researchers around the world who helped AveXis and most importantly the families and children with SMA who supported AveXis and me in our infancy. I look forward to the continued growth and success of the company as I watch from a new vantage point.”
AveXis is committed to improving the lives of patients who suffer from severe genetic and orphan conditions, whose treatment has major unmet medical needs. The autosomal-recessive genetic disease SMA is the primary focus of the company, which is implementing the restructuration plan in its leadership to support the development of chariSMA, the first human gene treatment in the world for pediatric SMA.
“We are pleased to be in a position to recruit a world-class leadership team to build the company to the next level,” said Mr. Harkey. “We are very encouraged by the progress to date in developing our ChariSMA gene therapy program for SMA in collaboration with Nationwide Children’s Hospital and Ohio State University, and remain completely dedicated to advancing this new treatment as rapidly as possible.”
Last October, the company concluded dosage in the first cohort of their phase 1 clinical trial of chariSMA, which provides functional SMN genes through self-complementary AAV9. The non-randomized study began in April to evaluate both the safety and efficacy of the intravenous therapy, as the company expects to be able to improve survival rate and motor function of the dosed patients. Preclinical studies had already demonstrated its effectiveness in crossing the blood-brain-barrier and delivering the drug to motor neurons.