Dallas, Texas-based AveXis Inc. has established a license agreement with Asklepios Biopharmaceutical Inc. in an effort to advance a potential treatment for spinal muscular atrophy (SMA). The agreement gives AveXis rights to a self-complementary (SC) technology from AskBio called Duplex vectors.
AveXis aims to use the novel technology in its phase 1 Adeno-Associated Virus (AAV) gene transfer clinical trial that includes patients who suffer from the hereditary disease of the motor neurons known as SMA. “AveXis is committed to the development of novel treatments for patients with SMA,” explained AveXis’ scientific founder Brian K. Kaspar, PhD, in a press release.
“The self-complementary technology allows for the rapid production of the survival motor neuron (SMN) protein which is critical for SMA patients where motor neuron dysfunction and death along with muscle weakness progresses rapidly after the initial onset of symptoms. We believe this technology is a key component to the promising data we have seen thus far and we are pleased to establish this agreement with AskBio,” he added.
The terms of the agreement determine that AveXis will hold non-exclusive access to the Duplex vectors and may use them in the development and commercialization efforts of its SMA therapy. The specific financial terms of the deal were not disclosed by the companies, but it is known that AskBio will be granted an upfront payment, as well as future payments according to development and commercial milestones, and potential royalty to the products.
“AskBio’s duplex vectors have a long-standing proven track record of demonstrating faster onset and higher levels of therapeutic gene expression. As an AAV platform technology AskBio’s self-complementary technology has been shown to be effective at decreasing dosing requirements. In turn this reduces manufacturing demands across a broad range of therapeutic areas,” said AskBio’s MPM vice president Jade Samulski.
“This transaction is further validation of the diverse applicability of another one of AskBio’s platform technologies and represents another opportunity to positively affect patient outcomes. We are pleased to be formally associated with an effort designed to provide potentially therapeutic results for such a vulnerable patient population and one in such obvious and significant need,” continued Samulski.
AskBio is focused on the development of gene-delivery technologies able to treat conditions of the central nervous system, while AveXis’ primary goal is to offer a novel therapy to SMA patients. The life-threatening disease causes the progressive weakening of the muscles and AveXis is currently working on the first human gene treatment in the world for pediatric SMA in its study chariSMA.
Last month, the company 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 SMA therapy. Mr. Carbona remained as a director of the company, as 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.