AveXis presented other research at AAN that furthered the scientific understanding of AVXS-101:
· Dr. Brian Kaspar, Senior Vice President and Chief Scientific Officer of AveXis, presented data from a dose-response study for the cerebrospinal fluid delivery of AVXS-101 that offers insight into vector distribution and its correlation with transgene expression, providing guidance for future AAV9-based clinical trials in SMA, as well as other neurodegenerative disorders.
· Dr. Douglas Sproule, Vice President of Clinical Development and Medical Affairs of AveXis, presented data suggesting pre-existing antibodies to AAV9 are quite uncommon in the pediatric population and should not impact use of gene therapy for the vast majority of SMA Type 1 patients.
· Dr. Linda Lowes, Director of Clinical Therapies Research and a member of the Center for Gene Therapy at the Research Institute of Nationwide Childrens Hospital, presented research providing further evidence of the correlation between motor function and motor milestone achievement in patients with SMA Type 1, demonstrating that the degree of treatment outcome appears to be influenced by age at dosing and baseline motor function.
Announced Exclusive Worldwide License Agreement for Two Rare Neurological Monogenic Disorders Using NAV AAV9 Vector: On June 7, AveXis and REGENXBIO Inc. announced an exclusive worldwide license agreement for AveXis to develop and commercialize gene therapy treatments using REGENXBIOs NAV AAV9 vector to treat Rett syndrome and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 gene. AveXis intends to provide more details on these programs in the second half of 2017.
Announced Alignment with U.S. Food and Drug Administration (FDA) on Good Manufacturing Practice (GMP) Commercial Manufacturing Process: On June 14, AveXis announced alignment with the FDA regarding the process for producing the intended commercial scale GMP-derived gene therapy product, following the receipt of minutes from the Type B Chemistry Manufacturing and Controls meeting held on May 1. This alignment includes support for the proposed commercial manufacturing process, the proposed analytical methods and corresponding qualification and validation plans inclusive of key release assays such as potency, purity and identity and the proposed comparability protocol, which helps assess how similar the product derived from the GMP process is to the original product used in the Phase 1 trial of AVXS-101 in patients with SMA Type 1.
· In the meeting minutes, the FDA made a request that the company complete implementation of its potency assay qualification plan, presented in the meeting, prior to initiation of upcoming clinical studies. This assay utilizes the Delta 7 mouse model, which has been used historically to assess AVXS-101 potency but now incorporates additional elements to make it acceptable to global regulatory authorities. The company has already initiated the work necessary to address this request and expects to have the data ready to submit to the FDA in August.
· AveXis plans to initiate a pivotal study trial of AVXS-101 in SMA Type 1 in the U.S. and a Phase 1/2a trial of AVXS-101 in SMA Type 2 in the U.S. later in the third quarter of 2017, pending agreement from the FDA that these data are sufficient. AveXis expects to provide an update once the company has received FDA agreement that these data are sufficient to initiate the studies. The company also expects to provide design details for these studies at that time.
· Additionally, FDA is aligned with the companys proposed comparability protocol to assess the similarity of key characteristics of the Nationwide Childrens Hospital product, used in the Phase 1 SMA Type 1 study, with the product derived from the new GMP manufacturing process. Data from this comparability work is ongoing and will include the above-mentioned potency