By Alex Keown
This morning Regenxbio said the payment was related to the development and commercialization of products to treat spinal muscular atrophy (SMA). The accelerated license payment consisted of $60 million in annual fees and a commercial milestone fee of $40 million. There is an additional potential $80 million waiting in the wings from potential commercial milestones and royalties. The payment came nearly six months after Regenxbio and AveXis strengthened their cooperative partnership in SMA.
In January the two companies amended the 2014 license agreement for Regenxbio’s gene therapy technology. The amended agreement gave AveXis (and now Novartis) exclusive, worldwide rights to all vectors in Regenxbio’s NAV Technology Platform for the treatment of SMA. Regenxbio’s NAV Technology Platform is a proprietary adeno-associated virus (AAV) gene delivery platform.
In January AveXis announced it had expanded its clinical development for is initial SMA gene therapy candidate, AVXS-101. ). In addition to the ongoing pivotal trial in SMA Type 1 (STR1VE), which is the most severe form of the disease, and the ongoing Phase I trial in SMA Type 2 (STRONG), the company plans to initiate three studies to further evaluate AVXS-101, including in new SMA patient populations. In April 2018, AveXis reported that the six SMA patients who were at least one-month post gene transfer in the pivotal trial for AVXS-101 were exhibiting motor function improvements.
AVXS-101 has received Orphan Drug designation as well as Breakthrough Therapy designation. Novartis is already eying a potential filing for regulatory approval of AVX-101. The companies expect to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the second half of this year, with U.S. launch sometime in 2019, if approved.
If AVX-101 is approved, it would take on Biogen’s Spinraza, the only approved treatment for SMA in the United States. Spinal muscular atrophy is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for the survival of motor neurons. According to the Muscular Dystrophy Association, “babies born with the most severe type of SMA — Type 1 — are not able to sit up unsupported and the majority don’t survive beyond the age of two years.” SMA appears in about one out of every 60,000 babies, the MDA said on its website.
For AveXis April was a pivotal month. Not only was it acquired by Novartis for $8.7 billion, the company released strong data from its Phase I trial in Type 1 SMA. Following a two-year post gene therapy treatment, all patients in the therapeutic dose cohort in the Phase 1 trial were alive and event-free. Patients observed in the long-term follow-up continued to gain strength. Two of the patients were able to stand with assistance, AveXis said. Also in April AveXis announced the first patient dosed in its Phase III trial in pre-symptomatic patients with spinal muscular atrophy Types 1, 2 and 3. The SPRINT trial is expected to enroll approximately 44 pre-symptomatic patients with a bi-allelic deletion of SMN1; two, three or four copies of SMN2; and who are less than six weeks of age at the time of gene therapy.