AveXis Inc(NASDAQ:AVXS) stock lost over 9.9% on Jan 16th, 2018 (As of 1:07PM EST; Source: Google finance) as the group intends to offer and sell, up to $400 million of shares of its common stock in an underwritten public offering.
The group expanded their clinical development program for their initial gene therapy candidate, AVXS-101, for treating spinal muscular atrophy (SMA). Moreover, with the ongoing pivotal trial in SMA Type 1 (STR1VE) and the ongoing Phase 1 trial in SMA Type 2 (STRONG), AveXis intends to start three studies to further evaluate AVXS-101, including in new SMA patient populations. Moreover, the first patient has been dosed in the Phase 1 trial of AVXS-101 in SMA Type 2. AveXis sees the potential of this to be a major clinical progress as they continue towards their ultimate goal of bringing AVXS-101 to the patients and families devastated by SMA.
The group made an amendment with REGENXBIO Inc. (Nasdaq:RGNX) to expand upon the exclusive, worldwide license agreement they entered into in March 2014 (2014 License Agreement) for developing and commercialization of products to treat spinal muscular atrophy (SMA). This shows the group’s commitment to develop and commercialize novel gene therapy treatments for SMA, and serves as a further validation of their NAV Technology Platform and its potential utility in this therapeutic area.
REGENXBIO granted AveXis exclusive, with worldwide rights to all vectors in REGENXBIO’s NAV Technology Platform for treating SMA, REGENXBIO would get royalties owed under the original 2014 License Agreement, an upfront payment of $80 million, and a further payment of $30 million after one year and another $30 million after two years. REGENXBIO would also get potential commercial milestone payments of up to $120 million. REGENXBIO would also get mid-single to low double-digit royalties on net sales and for any product developed for treating SMA using a NAV vector, other than NAV AAV9, REGENXBIO would get a low double-digit royalty on net sales.
The group is positive over using the NAV Technology Platform from REGENXBIO in infants with SMA Type 1 and expects results in their initiated Phase 1 trial in SMA Type 2. The expanded partnership with REGENXBIO, would offer exclusive rights to the entire REGENXBIO NAV Technology Platform, boosting position in gene therapy treatments for this devastating disease.