Type of information: Milestone
Compound: products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors
Company: RegenXBio (USA - MD) Avexis (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: spinal muscular atrophy
- • On April 1, 2014, RegenX Bio and AveXis announced that they have entered into an exclusive agreement for the development and commercialization of products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors. Under the terms of the agreement, RegenX granted AveXis an exclusive, worldwide license, with rights to sublicense, to Regenx’s NAV rAAV9 vector for treatment of SMA disease in humans. In return for these rights, RegenX receives an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV9. “AveXis is committed to the development of new treatments for patients with SMA using NAV-vector technology. We feel rAAV9 is the most promising vector to achieve this goal. We call it our ‘special snowflake’, because, AAV9 has unique properties that allow us to develop novel targeted treatments for infants with SMA. We’ve named the product chariSMA® from Greek origin meaning ‘a gift of grace’,” said John A. Carbona, CEO of AveXis.
- • On June 11, 2018 , RegenXBio announced that it has received an accelerated license payment of $100 million under its license agreement with AveXis, for the development and commercialization of products to treat spinal muscular atrophy (SMA), due to the acquisition of AveXis by Novartis.
- The accelerated license payment consisted of $60 million in annual fees and a commercial milestone fee of $40 million. Under the License Agreement, in addition to the accelerated license payment, RegenXBio remains eligible to receive a potential commercial milestone fee of $80 million and certain royalties on net sales for any product developed for the treatment of SMA using RegenXBio's NAV technology.
- Novartis now holds exclusive rights to the NAV Technology Platform for the development of treatments for SMA, including AVXS-101, which uses RegenXBio's NAV AAV9 vector. 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.
- • On January 8, 2018, RegenXBio and AveXis announced that they have entered into an amendment which expands upon the exclusive, worldwide license agreement they entered into in March 2014 (2014 License Agreement) for the development and commercialization of products to treat spinal muscular atrophy (SMA). AveXis’ initial proprietary gene therapy candidate, AVXS-101, is in a pivotal trial for the treatment of SMA Type 1, and a Phase 1 trial for SMA Type 2. AVXS-101 uses RegenXBio’s NAV AAV9 vector.
- Under the terms of the amendment, RegenXBio granted AveXis exclusive, worldwide rights to all vectors in RegenXBio’s NAV Technology Platform for the treatment of SMA in addition to adding and amending certain terms of the 2014 License Agreement, including the modification of the assignment provision to now permit assignment in the event of a change of control by AveXis without RegenXBio’s consent. RegenXBio will receive, in addition to the payments and royalties owed under the original 2014 License Agreement, an upfront payment of $80 million, an additional payment of $30 million after one year and an additional payment of $30 million after two years, and RegenXBio is eligible to receive potential commercial milestone payments of up to $120 million. For any product developed for the treatment of SMA using the NAV AAV9 vector, RegenXBio will continue to receive mid-single to low double-digit royalties on net sales as defined in the original 2014 License Agreement, and for any product developed for the treatment of SMA using a NAV vector, other than NAV AAV9, RegenXBio will receive a low double-digit royalty on net sales.