SEC Filings

AVEXIS, INC. filed this Form 10-K on 02/28/2018
Entire Document

We are required to use commercially reasonable efforts to research, develop, commercialize and sell AskBio licensed products for the treatment of SMA in humans throughout the term of the AskBio License. We do not have the right to control prosecution of the in‑licensed patent applications, and AskBio has the sole right, but not the obligation, to enforce the in‑licensed patents. In addition, our rights under the AskBio License are not assignable without the prior written consent of AskBio.

Unless terminated earlier, the AskBio License automatically expires on the date on which we no longer have any payment obligation under the AskBio License. The AskBio License may be terminated prior to its expiration:


by us upon six months advance written notice to AskBio;


by either party upon the other party’s breach of the AskBio License that is not cured within the specified cure period based on the nature of such breach;


by either party in the event of either party’s bankruptcy, insolvency or certain similar occurrences;


by AskBio if, during a specified period of time and subject to certain conditions being met, we research, develop or commercialize an AAV‑based treatment for hemophilia or we undergo a change of control with, or are otherwise acquired by, a third party that conducts such activities; or


by AskBio if we bring any action or proceeding challenging the validity or enforceability of any of the licensed patents.

As of December 31, 2017, we have paid AskBio the $1.0 million upfront license fee and $0.6 million for a clinical development milestone payment.

Preclinical Programs for Rett Syndrome and Genetic ALS


We have also entered into three license agreements relating to our planned new programs for Rett syndrome and genetic ALS.



Effective June 7, 2017, we entered into an exclusive license agreement with REGENXBIO under certain patents and patent applications owned by the Trustees of the University of Pennsylvania and licensed to REGENXBIO, for the development and commercialization of products to treat Rett syndrome and genetic ALS using the AAV9 vector, or the REGENXBIO Rett and ALS License. Under the REGENXBIO Rett and ALS License, REGENXBIO granted us an exclusive, worldwide license under the licensed patent rights to make, have made, use, import, sell and offer for sale any products covered by the REGENXBIO Rett and ALS License, or the REGENXBIO Rett and ALS licensed products, in the field of the treatment of (i) Rett syndrome in humans by in vivo gene therapy using AAV9 delivering the gene encoding for methyl CpG binding protein 2, and (ii) ALS caused by SOD1 mutation in humans by in vivo gene therapy using AAV9 delivering the gene encoding for SOD1, subject to certain rights reserved by REGENXBIO and its licensors. The patent rights exclusively in-licensed include an issued United States patent, which expires in 2026. We have the right to sublicense the licensed technology to third parties subject to certain conditions as specified in the REGENXBIO Rett and ALS License. Under the REGENXBIO Rett and ALS License we grant a non-exclusive, worldwide, royalty-free, transferable, sublicenseable, irrevocable, perpetual license back to REGENXBIO to (a) use any patentable modifications and improvements to the licensed technology that we or our affiliates or sublicensees develop, or licensed back improvements, and (b) practice the licensed back improvements in connection with AAV9 outside of our fields of use.

 Under the terms of the REGENXBIO Rett and ALS License, we have paid or are required to pay:


an initial fee of $6.0 million;



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