SEC Filings

10-K
AVEXIS, INC. filed this Form 10-K on 02/28/2018
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Our Strategy

We are building a patient‑centric business with the goal of developing innovative gene therapy treatments that transform the lives of patients and their families suffering from rare and life‑threatening neurological genetic diseases. In order to accomplish this goal, we plan to execute on the following key strategies:

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Rapidly advance our SMA Type 1 program through clinical trials in the United States.  We are conducting a pivotal trial of AVXS-101 in SMA Type 1 using the IV formulation produced by our good manufacturing practice, or GMP, commercial manufacturing process. Five patients have been dosed in this clinical trial to date. In January 2017, we announced the completion of our Phase 1 clinical trial of AVXS‑101 in patients with SMA Type 1, in which we observed a favorable safety profile and that AVXS-101 was generally well-tolerated. As of August 7, 2017, all patients in the trial survived event-free at 20 months of age, in contrast to the 8% event-free rate at 20 months demonstrated in an independent, peer-reviewed natural history study of patients with SMA Type 1. The FDA has granted AVXS‑101 orphan drug designation for the treatment of all types of SMA, fast track designation for the treatment of SMA Type 1 and breakthrough therapy designation for the treatment of SMA Type 1 in pediatric patients. We intend to request a pre-BLA meeting for AVXS-101 for SMA Type 1 with the FDA in the second quarter of 2018.

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Further expand the development of AVXS‑101 for the treatment of SMA.  Based on preclinical data and our preliminary clinical observations to date, we believe AVXS‑101 may also have the ability to treat patients with SMA Types 2, 3 and 4, which result from the same genetic defect as SMA Type 1. We are conducting a Phase 1 safety and dosing escalation study of AVXS‑101 via IT delivery in patients with SMA Type 2. Two patients have been dosed in this clinical trial to date. Furthermore, we intend to initiate a trial for patients with two, three and four copies of the SMN2 backup gene, who are less than six weeks of age and pre-symptomatic at the time of gene therapy, to evaluate appropriate clinical endpoints of a one-time IV dose of AVXS-101 in the first half of 2018. We also intend to initiate a pediatric "all comers" trial for approximately 50 patients between approximately six months and 18 years of age who do not qualify for other AVXS-101 trials at the time of gene therapy, to evaluate a one-time IT dose of AVXS-101 in the late fourth quarter of 2018 or early 2019.

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Advance the development of AVXS‑101 outside of the United States.  The incidence, standard of care and prognosis of SMA globally are generally consistent. Despite a treatment being approved in Europe during 2017, we believe there is significant unmet need for patients suffering from SMA outside the United States. In January 2017, we announced that the EMA granted AVXS‑101 access into its PRIME program for the treatment of SMA Type 1. We intend to initiate our European pivotal trial of AVXS‑101 for SMA Type 1 during the first half of 2018. In February 2017, we announced that this pivotal trial will reflect a single‑arm design, using natural history of the disease as a comparator, and will enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy.

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Build a pipeline of gene therapy treatments for other rare and life‑threatening neurological genetic diseases, including Rett syndrome and genetic ALS.  In addition to our programs in SMA, we also intend to identify, acquire, develop and commercialize novel product candidates for the treatment of other rare and life‑threatening neurological genetic diseases that we believe can be treated with gene therapy. We intend to employ a targeted approach to acquisition and licensing transactions reflecting our goal to be a leading gene therapy company focused on the treatment of rare and life‑threatening neurological diseases. In June 2017, we announced that we had entered into an exclusive, worldwide license agreement with REGENXBIO for the development and commercialization of gene therapy using the recombinant AAV9 vector to treat Rett syndrome and genetic ALS.  We expect to move forward with initiating investigational new drug application, or IND, enabling studies in both Rett syndrome and genetic ALS and submit IND applications for both indications in late 2018 or early 2019.

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Continue to invest in and develop robust and sustainable manufacturing processes, as well as multiple supply sources, to ensure the supply of high‑quality products.  We have built our own commercial scale

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