AveXis has provided the following community statement on AVXS-101.
Dear SMA Community,
At AveXis, the gene replacement therapy company developing a new approach to treat SMA known as AVXS-101, we recently announced our plans to start three new clinical trials (STR1VE EU, SPRINT, REACH) in addition to our ongoing clinical trials (STR1VE, STRONG).
The goal of these planned trials is to enhance our understanding of how well AVXS-101 works and more importantly, its safety profile in a broad range of people with SMA. The designs are still being finalized and may change over time. Therefore, we do not have all of the details available yet — including inclusion/exclusion criteria, such as whether or not a patient is eligible if they have previously used another therapy. This level of detail will be made available for each study when it opens for enrollment. If you have questions about our studies in the meantime, please contact us at [email protected].
We are eager to learn more about AVXS-101 through these studies, as it will assist in potentially making AVXS-101 available to as many SMA patients as safely and quickly as possible. That has always been our driving purpose!
Sincerely,
The AveXis Team
Overview of AVXS-101 Clinical Development Program
Ongoing AVXS-101 Studies
- OVERVIEW: STR1VE is an ongoing study of AVXS-101 in patients with SMA Type 1, studied at multiple centers across the U.S. which began enrolling in September 2017.
- ADMINSTRATION: In STR1VE, AVXS-101 is given one-time through a needle inserted into a vien, known as an intravenous (IV) infusion.
- WHO: STR1VE will enroll a minimum of 15 patients with SMA Type 1 who are less than six months of age at the time of gene therapy administration, and who have one or two copies of the SMN2 backup gene and bi-allelic SMN1 gene deletion or point mutations.
- OVERVIEW: STRONG is an ongoing study of AVXS-101 in patients with SMA Type 2, studied at multiple centers across the U.S. which began enrolling in December 2017.
- ADMINISTRATION: In STRONG, AVXS-101 is given one time through what is known as an intrathecal (IT) injection near the lower end of the spinal cord. STRONG is the first time AVXS-101 is being administered this way. Data from STRONG will help us understand how to best design the planned study, REACH.
- WHO: STRONG will enroll 27 infants and children who are symptomatic with a generic diagnosis consistent with SMA, including the bi-allelic deletion of SMN1 and three copies of SMN2 without the SMN2 genetic modifier who are greater than 6 months and less than 5 years old. Patients enrolled in STRONG must be able to sit but cannot stand or walk, and cannot have previously been able to stand or walk.
Planned Studies
STR1VE EU
- OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centers across the European Union.
- ADMINISTRATION: In STR1VE EU, AVXS-101 is administered through a one-time IV infusion.
- WHO: STR1VE EU will enroll approximately 30 patients with SMA Type 1 who are less than six months of age at the time of gene therapy administration.
SPRINT
- OVERVIEW: SPRINT is a multi-national trial expected to start enrolling patients in the first half of 2018 in pre-symptomatic patients with SMA Types 1, 2 and 3.
- ADMINISTRATION: In SPRINT, AVXS-101 is administered through a one-time IV infusion.
- WHO: SPRINT is expected to enroll approximately 44 patients with two, three and four copies of SMN2 who are less than six weeks of age and pre-symptomatic at the time of genetherapy administration.
REACH
- OVERVIEW: REACH is a multi-national trial expected to start enrolling patients late in 2018 or early 2019 in SMA Types 1, 2 and 3.
- ADMINISTRATION: In REACH, AVXS-101 is administered through a one-time IT injection. Data from STRONG (the first study of AVXS-101 delivered through IT injection) will help determine the final study design.
- WHO: REACH is expected to enroll approximately 50 patients with SMA Types 1, 2 and 3 who are between approximately six months and 18 years of age.
FREQUENTLY ASKED QUESTIONS
1. Will the REACH trial include patients who have previously or are currently taking nusinersen?
- The trial design for REACH has not yet been finalized, including whether or not a patient will be eligible for the trial if they have previously or at the time of study been on nusinersen
- Since the STRONG study is the first time that we will be giving AVXS-101 through intrathecal injection, we will use the data from STRONG to help us understand how best to design REACH.
- The full design details for REACH will be available when the study begins enrolling patients in late 2018 or early 2019.
- For detailed information about clinical trials and the clinical trial process, download Cure SMA’s care series booklet, Learning About Clinical Trials.
- It is important to speak to your healthcare team about your care or your child’s care, including all treatment decisions.
2. What about the older kids with SMA?
- AveXis is eager to study AVXS-101 in older children, and the REACH trial will include patients between approximately six months and 18 years of age.
3. What about adults with SMA?
- With the addition of these new trials, AveXis will now be able to evaluate the impact of AVXS-101 in a broader group of people with SMA. We understand the urgency for all patients with SMA, and continue to evaluate the potential of expanding our clinical development program into adults. Data from these planned and ongoing studies will help us understand how to best design future trials.
4. When will AVXS-101 be approved by the FDA?
- While we cannot speak on behalf of the FDA regarding whether they will decide to approve AVXS-101 and when, the goal of the ongoing and planned studies is to enhance our understanding of how well AVXS-101 works and more importantly, its safety profile in a broad range of people with SMA. The FDA then uses the information collected in these clinical studies to help determine whether AVXS-101 should be approved, and, if so, in what patients.
5. Who should I contact to see if my child is eligible for these studies?
- For more information on the ongoing studies, STR1VE and STRONG, please visit StudySMANow.com.
- While the trial design for the planned studies have not yet been finalized, if you have any questions regarding STR1VE EU, SPRINT or REACH, please contact us at [email protected].