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AveXis Announce Additional Trials of AVXS-101 for SMA

25 January 2018

AveXis have provided an overview of their expanded program of clinical development for their gene therapy drug AVXS-101 in SMA, which incorporates two new trials to include a broader set of SMA patients.

AVXS-101, which has “Breakthrough Therapy” status to facilitate the drug approval process (for more information click here), uses harmless, genetically engineered viruses to increase SMN protein levels. A Phase I trial of AVXS-101 with SMA Type 1 patients has recently been completed and showed that the therapy is safe, well tolerated, and able to improve and help sustain muscle function in young infants with the condition (for more information click here).

Following this successful Phase I trial, two pivotal trials of AVXS-101 were initiated in the US and EU, known as STR1VE and STR1VE EU, respectively (for further information, click here and here). Recently, a fourth clinical study called STRONG was initiated with SMA Type 2 patients in the US (for more information click here).

AveXis are now planning to start two additional clinical trials of AVXS-101: SPRINT will assess the safety and effectiveness of the drug in pre-symptomatic infants, and REACH will involve SMA patients under 18 years of age ineligible for the other four ongoing and planned trials.

The AveXis clinical development program now includes the following five trials:

SPRINT, planned for pre-symptomatic SMA Types 1, 2, and 3

Expected to include about 44 patients, this multi-national trial will assess the safety of AVXS-101 and its impact on developmental milestones and survival. Enrolled patients will be less than six weeks of age, have two to four copies of SMN2, and, importantly, must not yet show any signs of SMA. This trial is similar in nature to the NURTURE trial of nusinersen, in that it will test the impact of treatment in pre-symptomatic newborns that are very likely to develop SMA based on their genetics. AVXS-101 will be given as a single administration into the blood, and dosing is expected to begin in the first half of 2018.

REACH, planned for SMA Types 1, 2, and 3

This trial will be conducted internationally and enroll approximately 50 patients aged between six months and 18 years. Similar to the EMBRACE trial of nusinersen, REACH has been designed to trial AVXS-101 in patients who do not qualify for the other AVXS-101 studies. This trial will test administration of AVXS-101 directly into the spinal cord fluid, and is likely to start at the end of 2018 or early in 2019.

STR1VE EU, Phase 3 pivotal trial planned for SMA Type 1 in Europe

Including about 30 infants (<6 months old) with SMA Type 1 and testing administration into the bloodstream, this trial will be conducted in Europe and will assess the safety and efficacy of AVXS-101 (Click here for more information). STR1VE EU is to begin in the first half of 2018. It is currently unclear whether the STR1VE EU trial will be enrolling patients from the UK. We would advise regularly checking the website for the most up-to-date news.

STR1VE, on-going Phase 3 pivotal trial for SMA Type 1 in the US

Similar to STR1VE EU in design, STR1VE will include a minimum of 15 patients in the US, and has enrolled three patients to date (Click here for more information).

STRONG, on-going Phase I trial for SMA Type 2 in the US

Designed to assess safety and provide information on efficacy of one-time injections of AVXS-101 into the spinal cord fluid, STRONG will enroll about 27 patients with SMA Type 2. Two different doses of AVXS-101 will be tested, and the impact of the drug will be assessed against historical data of SMA Type 2 progression, rather than a placebo control (Click here for more information). The first patient has been dosed in this study.

Further Information

AveXis Press Release

STR1VE clinical trial page

STRONG clinical trial page

AveXis community statement of the trials