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Biogen and Ionis Pharmaceuticals Provide Update on Nusinersen (Formerly Known as ISIS-SMNRx)

31 January 2016

Biogen and Ionis Pharmaceuticals (previously Isis Pharmaceuticals) have provided an update on the clinical development of their antisense oligonucleotide drug Nusinersen, which used to be known as Isis-SMNRx.

Nusinersen, which is also now known as Ionis-SMNRx and ASO-10-27, works by targeting the backup Survival Motor Neuron 2 (SMN2) gene and coaxing it to produce more of the SMN protein that is lacking in SMA patients (click here for more information). Very encouraging effects of the gene therapy have been reported in mouse models of SMA, and Nusinersen is consequently being tested in a number of different clinical trials:

ENDEAR is the first Phase III trial of Ionis-SMNRx initiated in very young infants with SMA (click here for more information).

CHERISH is the second Phase III trial of the drug, which aims to test the safety and effectiveness of Nusinersen in approximately 120 children (aged 2-12) with symptom onset at or later than six months (click here for more information).

NURTURE is a Phase II trial that aims to evaluate the safety and tolerability of the drug in about 25 pre-symptomatic newborns that are very likely to develop SMA based on their genetics.

EMBRACE is a Phase II trial designed to assess the safety and exploratory efficacy of Ionis-SMNRx in approximately 20 young SMA patients not able to participate in the current Phase III trials.

SHINE is an open-label, Phase III study designed to extend the treatment regime of patients who have completed the ENDEAR or CHERISH trials (click here for more information).

In a letter to the SMA community published on the Cure SMA website, Biogen and Ionis provided up-to-date information on the status of enrolment for each of these trials.

The Phase III trials, ENDEAR and CHERISH, are reported to be on track for completion in the first half of 2017, with target patient enrolment already accomplished for CHERISH, and expected to be achieved within six months for ENDEAR.

The NURTURE Phase II trial has enrolled more than 33% of the intended number of patients, EMBRACE is fully enrolled in the USA, and SHINE has only just started.

Biogen and Ionis report that they are pursuing additional regulatory avenues to expedite Nusinersen development. In line with this, they are hoping to be able to initiate what they call an early access program for the drug and end the use of placebo treatment in its testing, which will hopefully speed up the process of bringing the potential therapy to SMA patients.

Further Information

Biogen and Ionis letter

Ionis Prese release on CHERISH trial