Page last updated: 10th September 2024

2024

10th September: Biogen reports topline results from the DEVOTE Study

11th March: Biogen Report Biomarker Data from the RESPOND Study.

2023

6th July: Biogen present latest results from NURTURE.

30th June: Biogen present an update on the RESPOND Trial.

2022

23rd June: Biogen present updates on the RESPOND and DEVOTE trials.

2021

17th September: Biogen announces plans to initiate ASCEND trial.

22nd April: Biogen presents new data from its DEVOTE trial.

11th January: First patient treated in RESPOND study.

2020

21st July: Biogen announces plans to initiate RESPOND study

12th June: Biogen announces further positive results in NURTURE study

6th April: First patient treated in DEVOTE trial

2019

24th October: Biogen confirms recent results in NURTURE study to the SMA Europe Community

10th May: Biogen announces positive results in NURTURE trial

21st May: Phase 1 and 2 Studies for Later-Onset SMA Type 2 and 3 show positive results

2017

15th December: Summary and comparison of results to date for nusinersen and gene therapy published – read more.

15th November: Updates on the trials presented at the 22nd International Annual Congress of the World Muscle Society (WMS) October 3rd-7th 2017: Poster 1 – CHERISH; Poster 2 – EMBRACE; Poster 3 – ENDEAR

25th April: Updates on the CHERISH and NURTURE clinical trials presented at the annual American Academy of Neurology meeting (April 22nd-28th)

2016

23rd December: The U.S. Food and Drug Administration approved Spinraza (nusinersen) – the first drug approved to treat children and adults with spinal muscular atrophy (SMA)

15th December:

• Nusinersen Phase 2 trial results (ENDEAR) published in the Lancet.
• Commentary on the Lancet article by Tom Gillingwater.
• Stanford News article.

31st October: FDA accepts nusinersen for priority review

11th May: Ionis Pharmaceuticals released updated information on EMBRACE.

31st January: Biogen and Ionis Pharmaceuticals provide updates on trials with nusinersen – also now known as Ionis-SMNRx and ASO-10-27 and previously known as ISIS-SMNRX – ENDEAR; CHERISH; NURTURE; EMBRACE; SHINE.

2015

23rd October: ISIS Pharmaceuticals initiates SHINE study

16th June: ISIS Pharmaceuticals announces update on its ongoing Phase 11 study showing that ISIS-SMNRx continues to be well tolerated and appears to be having a positive effect on the disease progression.

2014

14th October: at the 19th International World Muscle Society Congress, ISIS Pharmaceuticals released detailed positive results from their Phase II clinical trials with infants with SMA Type 1 and other children with SMA type II

8th August: ISIS Pharmaceuticals announces beginning of Phase 111 study

2012

7th November: Isis Pharmaceuticals started a Phase Ib/IIa clinical trial of ISIS-SMNRx in 24 children with SMA (ages 2-15)

5th January: Biogen Idec entered a global agreement with ISIS Pharmaceuticals to develop and commercialise their compound ISIS-SMNRx.

2011

9th December: ISIS Pharmaceuticals had recently begun a Phase I clinical trial of their antisense oligonucleotide drug ISIS-SMNRx in medically stable SMA patients between 2-14 years of age.

The US Food and Drug Administration (FDA) had granted Orphan Drug Status with Fast Track Status to ISIS-SMNRx for the treatment of SMA patients.

Injection of a similar drug was safe and well tolerated by patients with a type of adult-onset motor neuron disease called amyotrophic lateral sclerosis (ALS), which is positive for the ISIS-SMNRx Phase I trial.

In the reported study, ISIS-SMNRx administration in SMA mice caused an increase in SMN protein levels, which in turn led to increased survival, reduced motor neuron loss and behavioural improvements. Furthermore, in a separate study, intrathecal administration of ISIS-SMNRx into cynomolgus monkeys resulted in putative therapeutic drug levels throughput the spinal cord (a key area of the body in the disease).

ASOs were used in mice to show that it is likely that SMN protein levels will need to be restored throughout the body, and not just the nervous system, in order to have the best chances of successfully treating the disease in patients.