Roche Announces Some Results from their Firefish and Sunfish Trials

09 October 2018

The Firefish Trial

This is one in a series of trials being performed by Roche, testing out this oral (by mouth) treatment ‘risdiplam’ aimed at increasing production of ‘survival of the motor neuron’ protein (SMN), which is deficient in individuals with SMA.

The medication was given to infants with Type 1 SMA (non-sitters), and it was discovered that these individuals achieved more developmental milestones.  Roche revealed that after eight months of treatment, out of 14 children who had received treatment:

  • 6 were able to sit; three of these without support.
  • 10 achieved full or partial head control (without treatment, the neck muscles are usually weak in infants with SMA Type 1)
  • 4 could roll to the side.

The results from physiotherapy assessment tools which monitor the ability to perform tasks (CHOP INTEND) increased positively over time. At the point of writing the press release, the vast majority of participants (19 out of a total of 21) were still alive, with three of these now older than 2 years of age.

These studies also look to see how many participants have ‘significant events’, such as needing help with breathing (ventilation). 90% of participants treated with the medication had no such events, which is much better than in other studies looking at untreated participants in a group of similar infants where the rate was 50%.

The Sunfish Trial

This study is broken into two parts and is with individuals with SMA Type 2 or 3, ages from 2 - 24 years. The results of the first part of the trial in which 30 participants were treated for one year, showed that the levels of SMN protein in the blood after 12 months were twice as high as before treatment.

Participants motor function was measured and showed an improvement of 3.1 points on the scale used, though the authors don’t state if this was significant. Comparing different age groups (less than 12 years old versus older than 12 years), slightly more improvement was seen in the younger group, but the study found improvements in both.

Although participants were found to have several ‘adverse events’, the studies didn’t find any ‘significant adverse events’ related definitively to the drug.

You can read more detail about the results in Roche’s media release here.