Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA) - Seite 2

Results also showed that treatment with risdiplam led to a median two-fold increase in blood SMN protein levels after four weeks, which was sustained for at least 24 months. This is consistent with previously reported results through 12 months of treatment. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons, which transmit movement signals from the central nervous system to the muscles.

These results are consistent with the results of the pivotal Part 2 of the trial at 12 months in non-ambulatory patients which demonstrated that change from baseline in total MFM32 score was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156).

The most common adverse events in Part 1 of the SUNFISH study were fever (pyrexia; 55%), cough (35%), vomiting (33%), upper respiratory tract infections (31%), cold (nasopharyngitis; 24%) and sore throat (oropharyngeal pain; 22%). The most common serious adverse event that occurred in three of the 51 patients exposed to risdiplam was pneumonia. To date there have been no treatment-related safety findings leading to withdrawal.

Enrolment for the JEWELFISH study, assessing safety and pharmacodynamic data in previously treated patients with SMA, who are now receiving risdiplam, is complete (n=174). Among the patients who completed 12 months of treatment with risdiplam, a median two-fold increase in SMN protein versus baseline was observed (n=18). An early assessment of safety showed a consistent safety profile compared to treatment-naive patients.

Of the 174 patients enrolled, 76 were previously treated with nusinersen and 14 with onasemnogene abeparvovec. The remaining 83 patients had been treated with compounds then being developed by Roche.

The most common adverse events were upper respiratory tract infections (13%), headache (12%), fever (8%), diarrhea (8%), nasopharyngitis (7%) and nausea (7%). To date there have been no drug-related safety findings leading to withdrawal from the JEWELFISH trial and the overall adverse event profile is similar to that observed in risdiplam trials of patients  not previously treated with a SMA-targeting therapy.

Risdiplam’s comprehensive clinical trial programme (birth to 60 years old) was designed to represent the broad, real-world spectrum of people living with this disease with the aim of ensuring access for all appropriate patients. Roche leads the clinical development of risdiplam, an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for SMA, as part of a collaboration with the SMA Foundation and PTC Therapeutics.