Genentech Roche provided the following community statement with clinical trial updates for SUNFISH (Type 2/3), FIREFISH (Type 1) and JEWELFISH.
Dear SMA community,
At Genentech (A Member of the Roche Group) we are committed to addressing the urgent needs of people living with SMA. We are happy to share this update on our two investigational oral molecules in development. olesoxime and RG7916, and the three trials with RG7916 that are currently recruiting- SUNFISH (Type 2/3), FIREFISH (Type 1) and JEWELFISH (non-na”ive Type 2/3).
Olesoxime
- Olesoxime is an orally administered compound that may maintain mitochondrial function and support the continued function of cells
- The European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have recommended that we generate additional benefit/risk data in a Phase Ill study
- We are considering EMA and FDA feedback on potential plans for a Phase Ill study with olesoxime and will keep you updated in the coming months
RG7916
- RG7916 is an SMN2 splicing modifier that is taken orally and distributes widely throughout the whole body
- It is in clinical development in collaboration with PTC Therapeutics and the SMA Foundation
- RG7916 has received Orphan Drug Designation from the FDA
What clinical trials with RG7916 are currently recruiting?
Three clinical trials with RG7916 are currently recruiting participants: SUNFISH, FIREFISH and JEWELFISH. Potential participants would need to meet all the inclusion criteria before enrollment in any RG7916 tria1.
SUNFISH
Aim: To assess the safety and efficacy of RG7916 in patients with Type 2 or 3 SMA.
- Part 1 will assess how safe and well tolerated RG7916 is at 2 different dose levels
- Part 2 will assess the efficacy and safety of RG7916 at the dose selected from Part 1
Who could enroll? Children and young adults (2-25 years old) with Type 2 or 3 SMA.
Current status: Planned enrollment is complete for Part 1 Dose level 1 (low dose) of RG7916 in 1o adults and adolescents (Group A). and in 9 children (Group B). Part 1 Dose level 2 (higher dose) is currently enrolling patients.
Safety review: The safety of study participants is a priority for us. As pre-planned in the study protocol, a Safety Monitoring Committee reviews all safety information from all SUNFISH participants. After reviewing the safety information from the lower dose given to group B in Part 1, the Committee endorsed SUNFISH to advance as planned.
Future development: Part 2 is expected to begin in the second half of 2017, including additional countries and sites.
FIREFISH
Aim: To assess the safety and efficacy of RG7916 in infants aged 1 to 7 months with Type 1 SMA.
- Part 1 will assess the safety and efficacy of RG7916 at 2 different dose levels
- Part 2 will assess the efficacy and safety of RG7916 at the dose selected from Part 1
Who could enroll? Infants aged 1 to 7 months with Type 1 SMA.
Current status: The first infant was dosed with RG7916 in December 2016, and the study is progressing in Europe. FIREFISH Part 1 will open in additional countries and sites, including the US, in the coming months.
Safety: The safety of study participants is a priority for us. As pre-planned in the study protocol, a Safety Monitoring Committee reviews all safety information from all FIREFISH participants
Future development: Part 2 is expected to begin in the second half of 2017, including additional countries and sites.
JEWELFISH
Aim: To assess the safety and tolerability of RG7916.
Who could enroll? Patients with SMA who have previously received an SMN2-targeting therapy (either as part of the MOONFISH study with RG7800 or a study with another SMN2 targeting therapy).
Current status: The first patient has received their first dose at a US site.
Safety: Safety of study participants is a priority for us. As pre-planned in the study protocol, a Safety Monitoring Committee reviews all safety information from all JEWELFISH participants
Future development: Further screening and enrollment of patients at sites across the US and Europe is planned.
How can I or my family find out how to participate in SUNFISH, FIREFISH or JEWELFISH?
Please contact your physician if you think you or a family member could be suitable for one of these trials.
You can also visit http://www.roche-sma-clinicaltrials.com to read more about our program.
Additionally, your local patient group may have more information and resources. For a list of organizations go to: http://www.smafoundation.org/about-sma/sma-organizations-worldwide.
How can I find out more?
You can read more about SUNFISH, FIREFISH and JEWELFISH at www.clinicaltrials.gov and https://www.clinicaltrialsregister.eu/.
We will continue to share updates and developments about our studies
If you have any questions, or would like to discuss this further, please contact [email protected].