Genentech, a member of the Roche Group, announced today that new data will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting from May 4-10 in Philadelphia, PA. Presentations include data from a pivotal study for risdiplam in spinal muscular atrophy (SMA), which has the potential to become the first oral treatment for this community.
Risdiplam is an investigational oral survival motor neuron 2 (SMN2) splicing modifier for SMA, which is designed to increase and sustain SMN protein levels in the central nervous system (CNS) and throughout the body. Platform presentations include one-year data from the dose-finding Part 1 of the pivotal FIREFISH study on key motor milestones, motor function and survival in infants with Type 1 SMA. Data show infants with Type 1 spinal muscular atrophy achieve key motor milestones and improved survival after one year of treatment. Updated safety, tolerability and pharmacokinetics / pharmacodynamics (PK/PD) data, as well as an exploratory efficacy analysis from Part 1 of the pivotal SUNFISH study in people aged 2-25 years with SMA Types 2 or 3 will also be presented. Risdiplam data from Part 1 of pivotal FIREFISH study show infants with Type 1 spinal muscular atrophy achieve key motor milestones and improved survival after one year of treatment.
Risdiplam is an investigational, oral medicine that is systemically distributed and designed to increase SMN protein levels in the central nervous system (CNS) and throughout the body. It is designed to help the SMN2 gene produce more functional SMN protein, to better support motor neurons and muscle function. Roche and Genentech are leading the clinical development of risdiplam in collaboration with the SMA Foundation and PTC Therapeutics. Risdiplam is currently being evaluated in three multicenter trials in people with SMA:
- FIREFISH – an open-label trial in infants aged one to seven months with Type 1 SMA
- SUNFISH – a double-blind, placebo-controlled trial in children and young adults (two to 25 years old) with Type 2 and 3 SMA
- JEWELFISH – an open-label exploratory trial in people aged 12–60 with Type 2 or 3 SMA who have been previously treated with SMN-targeting therapy as part of a clinical study
A new trial, RAINBOWFISH in pre-symptomatic SMA, was initiated in early 2019.
FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose escalation study in 21 infants. The primary objective of Part 1 was to assess the safety profile of risdiplam in infants and determine the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in approximately 40 infants with Type 1 SMA for 24 months, followed by an open-label extension. The primary objective of Part 2 is to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds).
SUNFISH is a two-part double-blind, placebo-controlled pivotal clinical trial in children and young adults (two to 25 years old) with Type 2 and 3 SMA. Part 1 determined the dose for the confirmatory Part 2, and enrollment in Part 2 completed in September 2018.