Genentech/Roche announced today that new data on its approved and investigational medicines for neurological conditions will be presented during the 70th American Academy of Neurology (AAN) Annual Meeting from April 21-27 in Los Angeles, California. The data will include investigational research from Genentech on drugs in development for spinal muscular atrophy (SMA). The SMA presentations include late-breaking interim data on the increase in survival of motor neuron (SMN) protein levels following treatment with RG7916 in infants with Type 1 SMA as well as data regarding the clinical trial of Olesoxime.
RG7916 is an investigational oral SMN2 splicing modifiers being studied in three ongoing clinical studies, FIREFISH, SUNFISH and JEWELFISH. RG7916 is being developed in collaboration with PTC Therapeutics and the SMA Foundation.
The following three presentations will be given regarding the development of RG7916:
- RG7916 Significantly Increases SMN Protein in SMA Type 1 Babies
- Updated Pharmacodynamic and Safety Data from SUNFISH Part 1, a Study Evaluating the Oral SMN2 Splicing Modifier RG7916 in Patients with Type 2 or 3 Spinal Muscular Atrophy
- Preliminary Evidence for Pharmacodynamics Effects of RG7916 in JEWELFISH, a Study in Patients with Spinal Muscular Atrophy Who Previously Participated in a Study with Another SMN2-Splicing Targeting Therapy
In addition to the updates about RG7916, Roche/Genentech will present data from their clinical trial of Olesoxime in a presentation titled, “A Long-term, Open-label Follow-up Study of Olesoxime in Patients with Type 2 or Non-ambulatory Type 3 Spinal Muscular Atrophy Who Participated in a Placebo-controlled Phase 2 Trial”. Olesoxime supports the function of mitochondria, the power houses of cells, therefore supporting the continued function of cells.
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