Roche (SIX:ROG; OTCQX:RHHBY) said it will discontinue development of spinal muscular atrophy candidate olesoxime (RG6083, TRO19622). In April, Roche presented data at the American Academy of Neurology meeting in Los Angeles from the long-term follow-up OLEOS study showing that the candidate led to a worsening in motor function.
The pharma also cited in its reasoning difficulties in formulating olesoxime and identifying an appropriate dose, as well as changes in the SMA treatment landscape.
Roche has another SMA candidate, RG7916, in the Phase II JEWELFISH and Phase II/III SUNFISH trials to treat adults and children with SMA types 2 and 3, and the Phase II/III FIREFISH trial to treat infants with SMA type 1. It is an oral survival of motor neuron 2 centromeric (SMN2) splicing modifier.
Olesoxime is a small molecule with cholesterol-like structure that interacts with the mitochondrial permeability transition pore (mPTP). Roche gained the candidate when it acquired Trophos S.A. in 2015 (see BioCentury Extra, Jan. 16, 2015).