Roche’s lead compound to treat spinal muscular atrophy (SMA) is no more. The pharmaceutical company has sent a letter to the SMA community informing them that they will no longer be developing olesoxime to treat SMA.  As they state in the letter, 18 month data from the phase 2/3 OLEOS study showed a worsening of motor function in SMA patients receiving olesoxime.

Olesoxime is a neuroprotective agent that was purchased by Roche in 2015 from Tophos. To date, it has proven to not be effective in helping patients with either amyotrophic lateral sclerosis nor SMA.

While this is a devastating blow to the SMA community, Roche is more hopeful about their compound RG7916, a SMN2 splicing modifier that is currently being evaluated in 3 clinical trials.

SMA is a rare neurological disorder due to a defect in the SMN1 gene that results in a reduction in SMN protein. That leads to a loss of motor neurons in the spinal cord and lower brain stem. Children with the most severe form of SMA (SMA type 1) seldom live past their 2nd birthday while those with SMA type 2 or 3 are often confined to a wheelchair.

Currently, there is only 1 drug approved to treat SMA patients and that is Spinraza, an antisense oligonucleotide that alters a second, normally silent gene, SMN2, to produce functional SMN protein.

In Canada, access to Spinraza is limited to an extremely small portion of the SMA population and more treatment options are desperately needed for these patients. Until those treatment options become available, Canadians are urged to sign a petition by CureSMA Canada that would allow Spinraza to be made available to all eligible SMA patients.