Biogen and Ionis Pharmaceuticals published new data on Monday regarding its experimental treatment for the rarest from of spinal muscular atrophy.
The drug, Spinraza, achieved the primary endpoint in a Phase 3 study evaluating its effects on Type 2 SMA. Volunteers running the trial saw a statistically significant improvement in motor function for patients taking the drug when compared to those on placebo, according to Xconomy.
Type 2 SMA is a rare genetic condition caused by a defective version of the SMN1 gene, which doesn’t produce enough of the SMN protein to keep muscles strong. Spinraza is an RNA-based drug that seeks to boost production of this protein leading to an improvement in patient’s motor function.
“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, M.D., Ph.D., executive vice president, head of Biogen’s Research and Development department, in a statement. “We will make regulators around the globe aware of this data and will continue working closely with them to bring SPINRAZA to families affected by SMA as quickly as possible.”
Biogen and Ionis will end this trial and provide participants with the option of switching over to an open-label extension study so they can all gain access to the drug, wrote Xconomy.
Currently, there are no approved drugs for SMA, but this data could help both companies make a compelling case. Biogen is planning for a potential launch of Spinraza in the U.S. either at the end of the year or the beginning of 2017.