Vertex halts phase 2b spinal injury trial for futility

Vertex has halted development of VX-210 after a phase 2b acute spinal cord injury trial failed an interim analysis. The lack of efficacy seen in the phase 2b is another, potentially terminal, blow to the prospects of the Rho inhibitor.

VX-210, formerly known as BA-210 and cethrin, has taken a circuitous route to this point. Work on the drug dates back to 2000 and Alseres Pharmaceuticals took it into the clinic in 2007. Alseres filed to run a phase 2b the following year but the study never got underway. Instead, Alseres returned the rights to the drug’s originator, BioAxone, which then tried to partner it with another company.   

Vertex became that company, eventually. In 2015, more than seven years after the drug entered the clinic, Vertex revealed it had paid BioAxone $10 million to enter into a license and collaboration agreement. The deal was tied to up to $90 million in milestones and fees.

The agreement got VX-210 moving again after years in stasis. Vertex filed to run a 150-patient phase 2b/3 trial early in 2016. Now, an interim analysis of that study has persuaded Vertex that its small bet on the Rho inhibitor won’t pay off. 

Vertex revealed the setback in a short statement in its third quarter results. The statement contains no data but what information there is paints VX-210 as a dud. While the safety results came in clean, the data monitoring board recommended stopping the trial early due to futility. That led Vertex to halt development of VX-210.

Where that leaves the asset is unclear. Vertex plans to discuss the next steps with BioAxone but, unless someone spots a big flaw in the trial, it is questionable whether anyone will be willing to put money into an old drug that failed in phase 2b.

If VX-210 has reached the end of the road, it will also deal a blow to the idea of targeting Rho to treat spinal cord injuries. The approach, like Novartis’ targeting of Nogo, rested on evidence that particular proteins stop nerves from regrowing. By blocking these proteins, molecules such as VX-210 were designed to facilitate nerve regrowth in people with damaged spinal cords.