GABAB PAM for the treatment of CMT1A.
Our license agreement with Indivior provides for a funded research program, under which we have the right to select drug candidates for exclusive development in certain indications outside of addiction, including CMT1A, a rare disease indication.
We expect to deliver drug candidates by end of 2020.
We believe an oral small molecule GABAB PAM with a once-a-day dosing and without the adherence-limiting side effects of baclofen, which is currently used off label, could bring significant benefit to patients and consequently present a strong commercial opportunity for us.
We also plan to pursue orphan drug designation for a selected drug candidate for CMT1A.