GABAB PAM for CMT1A

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 have started the clinical candidate selection phase and expect IND enabling studies to be initiated in 2022. 

Subject to regulatory approval, 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. 

Walso plan to pursue orphan drug designation for a selected drug candidate for CMT1A.