Safe­ty qualms in MyoKar­dia mid-stage study cause in­vestors to pause

Ahead of the piv­otal da­ta read­out of MyoKar­dia’s lead drug, mava­camten, in pa­tients with ob­struc­tive hy­per­trophic car­diomy­opa­thy (HCM), in­vestors took is­sue with the drug’s safe­ty pro­file in a sep­a­rate Phase II non-ob­struc­tive HCM study.

Mava­camten is ex­pect­ed to break new ground in heart dis­ease — a field mo­nop­o­lized by large phar­ma­ceu­ti­cal com­pa­nies large­ly due to the long, ar­du­ous and ex­pen­sive tri­als that are com­mon­place in heart drug de­vel­op­ment. Un­like oth­er de­vel­op­ers fo­cus­ing on com­mon heart dis­or­ders, MyoKar­dia’s treat­ment is al­so tar­get­ing a so-far un­tapped dis­ease — hy­per­trophic car­diomy­opa­thy — a ge­net­ic con­di­tion in which heart mus­cle thick­ens, mak­ing it hard­er for the or­gan to pump blood to the rest of the body.

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