Santhera Pharmaceuticals (SIX: SANN) today notes topline results from a two-part Phase 1 clinical study of AGAMREE® conducted by its North American licensing partner, Catalyst Pharmaceuticals, Inc. (“Catalyst”). The study suggests that AGAMREE® delivers glucocorticoid and anti-inflammatory activity, while avoiding significant immunosuppressive effects, supporting its potential use as a treatment across a broad range of chronic inflammatory rare diseases.
The study, conducted in healthy adult volunteers, evaluated equipotency between deflazacort and AGAMREE® and assessed the clinical immunosuppressive potential of AGAMREE® across ascending doses. In part A, both AGAMREE® and deflazacort demonstrated expected on-target glucocorticoid receptor activity and comparable cortisol suppression at clinical doses, with AGAMREE® showing less pronounced immunosuppressive biomarker effects, consistent with the currently labelled dosing of AGAMREE® in the treatment of Duchenne muscular dystrophy (DMD). Part B demonstrated that clinically relevant immunosuppressive effects were observed only at the highest dose level, above currently approved AGAMREE® dosing. No relevant immunosuppressive effects were observed at lower dose levels.
Under an exclusive license agreement entered into in 2023, Catalyst holds commercialization rights to AGAMREE® in North America for DMD and all potential future indications, while Santhera retains a right of first negotiation for any rights outside North America relating to new indications. Under the terms of the license agreement, Santhera is eligible to receive sales-based milestone payments as well as royalties on net sales across all commercialized indications