BLU-782, an Investigational Selective ALK2 Inhibitor

Presenter: Alison J. Davis, Ph.D. | Senior Scientist II, Blueprint Medicines

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling and ultimately life-shortening disease characterized by progressive replacement of skeletal muscle and connective tissue by heterotopic bone. FOP is caused by gain-of-function mutants of the activin receptor-like kinase 2 (ALK2) protein, the most common of which is ALK2R206H. BLU-782 is a potent and selective ALK2R206H inhibitor developed by Blueprint Medicines for the prevention of heterotopic ossification (HO) in FOP.

We used an ALK2R206H transgenic mouse model of FOP, coupled with linear MR and mCT imaging of the HO process, to document the course of disease and to determine the efficacy, mechanism of action and optimal dosing paradigm of BLU-782 to prevent HO. These data support the development of BLU-782 as a prophylactic, oral, once-daily therapeutic for people with FOP.

During this webinar, the presenter will:

Show BLU-782 is a potent and selective inhibitor of ALK2R206H, the causative mutation in FOP
Reveal how BLU-782 prevents HO in two different ALK2R206H injury models
Explain how ALK2R206H disease activity begins early after injury and is sustained throughout the HO process, suggesting chronic dosing of BLU-782 would be most efficacious for preventing HO in FOP patients

Dr. Davis is a Senior Scientist in the Biology Department at Blueprint Medicines and is currently the biology lead on three different preclinical and clinical programs. In this role, she collaborates across disciplines with biologists, chemists, pharmacologists, clinicians and regulatory experts to first test kinase inhibitors in relevant in vitro and in vivo disease models and then drive the best drug candidates into the clinic.

Dr. Davis holds a Ph.D. from the Johns Hopkins University School of Medicine with a focus in cell biology. She was an Assistant Professor at The Tufts University School of Medicine where she studied infection mechanisms of bacterial pathogens. In her tenure at Blueprint Medicines, she has contributed to four successful Investigational New Drug applications to the FDA, and recently celebrated the approval of her first FDA-approved drug, AYVAKIT.

Dr. Davis is a dedicated drug hunter using her skills to get the best therapeutics to patients, balancing urgency and thoughtfulness.

BLU-782, an Investigational Selective ALK2 Inhibitor in Development for Fibrodysplasia Ossificans Progressiva (FOP)

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ALISON J. DAVIS, PH.D.

Senior Scientist II, Blueprint Medicines