CERC-006

CERC-006 is a dual mTOR inhibitor (a class of drugs that inhibit the mammalian target of rapamycin)

CERC-006

Targeting Complex Lymphatic Malformations

CERC-006 is targeted towards complex Lymphatic Malformations (LM).  Lymphatic Malformation patients often have activating mutations along the PI3K/AKT/mTOR pathway and sirolimus is an mTORC1 inhibitor that has demonstrated clinical utility in LM. CERC-006 has the potential to improve upon both the safety and efficacy of mTOR inhibition in LM. Currently the main therapeutic options for treating a lymphatic malformation are percutaneous drainage, surgery, sclerotherapy, laser therapy, radiofrequency ablation, or medical therapy. Cerecor seeks to initiate a Phase 1b/2a proof-of-concept study of CERC-006 in LM patients in late 2020. 

Attributes
of CERC-006

  • High potency, dual mTOR inhibitor maximizing impact of mTOR blockade with potential for improved efficacy and tolerability relative to sirolimus
  • Active against a broad panel of tumor cell lines, including models resistant to rapalogs
  • Evaluated in Phase 1 study; good PK and significantly lower doses than MTD likely required to treat Lymphatic Malformations

About Complex Lymphatic Malformations

Lymphatic malformations are rare, non-malignant masses consisting of fluid-filled channels or spaces thought to be caused by the abnormal development of the lymphatic system. These malformations are usually apparent at birth or by two years of age. Lymphatic malformations can affect any area of the body (except the brain), but most commonly affect the head and neck. When evident at birth (congenital), lymphatic malformations tend to be soft, spongy, non-tender masses. The specific symptoms and severity of lymphatic malformations varies based upon the size and specific location of the malformation. Some lymphatic malformations can be massive. Lymphatic malformations, regardless of size, can potentially cause functional impairment of nearby structures or organs and disfigurement of affected areas.