Lilly's JAK2 inhibitor is currently being evaluated in Phase I clinical trials for the treatment of myeloproliferative neoplasms, or diseases of the blood and bone marrow, called polycythemia vera, essential thrombocythemia and myelofibrosis.  

LY2940680: Halting Abnormal Signaling via the Hedgehog Pathway (Abstract # 2819)

Lilly scientists have identified an orally administered anti-cancer agent, LY2940680, which, in preclinical studies, disrupted the abnormal signaling of a key regulator of embryonic cell development. Data was also presented at AACR's New Small Molecule Therapeutics mini-symposium.  

This novel molecule has been shown to affect a cancer cell signaling pathway initiated by the Hedgehog (Hh) protein, which is essential for regulating normal cell differentiation and proliferation.  Abnormal Hh signaling has been implicated in several types of cancer, including brain, lung, breast, prostate and skin cancers.  Abnormal Hh pathway activation can result from genetic mutations in the pathway's protein components.

One such protein component, known as Smoothened (Smo), is a key regulator of the Hh signaling pathway and is seen as a potential target for therapies aimed at treating cancer by halting abnormal Hh signaling.

Lilly scientists have identified that Lilly's Hedgehog inhibitor is a small molecule antagonist of Smo, binding to it and inhibiting Hh signaling in a human medulloblastoma tumor cell.  (Medulloblastoma is the most commonly diagnosed form of brain tumor in children, but is rare in adults.(1))  In addition, when the compound was orally administered to transgenic mice that spontaneously develop medulloblastoma, it improved animal survival.

Lilly's Hedgehog inhibitor is currently being studied in Phase I clinical trials for solid tumors.

SOURCE Eli Lilly and Company