Rafael Pharmaceutical’s first-in-class clinical lead compound, CPI-613, targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. CPI-613 is being evaluated in multiple Phase I, I/II, and II clinical studies as a single agent, as well as in combination with standard drug therapies, in patients diagnosed with advanced solid tumors or blood cancers. The . Food and Drug Administration (FDA) has given Cornerstone approval to initiate pivotal clinical trials in pancreatic cancer and acute myeloid leukemia (AML), and has designated CPI-613 an orphan drug for the treatment of pancreatic cancer, AML, and myelodysplastic syndromes (MDS).
On 20 June 2014, Shire rejected a takeover attempt by AbbVie . AbbVie offered £ per share (£ billion or $ billion in total).  On 8 July the offer was increased to $ billion.  On 18 July, it was announced that AbbVie would acquire Shire for $ billion.  On 15 October, news broke suggesting AbbVie was reconsidering their proposed takeover deal due to changes in US "Tax Inversion" law  and on 16 October AbbVie's board recommended that shareholders vote against the deal.  This news sent Shire's share price down over 27%, however AbbVie will be subject to a $ billion break up fee, payable to Shire.  On 21 October the merger was called off.