Shire to acquire Lumena Pharmaceuticals

Shire adds to rare disease portfolio with acquisition of LumenaPharmaceuticals, bringing late stage compounds for rare GI/hepatic conditions * Acquisition of Lumena Pharmaceuticals, a biopharmaceutical company with late stage rare disease pipeline assets * Adds to Shire's rare diseases portfolio and leverages this expertise, and is a perfect combination with Shire's already strong Gastrointestinal (GI) presence * Adds LUM001 in Phase 2 development for four rare and devastating hepatic diseases, two pediatric and two adult with a potential 2016 approval and LUM002 a Phase 2-ready candidate for the treatment of non-alcoholic steatohepatitis (NASH) * Very attractive opportunity to develop treatments for significant unmet need in rare cholestatic liver diseases as well as a treatment for non-alcoholic steatohepatitis (NASH) * Shire will acquire Lumena Pharmaceuticals for an upfront payment of $260 million in cash, plus a payment for net cash at closing, and near-term contingent milestone payments related to ongoing clinical trials * These two compounds, and Shire's full portfolio, will be discussed in more detail at an Investor Day later in 2014. Dublin, Ireland and San Diego, U.S. - May 12th, 2014 - Shire plc (LSE: SHP,NASDAQ: SHPG) and Lumena Pharmaceuticals, Inc., a biopharmaceutical companywith rare disease pipeline assets, announce the acquisition of LumenaPharmaceuticals by Shire. Chief Executive of Shire, Flemming Ornskov, MD comments: "Our pipeline and strategic focus on rare diseases is even further strengthenedwith the acquisition of Lumena Pharmaceuticals, which also complements ourstrong GI presence. These attractive potential treatments may offer new hope topatients with rare cholestatic liver disease and further contribute to Shire'sfuture growth. We are excited by the possibilities of these new assets in liverdisease. We have the resources, the infrastructure and the operating capacityto invest in these new potential growth drivers which add further value toShire's innovative pipeline." President and CEO of Lumena Pharmaceuticals, Mike Grey comments: "I believe that this transaction is a significant win for all parties involved,especially the patients, and the future of LUM001 as a treatment for rarecholestatic liver diseases looks brighter than ever. Shire has deep raredisease experience, a global infrastructure, and the commercial expertise todeliver LUM001 to patients around the world. The Lumena team will work closelywith Shire to finish the ongoing Phase 2 clinical programs as part of ourcommitment to the patient populations we have championed since we formed LumenaPharmaceuticals." Strategic rationale and background on Lumena Pharmaceuticals The acquisition of Lumena strengthens Shire's already valuable and robustpipeline. It complements Shire's strategic focus on Rare Diseases and providesa future growth path for Shire's Gastrointestinal business, which generatedrevenues of over $800 million in 2013. In acquiring Lumena, Shire is gainingexperience in liver disease with the opportunity to leverage its existing GIcommercial infrastructure. In addition, there is a good fit with Shire's recentacquisition of Fibrotech, which has brought pipeline programs to address unmetpatient need in other fibrotic conditions including renal impairment. Lumena Pharmaceuticals brings to Shire two new oral therapeutic compounds;LUM001, in Phase 2 with four potential orphan indications and LUM002, ready toenter Phase 2 later in 2014. LUM001 and LUM002 are both inhibitors of the apical sodium-dependent bile acidtransporter (ASBT), which is primarily responsible for recycling bile acidsfrom the intestine to the liver. Blocking bile acid transport with ASBTinhibitors reduces bile acid absorption and has the potential to improve liverfunction and relieve disease symptoms (such as extreme itching associated withcholestatic liver diseases), and may slow disease progression. These rare cholestatic liver diseases are primarily treated by hepatologistsand gastroenterologists and can be covered by a small, specialty sales forceconsistent with Shire's model. Shire does not expect the acquisition of Lumena to result in a change to itspreviously stated earnings guidance for 2014. About LUM001 LUM001 is a novel, once-daily, orally-administered, potent and selective ASBTinhibitor that works by preventing recycling of bile acids back to the liverand is thought to reduce bile acid accumulation, improve liver function andpotentially relieve the extreme itching associated with cholestatic liverdisease. LUM001 is currently in Phase 2 clinical development for four rare cholestaticliver disease indications; two pediatric and two adult with a potential 2016launch. These potential indications are: Alagille syndrome (ALGS), progressivefamilial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC) andprimary sclerosing cholangitis (PSC). Some of the key characteristics of cholestatic liver diseases are elevated bileacids, leading to progressive liver damage that can cause liver failure, andpruritus, or severe itching. Pruritus is generally the most debilitatingsymptom afflicting children and adults with these diseases. Surgical intervention, which lowers bile acid levels, has been shown to relievesymptoms and slow disease progression in patients with ALGS and PFIC - this iscurrently the only treatment option for these patients. Patients withcholestatic liver diseases may ultimately require liver transplants. By reducing serum bile acids, LUM001 may offer a novel therapeutic approach foralleviating the pruritus and progressive liver damage associated withcholestatic liver diseases. The prevalence of each of the four diseases is as follows: Indication US prevalence EU prevalence ALGS 3 per 100,000 3 per 100,000 9476 individuals PFIC