Rassegna Stampa Industrie Farmaceutiche del 11 Gennaio 2016

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1 Rassegna Stampa Industrie Farmaceutiche del 11 Gennaio 2016 ROCHE 07/01/2016 PROGNOSIS Study Published in The New England Journal of Medicine Reveals Innovative Roche Blood Test Can Be Used as a Predictive Tool for Preeclampsia Study results validate the clinical value of measuring sflt-1/plgf ratios in women with suspected preeclampsia,1 accounting for more than 4 out of every 10 maternal deaths each year globally. The PROGNOSIS study is the first multi-center, large-sample study to demonstrate the prognostic value of the Roche Elecsys preeclampsia test to predict the absence of preeclampsia for one week, and the development of preeclampsia within the subsequent four weeks in women with clinical suspicion of the syndrome. The addition of the sflt-1/plgf ratio measurement to proteinuria and blood pressure measures gives better prediction of preeclampsia, and can reduce hospitalization pre-diagnosis by 50%.3 The PROGNOSIS study has highlighted the clinical value of measuring sflt-1/plgf ratios in women with suspected preeclampsia. Roche today announced that the New England Journal of Medicine has published the results of PROGNOSIS, a groundbreaking clinical study demonstrating the prognostic value of the company's Elecsys sflt-1/plgf immunoassay ratio test in predicting which pregnant women are at highest risk of developing preeclampsia,1 one of the leading causes of death and complications for mothers and their unborn babies.2 "The emotional benefits of the test are very important: preeclampsia can develop quickly and symptoms can develop even in women who so far have had a healthy pregnancy," said Professor Harald Zeisler of the Department of Obstetrics and Gynecology at Medical University Vienna, Austria, and an investigator in the PROGNOSIS study. "If we can tell a patient with signs or symptoms that she has a low sflt-1/plgf value, and therefore a low risk of developing preeclampsia within short term, that's a big advantage. On the other hand, women with high sflt-1/plgf values can be referred to hospitals with neonatal and adult intensive care units, where they can receive the specialist care they need." As well as potentially saving lives, the more accurate diagnosis of preeclampsia may also have positive economic impacts on healthcare systems. In 2005, the average cost of preeclampsia, excluding normal delivery costs, was an estimated GBP 9,009 per pregnancy. With an estimated 8.5 million women affected by preeclampsia every year, the annual cost of preeclampsia worldwide is estimated to be GBP 76.6 billion (based on the 2005 estimate), representing a major financial burden.4 Clinical use of the sflt-1/plgf ratio to predict and diagnose preeclampsia could help reduce the associated health care costs, by cutting both inappropriate discharges and unnecessary hospitalizations. Routine use of the Roche preeclampsia test could reduce by 50% the number of women hospitalized prior to preeclampsia diagnosis, leading to a cost savings of approximately 400GBP per patient.3 "The PROGNOSIS study is the first to demonstrate that the Elecsys sflt-1/plgf immunoassay ratio can reliably rule out preeclampsia for one week," said Roland Diggelmann, Chief Operating Officer, Roche Diagnostics. "We're delighted to see the

2 results published in such a world-renowned journal as the New England Journal of Medicine, and confident the findings will have a positive impact on the prediction and clinical management of this serious medical condition. They also support our vision to bring medical value to patients, which will enable proactive disease management and better patient care." About PROGNOSIS The PROGNOSIS study has demonstrated that low ratios of the proteins sflt-1 and PlGF in the blood of women showing the signs and symptoms of preeclampsia can predict the absence of the condition within a period of one week (the rule-out claim). The data published today show that an sflt-1/plgf ratio of 38 and below can rule out the development of preeclampsia within the next week with a very high confidence level of 99.3%.1 Identifying women who are unlikely to develop preeclampsia in the short term will save them from the stress of monitoring and the disruption to their home life caused by a stay in hospital. PROGNOSIS also demonstrated that an sflt-1/plgf ratio greater than 38 may help predict whether women with suspected preeclampsia will develop the condition within four weeks (the rule-in claim),1 allowing doctors to identify at-risk patients who need close monitoring. These important new findings represent a step-change in the prediction of preeclampsia in the clinical setting, where the gold standard has traditionally relied on the measurement of proteinuria (protein in the urine) and blood pressure. Unfortunately, both are sub-optimal predictors of which women will develop preeclampsia and how the disease will progress.5 As a consequence, many women with signs and symptoms of the disease are unnecessarily admitted to hospital for intensive observation and monitoring, resulting in worry for them and their families, and additional costs to the health provider.... GILEAD 05/01/2016 Gilead Terminates Phase 2 Study of Simtuzumab in Patients With Idiopathic Pulmonary Fibrosis FOSTER CITY, Calif. --(BUSINESS WIRE)--Jan. 5, Gilead Sciences, Inc. (Nasdaq:GILD) today announced that the company is stopping its Phase 2 clinical study of the investigational monoclonal antibody simtuzumab among patients with idiopathic pulmonary fibrosis (IPF). This decision follows an analysis of unblinded efficacy and safety data by the study's Data Monitoring Committee (DMC), which recommended that the study be terminated early due to lack of efficacy. Gilead has also reviewed the data and determined the study has not shown evidence of a treatment benefit in the group of patients randomized to receive simtuzumab. Separately, Phase 2 studies of simtuzumab are ongoing in patients with non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC). The DMC for these studies also met and recommended the continuation of the studies, which have a 96-week endpoint. CTI BIOPHARMA 05/01/2016 CTI BioPharma And Baxalta Complete Submission Of New Drug Application For Pacritinib For Unmet Medical Need In

3 Myelofibrosis SEATTLE and BANNOCKBURN, Ill., Jan. 5, 2016 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxalta Incorporated (Baxalta) (NYSE: BXLT) today announced the completion of the rolling submission of the New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. CTI BioPharma and Baxalta are requesting U.S. marketing approval of pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/ L) a specific patient population for which there is an existing unmet medical need. The Companies are seeking accelerated approval and have requested a Priority Review of the application. Pacritinib is an investigational treatment being developed for patients with myelofibrosis regardless of their platelet counts. If approved, pacritinib would be the first JAK2 inhibitor indicated for the treatment of patients with myelofibrosis and baseline platelet counts of less <50,000/ L. "We are pleased to have completed the rolling submission and look forward to working with the FDA during the review process with the goal of bringing this important treatment to people living with myelofibrosis, including those with low platelet counts," said James Bianco, M.D., president and chief executive officer of CTI BioPharma. Myelofibrosis is a rare, but serious and life-threatening chronic leukemia that disrupts the normal production of blood cells and results in scarring of the bone marrow, limiting the ability to produce new blood cells and prompting the spleen and other organs to take over this function. The disease often leads to an enlarged spleen and lower than normal counts of blood cells including red blood cells and platelets, which are essential for blood clotting. "Pacritinib has the potential to change the treatment paradigm for people with intermediate and high-risk myelofibrosis, particularly those patients with cytopenias," said David Meek, executive vice president, president of Oncology at Baxalta. "Together with CTI BioPharma, we are continuing to develop this potential new treatment for more people in need around the world." About the Pacritinib NDA The NDA includes data from the PERSIST-1 Phase 3 trial as well as data from Phase 1 and 2 studies of pacritinib. Submission of an NDA after a single Phase 3 trial under accelerated approval, instead of waiting to complete two Phase 3 trials, could potentially reduce time to market by up to 14 months. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high-risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment emergent thrombocytopenia on another JAK2 therapy; or patients who are intolerant to or whose symptoms are not well controlled (or sub-optimally managed) on another JAK2 therapy. CTI BioPharma and Baxalta are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Baxalta will jointly commercialize pacritinib in the U.S. while Baxalta has exclusive commercialization rights for all indications outside the U.S. NOVO NORDISK 07/01/2016 Novo Nordisk files for regulatory approval of long-acting factor IX in the EU for the treatment of haemophilia B Bagsv rd, Denmark, 7 January Novo Nordisk today announced

4 the submission to the European Medicines Agency of the Marketing Authorisation Application for the approval of long-acting factor IX, nonacog beta pegol. Nonacog beta pegol is a glycopegylated recombinant factor IX with a significantly improved pharmacokinetic (PK) profile, developed for patients with haemophilia B. Novo Nordisk expects to file the Biologics License Application (BLA) for nonacog beta pegol to the US Food and Drug Administration during first half of The filing of nonacog beta pegol is based on the results from the paradigm clinical trial programme, which involved 115 patients with severe or moderately severe haemophilia B. Nonacog beta pegol was found to be efficacious in routine prophylaxis, treatment of bleeding episodes and surgery for adults, adolescents and children. Furthermore, nonacog beta pegol appeared to be well-tolerated and no safety concerns were identified. Compared to standard factor IX products, nonacog beta pegol has a five times longer half-life. Patients in the paradigm study achieved a higher level of factor IX in the blood despite less frequent dosing of nonacog beta pegol. In the phase 3 trials, once-weekly administration of 40 IU/kg nonacog beta pegol maintained factor IX activity levels above 15%, reduced the median annualised bleeding rate (ABR) to 1.0 and showed a potential to prevent bleeds in target joints. Furthermore, these patients reported an improvement in quality of life during the trial. "With the regulatory filing of our long-acting factor IX, patients with haemophilia B are one step closer to having a new treatment option" said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. "With its high factor activity level, less frequent dosing and very low ABRs, nonacog beta pegol has the potential to improve the quality of life for both patients and their families." About nonacog beta pegol Nonacog beta pegol is an extended half-life factor IX molecule intended for replacement therapy in patients with haemophilia B. Glycopegylation, the prolongation technology used for the half-life extension, is a novel approach in haemophilia B, already proven safe and efficacious in haemophilia A and other therapeutic areas. About the paradigm clinical programme The paradigm clinical trial programme for nonacog beta pegol enrolled children and adults with severe or moderately severe haemophilia B. A total of 115 previously treated patients with a total of more than 8,800 exposure days for up to 2.7 years of treatment with nonacog beta pegol. The paradigm 1 PK trial (16 people treated) - a single-dose escalation trial evaluating safety and PK of nonacog beta pegol compared with marketed recombinant and plasma-derived factor IX products. Nonacog beta pegol showed up to twofold increase in recovery, higher activity levels and a fivefold prolongation of half-life compared to existing treatment. The paradigm 2 pivotal trial (74 people treated) - a 52-week single-blinded randomised trial evaluating safety, efficacy and PK for adults and adolescents in routine prophylaxis and treatment of bleeds. When provided prophylactic at 40 IU/kg weekly, nonacog beta pegol appeared to have a safe and well-tolerated profile and showed a median annualised spontaneous bleeding rate of 0.0. Furthermore, 97% of breakthrough bleeds were treated successfully and 90% of target joints no longer classified as such. The paradigm 3 surgery trial (13 people treated) - a dedicated trial confirming safety and efficacy during and after major surgical procedures. In all patients, a single preoperative dose provided effective haemostatic coverage, and no patient required additional doses on the day of surgery. Additionally, three doses proved sufficient in maintaining haemostasis during the first two weeks following the procedure. The paradigm 4 extension trial (71 people treated) - a safety extension trial with longer-term exposure demonstrated a well-tolerated profile with no inhibitors or other safety signals identified. The paradigm 5 paediatric trial (25 people treated) - a 52-week single-arm trial evaluating once-weekly prophylaxis and treatment of bleeding episodes in previously treated children 1-12 years of age. Nonacog beta pegol appeared to have a safe profile, and all patients maintained mean factor activity levels

5 above 15% one week after dosing of 40 IU/kg and a median ABR of 0.0 and 2.0 for children aged 0-6 and 7-12 years old respectively. BAYER HEALTHCARE 08/01/2016 Bayer halves the price of its contraceptive implant Jadelle for women in developing countries Effective long-acting and reversible contraceptive method to support women in the world's poorest nations Berlin, January 8, 2016 Bayer will halve the price of its contraceptive implant Jadelle until 2023 for women living in poor countries around the world. According to the Reproductive Health Supply Coalition the demand for implants is increasing continuously in these countries. Bayer's "Jadelle Access Program" aims to make this underutilized method more affordable and accessible to women there, ultimately helping them to expand their choice of contraceptive options. "Our Jadelle Access Program' widens the contraceptive options available to women living in poor countries around the world, so they can choose a method that best suits their needs," said Dieter Weinand, member of the Board of Management of Bayer AG and President of its Pharmaceuticals Division. Ensure healthy lifes and promote well-being for all at all ages', is one of the Sustainable Development Goals (SDGs) ratified in September 2015 by the United Nations. These goals set a large number of ambitious targets to be achieved by They all recognize "health" as a driver of global economic growth. Although only specifically covered by one goal, health is implicitly linked to most of the other 16 SDGs. Bayer's activities in family planning directly relate to the "Health" goal, as it covers among others child and maternal mortality and reproductive health. As a market leader in oral contraceptives, Bayer now has nearly 50 years of expertise in the field of family planning. For women in developing countries, Bayer supplies its different hormonal contraceptive methods to partners in international development cooperation at a preferential price. At the same time, Bayer is developing new concepts like the "Jadelle Access Program" to give women more family planning options. ANACOR PHARMACEUTICAL 08/01/2016 Anacor Pharmaceuticals to Present Data From Long-Term Safety Study of Crisaborole Topical Ointment, 2% in Patients With Mild-to-Moderate Atopic Dermatitis at Upcoming Medical Conference PALO ALTO, Calif. -(BUSINESS WIRE)- Anacor Pharmaceuticals, Inc. (NASDAQ:ANAC) announced today that it will present data from the long-term safety study of crisaborole topical ointment, 2% in children and adults with mild-to-moderate atopic dermatitis at the Winter Clinical Dermatology Conference, being held in Koloa, Hawaii from January 15-20, About Crisaborole Topical Ointment, 2% Crisaborole topical ointment, 2%, is an investigational non-steroidal topical anti-inflammatory PDE-4 inhibitor in development for the potential treatment of

6 mild-to-moderate atopic dermatitis. Crisaborole is a novel boron-containing small molecule and, although the specific mechanism of action is not yet completely defined, Anacor believes that crisaborole inhibits PDE-4 in target cells, which reduces the production of pro-inflammatory cytokines thought to cause the signs and symptoms of atopic dermatitis. SANOFI 08/01/2016 Sanofi and Regeneron Announce Sarilumab Biologics License Application Accepted for Review by US FDA Paris, France and Tarrytown, New York - January 8, Sanofi and Regeneron Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for sarilumab. Per the Prescription Drug User Fee Act (PDUFA), the target action date is Oct. 30, Sarilumab is an investigational, human monoclonal antibody directed against the IL-6 receptor that is intended for the treatment of patients with active, moderate-to-severe rheumatoid arthritis (RA).[1] IL-6 is the most abundant cytokine in the serum and synovial fluid of patients with RA and levels correlate with both disease activity and joint destruction. [2] The BLA for sarilumab contains data from approximately 2,500 adults with active, moderate-to-severe RA who had an inadequate response to previous treatment regimens, including seven studies from the global SARIL-RA Phase 3 program. The goal of the ongoing global clinical development program is to evaluate the safety and efficacy of subcutaneous sarilumab, either as monotherapy or in combination with conventional disease-modifying anti-rheumatic drugs (DMARDs), including methotrexate (MTX), in reducing the signs and symptoms and inhibiting the radiographic progression of RA.[3],[4],[5],[6],[7],[8],[9] The safety and efficacy of sarilumab have not been fully evaluated by any regulatory authority. VDA Net All Rights Reserved