Your guide to the pharmaceutical world Volume 7 Issue 6 Depression Exposé A light in the dark Purchasing Finding the right price point Precision Medicine Targeted treatments Depression Exposé A light in the dark Purchasing Finding the right price point Precision Medicine Targeted treatments www.pbiforum.net 01.qxp_Layout 1 02/12/2019 10:09 Page 1INTERNATIONAL FoodDrink FoodDrink www.fdiforum.netOCTOBER 2019 £3.50 STERLING News The natural choice The natural choice For businesses that are serious about reducing their carbon footprint www.blmgroup.co.uk VOLUME 4 ISSUE 1 WWW.RENEWABLESANDENVIRONMENT.COM OCTOBER 2019 EAST MIDLANDS LEADING BUSINESS MAGAZINE www.blmgroup.co.uk WWW.EASTMIDLANDSBUSINESSLINK.CO.UK WWW.EASTMIDLANDSBUSINESSLINK.CO.UK £3.50 In the eye of the law Better. Smarter. Faster. Seeking help when growing 01.qxp_Layout 1 04/10/2019 10:44 Page 1 EMPOWER Your Business Your guide to the pharmaceutical world Volume 7 Issue 4 Antibiotic Resistance Exposé Resistance is futile Manufacturing Automate, automate, automate Vaccines A shot in the arm Antibiotic Resistance Exposé Resistance is futile Manufacturing Automate, automate, automate Vaccines A shot in the arm www.pbiforum.net 01.qxp_Layout 1 06/08/2019 09:25 Page 1 september 2019 £3.50 yorkshire & lincolnshire’s leading business magazine www.blmgroup.co.uk VOLUME 34 ISSUE 9 Boosting wellbeing TAKING STOCK TAKING STOCK Keeping up with the market Train old or train new? Often Imitated - Never Equalled www.blmgroup.co.uk 02.qxp_Layout 1 02/12/2019 10:10 Page 1@pbiforumwww.pbiforum.net company/pharma-business-international Contents Volume 7 Issue 6 4 12 16 32 Group Editor: Steve Fisher (s.fisher@blmgroup.co.uk) Editor: Michael Fisher (m.fisher@blmgroup.co.uk) Journalist: Dominic Cuthbert (d.cuthbert@blmgroup.co.uk) Tess egginton (t.egginton@blmgroup.co.uk) Sales Director: Angie Cooper (a.cooper@blmgroup.co.uk) Sales Manager: Sam Trott (s.trott@blmgroup.co.uk) Sales: vicky hunt (v.hunt@blmgroup.co.uk) Tel: +44 (0) 1472 310302 Accounts & Subscriptions: Angela Sharman (a.sharman@blmgroup.co.uk) Design & Production: Gary Jorgensen, Mark Casson, (studio@blmgroup.co.uk) Editorial: Tel: +44 (0) 1472 310305, Fax: +44 (0) 1472 310317 Part of BLMGroup: Armstrong house, Armstrong Street, Grimsby, north east lincolnshire Dn31 2Qe england All rights reserved. No part of this publication may be reproduced, transmitted, photocopied, recorded or otherwise without express permission of the copyright holder, for which application should be addressed first to the publisher. While every reasonable care is taken, neither the publisher nor its participating agents accept liability for loss or damage to prints, colour transparencies, negatives or other material of whatever nature submitted to this publication. The views expressed in this publication are not necessarily the views of those held by the publisher. C over P ho T o : © S hu TT er ST o C k / S ol A r S even News4 Stay up to date with the latest industry updates from the global pharmaceutical technology industry. M&A Round-up8 With Bristol-Myers Squibb having finally completed its historic merger with Celgene, Pharma Business International takes a closer look at the deal that has defined the industry’s M&A market in 2019. Depression Exposé12 For this month’s cover feature, Pharma Business International explores what’s happening in the depression pipeline, exploring new treatment options, delivery systems and breakthroughs. Precision Medicine16 Breakthroughs over the last few decades have paved the way for precision medicine, but there is still much more research needed before this approach becomes the norm. Paediatric Therapeutics20 The development and testing of paediatric therapeutics have historically been limited, with a habit of simply translating results from adults to children. Oncology24 Artificial intelligence is a hot topic in oncology, with its applications spanning from improving diagnosis to precision and personalised medicine. Partnerships28 Pooling skills is valuable in any industry, but the pharmaceutical industry, especially, has seen some high-profile collaborations in recent years. Purchasing32 There’s plenty to consider for health bodies when purchasing new treatments for patients, as we explore. Events36 We give a rundown of pharma events happening around the world. 03.qxp_Layout 1 02/12/2019 10:13 Page 1Janssen evolves digital clinical trial design Janssen has launched what it claims is the first-ever completely decentralised mobile indication-seeking clinical study as the Johnson & Johnson company embarks on “the next evolution of digital clinical trial design”. To accelerate the study and fast-track results, all contact with participants will be done virtually, with no in-person clinical visits required. Drawing on previous experience, Janssen is utilising smart technology and wearable devices to more quickly and efficiently gather and analyse real-world evidence to assess the effectiveness of canagliflozin in adults with heart failure, with or without type 2 diabetes. Through a collaboration with global research organisation PRA Health Sciences and its innovative mobile clinical trial platform, the CHIEF-HF study will examine the use of canagliflozin compared to placebo on quality of life improvement scales, in participants with either preserved or reduced ejection fraction heart failure. 4 Pharma Business International www.pbiforum.net Latest news NIH & Gates Foundation take aim at sickle cell disease & HIV The National Institutes of Health (NIH) and the Bill and Melinda Gates Foundation are each investing $100 million towards developing affordable, gene-based cures for sickle cell disease (SCD) and HIV. The collaboration sets out a bold goal of advancing safe, effective and durable gene-based cures to clinical trials in the US and relevant countries in sub-Saharan Africa within the next seven to 10 years. The ultimate goal is to scale and implement these treatments globally in areas hardest hit by these diseases. The collaboration will align aggressive, high-reward research efforts to accelerate progress on shared gene-based strategies to cure SCD and HIV. Both organisations will also continue to invest in other parallel research efforts on cures for SCD and HIV outside of this collaboration. Data shows efficacy & safety of Desvocy for PrEP Gilead Sciences has reported 96-week results from the DISCOVER trial evaluating the safety and efficacy of once-daily Descovy for HIV pre-exposure prophylaxis (PrEP) compared with Truvada for PrEP in men and transgender women who have sex with men and are at risk for sexually acquired HIV infection. Descovy (emtricitabine 200 mg & tenofovir alafenamide 25 mg) demonstrated non-inferior efficacy to Truvada (emtricitabine 200 mg & tenofovir disoproxil fumarate 300 mg) through 96 weeks and statistically significant differences over Truvada for certain key measurements of bone and renal safety assessed in the study, which were pre-specified secondary endpoints. Diana Brainard, Senior Vice President of HIV & Emerging Viruses at Gilead Sciences, said the results “offer new insights into the improved renal and bone safety profile of Descovy as measured by key bone and renal markers. As more at-risk people use PrEP for longer periods of time, the data affirm the value of Descovy for PrEP as a new HIV prevention option.” © Shutterstock/sdecoret © Shutterstock / angellodeco © Shutterstock / Marc Bruxelle 04-07.qxp_Layout 1 02/12/2019 10:16 Page 1EMA accepts keys to new Amsterdam base The European Medicines Agency (EMA) has taken charge of its newly built tailor-made premises in Amsterdam. EMA moved from London to Amsterdam in March 2019 following the result of the EU referendum and has since then been operating from temporary premises in Amsterdam Sloterdijk. Now that the Zuidas-based building has been handed over by Dutch authorities, technical equipment will be installed followed by IT configuration and testing. It is expected that staff can move into their new offices and workspaces in January 2020. Pharma Business International 5www.pbiforum.net Latest news Four drug companies settle opioid litigation in Ohio Teva Pharmaceutical has along with a trio of drug distributors agreed to pay a total of $60 million to settle opioid litigation in the Ohio counties of Cuyahoga and Summit. The three distributors – AmerisourceBergen, Cardinal Health and McKesson – will together pay $215 million in the first track of the multi-district opioid litigation. In a joint statement, they said: “The companies expect settlement funds to be used in support of initiatives to combat the opioid epidemic, including treatment, rehabilitation, mental health and other important efforts.” Teva, meanwhile, has also reached a settlement agreement with both Ohio counties. Under the terms of the settlement, it will pay a cash payment of $20 million over three years. Furthermore, Teva will provide the counties with the critical opioid treatment medication Suboxone (buprenorphine naloxone) valued at $25 million and distributed over three years to “help in the care and treatment of people suffering from addiction”. Parkinson’s organisations partner to advance potential new therapy The Parkinson’s Foundation has granted $250,000 towards a potential new therapy for people with Parkinson’s disease (PD) as part of a new partnership with Parkinson’s UK. Each year, Parkinson’s UK, their supporters and partners invest more than $5 million into Parkinson’s Virtual Biotech to rapidly develop and test the most promising research findings with the aim of turning them into treatments that will transform life for people with PD. This grant will help support a project seeking to discover new ways that may prevent brain cell death by stabilising the source of energy essential to cell survival, the mitochondria. This is the first international funding received by Parkinson’s UK for the Virtual Biotech and signals the start of a joint trans-Atlantic mission to fast-track the best scientific ideas towards ground-breaking © Shutterstock / sfam_photo © Shutterstock / thodonal88 © Shutterstock / Victor Moussa 04-07.qxp_Layout 1 02/12/2019 10:16 Page 26 Pharma Business International www.pbiforum.net Latest news Evotec & Vifor Pharma to develop nephrology therapeutics Evotec and Vifor Pharma are creating a new joint venture focussed on the discovery and development of novel nephrology therapeutics. The joint venture will focus on the discovery of nephrology therapeutics by analysing the clinical data provided by Evotec that is mining a unique UK kidney biobank – NURTuRE – in order to identify new targets for renal therapies and sharpen existing product profiles of an innovative pipeline. Evotec will focus on applying its drug discovery & development capabilities and will leverage Vifor Pharma’s proven commercial platform to create a robust pipeline of nephrology programmes. Upon completion of clinical trials for drugs in the newly-created, jointly-owned pipeline, products will be out-licensed to Vifor Pharma for registration and commercialisation to further expand Vifor Pharma Group’s strong nephrology pipeline. Initial funding of €25 million for pre-clinical development will be covered by Vifor Pharma so that multiple targets or candidates can be moved forward simultaneously. First new HIV strain identified in almost 20 years A team of scientists at Abbott have discovered a new subtype of ‘Group M’ HIV virus for the first time since guidelines for classifying new strains of HIV were established in 2000. To determine whether an unusual virus is in fact a new HIV subtype, three cases must be discovered independently. The first two samples of this subtype were discovered in DRC in the 1980s and the 1990s. The third, collected in 2001, was difficult to sequence at that time because of the amount of virus in the sample and the existing technology. Today, next-gen sequencing technology allows researchers to build an entire genome at higher speeds and lower costs. In order to utilise this technology, Abbott scientists had to develop and apply new techniques to help narrow in on the virus portion of the sample to fully sequence and complete the genome. Novartis to broaden portfolio reach in sub-Saharan Africa Novartis has announced a new strategy to broaden patient reach and availability of its portfolio of medicines in sub-Saharan Africa (SSA). As part of the new strategy, Novartis will pivot the current organisational focus in SSA from financial metrics such as sales performance and profits, to metrics that drive access to innovative medicines and strengthen health systems in the region. A new organisational unit will bring together the expertise and portfolio of its Sandoz Division, the Novartis Pharmaceuticals and Oncology business units comprising our Innovative Medicines Division and Novartis Social Business. Racey Muchilwa has been appointed as the Head of Global Health SSA, contributing her strong knowledge of the healthcare system and patient needs in the region. The SSA unit aims to maximise patient reach across the full income pyramid by focusing on tiered pricing models, competitiveness in tenders and scaling social business models as well as affordability strategies. © Shutterstock/crystal light © Shutterstock / gopixa © Shutterstock/Matic Stojs Lomovsek 04-07.qxp_Layout 1 02/12/2019 10:16 Page 3 EUROPE Conditional approval for Merck’s Ebola virus vaccine The European Commission (EC) has granted conditional approval to Merck for Erverbo for the active immunisation of adults to protect against Ebola Virus Disease (EVD). The approval is based on data submitted to the European Medicines Agency for accelerated assessment in March 2019. With this approval, the EC will grant a centralised marketing authorisation with unified labelling that is valid in the 28 countries that are members of the EU, as well as European Economic Area members, Iceland, Liechtenstein and Norway. Approval for Xospata for mutated AML Regulators have approved the oral once-daily therapy Xospata (gilteritinib) as a monotherapy for the treatment of adult patients with relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation (FLT3mut+). According to Astellas Pharma, the therapy has the potential to improve treatment outcomes for AML patients with two forms of the most common mutation – FLT3 internal tandem duplication and FLT3 tyrosine kinase domain (TKD) mutation. The approval is based on results from the Phase 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. Label update for Cosentyx The European Commission has rubber stamped a label update for the up-titration of Cosentyx (secukinumab) to 300 mg for patients with active ankylosing spondylitis (AS). The approval is based on data from MEASURE 3, a three-year study that explored the tolerability and efficacy of Cosentyx in patients with AS. The safety profile was consistent with previous studies, Novartis said. Satralizumab under review for NMOSD Roche’s submissions for satralizumab in neuromyelitis optica spectrum disorder (NMOSD) has been accepted by review by regulators in both Europe and the US. The European Medicines Agency has granted the submission Accelerated Assessment, signifying that the application is now under review by the Committee for Medicinal Products for Human Use. In the US, the Food and Drunk Administration has accepted the company’s Biologics License Application for satralizumab. The CHMP recommendation and the FDA decision are expected in 2020. UNITED STATES Adakveo approved to reduce pain crises in sickle cell disease Novartis has secured approval for Adakveo (crizanlizumab) to reduce the frequency of pain crises in patients with sickle cell disease. This is the first FDA-approved medicine in sickle cell disease that binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion. The approval is based on results of the SUSTAIN trial which showed that Adakveo significantly lowered the median annual rate of pain crises by 45%. First transdermal patch for treating schizophrenia approved Regulators have approved Secuado (asenapine), the first and only transdermal patch formulation for treating adults with schizophrenia. The once-daily transdermal drug delivery system provides sustained concentrations during wear time (24 hours) of the atypical antipsychotic drug asenapine (a well-established treatment for schizophrenia). In a Phase 3 study, Secuado achieved the primary endpoint of statistically significant improvement from baseline in the change of the total Positive and Negative Syndrome Scale compared to placebo at week six. Approval for new type of antibiotic for complicated UTIs Regulators have rubber stamped a new type of antibiotic to treat complicated urinary tract infections (cUTI) in adult patients with limited or no alternative treatment options. Approval was granted to Japan’s Shionogi for Fetroja (cefiderocol) for cUTI including pyelonephritis caused by: susceptible Gram-negative microorganisms: Escherichia coli, Klebsiella pneumoniae, Proteus mirabilis, Pseudomonas aeruginosa, and Enterobacter cloacae complex. The approval is based on data from the pivotal APEKS-cUTI study that evaluated the efficacy and safety of Fetroja versus imipenem/cilastatin (IPM/CS) in patients with cUTI. Shionogi anticipates making Fetroja commercially available in early 2020. US approves Pfizer’s Humira biosimilar The FDA has approved Pfizer’s Humira biosimilar for treating patients with multiple inflammatory conditions including adult Crohn’s disease, ulcerative colitis and psoriatic arthritis. The approval of Abrilada (adalimumab-afzb) was based on the review of a comprehensive data package, which demonstrated biosimilarity to the reference product. Pfizer said it is planning to launch Abrilada in the US in 2023. www.pbiforum.net Pharma Business International 7 04-07.qxp_Layout 1 02/12/2019 10:16 Page 4Breaking down the biggest deals © Shutterstock /IgorGolovniov 08-11.qxp_Layout 1 02/12/2019 10:19 Page 1Pharma Business International 9 M&A ROUND-UP The latter half of 2018 brought with it fears that the pharmaceutical mergers and acquisitions market was cooling, with a slowdown in deals compared with the busy start to the year. Days into 2019, however, and one of the industry’s biggest deals to date was announced. Bristol-Myers Squibb (BMS), the New York-based biopharmaceutical company, was acquiring Celgene for $74 billion. The aim was to create a “premier innovation biopharmaceutical company” bringing together both parties’ complementary portfolios in oncology, immunology & inflammation, and cardiovascular disease. Once completed, it was revealed, BMS shareholders would own around sixty-nine per cent of the merged company, with shareholders of Celgene owning the remaining thirty-one per cent. Yet the path towards completion was far from smooth. In late February, the deal hit a roadblock after the largest shareholder in BMS, investment management firm Wellington Management, refused to give its support. The firm, which holds an eight per cent stake in BMS, informed the company’s Board of Directors that it was “not supportive” of the then proposed deal. Analysts at the time were right on the money claiming that this was unlikely to scupper the deal but, in April, the merger was beset by a rebel investment group. Starboard Value, an American activist hedge fund, filed blue proxy materials intended to be voted on at an upcoming annual general meeting with the aim of rejecting the deal. However, the proposal was withdrawn, clearing the way for BMS to gain the backing of its shareholders. At a Special Meeting of Stockholders, shareholders voted to approve the issuance of shares of common stock With Bristol-Myers Squibb having finally completed its historic merger with Celgene, Pharma Business International takes a closer look at the deal that has defined the industry’s M&A market in 2019. connected with the merger. More than seventy-five per cent of those shares were voted in favour of the merger. At the time, the deal looked set to complete in the third quarter of 2019, but there were further issues ahead, this time from the Federal Trade Commission (FTC). In order to assuage competition concerns expressed by the FTC, and in order to further progress the deal, BMS agreed in June to divest Otezla (apremilast), a treatment for psoriasis and psoriatic arthritis, and one of its most lucrative drugs. It was acquired by Californian biopharmaceutical company, Amgen, in a deal worth up to $13.4 billion. According to the agency, this is the largest divestiture in a merger- enforcement case by the FTC or Justice Department. As well as satisfying concerns that the merger would otherwise harm the competition, the proceeds of the sale will enable BMS to pay off its debts faster and, as was previously announced, increase a planned post-merger share re-purchase from $5 billion to $7 billion. Following the divestiture, the FTC signed off on the merger and, in November, BMS secured antitrust approval, clearing the very last regulatory hurdle. It’s worth mentioning that the approval was hardly unanimous, with a 3- 2 vote split along the party lines and serving as an ill omen for major biopharma merger deals in the future. In the present, Celgene has become a wholly-owned subsidiary of BMS, the latter of which promptly divested Otezla. And so ends a near year-long process that has been the one constant across the M&A round-ups in 2019. 10 Á 08-11.qxp_Layout 1 02/12/2019 10:19 Page 2Next >