Your guide to the pharmaceutical world Volume 7 Issue 3 Funding discovery The price of innovation AI in Drug Discovery A prescription for precision Allergies Allergies no more Funding discovery The price of innovation AI in Drug Discovery A prescription for precision Allergies Allergies no more www.pbiforum.net 01.qxp_Layout 1 07/06/2019 12:53 Page 1FOR MANUFACTURING IN FOOD, BEVERAGES, PHARMACEUTICALS, TOILETRIES, COSMETICS AND MORE… 1-3 OCTOBER 2019 NEC, BIRMINGHAM THE COMPLETE PRODUCTION LINE EVENT ppmatotalshow.co.uk OWNED & ORGANISED BY FREE TO ATTEND REGISTER NOW 02.qxp_Layout 1 07/06/2019 12:54 Page 1@pbiforumwww.pbiforum.net company/pharma-business-international Contents Volume 7 Issue 3 News4 Stay up to date with the latest industry updates from the global pharmaceutical technology industry. M&A Round-up8 The proposed merger between Bristol-Myers Squibb and Celgene is once again dominating our M&A round-up, while other industry players secure long- term growth by swooping for oncology and gene therapy specialists. Gastrointestinal Disease Exposé12 With some forty per cent of the world’s population suffering from some kind of gastrointestinal disease, it’s clear that there’s a pressing medical need for new therapies and medications. With new research identifying the importance of the gut microbiome to health and wellbeing, new breakthroughs are just around the corner. Treating Allergies16 The allergies market has always been a profitable one for the pharma industry, but allergy immunotherapy may well be the silver bullet that patients, but not the pharma giants, want. AI in Drug Discovery20 Artificial intelligence is changing the face of drug discovery and development, saving time and money, and paving the way for precision medicine. Funding Discovery24 Pharma Business International explores the various ways in which medicines and drug discovery are funded. Intellectual Property and Patents28 Any pharmaceutical company is only as great as their intellectual property – but shakeups across the world are making the act of keeping an IP difficult. Biosimilars32 With more and more pharmaceutical firms shifting their focus to biosimilars, we explore the state of the market, what’s driving growth and what barriers still need to be overcome. Events36 We give a rundown of pharma events happening around the world. 4 12 20 24 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) 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, N E Lincs 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. COVER PHOTO: © SHUTTERSTOCK / HYWIT DIMYADI 03.qxp_Layout 1 07/06/2019 12:55 Page 1Trial okays aspirin for brain bleed stroke patients A major clinical trial has found that people who have suffered a stroke caused by a brain haemorrhage can take common medicines such as aspirin without increasing their risk of another stroke. Researchers say the findings are reassuring for the thousands of people who take such medicines to reduce their risk of heart attack and another common type of stroke caused by blood clots in the brain. Doctors had thought antiplatelet medicines – which include aspirin and clopidogrel – might make people with stroke due to brain haemorrhage more likely to suffer another bleed in the brain. Researchers led by the University of Edinburgh tracked outcomes from 537 people from across the UK who had suffered a brain haemorrhage while they were taking medicines to stop blood clotting. The team found that people who took antiplatelet medicines experienced fewer recurrences of brain haemorrhage compared with those who did not take these treatments. The study was published in The Lancet and The Lancet Neurology. 4 Pharma Business International www.pbiforum.net Latest news World’s first ‘Darwinian’ cancer drug programme to launch in London A new state-of-the-art building in London is set to house the world’s first ‘Darwinian’ drug discovery programme specially designed to tackle cancer’s ability to evolve resistance to treatment. The Institute of Cancer Research (ICR) will invest an initial £75 million in creating a global centre of expertise in anti-evolution therapies – which hold the promise of outsmarting cancer to improve cure rates. Cancer’s ability to constantly adapt, evolve and develop drug resistance is what makes it so lethal. But ICR scientists aim to harness evolutionary science within a new Centre for Cancer Drug Discovery to ‘herd’ cancers with anti-evolution drugs and combinations. They believe this new approach can deliver long-term control and effective cures, just as comparable approaches have with HIV. The ICR is seeking a further £15 million in donations to complete the new building and equip it with state-of-the-art instruments and computational technologies. You can donate by visiting www.icr.ac.uk. Aprinoia secures grant from Michael J. Fox Foundation Japanese biotech Aprinoia Therapeutics has secured a research grant from The Michael J. Fox Foundation for Parkinson’s Research for the development of alpha-synuclein PET imaging tracers. Aprinoia has established a proprietary small molecule platform targeting aggregated protein structures, including tau and alpha-synuclein. From its bespoke compound library, the company has identified a subset of molecules that selectively bind to alpha-synuclein aggregates and may be useful as PET imaging agents. A clinical alpha-synuclein PET tracer could be applied for precise diagnosis of alpha-synuclein-associated diseases – including Parkinson’s disease, dementia with Lewy bodies, and multiple system atrophy – and for facilitating development of treatments for patients of those diseases. Aprinoia CEO Ming-Kuei Jang said: “Our goal is to develop a tracer that could be widely accessible to the medical and research communities for advancing our knowledge of Parkinson’s disease and other alpha-synuclein related diseases and for developing new treatments for our patients.” © ICR © Shutterstock / White bear studio © Shutterstock / sfam_photo 04-07.qxp_Layout 1 07/06/2019 12:57 Page 1Record levels for clinical trial participants in England There’s been a rise in people benefitting from clinical research in England over the last year with numbers reaching record highs. During the past twelve months, over 870,000 participants were involved in clinical research studies supported by the National Institute for Health Research (NIHR). This takes significant steps towards the NHS Long Term Plans goal to reach one million people in trials by 2023/2024 – part of the Government’s overall strategy to improve care, treatment and NHS services in England. Health research for children has recruited the most participants this year with 81,892 people taking part. This was followed by studies delivered in primary care settings (78,533). Childbirth and sexual health research recruited the third highest number of participants at over 74,128 – up 20% from last year’s figures. The NIHR figures show that patients now have more opportunity than ever before to take part in clinical research and potentially benefit from new drugs and treatments. Pharma Business International 5 www.pbiforum.net Latest news Takeda launches new gastrointestinal-focussed biopharma Takeda and Frazier Healthcare Partners have launched Phathom Pharmaceuticals, a new biopharma focused on the development and commercialisation of novel treatments for gastrointestinal (GI) diseases and disorders. Takeda has granted a license to Phathom for the development and exclusive commercialisation rights to vonoprazan in the US, Europe and Canada in exchange for upfront cash and equity, as well as future cash milestones and royalties on net sales. Vonoprazan is a novel, orally active potassium competitive acid blocker (P-CAB) discovered and developed by Takeda. In connection with the license, Phathom has completed a $90 million crossover financing and $50 million debt facility. Joining Tachi Yamada and David Socks on the Phathom board of directors are James Topper, Managing General Partner of Frazier Healthcare Partners, and Jon Edwards, Partner of Medicxi. Gene therapy cures babies with ‘bubble boy’ disease Gene therapy developed at St. Jude Children’s Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells – including T cells, B cells and natural killer (NK) cells – for the first time in research detailed in the New England Journal of Medicine. The children were enrolled in an ongoing clinical trial of infants newly diagnosed with SCID-X1, the most common type of SCID, a rare, life-threatening genetic disorder that is sometimes called “bubble boy” disease. The name is a reference to measures taken to protect patients, who are born with little or no immune protection. Untreated, patients usually die early in life. Ten infants have received the experimental therapy. It was developed in research led by the late Brian Sorrentino of the St. Jude Department of Haematology. He is the senior author and died after the manuscript was submitted for publication. © Shutterstock / Natali_Mis © Shutterstock / Numstocker © Shutterstock / PopTika 04-07.qxp_Layout 1 07/06/2019 12:57 Page 26 Pharma Business International www.pbiforum.net Latest news Scancell & Cancer Research reach milestone for novel immunotherapy Scancell has reached an “important milestone” on SCIB2, a novel cancer immunotherapy it’s developing in partnership with Cancer Research UK. The company’s ImmunoBody vaccine is being developed as a potential treatment for patients with solid tumours, including non-small cell lung cancer. The ImmunoBody immunotherapy platform activates the body’s immune system by enhancing the uptake and presentation of cancer antigens to help target and eliminate cancer cells. SCIB2, Scancell’s second ImmunoBody therapy, targets an antigen called NY-ESO-1, which is expressed on a range of solid tumours. Pre-clinical studies have demonstrated that administration of SCIB2 as a liposomal nanoparticle results in potent immune responses and prolonged survival. The nanoparticle delivery system provides an alternative approach to electroporation, which has been used to deliver other ImmunoBody agents to patients. Cancer Research UK is now planning a clinical trial to investigate the safety and efficacy of the SCIB2- nanoparticle complex in patients with solid tumours. Bioquell launches ProteQ mobile room bio-decontamination system Bioquell, a leading global expert in reducing the risk of bio-contamination in the pharmaceutical, life science and healthcare markets, has launched the cost effective and high performance Bioquell ProteQ mobile room bio- decontamination system. Utilising the company’s approved 35% hydrogen peroxide vapour and boasting a modular, upgradeable design for a more configurable pricing structure, the Bioquell ProteQ is suited to a wide range of critical areas, including biopharmaceutical manufacturing facilities, GMP/GLP and bio-safety laboratories, animal facilities and cleanrooms. Developed as the next generation of the company’s previous mobile room decontamination offering, the rigorously tested Bioquell ProteQ incorporates powerful distribution technology for reduced cycle times with more efficient use of Bioquell’s approved 35% hydrogen peroxide vapour and less need for additional standalone distribution fans. It also offers new wireless functionality which can network multiple units, improving set up times and making it ideal for areas of any size. T3D secures grant to develop novel Alzheimer’s treatment T3D Therapeutics has secured a grant worth $9 million over four years to help fund a Phase 2 clinical study of T3D-959, its novel metabolic-focussed Alzheimer’s disease (AD) treatment. The grant comes from the National Institute on Ageing – part of the National Institutes of Health – and aims to help develop the drug into a new approach to treating AD. The Phase 2 PIONEER study is expected to initiate patient dosing in early 2020. It will enrol subjects who will receive one of three different doses of T3D-959 or a placebo for 24 weeks. “During my lengthy tenure treating AD patients I have seen first-hand the frustrations of caregivers and patients at the lack of an effective therapy with the plethora of recent drug development failures causing them to lose hope,” said Warren Strittmatter, Chief Medical Officer of T3D Therapeutics. “This award provides great support for our promising new therapy to give them renewed optimism.” © Shutterstock / Kateryna Kon © Shutterstock/Atthapon Raksthaput 04-07.qxp_Layout 1 07/06/2019 12:57 Page 3 EUROPE Zynquista approved for treating type 1 diabetes Zynquista (sotagliflozin) has been approved in Europe for use as an adjunct to insulin therapy to improve blood sugar control in adults with type 1 diabetes. Developed by Sanofi and Lexicon, Zynquista is an oral dual inhibitor of two proteins responsible for glucose regulation known as sodium-dependent glucose co- transporter types 1 and 2 (SGLT1 and SGLT2). Expanded breast cancer indication for Lynparza in Europe Lynparza has been approved in Europe as a monotherapy for treating adult patients with germline BRCA-mutated HER2- negative advanced breast cancer. Developed by AstraZeneca and Merck, Lynparza is the first PARP inhibitor approved in the EU for patients with this difficult-to-treat disease. The approval was based on data from the randomised, open-label, Phase III OlympiAD trial which tested Lynparza vs. physician’s choice of chemotherapy. This is the third indication for Lynparza in the EU. European approval for Pfizer’s Vizimpro The European Commission has given Pfizer’s Vizimpro (dacomitinib) the thumbs up for adult patients with epidermal growth factor receptor (EGFR) mutated non-small cell lung cancer (NSCLC). The approval was supported by data from the Phase 3 ARCHER 1050 study. European approval for Teva’s migraine treatment European regulators have approved Teva’s Ajovy, the first and only anti-CGRP treatment with both quarterly and monthly dosing for the prophylaxis of migraine in adults. Ajovy (fremanezumab) is a humanised monoclonal antibody (mAb) that binds to the calcitonin gene-related peptide (CGRP) ligand and blocks its binding to the receptor. It was evaluated in two pivotal Phase III clinical trials that enrolled patients with disabling migraine and studied fremanezumab as a prophylactic treatment for migraine in adults. UNITED STATES Novartis picks up approval for Piqray Piqray has become the first and only treatment specifically for patients with a PIK3CA mutation in HR+/HER2 advanced breast cancer approved in the US. The FDA approval is based on results of the Phase III trial, SOLAR-1, that showed Piqray plus fulvestrant nearly doubled median progression-free survival compared to fulvestrant alone in HR+/HER2- advanced breast cancer patients with a PIK3CA mutation. Benlysta first medicine approved for children with lupus in US Benlysta has become the first medicine in the US approved for children with lupus after GSK gained the green light from the FDA. The IV formulation of Benlysta (belimumab), a B- lymphocyte stimulator (BLyS)-specific inhibitor, has been approved under priority review. The approval extends the current indication in the US for the IV formulation of Benlysta in adults to patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus who are receiving standard therapy. FDA approves Praluent for CV patients Regeneron Pharmaceuticals and Sanofi have secured FDA approval for Praluent (alirocumab) to reduce the risk of heart attack, stroke and unstable angina requiring hospitalisation in adults with established cardiovascular (CV) disease. The approval is based on data from ODYSSEY OUTCOMES, which assessed the effect of adding Praluent to maximally- tolerated statins on CV outcomes in 18,924 patients who had an acute coronary syndrome within a year of enrolling in the trial. Pfizer’s male breast cancer treatment approved in US Regulators in the US have approved Pfizer’s supplemental New Drug Application to expand the application of Ibrance (palbociclib) for treating men with HR+ and HER2 metastatic breast cancer. With this approval, Ibrance is the first and only CDK 4/6 inhibitor indicated in combination with an aromatase inhibitor for the first-line treatment of men with HR+, HER2- metastatic breast cancer in the US. www.pbiforum.netPharma Business International 7 04-07.qxp_Layout 1 07/06/2019 12:57 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net 08-11.qxp_Layout 1 07/06/2019 12:58 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP L et’s begin this issue’s mergers and acquisitions round-up by getting the big news out of the way from the get- go with the now obligatory update on the fate of Bristol-Myers Squibb’s (BMS) $74 billion acquisition of Celgene. Its inclusion shouldn’t surprise readers, having dominated the M&A space since it was first announced back in January. Of course, a deal of this magnitude has several regulatory hurdles to clear, not to mention gaining the backing of shareholders and all relevant parties. But recently the deal took an important step forward at a Special Meeting of Stockholders. Here BMS shareholders voted to approve issuance of shares of common stock in connection with the planned merger. It’s a move the BMS board are obviously in favour of, but one which has gained the majority backing of shareholders, with over seventy-five per cent of the shares voted at the meeting in favour of the merger agreement. That was in mid-April and, as of publishing, was the latest update from the boards of both parties. Suffice it say, BMS Chairman Giovanni Caforio was pleased, thanking shareholders for their support. The deal, which Caforio says will result in a “premier innovative biopharma company” remains on-track to close in the third quarter of 2019 subject to the typical closing conditions and regulatory approvals. So by the next issue of Pharma Business International , the major merger deal that has become a mainstay of our pages may well have completed. Moving on, we find Merck bolstering its oncology pipeline by swooping for Texan biopharma Peloton Therapeutics in a deal worth up to $2.20 billion. To date, Merck is the largest pharma company to acquire a firm with the purpose of fortifying its pipeline of cancer treatments. It’s a smart move for Merck who will gain access to 10 Á Nearing completion The proposed merger between Bristol-Myers Squibb and Celgene is once again dominating our M&A round-up, while other industry players secure long-term growth by swooping for oncology and gene therapy specialists. 08-11.qxp_Layout 1 07/06/2019 12:58 Page 2Next >