Your guide to the pharmaceutical world Volume 9 Issue 2 Blood diseases exposé Gene editing gathers pace Security Securing the supply chain Manufacturing Printing pills PharmaBUSINESS INTERN ATION AL The total package From development to dispensing The total package From development to dispensing Blood diseases exposé Gene editing gathers pace Security Securing the supply chain Manufacturing Printing pills www .pbiforum.net 01.qxp_Layout 1 08/04/2021 12:09 Page 1 INTER N Food Food &&& Food Food & N ATI O N &&Drin Drin Drin &&Drin AL nk nk nkk app A r f h hi ts a ews Ingredient spotlight End offline packkaaging For businesses that are serious about reducing their carbon footprint www.blmgroup.co.uk VOLUME 4 ISSUE 1 W W W. R E N E W A B L E S A N D E N V I R O N M E N T. C O M E A S T M I D L A N D S L E A E A D I N G B U S I N E S S M A G A A Z I N E APRIL 2020 0 £3. .50 BUS SUP SINES T PPOR SS TING B THCART HEAL YY FACTOR RE OFF FICE ea Stopping the spr THCAR WW HEAL d thinkin Forwar TT AUTOMA Y AFACTOR ad WW EASTMIDL RE A hea SOL OFF ng factories LANDSBUSINE TION ficef althy of LUTIONS ESSLINK CO U FICE w UK www.blmgroup.co.uk EMPOWER Your Business Focus on T A Pharm rld o our guide to the pharmaceutical w Y B U S I N E S S I N T E R N A L ma olume 8 Issue 1 V T I O N declares g rrld Hea oooW coron F ganisa gg l b l g l h O virus na g gency ti medi Nucle Prev aaccin V The nuclea b f treatment ve The P l t k aage n e t eelopment t vv om de rF w w w di i k . p b i f o r u m . n yorkshire & lincolnsh hire’s leading business ma magazine april 2020 £3.5 50 TO T RES C THE SPOND CRISIS DING S YY FACTOR C TT HEAL OFF THCARE FICE safety Ensuring e TT HEAL e of factories The futur TION T AUTOMA YY AFACTOR fices healthy f Keeping of SOLUTIONS OFFICE employee THCARE V w VOLUME 35 ISSUE 4 .blmgroup.co.uk www Often Imitated - Never Equalled www.blmgroup.co.uk 02.qxp_Layout 1 08/04/2021 12:10 Page 1Contents Volume 9 Issue 2 12 16 20 28 Group Editor: Steve Fisher (s.fisher@blmgroup.co.uk) Editor: Michael Fisher (m.fisher@blmgroup.co.uk) Assistant Editor: Dominic Cuthbert (d.cuthbert@blmgroup.co.uk) Journalist: Tess Egginton (t.egginton@blmgroup.co.uk) Sales Director: Angie Cooper (a.cooper@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. News4 Stay up to date with the latest industry updates from the global pharmaceutical technology industry. M&A round-up8 Reinforcing, rather than diversifying, is the name of the game for the latest slew of major deals in the pharmaceutical sector. Blood diseases exposé12 The buzz in treating blood disorders is firmly focused on genetic treatments, with a plethora of studies showing promising results. Finance16 We explore the various ways in which drug discovery and development can be funded. Security20 The security of pharmaceutical supply chains has been thrust into the spotlight during the COVID-19 pandemic, while regulation is clamping down on counterfeit drugs and promoting better traceability. Packaging24 Packaging has a key role to play in battling counterfeit medicines, with manufacturers increasingly relying on track and trace systems. Manufacturing28 Pharma Business International explores the proliferation of 3D printing in drug manufacturing, what’s driving the growth and what challenges still remain to overcome. Emerging diseases32 COVID-19 has dominated global news and has many people asking if we can’t predict the next emerging pandemic. Research is already underway. Events 36 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy: Shutterstock / Ezume Images 03.qxp_Layout 1 08/04/2021 12:11 Page 1Clinigen signs Africa and Asia distribution agreement with Amgen Clinigen Group, the global pharmaceutical Products and Services company, has signed a distribution agreement with Amgen to supply part of its product portfolio to multiple countries across Africa and Asia. Under the terms of the distribution agreement, Clinigen will provide physicians and pharmacists in certain territories with access to medicines which are not currently commercially available. Clinigen will act as the main point of contact for healthcare professionals wanting to access the medicines, managing all elements of the process including order and enquiry management, regulatory compliance, and logistics. The agreement will help support both companies’ missions of increasing access to medicines for patients across the world. Sam Herbert, Chief Operating Officer, Clinigen, said: “This agreement will help to ensure healthcare professionals can obtain compliant access to critical medicines that are not commercially available in their country.” 4 Pharma Business International www.pbiforum.net Latest news CN Bio awarded grant to develop single and multi-organ models for COVID-19 research CN Bio, a developer of single and multi-organ microphysiological systems (MPS), otherwise known as organ-on-a-chip, has received a grant from Innovate UK, the UK’s innovation agency, to develop human- relevant MPS to advance investigations into SARS-CoV-2 infection. The funding will be used to develop and test advanced cell culture models that represent different regions of the lung and subsequently linking these models to other organ systems, such as liver and gut, to inform the rapid development of novel therapeutics. Dr Tomasz Kostrzewski, Director, Biology, CN Bio, said: “Through Innovate UK’s recognition of our technology’s potential, CN Bio is proud to be working on an additional COVID-19 project, to support the global effort in tackling the pandemic. “This funding will enable us to develop and evaluate humanised single and multi-organ lung models, with the aim of deploying these to researchers investigating COVID-19 infection and immunology, and ultimately strengthen the fight against the spread of the virus.” Merck to add single-use assembly production to life science centre Merck is accelerating its European expansion plans for single-use products critical to manufacturing vaccines and lifesaving therapies. The life science company is adding a single-use assembly production unit at its Life Science Center in Molsheim, France. With the €25 million investment, the company is accelerating its European expansion plans for this key technology, which is used for the production of COVID-19 vaccines and other lifesaving therapies. Molsheim will be the first site in Europe where Merck manufactures the product. Further production sites are located in Danvers, Massachusetts, USA, and Wuxi, China. Belén Garijo, Vice Chair of the Executive Board and Deputy CEO of Merck, said: “Our new European production unit will increase capacity of the urgently needed supply of single-use assemblies to vaccine and therapeutic manufacturers worldwide.” © Shutterstock/pogonici © Shutterstock / Dmitry Kalinovsky © Shutterstock/Andrii Vodolazhskyi 04-07.qxp_Layout 1 08/04/2021 12:13 Page 1Eisai and DZNE to create potential novel treatments for neurodegenerative disorders Eisai and Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE) had entered a research collaboration aiming to create potential novel treatments for neurodegenerative disorders including Alzheimer’s disease (AD) which modulate immune competence in neurons and glia cells. DZNE is a major interdisciplinary public research organization constituted with ten research sites in Germany that investigates methods for prevention, diagnosis and treatment against neurodegenerative diseases. The collaboration will incorporate DZNE’s expertise in the fundamental and clinical research with Eisai’s abundant experience in drug discovery for neurodegenerative diseases to accelerate development of novel drug candidates. Prof. Pierluigi Nicotera, Chairman of the DZNE’s Executive Board, said: “We think that this collaboration will be able to accelerate creation of new treatments for neurodegenerative disorders, including those for Alzheimer’s and Parkinson’s.” Pharma Business International 5 www.pbiforum.net Latest news Aker BioMarine enters into research collaboration for Alzheimer’s Global biotech innovator Aker BioMarine enters into a research collaboration with Université de Sherbrooke Department of Medicine, to investigate if LPC-bound EPA/DHA is effective in preventing cognitive decline linked to Alzheimer’s disease. Aker BioMarine will supply the research team its krill-based LPC EPA/DHA product, LYSOVETA. While there is strong evidence linking DHA to better cognitive scores and lower risk of Alzheimer’s disease, clinical trials using fish oil as the source for EPA/DHA have not been conclusive. To help fill this gap, a research team from the Université de Sherbrooke in Canada will test if LYSOVETA, Aker BioMarine’s new delivery platform based on LPC-bound EPA/DHA from krill, is more effective in increasing the concentration of DHA in the brain and improving cognition. Matts Johansen, CEO, Aker BioMarine, said: “Alzheimer’s is a disease without a cure, which is why we are actively exploring whether LYSOVETA can help improve cognitive abilities among those who are affected by it.” CEPI launches $3.5bn plan to tackle risk of future pandemics and epidemics CEPI, the Coalition for Epidemic Preparedness Innovations, has launched a US$3.5 billion plan to dramatically reduce or even eliminate the future risk of pandemics and epidemics, potentially averting millions of deaths and trillions of dollars in economic damage. CEPI’s five-year plan sets out ambitious initiatives to build a future world that is better equipped to deal with these devastating diseases. As part of the plan, CEPI announced its “moonshot” objective to help compress vaccine development timelines to 100 days, about a third of the time that it took the world to develop a COVID-19 vaccine. CEPI is calling on governments, global health organisations, and strategic partners to back this critical investment in global health security, taking advantage of the revolution in vaccinology that has been catalysed by COVID-19. © Shutterstock / zabanski © Shutterstock / Atthapon Raksthaput © Shutterstock / PopT ika 04-07.qxp_Layout 1 08/04/2021 12:13 Page 26 Pharma Business International www.pbiforum.net Latest news NIH awards grants to support bacteriophage therapy research The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, has awarded $2.5 million in grants to 12 institutes around the world to support research on bacteriophage therapy. These awards represent NIAID’s first series of grants focused exclusively on research on this therapy, an emerging field that could yield new ways to fight antimicrobial- resistant bacteria. Bacteriophages (or “phages”) are viruses that can kill or incapacitate specific kinds of bacteria while leaving other bacteria and human cells unharmed. By gathering naturally-occurring phages, or by modifying or engineering phages to display certain properties, researchers hope to create novel anti-bacterial therapeutics. Because phages eliminate bacteria by infecting them, rather by generating compounds like antibiotics which kill bacteria, phages can be used to treat antibiotic-resistant infections. In addition, some evidence suggests that combination therapy containing both phages and antibiotics could prevent bacteria from becoming drug resistant. Although scientists have been aware of phages and their ability to kill bacteria since 1917 the first US-based clinical trials of phage therapy have only recently begun. Individual US patients have received phage therapy, but only under emergency investigational new drug protocols. In eastern Europe, where the use of phage therapy is more prevalent, its efficacy has not been rigorously demonstrated. The new NIAID grants support research to address key knowledge gaps in the development of phages as preventative and therapeutic tools for bacterial infections. Basic research supported by these grants will include a study characterizing different types of phages; a project studying how phages combat sticky, sheet-like colonies of microorganisms called biofilms, which can be difficult to treat with antibiotics; and research to determine how to identify new, potentially useful phages. Some translational research supported by these grants will study how to exploit the interaction between phages and bacteria to create lasting, re-usable therapeutics; and engineering viruses to combat Staphylococcus bacteria. Cancer Research UK and Teon advance new first-in-class cancer drug into clinical trial Cancer Research UK and Teon Therapeutics have signed a collaboration agreement to progress the early phase clinical development of the lattrr’s first-in-class small molecule adenosine A2B receptor antagonist, TT-702. Under the terms of the clinical development partnership, Cancer Research UK will sponsor the first-in-human Phase I/II clinical trial of TT- 702, which will be led by a team at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust. TT-702 targets cancer’s ability to evade the human immune response, enabling the immune system to expose and then destroy cancer cells. The hope is that TT-702 could be used to treat patients with advanced hard-to-treat solid tumours, including metastatic castrate-resistant prostate cancer, which is an aggressive form of cancer which has progressed despite surgery and hormone therapy. Cancer Research UK’s Centre for Drug Development will work closely with Teon throughout the collaboration and share clinical insights as they emerge to help guide TT-702’s future development strategy. Teon retains the rights to further develop and commercialise TT-702 and will receive the results of the clinical trial from Cancer Research UK in return for undisclosed success-based milestone and royalty payments. TT-702 is an adenosine receptor antagonist and specifically targets the A2B receptor, which is over expressed on various types of tumour cells and immune cells. The Centre for Drug Development, Teon, and a team of clinical investigators led by Professor Johann de Bono are currently preparing to open a first- in-human clinical trial in the second half of 2021. The researchers will test the safety and efficacy of TT-702 in multiple cancer indications, including hard-to-treat prostate cancer and triple negative breast cancer. TT-702 will be evaluated as both a monotherapy and in combination with anti-PD1 immunotherapy or hormonal therapy. © NIAID A computer-generated rendition of a bacteriophage © Shutterstock / luchschenF 04-07.qxp_Layout 1 08/04/2021 12:13 Page 3Eye on approvals US approves PONVORY for relapsing multiple sclerosis US regulators have approved PONVORY (ponesimod), Janssen’s once-daily oral selective sphingosine-1-phosphate receptor 1 (S1P1) modulator, to treat adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. The FDA approval is based, in part, on a two-year, head-to-head Phase 3 clinical trial in which PONVORY 20 mg demonstrated superior efficacy in significantly reducing annual relapses by 30.5% compared to teriflunomide (Aubagio) 14 mg in patients with relapsing MS. Lilly’s bamlanivimab-etesevimab combo granted emergency use for COVID-19 The US FDA has granted Emergency Use Authorization (EUA) for Eli Lilly’s investigational bamlanivimab (LY-CoV555) 700 mg and etesevimab (LY-CoV016) 1400 mg together for COVID-19. The therapy is authorised for the treatment of mild to moderate COVID-19 in patients aged 12 and older who are at high risk for progressing to severe COVID-19 and/or hospitalisation. Bamlanivimab and etesevimab should be administered together via a single intravenous infusion as soon as possible after a positive COVID-19 test and within 10 days of symptom onset. ViiV secures European marketing authorisation for fostemsavir Authorisation of Rukobia (fostemsavir), a first-in-class attachment inhibitor in combination with other antiretrovirals for the treatment of adults with multidrug-resistant HIV. Specifically, the treatment is approved for use in combination with other antiretroviral (ARV) therapies for the treatment of adults with multidrug-resistant HIV-1 infection, for whom it is otherwise not possible to construct a suppressive anti-viral regimen. The Marketing Authorisation Application (MAA) for fostemsavir is supported by data from the pivotal phase III BRIGHTE study, which evaluated the safety and efficacy of fostemsavir in combination with an optimised background therapy (OBT) in heavily treatment experienced (HTE) adults living with multidrug-resistant HIV, many of whom had advanced HIV disease at study entry. CDC Advisory Committee recommends first single-shot COVID-19 vaccine for US adults The US Centres for Disease Control and Prevention’s (CDC) Advisory Committee on Immunisation Practices (ACIP) has recommended the first single-shot COVID-19 vaccine – developed by the Janssen Pharmaceutical Companies of Johnson & Johnson – for individuals 18 years of age and older under the Emergency Use Authorization (EUA) issued by the US FDA. www.pbiforum.net Pharma Business International 7 Latest news UCB & Microsoft expand collaboration to accelerate drug discovery & development UCB and Microsoft have struck a new multi-year, strategic collaboration to combine Microsoft’s computational services, cloud, and AI with UCB’s drug discovery and development capabilities. As several drug discovery activities require the analysis of high-dimensional data sets or multi- modal unstructured information, Microsoft’s platform can support UCB’s scientists, including its data scientists, to discover new medicines in a more efficient and innovative way. The collaboration builds on the work UCB and Microsoft have already embarked on around COVID-19. As part of the COVID Moonshot project, UCB’s medicinal and computational chemists contributed compound designs to this worldwide open-science project to create an orally bioavailable anti-viral for COVID-19 – with the most potent series of compounds coming from UCB designs. This combination of cutting-edge science, computing power, and AI algorithms aims to significantly accelerate the iteration cycles required to explore a vast chemical space to test many hypotheses and identify more effective molecules. The collaboration plans to extend this model and identify other areas where computing power, AI, and science can accelerate the development of life changing therapies for people living with severe diseases in immunology and neurology. The work will augment UCB’s scientists, subject matter experts and research partners across every part of the drug discovery and delivery value chain by harnessing diverse research information and AI models alongside human expertise and creativity. UCB and Microsoft will explore how to combine diverse research data sets with four strategic objectives in mind, allowing UCB to: improve a patient’s overall journey; increase the impact of a treatment through a deeper understanding of the biological causes of the diseass; systematically provide better research data-driven insights to enable the faster discovery of therapeutic molecules; accelerate clinical development timelines. © Shutterstock / Gorodenkoff 04-07.qxp_Layout 1 08/04/2021 12:13 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net We begin our second mergers and acquisitions round-up of the year with the completion of Angelini Pharma’s acquisition of Arvelle Therapeutics, a Swiss-based biopharmaceutical company focused on bringing innovative treatments to patients suffering from central nervous system (CNS) disorders. $370 million has been paid with the completion of the deal, while an additional $240 million will be paid following regulatory approval. “The acquisition of Arvelle represents a milestone in our growth path,” said Pierluigi Antonelli, CEO Angelini Pharma, part of the private Italian Angelini Group, at the time of completion. Since its founding in 2019, Arvelle has been focused on bringing cenobamate (sold under the brand name Xcopri; a medication used for the treatment of partial-onset seizures in adults) to people with epilepsy in Europe. The company had held commercial rights to cenobamate under a licensing agreement with SK Biopharmaceuticals. According to Antonelli “Cenobamate showed significant efficacy, with more patients achieving a 50% or greater reduction in seizure frequency and unprecedented rates of seizure freedom of up to 21% compared to placebo.” The company now has the exclusive license to commercialise cenobamate in the EU and other countries in the European Economic Area (Switzerland and the United Kingdom). Following the deal, Angelini Pharma has become a major player in the market for the treatment of CNS and mental health disorders. Additionally, direct affiliates of Angelini Pharma will open direct affiliate in France, UK, Nordic countries and Switzerland by 2022. In early March, Takeda, the major Japanese pharmaceutical firm, announced that it was acquiring Maverick Therapeutics, a private biopharmaceutical company pioneering conditionally active bispecific T-cell targeted immunotherapies. The deal, for up to $525 million, follows an initial collaboration between the two companies to develop conditionally active T-cell engager therapies back in 2017. Through the deal, Takeda will obtain Maverick’s T-cell engager COBRA platform and a broad development portfolio, including Maverick’s lead Reinforcing, rather than diversifying, is the name of the game for the latest slew of major deals in the pharmaceutical sector. Pipeline reinforcement 10 Á 08-11.qxp_Layout 1 08/04/2021 12:14 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP © Shutterstock /Michael V i 08-11.qxp_Layout 1 08/04/2021 12:14 Page 2Next >