Your guide to the pharmaceutical world Volume 9 Issue 4 Respiratory disease Respiratory disease research races ahead Regulation Changing hurdles Labelling Counteracting counterfeits PharmaBUSINESS INTERN ATION AL The total package From development to dispensing The total package From development to dispensing Respiratory disease Respiratory disease research races ahead Regulation Changing hurdles Labelling Counteracting counterfeits www .pbiforum.net 01.qxp_Layout 1 13/08/2021 11:06 Page 1SCAN TO REGISTER ppmashow.co.uk See the latest processing equipment, packaging machinery, robotics and machine vision in action Gain a competitive advantage through the latest innovations and seminars NEC, BIRMINGHAM WHERE MATTERS 02.qxp_Layout 1 13/08/2021 11:07 Page 1Contents Volume 9 Issue 4 14 20 24 36 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 Major deals abound in this issue’s M&D round-up, including the completion of 2020’s biggest acquisition in the biopharma space. A Life in a Day Q&A12 We spoke with Mark Doyle, co-founder of The Method and creator of the innovative learning and development programme, A Life in a Day. Respiratory diseases14 Pharma Business International details some of the most recent breakthroughs in respiratory disease research. uMed Q&A18 We spoke with Dr Matt Wilson, founder of uMed about the company’s mission of helping GPs and their patients take part in clinical research, as well as digital automation and much, much more. Regulation20 With all the changes caused by COVID-19, how has the regulatory market fared? Will there be any changes in the future? Labelling24 Advances in labelling are helping to stem the rising tide of counterfeit medicines, securing the supply chain and keeping patients safe. Distribution28 Perhaps more than any other technology, blockchain is taking pharmaceutical and medical distribution into the future. Controlling COVID32 As new variants and waves of COVID-19 spread across the world, vaccinations and testing are crucial to managing the virus - especially as countries look to lift restrictions. Automation36 Keeping up with changes in manufacturing can be difficult, especially in the midst of a pandemic, which is why so many outsource such concerns to others. PPMA Show 2021 40 Taking place at the NEC, Birmingham, from 28th-30th September 2021, the exhibition showcases the latest innovations in processing and packaging machinery Events 42 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy: Shutterstock / Image Point Fr 03.qxp_Layout 1 13/08/2021 11:07 Page 14 Pharma Business International www.pbiforum.net Latest news Origin Group launches 100% recyclable single- dose sachet for the health sector The Origin Group’s new business unit, SnappD is now offering the UK’s first 100% recyclable single-dose sachet which is fully brandable for the health sector. SnappD is a single-dose pouch designed for the hygienic and accurate dispensing of liquid and semi-liquid medicines and healthcare products, including painkiller, antiseptic cream, and vitamin supplements. The sachets can be opened with a single hand movement. As well as being convenient for the user, this new concept helps to address the challenges of the “sachet economy” which continues to generate massive quantities of plastic waste globally. There’s currently no legislation in place in the UK or EU to tackle the environmental impact of plastic sachets, but pharmaceutical and healthcare brands need to think ahead of the curve to avoid costly compliance challenges in future and to keep in line with changing legislation and consumer views on sustainability. SnappD sachets, made with more than 30% Post-Consumer-Recycled materials (PCR), are manufactured and precision filled in an ISO 22716 environment, within a purpose-built room which simulates that of a cleanroom facility, in Melton, East Riding of Yorkshire. SnappD also delivers eco-savings during transportation. The size and shape of SnappD sachets mean large quantities can be easily transported with minimal space wastage. For example, 250 Snapped 50x50 sachets can fit in 1 POS box – totalling up to 108,000 in one pallet. Designed for universal ease of use, the SnappD patented sachet can be used as part of brands’ current offering, or as samples to increase brand reach and attract new customers. Customers can choose from a variety of customised opening functions for liquids, semi-liquids, and high viscosity products. Additionally, there is a double-cut option for brands to package two products in a dual-chamber, to be dispensed together. Any sachet size between 40x50 mm and 100x100 mm is possible, delivering a minimum filling volume of 0.2 ml and up to 40 ml depending on the product category. Pharmaceutical-grade filling system is offered for filling volumes below 0.5 ml that require more precision, and for higher shelf-life requirements, barriers of aluminium and siox silica coating are available. SnappD’s in-house design technologists offer personal consultations to advise on the best options, using a 3D visualiser tool to maximise visual impact and reduce project timelines. Rich Quelch, Global Head of Marketing at SnappD, said: “Building on The Origin Group’s heritage of manufacturing exceptional glass packaging, plastic containers, measuring devices and child-resistant solutions, we decided that one-dose sachets were a logical extension of our product range. Created in response to the increased demand for hand sanitiser during the coronavirus pandemic, SnappD was subsequently redesigned by our engineers to suit a wide range of applications and sectors, while supporting global efforts to reduce environmental harm from single-use plastic sachets.” Takeda and Frazier forge new company to develop norovirus vaccine candidate Takeda and Frazier Healthcare Partners are together launching HilleVax, a biopharmaceutical company to develop and commercialise the former’s norovirus vaccine candidate. Takeda has granted a license to HilleVax for the exclusive development and commercialisation rights to its norovirus vaccine candidate, HIL-214 (formerly TAK-214), worldwide outside of Japan. Takeda will retain commercialisation rights in Japan and HilleVax will integrate certain Japan development activities into its global development. Takeda remains committed to vaccines and this collaboration allows Takeda to focus primarily on dengue, COVID-19, pandemic influenza and Zika. HIL-214, which is a virus-like particle (VLP) based vaccine candidate, completed a randomized, placebo-controlled Phase 2b field efficacy study in 4,712 adult subjects in which HIL-214 was well-tolerated and demonstrated clinical proof of concept in preventing moderate-to-severe cases of acute gastroenteritis from norovirus infection. To date, the candidate has been studied in nine human clinical trials with safety data from over 4,500 subjects and immunogenicity data from over 2,000 subjects. © Shutterstock / Kateryna Kon 04-07.qxp_Layout 1 13/08/2021 11:09 Page 1Biocon to develop antibody for COVID via Adagio partnership Biocon Biologics, a subsidiary of Biocon, has secured an exclusive licence from Adagio Therapeutics to manufacture and commercialise an antibody treatment based on ADG20 for India and select emerging markets. ADG20, a novel monoclonal antibody targeting the spike protein of SARS-CoV-2 and related coronaviruses, is in global clinical development by Adagio as a single agent for both the treatment and prevention of COVID-19, its variants, as well as future variants that may emerge. Initial data indicate that ADG20 could provide both rapid and durable protection against COVID-19 for up to one year. With its potential to address resistant variants, including the Delta variant, and its ability to be administered easily as a single, intramuscular injection in the outpatient setting, ADG20 is uniquely poised to address the current need for an effective, safe and convenient therapy for COVID-19. Pharma Business International 5 www.pbiforum.net Latest news Prime Medicine launches with $315m to deliver on promise of Prime Editing Prime Medicine, a company delivering on the promise of Prime Editing to provide lifelong cures to patients, has launched this month with $315 million in financing. The financing comprised a $115 million Series A; based on the rapid progress of the science and the company, Prime Medicine expanded its syndicate support with a $200 million Series B financing approximately nine months after the company began operations. The funds will be used to continue building the company, rapidly advance towards clinical indications, expand the capabilities of the platform, and to further enhance the exceptional promise of Prime Editing. Prime Editing is a next-generation gene editing technology that acts like a DNA word processor to “search and replace” disease-causing genetic sequences at their precise location in the genome, without resulting in double-strand DNA breaks that cause unwanted cellular changes. By the end of 2021, Prime Medicine expects to employ more than 100 people full-time. Regeneron to invest $1.8bn to extend Westchester County campus Regeneron, New York’s largest biotech company, will invest approximately $1.8 billion over six years to expand its research, preclinical manufacturing and support facilities at its Westchester County campus in Tarrytown and create 1,000 new full-time, high- skill jobs in the Mid-Hudson Region over the next five years. The company intends to substantially expand its research and development capabilities in Tarrytown, which will include the addition of new preclinical manufacturing and process development suites, laboratories and office space. This project will encompass the design and construction and fit out of up to eight buildings, three parking garages and a central utility plant totalling approximately 900,000 square feet. Road infrastructure improvements will be necessary to support additional buildings at their existing Headquarters and R&D campus. The project is planned to take place in 2 phases over 6 years. © Shutterstock / Gorodenkoff © Shutterstock / angellodeco © Shutterstock / Eetu Mustonen 04-07.qxp_Layout 1 13/08/2021 11:09 Page 26 Pharma Business International www.pbiforum.net Latest news GSK unveils plan for one of Europe’s largest life science campuses GlaxoSmithKline (GSK) has unveiled plans to develop one of Europe’s largest life science campuses. Specifically, the company is looking to transform land within its existing 92-acre R&D site in Stevenage, England into a major cluster for biotech and other early-stage life science companies. The vision for the new campus has been developed in partnership with Stevenage Bioscience Catalyst (SBC), the UK Government, Stevenage Borough Council and the Local Enterprise Partnership (LEP), and would build on the existing presence of GSK, the Cell and Gene Therapy Catapult and the SBC on the site. As part of proposals, GSK is looking to sell the 33 acres of land, with an aim to subsequently unlock up to £400 million in new investment from a private sector developer to build the new campus and potentially create up to 5,000 highly-skilled jobs, over the next five to ten years. The company expects to select a development partner later this year, with a view for work to begin on master planning for the new campus in 2022. © Shutterstock / Golden Shrimp Verge Genomics & Lilly to develop Novel ALS treatments Verge Genomics, a biotech company pioneering all-in-human, artificial- intelligence-powered drug discovery and development, has entered a collaboration with Eli Lilly to research and develop novel therapies for the treatment of amyotrophic lateral sclerosis (ALS). Under the terms of the three-year agreement, Verge will receive up to $25 million in upfront, equity investment and potential near-term payments, with additional milestone value of $694 million and potential downstream royalties. In this collaboration, Verge will apply its all-in-human platform to discover and validate new targets for ALS. The all-in-human platform is based on a proprietary collection of patient brain transcriptomes across a variety of neurodegenerative diseases. Through its application, the all-in-human platform provides insights into novel causal disease mechanisms in genetically segmented patient populations, and enables the discovery of therapeutic targets. Based on these insights, Verge will apply its human-based discovery capabilities to validate targets. Lilly will select up to four targets identified by Verge with plans to advance through clinical development and commercialisation. Sanofi launches dedicated vaccines mRNA Center of Excellence Sanofi is to invest €400 million annually in a first-of-its kind vaccines mRNA Center of Excellence. The Center will work to accelerate the development and delivery of next-generation vaccines by bringing together approximately 400 dedicated employees integrating end-to-end mRNA vaccine capabilities with dedicated R&D, digital, and chemistry, manufacturing and controls (CMC) teams across sites at Cambridge, MA (US) and Marcy l’Etoile, Lyon (France). Jean-Francois Toussaint, Global Head of Research and Development, Sanofi Pasteur, said: “During the COVID-19 pandemic, mRNA technologies demonstrated potential to deliver new vaccines faster than ever before. “However, key areas of innovation such as thermostability and tolerability improvements will be critical to unlock the applications of mRNA in routine vaccination against a broader set of infectious diseases and across all ages.” The Center of Excellence will enable acceleration of the vaccines mRNA portfolio developed through the Translate Bio collaboration established in 2018 and expanded in 2020. © Shutterstock / HJBC © Shutterstock / Green Apple 04-07.qxp_Layout 1 13/08/2021 11:09 Page 3Eye on approvals Skysona becomes first and only gene therapy approved in EU for CALD Skysona has become the first and only gene therapy approved in the EU to treat early Cerebral Adrenoleukodystrophy (CALD), a rare neurodegenerative disease that can lead to progressive, irreversible loss of neurologic function, and death. The European Commission (EC) granted marketing authorisation for Skysona (elivaldogene autotemcel, Lenti-D) to bluebird bio for the treatment of early CALD in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. Europe approves Aubagio as the first oral MS therapy for paediatric patients The European Commission has approved Sanofi’s Aubagio (teriflunomide) for the treatment of paediatric patients 10 to 17 years of age with relapsing-remitting multiple sclerosis (RRMS). Based on data from the Phase 3 TERIKIDS study, the approval confirms Aubagio as the first oral multiple sclerosis (MS) therapy for first-line treatment of children and adolescents with MS in the European Union. Aubagio was initially approved in the EU in 2013 for the treatment of adult patients with RRMS and the EC approval for the paediatric indication provides an additional year of marketing protection in the European Union. Comirnaty receives first authorisation in EU for COVID vaccine in adolescents Conditional Marketing Authorisation (CMA) for Pfizer and BioNTech’s Comirnaty has been expanded to included individuals 12 to 15 years of age in the EU. This follows the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion to authorize the vaccine in this age group. The extended indication for the CMA for Comirnaty is valid in all 27 EU member states. Comirnaty was the first COVID-19 vaccine to receive authorisation in the EU and is the first to have its CMA extended to adolescents. Distribution and administration by the EU member states will continue to be determined according to the populations identified in the EU and per national guidance. Europe approves Ponvory for elapsing multiple sclerosis European regulators have approved Janssen’s Ponvory (ponesimod) for the treatment of adult patients with relapsing multiple sclerosis (RMS) with active disease defined by clinical or imaging features. The European Commission approval of ponesimod is based on data from the Phase 3 OPTIMUM trial, a multicentre, randomised, double-blind, parallel-group, active-controlled superiority study of 1,133 adult patients (aged 18-55 years) in 28 countries. The EC approval follows the positive CHMP opinion for ponesimod in March 2021 and the announcement of the U.S. Food and Drug Administration (FDA) approval of ponesimod for use in adults with relapsing forms of MS. Roche’s Actemra/RoActemra receives FDA Emergency Use Authorisation The US FDA has issued an Emergency Use Authorization (EUA) to Roche for intravenous Actemra/RoActemra (tocilizumab) for the treatment of COVID-19 in hospitalised adults and paediatric patients (two years of age and older) who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). The EUA is based on results from four randomised, controlled studies that evaluated Actemra/RoActemra for the treatment of COVID-19 in more than 5,500 hospitalised patients. The results of these studies suggest that Actemra/RoActemra may improve outcomes in patients receiving corticosteroids and requiring supplemental oxygen or breathing support. Alkermes secures FDA approval of Lybalvi for schizophrenia and bipolar I disorder Alkermes has secured US FDA approval for Lybalvi (olanzapine and samidorphan) for the treatment of adults with schizophrenia and for the treatment of adults with bipolar I disorder, as a maintenance monotherapy or for the acute treatment of manic or mixed episodes, as monotherapy or an adjunct to lithium or valproate. Lybalvi is a once-daily, oral atypical antipsychotic composed of olanzapine, an established antipsychotic agent, and samidorphan, a new chemical entity. Alkermes expects to make Lybalvi available for patients in the fourth quarter of 2021. FDA expands authorised use of REGEN-COV The US FDA has updated the Emergency Use Authorization (EUA) for Regeneron’s investigational COVID-19 antibody cocktail REGEN-COV (casirivimab and imdevimab). The authorization now includes post-exposure prophylaxis in people at high risk for progression to severe COVID-19, who are not fully vaccinated or are not expected to mount an adequate response to vaccination, and have been exposed to a SARS-CoV-2 infected individual, or who are at high risk of exposure to an infected individual because of infection occurring in the same institutional setting (such as in nursing homes or prisons). In those who require repeat dosing for ongoing exposure, REGEN- COV can also now be administered monthly. www.pbiforum.net Pharma Business International 7 04-07.qxp_Layout 1 13/08/2021 11:09 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net Starting this issue’s mergers and acquisitions round-up with a bang, AstraZeneca has completed its historic $39 billion takeover of rare diseases specialist, Alexion. The deal was originally announced in December last year, making it 2020’s biggest in the biopharmaceutical space. It sees Alexion apply its complement-biology platform across areas of AstraZeneca’s broader early stage pipeline. AstraZeneca is betting big on rare diseases in what CEO Pascal Soriot has dubbed “a new chapter” for the business. “Our sustained R&D investment in oncology, cardiovascular and renal, as well as respiratory and immunology, has powered AstraZeneca’s transformation and now we add rare diseases, where fewer approved treatment options exist,” he said. Next up, Fortune 500 science and technology innovator, Danaher Corporation, has entered into a definitive agreement to acquire privately-held Aldevron for $9.6 billion. Founded in 1998 by Michael Chambers and John Ballantyne, Fargo, North Dakota-headquartered Aldevron manufactures plasmid DNA, mRNA, and proteins, serving biotechnology and pharmaceutical customers across research, clinical and commercial applications. The deal will see Aldevron will operate as a standalone operating company and brand within Danaher’s Life Sciences segment. Rainer M. Blair, Danaher President and CEO, said the deal will expand the company’s capabilities “into the important field of genomic medicine and help us support our customers and their critical mission to bring more life- saving therapies and vaccines to market faster.” Back to the top ten big pharma firms where Sanofi is accelerating its mRNA deployment with the $3.2 billion acquisition of Translate Bio. The company is aggressively fortifying its mRNA capabilities. This latest deal follows only a few months after the company swooped for Tidal Therapeutics in a deal worth up to $470 million. That acquisition further accelerates Sanofi’s efforts to develop transformative A flurry of activity Major deals abound in this issue’s M&D round- up, including the completion of 2020’s biggest acquisition in the biopharma space. 08-11.qxp_Layout 1 13/08/2021 11:10 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP medicines using mRNA technology. This latest deal builds on the collaboration and exclusive license agreement struck with Translate Bio in 2018. The agreement, to develop mRNA vaccines, was further expanded in 2020 to broadly address current and future infectious diseases. There are two ongoing mRNA vaccine clinical trials under the collaboration, the COVID- 19 vaccine Phase 1/2 study with results expected in Q3 2021 and the mRNA seasonal influenza vaccine Phase 1 trial with results due in Q4 2021. The acquisition builds on Sanofi’s establishment of a first-of-its kind vaccines mRNA Centre of Excellence. On the therapeutic side, Translate Bio has an early-stage pipeline in cystic fibrosis and other rare pulmonary diseases. In addition, discovery work is ongoing in diseases that affect the liver, and Translate Bio’s MRTTM platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as oncology. “Translate Bio adds an mRNA technology platform and strong capabilities to our research, further advancing our ability to explore the promise of this technology to develop both best-in-class vaccines and therapeutics,” said Sanofi CEO Paul Hudson. “A fully owned platform allows us to develop additional opportunities in the fast-evolving mRNA space. We will also be able to accelerate our existing partnered programs already under development. Our goal is to unlock the potential of mRNA in other strategic areas such as immunology, oncology, and rare diseases in addition to vaccines.” August also saw Amgen announce its © Shutterstock /Michael V i © Shutterstock /HJBC 10 Á As we were finishing with this issue, news broke that Bayer was acquiring US biopharmaceutical company, Vividion Therapeutics. The deal, worth up to $2 billion, sees Bayer strengthen its drug discovery platform, by taking charge of Vividion’s platform, which can produce a variety of small molecule therapies across indications. 08-11.qxp_Layout 1 13/08/2021 11:10 Page 2Next >