Your guide to the pharmaceutical world Volume 9 Issue 5 Supply chain Securing the supply chain Ethics The ethics of gene editing Business growth Bankrolling innovation PharmaBUSINESS INTERN ATION AL The total package From development to dispensing The total package From development to dispensing Supply chain Securing the supply chain Ethics The ethics of gene editing Business growth Bankrolling innovation www .pbiforum.net 01.qxp_Layout 1 12/10/2021 13:38 Page 1 INTER N Food Food &&& Food Food & N ATI O N &&Drin Drin Drin &&Drin AL nk nk nkk app A r f hh 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 TT 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 ANDSBUSINE TION ficef althy of LUTIONS ESSLINK CO U FICE w UK www.blmgroup.co.uk EMPOWER Your Business Focus on TA 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 ooo W coron F ganisa geg l b l g l h O virus na g gency ti medic Nucle Prev aaccinV The nuclea e b f treatment ve The P l t kaage n e t eelopment t v om de rF w w w di i k . p b i f o r u m . n yorkshire & lincolnshhire’s leading business mamagazine april 2020 £3.5 50 TO T RES C THE SPOND CRISIS DING S ACTOR C 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 12/10/2021 13:39 Page 1Contents Volume 9 Issue 5 12 16 20 32 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 There’s been a flurry of billion-dollar M&A activity in the pharmaceutical sector over the last two-month period with several of the biggest industry players moving into new therapeutic areas, or expanding their expertise as we explore. Supply chain12 Two of the biggest supply chain concerns for pharmaceutical companies – doubly so since the pandemic – are its fleet and the constant threat of counterfeits. Animal health16 The animal health sector continues to expand and biopharmaceutical companies are innovating - seeing new vaccines, cancer treatments, and alternatives to antibiotics come to fruition. Efficiency and optimisation20 The possibilities offered by the fourth industrial revolution to the pharmaceutical industry are among the most exciting, with 3D printing technology enabling scientists, healthcare professionals and medical manufacturers to custom print everything from synthetic bone, to pills and precision equipment. PPE 24 The essential role PPE plays is now recognised more than ever and as a result, it is evolving. Cold chain28 While new public attention is brought on the cold chain due to the distribution of vaccines during the pandemic, the room for improvement is as clear as ever. Ethics32 Gene editing remains one of the most controversial topics in medicine. Despite first emerging in the seventies, it continues to present a unique ethical quandary for the scientific and medical communities. Business growth36 Setting aside the regulatory and ethical challenges involved in new discoveries and bringing new medical and pharmaceutical products to market, there remains the issue of funding – specifically who’s bankrolling the research, materials, staff, clinical trials and so on. Events 40 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy: Shutterstock / CI Photos 03.qxp_Layout 1 12/10/2021 13:41 Page 14 Pharma Business International www.pbiforum.net Latest news Theramex launches first and only body-identical combined hormone therapy treatment for post-menopausal women Theramex has launched Bijuva® / Bijuve® (1mg estradiol / 100mg progesterone) across Europe and the UK. Bijuva® - known as Bijuve® in the UK – is the first and only body- identical hormone therapy treatment (HRT) for oestrogen deficiency symptoms in post-menopausal women with intact uterus and at least 12 months since last menses, which combines oestradiol and progesterone in a single daily oral capsule. Unlike synthetic hormone products, the oestradiol and progesterone found in Bijuva® / Bijuve® are chemically and biologically identical to the hormones naturally circulating in the woman’s body. Real-world evidence, recognised by NICE and the British Menopause Society, suggests that use of body-identical hormones is associated with lower risks compared to synthetic versions. In particular, the evidence base demonstrates that body- identical hormones have not been shown to increase the risk of venous thromboembolism and are safer forms of HRT with respect to the risk for breast cancer, heart disease, heart attack, and stroke. To find out more, visit www.theramex.com. Exscientia and Gates Foundation to develop therapeutics against coronavirus and pandemic potential viruses Exscientia, an AI-driven pharmatech company, has entered a four- year collaboration with the Bill & Melinda Gates Foundation to develop small molecule therapeutics that tackle the current coronavirus pandemic and help prepare for future pandemics. The collaboration will initially focus on developing broad-spectrum coronavirus agents (e.g., SARS-CoV-2 and its variants, MERS), including accelerating Exscientia’s lead program, which targets the main protease (Mpro) of SARS-CoV-2, the virus responsible for COVID-19. Subsequently, the collaboration will expand to develop therapeutics for influenza and Paramyxoviridae (e.g., Nipah), with the potential to develop additional programs as identified by the joint team. Exscientia’s AI-driven platform enables scientific concepts to be rapidly translated into precision-designed therapeutic candidates, with a goal of increasing probability of success and an accelerated path to the patient. Andrew Hopkins, CEO of Exscientia, said: “We believe that our AI-driven platform can accelerate the creation of better, more effective therapeutics that can address some of the world’s most critical and emerging health risks.” © Shutterstock / everything possible © Shutterstock / fizkes GentiBio raises $157m to advance engineered Treg therapies GentiBio, a biotherapeutics company developing engineered regulatory T cells (Tregs) therapies for immunology, has closed on a Series A financing generating immediate proceeds of $157 million. The financing was led by Matrix Capital Management with participation by Avidity Partners, JDRF T1D Fund, seed investors OrbiMed, RA Capital Management, and Novartis Venture Fund, and Seattle Children’s Research Institute. GentiBio, leveraging its highly differentiated platform, is able to create multiple, tunable Treg phenotypes from more abundant autologous and allogeneic cell sources that can be manufactured at scale. GentiBio has raised $177 million since 2020 to transcend technical hurdles that have limited the potential of therapeutic Tregs. Adel Nada, President and CEO of GentiBio, said: “With the Series A financing, we are focused on advancing our programs with a uniquely scalable manufacturing process that produces stable and disease specific engineered Tregs that are also tunable once infused in patients.” © Shutterstock / Saiful52 04-07.qxp_Layout 1 12/10/2021 13:43 Page 1Pharma Business International 5 www.pbiforum.net Latest news Janssen presents preliminary data of study evaluating amivantamab in combination with lazertinib The Janssen Pharmaceutical Companies of Johnson & Johnson has announced preliminary results from the Phase 1b CHRYSALIS-2 study evaluating amivantamab in combination with lazertinib in the treatment of patients with non-small cell lung cancer (NSCLC) characterised by epidermal growth factor receptor (EGFR) exon 19 deletion or L858R mutations whose disease had progressed after treatment with osimertinib and platinum chemotherapy. While previously reported results have demonstrated durable responses with amivantamab in combination with lazertinib in chemotherapy-naïve patients previously treated with osimertinib, these new data suggest that intervening chemotherapy does not impact activity with the combination. Catherine A. Shu, M.D., Clinical Director of the Thoracic Medical Oncology Service, Columbia University Herbert Irving Comprehensive Cancer Center, and presenting study investigator, said: “We are encouraged by these data showing that the combination of amivantamab and Lazertinib elicited antitumour activity, even in a heavily pre-treated patient population.” Kite and Appia Bio to develop allogeneic cell therapies for cancer Kite Pharma and Appia Bio, an early stage biotechnology company developing engineered allogeneic cell therapies from hematopoietic stem cells (HSCs) for cancer patients, will together research and develop HSC-derived cell therapies directed toward haematological malignancies. Kite, a Gilead company, and Appia Bio will develop chimeric antigen receptor (CAR)-engineered invariant natural killer T (CAR- iNKT) cells using Appia Bio’s ACUA technology platform for allogeneic cell therapy. With its ACUA technology platform, Appia Bio leverages the biology of lymphocyte development to generate CAR-iNKT cells from HSCs. The ACUA platform offers the potential for improved efficacy and safety, streamlined manufacturing, and off-the-shelf accessibility of HSC-derived CAR iNKT-cell therapies. Appia Bio will receive an upfront payment, an equity investment, and additional milestone payments for a total value of up to $875 million as well as tiered royalties. Kite will be responsible for the development, manufacturing, and commercialisation of the product candidates identified through the collaboration. © Shutterstock / Giovanni Cancemi © Shutterstock / MaximP © Shutterstock / Magic mine Eye care collaboration for AbbVie and REGENXBIO AbbVie and REGENXBIO have partnered to develop and commercialise RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases. RGX-314 is currently being evaluated in patients with wet AMD in a pivotal trial utilising subretinal delivery, and in patients with wet AMD and DR in two separate Phase II clinical trials utilising in-office suprachoroidal delivery. Under the collaboration, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314. AbbVie and REGENXBIO will collaborate and share costs on additional trials of RGX-314, including the planned second pivotal trial evaluating subretinal delivery for the treatment of wet AMD and future trials. AbbVie will lead the clinical development and commercialisation of RGX-314 globally. REGENXBIO shall participate in US commercialisation efforts as provided under a mutually agreed upon commercialisation plan. 04-07.qxp_Layout 1 12/10/2021 13:43 Page 26 Pharma Business International www.pbiforum.net Latest news Gritstone to develop COVID-19 variant vaccine with CEPI funding CEPI, the Coalition for Epidemic Preparedness Innovations, has entered into a funding agreement with Gritstone bio to advance development of a self- amplifying mRNA vaccine candidate against COVID-19 variants. CEPI will provide up to $20.6 million to support a phase 1 clinical trial, including manufacturing of clinical trial materials as well as supporting preclinical studies and optimisation of manufacturing processes. This funding forms part of CEPI’s programme to develop “next-generation” COVID-19 vaccines that are differentiated from those already in advanced development and can be used against COVID-19 variants. CEPI’s portfolio of “next-generation” vaccines currently includes SK bioscience’s nanoparticle vaccine candidate (GBP510), the University of Hong Kong’s intranasal vaccine candidate, VBI Vaccines’ virus-like particle candidate, and ZerunBio’s recombinant vaccine candidate. Dr Andrew Allen, co-founder, President and CEO of Gritstone, said: “With this unique antigenic breadth, our CORAL vaccine may offer protection against emerging spike variants of SARS-CoV-2 that appear challenging for first generation vaccines.” © Shutterstock / creativeneko Janssen presents results from Phase 1b/2 NORSE study in patients with metastatic or locally advanced urothelial carcinoma treated with erdafitinib in combination with cetrelimab The Janssen Pharmaceutical Companies of Johnson & Johnson has announced results from the Phase 1b/2 NORSE (NCT03473743) study evaluating erdafitinib in combination with cetrelimab, an anti-programmed cell death protein 1 (PD-1) monoclonal antibody, compared to erdafitinib monotherapy in patients with locally advanced or metastatic urothelial carcinoma (mUC) with fibroblast growth factor receptor FGFR3 or FGFR2 genetic alterations who are ineligible for cisplatin, a current standard of care treatment for mUC. Preliminary findings suggest robust clinical activity and depth of response in patients treated with erdafitinib in combination with cetrelimab. The overall safety of treatment with erdafitinib in combination with cetrelimab was generally consistent with erdafitinib monotherapy and aligned with the known safety profile of approved anti–PD-1 therapies. “The NORSE clinical study marks another important step forward in changing the prognosis for people living with specific genetic alterations associated with advanced bladder cancer,” said Dr Ignacio Durán, Medical Oncologist, Margues de Valdecilla University Hospital, Santander, Spain. AI therapeutics company raises $180m Deep Genomics, an artificial intelligence (AI) therapeutics company, has closed a $180 million Series C financing round. SoftBank Vision Fund 2 led the financing with participation from new investors, Canadian Pension Plan Investment Board (CPP Investments), Fidelity Management & Research Company LLC, Alexandria Venture Investments, and existing investors Amplitude Ventures, Khosla Ventures, Magnetic Ventures, and True Ventures. “This financing further validates the significant advances in our AI discovery platform and growth of our proprietary preclinical pipeline,” said Brendan Frey, founder & CEO of Deep Genomics. “It is rewarding to work with investors who recognise the long-term potential of our AI platform as we continue to identify novel targets and develop transformative medicines for patients.” Since its founding in 2015, Deep Genomics has built a suite of predictive systems known as the AI Workbench, which has made billions of predictions across the entire human genome, for millions of genetic variants, and hundreds of millions of novel compounds. © Shutterstock / VectorPot 04-07.qxp_Layout 1 12/10/2021 13:43 Page 3Eye on approvals First ophthalmology biosimilar approved in US Samsung Bioepis and Biogen have secured FDA approval for Byooviz (ranibizumab-nuna), the first ophthalmology biosimilar approved in the US. Byooviz is a biosimilar referencing Lucentis (ranibizumab) and has been approved for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), and myopic choroidal neovascularization (mCNV). In addition to the US approval, Byooviz was approved in Europe and the UK in August. Health Canada approves Moderna’s COVID-19 vaccine Health Canada has approved Moderna’s New Drug Submission (NDS- CV) for its COVID-19 vaccine. Spikevax – which has been known as COVID-19 Vaccine Moderna – is now approved for active immunisation to prevent COVID-19 in individuals 12 years of age and older. The approval is based on clinical data from the Phase 3 COVE study of the Moderna COVID-19 vaccine, which enrolled more than 30,000 participants in the US. In final analysis of Phase 3 COVE study data, Spikevax showed 93% efficacy, with the efficacy remaining durable through six months after administration of the second dose. The safety profile based on extended safety follow-up was consistent with the Phase 3 COVE study primary results. Janssen gains US approval for Invega Hafyera for schizophrenia Janssen has secured FDA approval long-acting atypical antipsychotic Invega Hafyera (6-month paliperidone palmitate), the first-and-only twice- yearly injectable for the treatment of schizophrenia in adults. The approval is based on the results of a 12-month, randomised, double-blind, non- inferiority Phase 3 global study that enrolled 702 adults (ages 18-70) living with schizophrenia from 20 countries. The results showed non- inferiority of Invega Hafyera compared to Invega Trinza on the primary endpoint of time to first relapse at the end of the 12-month period. Results found that 92.5% of patients treated with Invega Hafyera and 95% treated with Invega Trinza were relapse-free at 12 months. The safety profile observed in the trial was consistent with previous studies of Invega Sustenna and Invega Trinza with no new safety signals emerging. FDA accepts NDA for Teva and MedinCell’s schizophrenia drug US regulators have accepted Teva Pharmaceuticals and MedinCell’s New Drug Application (NDA) for TV- 46000/mdc-IRM (risperidone extended-release injectable suspension for subcutaneous use) for the treatment of schizophrenia. The acceptance is based on Phase 3 data from two pivotal studies: TV46000-CNS-30072 (the RISE Study – The Risperidone Subcutaneous Extended-Release Study) and TV46000-CNS-30078 (the SHINE Study – A Study to Test if TV-46000 is Safe for Maintenance Treatment of Schizophrenia). These studies evaluated the efficacy and long-term safety and tolerability of TV-46000 as a treatment for patients with schizophrenia. Results will be shared at future scientific conferences and in peer-reviewed publications. European approval for UCB’s Bimzelx for plaque psoriasis UCB has secured European approval for Bimzelx (bimekizumab) for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. Bimekizumab is the first approved treatment in the European Union (EU) for moderate to severe plaque psoriasis that is designed to selectively and directly inhibit both IL-17A and IL-17F, two key cytokines driving inflammatory processes. Bimekizumab is approved at a recommended dose of 320 mg, administered by two subcutaneous injections every four weeks to week 16 and every eight weeks thereafter. AbbVie secures European approval for Rinvoq AbbVie has secured European approval for Rinvoq (upadacitinib), an oral, selective and reversible JAK inhibitor, for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy. The approval from the European Commission (EC) is supported by data from one of the largest registrational Phase 3 programs in atopic dermatitis with more than 2,500 adults and adolescents with moderate to severe disease. UK authorises Regeneron antibody cocktail for acute COVID-19 The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted Conditional Marketing Authorisation (CMA) to Regeneron for the casirivimab and imdevimab antibody cocktail to prevent and treat acute COVID-19 infection. In addition to the CMA, which affects people in England, Scotland and Wales, the MHRA has also authorised emergency supply of the antibody cocktail – known as REGEN-COV in the US and Ronapreve in the UK and other countries – to prevent and treat acute COVID-19 infection for people in Northern Ireland. www.pbiforum.net Pharma Business International 7 04-07.qxp_Layout 1 12/10/2021 13:43 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net © Shutterstock /HJBC Expansion, expansion, expansion There’s been a flurry of billion-dollar M&A activity in the pharmaceutical sector over the last two-month period with several of the biggest industry players moving into new therapeutic areas, or expanding their expertise. 08-11.qxp_Layout 1 12/10/2021 13:45 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP Starting with a bang this issue with the headline announcement that Sanofi, the French-headquartered big ten pharma firm, is acquiring Translate Bio for $3.2 billion. Announced in early August, the deal for the Lexington, Massachusetts-based messenger RNA (mRNA) therapeutics company advances Sanofi’s deployment of mRNA to develop therapeutics and vaccines. The acquisition builds on Sanofi’s establishment of a first-of-its kind vaccines mRNA Centre of Excellence and its $470 million acquisition of Tidal Therapeutics back in April 2021. “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 of the deal. “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.” In June 2018, Sanofi and Translate Bio entered into a collaboration and exclusive license agreement to develop mRNA vaccines which was further expanded in 2020 to broadly address current and future infectious diseases. 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 MRT platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as oncology. Less than a month later, Sanofi announced that it was further strengthening the growth of its transplant business with the $1.9 billion acquisition of Kadmon Holdings, a US biopharmaceutical company that discovers, develops, and markets transformative therapies for disease areas of significant unmet medical needs. The acquisition supports Sanofi’s strategy to continue to grow its General Medicines core assets and will immediately add Rezurock (belumosudil) © Shutterstock /diy13 10 Á 08-11.qxp_Layout 1 12/10/2021 13:45 Page 2Next >