Your guide to the pharmaceutical world Volume 12 Issue 5 Logistics Finding the right balance Machinery and equipment More than a machine Environmental Responding to the crisis Logistics Finding the right balance Machinery and equipment More than a machine Environmental Responding to the crisis www.pbiforum.net NTERN NATION AL Fut pro Fut ture fi g coding bility ce g Labelling, c and traaceab Food scienc Environmen spotlight ntal agingkc aa P rld o our guide to the pharmaceutical wY Issue 4 1olume 1 V mpeeee T profitable Sustainable, ature er bl a Unyielding i contro em l ll a Handlingth handli M net www .pbiforum.ne SEPTEMBER 2023 EAST MIDLANDS LEA £3.50 EADING BUSINESS MAGA AZINE een going gr WW The business case eo DMS Comms, Collab WW the barriers Organisatio bs and ANDSBUSINE rstoexceence ning - onal lear ESSLNKCOU w UK .co.uk www EMPOWER Your Business T OCTOB £2.95 BER2023 BreastC Hair & B Cce Bty WithLou In Love Month Awaren B th BLM GROUP www.blmgroup.co.uk Heritage Lincolns hi WWW.LINCWWW.LINC OLNSHIRETOOLNSHIRETO ODAYNEODAYNE september 2023 yorkshire & lincolnsh £3.50 shire’s leading business ma magazine VOLUME38SSUE09 .blmgroup.co.ukwww organ c Over Automation driving adaption success Driving business ning isational lear comngbaeso V w Often Imitated - Never Equalled www.blmgroup.co.uk4 12 20 24 Steve Fisher (s.fisher@blmgroup.co.uk) Michael Fisher (m.fisher@blmgroup.co.uk) Tess Egginton (t.egginton@blmgroup.co.uk) Angie Cooper (a.cooper@blmgroup.co.uk) Tel: +44 (0) 1472 310302 Angela Sharman (a.sharman@blmgroup.co.uk) Gary Jorgensen, Mark Casson, (studio@blmgroup.co.uk) Tel: +44 (0) 1472 310305, Fax: +44 (0) 1472 310317 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 Since our last issue, a flurry of mergers and acquisitions have been revealed within the pharmaceutical industry, with the focus on smaller transactions. Traceability12 Proper traceability systems not only help prevent a crisis, but also put criminals on the backfoot. Machinery and equipment16 Automation is about more than replacing human workers with machines – it is about achieving more than humans ever could and creating new opportunities for the company because of it. Vision, inspection and detection20 Quality assurance and health and safety both rely on good inspection, and we are long past the point where the human eye can be of any practical use in a factory setting. As demand for machine vision systems increase however, the complexity of the task rises in tandem. Environmental24 Reducing environmental waste and greener business has been a buzzword for years but recent disasters made worse by climate change may force it into reality. Logistics28 Lean, efficient, resilient. That’s the focus of logistics and transport nowadays, and one that has to be considered for the future. Events 32 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy: stock.adobe.com/ Mike Mareen4 Pharma Business International www.pbiforum.net Latest news Genespire raises €46.6m to advance its first pediatric in-vivo gene therapy into the clinic Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, has closed a €46.6 million Series B financing, co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital through its Large Venture Fund forming a strong syndicate with Indaco SGR. The financing will enable the development of GENE202, the company’s lead candidate, up to a Phase I/II clinical trial for the treatment of MMA, a devastating genetic disorder impairing the metabolism of certain amino acids and fats. MMA has onset in early infancy, is associated with significant mortality and morbidity, and is characterized by severe clinical manifestations including muscular weakness, seizures, developmental delays and organ damage. Currently, there are no disease-modifying treatments for MMA. GENE202 is a pioneering off-the-shelf gene therapy which harnesses the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLVs are designed to be used intravenously, enabling the patient’s liver to produce the therapy throughout its lifetime. Orbis Medicines and Vivtex form research & exclusive licensing collaboration to support next- generation orally dosable ‘nCycles’ Orbis Medicines, a leader in oral macrocycle drug discovery, has announced a research collaboration and option to an exclusive license with Vivtex, a biotech company aiming to transform the development of oral biologic therapies for major diseases. The collaboration will enable Orbis to utilize Vivtex’s GI-ORIS™ platform for gut permeability screening to evaluate the oral bioavailability of Orbis’ portfolio of next-generation macrocycle drugs it calls ‘nCycles’. “This collaboration extends our ability to design for oral bioavailability into later stages of preclinical development with Vivtex’s extensively validated ex vivo porcine gut model, which is highly informative for human gut interactions. Our goal is faster development of the highest quality candidates possible for clinical entry as we work towards bringing a new generation of macrocycle drugs to patients,” said Simon Feldbæk, Managing Director and Chief Development Officer at Orbis. Under the agreement, the companies will collaborate on permeability screens of Orbis’ nCycles. nCycles are systematically designed by Orbis’ “nGen” platform to be orally bioavailable and membrane permeable, both decades-long challenges in macrocycle drug design. ILC Therapeutics raises £2.5m ILC Therapeutics, a specialist developer of hybrid interferons, a new drug class engineered for improved therapeutic profiles across multiple indications, has successfully completed a fundraise of £2.5m. The fundraise, led by St Andrews-based impact investment firm Eos, Scottish Enterprise, Medical Incubator Japan and Los Angeles-based lawyer and investor Robert Kopple, will support the continued pre-clinical development of the company’s two lead assets, Alfacyte™ and Dermacyte™ as they move towards the clinic. Alfacyte™ is being developed for the treatment of upper respiratory tract infections and, as a broad-spectrum antiviral, is a promising novel therapeutic to tackle the threat of future pandemics, including coronaviruses. Alfacyte™ has demonstrated activity against multiple viruses and is currently being developed for respiratory syncytial virus (RSV), with further proof-of-concept studies intended in other indications. Dermacyte™ represents a novel approach to psoriasis, with a multi-pathway and multi-cellular mode of action. Wide-ranging in vitro data has shown Dermacyte™ inhibits key cytokines and chemokines involved in psoriasis, with in vivo proof-of-concept studies now underway. © stock.adobe.com/ immimagery © stock.adobe.com/ Viktoriia © stock.adobe.com/ killykoonPharma Business International 5 www.pbiforum.net Latest news NanoVation Therapeutics partners with Novo Nordisk to develop genetic medicines targeting cardiometabolic and rare disease NanoVation Therapeutics, a platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Novo Nordisk to advance the development of novel genetic medicines targeting cardiometabolic and rare diseases. The partnership combines NanoVation Therapeutics’ proprietary long-circulating lipid nanoparticle (lcLNP™) technology for RNA delivery to cells outside of the liver with Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation. Under the terms of the agreement, Novo Nordisk and NanoVation will collaborate on two lead programs to develop base-editing therapies for certain rare genetic diseases, and up to five additional future targets for cardiometabolic and rare diseases. Novo Nordisk will receive a defined exclusive, worldwide license to use NanoVation’s LNP technology for the two lead programs. NanoVation will receive research funding and is eligible to receive up to approximately US$600 million in up-front cash and potential milestone payments, as well as tiered royalties on future product sales as part of the multi-year deal. © stock.adobe.com/ Scott Cornell New Advanced Therapy Treatment Centre established in London to improve clinical trial infrastructure The Cell and Gene Therapy Catapult (CGT Catapult) and the Advanced Therapy Treatment Centre (ATTC) network have welcomed a new centre, the London Advanced Therapies ATTC. The network will work to accelerate the delivery of cell and gene therapy clinical trials, aiming to help more patients access transformative treatments. The London Advanced Therapies ATTC is made up of its four partner organisations: Imperial College London, King’s College London, Queen Mary University of London and University College London (UCL), and their NHS Trusts. It will build on the reputation of these world-leading institutions, and of London as a whole, as a global hub for advanced therapies research and commercial clinical trials. The focus of the centre will be working with the healthcare providers connected to its partner organisations to improve the infrastructure for advanced therapy clinical trials. This aims to increase the number and speed of trials as well as help more patients take part in these trials and access potentially life-changing cell and gene therapies. © stock.adobe.com/ Sebastian Kaulitzki Orion and Aitia enter AI-driven drug discovery and drug simulation collaboration in oncology Orion Corporation and Aitia have entered into an agreement to create new Gemini Digital Twins to help in the fight against cancer. The global cancer burden is projected to rise significantly, with new cases expected to reach 35 million annually by 2050 if current trends continue, driven by population aging and growth. This growing challenge underscores the urgent need for innovative treatments. Under the terms of the agreement, Orion and Aitia will work together to focus on translational questions to discover and validate novel drug targets, and to develop drug candidates across multiple oncologic indications through the utilisation of Aitia’s Digital Twins combined with Orion’s pre-clinical and clinical data and expertise. Orion will receive an exclusive option to research, develop, and commercialise products directed against specific novel drug targets identified by Aitia. In addition, Aitia is eligible to receive certain upfront payments, and development and regulatory milestone payments totalling to more than USD 10 million per drug target, as well as tiered single-digit royalties on sales of products covered by the agreement. © stock.adobe.com/ kittyfly6 Pharma Business International www.pbiforum.net Latest news Agios’ tebapivat granted FDA Orphan Drug Designation for treatment of myelodysplastic syndromes The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Agios Pharmaceuticals’ novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS). “Receiving Orphan Drug Designation for tebapivat in MDS underscores the importance of bringing new oral treatment options to patients suffering from this rare disease,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head R&D at Agios. “We aim to deliver the first oral therapy that addresses anemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000-80,000 patients in the U.S. and EU5 and accounts for approximately 70% of MDS cases.” Agios completed a Phase 2a study of tebapivat in lower-risk MDS late last year and is currently initiating a Phase 2b study of tebapivat in lower-risk MDS. Under the Orphan Drug Act, Orphan Drug Designation qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval. U.S. FDA grants Orphan Drug Designation to Actimed Therapeutics’ ALS treatment The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Actimed Therapeutics’ S-oxprenolol (ACM-002) for the treatment of amyotrophic lateral sclerosis (ALS). ALS (also known as Lou Gehrig’s disease) is a progressive, fatal neuromuscular disease that affects nerve cells in the brain and spinal cord, eventually leading to a loss of muscle control and paralysis. ALS prevents motor neurons sending impulses from the brain and spinal cord to the muscles, causing muscle atrophy and weakness and has a mean survival of 3.5 years. Voluntary muscle action is progressively lost and in late-stage ALS patients may have difficulty communicating, swallowing, moving, and breathing. There is currently no cure for ALS. Cachexia, a complex wasting disease, may occur in the early stages of ALS and has a significant impact on quality of life, proving terminal in some patients. In pre-clinical ALS models, S-oxprenolol has demonstrated significant benefits including slowing disease progression, improvement in survival, attenuation of muscle loss, motor neuron protection, reduction in loss of body mass and improvements in lean body mass. $1.8bn investment sees Lilly expand manufacturing footprint in Ireland Eli Lilly and Company has announced a $1 billion expansion of its Limerick, Ireland, manufacturing site to increase production of biologic active ingredients, including those for its recently approved treatment for early symptomatic Alzheimer’s disease. The company also unveiled its new $800 million facility expansion in Kinsale, Ireland, which began making medicines last year to meet demand for Lilly’s latest diabetes and obesity treatments. This additional investment is part of the most ambitious manufacturing expansion agenda in the company’s history. Since 2020, Lilly has committed more than $20 billion to build, expand and acquire manufacturing facilities in the U.S. and Europe. “These investments will boost the production of some of our medicines, helping millions of people with diabetes, obesity and Alzheimer’s disease live the healthiest lives possible,” said Edgardo Hernandez, executive vice president and president of Lilly Manufacturing Operations. “We won’t stop there – these state-of-the-art facilities will also be equipped to support our promising pipeline molecules of the future.” © stock.adobe.com / Nuttapong punna © stock.adobe.com/ PH alex aviles © stock.adobe.com/ Tada Images European Commission approves LEO Pharma’s Anzupgo cream for adults with moderate to severe chronic hand eczema The European Commission (EC) has granted marketing authorization for LEO Pharma’s Anzupgo (delgocitinib) cream for the treatment of adult patients with moderate to severe chronic hand eczema (CHE) for whom topical corticosteroids are inadequate or inappropriate. CHE is a heterogeneous, fluctuating, inflammatory skin disease with key symptoms of itch and pain. Its pathophysiology is characterized by skin barrier dysfunction, skin inflammation, and skin microbiome alterations. The condition can have a high psychological, social, and occupational burden. Anzupgo is a topical pan-JAK inhibitor for the treatment of moderate to severe CHE. It inhibits the activation of JAK-STAT signaling, which plays a key role in the pathogenesis of CHE. Currently, there are no other topical treatments specifically approved for adults with moderate to severe CHE for whom topical corticosteroids are inadequate or inappropriate. Fresenius Kabi and Formycon receive U.S. FDA approval for biosimilar Otulfi The U.S. Food and Drug Administration (FDA) has approved Fresenius Kabi and Formycon’s Otulfi (ustekinumab-aauz), the ustekinumab biosimilar referencing Stelara (ustekinumab). Otulfi is approved for the treatment of Crohn’s disease, ulcerative colitis, moderate to severe plaque psoriasis and active psoriatic arthritis. Ustekinumab is a human monoclonal antibody that targets the cytokines interleukin-12 and interleukin-23 which play an important role in inflammatory and immune responses. The FDA approval of Otulfi (ustekinumab-aauz) is based on a thorough evaluation of a comprehensive data package including analytical, pre-clinical, clinical and manufacturing data. Otulfi demonstrated comparable efficacy, safety, pharmacokinetics and immunogenicity to the reference drug Stelara in patients with moderate to severe psoriasis vulgaris (plaque psoriasis). Otulfi was approved for both subcutaneous and intravenous formulations which will offer a comprehensive, alternative treatment solution for health care professionals and patients treated with ustekinumab in the U.S. U.S. FDA approvals for UCB’s BIMZELX for psoriatic arthritis, non-radiographic axial spondyloarthritis and ankylosing spondylitis The U.S. Food and Drug Administration (FDA) has approved UCB’s BIMZELX (bimekizumab-bkzx) for the treatment of adults with active psoriatic arthritis (PsA), adults with active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation, and adults with active ankylosing spondylitis (AS). Bimekizumab-bkzx is the first approved treatment for these three indications that is designed to selectively inhibit two key cytokines driving inflammatory processes – interleukin 17A (IL-17A) and interleukin 17F (IL-17F). These newly approved indications follow the first U.S. approval for BIMZELX in October 2023 for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. “The approval of BIMZELX in the U.S. across three new indications – active psoriatic arthritis, active non-radiographic axSpA with objective signs of inflammation, and active ankylosing spondylitis – highlights the clinical benefit of dual inhibition of both IL-17A and IL-17F for patients, and provides an opportunity for more people living with chronic inflammatory diseases to achieve meaningful outcomes,” said Emmanuel Caeymaex, executive vice president, head of Patient Impact and chief commercial officer, UCB. “In psoriatic arthritis and across the spectrum of axSpA, clinical study results and real-world experience outside the U.S. have highlighted that BIMZELX can help patients achieve high thresholds of clinical response that are rapid in onset and sustained up to two years.” U.S. FDA approves OCREVUS ZUNOVO as the first and only twice-a-year 10-minute subcutaneous injection for people with relapsing and progressive multiple sclerosis The U.S. Food and Drug Administration (FDA) has approved Roche’s OCREVUS ZUNOVO (ocrelizumab & hyaluronidase-ocsq) for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). OCREVUS ZUNOVO is the first and only twice-a-year, healthcare professional (HCP)-administered approximately 10-minute subcutaneous (SC) injection approved for both these forms of multiple sclerosis, giving people living with MS more treatment options. After the first dose, the time for treatment with OCREVUS ZUNOVO could be as little as 55 minutes. Patients will be required to take premedications at least 30 minutes prior to each dose. Following the first dose, patients will be monitored by their HCP for at least 60 minutes. Patients will be monitored for at least 15 minutes post-injection for subsequent doses. “OCREVUS ZUNOVO gives patients and providers another option for receiving OCREVUS, building on a decade of robust safety and efficacy data for OCREVUS in multiple sclerosis,” said Levi Garraway, M.D., Ph.D., Roche’s chief medical officer and head of Global Product Development. “This approval may offer greater flexibility for healthcare providers and people living with multiple sclerosis, based on their individual treatment needs.” U.S. FDA approves first treatment for Niemann-Pick disease, type C The U.S. Food and Drug Administration (FDA) has approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved by the FDA to treat NPC. NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the necessary transport of cholesterol and other lipids within a cell. As a result, these cells do not function as they should, ultimately causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years. The FDA granted approval of Miplyffa to Zevra Therapeutics. www.pbiforum.netPharma Business International 7M&A ROUND-UP 8 Pharma Business International www.pbiforum.net W ith summer over and as we move deeper into the second half of the year, sparing a couple of outliers (which themselves are certainly not mega-mergers), the pharma industry is showing a focus on smaller acquisitions. Pharma Business International reflects on key deals since our last issue. Otsuka Pharmaceutical - Jnana Therapeutics Otsuka Pharmaceutical is set to acquire Jnana Therapeutics through its subsidiary, Otsuka America, paying $800 million to the shareholders of Jnana upon completion, as well as up to an additional $325 million in development and regulatory milestones. Jnana has a novel approach to drug discovery enabled by RAPID – the company’s next-generation chemoproteomics platform designed to discover medicines for highly validated but challenging-to- drug targets. The platform leverages a high- throughput, binding-based screening approach that is inherently flexible, enabling the discovery of binding sites across the surface of a target protein and the identification of small molecules that elicit diverse pharmacologies. Jnana has used RAPID to successfully identify first-in-class compounds and address a range of historically challenging-to-drug target classes, including solute carriers, transcription factors, and signaling scaffold proteins. While Jnana’s RAPID platform is not limited to any specific therapeutic area, the company has built a unique competitive position by concentrating on Phenylketonuria (PKU), a rare inherited metabolic disorder in which phenylalanine accumulates to abnormally high levels in the blood, and autoimmune diseases, where small molecule drug discovery has been challenging. Jnana’s technology generated JNT-517, an allosteric small molecule inhibitor of SLC6A19, an SLC (solute carrier) that regulates amino acid reabsorption in the kidney. JNT-517 has the potential to become a first-in-class oral treatment for PKU, as it has been shown to be effective, well-tolerated, and safe in a Phase 1b/2 study. A majority of the PKU population is not effectively treated with existing therapies, and JNT- 517 is an approach that could address individuals of all ages across the spectrum of mild to severe disease. Organon - Dermavant Organon, meanwhile, has entered into a definitive agreement, to acquire Dermavant, a Roivant company developing and commercializing innovative therapeutics in immuno-dermatology. Dermavant’s novel product, VTAMA (tapinarof) cream, 1%, for the topical treatment of mild, moderate, and severe plaque psoriasis in adults, was approved by the U.S. Food and Drug Administration (FDA) in May 2022. The FDA is reviewing a Supplemental New Drug Application (sNDA) for VTAMA cream as a potential treatment for atopic dermatitis (AD) in adults and children two years of Setting sights on smaller deals Since our last issue, a flurry of mergers and acquisitions have been revealed within the pharmaceutical industry, with the focus on smaller transactions. 10 ÁPharma Business International 9 www.pbiforum.net M&A ROUND-UPNext >