Your guide to the pharmaceutical world Volume 11 Issue 6 Depositors and filling Accurate filling Outsourcing Out of house End of year success stories A market ripe for picking Depositors and filling Accurate filling Outsourcing Out of house End of year success stories A market ripe for picking www.pbiforum.net8 20 24 28 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) Vicky Hunt (v.hunt@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 Pharma Business International highlights major acquisitions since our last issue as the giants continue to strike. End of year success stories12 There’s no denying the economical outlook has been troubling for the year, but those able to navigate the choppy waters have reaped dividends. As 2023 comes to an end, we look at what went right and what trends might be in stock for 2024. Finance and investment16 Finance is a tricky subject for pharma every year. At the end of 2022 we talked of the struggles the world has faced with regards to economies, inflation and economic pressure – and that’s only gotten worse through 2023. Depositors and fillers20 Accurate measurement, efficient filling and maximum hygiene all add pressure to the depositors and filling sector, and there are plenty of options available to suit any size of operation. Process control and automation24 The problem with automation is not that it is overly complicated or expensive, but that it requires continued monitoring to make sure the system is running optimally, and it’s possible to have an inefficient automated system and never realise it. Outsourcing28 The strain placed on manufacturers by worsening economic conditions is causing many to look at outsourcing their requirements rather than investing in costly new facilities, and even costlier recruitment processes to fill them. 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/guteksk74 Pharma Business International www.pbiforum.net Latest news Aragen to set up $30 million bio-manufacturing site in India Aragen, a Contract Research Development and Manufacturing Organization (CRDMO) for small molecules and biologics, is setting up a new biologics manufacturing facility in Bangalore, India. The state-of-the-art facility is part of a $30m investment, spanning nearly 160,000 sqm, and includes process development labs and multiple GMP manufacturing suites with supporting functions like quality control labs (analytical and microbiology). The manufacturing suites will be equipped for intensified processing using single-use bioreactors and advanced downstream purification capability to serve its rapidly expanding biologics customer base. The facility will offer integrated solutions from process development, process validation, analytical development, pilot production, large-scale DS manufacturing and stability services. It will have the capability to develop and manufacture mAbs, therapeutic proteins and fusion proteins to augment the company’s bioproduction capability in California. The process development laboratory is scheduled to be operational by the third quarter of FY 24 while the first manufacturing suite will open in the third quarter of FY 25. BeiGene and Ensem Therapeutics in $1.33bn agreement to advance CDK2 inhibitor BeiGene and Ensem Therapeutics have announced an agreement for BeiGene to acquire an exclusive global license to an Investigational New Drug (IND) application- ready oral cyclin-dependent kinase 2 (CDK2) inhibitor. “We are committed to developing novel molecules with the potential to transform the therapeutic landscape for cancer patients, and this partnership fits well with our strategic focus on breast cancer as an area with tremendous unmet need for innovative treatment options,” said Lai Wang, Ph.D., global head of R&D at BeiGene. “This CDK2 inhibitor from ENSEM complements our internally discovered Phase 1 CDK4 inhibitor, which has the potential to improve upon current CDK4/6 inhibitors in some breast cancer patients, and strengthens our early development pipeline in breast cancer and other solid tumors. We are excited to work with ENSEM to bring this molecule into the clinic in the near future.” ENSEM will receive an upfront payment and will be eligible for additional payments upon the achievement of certain development, regulatory, and commercial milestones, totaling up to $1.33 billion, in addition to tiered royalties. © stock.adobe.com/ Gorodenkoff 1nhaler raises £2m to develop single-use sustainable inhaler 1nhaler, a Scotland-based drug delivery device developer, has raised £2 million to develop its single-use dry powder inhaler (DPI). Inhalable medicines are already widely used to treat conditions such as asthma, epilepsy, allergies and Parkinson’s but, to date, delivery options have been limited due to user accessibility, cost, performance and impact on the environment. 1nhaler has developed a way of enabling many different dry powder drugs to be readily delivered in a sustainable, low-cost delivery device. Other inhalers are typically made from bulky plastic and are expensive to manufacture and distribute. 1nhaler’s revolutionary sustainable DPI is cardboard-based with the same dimensions as a credit card. The discreet platform technology includes a breathable membrane which can be tailored to deliver individual drugs to patients. Investment will allow 1nhaler to work with innovative companies around the world, allowing them to bring new and existing drugs to patients in a more efficient and affordable way, opening up the next generation of inhaled products. © stock.adobe.com/ James Thew The 1nhaler device - a single dose, dry powder inhalerPharma Business International 5 www.pbiforum.net Latest news Novo Nordisk invests in expansion of Chartres production facilities Novo Nordisk is set to invest more than 16 billion Danish kroner (2.1 billion euros) to expand its existing production site in Chartres, France, for its current and future product portfolio within serious chronic diseases. The investment will significantly increase the capacity of the manufacturing site, adding aseptic production and finished production processes and an extension of the current Quality Control Laboratory. The investment, which includes capacity for GLP-1 products, will increase Novo Nordisk’s ability to meet future demands for innovative medicines. The new facilities will more than double the footprint of the site. The facility will be designed as a multi-product facility to accommodate current and future processes and displaying state-of- the-art technology and working environment. As a future-proof and cost-effective facility, the construction will focus on delivering the highest quality to patients globally in an efficient and environmentally sustainable way. © stock.adobe.com/jetcityimage © stock.adobe.com/Ascannio Biotech company awarded Innovate UK grant to expand search for rare disease therapies Oxford-based SynaptixBio, whose aim is to find a treatment for TUBB4A- related leukodystrophies, has been awarded a £490,000 BioMedical Catalyst grant from Innovate UK to tackle less common variants of the disease. TUBB4A-related leukodystrophies are a group of rare neurodegenerative diseases primarily affecting young children. They are caused by a mutation in the TUBB4A gene, resulting in disruption to the signals between nerve cells in the brain. Currently, there is no cure; SynaptixBio is said to be the first and only company developing a therapy. Hypomyelination with Atrophy of the Basal ganglia and Cerebellum (H-ABC) is the most severe form of the disease, and SynaptixBio is already in the process of developing a therapy that will hopefully go into clinical trials next year; the other forms range in severity but all have life-changing impacts for those affected and their families. Laure Humbert, head of Research and Preclinical Development at SynaptixBio, said: “This funding will enable us to give hope to more patients and their families, who currently have none. We are extremely grateful to Innovate UK.” © stock.adobe.com/ TuMeggy Legend Biotech Ireland enters into exclusive, global license agreement with Novartis for CAR-T cell therapies Legend Biotech Ireland has entered into an exclusive, global license agreement with Novartis Pharma for certain Legend Biotech chimeric antigen receptor T-cell (CAR-T) cell therapies targeting DLL3 (Delta-like ligand protein 3), including its autologous CAR-T cell therapy candidate, LB2102. The License Agreement grants Novartis the exclusive worldwide rights to develop, manufacture and commercialize these cell therapies, and Novartis may apply its T-Charge platform to their manufacture. Legend Biotech is initiating clinical development of LB2102 for the treatment of extensive stage small cell lung cancer and large cell neuroendocrine carcinoma after the U.S. Food and Drug Administration (FDA) cleared its investigational new drug application in 2022. In 2023, the FDA granted the product candidate Orphan Drug Designation. Under the terms of the License Agreement, Legend Biotech will receive a $100 million upfront payment and will be eligible to receive up to $1.01 billion in clinical, regulatory and commercial milestone payments and tiered royalties. 6 Pharma Business International www.pbiforum.net Latest news XPHOZAH granted Orphan Drug Designation by U.S. FDA for pediatric hyperphosphatemia The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Ardelyx’s XPHOZAH (tenapanor) for the treatment of pediatric hyperphosphatemia. XPHOZAH is a single tablet taken twice daily that offers a first-in-class mechanism of action that blocks phosphate absorption through its primary pathway. “We are pleased that the FDA has recognized the important need among pediatric patients with hyperphosphatemia for treatment options,” said Mike Raab, president and Chief Executive Officer of Ardelyx. “This designation highlights the importance of evaluating treatment options for all patients with hyperphosphatemia, and we look forward to sharing more details on this important designation by FDA in the future.” In October, XPHOZAH was approved by the FDA to reduce serum phosphorus in adults with chronic kidney disease (CKD) on dialysis as add-on therapy in patients who have an inadequate response to phosphate binders or who are intolerant of any dose of phosphate binder therapy. AstraZeneca licenses novel agent for the treatment of obesity AstraZeneca and Eccogene have entered into an exclusive licence agreement for ECC5004, an investigational oral once-daily glucagon-like peptide 1 receptor agonist (GLP- 1RA) for the treatment of obesity, type-2 diabetes and other cardiometabolic conditions. Preliminary results from the Phase I trial have shown a differentiating clinical profile for ECC5004, with good tolerability and encouraging glucose and body weight reduction across the dose levels tested compared to placebo. Under the terms of the agreement, Eccogene will receive an initial upfront payment of $185m. In addition, Eccogene will also receive up to an additional $1.825bn in future clinical, regulatory, and commercial milestones and tiered royalties on product net sales. Under the agreement AstraZeneca is granted exclusive global rights for the development and commercialisation of Eccogene’s ECC5004 small molecule GLP-1RA for any indication in all territories except China, where Eccogene has the right to co-develop and co-commercialise in China alongside AstraZeneca. © stock.adobe.com/neirfy British Heart Foundation and UK Dementia Research Institute reveal plans for Centre for Vascular Dementia Research In partnership with the UK Dementia Research Institute (UK DRI), the British Heart Foundation has revealed plans to establish the UK’s first research Centre dedicated to finding new treatments to prevent, halt and ultimately cure vascular dementia. The British Heart Foundation aims to invest £7.5m over the next five years so the Centre can drive forward pioneering research into the devastating condition. UK DRI will contribute £1.5m as well as enable access to its cutting-edge technology and research centres. The Centre will be the UK’s flagship investment for vascular dementia research, uniting leading researchers from around the world under a single virtual ‘roof’. Vascular dementia is the second most common type of dementia, affecting around 150,000 people in the UK. It is caused by poor blood flow to the brain, which starves nerve cells of oxygen and nutrients they need to live. This causes nerve cells in the affected areas of the brain to die, and symptoms include memory loss and difficulty concentrating, which worsen over time. © stock.adobe.com / Graphicroyalty © stock.adobe.com / Vitalii FDA approves first therapy for rare type of non-cancerous tumors The U.S. Food and Drug Administration (FDA) has approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas. Desmoid tumors are non-cancerous but can be locally aggressive. The tumors may invade into surrounding structures and organs, resulting in pain, issues with being able to move, and decreased quality of life. Although surgical removal has historically been the treatment of choice, there is a high risk that the tumor will return or that other health challenges will occur after removal; therefore, systemic therapies (cancer treatment targeting the entire body) are being increasingly evaluated in clinical trials. The FDA granted the approval of Ogsiveo to SpringWorks Therapeutics Inc. Truqap plus Faslodex approved in the US for patients with advanced HR-positive breast cancer AstraZeneca’s Truqap (capivasertib) in combination with Faslodex (fulvestrant) has been approved in the US for the treatment of adult patients with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1 or PTEN). Eligible patients will have progressed on at least one endocrine-based regimen in the metastatic setting or experienced recurrence on or within 12 months of completing adjuvant therapy. The approval by the Food and Drug Administration (FDA) was based on the results from the CAPItello-291 Phase III trial published earlier this year in The New England Journal of Medicine. In the trial, Truqap in combination with Faslodex reduced the risk of disease progression or death by 50% versus Faslodex alone in patients with tumours harbouring PI3K/AKT pathway biomarker alterations (based on hazard ratio of 0.50, 95% confidence interval 0.38-0.65; p=<0.001; median progression-free survival (PFS) 7.3 versus 3.1 months). Concurrently with this approval, the FDA also approved a companion diagnostic test to detect relevant alterations (PIK3CA, AKT1 and PTEN). Jemperli plus carboplatin and paclitaxel approved in Canada for primary advanced or recurrent dMMR/MSI-H endometrial cancer Health Canada has approved a new indication for GSK’s Jemperli (dostarlimab for injection) in combination with carboplatin and paclitaxel for the treatment of adult patients with primary advanced or recurrent mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) endometrial cancer who are candidates for systemic therapy, based on Part 1 of the pivotal RUBY study. “Today’s approval supports our ambition for Jemperli to advance the standard of care for Canadian adult patients with primary advanced or recurrent dMMR/MSI-H endometrial cancer,” said Marni Freeman, country medical director at GSK Canada. “This expanded approval of Jemperli offers a new care option to healthcare professionals treating this patient population with high unmet need.” In December 2021, Jemperli had previously been granted Notice of Compliance with conditions by Health Canada as a monotherapy for the treatment of adult patients with dMMR or MSI-H recurrent or advanced endometrial cancer, that has progressed on or following prior treatment with a platinum containing regimen. First CRISPR/Cas9 gene-edited therapy authorised by the UK MHRA for sickle cell disease and transfusion-dependent beta thalassemia The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ CASGEVY (exagamglogene autotemcel [exa- cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). CASGEVY has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available. There are an estimated 2,000 patients eligible for CASGEVY in the UK. “Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR- based therapy in the world,” said Reshma Kewalramani, M.D., Chief Executive Officer and president of Vertex. “I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said Samarth Kulkarni, Ph.D., chairman and Chief Executive Officer of CRISPR Therapeutics. EC approval for RYEQO for the symptomatic treatment of endometriosis The European Commission (EC) has granted approval of a Type II Variation application for Gedeon Richter and Sumitomo Pharma’s RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethisterone acetate 0.5 mg) for the symptomatic treatment of endometriosis in women with a history of previous medical or surgical treatment for their endometriosis. RYEQO was initially approved by the EMA in July 2021 for the treatment of moderate-to-severe symptoms of uterine fibroids in adult women of reproductive age. Adele Gulfo, Chief Executive Officer, Biopharma Commercial Unit at Sumitomo Pharma America, said: “Approximately 14 million women in the European Union are living with endometriosis, a chronic condition whose painful symptoms can impact a woman’s ability to engage in certain daily activities. We are pleased that as a result of this decision women in the EU living with endometriosis now have another safe and effective symptomatic treatment option.” www.pbiforum.netPharma Business International 7M&A ROUND-UP 8 Pharma Business International www.pbiforum.net A s another year of dealmaking nears its close, 2023 has seen its fair share of significant acquisitions. It has invigorated hope of recovery for M&A in the pharmaceutical industry following a slowdown in years past, with 2023 welcoming Pfizer’s $43 billion swoop for Seagen, Merck’s $10.8 billion acquisition of Prometheus Biosciences, and Biogen’s $7.3 billion transaction for Reata Pharmaceuticals. Robert Newman, corporate partner at law firm DLA Piper, reflected as the sector heats up: “Certain sectors remain buoyant in M&A, notwithstanding the data suggesting a general trend of a slightly slowing global M&A market. One of these ‘hot’ sectors is ‘big pharma’ M&A, where it was reported back in June 2023 that $85 billion had been spent in the first five months of the year, kick- started largely by the Amgen acquisition of Horizon…. We have also seen the announced Roche acquisition of Telavant for $7.1 billion, whilst both Novo and Eli confirmed a combined war chest of $3.5 billion to deploy in obesity related treatments. We expect this level of spending from big pharma to continue as they look to utilise cash reserves (as was reported by EY) amassed during the COVID period, diversify their product portfolio and to help mitigate the well-publicised patent cliff at the end of this decade. Novel and innovative areas such as immunology and obesity appears to be a particular target area given the large number of patients globally affected and the recent advancement in science and our understanding of how to better target these diseases.” While further dealmaking is eyed, Pharma Business International reflects on recent moves. AbbVie - ImmunoGen In the biggest deal since our last issue, AbbVie is set to acquire ImmunoGen and its flagship cancer therapy ELAHERE (mirvetuximab soravtansine-gynx), a first- The giants strike Pharma Business International highlights major acquisitions since our last issue as the giants continue to strike.Pharma Business International 9 www.pbiforum.net M&A ROUND-UP © stock.adobe.com/diy13 in-class antibody-drug conjugate (ADC) approved for platinum-resistant ovarian cancer (PROC). The $10.1 billion acquisition will accelerate AbbVie’s commercial and clinical presence in the solid tumor space, while ImmunoGen’s follow-on pipeline of promising next- generation ADCs further complements AbbVie’s ADC platform and existing programs. Richard A. Gonzalez, chairman and CEO, AbbVie, said: “The acquisition of ImmunoGen demonstrates our commitment to deliver on our long-term growth strategy and enables AbbVie to further diversify our oncology pipeline across solid tumors and hematologic malignancies. Together, AbbVie and ImmunoGen have the potential to transform the standard of care for people living with cancer.” ImmunoGen’s oncology portfolio has the potential to help drive long-term revenue growth for AbbVie’s oncology franchise. Ovarian cancer is the leading cause of death from gynecological cancers in the U.S. ELAHERE is the first targeted medicine to show meaningful survival benefit in PROC. As a fast- growing solid tumor therapy, ELAHERE offers AbbVie a potential multi-billion- dollar on-market medicine with expansion opportunities in earlier lines of therapy and larger segments of the ovarian cancer market. The boards of directors of both companies have approved the transaction, which is expected to close in the middle of 2024, subject to ImmunoGen shareholder approval, regulatory approvals, and other customary closing conditions. Roche - Telavant Meanwhile Roche’s aforementioned agreement to acquire Telavant, owned by Roivant Sciences Ltd. and Pfizer Inc., will see the giant pay $7.1 billion upfront and a near-term milestone payment of $150 million. The agreement includes the 10 ÁNext >