Your guide to the pharmaceutical world Volume 12 Issue 2 End of line packaging Sustainable, traceable, profitable Transport and logistics Governance and transport Clinical trials Digital trials End of line packaging Sustainable, traceable, profitable Transport and logistics Governance and transport Clinical trials Digital trials 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.uk8 12 20 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 Following a slow few years for M&A in the pharma industry, dealmaking is picking up pace. Transport and logistics12 Given the importance of the transport industry to just about any country, logistics groups are asking if it isn’t time for a more direct line between government and industry. Labelling, coding and marking16 Labelling, coding, and marking are key in the pharmaceutical sector - to product traceability, integrity, and safety. End of line packaging20 By the year 2032, the end of line packaging market is anticipated to reach $7.9 billion, and while much of that will be in food and beverage as well as pharma, it’s still an increase of 4.3% CAGR. With such growth comes change, however, and the end of line packaging industry has to adapt to current conditions. Clinical trials24 As new technology evolves, so too must the mechanisms we use. While AI is useful for many aspects of drug design, human testing will always be an inevitable facet of clinical trials. That doesn’t mean technology can’t be used to screen for and recruit applicants, however. Waste management28 As the pharmaceutical industry faces closer scrutiny for its waste disposal, and its environmental impact is more widely recognised, responsible waste management is vital. 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/Artinun4 Pharma Business International www.pbiforum.net Latest news MiNA Therapeutics enters research collaboration with Nippon Shinyaku to develop RNAa therapeutics targeting rare neurodegenerative diseases MiNA Therapeutics, a pioneer of small activating RNA (RNAa) therapeutics, has announced a research collaboration and licensing agreement option with Nippon Shinyaku, a Japanese-based pharmaceutical company focused on disease areas of high unmet medical need. The collaboration will allow for the discovery, and potential development and commercialization of RNAa therapeutic candidates targeting rare neurodegenerative diseases for which there are currently no treatment options. Under the agreement, MiNA Therapeutics will utilize its proprietary RNAa algorithm and technology platform to identify and characterise RNAa molecules targeting rare genetic diseases of the central nervous system (CNS). Nippon Shinyaku will also have the option to exercise an exclusive license to further research, develop and commercialize pharmaceutical candidates based on early-stage preclinical outcomes. Nippon Shinyaku will pay MiNA an upfront payment and development milestone associated with delivering characterized RNAa molecules and, subject to exercising an exclusive option fee for further development and commercialization, milestones payments and royalties commensurate with progress and sales of the product(s) after launch. US FDA Rare Pediatric Disease Designation granted to Lisata Therapeutics’ LSTA1 for the treatment of osteosarcoma The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to LSTA1, Lisata Therapeutics’ lead product candidate, for the treatment of osteosarcoma, a rare cancer that can develop in children, adolescents and young adults. “LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumors, and we believe that potential extends to a beneficial impact in osteosarcoma, a rare cancer that usually develops in the osteoblast cells that form bone,” stated Kristen K. Buck, M.D., executive vice president of R&D and chief medical officer of Lisata. “Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1, and, more importantly, it underscores the high unmet medical need of this patient population.” A substantial benefit of a RPDD is receipt of a priority review voucher, often referred to as a “golden ticket,” once the FDA approves the new drug application (NDA) for the product and indication having received the RPDD © stock.adobe.com/Jeniffer Sequani invests in people and facilities as it eyes future growth As part of their strategic plans for growth and to meet the evolving needs of modern product developers, Ledbury-based Contract Research Organisation, Sequani has made a significant investment in a new custom designed facility incorporating 820 sq m of laboratory space and a 910 sq m modern vivarium, whilst also appointing Lee Coney as CSO. With over 30 years of product development and senior managerial experience in the sector, Lee will take a leading role in working with the company’s customers to develop a wide range of pharmaceutical, crop protection and chemical products. Speaking about the new facility, Lee said: “The new state-of-the-art lab and vivarium will enable us to be more flexible and allow us to support the evolving needs of our customers and worldwide regulatory authorities. Amongst other advantages, co-locating the laboratory and animal facilities into a single building offers enhanced biosecurity for sample flows from animal unit to analytical laboratory. It’s more efficient and will allow us to deliver a superior service.” © stock.adobe.com/Fabián MontañoPharma Business International 5 www.pbiforum.net Latest news Boehringer Ingelheim and Sosei Heptares join forces to develop schizophrenia treatments Boehringer Ingelheim and Sosei Heptares have entered a global collaboration and exclusive option-to-license agreement. At the center is a joint mission to develop and commercialize Sosei Heptares’ portfolio of first-in-class GPR52 agonists, a novel G protein-coupled receptor (GPCR) target, with the intent to improve patient outcomes by simultaneously addressing positive, negative, and cognitive symptoms of schizophrenia. Schizophrenia is a serious condition that affects about 1 in 100 people worldwide. It is characterized by three clusters of symptoms: ‘positive’ symptoms, such as psychosis, delusions and hallucinations, ‘negative’ symptoms, such as social withdrawal and apathy, and cognitive symptoms, such as attention, planning and memory deficits. The impact of these symptoms on people’s ability to cope with normal day-to-day life is significant and the related burden on carers and society at large is substantial, especially since the age of onset of the disease is typically in the 20’s. While ‘positive’ symptoms can be stabilized with antipsychotics, some of which can have side effects, there are currently no approved medicines for ‘negative’ or cognitive symptoms. © stock.adobe.com/ Rapeepat Lamassu awarded grant for breakthrough cancer treatment Lamassu Bio has been awarded a grant from the National Institutes of Health (NIH) and National Cancer Institute (NCI) for the development of their groundbreaking treatment for p53 wild-type sarcomas. The $2.05 million grant will help fund the clinical trial integral to this new cancer treatment. The trial will be conducted in collaboration with Cleveland Clinic Taussig Cancer Center and Cleveland Clinic Children’s Pediatric Hematology and Oncology Department. P53 is a crucial tumor suppressor gene commonly mutated in human cancers. Its role in preventing tumor formation by inducing programmed cell death in response to cellular stress makes it a key target for cancer therapy. The project focuses on advancing SA53, a novel therapeutic that targets p53 wild- type sarcomas, malignant tumors of connective or non-epithelial tissue. SA53 has demonstrated remarkable potency, efficacy and safety in preclinical models, positioning it for an Investigational New Drug (IND) submission. This innovative approach offers promising prospects for addressing chemo-resistant cancer and presents a significant pathway for advancing cancer care. © stock.adobe.com/huenstructurebio.com Ipsen and Sutro Biopharma reveal exclusive global licensing agreement for ADC targeting solid tumors Ipsen and Sutro Biopharma have announced an exclusive global licensing agreement for STRO-003. STRO-003, an antibody-drug conjugate (ADC) in the final stages of pre-clinical development, targets the ROR1 tumor antigen which is known to be overexpressed in many different cancer types including solid tumors and hematological malignancies. The agreement gives Ipsen exclusive worldwide rights to develop and commercialize STRO-003 and will be the first ADC candidate joining Ipsen’s expanding portfolio. “The potential for ADCs in oncology is well-documented and we are excited by the addition of STRO-003, Ipsen’s first ADC candidate with best-in-class potential,” said Mary Jane Hinrichs, SVP and head of Early Development at Ipsen. “STRO-003 is a next-generation ROR1 ADC, leveraging Sutro’s site-specific technology to generate a highly stable conjugate, coupled with exatecan payloads, that have shown significant potential in solid tumors. This is our focus as we prepare to enter Phase I, harnessing Ipsen’s global expertise in oncology development, while also reinforcing our commitment to bringing new medicines to patients with few treatment options.” © stock.adobe.com/Feng Yu6 Pharma Business International www.pbiforum.net Latest news Getinge and CellRev launch pioneering adherent cell expansion platform CellRev, in partnership with Getinge, has launched a breakthrough adherent cell expansion platform, Livit ACE. Designed to eliminate many of the limitations and inefficiencies associated with adherent cell culture, Livit ACE reduces costs, enhances productivity and accelerates development times. As well as removing a significant amount of single-use consumables from adherent cell culture workflows, Livit ACE can decrease labour costs and reduce the overall cost per dose by more than 50%. CellRev, an innovative supplier of processing solutions to improve the efficiency of cell manufacturing, has worked in partnership with the global medtech company, Getinge, to bring to market the Livit ACE, a disruptive continuous manufacturing platform to transform cell-based vaccine and therapy manufacturing. Getinge provides hospitals and life science institutions globally with products and solutions that aim to improve clinical results and optimise workflows. With the ambition to bring innovative technologies to market, Getinge teamed up with CellRev, for development and commercialisation of an industry-first continuous adherent cell expansion platform. Livit ACE combines the best of CellRev’s proprietary processing technologies and Getinge’s bioprocessing expertise and equipment into one solution. Lonza to acquire large-scale biologics site from Roche Lonza, a global manufacturing partner to the pharmaceutical, biotech and nutraceutical markets, has signed an agreement to acquire the Genentech large-scale biologics manufacturing site in Vacaville, California (US) from Roche for $1.2 billion. The acquisition will significantly increase Lonza’s large-scale biologics manufacturing capacity to meet demand for commercial mammalian contract manufacturing from customers with existing commercial products, and molecules currently on the path to commercialization within the Lonza network. The Vacaville (US) facility currently has a total bioreactor capacity of around 330,000 liters, making it one of the largest biologics manufacturing sites in the world by volume. Under the agreement, approximately 750 Genentech employees at the Vacaville (US) facility will be offered employment by Lonza. Jean-Christophe Hyvert, president, Biologics, Lonza, said: “The Vacaville site is a highly valuable strategic acquisition that will make capacity immediately available for our customers and unlock future growth for our Biologics division. It will support us in providing a commercialization path to existing customers and incremental large-scale commercial capacity to our partners.” Cybin receives FDA Breakthrough Therapy Designation for novel psychedelic molecule for Major Depressive Disorder Cybin, a clinical-stage biopharmaceutical company committed to revolutionizing mental healthcare by developing new and innovative next-generation psychedelic- based treatment options, has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to CYB003, its proprietary deuterated psilocybin analog in development for the adjunctive treatment of Major Depressive Disorder (MDD). If approved by the FDA, CYB003 would be the first known adjunctive psychedelic-based therapeutic for the treatment of MDD. The company also announced that its Phase 2 trial of CYB003 in MDD demonstrated robust and sustained improvement in depression symptoms at four months with 75% of participants receiving two 16mg doses achieving remission and no longer showing signs of depression. BTD provides an expedited review pathway, as well as increased access to FDA guidance on trial design, with the potential to reduce drug development timelines. It is reserved for drug candidates that target serious conditions and demonstrate substantial improvement on a clinically significant endpoint over available therapies. © stock.adobe.com/Robert © stock.adobe.com / Wellnhofer Designs US FDA approves antibiotic ZEVTERA for three indications The US Food and Drug Administration (FDA) has approved Basilea Pharmaceutica’s ZEVTERA® (ceftobiprole medocaril sodium for injection), for the treatment of adult patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including those with right-sided infective endocarditis, and adult patients with acute bacterial skin and skin structure infections (ABSSSI) and for adult and pediatric patients with community- acquired bacterial pneumonia (CABP). Dr. Marc Engelhardt, chief medical officer of Basilea, said: “We are very pleased that the FDA approved ZEVTERA for all three indications that were submitted with the NDA (New Drug Application), including a pediatric labelling. This approval is a landmark for ceftobiprole and reflects its broad clinical utility. The indication in adult patients with Staphylococcus aureus bacteremia, including those with right-sided infective endocarditis, caused by methicillin-susceptible and methicillin-resistant isolates, MSSA and MRSA, addresses a real medical need, as current treatment options are limited.” The NDA was supported by clinical efficacy and safety data from the phase 3 studies ERADICATE (SAB) and TARGET (ABSSSI), and a phase 3 study in CABP. Abilify Maintena approved in EU as first once-every-two-months long-acting injectable for schizophrenia The European Commission (EC) has approved Otsuka Pharmaceutical Europe and H. Lundbeck’s Abilify Maintena® 720mg/960mg (aripiprazole) as a once-every-two-months long-acting injectable (LAI) formulation for the maintenance treatment of schizophrenia in adult patients stabilised with aripiprazole. Aripiprazole once-every-two-months LAI is a new formulation containing 960mg aripiprazole provided in a single-chamber prefilled syringe that does not require reconstitution. It is intended for dosing once every two months via intramuscular injection into the gluteal muscle and is the first once- every-two-months LAI antipsychotic licensed in the EU for this indication. The EC based its approval on a 32-week pharmacokinetic bridging trial, which also evaluated the safety and efficacy of the drug as primary and secondary endpoint respectively. Aripiprazole once-every-two-months LAI was shown to provide similar plasma concentrations, and therefore similar effectiveness, as well as a similar safety and tolerability profile to aripiprazole once-monthly LAI (Abilify Maintena® 400mg) in 266 adults, of whom 185 were diagnosed with schizophrenia. ADZYNMA approved in Japan for patients with congenital thrombotic thrombocytopenic purpura The Japanese Ministry of Health, Labour and Welfare has approved the use of Takeda’s ADZYNMA (apadamtase alfa/cinaxadamtase alfa) for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) for individuals 12 years of age and older. ADZYNMA is the first and only approved recombinant ADAMTS13 protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme. cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme. It is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache and abdominal pain. When left untreated, acute TTP events have a mortality rate of >90%. The approval is supported by the totality of the evidence provided from an interim analysis of efficacy, pharmacokinetic, safety and tolerability data from the first randomized, controlled, open-label, crossover Phase 3 trial in cTTP patients ages 12-68, which includes five Japanese patients and supported by long-term safety and efficacy data from a continuation study. US FDA approves EDURANT PED for pediatric patients living with HIV-1 The US Food and Drug Administration (FDA) has approved Johnson & Johnson’s EDURANT® PED (rilpivirine) for the treatment of HIV-1 in combination with other antiretroviral therapies (ARVs) in treatment-naïve children (with HIV-1 RNA <100,000 copies/mL) at least 2 years of age and weighing at least 14 kg and less than 25 kg. “Decades of experience with the global HIV epidemic have made it clear that new and improved treatment options are needed to support the diverse population of people living with HIV on their treatment journey,” said Penny Heaton, M.D., global therapeutic area head, Infectious Diseases and Vaccines at Johnson & Johnson. “While the population of young children living with HIV is small, additional treatment options remain key to ensuring that each person living with HIV can be matched to a treatment regimen that is right for them.” The FDA’s decision is based on results from the PAINT and PICTURE studies in pediatric subjects, which showed that rilpivirine, in combination with other ARVs, effectively suppresses the virus in treatment-naïve (with HIV-1 RNA <100,000 copies/mL) pediatric patients. VYLOY approved in Japan for treatment of gastric cancer Japan’s Ministry of Health, Labour and Welfare has approved Astellas Pharma’s VYLOY™ (zolbetuximab), an anti-claudin 18.2 (CLDN18.2) monoclonal antibody, in combination with chemotherapy, for patients with human epidermal growth factor receptor 2 (HER2)-negative, CLDN18.2 positive, unresectable, advanced or recurrent gastric cancer. VYLOY is the first and only CLDN18.2-targeted therapy approved by any regulatory agency in the world. Despite efforts to reduce its impact, gastric cancer is the third deadliest cancer in Japan, with 126,724 cases diagnosed in 2022. The approval is based on results from the Phase 3 SPOTLIGHT and GLOW clinical trials for first-line treatment in patients with locally advanced unresectable or metastatic HER2- negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors were CLDN18.2 positive. Both trials met their primary endpoint, progression- free survival, as well as a key secondary endpoint, overall survival, showing statistical significance in patients treated with VYLOY plus chemotherapy compared to placebo plus chemotherapy. www.pbiforum.netPharma Business International 7M&A ROUND-UP 8 Pharma Business International www.pbiforum.net © stock.adobe.com/volody10 A revival in dealmaking Following a slow few years for M&A in the pharma industry, dealmaking is picking up pace. Pharma Business International 9 www.pbiforum.net M&A ROUND-UP M ergers and acquisitions are a key aspect in drug development, yet plunged as the pandemic ended. Now, however, a revival in dealmaking has begun, in line with companies’ expansion plans and patent cliffs. Pharma Business International reflects on some of the significant transactions since our last issue. Gilead Sciences - CymaBay Therapeutics Gilead Sciences has acquired CymaBay Therapeutics in a $4.3bn deal, with CymaBay’s investigational lead product candidate seladelpar, for the treatment of primary biliary cholangitis (PBC) including pruritus, complementing Gilead’s existing liver portfolio. Seladelpar is an investigational, oral, selective peroxisome proliferator- activated receptor delta (PPAR?) agonist, shown to regulate critical metabolic and liver disease pathways. The United States Food and Drug Administration (FDA) has completed its filing review and accepted a New Drug Application for seladelpar and granted priority review with a Prescription Drug User Fee Act target action date of August 14, 2024. Seladelpar received FDA Breakthrough Therapy Designation for use in the treatment of PBC including pruritus in patients without cirrhosis or with compensated cirrhosis and PRIME status (EMA), as well as Orphan Drug Designation in the US and Europe for the treatment of patients with PBC. Daniel O’Day, chairman and Chief Executive Officer, Gilead Sciences, said: “We are looking forward to advancing seladelpar by leveraging Gilead’s long- standing expertise in treating and curing liver diseases. Building on the strong research and development work by the CymaBay team to date, we have the potential to address a significant unmet need for people living with PBC and expand on our existing broad range of transformational therapies.” Novartis - MorphoSys Meanwhile, Novartis is set to strengthen its oncology pipeline with the €2.7bn acquisition of MorphoSys, a biopharmaceutical company developing innovative medicines in oncology. Upon completion of the acquisition, Novartis will own pelabresib (CPI-0610), a novel and potentially practice changing treatment option with a well-tolerated safety profile provided in combination with ruxolitinib for patients with myelofibrosis (MF). It will also include tulmimetostat (CPI-0209), an early-stage investigational dual inhibitor of enhancer of zeste homolog 1 and 2 (EZH1 and EZH2) proteins currently being tested in patients with solid tumors or lymphomas. “We are excited about the opportunity of bringing pelabresib, a potential next- generation treatment combined with ruxolitinib, to people living with myelofibrosis, a rare and debilitating form of blood cancer,” said Shreeram Aradhye, M.D., president, development and chief medical officer of Novartis. “With the planned acquisition of MorphoSys, we aim to further strengthen our leading pipeline and portfolio in oncology, adding to our capabilities and expertise. Building on our long-standing development partnership with MorphoSys, we look forward to continuing our work together to realize the full impact and value of their investigational medicines for patients with unmet needs.” AstraZeneca - Fusion Pharmaceuticals Furthermore, AstraZeneca has entered into a definitive agreement to acquire Fusion Pharmaceuticals, a clinical-stage biopharmaceutical company developing next-generation radioconjugates (RCs), for $2.4bn. The deal marks a step forward in AstraZeneca delivering its ambition to transform cancer treatment and outcomes by replacing traditional regimens like chemotherapy and radiotherapy with more targeted treatments. RCs have emerged as a promising modality in cancer treatment over recent years. These medicines deliver a radioactive isotope directly to cancer 10 ÁNext >