Your guide to the pharmaceutical world Volume 10 Issue 4 Packaging The war against counterfeit drugs Temperature control Unyielding integrity Software and IT systems Is the future yet? Packaging The war against counterfeit drugs Temperature control Unyielding integrity Software and IT systems Is the future yet? www.pbiforum.net 01.qxp_Layout 1 15/08/2022 09:04 Page 102.qxp_Layout 1 11/10/2022 12:01 Page 18 12 20 30 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 After a slow 2021 for M&A in the pharmaceutical industry, and a relatively calm start to 2022, deals are set to gather pace. Packaging12 The counterfeit drugs market is one of the largest in the world and yet many who take them are unawares of their illegal nature. Packaging may well be the solution to making customers aware of the problem. Temperature control16 Maintaining the cold chain is vital to ensure the efficacy of vaccines and vital medical products. As well as an upsurge in growth over the last decade, the sector was called on in the safe delivery and transportation of COVID-19 vaccines, and this pressure has not yielded with further pandemic fears in the future. Software and IT systems20 With advances in technology across the board, AI – or more specifically machine learning – were bound to come up sooner or later. We ask the questions of how viable they might be in the pharma industry. Materials handling24 Materials handling is more important in pharma than any other industry and the explosive growth expected for the sector proves it. Events 28 We give a rundown of pharma events happening around the world. Marketing 30 Market research is one of the most important aspects of marketing, and understanding social media trends is integral to understanding core demographics. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy:stock.adobe.com/mguido 03.qxp_Layout 1 15/08/2022 09:06 Page 14 Pharma Business International www.pbiforum.net Latest news NIH and GreenLight Biosciences to collaborate on COVID-19 vaccine development for new variants GreenLight Biosciences is collaborating with the National Institutes of Health (NIH) to develop COVID-19 vaccines that are more broadly protective against new variants and with longer- lasting effects. “This is a tremendous opportunity for us to partner with a premier vaccine discovery team and use our mRNA platform to accelerate the development of coronavirus vaccines which are more broadly protective and durable than those currently available,” says Amin Khan, PhD, GreenLight’s chief science officer, Human Health. SARS-CoV-2, the virus that causes COVID-19, continues to evolve and accumulate genomic mutations with the potential to negatively affect the efficacy of existing medical countermeasures. For the current COVID-19 pandemic and future coronavirus-related pandemics, the rapid development and deployment of vaccines active against a range of coronaviruses, including variants of SARS-CoV-2, will be vital for public health. Research into effective lung fibrosis treatment receives funding boost New treatments for lung fibrosis, being developed by researchers at the University of Nottingham, are being advanced further with investment in a new spin-out company. o2h Ventures has announced SEIS and EIS investment, alongside co-investment from the University of Nottingham’s Invention Fund, into spin-out company Alevin Therapeutics to develop a platform of novel small molecule RGD integrin inhibitors with superior drug-like qualities. The discovery-stage compounds have broad therapeutic applications in areas that currently have limited or ineffective treatments – including lung fibrosis, kidney disease and cancer – with the most advanced being an inhaled drug for the treatment of Idiopathic Pulmonary Fibrosis (IPF). The investment provides the company with funding of close to £1m to progress and de-risk its pipeline assets. Alevin Therapeutics has arisen from research driven by the Business Partnership Unit and School of Chemistry at the University of Nottingham. Originally the subject of a long-standing collaboration between academia and big pharma, scientists developed the platform of novel compounds with the aim of treating life-threatening conditions by limiting the activity of a key signalling pathway that is targeted by the integrin inhibitors. © stock.adobe.com/ skunkeye © stock.adobe.com/AntonioDiaz Ipsen and Marengo Therapeutics announce strategic partnership to advance two precision immuno-oncology candidates into the clinic Ipsen and Marengo Therapeutics have formed a strategic partnership to advance two of Marengo’s preclinical STAR platform-generated candidates into the clinic. The collaboration will leverage Marengo’s proprietary R&D expertise of a novel mechanism of T cell activation with Ipsen’s global oncology footprint for clinical development and commercialization. Under the terms of the agreement, Ipsen will make an upfront payment of $45 million, together with potential payments up to a total of $1.592 billion if all milestones are met in addition to tiered sales royalty payments. Marengo will lead the preclinical development efforts and will expense related costs until the submission of an Investigational New Drug (IND) application to the U.S. FDA. Ipsen will assume responsibilities for clinical development and commercialization. “Marengo’s foundational discovery of activation of T cell subsets via TCR V? is unprecedented and highly differentiated from the current immuno- oncology technologies we have seen,” said Howard Mayer, M.D., executive vice president and head of R&D at Ipsen. © stock.adobe.com/ krisana 04-07.qxp_Layout 1 15/08/2022 09:14 Page 1Pharma Business International 5 www.pbiforum.net Latest news NIH launches clinical trial of mRNA Nipah virus vaccine The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, has launched an early-stage clinical trial evaluating an investigational vaccine to prevent infection with Nipah virus. The experimental vaccine is manufactured by Moderna, and was developed in collaboration with NIAID’s Vaccine Research Center. It is based on a messenger RNA platform. NIAID is sponsoring the Phase 1 clinical study, which is being conducted at the NIH Clinical Center in Bethesda, Maryland. Nipah virus infection is a zoonotic disease. Fruit bats are the natural host for the virus. The first known Nipah outbreak occurred in 1998 in Malaysia and Singapore and resulted in 265 human cases and 105 deaths. Since 1999, outbreaks have occurred annually in Asia. The virus can cause mild-to-severe disease rapidly progressing from respiratory infection symptoms to encephalitis leading to coma or death. An estimated 40 to 75 percent of people infected with Nipah virus die. Although most cases are transmitted via animals, person-to-person transmission can occur. Currently, there is no licensed vaccine or treatment for Nipah virus infection. Silverback Therapeutics and ARS Pharmaceuticals to merge Silverback Therapeutics and ARS Pharmaceuticals have entered into a definitive agreement under which ARS will merge with Silverback in an all-stock transaction. The combined company will focus on the potential regulatory approval and commercialization of neffy, ARS’ investigational epinephrine nasal spray for the treatment of Type I allergic reactions including anaphylaxis. The combined company is expected to have approximately $265m in cash, cash equivalents and marketable securities at closing. Upon stockholder approval, the combined company is expected to operate under the name ARS Pharmaceuticals. The merger is currently anticipated to close in the fourth quarter of 2022. “We are extremely pleased to announce this proposed merger with Silverback, which we believe enables ARS to maximize the paradigm-changing opportunity of neffy,” said Richard Lowenthal, M.Sc., MSEL, co-founder and Chief Executive Officer of ARS. “neffy is on the cusp of achieving what has not been possible before – the ability to deliver epinephrine with comparable pharmacokinetics to an intramuscular injection, but with a simple to administer nasal spray.” © stock.adobe.com/ Blue Planet Studio © stock.adobe.com/Yurii Kibalnik © stock.adobe.com/Kateryna_Kon AVROBIO receives orphan drug designation for Hunter syndrome gene therapy The U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVROBIO’s AVR-RD-05, a gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys. Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules. The gene therapy uses a patient’s own hematopoietic stem cells (HSCs) that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. The company’s planned collaborator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in 2023 under the company’s collaboration with the University of Manchester, UK. The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, who has previously published preclinical data demonstrating that HSC gene therapy deploying an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology. 04-07.qxp_Layout 1 15/08/2022 09:14 Page 26 Pharma Business International www.pbiforum.net Latest news Myovant Sciences and Pfizer receive U.S. FDA approval of MYFEMBREE for endometriosis pain The U.S. Food and Drug Administration (FDA) has approved Myovant Sciences and Pfizer’s MYFEMBREE (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) as a one-pill, once-a-day therapy for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, with a treatment duration of up to 24 months. The approval is supported by one-year efficacy and safety data, including 24-week data from the Phase 3 SPIRIT 1 and SPIRIT 2 trials, which were published in The Lancet, and the first 28 weeks of an open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2. MYFEMBREE also is approved for heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. Myovant and Pfizer will continue to jointly commercialize MYFEMBREE in the U.S. “Endometriosis is a painful, chronic disease with limited therapies to manage symptoms,” said Juan Camilo Arjona Ferreira, M.D., chief medical officer of Myovant Sciences, Inc. “The new MYFEMBREE indication helps advance our mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis.” Poseida Therapeutics to collaborate with Roche to develop allogeneic CAR-T cell therapies for hematologic malignancies Poseida Therapeutics has entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. Roche will receive either exclusive rights or options to develop and commercialize a number of allogeneic CAR-T programs in Poseida’s portfolio directed to hematologic malignancies, including P-BCMA-ALLO1, an allogeneic CAR-T for the treatment of multiple myeloma and for which a Phase 1 study is underway, and P-CD19CD20-ALLO1, an allogeneic dual CAR-T for the treatment of B-cell malignancies with an IND expected in 2023. The parties will also collaborate in a research program to develop next-generation features and improvements for allogeneic CAR-T therapies, from which they would develop additional allogeneic CAR-T product candidates directed to existing and new hematologic targets. Poseida will receive $110m upfront and could receive up to $110m in near-term milestones and other payments in the next several years. Poseida is eligible to receive research, development, launch, and net sales milestones and other payments potentially up to $6 billion in aggregate value, as well as tiered net sales royalties into the low double digits, across multiple programs. © stock.adobe.com/Sundry Photography © stock.adobe.com/Drazen University signs licensing deal with COVID-19 test provider to commercialise novel testing method A unique testing method invented at the University of Birmingham (UK) is set for a global rollout after its commercial rights were exclusively licensed to the world’s largest COVID-19 test provider, Innova Medical Group, Inc. Known as Reverse Transcription-Free EXPAR (RTF-EXPAR) testing, this new technology offers detection in as little as 10 minutes. The speed of the test is based, in part, on its avoidance of slower, reverse transcriptase-based technologies, and provides the ease of use and speed of lateral flow tests with the sensitivity of PCR testing. Detailed test evaluations reveal the method delivers a fast, accurate, highly sensitive and simple test for COVID-19, meaning the test could be deployed at entertainment venues, airport arrival terminals, and in remote settings where clinical testing laboratories are not available. This new RTF-EXPAR testing platform is also being adapted for the detection of other viruses, meaning the tests can be quickly adapted to cover both new variants and new viruses. © stock.adobe.com/BASILICOSTUDIO STOCK 04-07.qxp_Layout 1 15/08/2022 09:14 Page 3 U.S. FDA approves Incyte’s Opzelura cream for vitiligo The U.S. Food and Drug Administration (FDA) has approved Incyte’s Opzelura (ruxolitinib) cream 1.5% for the topical treatment of non-segmental vitiligo in adult and pediatric patients 12 years of age and older. Opzelura is the first and only FDA-approved treatment for repigmentation in patients with vitiligo, and the only topical formulation of a Janus kinase (JAK) inhibitor approved in the United States. Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin. In patients with non-segmental vitiligo, Opzelura is approved for continuous topical use twice daily to affected areas of up to 10% body surface area. Satisfactory patient response may require treatment with Opzelura for more than 24 weeks. New breast cancer drug approved by the Philippines’ Food and Drug Administration A new drug shown to significantly reduce the risk of cancer recurrence or death in an aggressive form of breast cancer has been approved by the Food and Drug Administration of the Philippines. The drug, NERLYNX (neratinib) is an oral medication taken daily for 12 months by women who have been diagnosed with early-stage HER2-positive (HER2+) breast cancer and who have received prior trastuzumab-based therapy. The greatest benefit is seen in women who are hormone-receptor positive (HR+) and who initiate NERLYNX therapy within 12 months of completing trastuzumab-based therapy. Their five-year risk of recurrence is reduced by 42% after completing 12 months of NERLYNX therapy. NERLYNX is being made available by Specialised Therapeutics. Xenpozyme approved by EC as first treatment for ASMD The European Commission (EC) has approved Sanofi’s Xenpozyme (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B. The approval is based on positive data from the ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed substantial and clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and reduction of spleen and liver volumes, with a well-tolerated safety profile. Given the urgent unmet medical needs of the ASMD community, the European Medicines Agency (EMA) granted Xenpozyme PRIority MEdicines (PRIME) designation. Xenpozyme has also received special breakthrough designations from several other regulatory agencies around the world. MHLW in Japan grants approval for BioMarin’s Voxzogo for children with achondroplasia The Ministry of Health, Labor and Welfare (MHLW) in Japan has granted approval of the registration of BioMarin Pharmaceutical’s Voxzogo (vosoritide) for injection, indicated for the treatment of achondroplasia in children of all ages, whose growth plates are not closed. Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation. “We are delighted to offer children in Japan of all ages with achondroplasia access to a treatment option that addresses the underlying genetic mechanism of the condition,” said Jean-Jacques Bienaimé, chairman and CEO of BioMarin. “CNP was discovered as a natural regulator of bone growth in Japan in 1990 so we are especially proud to be able to offer a therapeutic choice there. We look forward to nurturing our partnerships with advocates and the achondroplasia community in Japan and beyond.” EC approves PTC’s gene therapy Upstaza for AADC deficiency PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has been granted marketing authorization by the European Commission (EC). Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older. During Upstaza clinical studies, patients went from not achieving any developmental motor milestones to demonstrating clinically meaningful motor skills from as early as three months following treatment, with transformational improvements shown to continue up to ten years after treatment. In addition, cognitive skills improved in all treated patients. Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications. MHRA grants marketing authorization for Oxbryta to treat hemolytic anemia in patients with sickle cell disease 12 and older The Medicines and Healthcare products Regulatory Agency (MHRA) has granted Great Britain marketing authorization for Global Blood Therapeutics’ Oxbryta (voxelotor) for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adult and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (hydroxyurea). Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle hemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of red blood cells in SCD. www.pbiforum.netPharma Business International 7 04-07.qxp_Layout 1 15/08/2022 09:14 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net Momentum picks up for M&A After a slow 2021 for M&A in the pharmaceutical industry, and a relatively calm start to 2022, deals are set to gather pace. 08-11.qxp_Layout 1 15/08/2022 09:16 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP A s momentum picks up for mergers and acquisitions in the pharmaceutical industry following a scarcity of swoops in 2021, deals are expected to gather further pace through the second half of 2022. PwC notes that large pharma companies are abundant with cash, especially those with COVID-19 treatments under their wing, biotech valuations have been normalising, and many firms are seeking new assets to expand their pipelines as the 2025 patent cliff approaches, meaning earlier stage companies are anticipated to be sought after. “All of the stars are aligned for there to be a flurry of deals activity across all areas of the sector,” says PwC. With pharma’s current appetite for smaller deals, recent transactions have not hit as high a value as Pfizer’s $11.6 billion swoop for Biohaven Pharmaceuticals highlighted last issue, but there remains plenty to report on. Amgen - ChemoCentryx Amgen is set to acquire ChemoCentryx for $52 per share in cash, representing an enterprise value of approximately $3.7 billion. The transaction is expected to close in the fourth quarter of 2022. “The acquisition of ChemoCentryx represents a compelling opportunity for Amgen to add to our decades-long leadership in inflammation and nephrology with TAVNEOS, a transformative, first-in-class treatment for ANCA-associated vasculitis,” said Robert A. Bradway, chairman and Chief Executive Officer at Amgen. “We are excited to join in the TAVNEOS launch and help many more patients with this serious and sometimes life-threatening disease for which there remains significant unmet medical need. We also look forward to welcoming the highly skilled team from ChemoCentryx that shares our passion for serving patients suffering from serious diseases.” TAVNEOS is an orally administered selective complement component 5a receptor inhibitor approved by the U.S. Food and Drug Administration in 2021 as an adjunctive treatment for adult patients with severe active ANCA-associated vasculitis, specifically granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) - the two main forms of ANCA-associated vasculitis - in combination with standard therapy. U.S. sales of TAVNEOS in the first quarter of 2022, the first full quarter of sales, were $5.4 million. TAVNEOS is also approved in major markets outside the U.S., including the European Union and Japan. Vifor Fresenius Medical Care Renal Pharma Ltd. will retain exclusive rights to commercialize TAVNEOS outside the U.S., except in Japan where Kissei Pharmaceutical Co., Ltd. holds commercialization rights and Canada where Otsuka Canada Pharmaceutical holds commercialization rights. ChemoCentryx also has three early- stage drug candidates that target chemoattractant receptors in other inflammatory diseases and an oral checkpoint inhibitor for cancer. Gilead Sciences - MiroBio Gilead Sciences meanwhile will acquire MiroBio for approximately $405 million in cash, subject to customary adjustments. The deal will provide Gilead with 10 Á 08-11.qxp_Layout 1 15/08/2022 09:16 Page 2Next >