Your guide to the pharmaceutical world Volume 9 Issue 3 Antivirals exposé Promising treatments Logistics & distribution Maintaining integrity Testing Taking clinical testing into tomorrow PharmaBUSINESS INTERN ATION AL The total package From development to dispensing The total package From development to dispensing Antivirals exposé Promising treatments Logistics & distribution Maintaining integrity Testing Taking clinical testing into tomorrow www .pbiforum.net 01.qxp_Layout 1 10/06/2021 09:50 Page 1 INTER N Food Food &&& Food Food & N ATI O N &&Drin Drin Drin &&Drin AL nk nk nkk app A r f h hi ts a ews Ingredient spotlight End offline packkaaging For businesses that are serious about reducing their carbon footprint www.blmgroup.co.uk VOLUME 4 ISSUE 1 W W W. R E N E W A B L E S A N D E N V I R O N M E N T. C O M E A S T M I D L A N D S L E A E A D I N G B U S I N E S S M A G A A Z I N E APRIL 2020 0 £3. .50 BUS SUP SINES T PPOR SS TING B THCART HEAL YY FACTOR RE OFF FICE ea Stopping the spr THCAR WW HEAL d thinkin Forwar TT AUTOMA Y AFACTOR ad WW EASTMIDL RE A hea SOL OFF ng factories LANDSBUSINE TION ficef althy of LUTIONS ESSLINK CO U FICE w UK www.blmgroup.co.uk EMPOWER Your Business Focus on T A Pharm rld o our guide to the pharmaceutical w Y B U S I N E S S I N T E R N A L ma olume 8 Issue 1 V T I O N declares g rrld Hea oooW coron F ganisa gg l b l g l h O virus na g gency ti medi Nucle Prev aaccin V The nuclea b f treatment ve The P l t k aage n e t eelopment t vv om de rF w w w di i k . p b i f o r u m . n yorkshire & lincolnsh hire’s leading business ma magazine april 2020 £3.5 50 TO T RES C THE SPOND CRISIS DING S YY FACTOR C TT HEAL OFF THCARE FICE safety Ensuring e TT HEAL e of factories The futur TION T AUTOMA YY AFACTOR fices healthy f Keeping of SOLUTIONS OFFICE employee THCARE V w VOLUME 35 ISSUE 4 .blmgroup.co.uk www Often Imitated - Never Equalled www.blmgroup.co.uk 02.qxp_Layout 1 10/06/2021 09:51 Page 1Contents Volume 9 Issue 3 8 12 22 30 Group Editor: Steve Fisher (s.fisher@blmgroup.co.uk) Editor: Michael Fisher (m.fisher@blmgroup.co.uk) Assistant Editor: Dominic Cuthbert (d.cuthbert@blmgroup.co.uk) Journalist: Tess Egginton (t.egginton@blmgroup.co.uk) Sales Director: Angie Cooper (a.cooper@blmgroup.co.uk) Sales: Vicky Hunt (v.hunt@blmgroup.co.uk) Tel: +44 (0) 1472 310302 Accounts & Subscriptions: Angela Sharman (a.sharman@blmgroup.co.uk) Design & Production: Gary Jorgensen, Mark Casson, (studio@blmgroup.co.uk) Editorial: Tel: +44 (0) 1472 310305, Fax: +44 (0) 1472 310317 Part of BLMGroup: Armstrong House, Armstrong Street, Grimsby, North East Lincolnshire DN31 2QE England All rights reserved. No part of this publication may be reproduced, transmitted, photocopied, recorded or otherwise without express permission of the copyright holder, for which application should be addressed first to the publisher. While every reasonable care is taken, neither the publisher nor its participating agents accept liability for loss or damage to prints, colour transparencies, negatives or other material of whatever nature submitted to this publication. The views expressed in this publication are not necessarily the views of those held by the publisher. News4 Stay up to date with the latest industry updates from the global pharmaceutical technology industry. M&A round-up8 M&A activity is heating up in the pharmaceutical sector. We take a closer look at some of the biggest deals over the last few months. Antivirals exposé12 While vaccines are rolled out, the importance of antivirals in treating COVID-19 should not be forgotten. Pharma Business International shines a light on recent developments showing promise. Theramex Q&A16 We recently spoke with Robert Stewart, CEO of Theramex, a global speciality pharmaceutical company solely committed to supporting the health needs of women through every stage of life. Logistics & distribution18 When it comes to maintaining the temperature integrity of pharmaceutical products, it’s best to look at the supply chain – and in particular storage and distribution centres. Testing22 It’s notoriously difficult to get a potential new therapeutic through clinical trials and testing, but digital technologies are helping to make testing more efficient. From discovery to market26 It goes without saying that drug discovery and bringing new products to market is a gruelling process. Recent developments, in no small part driven by COVID-19, may make this process easier in future. Innovation30 Innovations in artificial intelligence are already having a profound effect on drug discovery, aiding research and shortening time to market. Capsules34 A popular drug delivery dosage form holding a number of benefits for both drug developers and patients, capsules continue to evolve and are in high demand. Events 38 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pbiforum Cover photo courtesy: Shutterstock / A-photographyy 03.qxp_Layout 1 10/06/2021 09:52 Page 1FDA expands collaboration with CN Bio to investigate lung- on-a-chip model for inhaled drug evaluation applications CN Bio, a developer of single- and multi-organ-on-chip microphysiological systems (MPS) that improve the accuracy and efficiency of drug discovery, has announced the U.S. Food and Drug Administration (FDA) has extended their research collaboration, for a further three years. The scope of the research carried out with the FDA’s Center for Drug Evaluation and Research (CDER) has now been broadened to include the exploration of CN Bio’s lung-on-a-chip using the PhysioMimix™ MPS platform, to appraise the system’s use for the evaluation of inhaled drug products and assessing additional applications for CN Bio’s liver model. The extension follows the success of CN Bio and the FDA’s initial 4-year agreement, culminating in a co-publication that demonstrated the advanced performance of the Company’s liver-on-a-chip MPS; the first co-authored, peer-reviewed article between a microphysiological system provider and a regulator. 4 Pharma Business International www.pbiforum.net Latest news Vertex and Obsidian to discover novel therapies that regulate gene editing Vertex Pharmaceuticals and Obsidian Therapeutics have entered into a strategic research collaboration and licensing agreement focused on the discovery of novel therapies that regulate gene editing for the treatment of serious diseases. The collaboration leverages Obsidian’s cytoDRiVE platform technology to discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules and Vertex’s established scientific and clinical capabilities in small molecule, cell and genetic therapies to more rapidly bring these approaches to patients. “This collaboration with Obsidian builds upon and will expand Vertex’s leadership in small molecule and genetic therapies, and we’re excited to partner with the team at Obsidian to explore the capabilities of their technology,” said Vertex’s David Altshuler, Executive Vice President, Global Research and Chief Scientific Officer. “The ability to tune gene-editing activity to a specific level is an important innovation that has the potential to address several serious diseases.” Under the terms of the agreement, Obsidian will use its cytoDRiVE technology to develop novel regulated gene editing therapy candidates for multiple serious diseases. Obsidian grants Vertex the exclusive option to license worldwide rights to candidates discovered and developed under the collaboration. Following Vertex’s exercise of its options, Vertex will be responsible for further preclinical and clinical development and commercialization. Vertex will pay Obsidian up to $75 million in upfront payments and research milestones that may be paid during the research term, including an equity investment in Obsidian. Obsidian is eligible to receive up to $1.3 billion in potential payments based upon the successful achievement of specified research, development, regulatory, and commercial milestones across up to five potential programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. Specific diseases that are the subject of this collaboration are not disclosed. Pfizer and BioNTech to supply EU with 1.8bn additional Comirnaty doses Pfizer and BioNTech have announced a new agreement with the European Commission (EC) to supply 900 million doses of Comirnaty, the companies’ COVID-19 vaccine, to the European Union (EU), with an option for the EC to request up to an additional 900 million doses. This new agreement is in addition to the 600 million doses that have already been committed to the EU through 2021. The additional 900 million agreed doses are expected to be delivered on a monthly schedule beginning December 2021 and continuing into 2023. As part of the agreement, the EC also has an option to increase the number of doses delivered by up to an additional 900 million, bringing the total number of potential doses delivered to the EC, inclusive of all agreements, to up to 2.4 billion. All doses for the EC are planned to be manufactured in the EU. © Shutterstock/Illus_man © Shutterstock / Nadezda Murmakova © Shutterstock/CI Photos 04-07.qxp_Layout 1 10/06/2021 09:54 Page 1Lilly & MiNA Therapeutics forge saRNA research collaboration Eli Lilly and London-based RNA activation therapeutic specialist, MiNA Therapeutics, have entered a global research collaboration to develop novel drug candidates using the latter’s proprietary small activating RNA (saRNA) technology platform. Under the terms of the agreement, MiNA will utilise its saRNA platform to research up to five targets selected by Lilly that aim to address diseases across Lilly’s key therapeutic focus areas. Lilly will be responsible for preclinical and clinical development of candidates and will retain exclusive commercialisation rights for any products resulting from the collaboration. MiNA will receive a $25 million upfront payment and is eligible to receive potential development and commercialisation milestones up to a total of $245 million per target, as well as tiered royalties from the low-single to low-double digits on product sales resulting from the collaboration. Pharma Business International 5 www.pbiforum.net Latest news Sandoz to strengthen European antibiotics manufacturing Sandoz is further expanding production capabilities in Kundl, Austria and in Palafolls, Spain as it looks to strengthen its European antibiotics manufacturing. By modernising and simplifying its manufacturing setup, Sandoz will improve its ability to consistently deliver high-quality medicines to patients, while remaining cost-competitive on the global market. In line with plans announced with the Austrian government in July 2020, Sandoz confirms that in a first step it will proceed to invest more than €100 million to introduce new manufacturing technology for the production of oral amoxicillin, an active pharmaceutical ingredient (API) for its leading penicillin product. This investment will allow Sandoz, a Novartis division, to leverage its position in Kundl as the hub and centre of the only major end-to-end antibiotics supply chain in Europe, covering all production steps from API to Finished Dosage Forms (FDF) for many leading antibiotics. Additionally, Sandoz will expand its Palafolls site in Spain. Biogen and Bio-Thera Solutions to develop Actemra biosimilar Biogen and Bio-Thera Solutions have entered into a commercialisation and license agreement to develop, manufacture and commercialise BAT1806, a Phase 3 clinical stage anti-interleukin-6 (IL- 6) receptor monoclonal antibody that is a proposed biosimilar referencing Actemra (tocilizumab). Actemra’s primary indication is for moderate to severe rheumatoid arthritis in adults as well as juvenile idiopathic polyarthritis, systemic juvenile idiopathic arthritis, giant cell arteritis and cytokine release syndrome. In 2020 global sales of Actemra were 2.8 billion CHF.2 Biogen will gain exclusive regulatory, manufacturing and commercial rights to BAT1806 in all countries excluding China (including Hong Kong, Macau and Taiwan). Biogen will expand its global biosimilars footprint with the potential approval of BAT1806. Biogen will make an upfront payment of $30 million to Bio-Thera Solutions, contingent upon Bio-Thera Solutions’ Phase 3 for BAT1806 achieving satisfactory results. Should certain commercial milestones be achieved, Bio-Thera Solutions will be eligible to receive potential milestone payments. Biogen will also pay Bio-Thera Solutions tiered royalties. © Shutterstock / Corona Borealis Studio © Shutterstock / nobeastsofierce © Shutterstock / Fahroni 04-07.qxp_Layout 1 10/06/2021 09:54 Page 26 Pharma Business International www.pbiforum.net Latest news Excenstia forges $1.2bn AI drug discovery deal with BMS Exscientia, the clinical stage, AI-driven pharmatech company, announced today that it has entered into a collaboration agreement with Bristol-Myers Squibb (BMS). This expanded collaboration has the potential to add to the BMS drug pipeline whilst enhancing Exscientia’s portfolio of shared assets. The collaboration will use AI to accelerate the discovery of small molecule therapeutic drug candidates in multiple therapeutic areas, including oncology & immunology. The agreement includes up to $50 million in upfront funding, up to $125 million in near to mid-term potential milestones, and additional clinical, regulatory and commercial payments that take the potential value of the deal beyond $1.2 billion. Exscientia will also receive tiered royalties on net sales of any marketed drug products resulting from the collaboration. This expanded collaboration builds upon Exscientia’s existing collaboration with Bristol Myers Squibb that was initiated in 2019 with Celgene prior to Celgene’s acquisition by BMS. © Shutterstock / IRINA SHI Beckman Coulter launch workflow automation for mid-volume labs Beckman Coulter, a clinical diagnostics leader, has announced the European launch of the DxA 5000 Fit, a workflow-automation solution designed to fit into medium- sized labs that run fewer than 5,000 tests a day. The clinical laboratory has gone through trial by fire in 2020, where total testing was 245% of baseline volumes, with ~55% being SARS-CoV-2 molecular tests. Despite vaccines, many industry players suggest SARS- CoV-2 testing volumes won’t be impacted in the near term, and the demand for COVID-19-related testing will continue through 2021 and potentially into 2022. This means that testing-volume pressure on labs is going to continue. Labs of all sizes are facing the same challenges: shortage of laboratory technologists and technicians , historic financial pressures on hospitals and health systems , and physically and mentally exhausted laboratory professionals – a pre- pandemic problem that has since been exacerbated. However, the TLA solutions that cater to labs processing high-sample volumes are out of reach for smaller volume labs due to space requirements and infrastructure constraints. Subtitle: The right fit for mid-sized labs In a survey, lab directors and managers were asked to identify the key challenges hospital labs are facing. The results found that staffing (26%) and turnaround time (23%) were identified as top priorities. Automation plays a key role in helping to address many of these issues while enabling precious resources to focus on high-value, clinical tasks – this has proven particularly true during the COVID-19 pandemic. “For large laboratories, TLA is a reality today. However, for medium-volume labs like ours that process fewer than 4,000 tests per day, comprehensive workflow-automation solutions haven’t been an option,” said Susan Enciso, System Supervisor of Chemistry, Excela Health. “Beckman Coulter’s DxA 5000 Fit solution is the right fit for us. It has a flexible design which is important in a lab like ours where space is a precious commodity; and reduces our manual process steps by up to 80%, helping us meet today’s unprecedented need for COVID-19 and routine tests.” To learn more about Beckman Coulter’s family of automation solutions and the DxA 5000 Fit, visit www.beckmancoulter.com/en/products/automation/workflow-automation-system-dxa-5000-fit. 04-07.qxp_Layout 1 10/06/2021 09:54 Page 3Eye on approvals FDA accepts Airway’s IND to develop novel recombinant protein for COVID-19 The US FDA has accepted Airway Therapeutics’ Investigational New Drug (IND) application to develop AT-100 (rhSP-D) as a treatment for COVID-19. Cincinnati biopharmaceutical company, Airway Therapeutics is leveraging the FDA’s Coronavirus Treatment Acceleration Program (CTAP) that is aimed at evaluating new treatment options for COVID-19. Airway will initiate a Phase 1b clinical trial to confirm the feasibility of intratracheal administrations of AT-100 and its beneficial safety and tolerability profile. Airway’s novel human recombinant protein AT-100 – an engineered version of an endogenous protein – has been shown in preclinical studies to safely reduce inflammation and infection while modulating the immune response across a range of respiratory diseases inside and outside the lung. ViiV initiates rolling submission of NDA for injectable PrEP in US ViiV Healthcare has initiated a rolling submission of a new drug application (NDA) with the US Food and Drug Administration (FDA) for investigational, long-acting, injectable cabotegravir (pre-exposure prophylaxis, or PrEP) for the prevention of HIV. The rolling submission allows ViiV Healthcare to submit portions of the regulatory application to the FDA as they are completed, rather than waiting until every section of the NDA is complete to submit the entire application for review. The complete submission will be based on results from two phase IIb/III studies, HPTN 083 and HPTN 084. HPTN 083 evaluated the safety and efficacy of long- acting cabotegravir for HIV prevention in men who have sex with men and transgender women. Farxiga approved in the US for the treatment of chronic kidney disease AstraZeneca’s Farxiga (dapagliflozin), a sodium-glucose cotransporter 2 (SGLT2) inhibitor, has been approved in the US to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease (ESKD), cardiovascular (CV) death and hospitalisation for heart failure (hHF) in adults with chronic kidney disease (CKD) at risk of progression. The approval by the FDA was based on positive results from the DAPA-CKD Phase III trial. The decision follows the Priority Review designation granted by the FDA earlier this year. WHO issues Emergency Use Listing for Moderna’s COVID vaccine The World Health Organization (WHO) has issued Emergency Use Listing (EUL) for Moderna’s COVID-19 vaccine to prevent COVID-19 in individuals 18 years of age and older. The WHO based its decision on the totality of scientific evidence shared by the Moderna, including a data analysis from the pivotal Phase 3 clinical study announced on November 30, 2020. Moderna will continue to share data with the WHO as it becomes available. Jemperli becomes first anti-PD-1 therapy available for endometrial cancer in Europe The European Commission has granted conditional marketing authorisation to GlaxoSmithKline (GSK) for Jemperli (dostarlimab), making it the first anti-PD-1 therapy available for endometrial cancer in Europe. Jemperli is a programmed death receptor-1 (PD-1)-blocking antibody, for use in women with mismatch repair-deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer who have progressed on or following prior treatment with a platinum containing regimen. FDA allows SPI to test ebselen as novel COVID-19 treatment The FDA has allowed two Phase 2 studies to begin testing ebselen (SPI-1005) in COVID-19 patients, Sound Pharmaceuticals, Inc. (SPI) has announced. SPI is developing SPI-1005, a novel anti-inflammatory drug which was recently shown to inhibit nCoV2 activity and viral replication. 120 adults with moderate or severe disease will be enrolled in two randomised, double-blind, placebo-controlled trials (RCTs) and treated for 7 or 14 days with the oral drug. Separately, SPI will be utilising the non-clinical and pre-clinical services program offered by the National Institute of Allergy and Infectious Diseases (NIAID). This will involve the expanded testing of ebselen both in vitro and in vivo, including a unique live animal model of COVID-19 transmission. www.pbiforum.net Pharma Business International 7 04-07.qxp_Layout 1 10/06/2021 09:54 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net Starting this issue’s M&A round-up with a bang, AstraZeneca’s proposed $39 billion acquisition of Alexion Pharmaceuticals has cleared the US Federal Trade Commission review. This follows the conclusion of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. To date, the deal has been cleared in Canada, Brazil, Russia and other countries globally. At the time of writing, regulatory clearances are still pending in the UK, the EU and Japan, among others. The proposed acquisition, first announced in December 2020, would enhance AstraZeneca’s scientific presence in immunology by adding Alexion’s innovative complement-technology platforms and strong pipeline. The acquisition remains expected to close in Q3 2021. Continuing with the major billion-dollar-plus deals, Thermo Fisher Scientific announced its intention towards the end of April to acquire PPD, provider of clinical research services to the pharma and biotech industry. The deal – for up to $20.9 billion – establishes Thermo Fisher as one of the foremost leaders in the clinical research services industry. PPD provides a broad range of clinical research and laboratory services to enable customers to accelerate innovation and increase drug development productivity. In 2020, the company generated revenue of $4.7 billion. Upon close of the transaction, PPD will become part of Thermo Fisher’s Laboratory Products and Services Segment. “The acquisition of PPD is a natural extension for Thermo Fisher and will enable us to provide these customers with important clinical research services and partner with them in new and exciting ways as they move a scientific idea to an approved medicine quickly, reliably and cost effectively,” said Marc N. Casper, Chairman, President and CEO of Thermo Fisher Scientific at the time of the Heating up M&A activity is heating up in the pharmaceutical sector. We take a closer look at some of the biggest deals over the last few months. 10 Á 08-11.qxp_Layout 1 10/06/2021 09:54 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP © Shutterstock /askarim 08-11.qxp_Layout 1 10/06/2021 09:54 Page 2Next >