Your guide to the pharmaceutical world Volume 8 Issue 5 Blockchain The benefits of blockchain Stem cell therapies Therapies for the future Precision medicine Tailoring treatments PharmaBUSINESS INTERN ATION AL The total package From development to dispensing The total package From development to dispensing Blockchain The benefits of blockchain Stem cell therapies Therapies for the future Precision medicine Tailoring treatments www .pbiforum.net 01.qxp_Layout 1 12/10/2020 10:54 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 12/10/2020 10:55 Page 1Contents Volume 8 Issue 5 4 16 28 32 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 Manager: Sam Trott (s.trott@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 After a quiet period of M&A activity for the industry, Pharma Business International explores a slew of multibillion-dollar-deals. Precision medicine exposé12 Pharma Business International delves into recent strides for precision medicine, highlighting studies that are expanding access to the approach, and displaying its potential. Stem cell therapies16 The cutting-edge stem cell therapy market continues to grow as the pandemic intensifies; now scientists are taking aim at coronavirus and, in so doing, bolstering our response to future pandemics. Finance20 We explore the various ways in which drug discovery and development can be funded. Fill finish manufacturing24 The coronavirus pandemic has brought extra demand to fill finish manufacturing, an area already seeing growth as well as new challenges and opportunities due to the rising number of drugs on the market, targeted therapeutics and smaller batch sizes. Inhibitors28 In the wake of the pandemic, inhibitors are being used in the ongoing fight against COVID-19 as Pharma Business International explores. Blockchain 32 With blockchain becoming one of the pharmaceutical sector’s biggest trends, we explore how the technology is bolstering traceability, reliability and safety across the entire supply chain. @pbiforum www.pbiforum.net company/pharma-business- international Cover photo courtesy: Shutterstock / Sashkin 03.qxp_Layout 1 12/10/2020 10:56 Page 1BioNTech bolsters COVID production capacity with Novartis facility BioNTech is acquiring a GMP manufacturing site from Novartis to expand its COVID-19 vaccine production capacity in the first half of 2021. Based in Marburg, Germany, the manufacturing site will expand BioNTech’s COVID-19 vaccine production capacity by up to 750 million doses per year, or over 60 million doses per month, once fully operational. The transaction is expected to close in the fourth quarter of 2020. The company plans to be able to produce up to 250 million doses of BNT162b2 in the first half of 2021, thanks to the established team and well-established drug substance and drug product manufacturing capabilities at the site. The Marburg production site is a state-of-the-art, multi-platform GMP certified manufacturing facility that currently employs approximately 300 people. The acquisition will accelerate BioNTech’s efforts to scale-up its commercial manufacturing capacity to produce its mRNA COVID-19 vaccine candidate BNT162. 4 Pharma Business International www.pbiforum.net Latest news Horizon introduces single cell RNAseq-linked CRISPR screening service Horizon Discovery, a Cambridge, UK-based gene editing specialist, has introduced a single cell RNAseq-linked pooled CRISPR screening to its CRISPR screening services portfolio. The platform offers high-quality screening data and biological insight to address critical gaps in target identification and validation. This launch is the result of a collaboration – announced back in 2018 – to develop and apply this novel research tool to discover new biology essential for the development of future therapeutics. The biological insight gained from Horizon’s single cell RNAseq-linked CRISPR screening platform could help researchers to gain further understanding into complex biological models and speed up the time from discovery to validation by integrating the effect of gene editing with complex gene expression mapping. Horizon’s pooled format screens offer researchers access to highly robust whole-genome level analyses that yield quality data outputs. Currently, it can be challenging to adequately multiplex the data from these screens to evaluate complex biological phenomena occurring in a subset of edited cells. Boehringer & Click to develop digital therapeutic for schizophrenia Boehringer Ingelheim and Click Therapeutics will together develop and commercialise of a prescription-based digital therapeutic for schizophrenia. It will utilise cognitive and neurobehavioral mechanisms delivered through Click’s proprietary engagement platform with the goal of reducing cognitive deficits and impaired social functioning in patients with schizophrenia. Together the two companies will combine their expertise to develop a novel mobile application – CT-155 – which combines multiple clinically validated therapeutic interventions to help schizophrenia patients modify their behaviour to achieve positive clinical outcomes alone and in combination with pharmaceutical therapy options. The partnership aims to provide better tools and resources to those living with schizophrenia, where there remains a huge unmet need due to lack of access to psychosocial intervention therapies. © Shutterstock/Avatar_023 © Shutterstock / OneStockPhoto © Shutterstock/vchal 04-07.qxp_Layout 1 12/10/2020 10:58 Page 1Huadong Medicine & Exscientia to accelerate oncology drug discovery with AI Exscientia, an AI drug discovery company, and Chinese pharma firm, Huadong Medicine, have partnered to accelerate the discovery of breakthrough small-molecule therapeutics targeted at innovative approach to oncology. The first project selected in partnership is focused on transcription control of DNA damage response genes, to treat patients with defective DNA damage repair mechanism, leading to high mutation frequency, as seen is in patients with ovarian cancer and breast cancer. Under the collaboration, the pharmatech Exscientia and Huadong will share responsibilities, with Exscientia applying its advanced Centaur Chemist AI platform to design new molecules against project specific objectives and Huadong Medicine applying its China based experimental and managerial capabilities to generate data and progress Exscientia’s AI design in a rapid closed-loop cycle. The partnership will also support both Huadong’s strategy to introduce new medicines in key markets and Exscientia’s aims to advance AI-designed innovative medicines globally. Pharma Business International 5 www.pbiforum.net Latest news New partnership to take potential MND treatment into clinical trials Researchers from the University of Sheffield’s Institute for Translational Neuroscience (SITraN) have been awarded a $700,000 drug development grant to progress a potential targeted therapy for motor neurone disease (MND) into clinical trials. The grant, awarded by Australian charity FightMND, will support SITraN’s partnership with Aclipse Therapeutics to progress a disease-modifying drug candidate – M102 – into first in-human trials for patients with the neurodegenerative disease. Neuroprotective properties in M102 were discovered by SITraN researchers in 2013. Preclinical models evidenced the potential for M102 to stop and reverse MND progression, which affects a patient’s ability to walk, talk, eat and breathe. The drug candidate activates the NRF2 (nuclear factor erythroid 2-related factor 2) and HSF1 (Heat shock factor 1) signalling pathways, recently understood as important disease pathways in MND. M102 may lead to significant slowing and reversal of disease progression in both familial and sporadic MND patients. Search for new cancer treatments get £8m genetic boost Projects to find new treatments for cancer, using whole genome sequencing, have received a £8 million funding boost from UK Research and Innovation (UKRI). Working in partnership with Genomics England, the additional funding will allow whole genome sequencing to be applied to seven clinical trials involving a range of cancers. The seven clinical trials that have been funded are: * TRACERx Renal CR-UK and AstraZeneca (RAMPART Trial) * CR-UK Personalised Breast Cancer Programme, CR-UK and AstraZeneca (PARTNER Trial) * CR-UK PATHOS and BMS-funded NICO Trials in Oropharyngeal and Oral cancer * Tessa Jowell Brain Matrix Trial Brain Tumour Charity * OxPloreD blood cancer, Johnson and Johnson * Metastatic Melanoma, RMH Cancer Charity * ESCALATE Oesophageal Cancer Study The funding will speed up the adoption of whole genome sequencing – a form of genetic analysis – in the study of cancer. Genetic analysis is a critical tool that allows clinicians to select the most appropriate treatments for each patient. © Shutterstock / AshTproductions © Shutterstock / Elisanth © Shutterstock / CI Photos 04-07.qxp_Layout 1 12/10/2020 10:58 Page 26 Pharma Business International www.pbiforum.net Latest news Scientists share promising COVID-19 drug development breakthrough Infectious disease scientists at the University of Pittsburgh and University of Pittsburgh Medical Center (UPMC) have shared details of a promising breakthrough in COVID-19 drug development. Researchers at Pitt’s Centre for Antibody Therapeutics, with support from UPMC, panned their library of 100 billion antibodies to identify possible candidates for use against SARS-CoV-2 (the virus that causes COVID-19). Using the SARS-CoV-2 spike protein as bait, Dr Dimiter Dimitrov and his team had identified the most promising options in barely a week, and they used one of them to create a drug now called Ab8. Dr John Mellors, UPMC and Pitt’s chief of infectious diseases, and Dimitrov, Director of the Center for Antibody Therapeutics, collaborated with several universities throughout the United States and Canada to further study Ab8 with the hope that it can one day serve as both a treatment and a preventative measure against COVID-19. Takeda opens R&D cell therapy manufacturing facility Takeda is expanding its cell therapy manufacturing capabilities with the opening of a new 24,000 sq ft cell therapy manufacturing facility at its R&D headquarters in Boston, Massachusetts. The facility provides end-to-end research and development capabilities and will accelerate Takeda’s efforts to develop next- generation cell therapies, initially focused on oncology with potential to expand into other therapeutic areas. The R&D cell therapy manufacturing facility will produce cell therapies for clinical evaluation from discovery through pivotal Phase 2b trials. The current Good Manufacturing Practices (cGMP) facility is designed to meet all US, EU and Japanese regulatory requirements for cell therapy manufacturing to support Takeda clinical trials around the world. It will be instrumental in building Takeda’s cell therapy capabilities and capacity to advance multiple next-generation oncology cell therapy platforms and programs with world-class collaborators. Enesi & Adelaide Uni to develop Zika vaccine UK biotech, Enesi Pharma, has partnered with the University of Adelaide to develop a new thermostable, solid dose DNA vaccine for Zika virus to prevent infection of pregnant women and the resultant congenital effects in the unborn child. This project aims to employ Enesi’s ImplaVax formulation expertise and platform to create a protective Zika virus DNA vaccine in a solid dose implant format for transcutaneous delivery via a needle-free device. The innovative combination of this ImplaVax formulation and delivery system with the novel DNA vaccine construct developed by researchers at the University of Adelaide is expected to enhance the vaccine’s efficacy, thermal stability and allow for safe and rapid vaccination of target populations that are otherwise geographically and economically disadvantaged. The new AUD 1.35 million collaboration with the University of Adelaide aims to advance the development of a novel thermostable, solid dose Zika virus DNA vaccine to Phase 1 clinical trials. © Shutterstock/Andrii Vodolazhskyi © Shutterstock / sdecoret © Shutterstock/Jarun Ontakrai 04-07.qxp_Layout 1 12/10/2020 10:58 Page 3Eye on approvals Spravato approved for use to treat MDD in Scotland The Scottish Medicines Consortium (SMC) has accepted Spravato (esketamine) nasal spray for use within NHS Scotland less than a week after NICE ruled not to recommend the drug for use on the NHS in England. Specifically, the nasal spray is approved in combination with a selective serotonin reuptake inhibitor (SSRI) or serotonin-norepinephrine reuptake inhibitor (SNRI), for adults with treatment-resistant Major Depressive Disorder (TRD), who have not responded to at least two different treatments with antidepressants in the current moderate to severe depressive episode. EU approves Imfinzi for ES-SCLC AstraZeneca’s Imfinzi (durvalumab) has been approved in the European Union for the 1st-line treatment of adults with extensive-stage small cell lung cancer (ES-SCLC) in combination with a choice of chemotherapies, etoposide plus either carboplatin or cisplatin. The approval was based on positive results from the Phase III CASPIAN trial showing Imfinzi plus chemotherapy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit for the 1st-line treatment of patients with ES-SCLC. Opdivo plus Yervov and chemo recommended for EU approval The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Opdivo (nivolumab) plus Yervoy (ipilimumab) with two cycles of platinum-based chemotherapy for the first-line treatment of metastatic non-small cell lung cancer (NSCLC). The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation. Clear Labs granted EUA by FDA for SARS-CoV-2 novel descriptive diagnostic The FDA has granted Clear Labs Emergency Use Authorization (EUA) for its Clear Dx SARS- CoV-2 novel descriptive diagnostic. The system extends available options for labs to scale infectious disease diagnostic testing. Clear Dx offers an automated, nanopore-based NGS solution for simultaneous high-volume diagnosis of SARS-CoV-2 and deeper genomic characterization of the virus. It provides end-to-end results in hours. FDA clears Roche’s BK virus quantitative test of cobas systems Roche has secured US approval for the cobas BKV Test on the cobas 6800 and 8800 Systems to support better care for transplant patients. The test was previously granted FDA Breakthrough Device designation demonstrating the improved treatment or diagnosis of life-threatening diseases or conditions for transplant patients. It provides standardised, high-quality results that can help healthcare professionals better assess the risk of complications caused by the BK virus in transplant patients and identify effective treatment options. FDA approves additional doses of Trulicity for type 2 diabetes The US FDA has approved two additional doses of Eli Lilly’s Trulicity (dulaglutide) for the treatment of type 2 diabetes. The approval expands the label of once-weekly Trulicity to include 3.0 mg and 4.5 mg doses based on data from AWARD-11. The phase 3 trial showed the additional doses led to further benefits in A1C and body weight reduction when compared to Trulicity 1.5 mg in people with type 2 diabetes. FDA fast-tracks LEO’s investigational topic cream for CHE US regulators have fast-tracked LEO pharma’s delgocitinib cream, an investigational topical pan-Janus kinase (JAK)-inhibitor, for the potential treatment of adults with moderate-to-severe chronic hand eczema (CHE). There are currently no treatment options available in the US specifically developed and approved for the treatment of CHE. FDA expands emergency use of remdesivir The FDA has expanded the Emergency Use Authorization (EUA) enabling use of the investigational antiviral Veklury (remdesivir) to treat all hospitalised patients with COVID-19, in addition to the previous authorization for patients hospitalised with severe COVID-19. The expanded EUA is based on results from the Phase 3 SIMPLE trial evaluating Veklury in hospitalised patients with moderate COVID-19 pneumonia, as well as results from the National Institute of Allergy and Infectious Diseases (NIAID) ACTT-1 trial in hospitalised patients with a range of disease severity. www.pbiforum.net Pharma Business International 7 04-07.qxp_Layout 1 12/10/2020 10:58 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net Observant readers might have noticed that our previous issue forwent the usual inclusion of our mergers and acquisitions round-up. Rather than an oversight, this omission occurred for one simple reason: there was simply a dearth of deals across the industry and, of those, none over the $1 billion dollar mark. It was an altogether quiet two-month period and, honestly, who can blame companies for playing it safe in the wake of the coronavirus pandemic. Now that it’s been ten months since the World Health Organisation declared COVID-19 a Public Health Emergency of International Concern, and we’ve entered what governments and media have taken to calling the ‘new normal’, companies across the pharmaceutical, life sciences and biotech sectors are once more looking to growth rather than playing it safe. Proof, if it were needed, was the announcement of the one of the year’s biggest deals when Gilead Sciences announced in September that it was acquiring New Jersey-based Immunomedics in a $21 billion deal. The deal sees Gilead bolster its oncology offering, providing the Californian biopharma giant with Trodelvy (sacituzumab govitecan-hziy), a first-in-class Trop-2 directed antibody-drug conjugate (ADC). The drug was granted accelerated approval by the US Food and Drug Administration (FDA) in April for the treatment of adult patients with metastatic triple-negative breast cancer (mTNBC) who have received at least A rise in activity After a quiet period of M&A activity for the industry, Pharma Business International explores a slew of multibillion-dollar-deals. © Shutterstock /Jarun Ontakrai 08-11.qxp_Layout 1 12/10/2020 10:59 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP two prior therapies for metastatic disease. Immunomedics plans to submit a supplemental Biologics License Application (BLA) to support full approval of Trodelvy in the US in the fourth quarter of 2020 (when the deal is anticipated to close). Immunomedics is also on track to file for regulatory approval in Europe in the first half of 2021. “This acquisition represents significant progress in Gilead’s work to build a strong and diverse oncology portfolio,” said Daniel O’Day, Chairman and CEO of Gilead Sciences, when the deal was announced. “Trodelvy is an approved, transformational medicine for a form of cancer that is particularly challenging to treat. We will now continue to explore its potential to treat many other types of cancer, both as a monotherapy and in combination with other treatments.” We’ve not only got one multi-billion- dollar deal to explore this issue, but two. In early September, it was revealed that Nestlé Health Science (NHSc) was acquiring Californian biopharma Aimmune Therapeutics in a $2.6 billion deal to create a “global leader” in food allergy prevention and treatment. The acquisition is an extension of NHSc’s food allergy portfolio, creating a broader spectrum of solutions for children living with food allergies. It grants NHSc access to Aimmune’s Palforzia, the first and only FDA- approved treatment to help reduce the frequency and severity of allergic reaction to peanuts – including anaphylaxis – in children aged four through seventeed. © Shutterstock /Sundry Photography 10 Á 08-11.qxp_Layout 1 12/10/2020 10:59 Page 2Next >