Your guide to the pharmaceutical world Volume 8 Issue 3 M&A round-up Dealmaking in a pandemic Rare diseases Disrupting innovation Curing coronavirus M&A round-up Dealmaking in a pandemic Rare diseases Disrupting innovation Curing coronavirus www.pbiforum.net 01.qxp_Layout 1 09/06/2020 10:55 Page 1WHERE PRODUCTIVITY MATTERS See the latest processing and packaging machinery, robotics and machine vision in action Network with industry peers and gain valuable insight at our must attend seminars ppmashow.co.uk 29 SEPT - 1 OCT 2020 NEC, BIRMINGHAM t our ars OWNED AND ORGANISED BY PPMA Group of Associations is a trading brand of PPMA Limited. SCAN TO REGISTER PPMA Advert 210x297.indd 108/04/2020 12:27 02.qxp_Layout 1 09/06/2020 10:55 Page 1Contents Volume 8 Issue 3 4 12 24 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 Given the continuing coronavirus crisis impacting the global economy, it’s unsurprising that M&A activity has cooled in the pharmaceutical and life sciences sectors. Although dealmaking had expected to continue strongly for 2020, few could have foreseen not only the arrival of COVID-19, but how deeply it would affect the operations and outlooks of companies, big and small. Tuberculosis exposé12 Once treated with sunlight and fresh air in sanatoriums, TB treatment saw great change with the development of effective chemotherapy in the 1940s. Though breakthroughs in the TB space have since been slow, innovation is picking up pace. Finding a cure for coronavirus16 With the number of reported COVID-19 cases well past six million, pharmaceutical companies and researchers are working rapidly to develop vaccines and uncover effective treatments. Rare diseases20 The outbreak of COVID-19 has seen investment and research shift away from rare diseases, putting the lives of patients at risk. Outsourcing24 Although outsourcing production carried inherent risk for pharmaceutical companies, it is becoming industry standard for many companies. Funding28 Even the biggest pharmaceutical players are required to secure funding to bankroll drug development or to move a novel new substance into clinical trial. But for research firms and small-scale developers, funding is the very lifeblood of their research and development programmes. Logistics 32 Transport is a go-to location for cutting costs, but how much further can the industry be stretched? Events 36 We give a rundown of pharma events happening around the world. @pbiforum www.pbiforum.net company/pharma-business- international Cover photo courtesy: Shutterstock / Lightspring 03.qxp_Layout 1 09/06/2020 10:57 Page 1Antidepressant fluvoxamine could treat COVID-19 Researchers from Washington University School of Medicine are now conducting a clinical trial of antidepressant drug fluvoxamine as a potential treatment for the ‘cytokine storm’ associated with COVID-19. If found effective, this selective serotonin reuptake inhibitor (SSRI) could be rapidly made available to patients as it is already globally marketed and has a known safety record, says GlobalData. “Having a treatment for cytokine storm that is already so easily available will help reduce mortality and prevent progression from mild-to-severe COVID-19, as well as increase fluvoxamine’s market share,” said Johanna Swanson, Product Manager at GlobalData. “The drug will be competing with tocilizumab, baricitinib and sarilumab which have been approved for rheumatoid arthritis with global sales of 2,208 million, 203 million and 109 million, respectively.” 4 Pharma Business International www.pbiforum.net Latest news Gilead & Arcus partner to develop next-gen cancer immunotherapies Gilead Sciences and Arcus Biosciences, an oncology-focused biopharma working to create best-in-class cancer therapeutics, have entered into a ten-year partnership to co-develop and co- commercialise current and future therapeutic product candidates in Arcus’s pipeline. The agreement will also provide ongoing funding to support Arcus’s research and development programs. “By gaining access to its broad, diverse pipeline and Arcus’s clear strengths in discovery and development, we believe that our partnership with Arcus will significantly accelerate our progress in developing transformative new therapies for cancer,” said Daniel O’Day, Chairman and CEO of Gilead Sciences Arcus will receive $375 million upon closing, consisting of a $175 million upfront payment and a $200 million equity investment from Gilead. The company is eligible to receive up to $1.225 billion in opt-in and milestone payments with respect to its current clinical product candidates. CEPI invest further funds in Novavax for COVID-19 vaccine The Coalition for Epidemic Preparedness Innovations (CEPI) will invest up to $388 million of additional funding in next-gen vaccine maker, Novavax, to fund the development and manufacture of a COVID-19 vaccine. This latest funding follows an initial $4 million CEPI invested in the Maryland-based biotech back in March and will continue to fund the clinical development of NVX-CoV2373, Novavax’ coronavirus vaccine candidate against SARS-CoV-2. It will also support rapid scale-up of the NVX-CoV2373 vaccine antigen, as well as Novavax’ proprietary Matrix-M adjuvant, which is expected to enhance immune responses by stimulating high levels of neutralizing antibodies. In addition, the CEPI funding will allow Novavax to dramatically increase its large-scale manufacturing capacity for both antigen and adjuvant in multiple locations. © Shutterstock/kubicka © Shutterstock / CI Photos © Shutterstock / PhotobyTawat 04-07.qxp_Layout 1 09/06/2020 10:59 Page 1CiRA Foundation & CGT Catapult to collaborate on stem cell research CiRA Foundation and the Cell and Gene Therapy Catapult (CGT Catapult) are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation. The project aims to further the application of iPS cell technologies for the manufacture of regenerative medicine products. The potential of distinct iPS cell lines for differentiation into specific cell types, such as cardiac muscle cells or white blood cells, is usually biased towards some cell line-specificity which is very difficult to predict. In order to select an appropriate iPS cell line for clinical trials, it is currently necessary to differentiate several candidate cell lines, which requires a great amount of time and effort. By combining their expertise, the companies will explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products. Pharma Business International 5 www.pbiforum.net Latest news Dyno launches to transform gene therapy using AI Dyno Therapeutics, a Cambridge, Massachusetts-based biotech applying AI to gene therapy has officially launched from stealth mode with its proprietary platform, CapsidMap. The platform enables the design of novel Adeno-Associated Virus (AAV) vectors that significantly improve upon current approaches to gene therapy and expand the scope of accessible diseases. Through its R&D and collaborations with biopharmaceutical companies, Dyno has active programs focused on novel gene therapy vectors for ophthalmic, muscle, central nervous system (CNS), and liver diseases. The company could potentially receive well over $2 billion in upfront payments, research support, option fees, as well as pre-clinical, clinical, regulatory and sales milestones under its collaboration agreements. The company launched in late 2018 with a $9 million financing co-led by Polaris Partners and CRV. Alan Crane, a co-founder of Dyno and Entrepreneur Partner at Polaris Partners, and Dylan Morris, General Partner at CRV, have joined Dyno’s board of directors, with Alan Crane serving as Dyno’s Executive Chairman. First generic remdesivir for COVID-19 treatment launched Beximco Pharmaceuticals, the Bangladesh-based manufacturer of generic pharma products and APIs, has launched the world’s first generic remdesivir for treating COVID-19. Remdesivir is an antiviral drug that was recently granted Emergency Use Authorisation by the US FDA Food for the treatment of COVID-19. The launch the grant of Emergency Use Authorisation by the Directorate General of Drug Administration (DGDA) – the regulatory authority in Bangladesh – for Beximco Pharma’s remdesivir IV injection (under the brand name Bemsivir) received on 21 May. Emergency approvals will help to broaden the use of remdesivir in hospitalised patients, especially in developing and least developed countries where access to breakthrough, advanced drugs remains a Originally developed by Gilead Sciences, remdesivir is a direct acting antiviral drug that inhibits viral RNA synthesis. Remdesivir is administered intravenously and is authorised for the treatment of hospitalised patients with severe COVID-19 disease. © Shutterstock / felipe caparros © Shutterstock / CI Photos © Shutterstock / metamorworks 04-07.qxp_Layout 1 09/06/2020 10:59 Page 26 Pharma Business International www.pbiforum.net Latest news Scientists explore using ‘own’ immune cells to target COVID-19 The engineering of specific virus-targeting receptors onto a patient’s own immune cells is now being explored by scientists from Duke-NUS Medical School (Duke-NUS) in Singapore, as a potential therapy for controlling infectious diseases, including the COVID-19-causing virus, SARS-CoV-2. This therapy that has revolutionised the treatment of patients with cancer has also been used in the treatment of other infectious diseases such as Hepatitis B virus (HBV), as discussed by the School’s researchers in a commentary published in the Journal of Experimental Medicine. This therapy involves extracting immune cells, called T lymphocytes, from a patient’s blood stream and engineering one of two types of receptors onto them: chimeric antigen receptors (CAR) or T cell receptors (TCR). TCRs are naturally found on the surfaces of T lymphocytes while CARs are artificial T cell receptors that are generated in the laboratory. These receptors allow the engineered T lymphocytes to recognise cancerous or virus infected cells. mRNA-based vaccine tech emerging as frontrunner against COVID-19 Messenger RNA (mRNA) based vaccine technology is emerging as one of the frontrunners among the technologies being evaluated to develop a vaccine for COVID-19, with eighteen candidates currently in development globally, according to GlobalData. “RNA vaccines are an innovative approach as they offer several advantages over conventional vaccines, particularly when responding to a pandemic threat,” said Keshalini Sabaratnam, Pharma Analyst at GlobalData. “While conventional vaccines developed with an attenuated or inactivated disease-causing pathogen or using proteins made by the pathogen (antigens) require long production times and have limited production capacity, RNA vaccines work by introducing an mRNA sequence that encodes the disease- specific antigen to trigger the body’s immune system. “A major advantage of RNA vaccines is that RNA can be produced rapidly and cheaply in the laboratory, offering the potential to scale up production to meet the demands of a pandemic. Other advantages include enhanced immunogenicity and better safety profile compared to classical and other types of vaccines, as has been proven in clinical trials.” Over 150 COVID-19 vaccines are currently under development by biopharmaceutical companies and research organizations around the world. Nine of them are in clinical trials. While SinoVac biotech’s inactivated SARS-CoV-2 vaccine and University of Oxford’s ChAdOx1-nCov19, a recombinant viral vector vaccine, were first to progress ahead of others to Phase I/II studies, RNA vaccine candidates were not far behind. BioNTech and Pfizer initiated Phase I/II clinical studies for its BNT162 RNA vaccine program in Germany on April 23, 2020. Trials for BNT162 will also be conducted in the US and in China. Moderna’s mRNA vaccine candidate mRNA-1273 is expected to enter a Phase II study in Q2 2020. Moderna was the first company to test a vaccine against COVID-19 in humans. AstraZeneca & Oxford University partner for COVID-19 vaccine AstraZeneca and the University of Oxford have announced a landmark agreement for the global development and distribution of the latter’s potential recombinant adenovirus vaccine aimed at preventing COVID-19. The collaboration aims to bring to patients the potential vaccine known as ChAdOx1 nCoV-19, being developed by the Jenner Institute and Oxford Vaccine Group, at the University of Oxford. Under the agreement, AstraZeneca would be responsible for development and worldwide manufacturing and distribution of the vaccine. Alok Sharma, UK Business Secretary, said: “This collaboration between Oxford University and AstraZeneca is a vital step that could help rapidly advance the manufacture of a coronavirus vaccine. “It will also ensure that, should the vaccine being developed by Oxford University’s Jenner Institute work, it will be available as early as possible, helping to protect thousands of lives from this disease.” © Shutterstock/fusebulb © Shutterstock / petarg © Shutterstock/joel bubble ben 04-07.qxp_Layout 1 09/06/2020 10:59 Page 3 Japan approves remdesivir for COVID-19 Gilead’s Veklury (remdesivir) has been approved in Japan as a treatment for COVID-19 under an exceptional approval pathway. The exceptional approval was granted in the wake of the COVID-19 pandemic and references the Emergency Use Authorisation of remdesivir in the United States. The approval is based on clinical data from the US National Institute of Allergy and Infectious Diseases’ global Phase 3 trial, Gilead’s Phase 3 SIMPLE trial in patients with severe manifestations of COVID-19, and available data from Gilead’s compassionate use program, including patients in Japan. Tecentriq gains first-line approval in NSCLC The FDA has approved Roche’s Tecentriq (atezolizumab) as a first-line monotherapy for the treatment of metastatic non-small cell lung cancer (NSCLC), marking its fourth approval in the indication and fifth in lung cancer overall. The approval is based on an interim analysis from the Phase III IMpower110 study, which showed that Tecentriq monotherapy improved overall survival by 7.1 months compared with chemotherapy in people with metastatic NSCLC whose tumours have high PD-L1 expression. Tecentriq is the first and only single-agent cancer immunotherapy with three dosing options, allowing administration every two, three or four weeks. Emergency use authorisation for Gilead’s remdesivir Gilead’s investigational antiviral remdesivir has been granted emergency use authorisation (EUA) by the FDA to treat COVID-19. The EUA will facilitate broader use of remdesivir to treat hospitalized patients with severe COVID-19 disease, enabling access to remdesivir at additional hospitals across the country. Allocation of the currently limited available supply of remdesivir will be made based on guiding principles that aim to maximize access for appropriate patients in urgent need of treatment, with direction from and in collaboration with the government. GSK picks up US approval for Zejula GlaxoSmithKline has gained FDA approval for its supplemental New Drug Application (sNDA) for Zejula (niraparib) making it the only approved once-daily PARP inhibitor in first-line monotherapy maintenance treatment for women with platinum-responsive advanced ovarian cancer regardless of biomarker status. This new indication is supported by data from the phase III PRIMA study (ENGOT-OV26/GOG-3012), which enrolled patients with newly diagnosed advanced ovarian cancer following a complete or partial response to platinum-based chemotherapy regardless of biomarker status. FDA approves first targeted treatment for rare bile duct cancer The FDA has granted accelerated approval to Incyte Corporation for Pemazyre (pemigatinib), the first treatment approved for adults with certain types of previously treated, advanced cholangiocarcinoma. Pemazyre’s approval was based on the results of a clinical trial that enrolled 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior treatment. Koselugo approved in US for paediatric patients for rare genetic condition US regulators have granted approval to AstraZeneca and Merck for the kinase inhibitor Koselugo (selumetinib) for the treatment of paediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). The approval by the FDA was based on positive results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)- sponsored Phase II SPRINT Stratum 1 trial coordinated by the NCI’s Center for Cancer Research, Paediatric Oncology Branch. This is the first regulatory approval anywhere in the world of a medicine for the treatment of NF1 PN, a rare and debilitating genetic condition. European approval for gene therapy to treat SMA European regulators have granted conditional approval to Novartis company AveXis for the gene therapy Zolgensma (onasemnogene abeparvovec) for the treatment of paediatric patients with 5q spinal muscular atrophy (SMA). “The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.” said Dave Lennon, President of AveXis. Breakthrough status for Enhertu in NSCLC AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of patients with metastatic non-small cell lung cancer (NSCLC). The FDA granted BTD based on data from the ongoing Phase II DESTINY-Lung01 trial currently testing Enhertu, a HER2-directed antibody drug conjugate, in patients with HER2-mutan metastatic NSCLC, and data from the Phase I trial published in Cancer Discovery. The overall safety and tolerability profile of Enhertu in the ongoing DESTINY-Lung01 trial is consistent with that seen in the Phase I trial. www.pbiforum.netPharma Business International 7 04-07.qxp_Layout 1 09/06/2020 10:59 Page 4M&A ROUND-UP 8 Pharma Business International www.pbiforum.net W here the billion-dollar-plus deals are concerned, there’s only one acquisition of note to explore this issue. Towards mid-May, Boston, Massachusetts-based Alexion announced its intention to expand its haematology, neurology and critical care commercial portfolio with the $1.4 billion acquisition of San Francisco biotech Portola Pharmaceuticals. It’s easy to see why Portola would be an attractive buy for Alexion. Andexxa (Ondexxya in Europe), Portola’s commercialised medicine, is the first and only approved Factor Xa inhibitor reversal agent and has demonstrated transformative clinical value by rapidly reversing the anticoagulant effects of Factor Xa inhibitors rivaroxaban and apixaban in severe and uncontrolled bleeding. Consequently, the acquisition will add near-term diversification to Alexion’s commercial portfolio and provides the opportunity to apply the company’s demonstrated global commercial excellence to create long-term value for patients and shareholders. “The acquisition of Portola represents an important next step in our strategy to diversify beyond C5,” said Ludwig Hantson, CEO of Alexion. “Andexxa is a strategic fit with our existing portfolio of transformative medicines and is well- aligned with our demonstrated expertise in haematology, neurology and critical care. “We believe Andexxa has the potential to become the global standard of care for patients who experience life-threatening bleeds while taking Factor Xa inhibitors apixaban and rivaroxaban. “By leveraging Alexion’s strong operational and sales infrastructure and deep relationships in hospital channels, we are well positioned to expand the number of patients helped by Andexxa, while also driving value for shareholders.” Given the continuing coronavirus crisis impacting the global economy, it’s unsurprising that M&A activity has cooled in the pharmaceutical and life sciences sectors. Although dealmaking had expected to continue strongly for 2020, few could have foreseen not only the arrival of COVID-19, but how deeply it would affect the operations and outlooks of companies, big and small. But that’s not to say there hasn’t been any deals since our last issue. Dealmaking in a pandemic 10 Á 08-11.qxp_Layout 1 09/06/2020 11:06 Page 1Pharma Business International 9 www.pbiforum.net M&A ROUND-UP © Shutterstock /eamesBot As we were finalising this issue, a Bloomberg report claimed that AstraZeneca approached remdesivir maker Gilead “over possible merger”. If the reports were correct, the £200 billion merger would be the biggest ever pharmaceuticals deal. For updates, keep an eye on our homepage. 08-11.qxp_Layout 1 09/06/2020 11:06 Page 2Next >