Your guide to the pharmaceutical world Volume 7 Issue 2Birth Control Exposé The new wave of male birth control Processing Automating excellence Gene Editing Redesigning DNABirth Control Exposé The new wave of male birth control Processing Automating excellence Gene Editing Redesigning DNAwww.pbiforum.net01.qxp_Layout 1 05/04/2019 13:21 Page 1FOR MANUFACTURING IN FOOD, BEVERAGES, PHARMACEUTICALS, TOILETRIES, COSMETICS AND MORE…1-3 OCTOBER 2019 NEC, BIRMINGHAM THE COMPLETE PRODUCTION LINE EVENTppmatotalshow.co.ukOWNED & ORGANISED BYFREE TO ATTEND REGISTER NOW02.qxp_Layout 1 05/04/2019 13:21 Page 1@pbiforumwww.pbiforum.netcompany/pharma-business-internationalContents Volume 7 Issue 2News4 Stay up to date with the latest industry updates from the global pharmaceutical technology industry. M&A Round-up8 2019 has had a strong start for the pharma M&A market with major deals completing and the announcement of one of the industry’s biggest acquisitions to date. Birth Control Exposé12 Despite a near sixty-year history, modern contraception has been aimed almost exclusively at women. At present, men are limited to condoms or a vasectomy, but a male hormone contraceptive might be closer to market than not. Processing16 With access to medicines continuing to advance, and patient care improving all round, new revenue streams are being made available to the market year on year. As with almost every other industry reliant on complex manufacturing, automated technology is having a transformative effect. Collaboration20 Collaborations between the pharmaceutical sector and digital technology specialists is bringing treatment and medicine into the digital age. Health, Safety & Hygiene24 The need for rigorous health and hygiene levels in the pharmaceutical industry can perhaps go without saying, but some areas require more stringent levels than others – namely cleanrooms. The Ethics in Gene Editing28 Since first emerging as a scientific tool in the seventies, gene editing has presented a unique ethical dilemma for the scientific community. Vitafoods Europe32 Taking place from 7-9 May at Palexpo in Geneva, Vitafoods Europe will bring the industry together to discuss key challenges such as sustainable sourcing, and wider trends including free-from, plant-based ingredients, and hemp extracts and CBD. Events34 We give a rundown of pharma events happening around the world.4121624Group Editor: Steve Fisher (s.fisher@blmgroup.co.uk) Editor: Michael Fisher (m.fisher@blmgroup.co.uk) Journalist: Dominic Cuthbert (d.cuthbert@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, N E Lincs 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.COVER PHOTO: © SHUTTERSTOCK / AREEYA_ANN03.qxp_Layout 1 05/04/2019 13:22 Page 1New global flu strategy launched by WHO The World Health Organisation (WHO) has launched a new Global Influenza Strategy for 2019-2030 to help protect people in all countries from the threat of flu. WHO said the goal of the strategy is to “prevent seasonal influenza, control the spread of influenza from animals to humans, and prepare for the next influenza pandemic”. The new strategy is the most comprehensive and far-reaching that WHO has ever developed for influenza. It outlines a path to protect populations every year and helps prepare for a pandemic through strengthening routine programmes. It has two overarching goals: * Build stronger country capacities for disease surveillance and response, prevention and control, and preparedness. * Develop better tools to prevent, detect, control and treat influenza, such as more effective vaccines, antivirals and treatments, with the goal of making these accessible for all countries.4 Pharma Business International www.pbiforum.netLatest newsAkili & Shionogi commercialising digital medicines in East Asia Akili Interactive has entered into a strategic partnership with Japan’s Shionogi to commercialise two digital medicines in Japan and Taiwan. AKL-T01 is currently under review with the US FDA as a potential digital treatment for children with ADHD. AKL-T02 is in late stage development as a potential digital treatment for cognitive dysfunction and related symptoms in children with Autism Spectrum Disorder (ASD). Both are delivered through immersive action video game experiences. Shionogi will be responsible for regulatory filings and has exclusive rights to the clinical development, sales and marketing of AKL-T01 and AKL-T02 in Japan and Taiwan. Akili will build and maintain control of a newly created Global Access Platform – an R&D and commercial platform including all global product development activities, distribution, technical support services. Shionogi has made upfront payments to Akili totalling $20 million who will be eligible to receive development and commercial milestones of up to $105 million.Getting to grips with regulatory compliance weighing in pharma manufacturing Weighing in pharmaceutical production strongly influences the quality of the final product. By selecting the right equipment and implementing correct calibration procedures, manufactures can ensure compliance with relevant regulatory bodies. Considerations include: • How to implement relevant regulations • How to select the right weighing system • How often to schedule routine test and calibration intervals Discover more about regulatory compliance by downloading the white paper – ‘Regulatory Compliance Weighing in Pharma Manufacturing’ and find out why regulations such as GMP, USP and ISO have implications on weighing systems. Register for the 30-minute webinar which explains more about interpreting and implementing relevant regulations. To download the white paper, visit www.mt.com/ind-pharma-regulations. © Akili Interactive Labs© Shutterstock / solarseven04-07.qxp_Layout 1 05/04/2019 13:23 Page 1Oxford Biomedica & Microsoft advancing gene therapy Oxford Biomedica is aiming to improve the yield and quality of next-gen gene therapy vectors using the cloud and machine learning in a new R&D collaboration with Microsoft. The collaboration will combine the expertise of Oxford Biomedica researchers and the team within the Station B initiative at Microsoft to explore new ways to increase the yield and improve the purity of Oxford Biomedica’s lentiviral vectors, while further reducing the cost. Oxford Biomedica will contribute large data sets for analysis via the Microsoft Azure intelligent cloud platform. Microsoft, working with Oxford Biomedica scientists, will utilise its cloud computing and machine learning capabilities to develop in silico models and novel algorithms to help advance the next generation of cell and gene delivery technology. The collaboration will run for an initial two-year period and may be extended by either party.Pharma Business International 5www.pbiforum.netLatest newsNew biotech targeting muscle diseases launches with $50m A new biotech company pioneering therapies for patients with serious muscle diseases has launched this week with $50 million series A finding. Dyne Therapeutics was founded, seeded and incubated and joined by Forbian and MP Capital in the financing round. Dyne is developing breakthrough medicines based on its proprietary product platform, which delivers nucleic acids and other molecules to skeletal, cardiac and smooth muscle with unprecedented precision. The company’s lead programs are designed to knock down gene expression for the treatment of rare, monogenic neuromuscular diseases. It is also developing therapeutics for patients with cardiac and smooth muscle diseases. It will initially focus on myotonic dystrophy type 1 (DM1), a rare, inherited disorder that causes muscle weakness and other life-limiting complications. No link between MMR vaccine and autism, study finds A major study involving researchers from Denmark’s Statens Serum Institut (SSI) shows that there’s no link between the measles, mumps and rubella (MMR) vaccine and children’s risk of developing autism. The results come as Europe is experiencing a tripling of measles cases in a year. For more than 20 years, claims have been made that the MMR vaccine increases children’s risk of developing autism. Researchers have now invalidated these claims for the second time. In 2002, SSI and Aarhus University performed a large study of 537,303 children. The study found no association between MMR vaccine and autism. Now SSI researchers have performed a brand-new and even bigger study that also concludes no association. The results are published in Annals of Internal Medicine.© Shutterstock / Zahraa Saleh© Shutterstock / Natali_Mis© Shutterstock / BigBlueStudio04-07.qxp_Layout 1 05/04/2019 13:23 Page 26 Pharma Business International www.pbiforum.netLatest newsPARP inhibitors could boost effects of immunotherapy A new study shows that PARP inhibitors have a previously unknown ability to boost the immune system, meaning it could help more patients benefit from immunotherapy. Scientists found that PARP inhibitors sparked a powerful immune response when used against cancer cells with weaknesses in repairing their DNA. The study changes our understanding of how PARP inhibitors work and suggests they could be used alongside immunotherapies to boost their effectiveness. Clinical trials have already started to assess this combination. Some patients have benefited dramatically from a new generation of immunotherapies – but often only between 10 and 20% of patients will respond, with many others’ cancers able to hide from the immune system. Study leader Professor Chris Lord, Professor of Cancer Genomics at The Institute of Cancer Research, London, said: “The findings of this study substantially change our understanding of how PARP inhibitors work.”The US National Institutes of Health (NIH) has begun the first-in-human trial of a universal flu vaccine candidate. The experimental vaccine – known as H1ssF_3928 – was developed by Scientists at the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH. It is designed to teach the body to make protective immune responses against diverse influenza subtypes by focusing the immune system on a portion of the virus that varies relatively little from strain to strain. The vaccine candidate was developed as part of a broader research agenda to create a so-called “universal” influenza vaccine that can provide long-lasting protection for all age groups from multiple influenza subtypes, including those that might cause a pandemic. NIAID Director Anthony S. Fauci, said: “This Phase 1 clinical trial is a step forward in our efforts to develop a durable and broadly protective universal influenza vaccine.”Novartis & Black Life Sciences launch new biopharma Novartis and Blackstone Life Sciences have launched Anthos Therapeutics, a new biopharma company focused on advancing next-gen targeted therapies for high-risk cardiovascular patients. Funds managed by private investment platform Blackstone Life Sciences provided $250 million for Anthos and will control the development of the products. Novartis will retain a minority equity interest in Anthos. As part of this launch, Novartis has licensed to Anthos MAA868, an antibody directed at Factor XI and XIa, key components of the intrinsic coagulation pathway. Founding members of Anthos’ Board of Directors include Blackstone MD Paris Panayiotopoulos, former CEO of Ariad Pharmaceuticals, and Blackstone Principal Ari Brettman, MD. Jonathan Freeman, MBA, Ph.D., Blackstone Senior Advisor and Anthos co-founder, joins as Chief Operating Officer. John Glasspool, a former leader of Novartis’ cardiovascular franchise with 30 years of experience in the biopharmaceutical industry, joins Anthos as CEO.© Shutterstock / Tero Vesalainen© Shutterstock / fusebulb© Shutterstock/studio formfreiFirst-in-human trial of universal flu vaccine candidate begins04-07.qxp_Layout 1 05/04/2019 13:23 Page 3 EUROPE Forxiga approved for type-1 diabetes Forxiga (dapagliflozin) has been approved as an adjunct to insulin in patients with type-1 diabetes. As well as being AstraZeneca’s first medicine approved for type-1 diabetes, Forxiga is also the first oral medicine approved in Europe as an adjunct to insulin. The approval is based on data from the Phase III DEPICT clinical programme for Forxiga in type-1 diabetes. Keytruda approved with chemo for NSCLC Regulators have approved Merck’s Keytruda in combination with chemotherapy as a first-line treatment for adults with metastatic squamous non-small cell lung cancer (NSCLC). Based on data from the Phase 3 KEYNOTE-407 trial, the approval allows marketing of the combination in all 28 EU member states plus Iceland, Lichtenstein and Norway. Expanded approval for Takeda’s Adcetris Marketing authorisation of Adcetris has been expanded in Europe to include the treatment of patients with previously untreated CD30+ Stage IV Hodgkin lymphoma. Adcetris is an antibody-drug conjugate jointly developed by Takeda and Seattle Genetics directed at CD30, a defining marker of Hodgkin lymphoma. The decision follows a positive opinion from the Committee for Medicinal Products for Human Use in December 2018. Roche’s MabThera approved for pemphigus vulgaris Roche’s MabThera (rituximab) has been approved for the treatment of adults with moderate-to-severe pemphigus vulgaris (PV). MabThera is the first biologic therapy approved by the European Commission for PV and the first major advancement in the treatment of the disease in more than 60 years. UNITED STATES Green light for postpartum depression treatment Zulresso (brexanolene) injection has become the first and only medicine specifically approved for treating postpartum depression. Sage Therapeutics said that Zulresso is expected to be available in late June following scheduling by the US Drug Enforcement Administration. Tecentriq/chemo combo approved for ES-SCLC Tecentriq has been approved in combination with chemotherapy for the first-line treatment of adults with extensive small cell lung cancer (ES-SCLC). Roche said the approval was based on results from the Phase III IMpower133 study which showed that the combo helped people live significantly longer compared to chemotherapy alone in the intention-to-treat population. Nasal spray approved for treatment-resistant depression Janssen has secured approval for Spravato (esketamine) CII nasal spray for use in conjuncture with an oral antidepressant in adults with treatment-resistant depression (TRD). Spravato uses the first new mechanism of action in decades to treat major depressive disorder. The medicine is administered as a nasal spray that is absorbed by the lining of the nasal passages and into the blood stream. It was studied in a robust Phase 3 clinical trial program with more than 1,700 adults with TRD. Botox generic approved in US, eyes Spring launch Jeuveau, a Botox generic from California’s Evolus, has become the first aesthetic-only neurotoxin approved in the US after getting rubber-stamped by the FDA. “Evolus is the first company in nearly a decade to enter the fast-growing US aesthetic neurotoxin market,” said David Moatazedi, President and CEO of Evolus. “We are pleased to introduce Jeuveau, the first FDA approved neurotoxin dedicated to aesthetics and manufactured in a state-of-the-art facility using Hi-Pure technology.” www.pbiforum.netPharma Business International 704-07.qxp_Layout 1 05/04/2019 13:23 Page 4M&A ROUND-UPSome of the world’s biggest pharmaceutical companies are using acquisitions to expand their positions in the burgeoning gene therapy field. The pharmaceutical mergers and acquisitions market has so far this year been dominated by companies consolidating, strengthening and expanding their presence and pipeline in the booming cell and gene therapy space. The last two months have yielded a number of high-profile deals indicating the direction of the industry and its most prominent players. The biggest by far has been Roche’s $4.3 billion acquisition of Spark Therapeutics, a Pennsylvanian company specialising in gene therapies for blindness, haemophilia and neurodegenerative diseases, among others. In 2017, it become the first company to secure Food and Drug Administration approval for a gene therapy aimed at a genetic disease. It’s an advantageous move for Roche who gains access to the company’s lead clinical asset, a novel gene therapy for treating haemophilia A, which is slated for Phase 3 trials later this year. Roche Gene therapy drives M&A 8 Pharma Business International www.pbiforum.net10 Á08-11.qxp_Layout 1 05/04/2019 13:24 Page 1Pharma Business International 9 www.pbiforum.netM&A ROUND-UP© Pedro Martinez Valera / Shutterstock08-11.qxp_Layout 1 05/04/2019 13:24 Page 2Next >