European Biotechnology News 9/2012 - Free Excerpt - Gene therapy rounds the bend in Europe

EuropeanBiotechnology

Science & Industry News

Nº 9 | Volume 11 | 2012 | 10.00 € | ISSN 1618-8276 | A 60711 |

CENTRAL EUROPE

Molecular Partners signs US$1.1bn deal with Allergan

NORTHERN EUROPE

Russian investors swallow Denmark's Affitech A/S

WESTERN EUROPE

Sanofi files to switch indications for alemtuzumab

SPECiAL

EuroBiotech Compass 2012Guide to Life Sciences Events

SOUTHERN EUROPE

Antibióticos in bioplastics deal with US developer Metabolix

EASTERN EUROPE

Polish insulin maker Bioton cashes in final Bayer cheque

SCiENCE & TECHNOLOgy

Research teams prove existence of cancer stem cells

Gen

e th

erap

y ro

unds the bend in Europe

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Euro|Biotech|NewsN º– 9 | Volume 11 | 2012 3

INtro

During the last thirty years, healthcare expenditure has been growing much more rapidly than GDP in OECD

countries, causing increasing concerns about the long-term sustainability of current trends. As we have seen with the global impact of the financial crisis, economic realities are moving faster than political ones. The standards of healthcare that European citizens are provided with today risk becoming economically unsus-tainable, if the efficiency of healthcare systems is not factored in when assessing the value of prevention and treatment of diseases.

Against this background, we need to look in more detail at the determinants of healthcare expenditure in the countries of Europe, explic-itly taking into account aspects like the role of income, the effects of an ageing population, life habits, technological progress, and institu- tional and budgetary variables.

When developing products and solutions for European patients today, the research-based pharmaceutical industry is responding to evi-dence -based requirements put in place by regu-latory agencies – which request proof of efficacy, quality and safety – and 30+ Health Technolo-gy Assessment (HTA) agencies across Europe, which want proof of relative effectiveness. In the area of prevention, the vaccine industry has to provide dedicated evidence to public authorities, which in turn make recommendations prior to any public prevention programme. There is a potential for unnecessary duplication between these different requirements, as they are actu-ally sourced from the same sets of data.

The need to secure timely, equitable and sus-tainable patient/citizen access to healthcare services has led to a growth in collaboration between HTA agencies recently, and this has been strongly supported by the European Com-mission. Industry believes there is room for this

initiative, whilst simultaneously keeping regu-latory and HTA processes separated and keep-ing pricing and reimbursement decisions fully at the national level.

Of particular note is the collaboration between HTA agencies, which is supported by the Eu-ropean Commission and will be formalised in a permanent network as of next year. The cur-rent network (EUnetHTA) is working on de-veloping methodological guidance for relative effectiveness assessment. Industry is support-ing this approach, and is contributing to this discussion in order to feed in experience and know-how. Moving forward, industry would certainly favour increased collaboration be-tween the regulatory world and the European collaboration on HTA, as this would ensure that evidence-based requirements receive the best scientific attention and are aligned across the range of decisionmakers, allowing for an ongoing dialogue between the parties. What is crucial is that consultation with stakeholders, including those in industry, is warranted.

Today, the key challenge for governments is designing pluralistic systems of healthcare delivery and financing. In this area, a well-balanced mix of public and private financing would put market forces to work promoting investment and innovation, without impos-ing unsustainable burdens on public budgets or denying prevention and care to the disad-vantaged population. B

Editorial

Challenges and answers: HTA in the economic crisis

Andrea rappagliosi, Co-Chair EFPIA HtA task Force, Sanofi Pasteur MSD, Lyon

Andrea rappagliosi is Vice President Market Access, Vaccine Advocacy & Medical Affairs at Sanofi Pasteur MSD, the only company in Europe totally dedicated to vaccines. During his career in both the public and the private sectors, he has focused on access issues that impact patients and healthcare systems in Europe. rappagliosi is also VP of the European Vaccine Manufacturers Group and co-chair of the EFPIA HtA task Force. the author would like to express a special thank-you to Edith Frenoy, Director at EFPIA, for her contributions to these reflections.

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CIRCULATION

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EuroBiotechNews covers the biotechnology sector of the current 27 EU member states, Norway and Switzerland. If you would like to subscribe, please refer to

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coNteNts

Imprint European Biotechnology News is published monthly by: BIOCOM AG, Lützowstr. 33–36, D-10785 Berlin, Germany, Tel.: +49-30-264921-0, Fax: +49-30-264921-11, E-Mail: [email protected]; Internet: www.eurobiotechnews.eu, Publisher: Andreas Mietzsch, Editorial Team: Thomas Gabrielczyk (Managing Editor), Derrick Williams (Co-editor), Dr. Patrick Dieckhoff, Dr. Philipp Graf, Dr. Bernd Kaltwaßer, Dr. Martin Laqua; Advertising: Oliver Schnell, +49-30-2649-2145, Advertising USA: Avani Media, Inc. , Leslie Hallanan, Tel.: +1-415-331-2150 , Fax: +1-415-289-0402, E-Mail: [email protected]; Distribution: Angelika Werner, +49-30-2649-2140; Printed at: Druckhaus Humburg, Bremen; Graphic Design: Michaela Reblin. European Biotechnology Science & Industry News is only regularly available through subscription at BIOCOM AG. Annual subscription fees: € 100.00, Students € 50.00 (subject to proof of enrolment). Prices include VAT, postage & packaging. Ordered subscriptions can be cancelled within 2 weeks directly at BIOCOM AG. The subscription is initially valid for one year. Subscriptions will be renewed automatically for one more year, respectively, unless they are cancelled at least 6 weeks before the date of expiry. Failures of delivery which BIOCOM AG is not responsible for do not entitle the subscriber to delivery or reimbursement of pre-paid fees. Seat of court is Berlin, Germany. As regards contents: individually named articles are published within the sole responsibility of their respective authors. All material published is protected by copyright. No article or part thereof may be reproduced in any way or processed, copied and proliferated by electronic means without the prior written consent of the publisher. Cover Photo: © fotolia.de ® BIOCOM is a registered trademark of BIOCOM AG, Berlin, Germany.

Is gene therapy finally coming into its own? 6

INsIght

Heard in Brussels 10

First EuroBiotechMonitor survey finds business climate good for biotech in EU 12

Commissioners to reform EU Clinical trials directive; New EMA registry 13

IP Flash; Biotech SMEs meet in Brussels 14

RegulatoRy affaIRs

Breaking news from the EMA 16

Update on clinical trials 17

PeRsPectIVes

Interview: Olivier Litzka, Edmond de Rothschild Invest. Partner 18

ecoNomIc

Focus on newsflow and dividends 19

Stock markets 20

RegIoNal News

Northern Europe denmark, Sweden, Finland, Norway, Iceland 22

Central Europe Germany, Austria, Switzerland 24

Western Europe UK, France, Belgium 26

Southern Europe Italy, Spain, Portugal 28

Eastern Europe Poland, Hungary, and Slovenia 30

After a balancing act that went down to the wire with regulators – and left its predecessor company bankrupt – uniQure biotherapies now looks set to become the first pharma company to gain approval for a gene therapy in Europe. the concept of ‘cur-ing’ rare inherited diseases by altering a patient’s genome in key cells has been around for decades. But disastrous, highly-publicised failures to harness the technology in the past frightened off developers and patients alike. Now that looks set to change.

seRVIces

Imprint 5

Biopeople News from Photocure, AdC therapeutics, FENS, Wilex, Ariana Pharma, and Horizon discovery 46

Company index 47

New products 48

Events What’s on in Sept.-October 2012 49

Encore 50

© f

oto

lia.d

e

Cover Story

SpeCial

EuroBiofairs CompassNavigating in the events season 33

BioSpain 2012, Pamplona 34

BioPartnering Future Europe, Brussels 36

Planet xMAP 2012, Monaco 38

euroPLX, Sorrento, Italy 40

Journées Internationales de Biologie, Paris 42

BIO-Europe 2012, Hamburg 44

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10 Euro|Biotech|News N º– 9 | Volume 11 | 2012

CoVer Story

Claire Skentelbery, Secretary General of the european Biotechnology Network

Heard in BrusselsM Fixing europe (again)

Brussels – Well ladies and gentlemen of the bio technology community, wel-come back after the silly season. I hope you had a nice one, whether it was in the sunny bit, the rainy bit or the bits that were on fire.As usual, summer in Europe was quiet. I see we still have a Eurozone, which is nice, and shows that the revolutionary, bio tech -driven solution I came up with earlier in the year is still working.But to business – what startling insights and wisdom can I provide from Brus-sels this month? Very little based on activity over the summer, so I shall in-stead turn once again to my crystal ball, and present a list of predictions based on my incurable optimism.European public funding: after a year of wrestling with the Framework pro-gramme and Innovative Medicines In-itiative, my gut tells me that the agencies will at last start plan ning their funding together, finally al lowing partners to plan an annual cal endar of collabora-tive project applications without having to worry about deadlines all landing in the space of a single month.National funding: countries in Eu-rope will suddenly remember, in the midst of reducing funding for inno-vation, that it is innovation that drives economic growth. If all your scientists are fired, particularly those in SMEs, you throttle economic progress, mak-ing early-stage research pointless and large company growth in your coun-try impossible.Policymakers: these will return from their holidays miraculously alive to the fact that some things are not worth ar-guing about any more – I refer of course to GM technology in food crops (just say yes or no), the EU patent law (just get it finished for crying out loud) and con-sumer genetic testing legisla tion (don’t let quacks and charlatans sell tests).

Big companies: my inner eye is partic-ular focussed here, foreseeing that the pipeline crisis and continued shrink-age of R&D capacity will revolution-ise the way that large companies work with others. There will be less of a ‘business on our terms’ approach to partnership and more quick move-ment, shared skills and earlier-stage partnerships.Small companies: those rascals, they're taking up a lot of space in my crystal ball. Biotech SMEs will certainly start working more closely together, rath-er than trying to nurse a single tech-nology through to blockbuster status. And they will start to understand the strength brought about by partnership with other SMEs – that they are strong-er together than alone.That's it for my post-summer pre-dictions – and given my astounding success at curing the Eurozone cri-sis earlier this year, I'm sure they will all have come to pass by Christmas. See you next edition – by which time that holiday tan will have faded and the silly season will be a distant me- mory. B

LPLD patients who had already had acute pancreatitis one or more times.

Euro|BioTech|News ?How will this reduction in the patient pop-ulation affect your business?

AldAg: !The patient population is smaller. I would estimate that 350-700 of LPLD patients in Europe have already had acute pancreati-tis. But that's not a limitation to our busi-ness, because we won’t treat hundreds of patients in the first year after approval. We will first have to identify patients that car-ry the gene defect and have had pancreas inflammations. To do that, we have to go to special hospitals in Europe where pa-tients with acute pancreatitis are treated and provide the doctors with our compan-ion diagnostics chip. Our aim is to identi-fy patients in about 20 hospitals through-out the EU.

Euro|BioTech|News ?What price do you think is realistic?

AldAg: !You have to look at prices for other orphan drugs on the market. Our approach has a big advantage: it's a one-time treatment that is effective for years. But if Glybera offers at least the same benefit as compa-rable orphan treatments, prices should be similar. Prices for orphan drugs like Prov-enge, Cere zyme or Naglazyme range from a100,000-a450,000 annually. Since our therapy has already proven efficacy over several years after just one treatment, I think it's reasonable to start at the high end of this range. I think you can do the math. Before we have any agreement in place with healthcare payers, I am, however, re-luctant to spell out pricing details in the media. I am sure you appreciate that.

Euro|BioTech|News ?What’s your outlook?

AldAg: !We have learnt an incredible amount throughout the approval process. Now we have a unique technology platform to make hundreds of medicines. B

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12 Euro|Biotech|News N º– 9 | Volume 11 | 2012

INSIGHT EUROPE

EUROPE

EuroBiotechMonitor: surprising optimism Most European biotechnology companies expect higher revenues in 2012 than in 2011. Even though access to external fi nancing, especially venture capital, remains a wearisome prospect on the continent, the vast majority of biotechs say they aren’t planning to cut any jobs this year. In an exclusive, EuroBiotechNews has received those top-line results from the 1st EuroBiotechMonitor survey. The poll across 2,100 European biotech compa-nies was commissioned by the Brussels-based European Bio technology Network. The results of the very fi rst business climate report dedicated to the European biotech sec-tor show that the fi eld remains optimistic despite a challenging business environment.

More cash than expected?

External � nancing was one problem most companies experienced. While public or larger companies have enough critical mass to attract signi� cant amounts of ex-ternal � nancing and licensing deals, the majority of smaller biotechs do not. Ac-cording to the survey, 21% of the respond-ents said that “bad access to � nancing hin-dered business development.” Around the same number said that the EU should in-crease its investment incentives to support biotech SMEs. Although many companies said that establishing EU-funded partner-ships was “easy” (48%) or “manageable”, around a quarter thought that improve-ments in R&D funding on the part of the EU and less bureaucracy (25%) could help the sector.

Surprisingly, 37% of the � rms speci� ed that they had enough cash to last for 12-24 months, while an additional 20% had enough for even longer. Around the same number said that access to external sourc-es of � nancing during the last 12 months was “good”, while a further 33% called it “satisfactory”. One in four said it was “bad” or “not relevant”.

Good general business climate

Regarding the entire European biotech sec-tor, the respondents felt positive. Over 90% appraised the overall business climate as good (58%) or satisfactory (34%). Howev-er, when it comes to future expectations, most � rms remain cautious. Only 24% be-lieved that the business climate will con-tinue to improve, while a majority of 63% thought it would remain as it is at the mo-ment. In another positive twist, many of the companies (40%) expect to hire more staff in the next 12 months. Over half speci-� ed that they are not planning any chang-es in staff numbers, and less than one in ten were reducing the employees on the payroll. According to the survey, 86% of the responding � rms employ less than 50 staff. 10% had 50-250 employees, and just 4% provided more than 250 jobs.

“The EuroBiotechMonitor survey gives us a tool to improve our unterstanding of the trends and challenges biotech companies are facing in Europe,” says study director Dr. Sebastian Delbrück. According to the survey1, which included all dedicated EU bio tech companies that met OECD crite-ria, more than two out of three (68%) re-spondents judged their enterprise’s cur-rent business situation as either “satisfac-tory” or “good”. Half of the mostly small and medium-sized companies said that their total revenues in 2011 outperformed those of the previous year, while 34% stat-ed that they had reached the same levels as

in 2010. In light of the demands for more money from national and European bio-tech interest groups, an unexpected result was the positive business outlook for next year. Just 7% of company management � gures believed that their business will decline in 2013, while 93% expected per-formance to remain constant or improve (Fig. 1A). Half of the responding � rms expected to make more money in 2012, while 35% anticipate that business will re-main stable (Fig. 1B). However, the most-ly small companies also said that support from the European Commission needed to be extended.

Fig. 1: Business outlook (A) and sales expectations (B) of EU biotech companies for 2012.

(A) Our business outlook for next year is … (B) Our revenues in 2012 are expected to be …

good satisfactory weak © E

urop

ean

Bio

tech

nolo

gy F

ound

atio

n

1 All data taken from 1st EuroBiotechMonitor, conducted from 11 May–13 July 2012.

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Euro|Biotech|NewsN º– 9 | Volume 11 | 2012 47

CompaNy aNd adVertiser iNdex

aAbbott Laboratories (USA) ………………… 16,44

Actavis (CH) …………………………………… 30

Actinium Pharmaceuticals Inc. (USA) ………… 28

ADC Therapeutics Sarl (CH) …………………… 46

Affitech (DK) …………………………………… 22

Allergan Inc. (USA) …………………………… 24

Amsterdam Molecular Therapeutics (NL) ……… 8

Amylin Pharmaceuticals Inc. (USA) …………… 44

Antibioticos S.A. (E) …………………………… 28

AOP Orphan Pharmaceuticals AG (A) ………… 16

Apeptico GmbH (A) …………………………… 25

Archer Daniels Midland Co. (ADM) (USA) …… 28

Ariana Pharma (F/USA) ………………………… 46

ASEBIO (E) ……………………………………34, 35

AstraZeneca (UK/S) …………………………… 44

BBASF AG (GER) ………………………………22, 46

Bayer Healthcare AG (GER) …………………… 30

Bayern Innovativ GmbH (GER) ……32BB Biotech

(CH) …………………………………………… 19

BIOCOM AG …………………………………… 25

BIO.NRW (GER) ………………………………… CP3

Bioquell Ltd. (UK) ……………………………… 23

BIOQUELL UK Limited ………………………… 48

BioSystems International (F) ………………… 46

Bioton S.A. (PL) ………………………………… 30

BioTOP Berlin-Brandenburg (GER) …………… 17

BioWin - The Health Cluster of Wallonia (B) 27, 49

Boehringer Ingelheim GmbH (GER) ………… 22

Bristol-Myers Squibb (USA) …………………… 44

CCelgene Europe Ltd. (USA) …………………… 16

Celltrion (KR) …………………………………… 16

Centocor BV (NL) ……………………………… 17

Chitinor (N) …………………………………… 22

co.don AG (GER) ……………………………… 22

Crucell N.V. (NL) ……………………………… 46

Cryo Innovation Ltd. (HUN) …………………… 30

Cryo Management Ltd. (HUN) ………………… 30

CSR plc. (UK) …………………………………… 18

dDBV Technologies (F) ………………………… 17

deCode genetics (IS) ………………………… 22

Deloitte & Touche GmbH (GER) ……………… 18

Diamyd Medical AB (S) ………………………… 22

Dievini Hopp Biotech Holding GmbH (GER) … 20

Donau-Universität Krems (A) ………………… 25

eEBD Group (GER) ……………………………44, 45

European Biotechnology Network …………… 13

EBEWE Arzneimittel GmbH …………………… 18

Edmond de Rothschild Investment Partners (F) 18

Elan Corp. (IRL) ………………………………… 21

Endocyte Inc. (USA) …………………………… 44

Endosense SA (CH) …………………………… 18

Evotec AG (GER) ………………………………… 8

F/GFIT Biotech Oyi Plc (FI) ………………………… 22

Galapagos NV (B) ……………………………44, 50

GE Healthcare (GER) …………………………… 48

Genmab A/S (DK) …………………………… 18, 20

Genomed Management SA (PL) ……………… 30

Genzyme (USA) ……………………………… 6, 26

GlaxoSmithKline (UK) ……………………… 17, 22

GPC Biotech AG (GER) ………………………… 25

Grünecker Patent- und Rechtsanwälte (GER) … 14

HHorizon Discovery Ltd. (UK) …………………… 18

Hospira (USA) ………………………………… 16

Hospira One 2 One Global Pharmaceutical (USA) 7

Hovione FarmaCiencia Sa (PT) ……………… 28

JJanssen AI (IRL) ………………………………… 21

Janssen Biologics LLC (IRL) …………………… 17

Janssen Biotech Inc. (USA) …………………… 20

Janssen-Cilag International NV (B) …………… 16

JenaValve Technology GmbH (GER) ………… 18

Johnson & Johnson (USA) …………………… 20

LLehman Brothers Inc. (USA) …………………… 44

LISA Vienna Region, Clustermanagement (A) 29

Luminex (I) ……………………………………38, 39

mMerck & Co. (USA) ……………………………… 44

Merck KGaA (GER) ………………………… 21, 50

Metabolix Inc. (USA) …………………………… 28

Microsynth AG (CH) …………………………… 19

Molecular Partners AG (CH) ………………… 8, 24

MPM Capital GmbH (GER) …………………… 25

NNeotope Bioscience (IRL) ……………………… 21

Newron Pharmaceuticals S.p.A. (I) …………… 28

Novartis AG ……………………………………… 6

Novartis Europharm Ltd. (UK) ………………… 16

Novexel S.A. (F) ………………………………… 18

Noxxon Pharma AG (GER) …………………… 18

oOno Pharma UK Ltd. …………………………… 50

pPfizer Inc. (USA) ………………… 6, 16, 18, 21, 22

PharmaMar (Grupo Zeltia) (E) ………………… 11

Pharming Group N.V. (NL) …………………… 18

Philogen S.p.A. (I) ……………………………… 28

Photocure ASA (N) …………………………… 18

Prexton Therapeutics SA (CH) ………………… 21

ProBioDrug AG (GER) ………………………… 18

Prometheus Labs (USA) ……………………… 37

PromoCell GmbH (GER) ……………………… 48

QQuartz Bio (CH) ………………………………… 21

Quintiles Limited (UK) ………………………… 16

rRauCon Business Development (GER) 33, 40, 41

Reed Expositions France (F) …………………42, 43

ReNeuron Group plc (UK) …………………… 17

Roche Diagnostics GmbH (GER) ……… CP 2, CP 4

sSALANS LLP (GER) …………………………… 15

Sanofi SA (F) ………………………… 3, 6, 22, 26

SAP AG (GER) ………………………………… 20

SciGen Ltd. (SG) ……………………………… 30

Seagarden ASA (N) …………………………… 22

Serono SA (CH) ………………………………… 21

SuperSonic Imagin (F) ………………………… 18

tTakeda Pharmaceuticals (JP) ………………… 16

Technology Vision Group (BPFE 2012)(USA) 36, 37

ThromboGenics NV (NL) ……………………… 17

Thomson Reuters (USA) …………………… 13, 50

Trans Nova Investments Ltd. (CY) …………… 22

UuniQure BV (NL) ………………………… 6, 8, 16

VViiV Healthcare (UK) …………………………… 22

Vitrolife AB (S) ………………………………… 30

WWatson Pharmaceuticals (USA) ……………… 30

Wilex AG (GER) ……………………………… 18, 20

xXcellerex Inc. (USA) ……………………………… 9

Xvivo Perfusion AB (S) ………………………… 30

ZZoran Corp. (USA) ……………………………… 18

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