Prosensa NV and researchers from Leiden University Medical Center (LUMC) under Prosensa's scientific advisor Prof. Gertjan van Ommen have published results from a Phase I clinical study with the 2-’O-methyl antisense compound PRO051 in four children (aged 10-13) with Duchenne Muscular Dystrophy (DMD) (N Engl J Med. 357(26):2677-86). Intramuscular injection of a single 80 mg shot of the compound, which is able to correct the open reading frame of the DMD gene by exon skipping and thus to restore dystrophin expression, was not associated with clinical adverse reactions, according to Prosensa. Additionally, PRO051 restored dystrophin expression in the treated muscle fibers of all four patients, who had been selected on the basis of their mutational status, muscle condition, and positive exon-skipping response to PRO051 in vitro. The compound will be suitable for 13% of all DMD patients (DMD incidence
1: 3,500), because it can treat a cluster of DMD mutations.
After stock markets’ worldwide strong start into 2007, last week of February saw the beginning of a correction phase. By 15 February, the WestLB EU Biotech index went up by 4.2%, again outperforming both the Nasdaq Biotech index...
After stock markets’ worldwide strong start into 2007, last week of February saw the beginning of a correction phase. By 15 February, the WestLB EU Biotech index went up by 4.2%, again outperforming both the Nasdaq Biotech index (+3.5%) and the AMEX stock exchange Biotech index (+3.4%). By 2 March, a few days after the correction began, the WLB Biotech Index is still ahead (-2.2%) versus both US indices (-4.6% and -4.2%). Interestingly, the Dow Jones STOXX Healthcare Index has performed better than all three Biotech indices. This potentially points to a more defensive orientation from investors. In our last comment we highlighted UCB as a core investment in our European MidCap Healthcare universe. Although UCB underperformed the WLB Biotech Index, with the uncertainty surrounding Cimzia’s fate in Crohn’s Disease playing a key role in our view, we remain positive for the reminder of 2007 given the anticipated incremental newsflow. The outcome of the FDA meeting on Cimzia in Crohn’s Disease is scheduled for March. We currently anticipate a US approval in the third quarter of 2007. Furthermore, the company reported positive data from the second Phase III trial for rotigotine in Restless Legs Syndrome (RLS). We estimate the product to be filed in the second half of 2007. Regarding Cimzia in Rheumatoid Arthritis (RA), we also anticipate filing for the second half of the year. Therefore, we still regard UCB as a core investment in our universe. Actelion, which is another important stock among our universe, has already experienced high volatility during the first months of 2007. Whereas shares moved up by 14% by 15 February, fuelled by pipeline newsflow, it has lost 14% since then. The company’s guidance for 2007 points to a slowdown in Tracleer growth. Furthermore, we estimate that there will be no decisive newsflow on the company’s most important pipeline projects in the course of 2007, with the shares therefore missing important newsflow triggers.
Investors becoming more
However, based on anticipated continued newsflow for many of the constituents of the WestLB EU Biotech index and further M&A speculation, we remain positive for its development during 2007. MorphoSys is guided for a strong increase in EBIT for 2007 and also remains in the limelight as a takeover target. For GPC Biotech we estimate plenty of clinical newsflow on satraplatin in the course of 2007 and the drug’s US approval in the second half of the year. While we think that the WestLB EU Biotech index will continue its out performance versus both of its US peers, the situation might look different versus the Dow Jones STOXX Healthcare index if investors become more defensively oriented.
Strasbourg – The European Science Foundation (ESF) launched a new call in March for its funding program EuroBioFund (www.esf.org/eurobiofund/eoi), set up in 2006 to identify future grand challenges in life science research,...
Strasbourg – The European Science Foundation (ESF) launched a new call in March for its funding program EuroBioFund (www.esf.org/eurobiofund/eoi), set up in 2006 to identify future grand challenges in life science research, which require a coordinated European approach for their financing and coordination. Scientists are invited to submit their ideas for large-scale research programmes by 28 May 2007. Six expressions of interest will be selected for discussion with funding organisations at the second annual EuroBioForum, scheduled to take place on December 5-7, 2007 in Lisbon, Portugal. By bringing together key stakeholders, EuroBioForum aims to set the scene for the future financing and implementation of new research programmes with annual funds of a5-10 million.
Brussels – The number of cancer cases in Europe is rising. According to a new study by the International Cancer Center (IACR), a WHO division based in Lyon (France), 3.2 million new cases were diagnosed in 2006, 300,000 more...
Brussels – The number of cancer cases in Europe is rising. According to a new study by the International Cancer Center (IACR), a WHO division based in Lyon (France), 3.2 million new cases were diagnosed in 2006, 300,000 more than in the previous survey two years before. Roughly 1.7 million people in Europe died from cancers, with lung cancer remaining the top killer (334,800 deaths) followed by colorectal cancer (207,400) and breast cancer (118.200). By 2015, the number of cancer deaths will rise by additional 10%, according to prognoses from the IARC. IACR head Peter Boyle said the numbers “are alarming”, because the number of cancer cases rose even though prevention and therapy options had been improved. He called for additional screening programs. Three reasons are responsible for the negative trend, according to the IARC: the ageing of the European population, smoking as well as refined cancer diagnoses, which identify cancers sooner than was previously possible.
“The IMI does not want to develop new medicines or new vaccines”, said EC representative Hoeveler during an IMI information meeting in Germany, which was organized in February by members from the Baltic See network ScanBalt. The...
“The IMI does not want to develop new medicines or new vaccines”, said EC representative Hoeveler during an IMI information meeting in Germany, which was organized in February by members from the Baltic See network ScanBalt. The overall goal of the initiative is to join the forces of all relevant stakeholders in the drug development process and to boost methodically standards, mainly in the field of pharmacology and toxicology. “This is still a pre-competitive area in which companies are interested in general standards and in investing significantly in academic research”, stressed Hoeveler, and this is also a reason for the European Commision to engage. “If we give money to such a public-private-partnership it must be clear, that our money only goes to academia or to small and medium-sized enterprises”, said Hoeveler outlining the Commission’s perspective. Nevertheless, the implementation of the IMI in the European bureaucracy is taking its time. “It was as early as 2004, when we first had the idea which was under the roof of the EFPIA”, remembers Børge Diderichsen, Novo Nordisk’s Vice President Corporate Research. At this moment, although the realisation seems likely to happen this year, there is still great impatience on the industrial side. “We know that”, Hoeveler agrees, “but much time has been spent on establishing a reliable system through which the European Commission can act with the industry.”
“The implementation of IMI as JTI seems realistic this year”
From January on, within FP7 such a system will be offered with the implementation of so-called ‘Joint Technology Initiatives’ which enable the European Commission, on the basis of Article 171 of the EC treaty, to combine private sector investment with national and European public funding, including funds from the EU‘s research framework programme, and to build up joint infrastructure. At present, around 30 public-private-initiatives in diffrent fields of action are in the process of applying for JTI status, and the IMI seems to be one of those that may finally be established this year. “A Strategic Research Agenda has been prepared by the EFPIA and the infrastructure and research funding system is clear, but there still remain some open questions, especially regarding the handling of IP matters”, said Hoeveler. Nevertheless, the timelime seems to be set: According to Hoeveler the European Commission is filing an official JTI application with the European Council in April, which is expected to be discussed two months later in the parliament. “The IMI Executive office could then begin work in Brussels in November”, Hoeveler said. As regards the budget he remains reserved: “Originally, the plans were around a3 billion, but a2 billion is a more realistic figure, as half of which will be provided by the Commission.”
Novo Nordisk A/S (Bagsværd, Denmark) will not seek regulatory approval for its human recombinant activated coagulation factor VII NovoSeven (Eptacog alfa) as a treatment for intra-cerebral haemorrhage, the most severe...
Novo Nordisk A/S (Bagsværd, Denmark) will not seek regulatory approval for its human recombinant activated coagulation factor VII NovoSeven (Eptacog alfa) as a treatment for intra-cerebral haemorrhage, the most severe complication of stroke. The drug, which interacts directly with factor X, missed the primary endpoint in a Phase III study on 821 patients. After 15 days of treatment, the company observed a significant reduction of intra-cranial bleeding and improvement of neurological impairment. However, after 90 days there was no significant reduction in mortality and severe disability. Thus, Novoseven remains limited to a narrow market niche. Since 1996, it has been approved for treatment of bleeding episodes and prevention of bleeding in patients with congenital haemophilia that have developed alloantibodies against factor VIII and IX, as well as treatment for congenital factor VII deficiency.
A Novartis Ophtalmics (Basel, Switzerland), the Novartis daughter, which is to distribute Genetech’s VEGF inhibitor Lucentis (ranibizumab) in Europe, has been given the green light from the European Commission. The monoclonal antibody fragment, which inhibits blood vessel spread in a retinal sublayer, the macula, is a breakthrough treatment for the wet form of age-related macula degeneration (AMD) – the most common age-dependent blindness. The drug, which is injected straight into the eye, has almost out-competed current treatments such as photodynamic therapy and an aptamer drug, by entering the US market six months ago with Q4/2006 revenues of roughly a quarter billion US$. However, the market success, which is based on a tremendous improvement of vision in AMD patients, is now shadowed by new study results indicating that patients that received the drug are at a higher risk of stroke. Thus, it is to be seen if ophthalmologists will switch treatment from the off-label treatment Avastin, a full length anti-VEGF mAb, which is said to show the same efficacy as Lucentis but at a price, 200 times lower than that of Lucentis.
A Roche AG’s (Basel, Switzerland) colorectal cancer blockbuster Avastin (bevacizumab) is expanding to additional indications. In February, the VEGF inhibitor was given a recommendation by the European Medicines Agency as a first line treatment for locally recurrent and metastatic breast cancer in combination with paclitaxel chemotherapy. Previous Phase III results indicated a doubling of the progression-free time of survival, from 6.7 months to 13.3 months. In another Phase III trial the antibody drug plus gemcitabine/cisplatin reached its primary endpoint of improving time of survival in patients with non-small cell lung cancer (NSMLC) at doses of 7.5 mg/kg and 15 mg/kg. In contrast to another trial, in which the angiogenesis inhibitor plus paclitaxel/carboplatin prolonged survival from 10.3 months to 12.3 months, Roche did not quantify the level of improvement.
A SR Pharma plc’s (London, UK) license partner Quark Biotech Inc. has initiated Phase I clinical testing with RTP-801i, a siRNA drug candidate against age-related macula degeneration (AMD), developed by SR Pharma’s subdivision Atugen. Last year, Alnylam Pharmaceuticals cancelled development of a siRNSA AMD treatment following Genentech’s successful market launch of Lucentis in AMD.
Three countries have pooled their expertise in the field of plant genomics in a transnational funding initiative that is unique within the European Union. The cooperation between the plant genome research programmes in Germany...
Frank Laplace, Ministry of Education and Research, Berlin
Rosa Rodriguez-Bernabe, Ministry of Education and Science, Madrid
Daniel Richard-Molard, Ministry of Technology and Research, Paris
The industries in each of the three countries have organised themselves into industry associations and platforms, which support the respective national plant genome research programmes and simultaneously develop industry-driven transnational research strategies at pre-competitive stages in the field of the knowledge-based bio-economy. In this fashion, they are able to continually strengthen both the individual, national and European competitive positions in this area, as well as playing a pivotal role in the preparation of joint calls from the three research ministries. These common transnational structures also provide the necessary backing for technology transfer. The strategic goals of the transnational cooperation reflect the respective national research programmes, allowing national research funding and transnational cooperation to reinforce each other. At the same time, this German, French and Spanish trilateral cooperation has established the basis for the founding of the ‘ERA-Net Plant Genomics’ – these countries currently form the application-oriented pillar of this particular ERA-Net. Other European countries are invited to take part in the further development of specific research and development goals, which has the aim of progressively strengthening European cooperation between business and industry.
Beginning in the Nineties
In the mid 1990s, it became clear that substantial progress in the breeding of optimized plants for use in agriculture and industry couldn’t be achieved without a comprehensive analysis of the functions of plant genes. Throughout the world, considerable amounts of government and private money were invested in relevant research. Such efforts were also made in some European countries, including France and Germany. Plant genome research programmes were launched in both countries in 1998. In 1999, the German and French Ministries of Research agreed to cooperate in their respective national plant genome research programmes, GABI and Génoplante. This was intended to pool the resources in the European Research Area and achieve a critical mass of know-how and research capacity, in the hope of being successful in the face of fierce international competition. Both research programmes are characterised by their corresponding target objectives and basic structures, a result of the close relationship between science, industry and research policies (public private partnership). The cooperation agreed between the two ministries had two objective targets: On the one hand, research capacities from both countries aimed at the functional analysis of genomes of important crop plants would be bundled and important resources for research and service infrastructures in both countries were to be made mutually available. On the other hand, cooperation between the steering committees of both programmes would be initiated and the transfer of research results into an application at the companies involved in both countries would be brought about through an efficient and demand-oriented technology transfer. To begin to tackle these major tasks, experts in the field put their heads together over a series of meetings, which began with a Franco-German workshop in Bonn in May 2000, where the general thematic frame of the planned cooperation was defined. In October 2000, experts from both sides met to discuss the issues of proprietary protection of research results and the rights of research facilities and companies involved in the research and development projects. Finally, a workshop was held in February 2001 in Montpellier, where definite research projects and the rules for their evaluation by a joint Franco-German panel of experts were established.
Due to the difficult legal issues that would have arisen on both sides through the cooperation of companies involved in bilateral projects, only four projects from fundamental research, with a three-year duration and financing totalling 4 million euros, were initially brought to funding in 2001. These projects aimed at achieving cooperation in Arabidopsis research and opening-up, as well as utilizing resources in both countries. As a pilot project for the cooperation between the companies involved in GABI and Génoplante, a large joint project – also funded within the framework of EUREKA – was applied for in parallel. This was financed with 13 million euros of public money. The focus of this project was the development and application of biomolecular tools for the improvement of silage quality in maize and quality wheat. At the same time, structures were established which were appropriate to guarantee the proper steering of the cooperation between GABI and Génoplante. The joint circle of experts and the immediate contacts between the managing offices of both programmes, as well as the establishment of a permanent working group to solve the legal issues within the cooperative undertaking, were of crucial importance.
A new stimulus from politics
At the Franco-German Science Forum on 12 February 2002, this development stage was deemed a good time to announce the two countries’ cooperation in the field of plant genome research – a first in the life sciences. This impulse accelerated both participants’ efforts to prepare additional projects with the involvement of businesses and to thus strengthen the intended application-oriented character of the cooperation. A prerequisite for this was to overcome the difficulties resulting from the quite different legal administrations of the two programmes and – deriving from these legal aspects – the various rights to be granted to the participating companies as regards the research goals accomplished. The permanent working group that was set up to deal with the legal issues has now achieved this prerequisite. The group set up super-ordinate rules for the utilisation of joint resources and for the handling of proprietary rights resulting from the work. The group also developed an agreement for the transfer of material as well as a secrecy agreement and stipulated regulations for a case-by-case process to resolve any project-specific problems. In this way, another five Franco-German joint projects were brought to funding in 2004. Four of these joint-ventures were carried out with the participation of French and German businesses. The funding in this case again amounted to 4 million euros. Industry supplemented approximately another 1.5 million euros from their own resources. The goals of these projects were scientific issues of interest to the participating plant breeding businesses.
Spain – the third partner
The dynamic development of the Spanish plant genome research programmes during these years provided the stimulus for an investigation into possible synergies and areas for cooperation between Germany, France and Spain in this research area. In May 2003, a workshop organised by the Spanish Ministry of Education and Science in Madrid, with over 250 participants, came to the conclusion that there are a multitude of shared standpoints between the goals and contents of the three countries’ programmes. Among others, this applied in particular to the research areas of improved biotic and abiotic stress tolerance, the improvement of physiological parameters of crop organs, and research on questions relevant to nutrition as well as on sustainable agricultural production. Under the title ‘Functional and comparative genomics approaches for the investigation and use of the natural diversity’, the participants at the workshop developed a total of 44 project concepts. The end result of the international assessment procedure was that nine joint projects were brought to funding by the participating research ministries in 2004. The funding sum totalled roughly 7.5 million euros. Experiences and procedures from the Franco-German cooperation formed the basis for the entire process of finding projects, evaluation and funding. To accompany the project implementation and to resolve issues quickly, a new steering committee was established, the so-called ‘6+6+6 Advisory Group’. Making up the committee, which meets regularly, are scientists from academia and businesses from the three countries, representatives from the three managing offices of the national programmes and representatives from the permanent working group dealing with the legal aspects of the cooperation. As with the French and German programmes, a further aim of the Spanish programme was to better position itself in the area of application-oriented research in order to strengthen the competitiveness of the industry. A first and crucial step in rising to this challenge was the formal constitution of INVEGEN, a Spanish association of private companies with interests in diverse sectors (agrofood, forestry, bio-energy, etc.), by the end of 2005.
To put the Franco-German-Spanish cooperation in plant genome research on a sustainable foundation, representatives from business, science and the various research ministries met in Weimar in March 2005 to discuss future subjects and strategy-finding processes. As a result, the representatives of the ministries signed a memorandum of understanding about the scientific cooperation. Within this memorandum, the trilateral cooperation was recognised as the core activity in application-oriented plant genome research in Europe, milestones for a further cooperation were defined and a strategy-finding process was initiated. The integration of the trilateral cooperation into the ERA-Net on plant genome research, which has been in place since 2004 and in which the three countries are also involved, was of special significance here. A milestone in the development of the trilateral cooperation was the decision by the industry platforms involved in the three respective national programmes to develop a strategy paper to define the overriding goal of the co-operation. It was also intended to describe the future development of the cooperation with regards to the application of research results in industrial practice, the strengthening of the competitiveness of the businesses involved and the reinforcement of the cooperation between science and economy. The strategy paper, titled ‘Genomic approaches for the investigation of genetic diversity in crop plants and its use for innovation’, was discussed and adopted at a trilateral meeting in October 2005 in Cordoba. Since then, it has provided the essential basis for other future joint activities between the three countries. The paper emphasises the contribution of plant genome research to the solving of central issues such as climate change, limitation of fossil resources, development of a sustainable agriculture and regional adaptation of crop plants. Simultaneously, it defines important fields of research and activities, which are of equal interest to the economy in the three countries involved. They are the following subjects in particular: improved quality in food and feed, and sustainable agriculture with improved productivity and yield stability. At the same time, the importance of plant research for the development of a knowledge-based bio-industry in Europe is emphasised.
The driving force behind the ERA-Net Plant Genomics
In 2004, eleven different European countries established the ERA-Net Plant Genomics, which serves to coordinate research and funding policies in participating countries in the field of plant genome research. Spain, France and Germany are among those involved in this ERA-Net. In 2006, the first funding call within the framework of this ERA-Net was announced. Spain, France and Germany participated with their very own call. The call, titled ‘From genome analysis to product innovation’, is based on a strategy paper from 2005 written by the industry platforms and is aimed at application-oriented subjects: improvement of food and feed quality, improvement of productivity and yield stability, development of plants for innovative applications such as energy providers or green factories, improvement and stabilisation of surrounding natural landscapes. The project management is to be executed preferably by the individual businesses. After an international assessment, a total of fourteen project proposals were recommended for funding. Alongside businesses and scientists from the three countries, individual partners from Portugal, the United Kingdom, Italy, Switzerland and the Netherlands also took part in a variety of projects. The total funding sum is estimated to be approximately 15 million euros. This will be supplemented by substantial funds from industry, amounting to roughly 5 million euros. These 20 million euros, which will go to specially designed cooperation projects, are made available under the potent national funding programmes of the participating countries. As regards the national strategic development of plant research, these funding programmes, which focus on the establishment of the knowledge-based bio-economy, are moving closer together and formulate common goals (see above). Transnational cooperation substantially aids this process. France, Spain and Germany are together mobilizing more than 70 million euros annually for plant genome research alone under their national programmes. This call resulted in the opening-up of this trilateral cooperation for further countries interested in application-oriented research within the framework of a public-private-partnership. The goal of these projects is to prepare the ground for the development of a knowledge-based bio-industry in Europe. The central concern is to secure the competitiveness of agriculture and industry, and thus jobs. In such a way, the three countries hope to continue to create visible momentum across Europe.D
Contact Prof. Dr. Frank Laplace Federal Ministry of Education and Research D-11055 Berlin Tel.: +49-1888-575111 eMail: firstname.lastname@example.org
Affimed Therapeutics AG (Heidelberg, Germany) announced that preclinical proof of concept has been established for Anti GPIIb/IIa, its anti-thrombosis product candidate as a treatment for cardiovascular disease in conjunction...
Affimed Therapeutics AG (Heidelberg, Germany) announced that preclinical proof of concept has been established for Anti GPIIb/IIa, its anti-thrombosis product candidate as a treatment for cardiovascular disease in conjunction with percutaneous coronary intervention (Circ. Res. 99:25-33). The fully human antibody, which can be produced as scFV or fab fragment in microorganisms, preferentially binds to the activated form of the GPIIb/IIIa receptor, according to Affimed. The GPIIb/IIIa receptor is the target of the marketed chimeric Fab2-fragment competitor drug Reopro (Abciximab, Centocor/Eli Lilly) and oral GPIIb/IIIa blockers such as tirofiban (Merck & Co.), a tyrosine derivative, which like all GPIIb/IIIa inhibitors blocks thrombocyte cross-linking and platelet aggregation. Preclinical studies have now demonstrated that the antithrombotic effect of Anti GPIIb/IIa is comparable to that of the non peptidic inhibitor tirofiban and the cyclic heptapeptide eptifibatide (GlaxoSmithKline). Affimed underlined that its antibody did not prolong bleeding times, which occur as a rare but severe side effect with the oral and injected competitor drugs.
Crucell N.V. (Leiden, The Netherlands) has announced two Phase I trials. One is a dose-escalation study with a heteromeric human monoclonal antibody (mAb) for the post-exposure prophylaxis (PEP) of rabies, which will involve 60...
Crucell N.V. (Leiden, The Netherlands) has announced two Phase I trials. One is a dose-escalation study with a heteromeric human monoclonal antibody (mAb) for the post-exposure prophylaxis (PEP) of rabies, which will involve 60 healthy volunteers. In the second trial, the company will test its AdVac®-based malaria vaccine, which is currently developed in collaboration with the US-National Institute of Allergy and Infectious Diseases (NIAID).
Serono S.A. (Geneva, Switzerland) and its US partner ZymoGenetics Inc. (Seattle) have started a proof-of-concept Phase II study with its protein drug atacicept (TACI-Ig) in 320 patients with rheumatoid arthritis (RA). The...
Serono S.A. (Geneva, Switzerland) and its US partner ZymoGenetics Inc. (Seattle) have started a proof-of-concept Phase II study with its protein drug atacicept (TACI-Ig) in 320 patients with rheumatoid arthritis (RA). The soluble TACI receptor is believed to reduce the production of autoantibodies by moping up two cytokines termed BLyS and APRIL, which control B lymphocyte maturation in mice, thus lowering humoral response in RA. In the study the companies will investigate the efficacy of different doses (25 mg, 50 mg or 150 mg) of atacicept plus methotrexate therapy in patients with an inadequate response to prior treatment with TNF inhibitors, which block the inflammation cascade. In contrast to the most approved biologicals, which target the T cell response in RA (such as Humira (Abbott), Enbrel (Wyeth), Remicade (Centocor), or Orencia (BristolMyerSquibbs) atacicept targets the B-cell response in autoimmune diseases and B-cell malignancies.
New results of a pharmacodynamic study with Nicox S.A.’s (Nice, France) osteoarthritis phase III drug candidate Naproxcinod have failed to demonstrate that its side effect profile is advantageous compared to the standard therapy...
New results of a pharmacodynamic study with Nicox S.A.’s (Nice, France) osteoarthritis phase III drug candidate Naproxcinod have failed to demonstrate that its side effect profile is advantageous compared to the standard therapy with non-steroidal anti-inflammatory drugs (NSAIDs). Despite the established efficacy of NSAIDs, the most common treatment of arthritis symptoms, NSAIDs raise blood pressure and interfere with the treatment of hypertension. Naproxcinod did not reach statistical significance for lowering systolic blood pressure, the primary endpoint of the trial (p=0.101). However, statistical significance was achieved for dropping diastolic blood pressure, the secondary endpoint (p=0.007).
7th Berlin Conference on IP in Life Sciences: Big Data, Big Drugs
The health care industry faces significant transformation, driven by a boom in knowledge within biomedical sciences and breakthrough technologies such as gene sequencing. The management of "big data“ will change the understanding of diseases, development of drugs and treatment of patients. more