May 28-29, 2009, Biberach an der Riss, Germany
and is hosted by ACTIP member company Boehringer Ingelheim.
The meeting will focus on critical issues in the relationships between CMOs and their clients, and on stem cell technology. For ACTIP member companies and invited observers only.
Reshaping R&D
News from the Commission
Biomanufacturing News
Stem cell news
Mergers and Acquisitions
Research and Development
Business News
Regulatory News
Agenda
Cost cutting has sustained pharma profits in the faltering global economy, however top executives are starting to realise that reducing R&D capacity may not have been the smartest move in an era of weak pipelines and impending patent expiry, according to a new report by Ernst & Young.
While drugmakers have continued to invest in R&D in the last few years, recent PhRMA figures indicate for example that US spending grew 20 per cent between 2004 and 2007, the focus has been on acquisitions and on making greater use of contractors to develop new products.
In contrast, internal R&D departments at big pharma firms have been subject to the same cost cutting initiatives and restructuring measures being employed in manufacturing and sales operations as the industry prepares for greater generic competition.
Although this strategy has proved effective in the short term, the growing concern among CEOs and observers alike is that the race to improve margins has weakened big pharma’s ability to innovate, which used to be the sector’s great strength.
For its latest study, E&Y quizzed executives at 15 of the world’s top pharmaceutical firms including Pfizer, GlaxoSmithKline and Johnson & Johnson and discovered that 72 per cent ranked the “thinness” of drug pipelines as their major concern.
This indicates a marked change in thinking compared with E&Y’s 2007 report which revealed that 92 per cent of the same group viewed cost reduction as their primary focus.
E&Y’s finding is in keeping with recent moves by firms like Wyeth, Pfizer, Eli Lilly and Bristol-Myers Squibb (B-MS) to maintain R&D spending but narrow their focus to therapeutic areas where demand is likely to grow, particularly in lifestyle-related illnesses expected to become prevalent in emerging markets.
Source: www.In-pharmatechnologist.com, November 13, 2009
Commission calls for a European focus on rare diseases
The European Commission adopted today a Communication and a proposal for a Council Recommendation on rare diseases setting out an overall Community strategy to support Member States in diagnosing, treating and caring for the 36 million EU citizens with rare diseases.
The Communication sets out a Community strategy for action in three main areas:
- improving recognition and visibility of rare diseases;
- supporting national plans for rare diseases in the Member States; and,
- Strengthening cooperation and coordination for rare diseases at European level.
European cooperation will help to bring together the scarce resources for rare diseases that are currently fragmented across individual countries in the EU. European action will help patients and professionals to collaborate across Member States in order to share and coordinate expertise and information. This will be achieved through for example, networks linking centres of expertise in different countries, and by making use of new information and communication technologies (“E-Health”). The Commission will build on successful existing actions, such as the previous health programme on rare diseases, the Research and Technological Development Framework Programmes, and the specific regulatory framework already in place to provide additional incentives for the development of ’orphan’ drugs for these conditions.
More reading: European Commission Website on Rare Diseases, with link to the Communication:
http://ec.europa.eu/health/ph_threats/non_com/rare_diseases_en.htm
Source: EC Press Release, November 11, 2008
Commission launches ’pharmaceutical package’
In December the European Commission launched its “pharmaceutical package”, which incorporates three separate initiatives under one umbrella, called the Communication on Safe, Innovative and Accessible Medicines
The Commission emphasizes in the Communication restoring the EU’s role as the natural home for pharmaceutical innovation and avoiding needless delays for patient access to innovative treatments. The pharmaceutical package also incorporates three separate legislative proposals which cover information to patients, pharmacovigilance and protection of patients from counterfeit medicines.
Today biotech medicines are estimated to account for approximately 20% of all marketed medicines and represent 50% of all medicines in the pipeline. “We are pleased to see the emphasis the Commission places on innovative medicines.” said Willy De Greef, EuropaBio’s Secretary General.
Source: EuropaBio Press Release, December 15, 2008
Research ministers agree to strengthen European Research Area (ERA)
On December 1-2 the EU Council agreed on Vision 2020 and updated the European Research Area (ERA) (1). Vision 2020 is a major step towards coordinating Europe’s national research programmes and sets out a political strategy to integrate Europe’s research efforts, the European Research Area. As such, Vision 2020 represents an important step forward in the development of a competitive knowledge-based economy, a key element of the Lisbon strategy. Innovative research is vital for the biotech industry which relies on Europe’s best and brightest scientists to develop ground breaking biotech products that will heal patients, clean up the environment and feed the world. The life sciences industry faces unique challenges to translate excellence in research into economic and societal value, because of its long research and development time lines. It can take 20 years or more for a product to emerge from the initial research breakthrough. An example is the work of one of this year’s Nobel Prize winners, Prof. zur Hausen, whose breakthrough research of the 1970s on HPVs led to the development of a cancer vaccine which was only recently put on the market.
Source: EuropaBio Press Release, December 11, 2008
More efficient protein manufacture in bacteria
US researchers have found a way to improve the yield of biopharmaceutical production by freeing up protein trapped in bacterial stores known as inclusion bodies.
Inclusion bodies are insoluble aggregates that are formed by bacteria to store excess recombinant protein produced during expression. These are hard to separate during bioprocessing and can mean that up to 95 per cent of the protein expressed by the bacterial culture is unusable.
Now, a team of scientists led by chemist David Weliky of Michigan State University have identified a way of delving into the secondary structure of these inclusion bodies using nuclear magnetic resonance (NMR) spectroscopy.
Typically, the protein is separated from host cell contaminants such as endotoxin by lysing the bacterial biomass, and separating the inclusion body using centrifugation or other approaches such as tangential flow filtration or microfiltration.
By using NMR in combination with labelling of protein with radioactive isotopes, the researchers were able to establish that — at least in Escherichia coli cells making a recombinant influenza virus known as FHA2 — much of the protein within the inclusion bodies is correctly folded.
That means that it may be possible to extract the protein from the inclusion bodies without separating and refolding, according to Weliky. The work is published in the Journal of the American Chemical Society.
J. Am. Chem. Soc., 2008, 130 (38), pp 12568–12569 http://pubs.acs.org/doi/abs/10.1021/ja8039426
Source: www.In-pharmatechnologist.com, January 5, 2009
Merck Serono expands biotech site
Merck Serono has begun work on the expansion of its Swiss biotech facility, with the additional capacity being used to meet rising demand for Erbitux (cetuximab).
The $374m expansion of the manufacturing facility in Corsier-sur-Vevey, Switzerland has also been earmarked for the production of treatments for autoimmune and inflammatory diseases, which are currently in clinical development.
When completed in 2010 the facility will have two new dedicated production suites with 120,000 litres of bioreactor capacity and an additional wastewater treatment station and logistic centre. Erbitux production is due to commence in 2012 following inspections from regulators.
Since opening in 1999 the facility has produced the active pharmaceutical ingredient for Rebif (interferon beta-1a) and therapeutic proteins for clinical trials. It currently has two production suites and employs 250 people.
Both Erbitux and Rebif have been performing well, with growth rates of 13 and 10 per cent respectively for the third quarter of 2008. This rising demand, and the need for capacity to manufacture other biologics in the pipeline, has led to Merck initiating the expansion.
Source: www.In-pharmatechnologist.com. November 13, 2009
Novartis recalls vaccines amid contamination concerns
Novartis has recalled two batches of meningitis C vaccine from the UK after the solvent used in their preparation was discovered to be contaminated with Staphylococcus aureus.
During a shipping test Staphylococcus aureus was detected in the batch of aluminium hydroxide solvent that was used in the preparation of the two lots. No contamination was detected at the time of the solvents release.
Novartis is now investigating the root cause of the solvent contamination and neither it, nor the Medicines and Healthcare Regulatory Agency (MHRA), have any evidence that any other batches are affected.
The MHRA has also stated that the two vaccine batches distributed in the UK passed all import tests, including one for sterility, and that the recall is merely precautionary. It is believed that around 20,000 doses will be recalled.
Novartis meningitis C vaccine, which is sold under the name Menjugate Kit, is manufactured in Italy and has been on the market in the UK since January.
Source: www.In-pharmatechnologist.com, February 26, 2009
Obama overturns stem cell research funding ban
The US green light for federally funded embryonic stem cell research is being hailed as a victory by the US drug industry, which predicts it will open the flood gates for novel medications and help revitalise the sector.
President Obama said that: “In recent years, when it comes to stem cell research, rather than furthering discovery, our government has forced what I believe is a false choice between sound science and moral values.
As a person of faith, I believe we are called to care for each other and work to ease human suffering. I believe we have been given the capacity and will to pursue this research and the humanity and conscience to do so responsibly.”
President Obama’s decision means that some of the $10bn (€7.8bn) allocated for healthcare research in the government’s stimulus package is likely to be earmarked for work on stem cells.
While not commenting directly, a statement issued by Pharmaceutical Research and Manufacturers of America (PhRMA) senior VP Ken Johnson to coincide with President Obama’s address stressed the importance of scientific freedom for the drug industry and researchers.
In its response, the National Institutes of Health (NIH) said it will “review existing NIH and other widely-recognized guidelines on human stem cell research and issue new NIH guidance within 120 days.”
Eight year long ban
In 2001, the previous US Administration blocked government funding for stem cell research on all but 21 pre-existing stem cell lines in a move that many observers considered was a sop to the Republican Party’s core voting demographic.
While some state organisations did provide investment, most notably the California Institute for Regenerative Medicine (CIRM) with its $3bn 10-year programme, amounts were limited and wider budgetary concerns always made such funding vulnerable.
The net affect of the ban and the difficulty guaranteeing funding from other sources was to increase the amount of research on adult stem cells, which were not covered, and to cause pharmaceutical firms to deprioritise stem cell programmes.
However, the tide began days just after President Obama’s inauguration in January when the Food and Drug Administration (FDA) became the first regulatory body anywhere in the world to clear human trials of a stem cell therapy, Geron Corps’ GRNOPC1 for spinal cord injury.
Source: www.In-pharmatechnologist.com, March 11, 2009
Newly found protein complex determines stem cell pluripotency
Scientists at the Stanford University School of Medicine have identified a protein complex that determines whether embryonic stem cells retain their pluripotency or begin to mature and specialize.
Embryonic-stem-cell BAF (esBAF) seems to work by adjusting the expression levels of c-Myc, Klf4, Oct4, and Sox2, which help change adult stem cells into an embryonic-like state. They suggest that EsBAF works by affecting the way DNA is packaged within the cells in chromatin. They also discovered that esBAF can both activate and inactivate the expression of the genes and posit that perhaps this refining function is what keeps embryonic stem cells in a state between self-renewal and differentiation.
These results stemmed from an investigation into the function of BAF after researchers had found that it was important in regulating how a cell’s genetic material is wrapped around histones. BAF is one of many so-called chromatin remodeling complexes that regulate accessibility to transcription factors in mammals.
The researchers are now investigating whether they can manipulate the subunits of the esBAF complex to make fibroblasts pluripotent and attempting to determine whether small molecules or chemicals modulate the complex’ function.
Two articles describing the work appeared in the online edition of the Proceedings of the National Academy of Sciences on March 2.
Source: www.genengnews.com, March 3, 2009
Pluripotent stem cells created without virus vectors
Researchers from Mount Sinai Hospital in Toronto, Canada, and others at the MRC Centre for Regenerative Medicine report discovering a method to create pluripotent stem cells without disrupting healthy genes. The technique uses a novel wrapping procedure to deliver specific genes to reprogram cells into an embryonic-like state instead of the usual viral-delivery mechanism.
iPS cells produced with this nonviral vector showed robust expression of pluripotency markers, indicating a reprogrammed state confirmed functionally by in vitro differentiation assays and formation of adult chimaeric mice, according to the team.
The investigators showed that nonviral transfection of a single multiprotein expression vector, which comprises the coding sequences of c-Myc, Klf4, Oct4, and Sox2 linked with 2A peptides, can reprogram both mouse and human fibroblasts. Moreover, the transgene can be removed once reprogramming has been achieved.
When the single-vector reprogramming system was combined with a piggyBac transposon, the scientists succeeded in establishing reprogrammed human cell lines from embryonic fibroblasts with robust expression of pluripotency markers.
The study appears in the March 1 edition of Nature.
Source: www.genengnews.com, March 2, 2009
Stem cells in bioreactors may solve platelet shortages
In the future it may be possible to grow supplies of blood platelets in bioreactors rather than relying on donated supplies, according to US researchers.
The discovery, published in the January 1 edition of Experimental Hematology, could avoid shortages in donated blood platelets and reduce the significant costs associated with processing them for use by patients.
Aside from their short shelf life, donated platelets have a number of other drawbacks. They require a huge logistical effort to collect, and also expensive methods required for collection, processing and testing for pathogens.
Researchers have produced platelets in the lab in suspension, in plastic vessels or on ’feeder layers’ of other cells. However, these have tended to give low yields of platelets that do not survive for very long, according to the scientists, led by Larry Lasky, associate professor of pathology at Ohio State University.
His team’s approach has been to use hematopoietic stem cells grown in a three-dimensional scaffold system made of a hydrogel or surgical-grade woven polyester fabric. The system does not require a feeder layer but does rely on coating the scaffold with platelet-boosting cytokines such as thrombopoietin and fibronectin.
After a few days, large bone marrow cells that produce platelets, called megakaryocytes, were produced in the culture system.
“We found production of functional platelets over 10 days with two-dimensional, 24 days with 3D scaffolds in wells, and more than 32 days in a single-pass 3D perfusion bioreactor system,” commented the authors, led by Larry Lasky of Ohio State University. They are now refining the technique to attempt to boost platelet yields.
Source: www.In-pharmatechnology.com, January 6, 2009
M&A fever sweeps drug industry in wake of Pfizer-Wyeth deal
Pfizer’s $68bn swoop for Wyeth looks set to start an epidemic of merger madness according to comments by Frost & Sullivan (F&S) analyst Shabeer Hussain.
Hussain, who heads up F&S’ pharmaceutical and biotechnology coverage team, said that the strong cash position of the drug industry, coupled with a reluctance to invest in R&D in the current economic climate and the impending loss of patent protection for blockbusters will accelerate the rate of M&A activity as companies try to survive.
He went on to suggest a range of potential deals, opining most notably that Sanofi-Aventis, which has been actively seeking to wrestle the top spot from Pfizer, may well forge a deal with Bristol-Myers Squibb (BMS) to maintain the momentum developed through the purchase of Zentiva last year.
Other potential tie-ups offered by Hussain include suggestions that: Sanofi may bid for cell culture specialist Crucell, now that Wyeth has dropped out; Eli Lilly may table an offer for Japan’s Takeda, given their collaboration on diabetes drugs; and that Johnson & Johnson (J&J) is well positioned for a run at Vertex Pharmaceuticals.
Hussain added however that growth through M&A’s was not a sustainable strategy long-term as it reduces the level of innovative R&D. He went on to describe Pfizer’s takeover of Wyeth as “a stopgap arrangement, [intended to help the firm] stay afloat during Lipitor patent expiration”.
Hussain concluded that such merger deals are “crisis management measures” designed to enhance stock prices and are perhaps an indication of wider problems within the drug industry.
One firm that looks unlikely to be involved in M&A talks any time soon is Switzerland’s Roche. Earlier this week the firm announced that its majority shareholder had agreed to keep its stake in one pool for an unlimited period, protecting the firm from potential takeover.
Roche’s move follows comments by Daniel Vasella, CEO of Novartis which has a 33 per cent holding in Swiss rival Roche, that a Novartis-Roche hybrid would be a “great company”.
Source: www.In-pharmatechnologist.com, January 29, 2009
Merck moves for Schering in $41bn mega-merger
The year of the mega-merger continues with Merck & Co’s move to buy Schering-Plough for $41.1bn, with the intention of boosting its pipeline, entering new markets and creating a “powerful biologics presence”.
Merck follows Pfizer and Roche to become the third company this year to launch a takeover bid worth over $40bn, drawing on their sizeable cash reserves in an attempt to equip themselves for challenging years ahead.
Although critics argue that mergers of scale do not maximise stockholder value and diminish R&D returns, many feel they are necessary to ensure earnings per share continue to grow in the current environment.
Merck is expecting the merger to generate annual savings of $3.5bn from 2011 onwards, with some of this probably being a result of integrating operations from their Vytorin (ezetimibe/simvastatin) joint venture.
Through the acquisition Merck will double the number of products it has in Phase III to 18, with this being supported by “high potential” early and mid stage pipelines.
In addition the life cycles of existing products could be extended, by creating new combinations or formulations using Merck’s and Schering’s respective therapeutics and technologies.
Merck also listed the boost in production capacity as a justification for the takeover, stating that it will realise synergies by looking at all operations in light of its “lean manufacturing and sourcing strategies”, which IBM said cut plant operating expenses by 20 per cent.
Although Merck has indicated that the merger will result in a 15 per cent reduction in staff there is currently no indication whether any manufacturing facilities will be sold, with the company instead focusing on the benefits of having additional biologics and sterile medicines capacity.
Merck, like the majority of the industry, is increasingly focusing on biologics, with the company hoping the combination of its novel proprietary biologics platform with Schering’s expertise will equip it for growth.
This expansion into new product markets will be accompanied by a furthering of Merck’s geographic reach, helping it to generate a larger proportion of its revenues from outside the US.
Schering generates 70 per cent of its revenue from countries other than the US and Merck hopes the resulting company will have a 50-50 split with the rest of the world.
Source: www.In-pharmatechnologist.com, March 10, 2009
Multinational deals set to continue in Indian pharma
The Indian pharmaceutical and biotech sectors may see more M&A activity this year with rumours that GSK, Sanofi Aventis and Merck KGaA are on the look out for potential deals.
Although GlaxoSmithKline said that speculation it is in talks with India’s Piramal healthcare is “unfounded”, recent comments by CEO Andrew Witty that the firm would focus on emerging markets fit with such a move.
In addition, comments by Sanofi Aventis chief Christopher Viebacher that the company wanted to expand its presence in India, coupled with his reservations about Pfizer-like mega deals, would seem to indicate that the firm is also on the look out for relatively small scale Indian acquisitions.
On a global scale, Indian drugmakers look like a very attractive proposition for acquisitions, particularly given their relatively low valuation compared to firms elsewhere. In addition, the country’s massive internal market is also likely to feature prominently in the thinking of any potential multinational suitors seeking to sustain revenue growth.
Evidence for the potential attractiveness of India’s drugmakers and its market first emerged in June last year with Daiichi Sankyo’s acquisition of Ranbaxy Laboratories for $4.6bn (€3.5bn).
The fact that Daiichi chose to proceed with the purchase despite Ranbaxy’s problems with the US Food and Drug Administration (FDA) and Health Canada suggests that the massive potential of the Indian market can overcome any short term concerns.
The prospect of more Indian deals was given further credence by the speculation that Wockhardt and Torrent Pharmaceuticals are among several firms to have been approached by multinational Pharma companies, according to a Livemint report.
An analyst said that: “Since the generic market is going to be more and more competitive, the growth prospects for companies that fails to focus on innovation seems bleak in the future.”
Source: www.In-pharmatechnologist.com, February 23, 2009
Merck & Co. to Pay $130M for Insmed’s FOBs and Production Plants
Merck & Co. is offering $130 million for Insmed’s pipeline of follow-on biologic (FOB) candidates and its commercial manufacturing facilities located in Boulder, CO. In December, Merck expressed interest in creating a franchise based on FOBs.
The Boulder facilities comprise 50,000 sq. ft. of biologics process development analytical laboratory and manufacturing plants.
The up-front payment will cover Phase III INS-19 and Phase I INS-20. INS-19, a recombinant granulocyte-colony stimulating factor (G-CSF), will be evaluated for its ability to prevent infections in patients with cancer receiving chemotherapy. INS-20 is a pegylated recombinant G-CSF designed to allow for less frequent dosing.
Insmed’s pipeline also includes IN-21, which is an interferon, and INS-22, which is a glycoprotein that stimulates the development and production of red blood cells and may be used in diseases like anemia.
Insmed will now focus on Iplex, its protein therapeutic platform. Iplex is being developed in a Phase II study of myotonic muscular dystrophy as well as in ALS. Early-stage research programs with Iplex includes retinopathy of prematurity.
Source: GEN News Highlights, February 12, 2009
Teva and Lonza JV targets biosimilars
Teva has made a big push into the biosimilars market through a joint venture with Lonza, which will give the generics giant expertise in biologics manufacture.
Lonza will collaborate with Teva on the development, manufacture and marketing of a portfolio of biosimilars, which they hope will put them in a strong position to succeed when a regulatory pathway is implemented.
The biosimilars market looks set to boom with the incoming US administration prioritising the creation of a regulatory pathway and major players, such as Merck – details here, setting up units to develop products.
Under the terms of the venture Teva and Lonza will collaborate on a selected portfolio of biogenerics. Names of the selected products and financial details of the deal are not being disclosed.
By defining the range of products they will collaborate the two companies have retained the ability to pursue biosimilar opportunities outside the scope of the venture independently or in other partnerships.
Source: www.In-pharmatechnologist.com, January 20, 2009
Biovitrum to divest R&D Segment Cambridge Biotechnology
Biovitrum decided to spin out its small molecule R&D subsidiary, Cambridge Biotechnology (CBT), and a range of primary care projects. Biovitrum will focus on specialist pharmaceuticals and protein therapeutics.
CBT’s pipeline includes a Phase II neuropathic pain project and obesity candidates in Phase II and Phase I trials. The company also has a leptin mimetic preclinical drug in obesity as well as a pain and inflammation preclinical program and four discovery programs in pain and inflammation.
Biovitrum, on the other hand, retains hemophilia and fat malabsorption clinical programs. Both are in mid-stage development. The firm reported net revenues from January to September 2008 of SEK 826.3 million, or $99.78 million. Its portfolio has therapies for oncology, rheumatoid arthritis, hemophilia, anticoagulation, and calcium hemostasis, including three biologics that it recently bought from Amgen for $150 million.
Source: Gen News highlights, January 22, 2009
Cellular analytics: Roche acquires innovatis AG
Roche announced that it has signed a definite agreement under which Roche will acquire 100% of innovatis, a privately held company based in Bielefeld, Germany. innovatis is a leading provider of automated cell analysis solutions, especially focussing on cell counting, viability testing, and cell function analysis in research, as well as bioproduction. The purchase price for this transaction is 15 million Euros.
“This acquisition is a further step in our strategy to strengthen our position as a complete solution provider in the cell analysis research market,” said Dr. Jürgen Schwiezer, CEO Division Roche Diagnostics. “Innovatis’ technology will complement the existing Roche cell analysis portfolio and is synergistic to the xCELLigence technology launched in 2008.” more
Source: Roche press release, March 16, 2009
Roche to acquire Memory Pharmaceuticals
Roche and Memory Pharmaceuticals today announced that the two companies have signed a definitive merger agreement for Roche to fully acquire Memory in an all-cash transaction at a price of approximately USD 50 million.
Memory develops innovative drug candidates for the treatment of debilitating central nervous system (CNS) disorders such as Alzheimer’s disease and schizophrenia. Memory’s nicotinic alpha-7 agonist drug candidates in these disease areas are already in partnered programmes with Roche: R3487 is in phase II clinical trials for Alzheimer’s disease and schizophrenia; R4996 is in phase I for Alzheimer’s disease.
“Acquiring Memory will enable Roche to secure the future development of its promising nicotinic alpha-7 agonists,” said William Burns, CEO Division Roche Pharmaceuticals. “The innovative work carried out by the scientists at Memory will be fully integrated into Roche’s R&D portfolio with the aim of providing new hope for patients and caregivers affected by devastating diseases such as Alzheimer’s.” more
Source: Roche Press Release, November 25, 2008
Roche and Genentech combine the two organizations
Roche (SWX: ROG.VX; RO.S) and Genentech (NYSE: DNA) announced today that they signed a merger agreement under which Roche will acquire the outstanding publicly held interest in Genentech for US $95.00 per share in cash, or a total payment of approximately US$46.8 billion to equity holders of Genentech other than Roche. The special committee of Genentech’s Board of Directors has approved the agreement and recommends that Genentech shareholders tender their shares in Roche’s tender offer.
Research and early development are to operate as an independent center. The South San Francisco site is to become headquarters of combined U.S. commercial operations. Furthermore, Genentech’s unique culture will be maintained
Innovation will be enhanced through a diversity of research approaches and sharing of IP, technologies, partnerships and other key assets
The transaction is expected to be EPS accretive in the first year after closing.
Source: Roche Press release, March 12, 2009
BioInvent International and Thrombogenics start phase II clinical trial
On February 23, BioInvent International and Thrombogenics announced they had started a phase II trial with a long-acting anti-coagulant, TB-402, for the proplylaxis of Deep Vein Thrombosis (DVT) following orthopedic surgery. TB-402, which is given as a single injection, could overcome major drawbacks such as bleeding and the need for extensive patient monitoring associated with current anti-coagulant therapy.
TB-402 is a recombinant human monoclonal antibody that targets Factor VIII. It only partially inhibits Factor VIII activity even when given in high doses. This novel mode of action is expected to reduce the risk of undesirable bleeding events and extensive patient monitoring.
The trial will enroll 300 patients across 36 centers, mainly in Central Europe.
Source: BioInvent Press Release, February 23, 2009
BioInvent presents positive preclinical data for BI-505 in Myeloma
BioInvent International AB (OMXS:BINV) has presented preclinical data demonstrating anti-tumour activity of its therapeutic cancer antibody BI-505 targeting ICAM-1, an adhesion molecule that is highly expressed in tumour cells. The data, presented at the XIIth International Myeloma Workshop in Washington DC, demonstrates that the compound is more efficacious in prolonging survival in animal models compared to bortezomib. The drug launched in 2003 for treatment of multiple myeloma, has shown strong performance in a market with estimated 40,000 new cases annually globally.
BI-505 is a fully human monoclonal antibody derived from BioInvent’s proprietary antibody library
n-CoDeR®. BI-505 was recently granted orphan drug designation by the European Agency for the Evaluation of Medicines (EMEA) for multiple myeloma, having previously been awarded Orphan Drug designation by the FDA in the US market. It is anticipated that the antibody could have an important role in the refractory or relapsed setting, due to up-regulation of ICAM-1 in patients who have developed resistance to chemotherapy. Presentation at: www.bioinvent.com
Source: BioInvent Press Release, February 27, 2009
Avastin and Tarceva in combination significantly improve progression free survival in lung cancer
Roche today announced interim results from a phase III study (ATLAS) in patients with advanced non small cell lung cancer (NSCLC). The study was stopped early because of the significance of the interim data. The results showed that Tarceva (erlotinib) plus Avastin (bevacizumab) given as first line maintenance treatment following initial therapy with Avastin plus chemotherapy extends the time patients live without their disease getting worse (progression free survival) compared to maintenance therapy with Avastin plus placebo. The results will be welcome news for patients and their physicians as extending the time patients live without their disease advancing is a key treatment aim in lung cancer. Most people with lung cancer are diagnosed with advanced stage disease and die within 12 months of diagnosis. more
Source: Roche Press Release, February 3, 2009
Bioceros joins PER.C6 network as only EU cell generation partner
Crucell and DSM Biologics have added fellow Dutch firm Bioceros to the PER.C6 vendor network to provide manufacturing and support capacity in line with increased demand for the cell line. Bioceros will provide the growing number European PER.C6 licensees with cell line generation and support services to help client further their PER.C6 manufacturing and research programs.
While further details are yet to be released, Bioceros’ previous work with Crucell’s STAR antibody platform indicate it is likely to work with licensees like Merck & Co, Morphosys and Eli Lilly which use PER.C6 for protein development and manufacture. Bioceros’ laboratories, which are housed at the University of Utrecht in Holland, have a track record of producing stable cell lines that can be transferred directly into research and industrial scale environments.
In the US, Crucell and DSM’s joint venture firm Percivia provides PER.C6 generation services for North American licensees along with Avid Bioservices, which joined the network in June 2008.
The PER.C6 platform, which comprises Crucell’s human cell line with DSM’s proprietary XD yield boosting technology, was pushed to a record yield of 27 g/L in June last year by a Percivia team working on immunoglobulins.
Source; www.In-pharmatechnologist.com, February 11, 2009
Merck launches biogenerics business
Merck & Co is moving into the follow-on biologics market as it searches for new revenue streams to reduce the damage caused by generic competition.
The company is forming a new business unit, Merck BioVentures, which anticipates having five compounds in late stage development by 2012.
Merck believes it is well equipped to compete in the follow-on biologics market having acquired the humanised GlycoFi yeast platform in 2006, which can produce proteins in yeast.
Merck has noted that the period through 2017 will see extensive patent expirations of leading biologics and is keen to profit from the opportunities this presents.
The GlycoFi platform allows for the creation of proteins with pre-specified and defined human carbohydrate side chains, which is said to offer improved speed, cost and quality over conventional methods. It can also be used to develop novel biologics.
Source: www.In-pharmatechnologist.com, December 10, 2008
$10bn savings from follow-on biologics; CBO report
Implementation of an abbreviated approval pathway for follow-on biologics could result in savings of almost $10bn over a decade, according to a report from the Congressional Budget Office (CBO).
The report predicts that between 2010 and 2019 having an abbreviated approval pathway in place would have a net effect on deficit of $9.2bn.
Additional savings of $2.8bn could be achieved by placing follow-on biologics and their brand name counterparts in the same billing code under Medicare Part B.
The CBO claims this would encourage more widespread use of follow-on biologics as payment rates to physicians are calculated as a weighted average of all products in a billing code.
Consequently those prescribing brand name products would not be fully reimbursed for the brand name products they prescribed when a generic alternative was available.
In turn physicians who prescribe follow-on biologics will benefit financially as they will retain the difference between the products cost and the Medicare payment.
By adopting this carrot and stick approach it is hoped that follow-on biologics will enter into widespread use shortly after they enter the market. As a follow-on biologic becomes more widely used its payment rate under Medicare Part B would decline, offering further savings to the state.
By streamlining the approval process the CBO believes a competitive follow-on biologics market can be created, with multiple manufacturers and a resulting downwards pressure on pricing.
The CBO claims that US spending on biologics exceeded $40bn in 2007 and that products accounting for three quarters of this spending are due to come off patent over the next decade.
This represents a sizeable opportunity for companies entering the follow-on biologics market, such as Merck & Co which is launching a new division for biogenerics, and could confer significant savings to the US.
Source: www.In-pharmatechnologist.com, January 5, 2009
Schering and GSK address the poor
Pandemic influenza vaccine manufacturing technology will be made available to developing countries through a collaboration between the WHO and Schering-Plough.
Under the terms of the deal the World Health Organization (WHO) will gain a non-exclusive licence to use Schering’s technology to manufacture seasonal and pandemic live, attenuated, influenza vaccines (LAIV).
The WHO can also sub-licence the technology, allowing manufacturers in developing countries to produce LAIVs using Schering’s embryonated chicken egg platform.
Schering has permitted the WHO to do this within the framework of its Global Vaccine Action Plan. This is the health body’s guide to preparing for a pandemic and includes the goal of increasing capacity in developing countries, which have so far been the worst affected by H5N1.
Manufacturers using the technology under the WHO’s sub-license will be able to provide vaccines to the public sector of developing countries on a royalty-free basis.
Schering’s offering to developing countries comes shortly after GlaxoSmithKline (GSK) outlined its new strategy, which intends to make medicines more affordable for the poor.
This strategy includes sharing patented processes and chemicals with other researchers to help expedite drug discovery programmes.
In addition scientists from other companies, non-governmental organisations (NGOs) or governments are being invited to work at GSK’s tropical disease research facility in Spain.
This boost to research will be supplemented by GSKs plan to improve hospitals, clinics and staff, which will be achieved by reinvesting 20 per cent of any profits made in the least developed countries back into those nations.
Source: www.In-Pharmatechnologist.com, February 25, 2009
Novartis gets $487m for US cell culture flu vaccin plant
Novartis will receive a further $487m (€366m) from the US HHS for its cell-culture flu vaccine plant in Holly Springs, North Carolina.
The facility, which will boost US vaccine production 25 per cent when fully operational in 2012, has been under construction since 2007 when Novartis won its original Department of Health and Human Services (HHS) contract.
The new deal calls for Novartis to develop two new vaccines, for either seasonal or pandemic use, and provides funding for clinical bridging studies designed to compare the firm’s vaccines to existing products in terms of safety and efficacy, potentially expediting their approval by the US Food and Drug Administration.
The ability to freeze cell cultures means that they can be made available for large scale production in a fraction of the time taken to procure the millions of hens eggs needed to make vaccine using the traditional approach, significantly reducing manufacturing lag.
Such flexability and speed means production can be delayed until the specific virus strain responsible for an epidemic, or pandemic, has been properly identified. This avoids the best-guess, blanket approach of combining several likely strains in a vaccine that is necessitated by the longer manufacturing times needed for egg-based production.
Novartis is at the forefront of cell-culture vaccines through its partnership with Dutch firm Crucell and access to the latter’s popular PER.C6 cell line. In 2007, Novartis’ Optaflu, production of which is due to be switched from the firm’s plant in Marburg, Germany to Holly Springs, became the first cell culture-derived vaccine to be cleared by European regulators.
Source: www.In-pharmatechnologist.com, January 19, 2009
UK report calls for nano regulations
The UK Royal Commission has issued a report on the spread of nanomaterials, stating that “urgent action [is] needed on testing and regulation”.
In its report the Commission found no evidence of nanomaterials causing harm to health or the environment but raised concerns that current levels of testing and regulation are inadequate.
Consequently the report calls for REACH to be expanded to make it more suitable for the regulation of nanomaterials. The report believes that REACH is a suitable framework for regulation but fails in some aspects specific to nanomaterials.
REACH’s one tonne threshold for registration is highlighted by the report as its most significant deficiency, believing this to be too high considering the size of nanoparticles.
The report also highlights REACH’s failure to treat nanoscale versions of existing substances as separate entities, despite the potential for them to possess different properties.
Despite these shortcomings the report argues that specific regulation for nanonmaterials is not required, believing that the legislative field is already crowded. Instead the report believes that current regulations should be adapted to cover the areas where legislation is currently inadequate to deal with nanomaterials.
Some of these issues may be discussed now the European Commission has requested that the Scientific Committee on Emerging and Newly Identified Health Risks (SCENIHR) undertakes a review of REACH in relation to nanomaterials.
Source: www.In-pharmatechnologist.com. November 2009
FDA officially opens its doors in China
The US Food and Drug Administration has opened offices in three Chinese cities – Beijing, Shanghai and Guangzhou – to help improve the safety of medicines and ingredients imported into the US.
The FDA’s main office will be in Beijing, with the Shanghai and Guangzhou locations serving as inspection stations. This is part of a major plan by the agency to expand its international presence, with additional branches due to open in Europe, Latin America and India before the end of the year.
The Beijing office was opened in a formal ceremony last week attended by US Secretary of Health and Human Services Michael Leavitt, FDA Commissioner Andrew von Eschenbach, and Shao Mingli, China’s deputy health minister and head of the State Food and Drug Administration (SFDA).
The opening of the new offices is timely, coming at the tail end of a series of scandals involving Chinese products in the US and other world markets, including contaminated medicines, foods, and personal care products such as toothpaste. China is also at the top of the list of countries for products which have had import permission to the US refused.
With a headcount of just eight, plus five Chinese nationals, the FDA’s presence in China is considered mainly advisory, providing advice on US quality standards and training local inspectors, although it will also maintain an inspection function.
The FDA has been under fire from US politicians in recent weeks, with questions raised about the agency’s oversight of the pharmaceutical supply chain in the wake of the heparin scandal, which saw product from around the world recalled after contaminated heparin sourced from China found its way into medicines, the ongoing probe into quality standards at India’s Ranbaxy and slew of food safety incidents.
One of the persistent criticisms is that the agency should do more inspections of overseas plants. The FDA carried out 13 visits to Chinese production facilities, out of more than 700 sites in 2007. That compared with 24 to France, which has 162 drug manufacturing plants.
Meanwhile, the European Union and China are also looking at working more closely in the future. The SFDA is planning to send a delegation to the EU next year to meet with regulators and industry groups.
The visits had been planned to take place in 2008, but were held back after the massive governmental budget drain which came about as a result of the earthquake in Chengdu earlier this year.
Source: www.In-pharmatechnologist.com, November 27, 2008
FDA clears transgenically produced therapeutic
The FDA gave the green light to the first ever transgenically produced therapeutic protein, also the first recombinant antithrombin, according to the drug’s manufacturer, GTC Biotherapeutics, and its partner, Ovation Pharmaceuticals. The companies expect ATryn® to be available in the second quarter.
ATryn® (Antithrombin [Recombinant]) was approved for the prevention of peri-operative and peri-partum thromboembolic events in hereditary antithrombin deficient patients. It is not indicated for treatment of thromboembolic events in hereditary antithrombin deficient patients.
Along with the approval of ATryn, the FDA’s Center for Veterinary Medicine also approved GTC’s New Animal Drug Application, the first of its kind to regulate genetically engineered animals. This is now required for a recombinant technology used to develop transgenic animals, such as the goats that produce recombinant antithrombin, the company reports.
“The approval of ATryn marks a significant milestone in the development of this innovative recombinant technology and delivers a new therapeutic option to benefit hereditary antithrombin deficient patients who are undergoing surgery or childbirth procedures,” said Geoffrey F. Cox, Ph.D., GTC’s chairman and CEO.
Source: GEN News highlights, February 9, 2009
EMEA adopts a new guideline on monoclonal antibodies
On 18 December 2008, the Committee for Medicinal Products for Human Use (CHMP) of the EMEA adopted a new guideline on “Development, production, characterization and specifications for monoclonal antibodies and related products”. Coming into effect on 1 July 2009, this guideline will replace the one on “Production and quality control of monoclonal antibodies” as well as the quality requirements for monoclonal antibodies set forth in the guideline on “Radiopharmaceuticals based on monoclonal antibodies”. You can read this 11-page document here.
Source: EuropaBio News, February, 2009
MabThera approved in the EU for patients with chronic lymphocytic leukaemia
Roche has announced that the European Commission has approved MabThera (rituximab) in combination with chemotherapy for use in patients with previously-untreated chronic lymphocytic leukaemia (CLL), the most common type of leukaemia to affect adults. The approval is based on compelling results from the pivotal phase III study CLL8.
“Data from the CLL8 trial suggest that MabThera used in combination with chemotherapy has the potential to become the standard of care for the treatment of CLL”, said Professor Michael Hallek, University of Cologne, Germany, who led the German CLL Study Group (GCLLSG) in conducting the CLL8 trial. “Today’s approval will make the best treatment, MabThera plus chemotherapy, available to patients with CLL across Europe”. more
Source: Roche Group Media Relations, February 27, 2009
RoACTEMRA approved in Europe to treat patients suffering from Rheumatoid Arthritis
Roche today announced that the European Commission has approved RoACTEMRA (tocilizumab, known as Actemra outside of the EU), to treat patients with rheumatoid arthritis (RA). RoACTEMRA, in combination with methotrexate (MTX), is indicated for the treatment of adult patients with moderate to severe RA who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists. In these patients, RoACTEMRA can be given as monotherapy in cases of intolerance to methotrexate (MTX) or where continued treatment with MTX is inappropriate. RoACTEMRA is the first interleukin-6 (IL-6) receptor-inhibiting monoclonal antibody developed for the treatment of RA and is a novel approach to help tackle this debilitating disease. more
Source: Roche Press Release, January 21, 2009
AGENDA
11 - 15 May 2009, Frankfurt am Main, Germany
29th International Exhibition-Congress on Chemical Engineering,
Environmental Protection and Biotechnology
Information: www.dechema.de/International_Events-lang-en.html
May 18-21, Atlanta, Georgia, USA
May 28-29, Biberach an der Riss, Germany
Only for ACTIP members
Info: actip-secretariat@telefonica.net
1st International Symposium and Advanced Course
APIB-2009
3-6 June 2009, University of Pavia, Italy
Information: www.edqm.eu/site/API-from-Bioprocess-Pavia-Italy-1359.html
19-22 July 2009, Montreal, Canada
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23-25 October 2009, Athens, Greece
www.eurasante.com/en/great-projects/eurobio-2009.html
June 24-28, 2009, Basel, Switzerland
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6-8 October, Hannover, Germany
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