The Year That Was

2. EU REGULATES REGENERATIVE MEDICINE

One of the bigger milestones for the European biotech industry in 2005 came toward the end of the year when the European Commission published long-awaited draft regulation governing what they call "advanced therapies" – including gene therapy, cell therapy and tissue engineering. The proposal, published in mid-November, aims to guarantee health protection for patients treated with these therapies, plus making it easier for companies to get products to market by establishing an EU-wide regulatory framework for their authorization, supervision and post-authorization vigilance.

"It's very important," says Aurelie Vandeputte, Manager of the Healthcare Council at EuropaBio, the EU biotech industry group. "It will help ensure patient safety because you have regulation at the EU level." It will also help even out a rugged playing-field for companies who currently have to contend with vastly differing regulations in different EU member states, she says.

3. 454: BUILT FOR SEQUENCING SPEED

© 2005 Nature Publishing Group

When 454 Life Sciences announced in May that it had inked a $62 million, 5-year deal with Roche for the promotion of its fast genome sequencing system, it was one of several high points in a good year for the Branford, Conn.-based subsidiary of CuraGen.

The 454 sequencing system uses picoliter-scale technology, light emitting sequencing chemistries and top-notch informatics to allow fast, whole-genome sequencing. The company sold its first system in March, to the Broad Institute of MIT and Harvard.

In September, the company gave the genomics world a black and white illustration of the technology's use. In a paper published in Nature, they described a shotgun sequencing and de novo assembly of the Mycoplasma genitalium genome with 96% coverage and 99.96% accuracy in one 4-hour run of the machine. That would have taken months 10 years ago.

4. BIOSIMILARS ARE DIFFERENT

With billions of dollars of biopharmaceuticals due to come off patent in the next few years, "biosimilars" – follow-on versions of original biologicals – are set to be increasingly big news. In 2005, the European Medicines Agency (EMEA) set the pace in developing a clear regulatory path for this type of drug, the biopharmaceutical version of generics.

"Biosimilars are in essence a new, third class of medicinal products," says Thomas Bols, director of government affairs for Europe at the US biotech firm Amgen. "We have now innovative, generic and biosimilar products. For biosimilars to get approved, a new legislative and regulatory pathway had to be created as both the approval schemes for innovative and generic medicines are not appropriate."

In this, the EU is taking a lead. "The EU is the first region in the world – before the US in any case – which has now adopted a legislative and regulatory pathway for the approval of biosimilar medicines," Bols points out.

5. CONFLICTED INTERESTS

After nearly two years of investigations, congressional hearings, and internal turmoil, the National Institutes of Health in August issued final conflict of interest regulations for its employees, easing restrictions on ownership of stock in drug and biotech companies and allowing most staff scientists to interact with colleagues in nonprofit and scientific organizations. The rules relaxed more stringent regulations announced in February that would have limited stock held by all NIH employees and totally ban all outside consulting, even with nonprofit organizations. Those rules were imposed after media and congressional reports revealed that some NIH scientists had accepted lucrative consulting contracts with outside companies. But the rules were relaxed after scientists reported widespread dissatisfaction and a few senior officials said they would quit. The new rules limit stock ownership for only 200 senior NIH scientists and employees but ban any consulting with pharmaceutical and biotech companies. Senior employees have until February 2006 to divest of their biotech and drug company holdings in excess of $15,000.

6. TYSABRI SUSPENDED

When Cambridge, Mass.-based firm Biogen-Idec voluntarily pulled its monoclonal antibody Tysabri (natalizumab) from the US market in late February, it was clearly very bad news. The decision to withdraw the multiple sclerosis drug from market was made after one fatal case and one additional case of progressive multifocal leukoencephalopathy (PML) were confirmed in patients receiving the drug. But as well as being a downer for the company and its marketing partner, Ireland's Elan Pharmaceuticals, the setback for Tysabri may have been something of a wakeup call for the biotech industry: Biologics can, despite their specificity, cause adverse events.

After reaching that nadir in February, however, there are signs the Tysabri story may have a happier ending. Safety testing this year has revealed just one other case of PML. J.P. Morgan Securities upgraded shares of the company to "overweight" from "neutral," and in October, Forbes suggested the drug may be back on the market before the end of 2006.

7. TERROR TACTICS EVOLVE

When the New York Stock Exchange postponed a planned listing of Huntingdon Life Sciences in September, the biotech industry saw it as a worrying sign that biomedical research had taken a backseat to the tactics of animal extremists. The decision had been preceded by a violent and threatening campaign against the company by the Animal Liberation Front, and prompted Biotechnology Industry Organization (BIO) chief executive James Greenwood to write to NYSE officials warning that "allowing terrorists to hobble the marketplace is equivalent to surrendering democracy."

The government is concerned as well: In May, John Lewis, US Federal Bureau of Investigations deputy assistant director for counter-terrorism, told the Senate Energy and Natural Resources Committee that the FBI is investigating more than 150 cases associated with animal rights and eco-terrorism in the United States.

Meanwhile, companies are fighting back, making more use of laws passed to protect them from terrorists like Stop Huntingdon Animal Cruelty-USA, which has been the subject of injunctions.

8. BIOTECH IN AFRICA

On the opening day of an agricultural science and technology meeting in Burkina Faso, on June 21, Pamela Bridgewater – US deputy assistant secretary of state for African affairs – announced that the presidents of four African countries had voiced their support for agricultural biotech. The heads of state of Mali, Niger, Ghana, and Burkina Faso all stressed to US officials how important they felt it was to focus on the uses of biotechnology to improve health and well-being of their populations, according to a report posted on several US Embassy Web sites.

The report makes no mention of any concrete actions being planned by those governments, but their statement comes at a time when recognition of the value of science is gaining political support in the continent of Africa. In September, the governments of more than 40 African countries pledged their support for a $160 million plan to boost science and technology across the continent. In terms of spending, that plan puts the "safe development and application of biotechnology" at the top of the pile, on an equal footing with securing and sustaining water.

9. MONEY FLOWS FROM OFF SHORE

In June this year, newspapers in Britain and beyond reported that a private equity group based in Bermuda – Celtic Pharma – was going to buy the British biotech company Xenova for up to £26.1 million. David Oxlade, chief executive of Xenova, told The Financial Times that the deal would ensure the company had enough funds to take its drugs to launch. "Public markets are not willing to provide those funds to the extent required," he said.

Oxlade went on to suggest that the deal might set a precedent in the biotech sector. Certainly Celtic, founded in December 2004, has its sights set on rapid expansion – it has raised some $300 million so far, and has its sights set on $1 billion by next year.

10. FDA APPROVES DRUG FOR BLACK PATIENTS

Nitromed, an emerging firm based in Lexington, Mass., made history in July this year when the Food and Drug Administration approved its BiDil drug for the treatment of heart failure in self-identified black patients. The first racially targeted drug, BiDil was said by the FDA to represent a step toward the holy grail of personalized medicine.

Robert Temple, FDA associate director of medical policy, said "the information presented to the FDA clearly showed that blacks suffering from heart failure will now have an additional safe and effective option for treating their condition. In the future, we hope to discover characteristics that identify people of any race who might be helped by Bidil."

But for John Hodgson of UK life sciences consultancy Critical I, the approval with a racial indication was "odd." "What the analysis didn't separate out was the very likely fact that the African-American group, matched age for age, were sicker in the first place," he says. In his view, the data suggest that the effect of the drug could be specific to the sicker population rather than a specific race group. "It's a very bad example, I think, of pharmacogenetics being used, and a very diverse group being lumped together in an inappropriate way."