AMSTERDAM/ZURICH Roman seems a typical six-year-old boy, smiling shyly next to his younger sister and mother Silvia as they open the door to their Amsterdam home.
But Roman has Duchenne muscular dystrophy (DMD), a genetic disorder that already makes it hard to walk long distances and will probably put him in a wheelchair by his teens. Lung and heart problems mean most DMD patients die aged 25 to 30 years.
"He knows that his muscles are not that strong, but he does not know it is progressive," says Dutch events manager Silvia. "He will ask about it himself, when he can handle it. That's the hard thing, his pain -- that's the hardest thing."
Yet there is a glimmer of hope. Roman's mother believes a small Dutch biotech company called Prosensa, located 40 kilometres away in the university town of Leiden, may finally have found a way to help Roman and others like him.
She's not the only excited one. GlaxoSmithKline (GSK.L) is throwing its weight behind the new drug and the two companies last month started a final-stage Phase III clinical study of a product that might help up to 13 percent of boys with DMD.
Silvia hopes Roman will be enrolled in subsequent trials.
Prosensa is one of a new wave of successful European biotechs, many of them in the Benelux countries and Scandinavia. They are stepping over a past generation of biotech failures and starting to win interest from investors.
Significantly, it focuses on rare diseases -- a red hot area in the wake of Sanofi-Aventis's (SASY.PA) $20 billion (12 billion pounds)-plus deal to buy Genzyme GENZ.O.
Europe's biotech sector has stumbled in the past but there is now renewed talk of stock market listings and trade sales.
Prosensa is preparing the ground for an initial public offering (IPO) and will choose its moment carefully.
"It is one of the options," Chief Executive Hans Schikan told Reuters.
Prosensa last year hired a chief financial officer with IPO experience, although Chief Business Officer Luc Dochez stresses a flotation "will be a choice, not a forced option" as Prosensa still has two years of funding.
Other young biotech companies are also eying the public markets, including Dutch rival Kiadis, a specialist in cell-based therapy, which bankers believe is again looking to list after pulling a planned IPO in 2007.
There are signs the market is recovering from recent trough years and 2011 could see more IPOs, with investor interest in places like Amsterdam and Brussels encouraging, although the overall picture is fragile.
"The market is improving. In Europe, a few biotech IPOs are being planned," one banker said.
The Netherlands and Belgium are a hotspot, helped by the sentiment-boosting $2.4 billion buyout of Dutch vaccine maker Crucell CRCL.AS by Johnson & Johnson (JNJ.N) and high hopes for Belgian biotech star performer ThromboGenics (THR.BR). Some German and Austrian IPOs are also on the cards, bankers say.
Nomura analyst Samir Devani detects a "power shift" in European biotech, particularly away from the UK, where a series of blow-ups with key pipeline products at companies like Antisoma ASM.L have taken a heavy toll on confidence.
Last year saw the emergence of a new group of well-financed European biotechs worth $500 million or more, including ThromboGenics, Sweden's Medivir (MVIRb.ST), Denmark's Bavarian Nordic (BAVA.CO), Sweden's Diamyd Medical DIAMb.ST and Norway's Algeta ALGETA.OL, he said.
The biotech casino remains high-risk, given the binary nature of the clinical trials that drive values. But the growing appetite of "Big Pharma" for biotech assets is underpinning the sector and analysts at Jefferies expect out-licensing deals and consolidation to remain key share price drivers in 2011.
For those companies braving the IPO market, however, the waters are still choppy.
Danish biotech Zealand Pharma (ZELA.CO), for example, got its issue away in November, but only after pricing it at the low end of the indicated range. The shares have fallen more than 20 percent since then.
More recently, Swiss-Italian biotech Philogen scrapped its plans to list in Milan after Bayer (BAYGn.DE) ended a partnership with the group.
Despite the setbacks, Thomson Reuters data shows an overall pick-up in biotech IPOs last year after a dramatic slowdown in 2008 and 2009.
U.S. biotech have also had mixed results this year, with Pacira Pharmaceuticals (PCRX.O) and Endocyte Inc (ECYT.O) going public, but Clarus Therapeutics pulling its IPO on February 11.
Anne Portwich, a partner at Life Sciences Partners, said many venture capitalists looking to realise biotech investment gains were now more focussed on M&A than IPOs.
"Companies that want an IPO have to be much more mature than they did in the past," she said.
BENELUX VS SWITZERLAND
After various high-profile setbacks, times are tough in Switzerland. Historically a centre of excellence, the talk these days among biotech executives is of survival.
Sander Slootweg, managing partner at venture capital firm Forbion, said that in the past, some over-priced Swiss biotechs had raised cash at "valuations that we would never invest in."
Tim Dyer, Chief Financial Officer of Swiss biotech Addex (ADXN.S), said a shortage of capital may mean some companies either have to close or merge to create critical mass.
"One of the strategies of these sub-100 million (Swiss francs) market cap biotechs could be to combine with other similarly sized companies to recreate the critical mass and put themselves back on the radar screen of larger funds," Dyer said.
Vontobel analyst Andrew Weiss agreed a wave of M&A could be brewing as a last resort for some companies.
For different reasons, he said Cytos Biotechnology CYTN.S, Cosmo Pharmaceuticals (COPN.S), Evolva (EVE.S) and also Addex could also look to team up, but not necessarily with each other.
Back in Belgium, Edwin Moses, the CEO of biotech Ablynx (ABLX.BR), which makes drugs based on llama DNA, finds the investor mood rather different -- helped by a strong retail interest that runs beyond the traditional wary Belgian dentist.
"I've found that people in the street, bakers and taxi drivers, they all follow companies like Ablynx, like ThromboGenics, very carefully in a way that I've not experienced in the larger countries," Moses said.
"There's quite a sophisticated retail market here where people pay attention even to complicated stories," he said. "At the moment the place to be seems to be here."
Whether the science will help boys like Roman remains to be seen. Prosensa and Glaxo are still recruiting the 180 patients they need for their key Phase III study and it will take 48 weeks of regular injections to determine if they really do have the first treatment to alter the course of DMD.
"Hope gives life," Roman's mother Silvia says.
(Additional reporting by Ben Deighton in Brussels, Ben Hirschler in London and Ludwig Burger in Frankfurt; Editing by Alexander Smith)
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