sábado, 20 de marzo de 2004

Un nuevo modelo de negocio en Farma

Alexis Borisy, alumno de Harvard y un colega, fundaron una start-up en el año 2.000 con un modelo de negocio innovador y mas eficiente en costes, para comercializar farmacos.
Un articulo en RedHerring, titulado "Old drug, new tricks", lo comenta:

A glass or two of wine doesn’t cause too many hangovers. But mix in a few shots of whiskey or tequila, and chances are, you’ll pay. This synergistic reaction – in which two relatively ineffective agents combine to pack a powerful punch – is the driving force behind a new pharmaceutical company, aptly named CombinatoRx.

Si deseais mas informacion, la web de esta empresa es CombinatoRx .

Old drug, new tricks
A pharma startup may have found a faster, cheaper way to market.

A glass or two of wine doesn’t cause too many hangovers. But mix in a few shots of whiskey or tequila, and chances are, you’ll pay. This synergistic reaction – in which two relatively ineffective agents combine to pack a powerful punch – is the driving force behind a new pharmaceutical company, aptly named CombinatoRx.

Founded in 2000 by CEO Alexis Borisy, a Harvard alum, and colleagues, CombinatoRx is taking a novel approach to creating and marketing drugs. Mr. Borisy and his fellow scientists have invented the first “combination drug discovery platform,” which systematically combines previously marketed drugs to look for new and potentially more effective compounds.

Taking advantage of previously approved drugs is the key to CombinatoRx’s business model – and the reason it’s making pharmaceutical waves. The strategy could dramatically truncate development and testing periods, significantly reduce the amount of risk involved, and save its developers a boatload of cash.

But as any entrepreneur will tell you, a successful pharmaceutical company needs a lot more than a great idea. Mr. Borisy and his colleagues will now have to prove they’ve got what it takes to traverse the U.S. Food and Drug Administration’s (FDA) tricky path to approval, which has tripped up even large pharma companies.

CombinatoRx’s approach is based on a simple premise: a disease of the human body isn’t confined to a single path, so why should a drug use only one path to treat it?

“These new kinds of drugs work together in a synergistic manner to attack the disease on multiple fronts,” says Mr. Borisy, who was named one of the top 100 innovators under 35 by MIT’s Technology Review magazine for his advances in the field. The typical human tumor is caused and maintained by 100 to 200 genetic mutations, he says, and the traditional view of drug development – “a single molecule going at a single target” – doesn’t stand much of a chance against such complexity.

Mr. Borisy and his team of scientists are trying to change the single pathway paradigm and bring a host of combination drugs to market. VCs are signing on: CombinatoRx has raised a total of $90 million in three rounds of private financing – $20 million in 2001, $40 million in the fall of 2003, and another $30 million this month. Third-round investors include Boston Millennia Partners, Yasuda Enterprise Development, Flagship Ventures, Canaan Partners, TL Ventures, and all existing institutional investors. Mr. Borisy says that he plans to go public sometime in 2005 or 2006.

For now, CombinatoRx is using the money to fund research into several combination compounds. The company’s technology platform combines two drugs at a time and analyzes its effect on certain diseases, at a multitude of different doses and ratios. The number of possible combinations is staggering – 2 million for each disease, including diabetes, cancer, and arthritis.

“Surely, out of 2 million in each area, there will be several significant new products,” says Mr. Borisy. The software screens several hundred thousand data points per day, and automatically flags interesting pairs.

The drugs that make up the interesting combinations are often completely ineffective against a particular condition on their own. For instance, neither an anti-psychotic nor an anti-parasitic drug have any effect against cancer – but together, they could achieve a synergistic reaction to create a new drug for cancer, and attack the disease in several ways.

Combining an anti-psychotic and an anti-parasitic drug to make an effective cancer treatment may seem counterintuitive – but that’s the point. CombinatoRx’s technology allows its scientists to search all possible combinations, rational or not. “A few people have stumbled happenstance onto an interesting combination, but no one is doing this,” says Mr. Borisy.

The aforementioned combination, named CRx-026, scored the company its first U.S. patent in May 2003. The drug, which has proven effective against tumors in mice by arresting cell division, is currently in Phase I of FDA trials. With a new drug, it would typically take at least four years to get to this stage – a testament to CombinatoRx’s competitive advantage.

“Because we are using existing drugs with known components, you already have a lot of information about them,” says Adeeb Mahmud, associate of corporate development. Having this information, including the drug’s potential side effects and its safety and effectiveness in humans, will save time, which – in pharmaceutics, especially – equals money.

Drug developers often gripe about the long FDA approval process, which can take up to 10 years. Pre-clinical stages, when a drug is researched and prepared for FDA review, can also take up to six years and cost tens of millions of dollars, according to Mr. Borisy – unless the drug is a combination of already-approved compounds, that is. His method could dramatically shorten this pre-clinical period to one or two years.

CombinatoRx currently has three drugs in various stages of clinical tests, which will double by the end of 2004, and “many, many in the pre-clinical pipeline,” says Mr. Borisy. He declined to estimate when the patented CRx-026 could make it to market because of the uncertainty regarding government approval.

CombinatoRx plans to look at every approved drug in all major disease areas and develop them into marketable drugs, either on their own or with the help of big pharma. “We are in talks with most of the major pharmaceutical companies,” says Mr. Borisy, though he declined to state any details. For now, CombinatoRx is focusing on drugs with expired patents. CombinatoRx has 70-plus patents of its own pending worldwide – one of which would prevent any company from copying its approach. “Because it’s novel and non-obvious, we get very, very strong intellectual property protection,” says Mr. Borisy. The patent, if granted, will prevent anyone from combining drugs in a systematic nature. “This is a unique capability,” he says, “it has taken 10 to 20 people several years and millions of dollars to build.”

As many a biotech startup has learned in the past, getting a drug through the FDA is expensive, and CombinatoRx still has to prove that there’s a market for its combination drugs. In any event, it will have an audience. “A lot of people are watching the company,” says Mr. Borisy, “because what we’re doing is unlike anything that’s been done before.”