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Filling need for small patient populations

October 22, 2007

Ovation Pharmaceuticals is creating a home for orphan drugs and developing new uses for older ones -- an innovative strategy that's aiding patients who have no or few options, and one that's helped it emerge as one of the fastest growing biopharmaceutical companies in the nation.

The Deerfield-based company acquires branded pharmaceutical medicines and promising late-stage drugs that are no longer priorities of big pharmaceutical companies, but that address unmet medical needs in small patient populations with severe illnesses.

It primarily targets drugs approved in Europe that need U.S. regulatory approval and solicits guidance on what medicines and drugs to pursue by working closely with specialty physicians.

Ovation recognized it needed to initially buy a group of drugs that could generate $100 million in sales pretty quickly. Its first 10 drugs did that. Sales have grown on average 50 percent a year at the privately held company.

Ovation Chief Executive Jeffrey Aronin explained, "While our focus really is developing new innovative medicines and lifesaving therapies, by having therapies that are already approved and by developing new uses for them, we've been able to take those earnings and invest them in our pipeline so that we can help more patients with unmet needs."

Ovation has 18 products on the market in 87 countries, including drugs to treat cancer, epilepsy, Parkinson's, lead poisoning in children and anxiety. One drug, Panhematin is used to treat the roughly 800 people who need it because of a rare enzymatic disorder.

A major focus area for Ovation is central nervous system illnesses. It has more late-stage epilepsy drugs in development than any other company, Aronin said.

Ovation is on track to launch four drugs to treat six different illnesses by 2012. At peak sales, those drugs have the potential to generate nearly $2 billion in sales.

Ovation expects to generate $800 million to $1 billion by 2012.

Among the products in its pipeline is clobazam, a drug to treat a rare form of childhood epilepsy in which patients lose all muscular power during the seizures; and a drug to treat infantile spasms, a devastating form of epilepsy that strikes infants, plaguing them with thousands of seizures a day. The latter patient population is less than 10,000, and there are no U.S. approved treatments for the infant condition, said Dr. Stephen Collins, Ovation chief scientific officer.

Ovation also is developing a drug to treat psychosis in Parkinson's patients, a drug used in Europe, but never approved anywhere else for this condition.

It's developing a drug to treat epileptic patients with complex partial seizures and an IV form of the anti-seizure drug carbamazepine. The latter would be the first and only injectable form of the medicine. It would meet the critical need of patients who temporarily must be taken off of the oral version of the medication, but who would be at risk of complications if switched to another drug.

"It was totally impossible to make carbamazepine into an intravenous formulation for many many years," Collins said. "We worked with investigators at the University of Minnesota to come up with a delivery trick. It's almost like a Trojan horse. There's a little cage of sugar the [drug] molecule goes in, and when you inject it into the blood vein, it pops out of the little cage. Now it's in the blood stream, and can't go back in the cage. It's very slick."