News & Observer | newsobserver.com | Company's aim: better drugs for poor

Published: Dec 27, 2007 12:00 AM
Modified: Dec 27, 2007 06:02 AM

Company's aim: better drugs for poor

Scynexis joins forces with group that battles often-neglected illnesses

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DEVASTATING DISEASES, FEW TREATMENTS

Effective, inexpensive or donated medicines are available for a large group of tropical diseases, such as leprosy. But treatment options for others are limited. Those diseases include:

BURULI ULCER: More than 40,000 cases of the bacterial infection have been reported in Africa, the Americas, Asia and the Western Pacific in the past 30 years. Infection leads to extensive destruction of skin and soft tissue, with the formation of large ulcers, usually on the legs or arms. Little is known about transmission.

CHAGAS DISEASE: An estimated 18 million people in Central and South America, most of them women, are infected by the parasite, which is transmitted through insect bites, blood transfusion or from mother to newborn. The disease can cause irreversible damage to the heart, digestive system and brain.

CHOLERA: The bacterial infection causes severe diarrhea and without treatment can cause death within hours. In 2005, more than 131,000 cases were reported worldwide.

SLEEPING SICKNESS: More than 300,000 people in sub-Saharan Africa are estimated to be infected with the parasite, which is transmitted by insect bites. Symptoms include disorientation and sleep disturbances. Left untreated, the disease is fatal.

LEISHMANIASIS: About 1.5 million people in Central and South America, Africa and Asia are infected every year. The parasitic disease is spread by infected sand flies and can cause skin sores, enlargement of the spleen and liver, and swelling of glands.

(WORLD HEALTH ORGANIZATION, DRUGS FOR NEGLECTED DISEASES INITIATIVE, U.S. CENTERS FOR DISEASE CONTROL AND PREVENTION)

INTERNATIONAL GROUP TARGETS SLEEPING SICKNESS

The players

* Scientists and researchers at Scynexis, a Durham drug chemistry company, headed by Yves Ribeill; Genzyme, a Boston biotech company; and Pace University in New York

* Drugs for Neglected Diseases Initiative, a nonprofit organization backed by Malaysia, Kenya and India, a Brazilian foundation, the French Institut Pasteur and the international humanitarian organization Medicins Sans Frontieres. It is funding the scientists' research.

The goal

* New treatments for sleeping sickness

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Diseases that infect millions of people too poor to pay for medicine have fascinated Yves Ribeill for about 20 years.

As a young chemist, he worked on malaria treatments at Rhone-Poulenc. After the French drug maker followed the lead of other pharmaceutical companies and dropped the research, Ribeill remained involved as a World Health Organization consultant on tropical diseases such as malaria, sleeping sickness and Chagas.

Now Ribeill, CEO of Durham drug chemistry company Scynexis, found a way to combine his passion with his business.

Last month, he worked out a deal with the Drugs for Neglected Diseases Initiative. The group is paying Scynexis $17 million over five years to help find safer medicines to treat sleeping sickness, also known as human African trypanosomiasis.

Creating a new medicine would bolster Scynexis' reputation, but success would not translate into more money. The mother lode of contract drug discovery is payments for experimental drugs that hit certain development milestones, as well as the royalties on sales. In this case, Scynexis can't expect either, because any drug that comes out of the agreement with Drugs for Neglected Diseases will cost very little, if anything.

"It's probably not the most lucrative business we have going, but it fairly compensates us," said Scynexis' executive director of market development, Terry Marquardt.

Twenty of Scynexis 130 employees are assigned to the project, which aims to create chemicals to treat sleeping sickness. Scientists at Boston biotech company Genzyme and Pace University in New York will help, using techniques refined in developing treatments for diseases prevalent in Western countries.

"We use research tools for diseases of the rich and come up with better drugs for the poor," Ribeill said.

Scynexis and chemistry

Founded in 2000 by 24 scientists who worked at Aventis CropScience's headquarters in Research Triangle Park, Scynexis has had unconventional deals in the past.

Scynexis helps pharmaceutical companies with the chemistry involved in creating experimental drugs, and it manufactures small amounts of drugs for testing. It has contracts to come up with promising treatments for two-legged and four-legged patients.

Drug makers in the U.S. and Japan have hired the company to look for antifungal medicines, cancer treatments and drugs to treat metabolic diseases. Scynexis' biggest contract is a deal worth as much as $200 million to find treatments for cats, dogs and horses.

The contracts are expected to generate $30 million in revenue this year, and Scynexis is considering a possible initial public offering in 2008.

Two years ago, with the business humming and turning a profit, Ribeill decided that Scynexis could turn its attention to tropical diseases.

As a Frenchman, he's aware of Europe's colonial past and said neither large drug makers nor Western governments have done much research on tropical diseases since pulling out of those countries. Once he turned his efforts to making a difference, he recruited Bakela Nare, a Zimbabwe-born biologist, from Merck to crank up research of parasites that cause tropical diseases.

Third World misery

The need for the effort is great. The World Health Organization estimates that at least 1 billion people -- about one in six worldwide -- is infected with at least one of the diseases. It estimates that there are currently 300,000 cases of sleeping sickness, most in the Democratic Republic of Congo, Angola, Uganda and the southern Sudan.

Sleeping sickness, which is transmitted by the tsetse fly, wreaks havoc as it invades the organs, immune system and brain. It is fatal if left untreated, but the medicines that treat it are toxic or old. Most require a series of injections, making the treatment difficult under the best of conditions.

The multibillion-dollar pharmaceutical industry has had little interest in researching treatments for sleeping sickness or other such diseases, because they affect people who have little money for medicines.

Foundations and nonprofit organizations have begun to step in in the past few years, Ribeill said.

"The world is becoming a smaller place," he said. "There's been a rising awareness that a healthy population is needed in places like Africa."

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