There is a New Clinical Trial of a Novel Drug for African Sleeping Sickness. Who Cares?

7/12/11

I do. Millions of Africans do. Two of the world’s wealthiest people do. And against all odds, two U.S. biotech companies do too.

The story starts in Africa where about 7,000 new cases of sleeping sickness happen each year. If diseases could be classified as cruel and unusual, sleeping sickness would be at the top of the list. In the early stage, most people don’t know they are infected and the disease can be very mild. In stage 2 of the disease, the parasite enters the brain and things get really nasty. Hallucinations start and sick people have been known to chase their neighbors with machetes or to scream in pain at the touch of water on their skin. Only at the end does the sick person slip into a coma prior to death.

The drug melarsoprol, discovered more than 60 years ago, is the primary treatment for the deadly stage 2 disease. A derivative of arsenic, it kills about 8 percent of those who receive it, fails to cure one-third of those treated, and has a nasty habit of burning the veins of patients. The treatment is 10 painful days of intravenous injections. The only new treatment in decades is a combination product that lessens toxicity but is still challenging to deliver in African settings.

The 37 countries at risk and the 7,000 people sickened by the disease each year are mostly the rural poor in countries at war or in crisis. Not an attractive market by any measure. I sometimes think about a company CEO defending a business plan to venture capitalists that includes African sleeping sickness. Non-starter!

And yet two successful biotechs—Palo Alto, CA-based Anacor Pharmaceuticals (NASDAQ: ANAC) and Research Triangle Park, NC-based Scynexis—are doing just this. How can this work?

Anacor has boron chemistry as its basic platform. It turns out that these compounds have activity against many targets, including parasites. From the beginning, CEO David Perry has been committed to finding a way to use the company’s core assets to pursue targets that would improve human health-even in areas where the market would not support the return on investment typically needed. There was no way Anacor could spend venture capital money on global health targets.

So Anacor went to the non-profit organization Drugs for Neglected Diseases initiative (DNDi), which is run by Bernard Pecoul and funded by the Bill & Melinda Gates Foundation, Doctors Without Borders, and several other organizations. DNDi is a product development partnership that has built the largest ever R&D portfolio for kinetoplastid diseases such as this one. DNDi partnered with Anacor and also brought their existing partner Scynexis into the mix.

Anacor had found compounds with activity against the whole parasite Trypanosoma brucei. Essentially you mix the compounds and the parasites together and see which compounds kill the parasites. More interestingly, the compounds appeared to be active against both early and late stages of the disease. Anacor and Scynexis began working together in 2008 to develop a preclinical candidate from this compound series. Scynexis worked, with funding from DNDi, with the compounds to reach the optimal mix of safety, efficacy, and availability in the brain in order to pass the blood-brain barrier and be effective against stage 2 of African sleeping sickness.

Now this product—the first new oral drug candidate in decades targeted specifically toward African sleeping sickness—is going into a Phase 1 clinical trial.

Who wins in this situation? Anacor gets a small amount of non-dilutive financing that allows it to cover its costs and learns more about its drug chemistry platform. It has also expanded its proprietary boron chemistry library for no cost. Scynexis conducts this work on a fee-for-service basis as a contract research organization (CRO) and is able to use and build its expertise in parasitology. To work on one of the great unmet medical needs of our times has engendered pride and loyalty in the employees of both companies that goes far beyond the gyrations of the stock prices. DNDi gets two strong partners with the technology, the will, and the scientific capabilities to more quickly advance compounds and develop drugs against one of the most neglected of the neglected diseases.

Then of course there are those people who suffer and will continue to suffer from a disease simply because they are too poor to create a paying market. With an effective drug, sleeping sickness could go the way of other diseases that are on the brink of being eradicated.

It is very cool to say that you created a drug that extended peoples lives by three months and that the market cap of your company rose into the billions. But how much cooler is it to say that you lead a commercially successful company that also provided the tools for the potential eradication of a disease that afflicts the poorest in our world? I often talk to companies who say when we reach “X” milestone then we can think about global health. But this kind of partnership shows that there doesn’t have to be a trade-off. The benefits and opportunities of partnering in global health have never been better.

Learn more about this new Phase 1 clinical trial in PLoS Neglected Diseases:

Read the BIO Ventures for Global Health case study about this partnership:

Melinda Moree is the CEO of BIO Ventures for Global Health, a non-profit organization whose mission is to save lives by accelerating the development of novel drugs, vaccines, and diagnostics coming from the biotechnology industry. Follow @

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