July News from BIO Ventures for Global Health

African sleeping sickness, more formally known as the two-stage human African trypanosomiasis (HAT), is spread via the bite of the tsetse fly. In stage 2, the disease attacks the brain, causing patients to hallucinate and fall into a coma. People with sleeping sickness have been known to attack their friends and suffer from agonizing pain simply by touching water. As the course of the terrible disease slowly grinds to a halt, patients become so weak that they cannot eat, they fall into a coma, and then meet death. For 25 years, the drug most commonly used to treat African sleeping sickness was melarsoprol. A derivative of arsenic, it killed roughly 8 percent of patients injected with it, failed to cure one-third of them, and could burn the veins of those who survived. The only new treatment in decades is a less toxic combination product that is difficult to deliver in remote African settings.

The discovery of a new boron-based compound has led a partnership among Anacor Pharmaceuticals, SCYNEXIS, and Drugs for Neglected Diseases initiative (DNDi) to one of the most promising developments in neglected diseases research: potentially the first inexpensive, easy-to-administer, oral treatment for African sleeping sickness. Pace University, the Swiss Tropical and Public Health Institute, the Sandler Center of the University of California, San Francisco, and Advinus Therapeutics have also contributed to these efforts. DNDi's Dr. Robert Don, Discovery & Preclinical Director and Head of Human African Trypanosomiasis Discovery & Preclinical Program, and Anacor Pharmaceuticals CEO David Perry spoke with BVGH about the importance of this new drug candidate, set to begin clinical trials soon, and how it could help lay the disease to rest forever.

This is an exciting development, especially for the world of public-private partnerships engaged in neglected diseases! Why do you believe this announcement is so important?

David Perry (DP): My view is that this is important because of the impact it can have on the disease. We all got into biotech and drug development to do something important, and this drug candidate, along with other efforts in global health that are going on, has the potential to eliminate African sleeping sickness -- not just treat patients, not just control the disease, but actually eliminate the disease. I don't know how it gets more exciting than that.

Robert Don (RD): It's important for a couple of reasons: one is that this is the first time in recent history that a new class of chemicals based on boron has been developed -- first by Anacor and more recently by DNDi and SCYNEXIS -- as a modern therapy for sleeping sickness. The second reason is that we at DNDi have now developed a pipeline of treatments for sleeping sickness. We've taken into account that there is a risk that drugs will fail during the development process, and rather than just focus on getting one new drug into clinical trials and hoping that will work, we're actually building a succession of compounds so that we're building in the capacity for them to fail.

When and why did Anacor begin testing this boron-based compound against neglected diseases?

DP: Nine years ago, Anacor was founded with the idea that incorporating the element boron into small molecules would have advantages in creating drugs. While we were testing that concept, we discovered that our technology platform applied well to killing parasites, which are the source of many neglected diseases that tend to infect people who don't have the money to pay for therapies. That is a daunting realization. On one hand, we realized that we had technology that could greatly impact world health -- that was the good news. On the other hand, we, like every other biotech, are funded by investors, and we have a responsibility to them to maximize their return.

How did you get from that point to where you are now in your partnership with DNDi?

DP: We developed a philosophy that if we have technology that can significantly impact world health, we also have a responsibility to apply that technology to deliver solutions. We are committed to using our people and our technologies to do that as long as we don't have to spend our investors' money on it. So we then began an effort to find people who could help fund this research, and we've set up a number of collaborations. DNDi was one of the first, and this drug candidate for sleeping sickness is the most advanced compound. We're also working with Medicines for Malaria Ventures on malaria, with OneWorldHealth on shigellosis, with the Bill & Melinda Gates Foundation on river blindness (onchocerciasis), and with the TB Alliance on tuberculosis.

How does DNDi effectively engage with biotech, and how did your organization get involved with Anacor's work on this drug candidate?

RD: We at DNDi appreciate that new drug discovery and development relies heavily on the pharmaceutical and biotech industries. It's critical for us to work with them -- there's no point in us reinventing the wheel and trying to do it by ourselves. With this sleeping sickness drug candidate, Anacor took a very proactive approach in seeking us out. We licensed the compound in very good condition, allowing us the freedom to work completely within the neglected diseases area on the drug candidate. We'd like to encourage companies in the pharmaceutical and biotech industries to have a look at their compounds and see if there could be any application to neglected diseases. It doesn't have to cost a lot, and it could lead to some very exciting results for people in need.

What advice would you give to biotech companies that want to collaborate with a product development partnership such as DNDi or other global health group on neglected disease R&D?

RD: For one, developing a drug to benefit people in the developing world who suffer from neglected diseases doesn't have to significantly impact a biotech or pharmaceutical company's bottom line. So if they have technology that is potentially applicable to a neglected disease, DNDi can give them advice, support, and even screen their compound libraries to see whether there is anything of use in the global health sphere. Not only is DNDi happy to work with them on the science, we're also happy to work under mutually beneficial licensing conditions to create a win-win for all. We understand these companies' commitments to their investors, and we're willing to work with them on that.

DP: Contact one of these product development partnerships -- it's as simple as that. Most biotech companies will undergo a similar process of screening compounds as we did. They will not feel like they can spend their investors' money on this type of research and development, but the good news is that there is money available. It takes some work to get to, but there are organizations set up to fund this kind of work, and if biotechs work at it, they not only can find the money, but do what most of us in biotech entered the field to accomplish: to cure devastating diseases.

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