Small companies may be key to big advances in malaria treatment
New approaches look hopeful -- but given past challenges, there is reason for skepticism
In May, the editors of the New Scientist pondered whether the M&A frenzy in the pharmaceutical industry bodes well for drug development in the area of neglected diseases, including tropical diseases such as malaria. Their opinion: No, not likely.
There is a reason for the skepticism. Writing in the June 8 edition of The New York Times, Dr. Francois Nosten, professor tropical diseases at the University of Oxford, recounted his experiences of watching history repeat itself when it comes to malaria: An outbreak would decimate vulnerable people in at-risk populations, after which an effective new drug would contain the outbreaks -- and then the inevitable mutations would develop, rendering the latest treatment ineffective.
In his piece, Nosten suggested a way forward: Rapid elimination of the parasite that causes malaria by taking preventive measures instead of only treating people who already have malaria. He explained that many people in high-risk regions carry the malaria parasite though they are not sick. So, based on his suggestion, rapid elimination of malaria would involve massive dosing of entire populations, including people who do not have malaria.
While that strategy may seem radical, approximately 207 million people worldwide have malaria and 627,000 people die from it each year -- mainly children under 5. Concern about these statistics has led multinational organizations to pledge billions of dollars towards creating and implementing more effective prevention and treatment strategies.
For example, in 2005, the President’s Malaria Initiative (PMI) was set up, with funding of $1.2 billion for a five-year period. The goal was to target the 15 hardest hit countries in Africa and reduce malaria-related deaths by 50%. Then in 2008, another $5 billion was pledged through the Lantos and Hyde Act, and most recently the Global Health Initiative was started under the Obama administration with the goal of taking on malaria in all of Africa. Ultimately funding flows through various channels, including the National Institutes of Health (NIH), which acts as a conduit to companies that are conducting innovative pharmaceutical research.
But which companies are getting that funding? When BioPharma Dive contacted Nosten, he reiterated his position that big pharma is generally not focused on or invested in developing antimicrobials.
“The pharma industry is more into drugs for chronic diseases than in infectious diseases, despite the looming disaster of resistance, as exemplified by the situation with malaria," he said.
As a result, much of the opportunity for targeted and innovative research belongs to smaller companies -- those with fewer than 80 employees and/or less than $200 million a year in revenues. While neglected diseases may be a negligible area of research for large companies, it could have good economies of scale for a smaller company. And in general, smaller companies with truly innovative or promising antimalarial treatments are also likely to benefit from small-business grants, orphan drug designation and a very savvy intellectual property strategy.
A biotech startup changes the game
DesignMedix is a Portland-based biotech startup that says it “focuses on globally important infectious diseases.” Its lead candidate for the treatment of malaria, DM1157, is being co-developed with Dr. David Peyton at Portland State University.
In April, DesignMedix received a $3 million grant to continue development of DM1157 -- the third grant the company has received in the last several years. DesignMedix Chief Operating Officer Sandra Shotwell has been involved in malaria research for the past 15 years and has 25 years of early-stage technology commercialization experience.Her company’s partnership with Peyton, listed as the company's chief scientific officer, has yielded some hard-earned breakthroughs.
In October 2008, the company received an exclusive license for its anti-resistance technology, developed at Portland State, as well as a round of funding from the Oregon Angel Fund. Then in April 2010, FDA granted orphan drug status to DM1157. That qualifies DesignMedix for funding from the agency's orphan products grants program, as well as tax credits for up to 50% of clinical testing expenses. In addition, DesignMedix will have reduced regulatory review costs, an expedited review timeline and seven years of marketing exclusivity.
The next step is to bring the drug and technology the company is developing into clinical trials and address issues related to capacity, which tends to be a concern for smaller companies. Once again, partnerships with external agencies and stakeholders will help to address that issue when it’s time.
DesignMedix has received the funding and support because its therapeutic approach to malaria is genuinely innovative. It turns out that mutations that lead to resistance cause rapid transport of drugs used to treat malaria out of the body, which renders those drugs ineffective. DesignMedix has developed technology that is designed to slow the passage of anti-malarial medication through malaria cells.
At the moment, it seems that all eyes are on Pfizer, AstraZeneca, Valeant and Allergan---but there are big stories happening at small companies, though these stories are often not front-page news. As DesignMedix continues to develop its anti-malarial therapies, people in Africa will continue to live in fear of the rainy season when cases of malaria spike. Shotwell and her colleagues are determined to change that situation by bringing a new drug to market and applying an innovative technology to the problem of resistance. Like Nosten, Shotwell’s goal is to overcome the scourge of malaria once and for all.