Last month, I posted a review of a report on “The Global Health Social Enterprise” (R4D report) written by a team at Results for Development Institute’s Center for Global Health R&D Policy Assessment” (“For Fun and Profit”). While I noted that the report was based on a limited information base that did not include any perspective from the many biotech/pharma industry analysts, I thought it provided a good description of the range of business models used by organizations/companies developing global health products. The report also had a nice appendix that summarized ten such companies and I thought it worthwhile to take a closer look at them.
First, the group (60° Pharmaceuticals, AltraVax, Anacor, DesignMedix, Genocea, GenVec, Inviragen, Napo, PaxVax, and Sequella) included five developing therapeutics and five vaccines (I’m not sure why no diagnostics companies). Second, all but one are still in the development stage and have no marketed or licensed products (Napo’s licensee, Salix, recently got FDA approval for the anti-diarrheal drug, crofelemer, although Napo is legal disputes with Salix [PR Newswire article] and its rest-of-world licensee, Glenmark [First Post article]). Two other companies (Anacor, through DNDi, and Sequella) have products in mid-late stage trials. Third, the companies vary widely in age from a few to multiple years (60° Pharmaceuticals was founded in 2010; Anacor in 2002) and size (DesignMedix has five employees, two of whom I know from my technology transfer days; Genocea has about 40). Fourth, and not surprisingly, the companies run the full range of funding levels and modes: modest funding through government grants (e.g., DesignMedix) to millions raised from professional VCs (Genocea has more than $60 million from nine investment firms but also Gates and DOD grants [Boston Globe article]) to multiple millions from a pharma partner and public sale of stock (Anacor).
Last, although the R4D report’s authors stated the companies were chosen because they were “explicitly dedicated to the social purpose of developing new medicines, treatments, or technologies for diseases of low- and middle-income countries,” I found the degree to which the companies were explicit about their dedication varied. My findings:
|Company||Global health mentioned in company mission statement||Number of global health projects/total number of projects*|
*Global health projects are those aimed at diseases afflicting many people throughout the world, e.g., HIV/AIDS, malaria, and hepatitis B, and not primarily in the developed world, e.g., herpes and peptic ulcers.
I find no fault in a company not being explicit in its dedication to a social mission or even mentioning or having a social mission. How a company presents itself, whether to the public, its employees, or investors, is less important than the effort it makes, first, in developing a product for a global health need, and, second, in getting that product used by the many people needing it. And as I noted above, while these companies are making progress, none has yet met an unmet need.
So what is needed as these companies get closer to marketing their products? Of course, therapeutics and vaccine companies face major hurdles from having a manufacturing system to meet demand, regulatory approvals in relevant countries, approved and accepted pricing, and distribution to customers. I expect that all of the companies mentioned have been on the hunt for partners to provide the capital and expertise to accomplish these tasks and have rationally assessed how they will get their products to market. Or have they? I put on my BD hat and took a closer look at PaxVax (PV) to see what it intends. First, I noted the company is taking a practical approach in that it is only working on orally-administered vaccines which will avoid the complexity of the distribution system needed for current vaccines and may simplify the “fill and finish” step in manufacturing. The approach will limit the diseases addressable by its products but increases the chances that the products will actually be used. Second, the company is using proven technology. For two of its products, vaccines for H5N1 ‘flu and HIV, PaxVax is using a proprietary technology (administration of antigen via live, replicating adenovirus but not the several of the 57 types that cause human respiratory and gastrointestinal illness) but one that has been used in humans to prevent adenoviral respiratory infections in the military (PV technology). For its cholera and dengue vaccines, the company is using whole, inactivated virus, a technology used in currently licensed vaccines. Third, PaxVax is backstopping its manufacturing by developing a robust and low cost process for its adeno-based vaccines, based on its own cell line that grows serum-free in suspension culture (GEN article). Fourth, the company has a done a good job with its funding. The company states it has raised more than $50 million from a VC firm, Seattle-based Ignition Capital (Ignition), and the Wellcome Trust and in grants from the US NIH and the Gates Foundation, although details are not provided. A bit disconcerting is that the company is the lone biotech investment of Ignition, meaning PaxVax is on its own for its partnering and commercialization effort. Also disconcerting is that the company has no VP for BD. But it looked to me as if Paxvax is addressing the lack of commercialization experience by hiring last year a vaccine industry veteran, Thomas Monath, who was CSO of Acambis, a Cambridge-based company, for 1992-2006 and who piloted registration of one or more of its products. I didn’t meet him when I interviewed for a BD job I did not get in 2005 but the company did well enough when it was acquired by Sanofi for more than $500 million in 2008. So on my grading card, PaxVax gets “A”s for mission, funding, and technology and a “C” for business development, but it is not too late to get that ball rolling.