Mapp Biopharmaceutical - A 2014 Fierce 15 Extra

Mapp Biopharmaceutical
Mapp's Ebola cocktail goes viral as outbreak spreads in Africa

CEO: Kevin Whaley
Based: San Diego
Founded: 2003
Clinical focus: Infectious diseases, Ebola
Website: www.mappbio.com

The scoop: Kevin Whaley isn't given to hyperbole. In fact, the CEO at Mapp isn't much given to publicly discussing ZMapp, the remarkable new treatment for Ebola, at all. At a time when every public biotech company with a preclinical program for Ebola is clamoring for attention, Whaley has given precious few interviews. And when he has talked about ZMapp, he's been careful to say that the company doesn't know whether it works and has lots more work to do. If anything, the air of mystery has only heightened the lurid 24/7 cable news attention given to ZMapp, which could help revolutionize the way in which outbreaks are treated in years to come.

ZMapp is a cocktail therapy made up of antibodies that Mapp's small team of 9 has assembled into a single treatment. For a vaccine, investigators would work on delivering antibodies that would prime the human immune system to fight off a lethal virus like Ebola. But for people who are already infected, facing about a 50% mortality rate, this new approach has the potential to provide a powerful and immediate response. So this year, FierceBiotech is going the extra mile on the Fierce 15, adding Mapp as a Fierce 15 Extra for its principled and pioneering stance.

What makes Mapp Fierce: ZMapp includes antibodies that were generated in mouse models exposed to an Ebola protein, then "humanized" to prevent rejection, transferred to tobacco plants through a benign plant virus--or vector--and grown in the genetically engineered tobacco leaves, which are harvested to produce the therapy. The cocktail includes antibodies licensed from Defyrus and USAMRIID, all drawn by the notion that a cocktail therapy would prove to be a patient's best shot at survival. That combination of antibodies in the cocktail represents the culmination of 10 years of work, and it was only arrived at in January.

The NIH unintentionally helped get the media frenzy started when they supplied a few doses to treat two Western Ebola victims, who appear to have responded very well and are now recovering. In a matter of weeks, Mapp nailed another impressive primate study, saving all the infected animals from a likely death. The U.S. government followed up with a contract worth up to $42 million to speed up work on production. The helter-skelter development effort was pointed down the path to a quick approval as Mapp's slow-motion progress of recent years collided with the fact that only one Ebola treatment was in the clinic, and that one had been under a clinical hold at the FDA before regulators immediately cleared it for production.

Enormous logistical issues remain. In addition to the clinical program that's needed to fully test the safety and efficacy of the treatment in humans, the treatment would need to be made in large quantities in order to combat the worst outbreak health officials have seen since Ebola first appeared in 1976. Currently, only one biologic is approved for manufacturing in plants, and that is Protalix's ($PLX) Gaucher's drug, which is made in plant cells. Kentucky BioProcessing currently makes ZMapp, using vector technology from Icon Genetics. But even with a huge effort, KBP would need months to scale up production.

South African officials say they have been approached about building a facility, which would take time, while Protalix has had to walk back some statements implying that they could adapt their manufacturing process to churn out ZMapp.

Regardless of how long it takes to contain the current outbreak, it will pass eventually. And Mapp will be able to go back to working without the cable news attention after making some quick and important advances. Whether ZMapp makes it to the goal line or not, their work with cocktail therapies has helped clarify the most promising approach to beating back one of the most challenging viral threats since HIV came along. And hopefully U.S. and global officials will have learned to consistently keep up the support needed to defeat Ebola once and for all.

Investors: The U.S. government; the Defense Threat Reduction Agency in the Department of Defense and the National Institute of Allergy and Infectious Diseases at the National Institutes of Health provided much of the funding and research support, with BARDA stepping in recently with a contract for manufacturing.

For more:
Feds pledge up to $42M to hit the gas on Mapp's Ebola drug
Mapp's Ebola treatment comes through in a preclinical study
Global fear of Ebola outbreak stokes a frenzy of new R&D, production plans

-- John Carroll (email | Twitter)

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Mapp Biopharmaceutical - A 2014 Fierce 15 Extra