Elusys Therapeutics scored another $50.2 million under a broader contract with the US government to advance the development of the privately held firm’s experimental inhalational anthrax antitoxin Anthim (ETI-204), a high-affinity deimmunized monoclonal antibody.
Anthim targets the protective antigen of Bacillus anthracis, the bacterium that causes anthrax, and neutralizes the lethal effects of anthrax toxins.
The agent is formulated as a solution suitable for either intraveneous or intramuscular administration, Elusys President & CEO Dr Elizabeth Posillico told Scrip.
The award is part of a five-year contract with the US Biomedical Advanced Research and Development Authority (BARDA) that could potentially total $143 million, of which Elusys already has banked $57.5 million.
Dr Posillico said the latest round of funding will be used to conduct pivotal efficacy studies in animals and complete validation of the commercial manufacturing process for Anthim.
The ultimate goal, Dr Posillico said, is to gain the US FDA’s approval and win a contract for Anthim to be included in the US Strategic National Stockpile (SNS) – a repository of medical products and equipment intended for use in the event of a natural disaster or bioterrorism attack.
Anthim is being developed under the FDA’s animal rule, a regulatory structure created in 2002 that permits a drug’s effectiveness to be demonstrated in animals when it is unethical or not feasible to conduct controlled clinical trials in humans.
Under the animal rule, a medication could be approved on the basis of adequate and well-controlled animal studies when the results establish that the product is reasonably likely to produce clinical benefit in humans.
In assessing the sufficiency of animal data, the FDA may take into account other data, including human data, available to the agency.
Under the rule, regulators rely on studies in animals to provide substantial evidence of a product’s effectiveness only when there is a reasonably well-understood mechanism of action against a target with known pathophysiology; the effect is demonstrated in more than one animal species; the animal study endpoint is clearly related to the desired benefit in humans; and the data on the kinetics and pharmacodynamics of the product or other relevant data in animals and humans allows selection of an effective dose in humans.
The efficacy and safety of Anthim is being studied in animals, with safety studies also being conducted with human volunteers.
Dr Posillico noted that Elusys recently completed a clinical dose-escalation study of Anthim in 108 human volunteers and collected additional safety and pharmacokinetic data at intravenous doses up to 16mg/kg.
The Pine Brook, New Jersey-based biotech also has completed two other dose-escalation safety studies in humans, in addition to several studies assessing activity of Anthim against anthrax in two animal species.
To date, five studies assessing the efficacy and safety of Elusys’ agent in animals with inhalational anthrax have been completed.
“We also are seeing some very good stability with this product,” Dr Posillico said, adding that the firm feels confident it can “provide the government with a product with good shelf life.”
The new $50.2 million is expected to carry Elusys’ activities for Anthim over the next 18 months, Dr Posillico said.
Those 18 months will be full of critical activities that will need to be completed to ensure the firm is ready for the next steps of assembling data for the biologics license application, and hopefully, the SNS contract.
But, Dr Posillico explained, the company must get through “a lot of gates” first to establish its readiness to qualify for a contract.
“The government wants to make sure we have a manufacturing process well established and also FDA agreement on pivotal study design,” she said.
Dr Posillico noted that Elusys has another separate five-year BARDA contract worth $68.9 million to advance Anthim as an agent to prevent disease and death from exposure to anthrax, which the firm won last September (scripintelligence, 12 September 2011).
“We are very excited about the product,” she said. “We think that it has a lot of potential.”
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