With the so-called "patent cliff" looming on the horizon, the inhalable therapeutics market has become "one of the fastest growing spaces," said Rob Neville, executive chairman of 2007 start-up Savara Pharmaceuticals.
"Companies are seeking new formulations and new routes of delivery," he said, and Savara's NanoCluster formulation technology, originally developed by company founder Cory Berkland at the University of Kansas, is designed as a next-generation pulmonary delivery platform aimed at overcoming some of the limitations of earlier inhaled products.
The company can take compounds - both marketed drugs and new chemical entities - and formulate them using the NanoCluster technology, which engineers particles out of the drug itself, negating the need for excipients. The concept is based on natural examples of aerosols such as spores from molds and fungi.
"We tried to mimic that, with something that looked like a spore" by changing the molecular structure of the drug, Neville said.
The goal is to create particles small enough to ensure adequate deposition of the drug into the lung. In most existing inhaled products - inhaled asthma drugs, for example - only 15 percent to 30 percent of the drug actually gets into the lungs, he said, and of that, only a fraction makes it into the deep lungs, the rest going into the throat and stomach where it can result in systemic effects.
With the NanoCluster technology, "we've been able to dial in the drug at between 1 and 3 microns," Neville told BioWorld Today. "That's the perfect range of those particles."
Best of all, he added, the technology "is very reproducible and scalable," and data to date have demonstrated a "high dose-to-dose repeatability."
Savara is working on a couple of programs on its own, namely in lung cancer prevention and lung cancer staging, using grant money, but most of its business development centers around partnerships in which it will apply the NanoCluster technology to other companies' compounds.
Those deals typically start with a feasibility phase, with Savara formulating the drug and returning it to partners. "Then they evaluate it, and we enter into licensing negotiations," Neville said.
Savara, which raised seed funding in August, recently closed a Series A round. Though the company did not disclose the amount raised, an SEC filing indicated that the firm pulled in $833,000 of a $1.4 million round, with a total of 13 investors. The individual investors were not disclosed.
Neville said the Series A funding should take the firm through the end of 2010. He added that Savara also has revenue coming in from out-licensing its particle engineering technology services to other companies for drug development purposes.
"That's the value of having a platform technology," he said. "We have a number of areas" to potentially pursue.
The company initially is focusing on indications "involving infections in the lung," he said, such as asthma, chronic obstructive pulmonary disease, pulmonary bacterial infections and is working on ways to deliver tuberculosis therapy to the lungs.
Savara also had some early positive data in cancer. An upcoming issue of Pharmaceutical Research is expected to highlight preclinical data showing that a chemotherapeutic agent could be delivered directly to the lungs using a NanoCluster-based formulation at a much lower dose, suggesting a method of possibly treating lung cancer without the usual systemic side effects, Neville said.
But is Savara going to get into the inhaled insulin space, which, after some notable failures, seems to have been re-ignited by positive data from MannKind Corp.'s Afresa (insulin human [rDNA origin]) inhalation powder?
"We do have a 100 percent insulation formulation" that's available for licensing, Neville said, though the company does not intend to pursue that product on its own.
For now, Savara is using its technology to develop dry powder inhaled therapeutics, though the company would like to expand into the nebulizer and propellant markets as well. "Those are both very big markets, and we believe our technology is applicable," he said.
Savara operates with five full-time employees, plus another 12 to 15 part-time workers and consultants, though Neville said the latest financing will allow the firm to hire at least three new business development people.
The company is based in Austin, Texas, though its wet-lab and technical operations are in Lawrence, Kan.