Back in September, I wrote in this space that if a global flu pandemic ever strikes, public health officials might turn to a Lexington, MA-based startup company called Pulmatrix.
The pandemic (a bit overblown, I must say) did strike. And yes, the public health officials have been calling Pulmatrix.
This company’s technology is nowhere near ready for prime time in big clinical trials, much less the marketplace, so isn’t all the fuss a bit premature? Maybe. Then again, most biotech companies work on pretty incremental advances over the standards of care, but Pulmatrix is one of those rare beasts that has a chance to transform how physicians think about treating many major respiratory diseases. The technology has attracted $18 million in initial equity financing from Polaris Venture Partners and 5AM Ventures, and a scientific advisory board that includes David Edwards of Harvard University and Robert Langer of MIT. It’s been a few months since we last wrote about this company, so I got an update from CEO Bob Connelly.
The concept at Pulmatrix challenges the status quo of antiviral treatment, in which a drug is engineered to kill a single infectious invader, which works for a while until that virus inevitably uses its evolutionary tricks to develop resistance. This is the “one drug, one bug,” method, as Pulmatrix puts it. Instead of going that route, Pulmatrix is developing a technique that’s supposed to stop any pathogen or flu strain that might find its way into the lungs. It calls this the “one drug, multiple bug” approach.
“The single drug for multiple bug approach is what’s really generating a lot of attention for us, even though we’ve been keeping a low profile,” Connelly says.
Pulmatrix is trying to do this by creating aerosols that have positively-charged ion-based compounds, like calcium and magnesium, that would be sprayed into the lungs. These compounds are supposed to do a couple of things. First, they stimulate immune defenses to prevent infection. Second, the aerosols are supposed to change the viscosity of the mucus that lines the lungs, which activates proteins in the lungs to form 3-D matrices that create a firewall of sorts that blocks pathogens of any kind from burrowing deep into lung tissue. So far, in animal and early human studies, this method hasn’t gummed up the mucus lining of the lungs, which could make it harder to breathe, or worse, create a haven for infectious bugs to thrive.
“Think of it as like a river with a light coating of ice on top, but with the river flowing smoothly underneath,” Connelly says. “It’s more difficult to penetrate the surface top layer, and there’s still clearance below.”
What’s more, Pulmatrix is supposed to change the properties of the airways, so that when people breathe in a pathogen—like swine flu—it doesn’t form into those tiny droplets that people can sneeze out in the direction of other people. Essentially, it should make flu a whole lot less contagious.
Sounds great, but where’s the proof? Pulmatrix hasn’t said a whole lot publicly on this point. It issued a press release on May 1 saying that it completed a Phase I clinical trial that assessed the safety and tolerability of single escalating doses of its first drug, PUR003, in eight patients. The drug was found to be well-tolerated, with no severe adverse events reported. Animal studies, presented at a National Institutes of Health antiviral workshop, have shown that in multiple species, and with multiple strains of flu viruses, the drug worked. That included swine flu. Because of the way the drug is designed to work, it is thought to be able to both treat and prevent flu infections.
Based on the animal tests, and the early safety trial in humans, Pulmatrix is moving into a tougher test, a study in which 25 healthy volunteers will be randomly assigned to get its drug or a placebo, and see how they do after they’ve been exposed to the flu in a controlled environment.
This study is expected to get started next month, and assuming no snags or enrollment delays, it should produce results as soon as October, Connelly says.
The study, while small, will produce a lot of evidence that people at the Centers for Disease Control and Prevention, the National Institutes of Health, and the higher reaches of the U.S. Deparment of Health and Human Services will want to see. People in those government agencies have been meeting with Pulmatrix to talk about its technology, and they weren’t just picking up the phone and hopping on the bandwagon after the “swine flu” headlines hit, Connelly says.
The company’s technology is obviously at very early stages. The Phase I study in eight patients was done in a liquid formulation that’s delivered via a nebulizer—a clunky contraption that patients need to inhale from for a few minutes, twice a day. These devices are currently used for patients with cystic fibrosis, a fatal lung disease that causes buildup of thick, sticky mucus in the lungs, but it’s probably not the most practical way to deliver a mass-marketed flu drug, Connelly says. So Pulmatrix is working on two main delivery technologies.
One is a liquid nebulizer form for severe lung diseases like cystic fibrosis and chronic obstructive pulmonary disease, in which a nebulizer isn’t too much of an inconvenience. The other is a dry powder form that could be made into a portable inhaler like the ones people carry around for asthma attacks. That more convenient package is expected to enter clinical trials in the first half of 2010, Connelly says.
“You could take one or two puffs in the morning before work,” Connelly says. For a market like flu, which is episodic rather than chronic, “two minutes on a nebulizer would be too inconvenient,” he says.
The list of potential applications is a long one, with asthma, allergies, and rhinovirus (common cold) also ranking as high priorities, Connelly says. All of the trials to prove these concepts would take money, of course. Unlike most entrepreneurs I’ve been talking to, this didn’t seem to worry him much. The company has grown from 14 employees in January 2008 to about 34 now, and it expects to continue to grow. Pulmatrix secured an additional $3 million from Polaris and 5AM Ventures in March, and the company expects to do a Series B financing later this year. “We’re in good shape now,” Connelly says. “The good news for us is our existing investors are passionate about the company.”
And the competition? That’s mostly from established players like Roche’s oseltamivir (Tamiflu) and GlaxoSmithKline’s zanamivir (Relenza), although neither one is designed to modify the mucus lining of the lungs or offer such broad potential as Pulmatrix. “We’re really very much unique in the approach we’re taking,” Connelly says.
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