Genzyme is best known for its rare disease work. So perhaps it’s no surprise that some of its former executives are helping to steer six-year-old Cambridge, MA-based startup Aura Biosciences in that direction as well.
Aura today is announcing it’s raised a $21 million Series B round from a group of VC firms and former Genzyme CEO Henri Termeer. Advent Life Sciences led the round, which included a group of new backers (Chiesi Ventures, Ysios Capital, and Alexandria Venture Investments) and some old ones (LI-COR Biosciences) as well. Aura has now raised a total of $37 million since its inception in 2009, according to founder and CEO Elisabet de los Pinos (pictured above).
Termeer isn’t the only ex-Genzymer involved. Alan Walts, former head of Genzyme Ventures, is today being named Aura’s executive chairman. Alison Lawton, another former Genzyme exec, has become Aura’s chief operating officer.
It’s all part of Aura’s strategic pivot to rare forms of cancer—specifically, eye cancers for which there are no current treatments. Aura isn’t saying which type of eye cancer it’s going after first, but it’s now got the cash to begin its first clinical trial this year, according to de los Pinos.
Aura is developing a technology that uses viral nanoparticles as tools to deliver cancer drugs. These particles aren’t actually viruses, so they don’t have DNA or hijack cells and replicate like a virus would. Rather, they’re empty protein shells that resemble viruses and are specially engineered to selectively target tumors.
Aura uses these particles like ultra-small smart bombs. In the same way that an antibody-drug conjugate (ADC) links a toxin to a guiding antibody, Aura’s protein shells are chemically linked to drugs that are shepherded to tumor cells. De los Pinos says that these nanoparticles can carry much more drug than antibody, and can be used for a broad range of cancers. But to prove those claims, the company is not going after the types of cancers ADCs are already approved to treat—like Hodgkin lymphoma (Adcetris, from Seattle Genetics) or breast cancer (Kadcyla, from Roche/Genentech and Immunogen)—or the pancreatic or HPV-associated cancers that were its initial focus.
Instead, de los Pinos says that it made more strategic sense for Aura to pursue a “rare disease business model applied to cancer.” That is, uncommon cancers with no approved treatments. Focusing here enables Aura to run small, quick, cost-efficient clinical trials, and also makes it easier to advance a drug forward without having to give up any rights to the program.
“We’re not thinking of partnering,” de los Pinos says. “The idea is, since we have the capital now, to get into the clinic and retain all the value.”
Aura has already applied for an orphan drug designation, which, if granted by the FDA, would give its prospective treatment longer market exclusivity, she says.
Eye cancers fit the bill for Aura because the need is significant: the only treatments are surgery, radiation, or laser therapy, each of which can potentially leave patients blind. It’s also rare, compared to other cancers. Statistics from the American Cancer Society show that some 2,580 patients in the U.S. are expected to be diagnosed with eye cancers—mainly melanomas—in 2015. (By comparison, the ACS expects 221,200 new cases of lung cancer this year.)
Aura believes its approach may be able to treat eye tumors without threatening vision. The concept would be to inject one of its nanoparticles into the eye. That nanoparticle would be tagged with an infrared dye—a product made by an Aura investor, LI-COR, called 700DX. Once the nanoparticle reaches the tumor, a clinician would shine a specific laser light over it. The dye then activates, and kills the tumor cell, leaving healthy tissue unharmed.
That’s the idea, at least. So far, Aura has tried this approach in rabbits, and was able to destroy tumors without damaging the retina. Humans are a different story, of course. But while there are big hurdles ahead, Aura will know fairly quickly if it’s onto something. If successful in its first trial, it aims to move straight to a pivotal study.
That’s “the beauty of this indication,” de los Pinos says.
