After Lux Biosciences announced in August 2010 that the FDA declined to approve its experimental drug to treat an inflammatory disorder of the eye called uveitis, the company pretty much went silent. No more press releases, few scientific publications, very little visibility beyond some presentations at medical meetings by outside investigators. Under the tutelage of CEO Dean Mitchell, a veteran of GlaxoSmithKline and Bristol-Myers Squibb who was brought in to deal with the regulatory challenge, Lux went about as deep into stealth mode as a company can go.
That’s why today’s announcement from the Jersey City, NJ-based company is likely to raise some interest in the burgeoning ophthalmology industry. Lux says in a statement that it has completed enrollment of the 155-patient clinical trial that it was required to complete before the FDA would reconsider approving its drug, called voclosporin (Luveniq). If all goes as planned, Mitchell says, Lux will release data from the trial early next year and resubmit the drug for approval in both the U.S. and Europe shortly after that.
Lux’s drug is designed to treat a subset of patients with uveitis (pronounced you-vee-EYE-tis) that affects the back or midsection of the eye. The disease mostly strikes women in their 40s and is the fourth-leading cause of blindness in the developed world. About 250,000 people in the U.S. and Europe have the condition, making voclosporin eligible for “orphan” designation and a condensed, six-month priority review by the FDA. That means the drug, which can be taken in pill form, could be ready to hit the market by the end of 2013.
Getting from idea to late-stage development has been arduous for the 16-person startup. Lux was founded in 2006 by four ophthalmology industry veterans who licensed the rights to develop voclosporin as an eye drug from Edmonton, Alberta-based Isotechnika. Lux ran two trials: one in patients with flare-ups of uveitis and the second in patients who were in remission.
The first trial worked, but the second didn’t, leading the FDA to demand that the company replicate the successful study before returning to seek approval. “It was bad—it meant a two-year delay,” Mitchell says. “But we knew that if we repeated the trial and it was successful, we’d be approved.”
Lux’s development team took some of the lessons they learned from the first approval attempt and applied them to the new trial, with the blessing of the FDA’s head of ophthalmology, Mitchell says. For example, the company carefully spelled out the criteria for determining whether or not patients are responding to the drug—a change that Mitchell says is designed to prevent physicians from allowing patients to drop out of the trial prematurely. The company also started measuring the response to the drug 12 weeks into the 24-week trial, rather than waiting until patients have been on it for 16 weeks. “We improved the study design to give us a greater probability of being successful,” Mitchell says.
Lux is testing voclosporin in conjunction with oral steroids, which are commonly prescribed to patients with uveitis. Because high doses of steroids are dangerous—they can cause adverse events ranging from diabetes to osteoporosis—ophthalmologists who treat the disease try to keep patients on the tiniest doses possible. “Our idea is by adding voclosporin, you can taper the steroids down,” Mitchell says.
Upon its founding, Lux raised $36 million from HBM Partners, Novo, Prospect Venture Partners, and SV Life Sciences. It raised a $50 million Series B round led by those investors in 2009. Mitchell says Lux’s investors recently put an undisclosed amount of funding into the company that would take it through FDA approval. After the new trial is completed, the company will weigh its options, which could range from inking commercialization partnerships to getting acquired outright, he says.
Investors, both private and corporate, have taken notice of ophthalmology of late—a trend that could help Lux as Mitchell’s team goes on the hunt for more funding. Last fall, New York-based biotech startup Imagen, which is developing a drug to treat age-related macular degeneration, raised $40 million in a Series A financing led by SV Life Sciences, Fidelity Biosciences, and Novo. Seattle-based Acucela has also raised $40 million and is gearing up for a late-stage trial in macular degeneration. Several Big Pharma companies, including Sanofi and GlaxoSmithKline, have been investing in eye products, too.
Mitchell saw firsthand how hot ophthalmology drug development can be, when, earlier this year, Bausch & Lomb agreed to buy cataract drugmaker Ista Pharmaceuticals for $500 million. Mitchell was a member of Ista’s board of directors. “It’s very clear there’s been quite a lot of consolidation of small ophthalmology companies,” Mitchell says. “I think if we choose to go in that direction, there will be some interest.”
Mitchell believes hunkering down for two years to complete the new trial was the best possible path Lux could have taken. “The company had a pretty high profile. There were expectations of approvals and discussions with other companies about potential partnerships,” he says. “I felt we needed to pull back from all that and get our act together.”