Assistant Managing Editor

With simultaneous filings for uveitis drug Luveniq in the U.S. and Europe, Lux Biosciences Inc. has done what few small, private biotechs have managed in today's tough economy: get a drug to market without the help of public funding or a big pharma partner.

Of course, it hasn't reached market yet. But Jersey City, N.J.-based Lux plans to request a priority review for Luveniq in the U.S., where it has both orphan and fast-track status for treating noninfectious uveitis that affects the intermediate or posterior segments of the eye.

"We're hoping for approval in Q3," Ulrich Grau, president and CEO, told BioWorld Today, adding that "we have funding to get us through that approval milestone."

After that, the company will have to seek additional funding. "We'll be exploring options," he said.

Those could include private or public financings or corporate partnerships.

But getting to market alone would be no small feat. In fact, BioWorld Insight recently reported that only one private firm in five years had been able to pull that off. Cambridge, Mass.-based Gloucester Pharmaceuticals Inc. made it all the way through approval with Istodax (romidepsin) for cutaneous T-cell lymphoma with about $100 million in venture money.

Shortly after Istodax cleared the FDA, Gloucester was snatched up by Summit, N.J.-based Celgene Corp. for $640 million. (See BioWorld Today, Dec. 8, 2009.)

Like Gloucester, Lux attributed its success to a very efficient use of capital. Since its inception in 2005, Lux has raised just under $100 million in venture capital, most recently closing on an impressive $50 million Series B round in October to support regulatory filings of Luveniq and commercial launch preparations. (See BioWorld Today, Oct. 20, 2009.)

The filings alone were a big task for a small firm. Grau said submissions comprised about 1 million pages, and filing simultaneously in the U.S. and Europe was "a bit of a challenge."

Lux was helped some by Isotechnika Inc., from which it licensed rights to Luveniq in ophthalmic indications. Isotechnika, of Edmonton, Alberta, retained rights to the drug, a next-generation calcineurin inhibitor known as voclosporin, in autoimmune indications such as psoriasis and organ transplant rejection.

The FDA allowed Lux to submit safety data from Isotechnika's psoriasis trials as part of the Luveniq application. "So our safety database includes 2,110 patients, which is unusually large given that uveitis is a small indication," Grau said.

Upon FDA approval, Lux would owe Isotechnika a $7.1 million milestone payment. An additional $3.5 million payment would be triggered by a nod from European regulators.

Efficacy data came from Lux's LUMINATE clinical program, which included two randomized trials involving different uveitis populations.

Results from the first study, designated LX211-01, enrolled subjects with uncontrolled uveitis. Data showed that, at the recommended dose of 0.4 mg/kg twice daily, Luveniq rapidly reduced inflammation in patients with moderately severe disease, either alone or in combination with systemic corticosteroids.

Those receiving the drug had a 50 percent reduction in mean vitreous haze vs. 29 percent in the placebo arm, and the addition of Luveniq also allowed for the withdrawal of immunosuppressive therapy and lessened the use of daily prednisone treatment.

Similar data emerged in the second trial, LX211-02, which tested the drug in patients whose disease was clinically quiescent at the time of enrollment. Again, data showed a 50 percent reduction in the rate of inflammatory exacerbations at the 26-week primary endpoint compared to those on placebo.

Uveitis affects about 300,000 people in the U.S., with about 10 percent of those suffering blindness. So "it's a very major medical need," Grau said, particularly the form of the disease that involves the posterior segment of the eye.

Topical drugs are not effective, so patients often have to resort to oral corticosteroids or methotrexate, or require actual injections into the eye, he said. But Luveniq could reduce inflammation, preserve vision and help eliminate the need for corticosteroid treatment.

Earlier in its pipeline, Lux is working on a nanomicellar formulation of voclosporin for dry eye syndrome, with a Phase I trial completed last year, and may look at moving into other chronic inflammatory ocular diseases.

Published  February 5, 2010