Step by step, a small biotech called Aldeyra Therapeutics has been trying to build a case for a new type of inflammation-fighting medicine. Data released this morning by the Lexington, MA-based company continue to support the idea.
Aldeyra said its drug, a medicine called NS2 meant to “trap” toxic aldehyde molecules, succeeded in a mid-stage trial in patients with a rare skin condition called Sjogren Larsson Syndrome. The disease causes neurological and eye problems and what’s known as ichthyosis—dry, rough, scaly skin that can lead to a number of medical complications like infections and chronic blistering, according to the Foundation for Ichthyosis & Related Skin Types. There are no FDA-approved treatments for SLS.
SLS is thought to arise due to abnormally high levels of toxic aldehydes, which are common metabolic byproducts. Normally, enzymes in the body chew up aldehydes and make acids or some other product, but when they erroneously build up in the body, they help drive an inflammatory response. Aldeyra uses small molecules to bind to these aldehydes—Aldeyra calls its molecules aldehyde “traps”—and degrade them. In theory, that normalizes aldehyde levels and reduces inflammation.
Aldeyra has tested this concept in two other mid-stage studies, for the eye diseases allergic conjunctivitis and noninfectious anterior uveitis. Both studies, which used an eye drop formulation of NS2, were successful. These trials are part of a fact-finding mission for Aldeyra to see how broad of an opportunity it might have with aldehyde traps. As CEO Todd Brady (pictured) told Xconomy earlier this year, the big idea, ultimately, is to treat broad, inflammatory conditions—perhaps, for instance, to supplement corticosteroids and allow for a lower dose and less troublesome side effects.
That’s a long way off, but today Aldeyra took another step forward: a skin-cream formulation of NS2 led to a statistically significant reduction in the severity of SLS patients’ ichthyosis in a small mid-stage trial, compared to a placebo. The trial enrolled a total of 12 patients with SLS and moderate to severe ichythosis. Half of them were given NS2 cream once a day for two months on a small area of skin, the other half a placebo. All six patients on NS2 improved over the course of treatment, and the treated skin areas for five out of six patients were assessed by an independent reviewer as “almost clear” or “mild.” There were no significant side effects, Aldeyra said.
Shares of Aldeyra (NASDAQ: ALDX) surged more than 29 percent, to $7.55 apiece, in pre-market trading. Still, the company trades below its $8 IPO price in May 2014. Domain Associates and Johnson & Johnson, which largely funded Aldeyra before it went public, remained its largest shareholders as of an April proxy filing, with ownership stakes of 20.51 percent and 18.02 percent, respectively.