August 9, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Aldeyra Therapeutics , headquartered in Lexington, Massachusetts, announced positive data from a clinical trial of its topical NS2 to treat skin conditions caused by Sjogren-Larsson Syndrome (SLS).
SLS is a rare disease caused by a problem with aldehyde metabolism. It results in mental retardation, spastic paraparesis, macular dystrophy, leukoencephalopathy, and ichthyosis. Ichthyosis is a dermatologic disease that results in dry, thickened, scaly skin. NS2 is being tested to treat ichthyosis in SLS patients.
The study was on 12 patients with SLS and moderate to severe ichthyosis. NS2 showed clinically meaningful results in cutting the severity of ichthyosis compared to patients treated with placebo.
“The results of this randomized and controlled clinical trial represent a significant advance in the effort to develop a treatment for SLS,” said William Rizzo, the principal investigator of the trial, in a statement. “The data from this trial suggest that NS2, when applied topically to the skin, has the potential to help patients suffering from SLS by lowering toxic aldehyde levels and preventing the dermal dysfunction that causes ichthyosis. I am excited about these results, and I am particularly excited for SLS patients and their caregivers.”
Aldeyra employs 12 people and has been noted for having Massachusetts’ smallest biotech initial public offering (IPO) in recent years. In May 2014, when the company had only two employees, it raised $12 million.
Aldeyra outsources all its laboratory and development work and labors to keep costs down. It has spent $19 million over the past two calendar years. It has conducted two previous clinical trials, both mid-stage studies, to test NS2 formulated as an eye drop in 100 patients with allergic conjunctivitis and 45 patients with anterior uveitis.
“This is our third trial (reported this year), and it’s worked in all three,” said Todd Brady, the company’s chief executive officer, to the Boston Business Journal. Speaking about today’s announcement, he said, “We’re particularly thrilled because this is an indication with no approved therapies.”
NS2 binds, or “traps” aldehydes, which are toxic and involved in inflammatory pathways in a number of diseases. NS2 appears to decrease the aldehyde load, which decreases inflammation and has potential treatment in diseases where aldehyde metabolism is not working properly.
Aldeyra is currently trading for $6.68 per share. The company is announcing its second-quarter financial results tomorrow.
Cowen and Company reiterated a “buy” rating for the company on Tuesday, May 10. Stifel Nicolaus issued a “buy” rating and a $16 price target on July 1. Aldeyra has an average “buy” rating and consensus price target of $13.13.
At this point, Brady told the Boston Business Journal that he plans to discuss development plans with the U.S. Food and Drug Administration (FDA). It’s not clear if the 12-patient study, given the rarity of the disease, will be enough to apply for regulatory approval. He also indicated he plans to talk to European regulators. SLS is more common in Sweden and Norway. And Aldeyra has plans to start a trial for an oral formulation of the drug next year, which it hopes might be effective in treating neurological components of the disease, not just dermatological symptoms.
