Aldeyra Therapeutics Confirms FDA Orphan Drug Designation for ADX-102 in Sjogren-Larsson

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Aldeyra Therapeutics, Inc. , a clinical-stage biotech company devoted to treating diseases thought to be related to endogenous aldehydes.

A naturally occurring class of pro-inflammatory and toxic molecules announced that the United States FDA has granted Aldeyra's novel compound ADX-102 orphan drug designation for the treatment of congenital ichthyosis, a severe skin disease characteristic of Sjögren-Larsson Syndrome (SLS).

There are no FDA-approved therapies specifically indicated for the treatment of SLS, and ADX-102 is believed to be the only potential SLS therapy in clinical development.

"The skin disease associated with SLS is physically and emotionally debilitating, leading to considerable social stigma and requiring significant daily care and medical resources," said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. "Receipt of orphan drug designation by the FDA is great step forward for both Aldeyra and patients suffering from this rare and difficult-to-treat disease."

SLS is caused by genetic mutations that lead to high levels of toxic aldehydes. By sequestering and facilitating the degradation of aldehydes, ADX-102 represents a novel, mechanistically directed potential therapy for SLS. In August of 2016, Aldeyra announced that ADX-102 improved ichthyosis in SLS patients in a randomized, double-blind, vehicle-controlled Phase 2 clinical trial in which ADX-102 was administered to skin for two months. Improvement from baseline was clinically and statistically significant, and the magnitude of improvement was statistically superior to that of vehicle treatment. A global, double-blind, pivotal Phase 3 study is expected to begin later this year. Future clinical trials of ADX-102 or other novel aldehyde traps may involve oral administration as a potential treatment for the neurological aspects of the disease.

The FDA Office of Orphan Products Development designates orphan status to drugs intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 people in the United States. Receiving Orphan Drug Designation provides Aldeyra with multiple benefits, including waiver of the Prescription Drug User Fee, post-approval marketing exclusivity for seven years, research tax credits, and assistance during the marketing registration process.

About Aldeyra Therapeutics
Aldeyra Therapeutics, Inc. is a biotechnology company devoted to improving lives by inventing, developing and commercializing products that treat diseases thought to be related to endogenous aldehydes, a naturally occurring class of pro-inflammatory and toxic molecules. Aldeyra's lead product candidate, ADX-102, is an aldehyde trap in development for ocular inflammation, as well as for Sjögren-Larsson Syndrome and Succinic Semi-Aldehyde Dehydrogenase Deficiency, two inborn errors of aldehyde metabolism. ADX-102 has not been approved for sale in the U.S. or elsewhere.

About Sjögren-Larsson Syndrome
Sjögren-Larsson Syndrome is a rare inborn error of aldehyde metabolism caused by mutations in fatty acid aldehyde dehydrogenase, leading to elevated toxic fatty aldehyde levels that are thought to contribute to severe ichthyosis (scaly, thickened, dry skin), neurological disorders, and retinal disease. There is no therapy for SLS that has been approved by the U.S. Food and Drug Administration.

Corporate Contact:
Stephen Tulipano
Aldeyra Therapeutics, Inc.
Tel: +1 781-761-4904 Ext. 205
Email Contact

Investor Contact: 
Chris Brinzey
Westwicke Partners
Tel: 339-970-2843

Media Contact:
Cammy Duong 
MacDougall Biomedical Communications 
781-591-3443