Aldeyra Therapeutics, Inc. Receives Orphan Drug Designation from the U.S. Food and Drug Administration for ADX-102 in Sjögren-Larsson Syndrome
"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
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.
About
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
Safe Harbor Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding Aldeyra's plans for its product candidates. Aldeyra intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as "may," "might," "will," "objective," "intend," "should," "could," "can," "would," "expect," "believe," "anticipate," "project," "target," "design," "estimate," "predict," "potential," "aim," "plan" or the negative of these terms, and similar expressions intended to identify forward-looking statements. Such forward-looking statements are based upon current
expectations that involve risks, changes in circumstances, assumptions and uncertainties. Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. Important factors that could cause actual results to differ materially from those reflected in Aldeyra's forward-looking statements include, among others, the timing of enrollment, commencement and completion of Aldeyra's clinical trials, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; the ability to obtain and maintain regulatory approval to conduct clinical trials and to commercialize Aldeyra's product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Aldeyra's product candidates; the size and growth of the potential markets for
Aldeyra's product candidates and the ability to serve those markets; Aldeyra's expectations regarding Aldeyra's expenses and revenue, the sufficiency of Aldeyra's cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra's product candidates; Aldeyra's expectations regarding competition; Aldeyra's anticipated growth strategies; Aldeyra's ability to attract or retain key personnel; Aldeyra's ability to establish and maintain development partnerships; Aldeyra's expectations regarding federal, state and foreign regulatory requirements; regulatory developments in
In addition to the risks described above and in Aldeyra's other filings with the
Corporate Contact:
Tel: +1 781-761-4904 Ext. 205
Email Contact
Investor Contact:
Tel: 339-970-2843
Email Contact
Media Contact:
781-591-3443
Email Contact
Source:
News Provided by Acquire Media