Aldeyra Therapeutics Receives Orphan Drug Designation From US Food and Drug Administration For ADX-2191 To Treat Primary Vitreoretinal Lymphoma
LEXINGTON, Mass .– (COMMERCIAL THREAD) –Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the United States Food and Drug Administration (FDA) has granted the company orphan drug designation for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). There is no approved treatment for PVRL, a rare, aggressive, high-grade cancer that affects approximately 2,800 people in the United States, with approximately 600 new cases diagnosed each year.
“PVRL is an often fatal cancer that creates unique challenges for diagnosis and treatment, ”said Todd C. Brady, MD, Ph.D., President and CEO of Aldeyra. “The FDA’s orphan drug designation promotes an important clinical development and commercialization pathway for ADX-2191, which is the first methotrexate preparation specifically formulated for intraocular injection. This designation is another important milestone for our retinal disease control program, complementing our ongoing clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy, the leading cause of related surgery failure. of the retina.
The FDA Orphan Drug Designation Program is designed to provide financial incentives to sponsors for the development of drugs and biologics for rare diseases and conditions, in part defined as affecting less than 200,000 people in the United States. Designated orphan drug sponsors are eligible for tax credits for clinical trial costs, user fee waivers for marketing applications and, upon approval, a seven-year marketing exclusivity.
About the ADX-2191
ADX-2191 (methotrexate for intravitreal injection) inhibits dihydrofolate reductase, an enzyme involved in cell replication and activation. Methotrexate is the most common intravitreal drug used for the treatment of PVRL. ADX-2191 previously achieved orphan drug status and FDA expedited designation for the prevention of proliferative vitreoretinopathy, a rare but serious vision-threatening retinal disease without approved treatment.
About Aldeyra Therapeutics
Aldeyra Therapeutics is a biotechnology company developing new immunomodulatory therapies to treat eye and systemic diseases. Two of the company’s main product candidates, reproxalap and ADX-629, target RASPs (reactive aldehyde species), which are inflammatory mediators based on pre-cytokine systems. Reproxalap is being evaluated in phase 3 clinical trials in patients with dry eye and allergic conjunctivitis. The company’s clinical pipeline also includes ADX-2191 (methotrexate for intravitreal injection), a phase 3 drug candidate for proliferative vitreoretinopathy. For more information visit https://www.aldeyra.com/ and follow us on LinkedIn, Facebook, and Twitter.
Safe Harbor Declaration
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding Aldeyra’s plans and expectations for its product candidates, including ADX-2191. Aldeyra intends that such forward-looking statements 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 instances, you may identify forward-looking statements by terms such as, but not limited to, “could”, “could”, “will”, “objective”, “intention”, “should”, “could”, “could”, “” believe ” , “anticipate”, “project”, “on track”, “planned”, “target”, “design”, “estimate”, “predict”, “potential”, “aim”, “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These forward-looking statements are based on current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Aldeyra is at an early stage of development and may not never have products generating r important events. All Aldeyra development schedules may be subject to adjustment based on recruitment rate, regulatory review, preclinical and clinical results and other factors that may delay the initiation or completion of clinical trials. Important factors that could cause actual results to differ materially from those reflected in Aldeyra’s forward-looking statements include, but are not limited to, the timing of recruitment, the commencement and termination of Aldeyra’s clinical trials, the timing and successful preclinical studies and clinical trials conducted by Aldeyra and its development partners; updated or refined data based on Aldeyra’s continuous review and quality control analysis of clinical data, Aldeyra’s ability to design clinical trials with acceptable protocols and endpoints for applicable regulatory authorities; delay or failure in obtaining regulatory approval for Aldeyra’s product candidates; the ability to maintain regulatory approval of Aldeyra’s product candidates and the labeling of all approved products; the risk that previous results, such as signals of safety, potency or durability of effect, observed in preclinical or clinical trials, may not be reproduced or continue in studies or trials ongoing or future clinical trials involving Aldeyra’s product candidates in clinical trials focusing on the same or different indications; the risk that the results of smaller clinical trials or parts of clinical trials will not accurately predict the results of larger trials or the remainder of a clinical trial; the scope, progress, expansion and costs of the development and commercialization of Aldeyra’s product candidates; uncertainty as to Aldeyra’s ability to market (alone or with others) Aldeyra’s product candidates after regulatory approval, if applicable; the size and growth of potential markets and prices for Aldeyra’s product candidates and the ability to serve these markets; Aldeyra’s expectations regarding Aldeyra’s expenses and income, the sufficiency or use of Aldeyra’s cash resources and additional financing requirements; political, economic, legal, social and health risks, including the recent COVID-19 outbreak and subsequent public health measures, which may affect Aldeyra’s business or the global economy; the rate and degree of market acceptance of any of Aldeyra’s product candidates; Aldeyra’s competition expectations; Aldeyra’s anticipated growth strategies; Aldeyra’s ability to attract or retain key personnel; Aldeyra’s limited sales and marketing infrastructure; Aldeyra’s ability to establish and maintain development partnerships; Aldeyra’s ability to successfully integrate acquisitions into its business; Aldeyra’s expectations regarding federal, state and foreign regulatory requirements; regulatory developments in the United States and in foreign countries; Aldeyra’s ability to obtain and maintain intellectual property protection for its product candidates; trends and anticipated challenges in Aldeyra’s business and the market in which it operates; and other factors described in “Risk Factors” and “Management’s discussion and analysis of the financial position and results of operations “of Aldeyra’s annual report on Form 10-K for the fiscal year ended December 31, 2020 and Form 10-Q for the quarter ended December 31 March 2021, which are filed with the Securities and Exchange Commission (SEC) and available on the SEC’s website at https://www.sec.gov/. Additional factors may be set out in sections of Aldeyra’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, which are expected to be filed with the SEC in the third quarter of 2021.
In addition to the risks described above and in other documents filed by Aldeyra with the SEC, other unknown or unforeseeable factors could also affect Aldeyra’s results. No forward-looking statement can be guaranteed and actual results may differ materially from such statements. The information contained in this press release is provided only as of the date of this press release, and Aldeyra does not undertake to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise, except if the law requires it.