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Applied Therapeutics, Inc. (Nasdaq: APLT) (the Company), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today announced that the Company completed a meeting with the U.S. Food and Drug Administration (FDA) to discuss the potential new drug application (NDA) submission for govorestat for the treatment of Charcot-Marie-Tooth Sorbitol Dehydrogenase (SORD) Deficiency (CMT-SORD) in the third quarter of 2025.
The Company is awaiting the receipt of official meeting minutes from the FDA in order to determine next steps. At this time, a path forward regarding a potential submission strategy for regulatory approval, including under the accelerated approval pathway, has not been determined.
The Company remains committed to addressing the unmet needs of patients living with CMT-SORD.
About Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD)
CMT-SORD is a rare, progressive, debilitating hereditary neuropathy that affects peripheral nerves and motor neurons. CMT-SORD is one of the most common forms of recessive hereditary neuropathy and affects approximately 3,300 patients in the U.S. and 4,000 patients in the EU. The disease is caused by a lack of the enzyme sorbitol dehydrogenase, responsible for the metabolism of sorbitol, which causes sorbitol to accumulate at high levels in blood and tissues. Intracellular sorbitol accumulation is implicated in significant disability, loss of sensory function, neuromuscular dysfunction, and decreased mobility.
About Govorestat (AT-007)
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and CMT-SORD. Govorestat has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia, phosphomannomutase 2 deficiency, and CMT-SORD; Rare Pediatric Disease designation for Galactosemia and PMM2-CDG; and Fast Track designation for Galactosemia.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company committed to the development of novel drug candidates against validated molecular targets in rare diseases. The Company's lead drug candidate, govorestat, is a novel central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
To learn more, please visit www.appliedtherapeutics.com and follow the company on X at @Applied_Tx.
Forward-Looking Statements
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding the strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," "predicts" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding the Company's plans for govorestat for the treatment of CMT-SORD, including expected interactions and submissions with the FDA and potential eligibility for accelerated approval pathway. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, (i) our plans to develop, market and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms and advance product candidates into, and successfully complete, clinical studies, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain and timing of regulatory approval of our current and future product candidates, (viii) the anticipated indications for our product candidates, if approved, (ix) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (x) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (xi) the implementation of our business model and strategic plans for our business and product candidates, (xii) our intellectual property position and the duration of our patent rights, (xiii) developments or disputes concerning our intellectual property or other proprietary rights, (xiv) our expectations regarding government and third-party payor coverage and reimbursement, (xv) our ability to compete in the markets we serve, (xvi) the impact of government laws and regulations and liabilities thereunder, (xvii) developments relating to our competitors and our industry, (xviii) our ability to achieve the anticipated benefits from the agreements entered into in connection with our partnership with Advanz Pharma and (xix) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including those described in the "Risk Factors" section contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
Julie Seidel / Andrew Vulis
(212) 600-1902
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
