Applied Therapeutics Stock Price, News & Analysis

Applied Therapeutics, Inc. (Nasdaq: APLT) is a clinical-stage biopharmaceutical company focused on the development of novel drug candidates against validated molecular targets in rare diseases. The company concentrates on central nervous system (CNS) rare metabolic diseases and other rare conditions, aiming to advance therapies where there is significant unmet medical need.

Applied Therapeutics’ lead drug candidate is govorestat (AT-007), described by the company as a novel CNS-penetrant Aldose Reductase Inhibitor (ARI). Govorestat is being developed 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). Across company disclosures, Applied Therapeutics consistently characterizes govorestat as its lead program and a central focus of its clinical and regulatory strategy.

Core focus on rare metabolic and genetic diseases

According to multiple company statements, Applied Therapeutics is dedicated to creating transformative treatments for rare diseases. Its work centers on rare metabolic and hereditary conditions where validated molecular targets have been identified. In CMT-SORD, the company notes that govorestat is being evaluated in patients with a rare, progressive hereditary neuropathy associated with sorbitol accumulation. In Classic Galactosemia and PMM2-CDG, Applied Therapeutics highlights govorestat as a potential treatment option within severe rare disease settings.

The company reports that govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for Galactosemia and CMT-SORD, and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Galactosemia, phosphomannomutase 2 deficiency, and CMT-SORD. Govorestat has also been granted Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia, reflecting regulatory recognition of the seriousness of these conditions and the unmet medical need.

Clinical development of govorestat

Applied Therapeutics describes govorestat as being evaluated in late-stage clinical programs. The company has reported data from the INSPIRE Phase 2/3 (also described as registrational Phase 3) trial in patients with CMT-SORD, a subtype of Charcot-Marie-Tooth disease caused by sorbitol dehydrogenase deficiency. In its communications, the company notes that govorestat has been generally safe and well tolerated in the INSPIRE study, with statistically significant lowering of blood sorbitol levels and statistically significant improvements in certain secondary and patient-reported outcome measures at 12 months, as well as maintenance of effects and observations related to slowing of disease progression via MRI at 24 months in treated patients.

Applied Therapeutics has also discussed an open-label extension of the INSPIRE trial, indicating that a substantial proportion of remaining patients elected to continue on govorestat treatment. The company states that it has engaged with the FDA via Type C meetings regarding the potential regulatory path for govorestat in CMT-SORD, including discussion of possible New Drug Application (NDA) strategies and endpoints.

In Classic Galactosemia, the company has disclosed that it previously submitted an NDA for govorestat and received a Complete Response Letter (CRL) and a warning letter from the FDA. Applied Therapeutics reports that it continues to evaluate its response to the CRL and related regulatory feedback, and has sought or planned additional FDA interactions to discuss the path forward for govorestat in this indication.

For PMM2-CDG, Applied Therapeutics has highlighted results from a single-patient, investigator-initiated trial conducted under an expanded access IND. The company reports that treatment with govorestat in this setting was well tolerated, with no adverse effects associated with treatment reported, and that dose-dependent decreases in whole blood sorbitol and improvements in clinical rating scales were observed. These data have been described as supporting continued clinical development in PMM2-CDG.

Additional pipeline and partnering activity

Beyond govorestat, Applied Therapeutics has referenced other drug candidates in its pipeline. Earlier descriptions and subsequent company announcements identify AT-001 as an investigational, novel Aldose Reductase Inhibitor developed as an oral therapy for diabetic cardiomyopathy. In a later corporate update, the company states that it entered into an out-licensing agreement with Biossil, Inc. for AT-001, granting Biossil exclusive worldwide rights to develop and commercialize AT-001, excluding a select number of rare disease indications. Under that agreement, Applied Therapeutics reports that it received an upfront payment and is eligible for potential future milestone and royalty payments.

Earlier company descriptions also refer to AT-003 as a candidate for diabetic retinopathy. Across its communications, Applied Therapeutics consistently frames its research and development efforts as focused on novel drug candidates targeting validated molecular pathways in indications of high unmet need, with a particular emphasis on rare diseases and metabolic or neurologic conditions.

Strategic review and planned acquisition

In a corporate update, Applied Therapeutics announced that its Board of Directors initiated a process to explore strategic alternatives, including potential mergers, acquisitions, partnerships, licensing arrangements, or other strategic transactions, with the stated objective of maximizing shareholder value and supporting the advancement of govorestat. In connection with this review, the company disclosed workforce reductions and other cost-containment and cash conservation measures.

Subsequently, the company reported that it entered into an Agreement and Plan of Merger with Cycle Group Holdings Limited and an indirect wholly owned subsidiary of Cycle. Under this agreement, Cycle’s subsidiary is expected to commence a tender offer to acquire all outstanding shares of Applied Therapeutics common stock for a specified cash amount per share plus a non-tradeable contingent value right (CVR). The CVR structure described by the company provides for potential additional cash payments tied to future regulatory approvals for govorestat in specified indications and to achieving certain net sales milestones, as well as a potential closing cash distribution, all subject to detailed conditions.

The company states that, if the tender offer is successfully completed and related conditions are met, Applied Therapeutics would be acquired by Cycle through a second-step merger, with Applied Therapeutics surviving as a wholly owned subsidiary. The Board of Directors of Applied Therapeutics unanimously approved the merger agreement and recommended that stockholders tender their shares in the offer, based on its evaluation of strategic alternatives and assessment of the transaction terms.

Capital markets and listing status

Applied Therapeutics’ common stock trades under the symbol APLT on The Nasdaq Stock Market. The company has disclosed that it received a Nasdaq notice regarding non-compliance with the minimum bid price requirement and that it was granted an additional compliance period, with its listing transferred from the Nasdaq Global Market to the Nasdaq Capital Market. The company has indicated that it would consider options, such as a potential reverse stock split, to address bid price deficiencies, while noting that there can be no assurance of regaining compliance or avoiding delisting.

In addition, Applied Therapeutics has reported entering into at-the-market (ATM) equity offering arrangements with sales agents, allowing it to issue and sell shares of common stock from time to time under an effective shelf registration statement, and has disclosed the termination of a prior ATM agreement in connection with entering a new sales agreement.

Business model and sector context

Based on its public disclosures, Applied Therapeutics operates as a clinical-stage biopharmaceutical company within the broader biotechnology and life sciences ecosystem. Its activities are centered on research and development, clinical trials, regulatory interactions, and partnering or licensing arrangements rather than on commercial product sales. The company’s revenue to date has included research and development services revenue and license-related revenue, while it reports significant research and development and general and administrative expenses typical of a company in clinical development.

Applied Therapeutics positions itself within the rare disease segment, emphasizing CNS rare metabolic diseases and other rare hereditary conditions. The company’s communications repeatedly stress its focus on validated molecular targets and on diseases with high unmet medical need, reflecting a strategy of concentrating on specialized indications where targeted therapies and regulatory designations such as orphan and rare pediatric disease status can be important elements of the development and potential commercialization pathway.

Geographic and organizational footprint

Applied Therapeutics identifies itself as a U.S.-based company, with SEC filings listing a principal office in New York, New York. The company’s disclosures reference interactions with U.S. and European regulators, including the FDA and EMA, reflecting a development strategy that spans major regulatory jurisdictions for rare disease therapies.

APLT stock: what investors track

Investors following APLT stock typically monitor Applied Therapeutics’ progress in several key areas described in its public statements: clinical trial outcomes for govorestat in CMT-SORD, Classic Galactosemia, and PMM2-CDG; regulatory interactions and potential NDA submissions or responses; the evolution and potential completion of the planned acquisition by Cycle Group Holdings Limited; the company’s capital structure and financing activities; and its ongoing ability to meet Nasdaq listing requirements. As a clinical-stage biopharmaceutical issuer, Applied Therapeutics’ valuation and risk profile are closely tied to clinical, regulatory, and strategic transaction milestones it reports.

FAQs about Applied Therapeutics, Inc. (APLT)

• What does Applied Therapeutics do?
  Applied Therapeutics is a clinical-stage biopharmaceutical company that states it is committed to developing novel drug candidates against validated molecular targets in rare diseases, with a primary focus on CNS rare metabolic diseases and other rare conditions.
• What is govorestat (AT-007)?
  Govorestat, also referred to as AT-007, is the company’s lead drug candidate. Applied Therapeutics describes it as a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) being developed for CNS rare metabolic diseases, including Classic Galactosemia, CMT-SORD, and PMM2-CDG.
• Which indications is govorestat being developed for?
  According to the company, govorestat is being developed for Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), all of which are rare diseases with significant unmet medical needs.
• What regulatory designations has govorestat received?
  Applied Therapeutics reports that govorestat has received Orphan Medicinal Product Designation from the EMA for Galactosemia and CMT-SORD, Orphan Drug Designation from the FDA for Galactosemia, phosphomannomutase 2 deficiency, and CMT-SORD, Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.
• What is the INSPIRE trial?
  The INSPIRE trial is described by Applied Therapeutics as a randomized, double-blind, placebo-controlled Phase 2/3 or registrational Phase 3 study evaluating govorestat in patients with CMT-SORD. The company has reported 12-month, 18-month, and 24-month data from INSPIRE, including safety, sorbitol reduction, functional outcomes, and MRI-based assessments of disease progression.
• What happened with the Classic Galactosemia NDA?
  Applied Therapeutics has disclosed that it received a Complete Response Letter (CRL) and a warning letter from the FDA in response to its NDA for govorestat in Classic Galactosemia. The company states that it is evaluating the FDA’s feedback and considering next steps, including potential additional meetings with the agency.
• What is AT-001 and what is its status?
  AT-001 has been described as an investigational, novel Aldose Reductase Inhibitor developed as an oral therapy for diabetic cardiomyopathy. Applied Therapeutics later announced an out-licensing agreement granting Biossil, Inc. exclusive worldwide rights to develop and commercialize AT-001, excluding certain rare disease indications, in exchange for an upfront payment and eligibility for future milestones and royalties.
• Is Applied Therapeutics being acquired?
  The company has announced that it entered into an Agreement and Plan of Merger with Cycle Group Holdings Limited and an indirect wholly owned subsidiary of Cycle. Under this agreement, a tender offer is expected to be commenced to acquire all outstanding shares of Applied Therapeutics common stock for cash plus a non-tradeable contingent value right. Completion of the transaction is subject to specified conditions, and the company has indicated that the transaction is expected to close following satisfaction of those conditions.
• Where is Applied Therapeutics listed?
  Applied Therapeutics’ common stock is listed on The Nasdaq Stock Market under the ticker symbol APLT. The company has reported that its listing was transferred from the Nasdaq Global Market to the Nasdaq Capital Market in connection with an extension of time to regain compliance with Nasdaq’s minimum bid price requirement.
• How does Applied Therapeutics generate revenue at this stage?
  As a clinical-stage company, Applied Therapeutics reports that its primary activities are research and development and related corporate functions. Its financial disclosures indicate that revenue to date has included license revenue and research and development services revenue, while it incurs substantial research and development and general and administrative expenses.
• What sector and industry does Applied Therapeutics belong to?
  Applied Therapeutics operates in the biotechnology and biopharmaceutical space. It can be classified within research and development in biotechnology and within the broader professional, scientific, and technical services sector.