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Here are 1-2 brief analogies for Applied Therapeutics (APLT):

• A clinical-stage drug developer, like a younger, independent version of a biotech giant such as Amgen or Gilead, focused on getting novel medicines through clinical trials.
• An independent, early-stage R&D company in the pharmaceutical space, similar to a specialized drug discovery unit that a large pharma like Merck or Pfizer might have, but focused on its own pipeline.

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• Gavestinel (AT-007) for Classic Galactosemia: An investigational aldose reductase inhibitor designed to prevent and treat neurological complications in patients with Classic Galactosemia.
• Gavestinel (AT-007) for SORD Deficiency: An investigational aldose reductase inhibitor aimed at treating peripheral neuropathy in patients with Sorbitol Dehydrogenase (SORD) Deficiency.

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Applied Therapeutics (APLT) is a clinical-stage biopharmaceutical company primarily focused on the research and development of novel drug candidates. As such, the company has not yet commercialized any products.

According to its latest financial filings, including the 2023 10-K filed with the U.S. Securities and Exchange Commission, Applied Therapeutics has not generated any revenue from product sales. Therefore, APLT does not currently have "major customers" in the traditional sense of companies or individuals purchasing its commercialized products or services.

The company's activities are centered on advancing its drug pipeline through clinical trials. Any funding or revenue generated at this stage typically comes from sources such as equity financing, research grants, or potential collaboration and licensing agreements with larger pharmaceutical partners, rather than from commercial product sales to customers.

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Les Funtleyder Interim Chief Executive Officer and Chief Financial Officer
Les Funtleyder has served as Interim Chief Executive Officer and Chief Financial Officer of Applied Therapeutics since November 2023. He also serves as a healthcare portfolio manager at E Squared Capital Management, a role from which he is currently taking a sabbatical. His previous experience includes serving as the director of strategic investments for Opko Health and holding roles as Strategist and Portfolio Manager for Miller Tabak Health Transformation Fund, Provident Advisors, and UBS Warburg. Funtleyder is the author of "Healthcare Investing: Profiting from the New World of Pharma, Biotech, and Health Care Services" and is an adjunct professor of public health at Columbia University. He currently sits on the boards of HealthEngine, Reviva Pharmaceuticals, Regain Lasers, Aceso Global (an advisory firm for health systems), and Zentynel (a South American life sciences venture fund).

John H. Johnson Executive Chairman
John H. Johnson is a recognized leader in the pharmaceutical and biotechnology industry, bringing 40 years of transformational leadership experience to Applied Therapeutics. He has held positions at global healthcare organizations such as Johnson & Johnson, Eli Lilly & Company, ImClone, and Pfizer, Inc. Johnson has a multi-decade track record of implementing turnaround plans to enable growth and value creation for shareholders and previously served as the CEO of Reaction Biology.

Corwin Dale Hooks Chief Commercial Officer
Corwin Dale Hooks brings over three decades of experience in the biopharmaceutical industry, with a particular focus on rare disease marketing and sales. He was appointed as Chief Commercial Officer at Applied Therapeutics.

Evan Prescott Bailey Chief Medical Officer
Evan Prescott Bailey was promoted to Chief Medical Officer of Applied Therapeutics, effective June 15, 2025. He previously served as Senior Vice President, Clinical Development at the company, having joined Applied Therapeutics approximately four years prior to his promotion. Dr. Bailey's background includes extensive experience in clinical development execution, encompassing both early and late-stage development programs, and he is also a practicing pediatrician.

Constantine Chinoporos Chief Operating Officer and Chief Business Officer
Constantine Chinoporos serves as both the Chief Operating Officer and Chief Business Officer for Applied Therapeutics.

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The key risks to Applied Therapeutics (APLT) are predominantly centered around its clinical-stage nature, requiring successful regulatory approvals and sustained financial viability.

1. Regulatory Approval and Clinical Trial Outcomes

   A primary risk for Applied Therapeutics stems from the inherent unpredictability and lengthy process of obtaining regulatory approvals for its product candidates. The company's business is significantly harmed if it fails to secure regulatory approval or marketing authorization from agencies like the FDA. This risk is underscored by the FDA's rejection of a New Drug Application (NDA) for govorestat in December 2024 for classic galactosemia, citing issues related to electronic data capture and a dosing error during a clinical study. Such setbacks can lead to substantial delays, increased costs, or even the complete failure of a product candidate. Furthermore, clinical trial results are subject to varying interpretations and may not always be conclusive, potentially leading to delays or preventing approval. In 2024, Applied Therapeutics also experienced a setback with its AT-001 (caficrestat) drug candidate for diabetic cardiomyopathy, which failed to meet its primary endpoint in a Phase 3 trial.

2. Financial Health and Liquidity

   Applied Therapeutics faces significant financial challenges, characterized by a history of substantial operating losses and minimal revenue generation since its inception. The company has demonstrated negative profitability metrics, including alarming operating and net margins, and has a distressed financial strength rating. Reports indicate a high risk of bankruptcy within the next two years, with its Altman Z-Score placing it in the distress zone. The company's cash and cash equivalents have been declining, raising concerns about its ability to fund ongoing operations and clinical trials without securing additional capital or partnerships. This financial instability led to the company evaluating strategic options and implementing a significant workforce reduction. A recent development includes a proposed acquisition by Cycle Group Holdings for a low cash price per share, further highlighting its constrained liquidity and the need for external financing to continue operations.

3. Dependence on a Limited Pipeline

   As a clinical-stage biopharmaceutical company, Applied Therapeutics is highly dependent on the successful development and eventual commercialization of a limited number of product candidates. Its pipeline includes AT-007 (govorestat) for rare metabolic diseases like galactosemia and CMT-SORD, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy. The failure of a single lead candidate, such as the negative FDA decision for govorestat in galactosemia, or the unsuccessful Phase 3 trial for AT-001, can have a disproportionately severe impact on the company's prospects and valuation due to the lack of diverse revenue-generating products to offset such setbacks.

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Applied Therapeutics (APLT) is a clinical-stage biopharmaceutical company developing novel product candidates for diseases with high unmet medical needs. Their main product candidates and their addressable markets are as follows:

Govorestat (AT-007)

• Indication: Classic Galactosemia
  • Addressable Patient Population: Approximately 3,300 patients in the U.S. and 4,400 patients in the EU.
  • Market Size (Global): The global Galactosemia treatment market was valued at USD 1.2 billion in 2024 and is projected to reach USD 2.1 billion by 2034. North America holds 42.3% of the global market share, and Europe is the fastest-growing region.
• Indication: Sorbitol Dehydrogenase (SORD) Deficiency
  • Addressable Patient Population: Approximately 3,500-5,000 patients in the U.S. and 4,000 patients in the EU.
• Indication: PMM2-Congenital Disorder of Glycosylation (PMM2-CDG)
  • Addressable Market: null

AT-001

• Indication: Diabetic Cardiomyopathy (DbCM)
  • Addressable Market: null (AT-001 failed to meet its primary endpoint in a Phase 3 trial, and the company is seeking a partner. There are currently no approved therapies for this indication.)

AT-003

• Indication: Diabetic Retinopathy
  • Addressable Market: null (This candidate is in early Phase 1 clinical studies.)

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Applied Therapeutics (APLT) is a clinical-stage biopharmaceutical company with several programs in development for rare diseases. Over the next 2-3 years, the company's future revenue growth is expected to be primarily driven by the following factors:

1. Commercialization of Govorestat (AT-007) for SORD Deficiency: Govorestat (AT-007) is in Phase 3 development for Charcot-Marie-Tooth Sorbitol Dehydrogenase (SORD) Deficiency. Positive 12-month interim data was reported in February 2024, and the company had an FDA meeting scheduled in Q3 2025 to discuss a potential New Drug Application (NDA) submission. Applied Therapeutics is focused on regulatory alignment and a near-term NDA submission for this indication, aiming to transition into a commercial organization. The company has also launched a sponsored Sorbitol Assay program to aid in patient identification, which could support market readiness.
2. Potential Future Approval of Govorestat (AT-007) for Classic Galactosemia: While the New Drug Application (NDA) for govorestat for Classic Galactosemia received a Complete Response Letter (CRL) from the FDA in November 2024, the company's Q2 2025 financial results (reported August 13, 2025) still mention an "ongoing review of govorestat development program for Classic Galactosemia progressing as planned." If Applied Therapeutics successfully addresses the issues raised in the CRL and secures regulatory approval, this could become a significant revenue driver, potentially by late 2026 or 2027, given the need to re-engage with the FDA.
3. Advancement of Govorestat (AT-007) for PMM2-CDG: Govorestat is also being developed for phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), an ultra-rare condition with no approved treatments. The program is Phase 2 ready, and results from an ongoing single-patient investigator-initiated trial were expected to be presented in October 2025, indicating continued progress that could lead to future revenue if successful.
4. Receipt of Pediatric Rare Disease Priority Review Voucher: If Govorestat (AT-007) is approved for Classic Galactosemia, it could garner a Pediatric Rare Disease Priority Review Voucher. These vouchers can be sold to other pharmaceutical companies, historically fetching significant sums (e.g., $100 million to $125 million), providing a substantial non-product revenue stream.

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Share Issuance

• In January 2020, Applied Therapeutics completed a public offering of 3,152,712 shares of common stock at $45.50 per share, generating approximately $143.4 million in gross proceeds.
• From August 2023 to August 2025, the company sold 20,615,976 shares through an "at the market" agreement with Leerink Partners LLC, resulting in approximately $49.3 million in net proceeds.
• In August 2025, Applied Therapeutics entered into a new "at the market" equity sales agreement with Cantor Fitzgerald & Co., allowing it to offer and sell shares of common stock with an aggregate offering price of up to $75 million.

Outbound Investments

• In July 2025, Applied Therapeutics entered into an out-licensing agreement with Biossil, Inc. for AT-001, granting Biossil exclusive worldwide rights to develop and commercialize AT-001 (excluding certain rare disease indications) in exchange for an upfront payment and eligibility for future royalties and milestone payments.
• The company also executed a commercial licensing arrangement with Advanz Agreement, which included an upfront payment of EUR 10.0M (approximately $10.7M) and potential future milestones of EUR 134M plus 20% royalties.