Applied Therapeutics, Inc. (APLT): History, Ownership, Mission, How It Works & Makes Money

Applied Therapeutics, Inc. (APLT) is a clinical-stage biopharma focused on rare diseases, but how do you assess a company that just posted a 719.7% revenue surge to $1 million in Q3 2025 while still operating with limited liquidity? Their core mission is advancing govorestat, an Aldose Reductase Inhibitor (ARI), for conditions like CMT-SORD and Classic Galactosemia, which represents a massive market opportunity if the drug clears regulatory hurdles. The reality is that despite narrowing their net loss by 72.3% to $18.99 million this quarter, their cash reserves stood at only $11.9 million as of September 30, 2025, so you defintely need to understand the foundational science and the critical Q4 2025 FDA meeting to map out the next move.

Applied Therapeutics, Inc. (APLT) History

You're looking for the bedrock of Applied Therapeutics, Inc., and honestly, it's a story of a classic biotech startup: big vision, a critical drug candidate, and a long, expensive road to regulatory approval. The company's journey, centered on developing small molecule drugs for rare metabolic diseases, is defined by a few key funding injections and a constant regulatory back-and-forth for its lead compound, govorestat (AT-007).

This is a clinical-stage biopharmaceutical company, meaning it makes money by raising capital and hitting milestones, not from product sales yet. To be fair, its trailing 12-month revenue as of June 30, 2025, was only about $122K, so capital is defintely the lifeblood right now.

Given Company's Founding Timeline

Year established

Applied Therapeutics, Inc. was founded in 2016.

Original location

The company is headquartered in New York, NY, United States.

Founding team members

The company was founded by Shoshana Shendelman, PhD, who has served as a key leader, including as Chairwoman and Chief Executive Officer during the company's evolution.

Initial capital/funding

While the precise seed capital isn't public, the company's first recorded funding event was in February 2017. The total capital raised historically is substantial, including a critical $100 million private placement in February 2024. The company went public on May 14, 2019, with an initial public offering (IPO) price of $10.00 per share.

Given Company's Evolution Milestones

Given Company's Transformative Moments

The company's trajectory is a clear example of the high-stakes, binary outcomes in clinical-stage biotech. The most transformative moments weren't just the large funding rounds, but the regulatory interactions that forced a strategic shift.

• The Galactosemia Regulatory Pivot: The FDA's 2022 Refusal to File for govorestat in Classic Galactosemia was a shock that changed the company's near-term focus. It forced a major reallocation of R&D spend toward the CMT-SORD Deficiency program, which was showing more promising data.
• The 2024 Capital Lifeline: The $100 million private placement in February 2024 was an absolute necessity. It bought the company critical time to execute the CMT-SORD trial extension and prepare for the next regulatory submission. Without it, the company's cash runway would have been dangerously short.
• Interim Leadership and Cost Control in 2025: As of November 2025, the company is under interim leadership with Les Funtleyder serving as both Interim CEO and CFO. This reflects an organizational focus on cost management and financial discipline as they navigate the final regulatory steps. The net loss for the third quarter of 2025 was $19.0 million, a significant improvement from the prior year's quarterly loss, but still a substantial burn.

The current strategy hinges entirely on the FDA's decision regarding govorestat for CMT-SORD Deficiency. The ongoing cash position of $11.9 million as of September 30, 2025, underscores the urgency. For a deeper dive into the 'why' behind these drug development choices, you should review the Mission Statement, Vision, & Core Values of Applied Therapeutics, Inc. (APLT).

Applied Therapeutics, Inc. (APLT) Ownership Structure

Applied Therapeutics, Inc. is controlled by a mix of institutional investors and individual shareholders, a typical structure for a publicly-traded, clinical-stage biopharmaceutical company. The majority of the company is held by professional money managers, which means strategic decisions are heavily influenced by the preferences of large funds.

Given Company's Current Status

Applied Therapeutics, Inc. is a publicly-traded, clinical-stage biopharmaceutical company focused on developing treatments for rare diseases. The company is listed on the NASDAQ Stock Market under the ticker symbol APLT.

As of the third quarter of 2025, the company's financial position showed cash and cash equivalents totaling $11.9 million as of September 30, 2025, with a reported net loss of $19.0 million for Q3 2025. This cash burn rate is a critical factor for investors, especially given the ongoing regulatory discussions with the FDA regarding their lead drug candidate, govorestat. Exploring Applied Therapeutics, Inc. (APLT) Investor Profile: Who's Buying and Why?

Given Company's Ownership Breakdown

Institutional investors hold the largest block of shares, giving them significant voting power over corporate governance matters, including board elections and major strategic moves. Insiders, while holding a smaller percentage, still maintain a meaningful stake that aligns their interests with the company's long-term success, or lack thereof.

Given Company's Leadership

The executive team is navigating a challenging regulatory environment, particularly with the FDA's feedback on govorestat for both Classic Galactosemia and Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD).

The leadership structure saw a recent change in November 2025, which can shake up strategy. Honestly, a leadership change during critical regulatory dialogue is defintely something to watch.

• Les Funtleyder: Serves as both the Interim Chief Executive Officer and Chief Financial Officer, a dual role he took on in late 2023.
• Evan Bailey: Chief Medical Officer, steering the clinical development programs.
• Dale Hooks: Chief Commercial, focused on preparing the market for potential drug approvals.
• Board Resignation: John H. Johnson resigned as Executive Chairman and as a director on November 9, 2025, a notable governance event.

Applied Therapeutics, Inc. (APLT) Mission and Values

Applied Therapeutics, Inc. (APLT) is fundamentally driven by a clear, singular mission: to create transformative treatments for patients suffering from rare, devastating metabolic diseases. This focus is expensive, though, with the company reporting a net loss of nearly $19 million in the third quarter of 2025 alone as they pursue their clinical goals.

You're looking for what this company stands for beyond the stock ticker, and honestly, their purpose is tied directly to their pipeline. The core of their culture is a deep commitment to the few, not the many, which is a high-stakes, high-cost endeavor.

Given Company's Core Purpose

The company's core purpose is clear in every public statement, mapping their scientific work directly to patient need. They are a clinical-stage biopharmaceutical company, so their entire operation centers on getting their lead drug, govorestat, through the regulatory finish line. Exploring Applied Therapeutics, Inc. (APLT) Investor Profile: Who's Buying and Why?

Official mission statement

The official mission statement, consistently used in their 2025 financial reports, is precise and actionable. It defines their scope-rare diseases-and their method-novel drug candidates against validated molecular targets.

• Be a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases.
• Focus on developing novel drug candidates against validated molecular targets.

This mission isn't just words; it dictates their cash burn. For instance, their Research and Development expenses for the third quarter of 2025 were $9.6 million, a direct investment into fulfilling this mission.

Vision statement

While Applied Therapeutics does not publish a separate, formal vision statement, their articulated long-term goal is to move beyond the clinical stage to successfully commercialize govorestat, transforming the treatment landscape for specific rare metabolic disorders.

• Transform the lives of patients with rare diseases, specifically Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and PMM2-CDG.
• Achieve regulatory alignment and approval for govorestat across multiple indications.
• Remove barriers to diagnosis and treatment, as seen with their sponsored sorbitol assay available to patients at no-cost.

Here's the quick math: their commitment to the CMT-SORD community, like offering the no-cost diagnostic assay, is an operational cost that supports their long-term vision of market adoption. It's smart, empathetic business.

Given Company slogan/tagline

Applied Therapeutics does not use a widely publicized, formal marketing slogan or tagline in its investor communications. The closest articulation of their operational value proposition is an unwavering focus on execution and progress in their clinical trials.

• 'Dedicated to creating transformative treatments for rare diseases.' (Used as a descriptive core purpose)

Their true 'tagline' is the action they take, like advancing govorestat through the FDA process, despite the financial pressure. They had only $11.9 million in cash and cash equivalents as of September 30, 2025, but they are defintely still pushing forward with multiple regulatory meetings.

Applied Therapeutics, Inc. (APLT) How It Works

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company that operates by discovering and developing novel small-molecule drugs, primarily Aldose Reductase Inhibitors (ARIs), to treat rare, genetically defined metabolic diseases. The company's core value creation comes from advancing its lead candidates, like govorestat (AT-007), through rigorous clinical trials and securing regulatory approval from the U.S. Food and Drug Administration (FDA) to address high unmet medical needs.

You can see the direct impact of this clinical-stage model in the Q3 2025 financials: total revenue was only $1.0 million, driven entirely by license revenue, but the net loss was a significant $18.99 million, which shows the heavy investment in R&D.

Given Company's Product/Service Portfolio

Given Company's Operational Framework

The company's operational process is a focused, high-risk, high-reward model typical of a clinical-stage biopharma. It centers on the efficient execution of clinical trials and proactive engagement with regulatory bodies like the FDA.

• Drug Discovery: Use structure-based drug design and proprietary screening to create novel small-molecule therapies that correct underlying metabolic dysfunction.
• Clinical Advancement: Execute multi-center, global Phase 3 trials for lead candidates like govorestat, which is in a planned confirmatory trial for CMT-SORD.
• Regulatory Navigation: Engage in frequent dialogue, such as the planned Q4 2025 FDA meeting to review govorestat for Classic Galactosemia, to clarify requirements for potential accelerated approval pathways.
• Financial Management: Direct most capital toward clinical development; for example, Q3 2025 Research and Development (R&D) expenses were $9.6 million.
• Revenue Generation: While primary revenue will come from drug sales post-approval, near-term capital is generated through licensing agreements, like the one that drove the $1.0 million in license revenue for Q3 2025.

Here's the quick math: with cash and cash equivalents at $11.9 million as of September 30, 2025, and a quarterly net loss of nearly $19.0 million, the runway is defintely short, so securing new financing or partnerships is a constant, critical operational task.

Given Company's Strategic Advantages

Applied Therapeutics' market success hinges on its ability to translate its unique scientific platform into approved, first-to-market therapies for underserved patient populations.

• CNS-Penetrant ARI Platform: The lead candidate, govorestat, is specifically engineered to cross the blood-brain barrier, which is a significant advantage for treating neurological symptoms in rare metabolic diseases like Galactosemia and SORD Deficiency.
• Orphan Disease Focus: Targeting rare metabolic diseases provides a clear regulatory path, including Orphan Drug Designation and Rare Pediatric Disease designation for indications like PMM2-CDG, which can help accelerate the review process.
• First-Mover Potential: Developing treatments for diseases like SORD Deficiency, where there are currently no approved therapies, positions the company to capture the entire market upon regulatory success.
• Intellectual Property: Leveraging advanced crystallography and structure-based design to develop new-generation ARIs gives them higher inhibitory activity and less off-target activity than older compounds.

For a deeper dive into the financial implications of this clinical-stage model, you should check out Breaking Down Applied Therapeutics, Inc. (APLT) Financial Health: Key Insights for Investors.

Applied Therapeutics, Inc. (APLT) How It Makes Money

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company, meaning it does not yet sell approved drugs to patients; its primary source of income is non-commercial, specifically through strategic partnerships and intellectual property (IP) licensing. For the first nine months of the 2025 fiscal year, the company's entire revenue stream was generated this way, not from product sales.

Applied Therapeutics, Inc.'s Revenue Breakdown

The company's revenue is currently concentrated in a single stream, reflecting its pre-commercial status where value is realized through out-licensing agreements for its drug candidates, such as govorestat (formerly AT-007).

For the third quarter of 2025, total revenue was $1 million, a substantial jump of 719.7% from the $122,000 reported in the same quarter a year prior. This growth is entirely attributed to License Revenue, which often comes from upfront payments or milestone payments from partners like Biossil, Inc..

Business Economics

As a clinical-stage biopharma, Applied Therapeutics, Inc.'s economics are defined by high upfront investment in drug development and minimal operating revenue, which is a common profile in this sector. The company's financial health is less about profit margins right now and more about the cash runway needed to achieve regulatory milestones.

• Pricing Strategy: The future pricing model will be a specialty pharmaceutical strategy, likely commanding premium prices due to the focus on rare metabolic diseases like Classic Galactosemia and Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), which have high unmet medical needs and small patient populations.
• Economic Fundamentals: The business model is a high-risk, high-reward proposition. Success hinges on a single, pivotal event: the U.S. Food and Drug Administration (FDA) approval of its lead candidate, govorestat. Until then, the company must raise capital through stock sales or licensing deals to fund its operations.
• Cost Structure: The vast majority of costs are operating expenses, specifically Research & Development (R&D) and General & Administrative (G&A), which outpace the current revenue by a significant margin. The operating margin is deeply negative, which is defintely expected for a company in this phase.

The core economic engine is the value of its intellectual property and clinical data. Every positive update on govorestat's regulatory path-like the planned Q4 2025 FDA meeting for Classic Galactosemia-increases the intrinsic value of that IP, making future licensing deals or a commercial launch more lucrative. You can read more about the company's long-term goals here: Mission Statement, Vision, & Core Values of Applied Therapeutics, Inc. (APLT).

Applied Therapeutics, Inc.'s Financial Performance

The company's financial performance as of the third quarter of 2025 highlights a focus on cost management while advancing its pipeline, but still underscores the capital-intensive nature of drug development.

• Net Loss: The net loss for Q3 2025 narrowed significantly to $18.99 million, a 72.3% reduction from the $68.59 million net loss in Q3 2024. This is a good sign for operational efficiency.
• Cash Position: The cash and cash equivalents stood at only $11.9 million as of September 30, 2025, a steep drop from $79.4 million at the end of 2024. Here's the quick math: the quarterly net loss of $18.99 million is significantly higher than the remaining cash, signaling a clear need for near-term financing.
• R&D and G&A Expenses: R&D expenses for Q3 2025 were $9.6 million, down from $14.8 million a year ago, reflecting a more focused approach to clinical trials. G&A expenses were $8.2 million for the quarter.
• Liquidity Risk: The company's Altman Z-Score-a measure of a company's financial distress-is in the distress zone, which is a critical risk factor for investors to consider. What this estimate hides is that a major licensing deal or a successful capital raise could instantly change this picture.

The key action for you as an analyst is to monitor the company's cash burn rate against the timeline for the next major regulatory milestone, like the planned Q4 2025 FDA meeting for govorestat in Classic Galactosemia.

Applied Therapeutics, Inc. (APLT) Market Position & Future Outlook

Applied Therapeutics, Inc. (APLT) is a high-risk, high-reward story right now, with its entire future hinging on a single molecule, govorestat (AT-007), and its upcoming regulatory decisions. The company's position is that of a potential first-mover in several ultra-rare metabolic disorders, but it is currently a pre-revenue, clinical-stage entity with significant liquidity concerns.

You're watching a company that is burning cash to chase a massive, unmet medical need. For the third quarter of 2025, the net loss was $19.0 million, a huge improvement, but cash and cash equivalents stood at only $11.9 million as of September 30, 2025. That cash position is defintely the immediate risk factor.

Competitive Landscape

The competitive landscape for Applied Therapeutics is unique because its lead drug, govorestat, targets diseases like Classic Galactosemia and Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) where no approved drug currently exists. This means their competition isn't an established blockbuster drug, but rather a race to be the first-in-class treatment against the current standard of care-which is primarily dietary management or supportive therapy.

Here's a snapshot of the competitive positioning in their key indications, keeping in mind that the market share for a specific therapeutic drug in these indications is 0% today.

Opportunities & Challenges

The next 12 to 18 months are critical for Applied Therapeutics. The strategic focus is entirely on securing regulatory approval for govorestat, which, if successful, would pivot the company from a clinical-stage entity to a commercial one with a potential US market opportunity in Galactosemia alone estimated between $222.2 million and $666.6 million annually.

But there are real, near-term hurdles. Honestly, the biggest challenge is the cash runway and the ongoing back-and-forth with the FDA on the regulatory path for both Classic Galactosemia and CMT-SORD.

Industry Position

Applied Therapeutics holds a precarious but potentially powerful position in the niche field of rare, central nervous system (CNS) metabolic disorders. They are a clinical-stage biotech, meaning they have no commercial operations to speak of, but their lead asset, govorestat, is a CNS-penetrant Aldose Reductase Inhibitor (ARI) that addresses the underlying pathology of several devastating conditions.

Their industry standing is defined by a few key points:

• Govorestat's mechanism is novel in these indications, targeting toxic metabolite accumulation (galactitol, sorbitol), which could offer a significant clinical advantage over symptomatic treatments.
• The company is actively engaged in regulatory discussions with the FDA in Q4 2025 for Classic Galactosemia and CMT-SORD, which are the main catalysts that will determine its commercial viability.
• The total revenue for Q3 2025 was only $1.0 million, primarily from a license agreement, underscoring the complete reliance on pipeline success.
• Analyst consensus as of November 2025 is mixed but leans toward a 'Buy' or 'Hold,' reflecting the high-risk/high-reward nature of their regulatory path.

If you want to dig deeper into who is betting on this trajectory, you should be Exploring Applied Therapeutics, Inc. (APLT) Investor Profile: Who's Buying and Why?

The next concrete step for you as a decision-maker is to track the outcome of the Q4 2025 FDA meetings for govorestat, as this will directly impact the company's ability to secure financing and execute its commercial launch plan.