Comprehensive Report: AG-2304

Disclaimer: This report focuses exclusively on AG-2304, an investigational drug developed by Ahn-Gook Pharmaceutical. Other products with similar designations, such as ARCT-2304 (an influenza vaccine candidate by Arcturus Therapeutics) and H1-Ag-2304 (an FDA reagent for influenza vaccine standardization), are distinct entities and are not the subject of this report.

1. Introduction and Overview of AG-2304

AG-2304 is an investigational drug candidate currently under development by Ahn-Gook Pharmaceutical Co., Ltd., a pharmaceutical company based in South Korea.[1] The compound is also referred to by the alternative names AG 2304 and AG2304 in various databases and clinical trial registries.[1] Ahn-Gook Pharmaceutical has a portfolio that includes treatments for a range of conditions, and AG-2304 represents one of its development programs in the cardiovascular and metabolic disease space.[2]

AG-2304 is classified as an antihyperlipidaemic agent and is being specifically developed for the treatment of hypercholesterolemia, with primary hypercholesterolemia being the targeted indication for its later-stage clinical trials.[3] Hypercholesterolemia, characterized by elevated levels of cholesterol in the blood, is a major risk factor for cardiovascular diseases. The development of new therapeutic options, particularly those offering improved efficacy, safety, or patient convenience, remains an area of active research.

The drug is intended for oral administration.[1] Currently, AG-2304 is in the preclinical stage of development for hypercholesterolemia in South Korea.[3] However, Ahn-Gook Pharmaceutical has plans to advance AG-2304 into human clinical trials, with a Phase 1 study in healthy volunteers (NCT06916169) and a Phase 3 study in patients with primary hypercholesterolemia (NCT06717360) slated to begin in 2025.[3]

According to AdisInsight, AG-2304 is designated as a New Molecular Entity (NME).[3] This designation is typically reserved for drugs containing an active moiety that has not been previously approved or marketed. However, clinical trial information suggests AG-2304 is likely a fixed-dose combination (FDC) of two components, AG23041 and AG23042. If these components are existing, approved drugs, the NME classification might reflect a novel aspect of the combination itself, such as a unique formulation, a new ratio of known drugs leading to an altered therapeutic profile, or the inclusion of a novel excipient or delivery technology that significantly modifies the drug's characteristics. Alternatively, one of the components within the FDC could itself be an NME. The "Mechanism of Action: Unknown" status further complicates the interpretation of the NME designation, as it leaves open the possibility of a novel pharmacological agent being part of the formulation. Clarification on the nature of its components is essential to fully understand this NME status and its implications for development and regulatory pathways.

Table 1: AG-2304 - Key Profile Summary

Feature	Details	Source(s)
Developer	Ahn-Gook Pharmaceutical Co., Ltd.	1
Alternative Names	AG 2304, AG2304	1
Therapeutic Area	Hypercholesterolemia (Primary Hypercholesterolemia)	3
Drug Class	Antihyperlipidaemic	3
Mechanism of Action	Undefined / Unknown	1
Route of Administration	Oral	1
Highest Reported Phase	Preclinical (Hypercholesterolemia); Phase 1 & Phase 3 planned for 2025	3
New Molecular Entity (NME)	Yes (according to AdisInsight)	3

2. Pharmacology and Mechanism of Action

2.1. Known Pharmacological Properties

AG-2304 is categorized as an antihyperlipidaemic agent, signifying its primary pharmacological role in reducing elevated lipid levels in the bloodstream.[3] This classification is consistent with its intended therapeutic application in managing hypercholesterolemia.

2.2. Mechanism of Action

The precise mechanism of action for AG-2304 remains "Undefined" or "Unknown" based on currently available public information.[1] This lack of a defined mechanism is noteworthy for a drug candidate for which Phase 3 trials are being planned.

If AG-2304 is indeed an FDC, the "unknown mechanism" might refer to the specific synergistic interactions or combined pharmacological effects of its constituent components that Ahn-Gook Pharmaceutical is investigating, rather than the individual mechanisms of well-established drugs. For instance, if the components were Rosuvastatin and Fenofibric acid, their respective mechanisms—HMG-CoA reductase inhibition by Rosuvastatin [8] and peroxisome proliferator-activated receptor alpha (PPARα) agonism by Fenofibric acid [10]—are well-documented. In such a scenario, the "unknown" aspect could pertain to novel combined effects on lipid metabolism, inflammation, or other relevant pathways, or it could imply that one of the components is a novel entity with an as-yet-undisclosed mechanism. The "New Molecular Entity: Yes" status reported by AdisInsight lends some credence to the possibility of a novel component or a significantly novel interaction or formulation technology that alters the drug's behavior.[3] Without explicit disclosure of AG-2304's components, a definitive elucidation of its mechanism is not possible.

2.3. Potential Targets

Given the undefined mechanism of action and the lack of confirmed identities for its components, the specific molecular targets of AG-2304 cannot be definitively stated. However, if the hypothesis that AG-2304 is a combination of Rosuvastatin and Fenofibric acid is accurate, then the primary pharmacological targets would be HMG-CoA reductase and PPARα, respectively.[8] Inhibition of HMG-CoA reductase reduces cholesterol synthesis, while activation of PPARα leads to increased lipoprotein lipase activity, enhanced fatty acid oxidation, and changes in apolipoprotein expression, collectively resulting in lowered triglyceride levels and increased HDL cholesterol.

3. Composition and Formulation

3.1. AG-2304 as a Fixed-Dose Combination (FDC)

Evidence strongly indicates that AG-2304 is being developed as an FDC. The design of the planned Phase 1 clinical trial (NCT06916169) involves a comparison of "AG2304" with the "coadministration of AG23041 and AG23042".[5] This type of study design is standard for establishing the bioequivalence of an FDC product against its individual active pharmaceutical ingredients administered concurrently. The goal is to demonstrate that the FDC delivers the active components to the systemic circulation in a manner comparable to taking them as separate entities, which is a common regulatory requirement for FDC approval.

3.2. Identity of AG23041 and AG23042

The specific chemical identities of AG23041 and AG23042, the presumed individual components of the AG-2304 FDC, are not explicitly disclosed in the available research materials. However, considering the therapeutic indication of hypercholesterolemia, the oral route of administration, and Ahn-Gook Pharmaceutical's existing intellectual property, a plausible hypothesis is that AG23041 and AG23042 are Rosuvastatin and Fenofibric acid, respectively, or vice versa.

Several points support this inference:

Rosuvastatin, a potent statin, and Fenofibric acid, the active metabolite of fenofibrate (a fibrate), are well-established lipid-lowering agents commonly used in combination to manage mixed dyslipidemia, addressing both elevated LDL-cholesterol and triglyceride levels, while also raising HDL-cholesterol.[12]
Ahn-Gook Pharmaceutical holds a patent (WO2011129579A2) for an oral pharmaceutical composition of fenofibric acid combined with an alkalifying agent, designed to enhance its bioavailability and reduce absorption variability.[15] This demonstrates the company's expertise and interest in optimizing fenofibric acid formulations.
A news report from South Korea mentioned Ahn-Gook Pharmaceutical developing a hyperlipidemia combination drug with a Phase 3 trial initiation that aligns with the projected timeline for AG-2304.[16]

While this hypothesis is based on logical deduction and circumstantial evidence, direct confirmation of the components' identities is pending.

3.3. Identity of AG2304T and AG2304R

The planned Phase 3 clinical trial (NCT06717360) for primary hypercholesterolemia lists the interventions as "AG2304T and AG2304R".[6] These designations likely refer to different dosage strengths or distinct formulations of the AG-2304 FDC. For example, "T" and "R" could denote variations in the milligram strength of one or both active components (e.g., Rosuvastatin 10mg/Fenofibric acid Xmg versus Rosuvastatin 20mg/Fenofibric acid Ymg, or different release profiles). Phase 3 trials often include arms with different dosing regimens to identify the optimal balance of efficacy and safety for the target patient population.

The development of an FDC such as AG-2304 typically aims to enhance patient compliance by reducing the number of pills taken daily. Furthermore, FDCs can sometimes offer synergistic efficacy or an improved side-effect profile compared to the individual components administered separately. The strategy of Ahn-Gook Pharmaceutical appears to involve leveraging potentially known active ingredients, possibly with an improved formulation for one component (e.g., fenofibric acid, based on their patent [15]), to create a competitive product in the mature antihyperlipidemic market. The success of such a strategy hinges on demonstrating clear clinical benefits over existing therapeutic options.

3.4. Formulation Details

AG-2304 is formulated for oral administration.[1] If fenofibric acid is indeed a component, the technology described in Ahn-Gook's patent WO2011129579A2, which involves an alkalifying agent to improve fenofibric acid's bioavailability, could be incorporated into the AG-2304 formulation.[15] Such an enhancement could provide a pharmacokinetic advantage, leading to more consistent drug absorption and potentially allowing for reduced dosages or improved efficacy.

4. Clinical Development Program

4.1. Preclinical Development

Ahn-Gook Pharmaceutical has conducted or is currently conducting preclinical trials for AG-2304 in South Korea, focusing on its effects in hypercholesterolemia.[3] These studies utilized an oral route of administration, consistent with the intended clinical formulation.[3] Specific findings from these preclinical investigations, such as detailed efficacy in animal models or comprehensive toxicology data, are not available in the public domain documents reviewed.

4.2. Clinical Trials

Two key clinical trials are planned for AG-2304, marking its transition into human testing: a Phase 1 pharmacokinetic study and a Phase 3 efficacy and safety trial.

4.2.1. Phase 1 Study (NCT06916169 / AG2304 BE)

Title: "An Open-label, Randomized, Fasting, Single, Group 2, Stage 2, Cross-over Study to Evaluate the Safety and Pharmacokinetics of AG2304 Compared to Coadministration of AG23041 and AG23042 in Healthy Adult Volunteers".[5]
Status: Not yet recruiting (as of April 2025).[1]
Sponsor: Ahn-Gook Pharmaceutical.[5]
Objectives: The primary objective is to evaluate and compare the pharmacokinetic (PK) characteristics and safety profiles of a single oral dose of the FDC AG2304 versus the co-administration of its individual components (AG23041 and AG23042) in healthy Korean subjects under fasting conditions.[7] The study focus is primarily on pharmacokinetics.[5]
Interventions:
Test Product: AG2304 (presumed FDC).[7]
Reference Product: Co-administration of AG23041 and AG23042 (individual components).[7]
Design: This is an open-label, randomized, single-dose, 2-sequence, 2-period, crossover study. Participants will be assigned to one of two sequences:
Sequence A: Receive co-administration of AG23041 and AG23042 in Period 1, followed by AG2304 in Period 2.[7]
Sequence B: Receive AG2304 in Period 1, followed by co-administration of AG23041 and AG23042 in Period 2.[7]
Population: Approximately 70 healthy adult volunteers, both male and female, aged between 19 and 65 years, are planned for enrollment.[7]
Key Timelines: The study is planned to commence enrollment in August 2025 [3], with an estimated primary completion date of September 2025.[1]
Probability of Success: Data from Ozmosi suggests a 23% probability of success for this trial.[1] This relatively low figure for a Phase 1 bioequivalence-type study may reflect general challenges associated with FDC development, potential complexities in achieving desired PK profiles for combined agents, or specific (undisclosed) concerns related to this particular combination.

4.2.2. Phase 3 Study (NCT06717360)

Title: "A Multicenter, Randomized, Double-blinded, Active-controlled, Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of AG2304T and AG2304R in Patients with Primary Hypercholesterolemia".[6]
Status: Not yet recruiting (as of December 2024/April 2025).[3]
Sponsor: Ahn-Gook Pharmaceutical.[6]
Objectives: The primary aim is to evaluate the efficacy and safety of two different forms or dosages of the FDC, AG2304T and AG2304R, in patients diagnosed with primary hypercholesterolemia.[6] The trial is designed for registrational purposes and to assess therapeutic use.[6]
Interventions:
Investigational Drugs: AG2304T and AG2304R (likely different strengths or formulations of the AG-2304 FDC).[6]
Comparator: The trial is active-controlled, meaning AG2304T/R will be compared against an existing therapy.[6] The specific active comparator is not identified in the available documents. The choice of this comparator is critical, as it will set the benchmark for assessing AG-2304's performance. For hypercholesterolemia, comparators often include established statin monotherapies (e.g., Rosuvastatin, Atorvastatin) or other lipid-lowering agents.
Design: The study will be a multicenter, randomized, double-blinded, active-controlled trial.[6]
Population: The trial will enroll adult patients aged 19 to 80 years with a diagnosis of primary hypercholesterolemia.[17]
Key Timelines: The planned start date for this Phase 3 trial is June 2025.[3] The primary completion date and overall duration are not specified, but Korean news sources suggest a potential conclusion in 2026, depending on recruitment and follow-up timelines.[16]

Table 2: Overview of Clinical Trials for AG-2304

Trial ID (NCT No.)	Phase	Title	Status (as of Apr 2025)	Interventions	Population	Key Objectives	Start Date (Planned)	Primary Completion (Planned)	Sponsor
NCT06916169	Phase 1	An Open-label, Randomized, Fasting, Single, Group 2, Stage 2, Cross-over Study to Evaluate the Safety and Pharmacokinetics of AG2304 Compared to Coadministration of AG23041 and AG23042 in Healthy Adult Volunteers	Not yet recruiting	AG2304 (FDC) vs. Co-administration of AG23041 + AG23042 (individual components)	70 Healthy Adults (19-65 years)	Evaluate and compare PK profiles and safety of AG2304 vs. its components.	Aug 2025	Sep 2025	Ahn-Gook Pharmaceutical
NCT06717360	Phase 3	A Multicenter, Randomized, Double-blinded, Active-controlled, Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of AG2304T and AG2304R in Patients with Primary Hypercholesterolemia	Not yet recruiting	AG2304T (FDC), AG2304R (FDC) vs. Active Comparator (unspecified)	Patients with Primary Hypercholesterolemia (19-80 years)	Evaluate efficacy and safety of AG2304T and AG2304R for primary hypercholesterolemia for registrational purposes.	Jun 2025	Not Specified	Ahn-Gook Pharmaceutical

5. Regulatory Status and Intellectual Property

5.1. Regulatory Status

As an investigational drug, AG-2304 has not received marketing approval in any jurisdiction. The planned Phase 1 and Phase 3 clinical trials indicate that Ahn-Gook Pharmaceutical is preparing or will submit the necessary Investigational New Drug (IND) applications or equivalent regulatory filings to health authorities, likely beginning with the Ministry of Food and Drug Safety (MFDS) in South Korea, to conduct these human studies.

5.2. Intellectual Property

No patents explicitly naming "AG-2304" as the core invention have been identified in the provided information. However, existing patents held by Ahn-Gook Pharmaceutical may be relevant to the composition of AG-2304, particularly if it is an FDC as hypothesized.

A key patent of interest is WO2011129579A2, titled "Oral pharmaceutical composition comprising fenofibric acid and an alkalifying agent".[15]

Assignee: Ahn-Gook Pharmaceutical Co., Ltd.
Content: This patent describes an oral formulation designed to improve the bioavailability of fenofibric acid and minimize variability in its absorption. Fenofibric acid is an active metabolite of fenofibrate, used in the treatment of hyperlipidemia, including hypercholesterolemia and hypertriglyceridemia.
Relevance to AG-2304: If fenofibric acid is one of the active components (AG23041 or AG23042) in the AG-2304 FDC, this patented formulation technology could be integral to AG-2304. Utilizing such a technology could offer a competitive advantage by potentially enhancing the pharmacokinetic profile of the fenofibric acid component, leading to more consistent drug exposure or allowing for different dosing. This proprietary formulation aspect could also contribute to the rationale behind AG-2304's designation as a New Molecular Entity by AdisInsight, even if fenofibric acid itself is a known compound.

Another patent associated with Ahn-Gook Pharmaceutical, US9464044B2, titled "Compound having ability to inhibit 11Beta-HSD1 enzyme..." [18], mentions utility in treating "lipid disorder." However, its primary focus on 11β-HSD1 inhibition for conditions like type II diabetes, obesity, and metabolic syndrome suggests it is likely related to a different drug candidate in Ahn-Gook's pipeline and is less directly relevant to AG-2304 as a primary hypercholesterolemia treatment with an "unknown mechanism."

The intellectual property strategy for AG-2304 likely hinges on the novelty of the combination itself, the specific ratios of its components, its clinical performance, and potentially the proprietary formulation technology for one or more of its constituents, such as the patented fenofibric acid delivery system.

6. Discussion and Future Outlook

6.1. Summary of Knowns and Unknowns

AG-2304 is an orally administered antihyperlipidaemic agent under development by Ahn-Gook Pharmaceutical, targeting primary hypercholesterolemia. It is advancing towards Phase 1 and Phase 3 clinical trials scheduled to commence in 2025. Evidence strongly suggests AG-2304 is a fixed-dose combination (FDC) of two active pharmaceutical ingredients, provisionally identified as AG23041 and AG23042, with different strengths or formulations (AG2304T and AG2304R) to be evaluated in later-stage trials.

Key information gaps persist, including:

The definitive chemical identities of the components AG23041, AG23042, and consequently the specific compositions of AG2304T and AG2304R.
A clear elucidation of AG-2304's mechanism of action, beyond its general classification as an antihyperlipidaemic.
The identity of the active comparator drug that will be used in the Phase 3 trial (NCT06717360).
Detailed results from preclinical efficacy and safety studies.
A comprehensive explanation for its designation as a New Molecular Entity, particularly if its components are known approved drugs.

6.2. Potential Significance and Competitive Landscape

The market for hypercholesterolemia treatments is substantial and well-established, featuring numerous effective monotherapies and some FDCs. FDCs are generally favored for their potential to improve patient adherence by reducing pill burden and, in some cases, by offering synergistic efficacy or improved tolerability.

If the hypothesis that AG-2304 is an FDC of Rosuvastatin and Fenofibric acid proves correct, it will enter a competitive space. Such combinations are aimed at patients with mixed dyslipidemia who require management of both LDL-cholesterol and triglycerides/HDL-cholesterol.[12] The clinical utility and differentiation of AG-2304 would depend on demonstrating a superior or comparable efficacy and safety profile to existing treatments, including potent statin monotherapies or other FDCs. Ahn-Gook Pharmaceutical's patented formulation technology for fenofibric acid, if incorporated into AG-2304, could provide a key pharmacokinetic advantage and a point of differentiation.[15] Improved bioavailability or more consistent absorption of fenofibric acid could lead to better therapeutic outcomes or allow for optimized dosing.

The development timeline suggests a potential market entry for AG-2304 around 2027-2028, assuming successful completion of clinical trials and regulatory review. Its ultimate place in therapy will be determined by the strength of its clinical data and the evolving landscape of hypercholesterolemia management, which may include newer classes of drugs by that time.

6.3. Key Milestones to Monitor

The progression of AG-2304 will be marked by several key milestones:

Successful initiation and completion of the Phase 1 pharmacokinetic/bioequivalence study (NCT06916169), with results anticipated post-September 2025.
Initiation of the Phase 3 efficacy and safety study (NCT06717360) in patients with primary hypercholesterolemia, planned for June 2025, and subsequent reporting of its outcomes.
Public disclosure by Ahn-Gook Pharmaceutical regarding the precise active ingredients of AG-2304, its detailed mechanism of action, and the rationale for its NME status.
Presentations of preclinical and clinical data at scientific conferences or in peer-reviewed publications.
Future regulatory submissions and outcomes in South Korea and potentially other international markets.
Further patent publications that may specifically cover the AG-2304 combination or its uses.

7. Conclusion

AG-2304, an investigational oral antihyperlipidaemic agent developed by Ahn-Gook Pharmaceutical, is poised to enter clinical development with planned Phase 1 and Phase 3 trials commencing in 2025. Current information strongly suggests AG-2304 is a fixed-dose combination, potentially leveraging known active ingredients such as Rosuvastatin and Fenofibric acid, the latter possibly benefiting from Ahn-Gook's patented formulation technology to enhance bioavailability.

While the precise composition and mechanism of action are yet to be fully disclosed, the clinical development program aims to establish its pharmacokinetic profile, efficacy, and safety for the treatment of primary hypercholesterolemia. The success of AG-2304 will depend on demonstrating a favorable risk-benefit profile compared to existing therapies in a competitive market. Future disclosures regarding its components, mechanism, and clinical trial results will be critical in assessing its therapeutic potential and place in the management of hypercholesterolemia. The "New Molecular Entity" designation, if it pertains to a novel component or a significantly altered characteristic of known components due to formulation, adds an intriguing dimension to its development narrative.

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AG-2304 Advanced Drug Monograph

Generic Name