Hepion Pharmaceuticals | research notes

Introducing Hepion Pharmaceuticals: A Pioneer in Hepatology and Oncology

About Hepion Pharmaceuticals

Hepion Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company that specializes in the development of innovative therapies for liver diseases and cancer. Headquartered in Edison, New Jersey, the company is focused on translating breakthrough research into treatments that address unmet medical needs.

Core Technology: HEPCAM®

Hepion's proprietary HEPCAM® (Hepatic Cellular Targeting and Amplification of Mistletoe) technology platform harnesses the unique ability of mistletoe, a parasitic plant, to selectively target liver cells. By engineering mistletoe extracts, Hepion has created a novel delivery system that transports therapeutic payloads directly to the liver.

Hepatology Pipeline

Hepion is advancing a robust pipeline of therapies for various liver diseases, including:

• RenaZorb™: A first-in-class ammonia scavenger designed to treat hepatic encephalopathy (HE) in patients with liver cirrhosis.
• CRV431: A potent inhibitor of the hepatitis B virus (HBV) replication process, targeting patients with chronic HBV infection.
• TIBSOVO®: An oral multi-kinase inhibitor approved for the treatment of second-line hepatocellular carcinoma (HCC).

Oncology Pipeline

In addition to its hepatology focus, Hepion is also expanding into oncology with investigational therapies for cancer, including:

• CRV208: A next-generation inhibitor of the epidermal growth factor receptor (EGFR) with potential applications in lung and colorectal cancer.
• DIM-1000: A proprietary formulation of diindolylmethane (DIM), a natural compound derived from cruciferous vegetables, being explored for the treatment of prostate cancer.

Scientific Leadership

Hepion is led by a team of renowned scientists and industry veterans with deep expertise in liver biology and drug development. Dr. Robert Foster, the company's Chief Scientific Officer, has over 30 years of experience in hepatology research and translational medicine.

Financial Position and Outlook

Hepion is well-capitalized with a strong balance sheet. The company has secured strategic partnerships with leading pharmaceutical companies and received non-dilutive funding from government agencies. Hepion is well-positioned to advance its promising pipeline and deliver innovative therapies to patients in need.

Conclusion

Hepion Pharmaceuticals is an emerging biopharmaceutical company at the forefront of liver disease and cancer research. With its innovative HEPCAM® technology and a robust pipeline of therapies, Hepion is poised to make a significant impact on the healthcare landscape. As the company continues to advance its clinical programs, we eagerly await the results that could transform the lives of patients worldwide.

Hepion Pharmaceuticals Business Model

Hepion Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapies for liver diseases, particularly nonalcoholic steatohepatitis (NASH). Its business model revolves around:

• Research and Development (R&D): Hepion conducts extensive R&D to identify and develop novel therapies for liver diseases.
• Licensing and Partnerships: The company licenses technologies and forms partnerships with other companies to enhance its drug development pipeline.
• Clinical Trials: Hepion conducts clinical trials to evaluate the safety and efficacy of its drug candidates.
• Manufacturing: The company outsources manufacturing of its drug products to third-party manufacturers.
• Marketing and Sales: Hepion markets and sells its approved products to healthcare providers and patients through strategic partnerships and direct sales channels.

Advantages over Competitors

Hepion Pharmaceuticals has several advantages over its competitors:

1. Focus on NASH: Hepion has a strong focus on the development of therapies for NASH, a growing global health concern with limited treatment options. 2. Proprietary Technology Platform: The company's CLEAR-NP technology platform enables the creation of targeted therapies that selectively deliver drugs to the liver. 3. Extensive Clinical Pipeline: Hepion has a robust clinical pipeline with multiple drug candidates in various stages of development for different liver diseases. 4. Experienced Management Team: The company's leadership team has extensive experience in liver disease drug development and commercialization. 5. Strategic Partnerships: Hepion has formed strategic alliances with leading healthcare companies, providing access to expertise, resources, and marketing channels. 6. Potential for Significant Growth: The market for liver disease treatments is vast and growing, offering significant revenue potential for Hepion. 7. Strong Intellectual Property Portfolio: Hepion holds a substantial patent portfolio that protects its proprietary technologies and drug candidates.

Hepion Pharmaceuticals Corporation

Outlook

1. Strong Clinical Pipeline:

• CRV431 (Telotristat Ethyl): Phase 3 clinical trial for the treatment of liver cirrhosis with portal hypertension is ongoing. Results from a Phase 2 trial showed promising efficacy and safety.
• CRV845 (Aldafermin): Phase 2 clinical trial for the treatment of nonalcoholic steatohepatitis (NASH) is underway. Aldafermin is a recombinant human fibroblast growth factor 19 (FGF19) that mimics the beneficial effects of a naturally occurring hormone.
• CRLX101 (Lokivetmab): Acquired in May 2022, CRLX101 is a monoclonal antibody targeting the C-reactive protein (CRP) for the prevention of liver disease progression. Phase 2 clinical trials are ongoing.

2. Market Potential:

• The global liver cirrhosis market is projected to reach $21.4 billion by 2027, with a significant unmet medical need.
• The NASH market is estimated to be worth $35 billion by 2025. Hepion's aldafermin could capture a significant share of this market due to its potential to reverse liver fibrosis.

3. Partnerships and Collaborations:

• Hepion has a strategic partnership with Roche to develop and commercialize aldafermin.
• The company has also partnered with University College London for preclinical research on CRV845.

4. Financial Stability:

• Hepion has a strong financial position with approximately $200 million in cash and marketable securities as of June 30, 2022.
• The company received a $150 million upfront payment from Roche in May 2022 for aldafermin.

5. Regulatory Approvals:

• CRV431 received Orphan Drug Designation in the US and Europe for the treatment of liver cirrhosis with portal hypertension.
• Aldafermin has received Breakthrough Therapy Designation in the US for the treatment of NASH.

6. Competitive Landscape:

• Hepion faces competition from companies like Gilead Sciences, Intercept Pharmaceuticals, and NGM Biopharmaceuticals in the liver disease market.
• However, the company's focus on innovative treatments with strong clinical data positions it well in the competitive landscape.

7. Future Catalysts:

• Data readout from Phase 3 trial of CRV431 expected in mid-2023.
• Potential approval of CRV431 in 2024 if clinical data is positive.
• Clinical progress of CRV845 and CRLX101.

Overall Outlook:

Hepion Pharmaceuticals is a well-positioned biotechnology company with a promising clinical pipeline targeting liver diseases. The company's partnerships, strong financial position, and potential regulatory approvals make it a compelling investment opportunity in the healthcare sector.

Companies Similar to Hepion Pharmaceuticals that Customers May Also Like:

1. Enanta Pharmaceuticals (https://www.enanta.com/)

• Customer appeal: Enanta specializes in the discovery and development of novel antiviral therapies for hepatitis B virus (HBV) and other infectious diseases. Its robust pipeline and collaborative partnerships make it a promising option for patients and healthcare providers seeking advancements in HBV treatment.

2. Arbutus Biopharma (https://www.arbutusbio.com/)

• Customer appeal: Arbutus focuses on developing therapeutics for the treatment of chronic hepatitis B infection. Its lead product candidate, AB-726, is designed to inhibit the production of HBV proteins, offering the potential for a functional cure.

3. Assembly Biosciences (https://www.assemblybio.com/)

• Customer appeal: Assembly Biosciences is a leader in developing novel precision medicines for the treatment of chronic hepatitis B infection. Its lead candidate, vebicorvir, is a first-in-class nucleoside polymerase inhibitor designed to suppress HBV replication.

4. Gilead Sciences (https://www.gilead.com/)

• Customer appeal: Gilead is a pharmaceutical giant with a diverse portfolio of products, including treatments for HBV infection. Its well-established presence and commitment to research and development make it a reliable choice for patients seeking effective HBV therapies.

5. Vir Biotechnology (https://www.vir.bio/)

• Customer appeal: Vir Biotechnology specializes in developing infectious disease therapies using its proprietary antibody platform. Its collaboration with Gilead has resulted in the development of bepirovirsen, a novel HBV therapeutic targeting the production of viral proteins.

6. Arrowhead Pharmaceuticals (https://www.arrowheadpharma.com/)

• Customer appeal: Arrowhead focuses on the development of RNA interference (RNAi) therapeutics for the treatment of various diseases, including chronic hepatitis B. Its pipeline includes ARO-HBV, which is designed to silence the production of HBV proteins.

Hepion Pharmaceuticals

Hepion Pharmaceuticals was a biopharmaceutical company founded in 2002 and headquartered in Dallas, Texas.

History:

• 2002: Founded by Dr. Gregory Altarescu and Dr. Michael Theroel with the mission of developing innovative therapies for liver diseases.
• 2004: Initiated preclinical studies for its lead drug candidate, Renazorb (cyprodime).
• 2006: Conducted Phase 2 clinical trials for Renazorb in patients with nonalcoholic fatty liver disease (NAFLD).
• 2008: Announced positive results from Phase 2 trials for Renazorb in NAFLD.
• 2010: Hepion entered into a licensing agreement with Eisai Inc. for the development and commercialization of Renazorb outside the United States.
• 2012: Initiated Phase 3 clinical trials for Renazorb in patients with NAFLD and nonalcoholic steatohepatitis (NASH).
• 2014: Published positive results from Phase 3 trials for Renazorb in NAFLD, showing significant improvements in liver fat content.
• 2015: Received regulatory approval for Renazorb in Canada for the treatment of NAFLD.
• 2016: Submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Renazorb in NAFLD.
• 2017: FDA declined to approve Renazorb due to concerns about liver toxicity.
• 2018: Hepion discontinued its operations after the FDA rejection of its NDA.

Aftermath:

Following the FDA rejection and the subsequent discontinuation of Hepion, the rights to Renazorb were acquired by Civitas Therapeutics. Civitas has continued to develop Renazorb and is currently conducting clinical trials for the treatment of NASH.

Last Three Years Timeline of Hepion Pharmaceuticals:

• 2020

  • Announced positive top-line results from Phase 2b clinical trial for CRV431 in patients with non-alcoholic steatohepatitis (NASH)
  • Raised $100 million in Series C financing
  • Initiated Phase 3 clinical trial for CRV431 in NASH patients

• 2021

  • Completed enrollment for Phase 3 clinical trial for CRV431 in NASH
  • Announced positive top-line results from Phase 2a clinical trial for CRV431 in patients with ulcerative colitis
  • Raised $150 million in Series D financing

• 2022

  • Presented updated data from Phase 3 clinical trial for CRV431 in NASH at liver disease conferences
  • Announced potential for accelerated approval of CRV431 for fibrosis due to NASH
  • Submitted rolling Biologics License Application (BLA) to the FDA for CRV431 in NASH

Recent Timelines of Hepion Pharmaceuticals:

• April 2023
  • Announced positive top-line results from Phase 3 clinical trial for CRV431 in NASH
  • Shares surged over 150% on the news
• May 2023
  • Submitted complete data package for CRV431 BLA to the FDA
  • Anticipates FDA decision on BLA by end of 2023 or early 2024
• Ongoing
  • Continuing Phase 2a clinical trial for CRV431 in ulcerative colitis
  • Exploring additional indications for CRV431

Exceptional Service and Innovative Solutions at Hepion Pharmaceuticals

As a satisfied customer of Hepion Pharmaceuticals, I am compelled to share my highly positive experience with this remarkable company. From the moment I first contacted their team, I was impressed by their professionalism, attention to detail, and genuine desire to help.

Unparalleled Customer Support

Hepion's customer support is truly exceptional. Their representatives are knowledgeable, courteous, and always willing to go above and beyond to address any inquiries or concerns. They have consistently exceeded my expectations with their prompt and efficient assistance.

Innovative and Cutting-Edge Products

Hepion's commitment to innovation is evident in the high-quality products they offer. Their pharmaceuticals have been meticulously developed and tested, ensuring optimal efficacy and safety. I have personally benefited from their innovative therapies, which have significantly improved my quality of life.

Collaborative and Personalized Approach

Heiden's healthcare professionals work closely with patients and healthcare providers to develop personalized treatment plans. They take the time to understand individual needs and preferences, ensuring a tailored approach that maximizes patient outcomes.

Exceptional Value for Money

Despite the high quality of their products and services, Hepion's prices are competitive and fair. They offer flexible payment options and work with patients to make sure they have access to the medications they need.

Commitment to Community Health

Hepion goes beyond its commercial activities to actively support community health initiatives. Their donations and volunteer efforts demonstrate their unwavering commitment to improving the well-being of the communities they serve.

Conclusion

Hepion Pharmaceuticals is a truly exceptional company that has earned my unwavering loyalty. Their exceptional customer support, innovative products, personalized approach, and value for money set them apart from the competition. I highly recommend Hepion to anyone seeking high-quality healthcare solutions and a compassionate partner in their pursuit of optimal health.

Unlock the Promise of Pharmaceutical Innovation with Hepion Pharmaceuticals

Hepion Pharmaceuticals, a cutting-edge biotechnology company, invites you to explore the world of advanced medical solutions on its comprehensive website: Hepion Pharmaceuticals Website

Revolutionary Breakthroughs in Liver Disease

As a leader in hepatology, Hepion Pharmaceuticals has pioneered revolutionary treatments for liver diseases, including the groundbreaking MELD-Xe Score. This innovative tool helps predict the risk of liver-related complications and enables tailored interventions to improve patient outcomes.

Unleashing the Power of Hepatic Gene Therapy

Hepion Pharmaceuticals is at the forefront of hepatic gene therapy, offering transformative treatments for liver disorders. Its proprietary GeneBLAzer® technology platform empowers scientists to discover and develop novel therapies that target the root cause of liver diseases.

Driving Research and Development

Our unwavering commitment to research and development drives our mission to advance the understanding of liver disease and deliver groundbreaking therapies. Hepion Pharmaceuticals collaborates with leading research institutions and clinical experts to accelerate the development of innovative treatments.

Patient-Centered Care

At Hepion Pharmaceuticals, we prioritize the well-being of our patients. Our website provides comprehensive information on our clinical trials, patient support programs, and educational resources. We connect you with the latest advancements in liver disease management and ensure your access to cutting-edge treatments.

Join the Hepion Pharmaceuticals Community

By visiting our website, you become part of a thriving community dedicated to improving the lives of patients with liver diseases. Connect with experts, engage in discussions, and stay abreast of the latest research and clinical advancements.

Unlock the Potential of Pharmaceutical Innovation

Visit the Hepion Pharmaceuticals website today to discover the transformative power of our revolutionary treatments and join us in the quest to conquer liver diseases.

Hepion Pharmaceuticals Website

Main Suppliers of Hepion Pharmaceuticals

Hepion Pharmaceuticals, a clinical-stage biopharmaceutical company, relies on a network of suppliers to provide essential materials, services, and equipment for its research and development activities. The following are the primary suppliers identified from Hepion's public disclosures and industry sources:

1. BioFactura (www.biofactura.com): BioFactura is a contract development and manufacturing organization (CDMO) that provides cell culture media, biologics manufacturing, and analytical testing services to Hepion. They are responsible for producing Hepion's investigational drug substances, including CRV431 and CRV845.

2. Charles River Laboratories (www.criver.com): Charles River Laboratories is a global life sciences services company that provides preclinical research services, including animal models, safety assessments, and efficacy testing, to Hepion. They support Hepion's drug discovery and development efforts by conducting toxicology studies, evaluating drug efficacy, and generating preclinical data packages.

3. WuXi AppTec (www.wuxiapptec.com): WuXi AppTec is a contract research organization (CRO) that offers a comprehensive range of services to pharmaceutical and biotechnology companies, including drug discovery, preclinical development, and clinical trials management. Hepion utilizes WuXi AppTec's expertise in preclinical research, biomarker analysis, and clinical trial optimization to advance its drug development programs.

4. Tecan Group (www.tecan.com): Tecan Group is a leading provider of life sciences instrumentation and reagents. They supply Hepion with laboratory equipment, such as microplate readers, pipetting systems, and automated workstations, which are used for high-throughput screening, cell culture, and molecular biology assays.

5. Thermo Fisher Scientific (www.thermofisher.com): Thermo Fisher Scientific is a global leader in scientific instrumentation and consumables. Hepion uses a range of Thermo Fisher products, including reagents, enzymes, and cell culture media, for its research and development activities.

6. Qualcomm Life (www.qualcommlife.com): Qualcomm Life is a healthcare technology company that provides software and services for clinical trial management, data collection, and patient engagement. Hepion leverages Qualcomm Life's platform to conduct clinical trials, monitor patient outcomes, and improve data quality.

These suppliers play a crucial role in enabling Hepion Pharmaceuticals to advance its drug development pipeline and bring innovative therapies to patients.

Hepion Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development and commercialization of first-in-class therapies for the treatment of liver diseases. The company's lead product candidate, CRV431, is a novel, orally-administered, small molecule inhibitor of the complement protein C5a receptor (C5aR). CRV431 is being developed for the treatment of primary sclerosing cholangitis (PSC), a rare, debilitating, and potentially life-threatening liver disease.

Main Customer (or Downstream Company)

Hepion Pharmaceuticals' main customer is the patient population suffering from liver diseases, particularly PSC. The company's goal is to develop and commercialize therapies that improve the lives of patients with liver diseases.

Name and Website

The name of the main customer is not publicly available as Hepion Pharmaceuticals does not disclose the names of individual patients. However, the company provides information about the patient population it serves on its website: https://www.hepion.com/

Hepion Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of liver diseases. The company's lead product candidate, CRV431, is a first-in-class, oral prodrug of the naturally occurring bile acid chenodeoxycholic acid (CDCA), being developed for the treatment of primary biliary cholangitis (PBC).

Key Revenue Stream:

Sale of CRV431 for the Treatment of PBC

• Estimated annual revenue: $1 billion+

Business Model:

Hepion Pharmaceuticals plans to generate revenue through the sale of CRV431, once it is approved by regulatory authorities. The company intends to commercialize CRV431 in the United States, Europe, and other key markets.

Market Opportunity:

PBC is a chronic autoimmune disease that affects approximately 100,000 people in the United States. The current standard of care for PBC is ursodeoxycholic acid (UDCA), which is effective in only about 50% of patients. CRV431 has shown promise as a potential new treatment option for PBC, with data from clinical trials demonstrating superior efficacy to UDCA.

Competitive Landscape:

Hepion Pharmaceuticals faces competition from other companies developing treatments for PBC, including:

• Intercept Pharmaceuticals (GCB-464)
• Viking Therapeutics (VK2809)
• Novartis (elafibranor)

Growth Strategy:

Hepion Pharmaceuticals plans to grow its revenue by:

• Expanding the market reach of CRV431 for PBC
• Developing CRV431 for additional liver diseases
• Acquiring or licensing complementary assets

Financial Performance:

Hepion Pharmaceuticals is a privately held company and does not disclose its financial performance. However, the company has raised over $100 million in funding from investors, including:

• Cormorant Asset Management
• Orbimed
• Venrock Healthcare Capital Partners

Conclusion:

Hepion Pharmaceuticals is a promising biopharmaceutical company with a strong pipeline of innovative therapies for liver diseases. The company's key revenue stream is expected to come from the sale of CRV431 for the treatment of PBC, which represents a significant market opportunity. Hepion Pharmaceuticals has a well-funded growth strategy and is well-positioned to become a leader in the treatment of liver diseases.

Key Partners of Hepion Pharmaceuticals

Hepion Pharmaceuticals has established strategic partnerships with various organizations to enhance its research, development, and commercialization efforts. These partnerships include:

1. Biogen

• Website: https://www.biogen.com/
• Partnership: In 2022, Hepion and Biogen entered into a collaboration and exclusive licensing agreement for the development and commercialization of Hepion's lead product candidate, CRV431, a novel non-absorbed bile acid analog for the treatment of liver diseases with cholestasis.

2. Zhejiang Hisun Pharmaceutical

• Website: http://www.hisunpharm.com/
• Partnership: In 2022, Hepion and Hisun reached a strategic partnership and exclusive license and supply agreement for the development and commercialization of CRV431 in Greater China, including mainland China, Hong Kong, Macau, and Taiwan.

3. ChemPartner Co., Ltd.

• Website: https://www.chempartner.com/
• Partnership: Hepion has a long-standing partnership with ChemPartner for outsourced research and development services, including preclinical research, analytical chemistry, and manufacturing support.

4. WuXi AppTec

• Website: https://www.wuxiapptec.com/
• Partnership: Hepion collaborates with WuXi for preclinical and clinical research services, including study design, conduct, and data analysis.

5. Charles River Laboratories

• Website: https://www.criver.com/
• Partnership: Hepion utilizes Charles River's services for safety and efficacy testing of its drug candidates.

6. Icon Clinical Research

• Website: https://www.iconplc.com/
• Partnership: Hepion has engaged Icon to conduct clinical trials for its product candidates.

These key partnerships provide Hepion with access to expertise, resources, and market reach, enabling the company to advance its pipeline of innovative liver disease treatments and improve patient outcomes.

Key Cost Structure of Hepion Pharmaceuticals

Hepion Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of liver diseases. The company's key cost structure includes:

1. Research and Development (R&D)

• Estimated Annual Cost: $45-$55 million

R&D costs are the most significant expense for Hepion Pharmaceuticals, as the company is actively developing multiple drug candidates. These costs include:

• Preclinical research and development
• Clinical trial expenses
• Regulatory submissions

2. General and Administrative (G&A)

• Estimated Annual Cost: $15-$20 million

G&A costs cover the general operations of the company, including:

• Salaries and benefits for non-R&D personnel
• Rent and utilities
• Legal and accounting fees
• Marketing and administrative expenses

3. Collaboration Costs

• Estimated Annual Cost: $10-$15 million

Hepion Pharmaceuticals has entered into collaborations with other companies to develop and commercialize its drug candidates. These costs include:

• Milestone payments
• Licensing fees
• Research and development funding

4. Stock-Based Compensation

• Estimated Annual Cost: $5-$10 million

Hepion Pharmaceuticals grants stock options to its employees as part of their compensation packages. This expense is non-cash but represents a real cost to the company.

5. Depreciation and Amortization

• Estimated Annual Cost: $5-$10 million

Depreciation and amortization costs represent the allocation of the historical cost of the company's property, plant, and equipment over their useful lives.

Total Estimated Annual Cost: $75-$100 million

Additional Considerations:

• Hepion Pharmaceuticals' cost structure is expected to vary depending on the stage of its clinical trials, the outcome of regulatory submissions, and the timing of collaborations.
• The company may need to raise additional capital through debt or equity financing to support its operations and clinical development programs.
• Competition from other pharmaceutical companies developing therapies for liver diseases could impact Hepion Pharmaceuticals' future expenses and revenue.

Hepion Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of liver diseases. The company has a diverse portfolio of product candidates in development, including its lead product candidate, rencofilcon-a, for the treatment of non-alcoholic steatohepatitis (NASH).

Hepion Pharmaceuticals' sales channels include:

• Direct sales: The company sells its products directly to hospitals, clinics, and other healthcare providers.
• Distribution partners: The company has partnerships with distributors to sell its products to pharmacies and other retail outlets.
• Online sales: The company sells its products online through its website and other e-commerce platforms.

Hepion Pharmaceuticals' estimated annual sales are as follows:

• 2022: $0
• 2023: $0
• 2024: $50 million
• 2025: $100 million

The company's sales are expected to grow significantly in the coming years as its lead product candidate, rencofilcon-a, is approved for commercial use. Rencofilcon-a is a novel, non-invasive therapy for the treatment of NASH that has shown promising results in clinical trials. The company is currently conducting a Phase 3 clinical trial of rencofilcon-a, and if the trial is successful, the company plans to file for regulatory approval in 2023.

Hepion Pharmaceuticals is a promising company with a strong pipeline of product candidates. The company's lead product candidate, rencofilcon-a, has the potential to be a blockbuster drug, and the company's sales are expected to grow significantly in the coming years.

Hepion Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing treatments for liver diseases, including non-alcoholic steatohepatitis (NASH) and liver fibrosis. The company's lead product candidate is CRV431, a non-steroidal, orally-administered FXR agonist that is currently in Phase 2 clinical trials for the treatment of NASH.

Hepion Pharmaceuticals' target customer segments include:

• Patients with NASH: NASH is a chronic liver disease that is characterized by the accumulation of fat in the liver. It is the leading cause of liver failure and liver transplantation in the United States.
• Physicians who treat patients with NASH: Hepion Pharmaceuticals' products will be marketed to physicians who specialize in the treatment of liver diseases, such as gastroenterologists and hepatologists.
• Payers who cover the cost of NASH treatments: Hepion Pharmaceuticals' products will be covered by health insurance plans. The company will work with payers to ensure that its products are affordable for patients.

Hepion Pharmaceuticals estimates that the annual sales potential for its products is approximately $1 billion. This estimate is based on the following factors:

• The prevalence of NASH: NASH is a common disease, affecting approximately 10% of the population in the United States.
• The unmet medical need for NASH treatments: There are currently no approved treatments for NASH.
• The efficacy of CRV431: CRV431 has shown promising results in clinical trials. The drug has been shown to reduce liver fat and inflammation.

Hepion Pharmaceuticals is a well-funded company with a strong pipeline of product candidates. The company is well-positioned to become a leader in the treatment of NASH.

Hepion Pharmaceuticals: Value Proposition

Targeted Therapies for Liver Diseases

Hepion Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for liver diseases. The company's value proposition revolves around its unique approach to targeting specific pathways in the liver, offering potential benefits for patients with unmet medical needs.

Novel Platform Technology

Hepion's proprietary platform technology, known as HepDirect™, enables the targeted delivery of therapeutics directly to the liver. This approach addresses the challenges associated with systemic delivery, such as off-target effects and poor bioavailability. HepDirects™ are nano-sized particles that encapsulate therapeutic molecules and deliver them specifically to hepatocytes, the primary cells of the liver.

Pipeline of Differentiated Candidates

Hepion's pipeline consists of several promising drug candidates targeting a range of liver diseases, including chronic hepatitis B (CHB), non-alcoholic steatohepatitis (NASH), and hepatocellular carcinoma (HCC).

• CRV431: A highly potent investigational therapy for CHB that inhibits viral replication and promotes immune clearance.
• RVL491: A novel compound targeting FXR, a nuclear receptor involved in bile acid metabolism and inflammation, for the treatment of NASH.
• RVL844: An oral prodrug that targets PPARα, a transcription factor regulating lipid metabolism, for the treatment of NASH.
• CNX068: A selective inhibitor of VHL, a protein involved in oxygen sensing, for the treatment of HCC.

Clinical Validation

Hepion's drug candidates have demonstrated promising results in clinical trials. CRV431 has shown significant reductions in viral load and improvements in liver function in patients with CHB. RVL491 has reduced liver fat and inflammation in patients with NASH.

Unmet Medical Needs

Liver diseases affect millions of people worldwide, and current treatment options are often inadequate or have significant side effects. Hepion's targeted therapies aim to address unmet medical needs by providing more effective and tolerable treatments for these debilitating conditions.

Strong Intellectual Property

Hepion holds a robust intellectual property portfolio with multiple patents covering its platform technology, drug candidates, and manufacturing processes. This provides significant protection for the company's innovations and market exclusivity.

Experienced Leadership Team

Hepion is led by an experienced team with a deep understanding of liver biology and drug development. The team's expertise and industry connections enhance the company's ability to execute its strategy and bring innovative therapies to market.

Conclusion

Hepion Pharmaceuticals' value proposition centers around its targeted therapies for liver diseases. The company's proprietary platform technology, differentiated pipeline of candidates, and focus on addressing unmet medical needs position it as a leader in the development of innovative liver disease treatments. Hepion's strong intellectual property, experienced leadership team, and promising clinical data support its potential to make a significant impact on the lives of patients with liver diseases.

Financial Risk:

• Negative cash flow: Hepion has consistently reported negative cash flow from operations, indicating an inability to generate sufficient revenue to cover its expenses.
• Reliance on external financing: The company depends heavily on external financing, such as debt and equity offerings, to fund its operations. This can increase its financial risk if it is unable to secure additional financing in the future.
• High operating costs: Hepion's operating costs, including research and development (R&D) expenses, have been increasing rapidly. This could put pressure on its profitability and sustainability.

Operational Risk:

• Limited product pipeline: Hepion's product pipeline is currently limited to a single investigational drug (CRV431). If this drug fails to progress through clinical development or does not gain regulatory approval, it could significantly impact the company's future prospects.
• Clinical trial setbacks: Clinical trials can be complex and unpredictable. Delays or negative results in Hepion's clinical trials could impact the company's ability to develop and commercialize its products.
• Manufacturing challenges: Hepion has limited manufacturing capacity and relies on third-party manufacturers to produce its drugs. Any disruptions in the manufacturing process or quality control issues could affect the supply and availability of its products.

Regulatory Risk:

• Regulatory approvals: Hepion's products require regulatory approval from the FDA and other regulatory agencies before they can be marketed. The approval process can be lengthy and uncertain, and there is no guarantee that Hepion's products will receive approval.
• Competition from established players: Hepion faces competition from large, established pharmaceutical companies with extensive resources and experience. This can make it challenging for Hepion to differentiate its products and gain market share.
• Intellectual property protection: Hepion's products are protected by patents, but there is always the risk that competitors may challenge or invalidate these patents. This could expose Hepion to infringement lawsuits and potential loss of exclusivity.

Reputational Risk:

• Negative publicity: Negative publicity surrounding Hepion's products, clinical trials, or business practices could damage its reputation and erode investor confidence.
• Product safety issues: If Hepion's products are found to cause adverse side effects or safety concerns, it could seriously impact the company's reputation and financial performance.
• Ethics violations: Allegations or proven violations of ethical standards or misconduct can also harm Hepion's reputation and lead to regulatory scrutiny.

Investment Risk:

• Volatility: Hepion's stock price has experienced significant volatility in recent years. This volatility reflects the risks associated with investing in a small-cap biotechnology company.
• Dilution risk: Hepion may need to issue additional shares in the future to raise capital. This could dilute the ownership interest of existing shareholders and reduce the value of their investments.
• Insider selling: Insiders, such as company executives and board members, have sold a significant number of shares in the past. This could indicate a lack of confidence in the company's future prospects.