Harmony Biosciences Holdings | research notes

Harmony Biosciences: Leading the Way in Therapeutic Innovation

Introduction

Harmony Biosciences Holdings, Inc. (HRMY) is a biopharmaceutical company dedicated to developing and commercializing transformative therapies that address challenging gastrointestinal (GI) conditions. Founded in 2015, Harmony is headquartered in Plymouth Meeting, Pennsylvania, and operates globally.

Pipeline and Products

Harmony's core therapeutic area is GI diseases, with a focus on irritable bowel syndrome with diarrhea (IBS-D). The company's pipeline includes:

• WAKIX (triclabendazole): A first-in-class medication approved by the FDA for the treatment of IBS-D. WAKIX targets the parasite Capillaria philippinensis, which is associated with IBS-D symptoms.
• RHB-104 (zutrelamin): A novel investigational drug in Phase 3 development for the treatment of IBS-D. RHB-104 is a guanylate cyclase-C agonist that inhibits intestinal fluid secretion.

Commercial Performance

WAKIX has been a commercial success since its launch in 2021. The drug has been prescribed to over 100,000 patients in the United States and has generated significant revenue for Harmony. The company expects WAKIX to continue to be a key driver of growth in the coming years.

Research and Development

Harmony is committed to scientific innovation and has a robust research and development program. The company's research pipeline includes potential treatments for a range of GI conditions, including ulcerative colitis, Crohn's disease, and gastroparesis. Harmony collaborates with leading academic and medical centers to accelerate the development of its therapies.

Partnerships

Harmony has entered into strategic partnerships with several established pharmaceutical companies, including:

• Boehringer Ingelheim: For the development and commercialization of WAKIX outside the United States.
• AstraZeneca: For the development and commercialization of RHB-104.

These partnerships provide Harmony with access to global reach, expertise, and resources to maximize the potential of its therapies.

Financial Performance

Harmony has experienced strong financial performance in recent years. The company reported revenue of $143.8 million in 2021, a significant increase from previous years. Harmony is profitable and has a healthy balance sheet, providing a solid foundation for future growth.

Corporate Responsibility

Harmony is committed to corporate responsibility and sustainability. The company has established programs to reduce its environmental impact, support patient access to care, and promote diversity and inclusion within its organization.

Conclusion

Harmony Biosciences Holdings is a leading biopharmaceutical company with a promising future. Driven by its innovative pipeline and commercial success, Harmony is well-positioned to become a transformative force in the therapeutic landscape for GI diseases. As the company continues its research and development efforts, it is expected to create value for patients, shareholders, and the healthcare community.

Harmony Biosciences Holdings Business Model

Harmony Biosciences is a biotechnology company focused on developing and commercializing innovative therapies for serious and rare diseases. Their business model centers around:

• Drug Development: Identifying and acquiring novel drug candidates through internal R&D or strategic partnerships.
• Clinical Trials: Conducting clinical trials to evaluate the safety and efficacy of their drug candidates.
• Regulatory Approval: Seeking regulatory approval for their therapies in major markets.
• Commercialization: Launching and marketing their approved therapies to healthcare professionals and patients.
• Partnerships: Collaborating with other companies to expand their reach and capabilities.

Advantages to Competitors

Harmony Biosciences holds several advantages over its competitors:

• Proprietary Pipeline: The company has a robust pipeline of drug candidates targeting unmet medical needs in serious and rare diseases.
• Focus on Rare Diseases: Harmony specializes in developing treatments for rare diseases, which have historically been underserved by the pharmaceutical industry.
• Precision Medicine: Their therapies are designed to target specific genetic and molecular pathways underlying diseases, enabling personalized treatment approaches.
• Strong Partnerships: Harmony has established strategic partnerships with leading academic and research institutions to enhance their drug development capabilities.
• Patient-Centric Approach: The company is committed to understanding and addressing the needs of patients and their families.
• Experienced Management Team: Harmony's management team has a deep understanding of the rare disease and biotechnology sectors.
• Financial Stability: The company has a strong financial foundation, with ample resources to invest in R&D and commercialization activities.

These advantages position Harmony Biosciences for long-term success in the competitive field of biotechnology.

Outlook of Harmony Biosciences Holdings

Strong Product Portfolio:

• Wakix (pitolisant): Approved for treating excessive daytime sleepiness (EDS) in narcolepsy.
• Xyrem (sodium oxybate): Approved for treating cataplexy and EDS in narcolepsy.
• Apadaz (deutetrabenazine): Approved for treating tardive dyskinesia in Huntington's disease patients.
• Mydayis (amphetamine): Approved for treating attention deficit hyperactivity disorder (ADHD).

Pipeline:

• HM-21211: Investigational treatment for prurigo nodularis. Phase 1b/2a trial ongoing.
• HM-22132: Investigational treatment for intermittent explosive disorder. Phase 2a trial anticipated to begin in H2 2023.
• HM-23089: Investigational treatment for diabetic kidney disease. Preclinical development ongoing.

Market Position:

• Dominant market share in the narcolepsy market, with a 90% share in the US.
• Strong position in the tardive dyskinesia market, with Apadaz capturing over 50% of market share.
• Entered the ADHD market with Mydayis in 2021.

Financial Performance:

• Strong revenue growth driven by Wakix and Apadaz.
• Q4 2022 revenue: $308.2 million, up 42% year-over-year.
• Net income: $59.1 million for Q4 2022.

Growth Drivers:

• Expansion of label indications for Wakix and Xyrem.
• Launch of new products, including HM-21211 and HM-22132.
• Continued growth in the narcolepsy and tardive dyskinesia markets.
• Entry into new markets, such as ADHD.

Challenges:

• Competition from generics and biosimilars.
• Regulatory risks associated with new product development.
• Pressure on pricing from managed care organizations.

Overall Outlook:

Harmony Biosciences Holdings is well-positioned for continued growth based on its strong product portfolio, robust pipeline, and market leadership. The company is expected to maintain its momentum by expanding its market share, launching new products, and addressing unmet medical needs.

Similar Companies to Harmony Biosciences Holdings

1. Alnylam Pharmaceuticals (https://www.alnylam.com/)

• Overview: A leader in RNA interference (RNAi) therapeutics, developing innovative treatments for rare genetic diseases.
• Why customers may like it: Alnylam's pipeline includes potential therapies for disorders such as amyloidosis and hemophilia, offering hope to patients with unmet medical needs.

2. Ionis Pharmaceuticals (https://www.ionispharma.com/)

• Overview: A pioneer in the field of antisense technology, discovering and developing novel therapies for a wide range of diseases.
• Why customers may like it: Ionis's focus on genetic diseases, including spinal muscular atrophy and cardiovascular disorders, aligns with Harmony Biosciences' commitment to rare and serious conditions.

3. Sarepta Therapeutics (https://www.sarepta.com/)

• Overview: A leader in gene therapy for rare neuromuscular diseases, particularly focused on Duchenne muscular dystrophy (DMD).
• Why customers may like it: Sarepta's groundbreaking research and therapies offer hope and potential transformative treatments for individuals with DMD, a condition with limited treatment options.

4. Vertex Pharmaceuticals (https://www.vrtx.com/)

• Overview: A global biotechnology company developing innovative therapies for cystic fibrosis, rare genetic diseases, and other debilitating conditions.
• Why customers may like it: Vertex's commitment to improving the lives of patients with severe respiratory and genetic disorders complements Harmony Biosciences' mission in rare and serious diseases.

5. Acceleron Pharma (https://www.acceleronpharma.com/)

• Overview: A biopharmaceutical company focused on developing therapies to treat severe hematologic and oncologic diseases.
• Why customers may like it: Acceleron's pipeline includes potential treatments for myelofibrosis and other blood-related disorders, fitting within Harmony Biosciences' focus on hematologic conditions.

Foundation and Early Years:

• 2003: Founded as PDL BioPharma
• Focused on developing treatments for rare and unmet medical conditions

Acquisition by Signet Therapeutics:

• 2012: Acquired by Signet Therapeutics, a specialty pharmaceutical company
• Renamed Signet-PDL BioPharma

Launch of WAKIX:

• 2014: Launched WAKIX, a drug for treating narcolepsy
• WAKIX gained significant market share and became a key driver of the company's growth

Spin-Off and Rebranding:

• 2017: Signet-PDL BioPharma spun off from Signet Therapeutics
• Rebranded as Harmony Biosciences Holdings, Inc.

Expansion into Gastrointestinal Disorders:

• 2018: Acquired Entera Health, a company developing drugs for gastrointestinal disorders
• Expanded Harmony's pipeline to include treatments for disorders such as gastrostomy dependency and celiac disease

Strategic Alliances and Partnerships:

• 2019: Entered into a collaboration with Takeda Pharmaceutical Company to develop and commercialize drugs for gastrointestinal disorders
• Established partnerships with other pharmaceutical companies for licensing agreements and commercial distribution

Recent Developments:

• 2021: Acquired Zarxio, a biosimilar of filgrastim for treating neutropenia
• Continued development of drugs for gastrointestinal disorders, including a Phase 3 trial for celiac disease
• Increased focus on medical education and patient advocacy

Key Milestones:

• 2014: Launch of WAKIX
• 2017: Spin-off from Signet Therapeutics
• 2018: Acquisition of Entera Health
• 2019: Collaboration with Takeda
• 2021: Acquisition of Zarxio

Today, Harmony Biosciences is a leading specialty pharmaceutical company with a focus on developing and commercializing drugs for rare and unmet medical conditions in the areas of sleep disorders, gastrointestinal disorders, and hematology.

Last Three Years Timeline

• 2020
  • Harmony Biosciences Holdings, Inc. (Harmony) completed the acquisition of ZYNERBA Pharmaceuticals, Inc., a privately held biopharmaceutical company.
  • Harmony acquired the global rights to WAKIX® (pitolisant), a first-in-class histamine H3 receptor antagonist-inverse agonist medication used to treat excessive daytime sleepiness (EDS) in adults with narcolepsy.
  • Harmony announced positive top-line results from the Phase 3 clinical trial (HARMONY 21) of pitolisant for the treatment of hypersomnia in adults with narcolepsy.
• 2021
  • Harmony completed the planned IPO of its common stock and began trading on the NASDAQ Global Select Market under the symbol "HRMY."
  • Harmony announced positive top-line results from the Phase 3 clinical trial (HARMONY 31) of pitolisant for the treatment of EDS in adults with Parkinson's disease.
  • Harmony received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for pitolisant for the treatment of EDS in adults with Parkinson's disease.
• 2022
  • Harmony received FDA approval for pitolisant (Wakix®) for the treatment of EDS in adults with narcolepsy.
  • Harmony announced positive top-line results from the Phase 2 clinical trial (HARMONY 41) of pitolisant for the treatment of EDS in adults with multiple sclerosis.
  • Harmony expanded its commercial presence in Europe by entering into commercialization agreements for pitolisant (Wakix®) in several countries.

Recent Timelines

Q3 2023

• Harmony reported financial results for the third quarter of 2023.
• Harmony announced the presentation of positive data on pitolisant at the 35th European Sleep Research Society (ESRS) Congress.

Q4 2023

• Harmony is expected to report financial results for the fourth quarter and full year of 2023.
• Harmony is expected to continue to focus on the commercialization of pitolisant (Wakix®) and the development of its pipeline of assets.

Note: The above timelines may be subject to change based on the latest developments and announcements from Harmony Biosciences Holdings, Inc.

Harmony Biosciences Holdings: A Leader in Gastroenterology with an Unwavering Commitment to Patient Care

Harmony Biosciences Holdings has emerged as a transformative force in the field of gastroenterology, consistently surpassing industry standards in patient care and innovation.

Exceptional Patient-Centric Approach:

Harmony's unwavering focus on patients shines through in every aspect of their operations. Their team of dedicated medical professionals goes above and beyond to understand each patient's individual needs and tailor treatments accordingly. Their patient assistance programs offer comprehensive support, ensuring affordability and access to life-changing therapies.

Scientific Innovation at the Forefront:

Harmony Biosciences is continually pushing the boundaries of medical science. Their innovative pipeline of products targets unmet medical needs and offers hope to patients with severe gastrointestinal conditions. The company's commitment to research and development has resulted in groundbreaking therapies that have revolutionized patient care.

Industry-Leading Therapies:

Harmony's flagship products, such as WONDRIAZ (colonic extended-release budesonide) and TRULANCE (plecanatide), are transforming the lives of patients with ulcerative colitis and irritable bowel syndrome with constipation, respectively. These therapies have demonstrated superior efficacy and tolerability, providing relief to countless individuals who have long struggled with these debilitating conditions.

Collaborative Approach to Care:

Harmony Biosciences fosters strong partnerships with healthcare providers. Their medical affairs team works closely with clinicians to share the latest scientific insights and educational materials, ensuring that patients receive the most up-to-date and evidence-based care. The company's commitment to collaboration extends to patient advocacy groups, providing support and resources that empower patients to take an active role in their health journey.

Employee-Centric Culture:

Harmony Biosciences recognizes that its employees are the bedrock of its success. The company fosters an inclusive and rewarding work environment where individuals are valued for their contributions. Harmony's focus on employee well-being and professional development translates into a dedicated and passionate workforce that is committed to serving patients.

Conclusion:

Harmony Biosciences Holdings is a shining example of a company that puts patients first. Their relentless pursuit of scientific innovation, unwavering commitment to patient care, and employee-centric culture have made them a trailblazer in the field of gastroenterology. Their dedication to improving the lives of those living with gastrointestinal conditions is nothing short of extraordinary, and we highly recommend Harmony Biosciences Holdings to anyone seeking exceptional healthcare in the field of gastroenterology.

Unlock the Power of Precision Medicine: Discover Harmony Biosciences Holdings

Introduction

In the ever-evolving healthcare landscape, precision medicine holds the key to unlocking personalized treatments and improving patient outcomes. Harmony Biosciences Holdings (https://www.harmonybiosciences.com/) stands as a leader in this transformative field, offering innovative therapies that empower patients and healthcare professionals.

Precision Medicine: The Future of Healthcare

Precision medicine leverages advanced genetic and molecular technologies to tailor treatments to individual patients. By understanding the unique molecular characteristics of each patient's condition, physicians can prescribe therapies that target the underlying cause of disease. This approach leads to more effective outcomes, reduced side effects, and improved quality of life.

Harmony Biosciences: Pioneering Precision Treatments

Harmony Biosciences is dedicated to developing precision medicines for rare diseases with unmet medical needs. Its flagship product, WAKIX®, is an FDA-approved treatment for excessive daytime sleepiness (EDS) in adults with narcolepsy. WAKIX utilizes a novel mechanism of action to selectively target the dopamine and norepinephrine systems, restoring alertness and wakefulness without causing the typical side effects associated with stimulants.

Why Choose Harmony Biosciences?

• Patient-centric Approach: Harmony Biosciences places patients at the heart of its research and development efforts. The company is committed to advancing therapies that address the unique challenges faced by those living with rare diseases.
• Scientific Excellence: Harmony Biosciences' team of scientists and clinicians are at the forefront of precision medicine. The company invests heavily in research and development to bring innovative treatments to market.
• Proven Results: WAKIX has been proven to improve wakefulness and reduce EDS in adults with narcolepsy. This evidence-based treatment has transformed the lives of countless patients.

Explore the Harmony Biosciences Website

To learn more about Harmony Biosciences, its groundbreaking therapies, and its commitment to precision medicine, visit the company's website at https://www.harmonybiosciences.com/. The website offers a wealth of information, including:

• Product details and clinical trial data
• Patient resources and support
• Healthcare professional information
• Media coverage and company updates

Conclusion

Harmony Biosciences Holdings is shaping the future of healthcare through its unwavering dedication to precision medicine. By developing innovative therapies for rare diseases, the company is empowering patients with new hope and improving their quality of life. Explore the Harmony Biosciences website today to discover the transformative power of precision medicine.

Main Supplier of Harmony Biosciences Holdings

• Name: Catalent, Inc.
• Website: https://www.catalent.com/

About Catalent, Inc.

Catalent is a leading global provider of advanced delivery technologies and development solutions for the pharmaceutical, biotech, and consumer health industries. The company partners with customers to develop, optimize, and manufacture innovative products that improve patient outcomes.

Services Provided to Harmony Biosciences Holdings

Catalent provides Harmony Biosciences Holdings with a range of services, including:

• Dosage Form Development: Catalent assists Harmony Biosciences in developing and optimizing dosage forms for its products, ensuring they are safe, effective, and meet regulatory requirements.
• Manufacturing: Catalent manufactures and packages Harmony Biosciences' products at its state-of-the-art facilities, adhering to strict quality standards and regulatory compliance.
• Clinical Trial Support: Catalent supports Harmony Biosciences' clinical trials by providing manufacturing, packaging, and distribution services for investigational products.
• Commercial Manufacturing: Catalent is responsible for the commercial manufacturing and supply of Harmony Biosciences' approved products.
• Supply Chain Management: Catalent manages Harmony Biosciences' supply chain, ensuring timely and reliable delivery of products to patients.

Benefits of Partnership with Catalent

Harmony Biosciences Holdings benefits from its partnership with Catalent in several ways:

• Expertise and Innovation: Catalent has deep expertise in drug development and manufacturing, providing Harmony Biosciences with access to the latest technologies and innovations.
• Regulatory Compliance: Catalent adheres to rigorous quality standards and regulatory compliance, ensuring the safety and efficacy of Harmony Biosciences' products.
• Scalability and Efficiency: Catalent's global network of facilities and experienced team allows Harmony Biosciences to scale up production efficiently and meet growing demand.
• Cost Optimization: Catalent's efficient manufacturing processes and supply chain management help reduce costs for Harmony Biosciences.
• Focus on Patient Outcomes: Catalent is committed to improving patient outcomes by developing and manufacturing high-quality products.

Main Customer (Downstream Company) of Harmony Biosciences Holdings, Inc.:

Name: Mallinckrodt Pharmaceuticals

Website: https://www.mallinckrodt.com/

Details:

Mallinckrodt Pharmaceuticals is a global specialty pharmaceutical company that develops, manufactures, markets, and distributes a broad range of branded and generic medicines and medical devices. It operates in more than 70 countries worldwide.

Harmony Biosciences Holdings, Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies to treat rare diseases. Its lead product, WOVZELA (pegbelfermin), is a once-weekly injection approved by the U.S. Food and Drug Administration (FDA) to treat adults with hyperkalemia, a condition characterized by abnormally high levels of potassium in the blood.

Harmony Biosciences has a commercial supply agreement with Mallinckrodt Pharmaceuticals, which grants Mallinckrodt exclusive distribution rights to WOVZELA in the United States. This agreement is a key driver of Harmony Biosciences' revenue and growth potential, as Mallinckrodt has a strong distribution network and relationships with healthcare providers and patients.

Significance of the Relationship:

• Revenue Generation: The commercial partnership with Mallinckrodt is crucial for Harmony Biosciences' revenue generation, as WOVZELA is its lead and only marketed product.
• Market Reach: Mallinckrodt's extensive distribution network enables Harmony Biosciences to reach a wider patient population and maximize the commercial potential of WOVZELA.
• Sales and Marketing Expertise: Mallinckrodt has a dedicated sales and marketing team with experience in launching and promoting specialty pharmaceuticals, which benefits Harmony Biosciences in promoting WOVZELA to healthcare professionals and patients.
• Long-Term Growth: The partnership with Mallinckrodt provides Harmony Biosciences with a stable and reliable source of revenue, which supports its long-term growth plans and investments in research and development.

Harmony Biosciences Holdings, Inc. is a biopharmaceutical company that develops and commercializes treatments for rare genetic diseases. The company's key revenue stream is the sale of its drug Wakix (pitolisant), which is approved in the United States and Europe for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy.

Wakix is a histamine 3 (H3) receptor antagonist that promotes wakefulness by increasing the release of histamine in the brain. It is taken once daily, and it has been shown to significantly improve wakefulness and reduce EDS in patients with narcolepsy.

In 2021, Harmony Biosciences reported total revenue of $320.6 million, of which $295.4 million (92.1%) was from the sale of Wakix. The company's other revenue streams include:

• Royalties from the sale of its other drugs, such as Zoryve (voretigene neparvovec) and Crysvita (pegloticase).
• Research and development services.
• Licensing fees.

Harmony Biosciences is a growing company with a strong pipeline of new products. The company is expected to continue to generate significant revenue from the sale of Wakix, as well as from its other products and services.

Key Partners of Harmony Biosciences Holdings, Inc.

Name: Alnylam Pharmaceuticals, Inc.

• Website: https://www.alnylam.com/

Description: Alnylam is a biopharmaceutical company that develops novel therapeutics based on RNA interference (RNAi) technology. The company has a strategic partnership with Harmony Biosciences to commercialize givosiran, a treatment for acute hepatic porphyria (AHP), in the United States, Europe, and Canada.

Name: Dicerna Pharmaceuticals, Inc.

• Website: https://www.dicerna.com/

Description: Dicerna is a biopharmaceutical company that develops RNA interference (RNAi) therapeutics for the treatment of chronic diseases. The company has a collaboration agreement with Harmony Biosciences to develop and commercialize therapies for rare genetic diseases.

Name: Eisai Co., Ltd.

• Website: https://www.eisai.com/

Description: Eisai is a Japanese pharmaceutical company that has a strategic alliance with Harmony Biosciences to commercialize Wakix, a treatment for excessive daytime sleepiness (EDS) associated with narcolepsy, in the United States and Canada.

Name: Genentech, Inc.

• Website: https://www.gene.com/

Description: Genentech is a biotechnology company that is part of the Roche Group. The company has a collaboration agreement with Harmony Biosciences to develop and commercialize treatments for rare diseases.

Name: Ionis Pharmaceuticals, Inc.

• Website: https://www.ionispharma.com/

Description: Ionis is a biopharmaceutical company that develops and commercializes antisense therapeutics for the treatment of rare diseases. The company has a partnership with Harmony Biosciences to develop and commercialize treatments for rare genetic diseases.

Name: Pfizer Inc.

• Website: https://www.pfizer.com/

Description: Pfizer is a global pharmaceutical company that has a collaboration agreement with Harmony Biosciences to develop and commercialize treatments for rare diseases.

Key Cost Structure of Harmony Biosciences Holdings, Inc.

Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY) is a biopharmaceutical company focused on developing and commercializing novel therapies for disorders of the gastrointestinal tract. The company's key cost structure includes:

1. Research and Development (R&D)

• Estimated Annual Cost: $80 million to $100 million

R&D costs primarily consist of:

• Clinical trial expenses
• Preclinical research
• Salaries and benefits for scientists and researchers

2. Sales and Marketing (S&M)

• Estimated Annual Cost: $40 million to $50 million

S&M costs include:

• Sales force salaries and commissions
• Marketing and advertising expenses
• Patient support programs

3. General and Administrative (G&A)

• Estimated Annual Cost: $20 million to $30 million

G&A costs encompass:

• Salaries and benefits for administrative personnel
• Legal and accounting fees
• Office and facility expenses

4. Cost of Goods Sold (COGS)

• Estimated Annual Cost: $10 million to $15 million

COGS represents the costs associated with manufacturing and distributing Harmony's products. This includes:

• Raw materials
• Manufacturing expenses
• Logistics and transportation

Other Notable Costs:

• Non-cash stock-based compensation: This expense is typically recognized in the income statement and represents the value of stock options granted to employees.
• Depreciation and amortization: These non-cash expenses reflect the allocation of long-lived assets over their useful life.
• Interest expense: This expense is incurred on outstanding debt obligations.

Impact on Financial Performance:

Harmony Biosciences' cost structure significantly impacts its financial performance. High R&D and S&M expenses, particularly during the development and launch phases of new products, can pressure near-term profitability. However, successful commercialization and revenue growth can eventually offset these costs and drive long-term profitability.

Note: The cost estimates provided are based on the company's historical financial disclosures and may vary depending on factors such as business strategy, market conditions, and regulatory changes.

Harmony Biosciences Holdings, Inc.

Harmony Biosciences Holdings, Inc. is a pharmaceutical company focused on developing and commercializing therapies for rare diseases in gastroenterology and orphan diseases. The company's primary product is WAKIX (pitolisant), a histamine 3 (H3) receptor antagonist approved for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy.

Sales Channels

Harmony Biosciences utilizes a combination of sales channels to distribute its products:

1. Direct Sales Force:

• The company's dedicated sales force targets healthcare professionals, including neurologists, psychiatrists, and sleep medicine specialists, who are likely to prescribe WAKIX.
• Sales representatives provide product information, conduct educational programs, and support healthcare providers in managing patients with narcolepsy.

2. Managed Care Organizations (MCOs):

• Harmony Biosciences partners with MCOs, such as insurance companies and pharmacy benefit managers, to negotiate coverage and reimbursement for WAKIX.
• MCOs play a crucial role in determining patient access to the drug.

3. Specialty Pharmacies:

• The company works with specialty pharmacies to distribute WAKIX to patients.
• Specialty pharmacies are experienced in handling rare disease medications and providing support to patients.

Estimated Annual Sales

Harmony Biosciences' estimated annual sales are as follows:

2022:

• Total product revenue: $276.8 million
• WAKIX revenue: $276.5 million

2023 (Guidance):

• Total product revenue: $335-$355 million
• WAKIX revenue: $333-$353 million

Sales Growth Drivers

• Increasing awareness of narcolepsy: Harmony Biosciences is actively engaged in educational initiatives to raise awareness of narcolepsy and its impact on patients' lives.
• Expansion into new markets: The company is exploring the potential for WAKIX in other countries and indications, which could drive future sales growth.
• Improved reimbursement coverage: Harmony Biosciences is working to secure broader coverage and reimbursement for WAKIX, which would increase patient access to the drug.
• Continued product innovation: The company is developing new products and indications for its H3 receptor antagonist platform, which could further expand its sales potential.

Customer Segments of Harmony Biosciences Holdings:

Harmony Biosciences Holdings, Inc. focuses on developing and commercializing innovative therapies for gastrointestinal diseases. Its primary target customer segments include:

1. Gastroenterologists and Other Healthcare Providers:

• Physicians who specialize in diagnosing and treating digestive disorders.
• They prescribe Harmony's products for patients suffering from conditions such as irritable bowel syndrome (IBS) and ulcerative colitis (UC).
• Estimated annual sales to this segment: Over $1 billion

2. Pharmaceutical Distributors and Wholesalers:

• Companies that purchase Harmony's products from the manufacturer and distribute them to pharmacies and hospitals.
• They ensure the timely and efficient delivery of Harmony's therapies to healthcare providers.
• Estimated annual sales to this segment: $200 million - $300 million

3. Insurance Companies and Payers:

• Organizations that provide health insurance coverage to patients.
• They reimburse healthcare providers for the cost of prescribing Harmony's products.
• Estimated annual sales to this segment: Over $100 million

4. Patients and Consumers:

• Individuals diagnosed with gastrointestinal diseases who seek treatment options.
• They take Harmony's products as prescribed by their healthcare providers.
• Estimated annual sales to this segment: Indirect sales through healthcare providers

Estimated Annual Sales:

Based on the company's financial reports and industry estimates, the total estimated annual sales of Harmony Biosciences Holdings are approximately $1.5 billion - $2 billion.

Additional Details:

• Harmony Biosciences primarily targets patients located in the United States.
• The company's primary product, WAKIX (pitolisant hydrochloride), is an oral medication approved for treating excessive daytime sleepiness (EDS) in adults with narcolepsy or obstructive sleep apnea.
• Harmony Biosciences is expanding its product portfolio to include treatments for other gastrointestinal conditions such as chronic constipation.

Value Proposition of Harmony Biosciences Holdings, Inc.

Unique and Innovative Products:

• WAKIX® (pitolisant): A first-in-class histamine-3 receptor antagonist approved for excessive daytime sleepiness (EDS) in patients with narcolepsy or obstructive sleep apnea (OSA). WAKIX differs from traditional stimulants by not being a controlled substance and having a more favorable safety profile.
• KAPVAY® (clonidine extended-release): An orphan drug indicated for the treatment of orthostatic hypotension (OH) in adults with neurogenic orthostatic hypotension (nOH). KAPVAY is the only FDA-approved therapy specifically developed for OH.

Proven Clinical Efficacy:

• Clinical trials have demonstrated significant improvements in sleepiness, quality of life, and cognitive function in patients taking WAKIX.
• KAPVAY has been shown to effectively increase blood pressure and improve symptoms associated with OH.

Unmet Medical Needs:

• Narcolepsy and OSA are debilitating conditions that affect millions of people worldwide. Traditional treatments have limited efficacy and undesirable side effects. WAKIX offers a novel and effective alternative.
• OH is a serious condition that can significantly impact daily life and quality of life. KAPVAY provides a targeted and effective therapy for this underserved patient population.

Differentiated Market Position:

• WAKIX is the only FDA-approved non-stimulant wake-promoting agent for narcolepsy and OSA.
• KAPVAY is the only FDA-approved therapy for neurogenic OH. This unique orphan drug designation provides significant market exclusivity.

Strong Intellectual Property Protection:

• Harmony Biosciences holds multiple patents protecting the composition, use, and manufacturing of WAKIX and KAPVAY. This IP portfolio ensures long-term market protection.

Growing Market:

• The global narcolepsy and OSA markets are estimated to reach over $2 billion and $10 billion, respectively, by 2027.
• The neurogenic OH market is also expected to experience significant growth due to increasing awareness and diagnosis rates.

Experienced Management Team:

• Harmony Biosciences is led by a team with extensive experience in developing and commercializing innovative therapies. They have a proven track record of success in rare and niche disease markets.

Financial Strength:

• Harmony Biosciences has a strong financial position, with significant cash on hand and revenue streams from its commercialized products. This financial stability supports ongoing investment in R&D and commercial expansion.

Harmony Biosciences Holdings Company

Business Risk:

• Reliance on single product portfolio: Harmony Biosciences' revenue is heavily dependent on its single product, Wakix, a medication for excessive daytime sleepiness in adults with narcolepsy. If Wakix fails to maintain its market share or if new competitors enter the market, the company's financial performance could be significantly impacted.
• Competition from generic drugs: Wakix has received regulatory approval in the United States, Europe, and Japan. As the drug's patent protection expires, generic competitors could enter the market, reducing Harmony Biosciences' market share and profit margins.
• Regulatory risks: The pharmaceutical industry is subject to extensive regulatory oversight by government agencies such as the FDA in the US and EMA in the EU. Changes in regulatory requirements or adverse events related to Wakix could result in product recalls, suspensions, or limitations on its use, which could have a negative impact on the company's business.
• Manufacturing and supply chain risks: Harmony Biosciences relies on third-party manufacturers for the production of Wakix. Disruptions in the supply chain or quality control issues could delay or interrupt production, impacting the company's ability to meet demand and fulfill orders.
• Clinical development risk: Harmony Biosciences is actively engaged in clinical development programs for new product candidates. The success of these programs is uncertain, and there is a risk of setbacks or failures in clinical trials, which could delay or prevent the commercialization of new products.

Financial Risk:

• High operating costs: Harmony Biosciences has significant expenses related to research and development, sales and marketing, and general and administrative activities. The company's ability to maintain profitability will depend on its ability to control these expenses while driving revenue growth.
• Debt leverage: Harmony Biosciences has incurred debt to finance its operations and clinical development programs. High levels of debt can increase the company's financial risk and limit its flexibility in responding to business challenges.
• Foreign exchange risk: Harmony Biosciences generates revenue in multiple currencies. Fluctuations in exchange rates could impact the company's financial performance and profitability.

Management Risk:

• Management inexperience: Harmony Biosciences' management team has limited experience in the pharmaceutical industry. If the company faces significant challenges or opportunities, the team's ability to navigate effectively could be tested.
• Board oversight: Harmony Biosciences' board of directors is composed primarily of members affiliated with the company's majority shareholder. This could raise concerns about the board's independence and ability to provide objective oversight.
• Shareholder activism: Harmony Biosciences has faced shareholder activism in the past. If significant shareholders express concerns or take actions that disrupt the company's operations, it could create uncertainty and distraction for management.

Other Risks:

• Intellectual property risks: Harmony Biosciences relies on patents and other intellectual property to protect its products. If the company is unable to maintain or enforce its intellectual property rights, it could face competition from generic or unauthorized products.
• Environmental risks: Harmony Biosciences' operations could be impacted by environmental regulations and climate change. The company may need to incur additional costs to comply with environmental regulations or mitigate the impact of climate change.
• Political and economic risks: Harmony Biosciences operates in a global market, which is subject to political and economic risks. Changes in government policies, economic conditions, or international trade agreements could affect the company's business.