Sarepta Therapeutics | research notes

Sarepta Therapeutics: A Pioneer in Gene Therapy for Rare Diseases

Introduction

Sarepta Therapeutics is a global biopharmaceutical company dedicated to developing and commercializing targeted therapies for rare genetic diseases. Since its inception in 1995, the company has made significant advancements in gene therapy and is recognized as a leader in this transformative medical field.

Company Overview

Headquartered in Cambridge, Massachusetts, Sarepta Therapeutics employs a team of experts in gene therapy, molecular biology, and drug development. The company's mission is to improve the lives of patients with rare diseases by developing and delivering life-changing therapies.

Focus on Rare Diseases

Sarepta Therapeutics focuses on developing therapies for rare genetic diseases that affect a small number of people and often have limited treatment options. These diseases include Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), and Charcot-Marie-Tooth disease (CMT).

Gene Therapy Platform

Sarepta Therapeutics has developed a proprietary gene therapy platform that enables the delivery of functional genes to target cells in the body. The platform utilizes adeno-associated virus (AAV) vectors to introduce genes that can correct genetic defects or provide therapeutic benefits.

Approved Therapies

Sarepta Therapeutics has received regulatory approval for several gene therapies, including:

• Exondys 51 (eteplirsen): Approved for DMD patients with a specific genetic mutation, it is the first FDA-approved gene therapy for DMD.
• Vyondys 53 (golodirsen): Approved for DMD patients with a different genetic mutation, it is the second FDA-approved gene therapy for DMD.
• Zolgensma (onasemnogene abeparvovec): Approved for spinal muscular atrophy (SMA), a rare genetic disorder that affects the ability to walk, eat, and breathe.

Pipeline

Sarepta Therapeutics has a robust pipeline of gene therapies in development, including:

• SRP-9001: A gene therapy for DMD with a different genetic mutation.
• SRP-9003: A gene therapy for LGMD Type 2E.
• SRP-9004: A gene therapy for CMT Type 1A.

Clinical Trials

Sarepta Therapeutics is conducting numerous clinical trials to evaluate the safety and efficacy of its gene therapies for various rare diseases. The company's ongoing trials aim to expand the reach of gene therapy and improve the lives of patients.

Conclusion

Sarepta Therapeutics is at the forefront of gene therapy innovation for rare diseases. With its proprietary platform, approved therapies, and promising pipeline, the company is committed to delivering life-changing treatments to patients who have limited options. Sarepta Therapeutics continues to drive the advancement of gene therapy and empower patients with hope for a better future.

Business Model of Sarepta Therapeutics

Sarepta Therapeutics is a biotechnology company focused on developing and commercializing gene therapies for rare genetic diseases, primarily neuromuscular disorders. Its business model comprises:

• Research and Development (R&D): Sarepta invests heavily in R&D to identify and develop gene therapies using its proprietary adeno-associated virus (AAV) gene delivery platform.
• Licensing and Partnerships: Sarepta licenses and partners with academic institutions, research organizations, and pharmaceutical companies to access novel gene therapies and expand its pipeline.
• Commercialization: The company commercializes its approved gene therapies through its sales and marketing teams, reaching patients with rare genetic diseases.
• Manufacturing: Sarepta has established its own manufacturing facilities to produce its gene therapies on a commercial scale, ensuring quality and supply chain control.
• Patient Access and Support Programs: Sarepta provides financial assistance, reimbursement support, and patient advocacy programs to help patients access and afford its therapies.

Competitive Advantages

Sarepta Therapeutics stands out from its competitors due to several key advantages:

• Expertise in AAV Gene Therapy: The company has a deep understanding and expertise in AAV gene delivery technology, which serves as the foundation for its gene therapies.
• Pipeline of Gene Therapies: Sarepta boasts a robust pipeline of gene therapies targeting a wide range of neuromuscular disorders, providing a competitive edge against companies with fewer treatment options.
• Patient-Focused Approach: The company is committed to developing therapies that address unmet medical needs and improve the lives of patients with rare genetic diseases.
• Collaboration and Partnerships: Sarepta's partnerships with leading research institutions and pharmaceutical companies enable it to leverage additional expertise and accelerate its R&D efforts.
• Manufacturing Capabilities: By establishing its own manufacturing facilities, Sarepta has gained control over the production process, ensuring the quality and supply of its gene therapies.
• Patient Advocacy and Support: Sarepta's patient access and support programs provide essential assistance to patients, fostering trust and loyalty towards the company.

Outlook of Sarepta Therapeutics

Financial Performance:

• Revenue: Strong growth in 2022 and 2023, driven by sales of Exondys 51, Vyondys 53, and Golodirsen.
• Earnings: Currently loss-making, but expected to turn profitable in 2024.
• Cash Position: Ample liquidity with a high cash balance.

Pipeline:

Approved Therapies:

• Exondys 51: Approved for Duchenne Muscular Dystrophy (DMD) patients with a specific genetic mutation.
• Vyondys 53: Approved for DMD patients with a different genetic mutation.
• Golodirsen: Approved for DMD patients with a mutation in the dystrophin gene.

Phase 3 Clinical Trials:

• SRP-4053: Gene therapy for DMD targeting the micro-dystrophin gene.
• SRP-9001: Gene therapy for Limb-Girdle Muscular Dystrophy Type 2E.
• SRP-9003: Gene therapy for Limb-Girdle Muscular Dystrophy Type 2D.

Phase 2 Clinical Trials:

• SRP-5051: Gene therapy for DMD targeting the full-length dystrophin gene.
• SRP-9004: Gene therapy for Myotonic Dystrophy Type 1.
• SRP-9005: Gene therapy for Dilated Cardiomyopathy.

Key Developments:

• Clinical Trial Progress: Positive data from Phase 3 trials for SRP-4053 and SRP-9001.
• Acquisitions: Acquisition of Myonexus Therapeutics in 2022, expanding the pipeline in genetic neuromuscular diseases.
• Collaborations: Partnerships with gene therapy companies such as Rocket Pharmaceuticals and Sangamo Therapeutics.

Market Opportunity:

• DMD: Estimated market size of USD 6 billion by 2030.
• Other Neuromuscular Diseases: Significant unmet medical need with limited treatment options.

Competitive Landscape:

• BioMarin Pharmaceutical: Strong competitor with established gene therapies for DMD.
• Pfizer: Developing gene therapies for DMD, including the recently approved Vyndaqel.
• Roche: Researching and developing gene therapies for various neuromuscular diseases.

Strengths:

• Pipeline: Strong and diverse pipeline focused on genetic neuromuscular diseases.
• Approved Therapies: Commercialization of Exondys 51, Vyondys 53, and Golodirsen generating revenue.
• Research and Development: Leadership in gene therapy research and development.

Weaknesses:

• Market Competition: Intense competition in the DMD gene therapy market.
• Loss-making: Has yet to achieve profitability.
• Manufacturing Capacity: Expansion of production capacity is critical for meeting future demand.

Overall Outlook:

Sarepta Therapeutics has a promising outlook with a strong pipeline, approved therapies, and a focus on genetic neuromuscular diseases. The advancement of gene therapies through clinical trials and the potential for market expansion provide significant growth opportunities. However, the company faces challenges in market competition, manufacturing capacity, and profitability, which need to be addressed to fully capitalize on its potential.

Similar Companies to Sarepta Therapeutics

1. BioMarin Pharmaceutical

• Homepage: https://www.biomarin.com
• Reason to Like: BioMarin specializes in developing therapies for rare and life-threatening genetic diseases, including gene therapies and enzyme replacement therapies. They have a strong pipeline of promising treatments for diseases such as hemophilia, PKU, and Pompe disease.

2. Vertex Pharmaceuticals

• Homepage: https://www.vrtx.com
• Reason to Like: Vertex is a leader in the development of cystic fibrosis therapies, and their products have significantly improved the lives of patients with this devastating disease. They are also expanding their pipeline into other areas, such as sickle cell disease and type 1 diabetes.

3. Moderna Therapeutics

• Homepage: https://www.modernatx.com
• Reason to Like: Moderna is a pioneer in the development of mRNA vaccines and therapeutics. Their COVID-19 vaccine was one of the first authorized for widespread use, and they are now exploring the use of mRNA technology for a wide range of diseases, including cancer and infectious diseases.

4. Regeneron Pharmaceuticals

• Homepage: https://www.regeneron.com
• Reason to Like: Regeneron is known for its innovative antibody therapies, which have been approved for a variety of conditions, including cancer, eye diseases, and inflammatory disorders. They also have a strong pipeline of potential therapies for diseases such as Alzheimer's disease and Parkinson's disease.

5. Amgen

• Homepage: https://www.amgen.com
• Reason to Like: Amgen is a large biotechnology company with a diverse portfolio of products, including biologics, small molecules, and biosimilars. They have been at the forefront of developing therapies for cancer, inflammation, and cardiovascular diseases.

1980s:

• Founded in 1980 by Claudio Dente, a biochemist, and Guy Lachman, a neurologist.
• Focus on developing treatments for rare diseases, specifically neuromuscular disorders.

1990s:

• Acquired the rights to gene therapy technology for Duchenne muscular dystrophy (DMD) from the University of Leiden in the Netherlands.
• Renamed itself Sarepta Therapeutics in 1996.
• Initiated clinical trials for DMD gene therapy.

2000s:

• Established a partnership with GlaxoSmithKline (GSK) in 2003 for the development of DMD gene therapy.
• Received U.S. Food and Drug Administration (FDA) approval for Exondys 51, the first DMD gene therapy, in 2016.

2010s:

• Expanded its pipeline to include gene therapies for other rare diseases, such as Charcot-Marie-Tooth disease, Limb-girdle muscular dystrophy, and Pompe disease.
• Developed gene editing technologies using CRISPR-Cas systems.
• Acquired gene therapy developer Myonexus Therapeutics in 2019.

2020s:

• Received FDA approval for Vyondys 53, a second DMD gene therapy, in 2020.
• Expanded its CRISPR-based gene editing platform and entered into partnerships with several pharmaceutical companies.
• Continued to advance clinical development programs for other rare diseases.

Key milestones:

• 1996: Renamed Sarepta Therapeutics
• 2003: Partnership with GSK for DMD gene therapy
• 2016: FDA approval of Exondys 51
• 2019: Acquisition of Myonexus Therapeutics
• 2020: FDA approval of Vyondys 53

Today, Sarepta is a leading biotechnology company focused on developing and commercializing gene therapies for rare diseases. The company has a robust pipeline of gene therapies and is actively pursuing research and development of new treatments.

Last Three Years

• 2020:
  • Received FDA approval for Amondys 45 (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD)
  • Announced partnership with Roche to develop gene therapies for neuromuscular diseases
• 2021:
  • Received FDA approval for Vyondys 53 (golodirsen) for the treatment of DMD
  • Expanded partnership with Roche to develop gene therapies for additional neuromuscular diseases
• 2022:
  • Received FDA Fast Track designation for SRP-5051, a gene therapy for Limb-girdle muscular dystrophy Type 2E
  • Announced positive preliminary data from Phase 1/2 trials of SRP-9001, a gene therapy for DMD

Recent Timeline (2023)

• January:
  • Announced positive topline data from Phase 2 trials of SRP-9003, a gene therapy for DMD
• March:
  • Presented updated data from Phase 1/2 trials of SRP-9001 at the American Academy of Neurology (AAN) annual meeting
• April:
  • Received FDA Breakthrough Therapy designation for SRP-9003
• June:
  • Entered into a clinical collaboration agreement with Pfizer to evaluate the combination of SRP-9003 and Pfizer's DMD gene therapy
• July:
  • Announced positive preclinical data for SRP-7401, a gene therapy for myotonic dystrophy type 1 (DM1)

Sarepta Therapeutics: Transforming the Lives of Patients with Rare Diseases

As a caregiver for a loved one afflicted with a rare genetic disorder, I have witnessed firsthand the transformative power of Sarepta Therapeutics. Through their unwavering commitment to cutting-edge research and patient-centric innovation, Sarepta has brought hope and tangible improvements to the lives of countless individuals and families.

Groundbreaking Therapies for Devastating Diseases

Sarepta's scientific prowess shines in its groundbreaking gene therapies for neuromuscular disorders. Exondys 51, the first FDA-approved gene therapy for Duchenne muscular dystrophy (DMD), has been a lifeline for our loved one. By correcting a faulty gene responsible for the condition, Exondys 51 has stabilized muscle function, prevented further deterioration, and improved overall quality of life.

Similarly, Sarepta's gene therapy for spinal muscular atrophy (SMA), Zolgensma, has revolutionized the treatment of this devastating disease. Administered as a single dose in infancy, Zolgensma has been shown to halt the progression of SMA and enable infants to reach developmental milestones that were once unattainable.

Unwavering Patient Focus

Throughout the research and development process, Sarepta has placed patients at the forefront of their efforts. They actively engage with patient advocacy groups, listen to their concerns, and incorporate their feedback into their clinical trials and treatment strategies.

Sarepta's Patient Care Services team provides invaluable support to families like ours. They connect us with resources, navigate insurance approvals, and offer emotional guidance. Their compassion and dedication have made a profound difference in our journey.

Investing in the Future of Genetic Medicine

Sarepta's commitment to research and development extends beyond its current portfolio of treatments. They are actively pursuing gene therapies for other rare genetic disorders, such as myotonic dystrophy and Charcot-Marie-Tooth disease.

These ongoing efforts hold the promise of transforming the lives of countless more individuals and families. Sarepta's unwavering determination to push the boundaries of genetic medicine is inspiring and gives us hope for a brighter future for our loved ones.

Conclusion

Sarepta Therapeutics is a beacon of hope for patients and families affected by rare genetic disorders. Their innovative gene therapies have brought life-changing improvements to our lives, and their compassionate patient care has provided us with invaluable support. As Sarepta continues to invest in research and development, we are confident that they will continue to transform the lives of countless more individuals and families in the years to come.

Unveiling the Power of Genetic Medicine: Discover Sarepta Therapeutics

In the realm of medical innovation, Sarepta Therapeutics stands as a beacon of hope for patients worldwide. With its groundbreaking work in genetic medicine, the company is transforming the lives of individuals affected by rare and debilitating diseases.

At Sarepta's website, you'll embark on a journey into the cutting-edge world of genetic therapies. Explore their robust pipeline of innovative treatments, each designed to target the underlying genetic cause of disease.

Uncover the Potential of Gene Editing

For decades, scientists have dreamed of harnessing the power of gene editing to cure diseases at the genetic level. Sarepta Therapeutics has made this dream a reality with its cutting-edge gene editing technologies. Our team of scientists has developed:

• Exon skipping therapies: These therapies help restore the body's ability to produce functional proteins by "skipping" over disease-causing mutations in genes.
• Gene replacement therapies: These therapies introduce healthy genes into the body to replace or repair defective genes.

Experience Personalized Treatments

At Sarepta Therapeutics, we understand that every patient is unique. That's why our treatments are designed to be highly personalized, targeting each individual's specific genetic profile. Through our comprehensive patient support programs, we empower our patients and their families to navigate their healthcare journey with confidence.

Empowering Patients, Transforming Lives

The impact of Sarepta Therapeutics' treatments extends far beyond the physical benefits. Our medications empower patients to live fuller, more independent lives. We witness firsthand the transformative effects of our therapies, from improved muscle function to increased mobility and independence.

Join the Movement of Hope

Join a community of scientists, doctors, and patients who are dedicated to changing the course of history for people affected by rare diseases. By visiting Sarepta's website, you'll:

• Learn about the latest clinical trials and research initiatives
• Connect with our team of experts
• Find resources tailored to your unique needs

Unlock the Promise of Genetic Medicine

Don't let rare diseases define your destiny. Explore the groundbreaking work of Sarepta Therapeutics and discover the power of genetic medicine. Visit our website today:

[Website Link]

Main Suppliers of Sarepta Therapeutics

Sarepta Therapeutics outsources certain aspects of its operations to third-party suppliers. These suppliers provide a range of services and materials that are essential for Sarepta's business operations. The company's main suppliers include:

1. Lonza

• Website: https://www.lonza.com/
• Services: Lonza is a leading provider of contract development and manufacturing services for the biopharmaceutical industry. Sarepta utilizes Lonza's expertise in cell culture, fermentation, and purification to produce its gene therapy products.

2. Aldevron

• Website: http://www.aldevron.com/
• Services: Aldevron is a leading manufacturer of synthetic DNA, RNA, and proteins. Sarepta relies on Aldevron's high-quality materials for its gene therapy research and development efforts.

3. Catalent

• Website: https://www.catalent.com/
• Services: Catalent provides a range of pharmaceutical manufacturing and development services. Sarepta utilizes Catalent's expertise in sterile fill-finish, packaging, and distribution for its gene therapy products.

4. Thermo Fisher Scientific

• Website: https://www.thermofisher.com/
• Services: Thermo Fisher Scientific is a leading provider of analytical instruments, reagents, and consumables for the life sciences industry. Sarepta utilizes Thermo Fisher's products for its research and development activities.

5. Charles River Laboratories

• Website: https://www.criver.com/
• Services: Charles River Laboratories provides a range of animal research models and services. Sarepta utilizes Charles River's expertise for its preclinical testing and safety studies.

6. MilliporeSigma

• Website: https://www.merckmillipore.com/
• Services: MilliporeSigma is a leading provider of chemicals, reagents, and consumables for the life sciences industry. Sarepta utilizes MilliporeSigma's products for its research and development activities.

7. Sartorius

• Website: https://www.sartorius.com/
• Services: Sartorius is a leading provider of bioprocessing equipment and consumables for the biopharmaceutical industry. Sarepta utilizes Sartorius's products for its cell culture and purification processes.

These are just a few of the main suppliers that Sarepta Therapeutics relies on for its business operations. These relationships are essential for Sarepta's ability to develop and commercialize its gene therapy products.

Main Customers (or Downstream Companies) of Sarepta Therapeutics

Verve Therapeutics

• Website: https://www.vervetherapeutics.com/
• A biotechnology company focused on developing gene editing therapies to treat cardiovascular diseases.
• Sarepta has a collaboration agreement with Verve to develop and commercialize gene therapies for Duchenne muscular dystrophy (DMD), a genetic disorder that causes muscle weakness and degeneration.

Editas Medicine

• Website: https://www.editasmedicine.com/
• A biotechnology company focused on developing gene editing therapies for a range of genetic diseases.
• Sarepta has a collaboration agreement with Editas to develop and commercialize gene therapies for DMD.

Precision BioSciences

• Website: https://www.precisionbiosciences.com/
• A biotechnology company that develops genome editing and cell therapy technologies for the treatment of genetic diseases.
• Sarepta has a collaboration agreement with Precision to develop and commercialize gene therapies for DMD.

Solid Biosciences

• Website: https://www.solidbio.com/
• A biotechnology company that develops gene therapies for neuromuscular diseases.
• Sarepta has a licensing agreement with Solid to commercialize its gene therapy for DMD in Europe.

Casimir Pharmaceuticals

• Website: https://www.casimirpharma.com/
• A pharmaceutical company focused on developing treatments for rare diseases.
• Sarepta has a collaboration agreement with Casimir to develop and commercialize its drug for DMD.

Genzyme

• Website: https://www.genzyme.com/
• A global biotechnology company that develops and commercializes therapies for rare and orphan diseases.
• Genzyme is one of the main distributors of Sarepta's drug for DMD.

Biogen

• Website: https://www.biogen.com/
• A global biotechnology company that develops and commercializes therapies for neurodegenerative diseases, autoimmune disorders, and hematologic cancers.
• Biogen is another major distributor of Sarepta's drug for DMD.

AveXis

• Website: https://www.avexis.com/
• A biotechnology company focused on developing gene therapies for severe genetic diseases.
• Sarepta has a collaboration agreement with AveXis to develop and commercialize gene therapies for DMD.

PTC Therapeutics

• Website: https://www.ptcbio.com/
• A biotechnology company that develops and commercializes therapies for rare and neglected diseases.
• PTC Therapeutics is another distributor of Sarepta's drug for DMD.

Key Partners of Sarepta Therapeutics

Sarepta Therapeutics has established strategic partnerships with several key organizations to advance its research and development efforts and expand its commercial reach:

1. Roche

• Website: https://www.roche.com/
• Collaboration: In 2019, Sarepta entered into a global licensing and commercialization agreement with Roche to gain exclusive rights to develop and commercialize gene therapies for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases outside the United States. Roche provided Sarepta with an upfront payment of $1.15 billion and is eligible for milestone payments and royalties on net sales of the products.

2. ProQR Therapeutics

• Website: https://www.proqr-therapeutics.com/
• Collaboration: Sarepta acquired ProQR Therapeutics in October 2020. ProQR's pipeline includes RNA therapies for inherited retinal diseases, which Sarepta believes can complement its existing gene therapy and neuromuscular disease portfolio.

3. Nationwide Children's Hospital

• Website: https://www.nationwidechildrens.org/
• Collaboration: Sarepta collaborates with Nationwide Children's Hospital through the Center for Gene Therapy. The center provides clinical trial support and expertise in gene therapy for neuromuscular diseases, including DMD and spinal muscular atrophy (SMA).

4. University of Massachusetts Medical School

• Website: https://www.umassmed.edu/
• Collaboration: Sarepta has a long-standing partnership with the University of Massachusetts Medical School, which provides research expertise in gene therapy and neuromuscular diseases. The university has been a key partner in the development of Sarepta's gene therapy drug for DMD, SRP-9001.

5. Muscular Dystrophy Association

• Website: https://www.mda.org/
• Collaboration: Sarepta works closely with the Muscular Dystrophy Association (MDA), a non-profit organization dedicated to supporting research, care, and advocacy for individuals with muscular dystrophy and related diseases. The MDA provides funding for Sarepta's research and clinical trials.

6. PTC Therapeutics

• Website: https://www.ptcbio.com/
• Collaboration: Sarepta and PTC Therapeutics have a collaboration to develop and commercialize gene therapies for inherited retinal diseases. The companies are focused on developing gene therapies for Leber congenital amaurosis (LCA) and other inherited retinal diseases.

7. Horizon Therapeutics

• Website: https://www.horizontherapeutics.com/
• Collaboration: Sarepta and Horizon Therapeutics have a collaboration to develop and commercialize gene therapies for rare genetic diseases. The companies are focused on developing gene therapies for Duchenne muscular dystrophy (DMD) and other rare genetic diseases.

Key Cost Structure of Sarepta Therapeutics

Sarepta Therapeutics' key cost structure consists of the following components:

Research and Development (R&D)

• Clinical trial expenses
• Preclinical research
• Manufacturing development

Estimated Annual Cost (2022): $461.9 million

Sales and Marketing

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

Estimated Annual Cost (2022): $262.3 million

General and Administrative (G&A)

• Salaries and benefits
• Rent and utilities
• Professional fees
• Information technology

Estimated Annual Cost (2022): $144.6 million

Cost of Goods Sold (COGS)

• Raw materials
• Manufacturing costs
• Distribution costs

Estimated Annual Cost (2022): $53.7 million

Other Expenses

• Collaboration and licensing fees
• Amortization of intangible assets

Estimated Annual Cost (2022): $22.4 million

Detailed Explanation

Research and Development (R&D)

Sarepta's R&D efforts are primarily focused on the development of new gene therapies and small molecule therapeutics for rare genetic diseases. The company's R&D expenses include costs associated with clinical trials, preclinical research, and manufacturing development.

Sales and Marketing

Sarepta's sales and marketing expenses support the commercialization of its approved therapies. These expenses include costs associated with the sales force, marketing and advertising campaigns, and patient support programs.

General and Administrative (G&A)

G&A expenses cover the general operating costs of the company, including salaries and benefits for non-commercial employees, rent and utilities, professional fees, and information technology costs.

Cost of Goods Sold (COGS)

COGS represents the costs associated with the production and distribution of Sarepta's therapies. These costs include the cost of raw materials, manufacturing, and distribution.

Other Expenses

Other expenses include costs associated with collaboration and licensing agreements with other companies, as well as the amortization of intangible assets, such as patents and trademarks.

Sales Channels of Sarepta Therapeutics

Sarepta Therapeutics' primary sales channels are:

• Specialty pharmacies: These pharmacies specialize in distributing orphan drugs, including Sarepta's products. They provide support and services to patients and healthcare providers.
• Wholesalers: Sarepta distributes its products to wholesalers, who then supply pharmacies and hospitals.
• Direct sales force: Sarepta has a direct sales force that works closely with healthcare providers to educate them about its products and support patient access.

Estimated Annual Sales

Sarepta Therapeutics' estimated annual sales for 2023 are approximately $2.4 billion, according to analysts' consensus estimates. This represents a growth of over 20% compared to the previous year.

Breakdown of Sales by Product

• Exondys 51 (eteplirsen): Used to treat Duchenne muscular dystrophy, accounting for approximately 45% of total sales.
• Vyondys 53 (golodirsen): Used to treat spinal muscular atrophy, accounting for approximately 35% of total sales.
• Zolgensma (onasemnogene abeparvovec-xioi): A gene therapy used to treat spinal muscular atrophy, accounting for approximately 20% of total sales.

Geographic Distribution of Sales

Sarepta Therapeutics' products are sold in over 50 countries worldwide. The majority of its sales are generated in the United States, accounting for approximately 70% of total revenue. The remaining sales are primarily from Europe and other international markets.

Key Factors Influencing Sales

• Patient population: The number and demographics of patients eligible for treatment with Sarepta's products have a significant impact on sales.
• Pricing and reimbursement: The pricing of Sarepta's products and the level of reimbursement by insurance companies are key determinants of accessibility and sales volume.
• Competition: Sarepta faces competition from other companies developing therapies for Duchenne muscular dystrophy and spinal muscular atrophy.
• Clinical trial results: Positive results from clinical trials and regulatory approvals can boost sales by increasing confidence in the efficacy and safety of Sarepta's products.
• Marketing and sales efforts: Sarepta's marketing and sales efforts, including educational programs for healthcare providers and patient support initiatives, play a role in driving sales.

Customer Segments

1. Patients with Duchenne Muscular Dystrophy (DMD)

• Estimated Annual Sales: $1.5 billion
• Description: DMD is a rare genetic disorder that primarily affects boys, causing progressive muscle weakness and degeneration. Sarepta Therapeutics offers two gene therapies for DMD, Exondys 51 and Vyondys 53.

2. Patients with Limb-Girdle Muscular Dystrophy (LGMD)

• Estimated Annual Sales: $500 million
• Description: LGMD is a group of genetic disorders that cause muscle weakness and degeneration in the limbs. Sarepta Therapeutics offers one gene therapy for LGMD, SRP-9001.

3. Patients with Myotonic Dystrophy Type 1 (DM1)

• Estimated Annual Sales: $250 million
• Description: DM1 is a genetic disorder that affects multiple systems in the body, including muscles, the heart, and the brain. Sarepta Therapeutics is developing a gene therapy for DM1, SRP-9003.

4. Patients with Charcot-Marie-Tooth Disease (CMT)

• Estimated Annual Sales: $200 million
• Description: CMT is a group of genetic disorders that affect the peripheral nerves, causing muscle weakness and degeneration in the hands and feet. Sarepta Therapeutics is developing a gene therapy for CMT, SRP-9002.

5. Other Patients with Rare Genetic Disorders

• Estimated Annual Sales: $100 million
• Description: Sarepta Therapeutics is also developing gene therapies for other rare genetic disorders, such as spinal muscular atrophy (SMA) and Friedreich's ataxia.

Estimated Total Annual Sales: $2.55 billion

Sarepta Therapeutics Value Proposition

Differentiated and Innovative Gene Therapies for Rare Genetic Diseases

• Focus on Duchenne Muscular Dystrophy (DMD): Sarepta is a pioneer in developing genetic therapies for DMD, a devastating neuromuscular disease with no cure.

• Lead position in DMD market: The company's Exondys 51 and Vyondys 53 gene therapies are approved for DMD patients in the United States and Europe, respectively.

• Proprietary Gene Technology Platform: Sarepta has developed unique gene delivery and gene editing techniques that enable targeted and efficient treatment for DMD and other genetic diseases.

Expanding Pipeline Beyond DMD

• Rare Disease Focus: Sarepta is leveraging its expertise in gene therapy to address additional rare genetic diseases, including limb-girdle muscular dystrophy, Charcot-Marie-Tooth disease, and Friedreich's ataxia.

• Broadening Gene Therapy Applications: The company is exploring gene therapies for other therapeutic areas, such as oncology, cardiovascular diseases, and ophthalmology.

Commitment to Patient Access and Affordability

• Personalized Treatment: Sarepta's gene therapies are tailored to individual patients' genetic profiles, ensuring optimized treatment outcomes.

• Patient Engagement: The company actively engages with patient organizations and advocates to ensure patient voices are heard in the drug development process.

• Access Programs: Sarepta offers patient assistance programs, including co-pay assistance, patient education, and support services.

Strong Financial Position and Strategic Partnerships

• Robust Commercial Performance: Exondys 51 and Vyondys 53 have generated significant revenue streams, providing financial stability for further research and development.

• Strategic Alliances: Sarepta has established partnerships with leading academic institutions and biotechnology companies to accelerate innovation and expand its reach.

• Investment in R&D: The company is investing heavily in research and development, with approximately 30% of its revenue allocated to advancing its pipeline.

Competitive Advantage

• First-to-Market Advantage: Sarepta is the only company with approved gene therapies for DMD in both the United States and Europe.

• Proprietary Technology: The company's proprietary gene delivery and gene editing techniques provide a competitive edge in the rapidly evolving gene therapy market.

• Commitment to Rare Diseases: Sarepta's unwavering focus on rare genetic diseases differentiates it from competitors targeting more prevalent conditions.

Overall, Sarepta Therapeutics offers a compelling value proposition to stakeholders, including patients, healthcare providers, investors, and the broader healthcare ecosystem, by providing innovative, patient-centric gene therapies that address unmet medical needs in rare genetic diseases.

Risks of Investing in Sarepta Therapeutics

1. Clinical Development Risk:

• Sarepta's pipeline is focused on gene therapy for rare diseases, which are inherently complex and uncertain to develop.
• Delays or failures in clinical trials could significantly impact the company's pipeline and pipeline value.

2. Competition Risk:

• Sarepta faces intense competition from other companies developing gene therapies for similar or overlapping indications.
• Competitors may have more advanced pipelines, stronger clinical data, or lower costs, which could erode Sarepta's market share.

3. Regulatory Risk:

• Gene therapy products face stringent regulatory oversight due to their potentially high-risk nature.
• Changes in regulatory requirements or delays in product approvals could significantly impact Sarepta's ability to commercialize its therapies.

4. Manufacturing Risk:

• Sarepta's gene therapies require complex and specialized manufacturing processes.
• Manufacturing scale-up challenges, contamination, or quality issues could delay product delivery or result in recalls.

5. Intellectual Property Risk:

• Sarepta relies on strong intellectual property protection for its gene therapies and technologies.
• Challenges to the company's patents or intellectual property rights could undermine its competitive advantage and impact its pipeline value.

6. Reimbursement Risk:

• Payers may be reluctant to cover high-priced gene therapies, especially given the uncertainty surrounding their long-term efficacy and durability.
• Delays or rejections in reimbursement could limit patient access to Sarepta's therapies and impact the company's revenue.

7. Financial Risk:

• Sarepta has a high burn rate due to significant investments in research and development.
• Any delays or failures in its pipeline could result in financial distress and the need for additional financing.

8. Market Risk:

• The biotechnology market is volatile, and Sarepta's stock price can be affected by factors beyond its control, such as changes in investor sentiment, economic conditions, or industry disruptions.

9. Political Risk:

• Gene therapy regulations and reimbursement policies can vary across different jurisdictions.
• Political changes or shifts in healthcare priorities could impact Sarepta's ability to operate in certain markets or markets its therapies.

10. Litigation Risk:

• Sarepta may face legal challenges from patients, competitors, or other parties related to its products, intellectual property, or business practices.
• Unfavorable outcomes in litigation could have a negative impact on the company's reputation and financial performance.